curepsp 2013 annual report

Making a Difference in the Lives of People with PSP, CBD and Other Atypical Parkinsonian Disorders

Annual Report3 1 0 2

ANNUAL REPORT 2013

TABLE OF CONTENTS

Dedication to Karen Strauss Cook . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 4

Vision, Mission and Overview of the Foundation . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 5

From the Former Chair of the Board of Directors . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 6

From the Current Chair of the Board of Directors . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 7

From the President-CEO . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 8

Strategic Vision FY 2014 . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 10

Leadership and Staff . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 12

Financials FY 2013 (Year Ended June 30, 2013) . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 14

Honor Roll of Donors FY 2013

• How Your Gifts Were Used . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 16

• Legacy Society . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 17

• Funding Research to Find a Cure - Major Gift Campaigns . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 20

• Vision for a Cure Campaign - Gifts and Pledges . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 21

• Named Funds . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 22

• Planned Gifts and Bequests . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 23

• Special Events and Fundraising . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 23

• Report of Gifts $1,000 and Above (July 1, 2012 – June 30, 2013) . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 24

Programs and Education Report 2013

• From the Chair of the Programs and Education Committee . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 26

• FY 2013 Program Activities and Accomplishments . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 27

Research Report 2013

• From the Chair of the Research Committee . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 30

• From the Director of Research and Clinical Affairs . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 31

• Research Road Map . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 33

• CurePSP Genetics Program and the Whole Exome Sequencing Project . . . . . . . . . . . . . . . . . . . . . . . . . . 35

• Prusiner Transgenic Rat Model . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 38

• Eloise H . Troxel Memorial Brain Bank . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 38

• 2013 International Research Symposium . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 38

• 2013 Funded Research Grants . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 40

• Comprehensive Inventory of Research Grants 1997 – 2013 . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 44

Methods of Supporting CurePSP . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 53

Michael Steinhardt Speech Memorializing Karen Strauss Cook . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 54

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It is a great privilege to accept the dedication of the 2013 CurePSP Annual Report in memory of my wife Karen Cook, who passed away this past October after nearly six years of living with symptoms caused by PSP . Though the last six years of her life were marked by increasingly compromised conditions, up until that time, Karen lived a very full life characterized by hard work, initiative and continual success . When she received a definitive diagnosis for PSP, we decided together as a family that we would do everything that we could to prevent this disease from occurring in others . Karen always lived her life with a sense of purpose and, given her disease prognosis, she wanted her misfortune to serve a purpose for others . She and I, along with our two sons, felt that there could be no higher purpose than assisting in the effort to find a cure or prevention for PSP . It has recently become apparent that such a cure or prevention could also address many other neurodegenerative diseases, including Alzheimer’s . I joined the board of CurePSP for the express purpose of fulfilling this objective .

We have come a long way in the two years that I have been involved with CurePSP . The recent advances in science suggest that a breakthrough is finally within our grasp . We still have a long way to go, but, with the same determination and proactivity that Karen applied to everything she did professionally, philanthropically, and personally, I am confident that we will prevail . She would not accept anything less . I hope you will join me in supporting a cause that meant so much to Karen and that could benefit so many .

At the back of this report is a reprint of the speech given at a recent event honoring Karen and her work . It portrays her dynamic life, one that was tragically cut short by PSP . Let’s work together to prevent a similar fate in others, thereby enabling a world free from these diseases .

Everett R . CookVice ChairCurePSP

The 2013 Annual Report Is Dedicated to Karen Strauss Cook

The Cook family - Everett, III, Karen, Everett, II, and Conor.

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VisionA World Free of PSP, CBD and Related Brain Diseases

MissionIncrease awareness of progressive supranuclear palsy, corticobasal degeneration, and other atypical Parkinsonian disorders; fund research toward treatment, cure and prevention; educate healthcare professionals; and provide support, information and hope for affected persons and their families .

Overview of the FoundationRESEARCH

CurePSP advances the understanding of progressive supranuclear palsy (PSP), corticobasal degeneration (CBD) and closely related brain diseases by funding research that identifies the causes and risk factors for these disorders, developing early stage diagnostic tests, and developing treatments or interventions to ameliorate symptoms and to prevent, slow, halt or reverse disease progression .

PATIENT AND CAREGIVER SUPPORT

CurePSP serves the needs of patients and caregivers managing with PSP, CBD, and other atypical Parkinsonian disorders in order to maintain well-being and quality of life . The Foundation serves as the core and link to a network of support, ensuring accessibility of information, education and counsel for each patient and caregiver confronted by PSP, CBD, and other atypical Parkinsonian disorders . CurePSP provides services including patient/caregiver educational resources, support groups, volunteer development, professional collaborations, online resources, webinars, social media, national conferences, and international leadership . The Foundation focuses on symptom management to improve the daily living of people who struggle to maintain dignity, self-reliance and hope . CurePSP offers patient care resources on PSP, CBD, and other atypical Parkinsonian disorders to healthcare institutions, peer organizations, and facilities serving the elderly and disabled .

EDUCATION FOR MEDICAL AND HEALTHCARE PROFESSIONALS

CurePSP provides scientific and clinical information/resources regarding the specific nature of PSP, CBD, and other atypical Parkinsonian disorders—including their diagnoses, symptoms, and treatments—to neurologists, physical therapists, occupational therapists, speech-language pathologists and nurses . The Foundation offers professional training, when appropriate and possible, through various print and web-based communications in addition to conferences held in association with professional organizations .

PATIENT ADVOCACY AND PUBLIC AWARENESS

CurePSP serves as a unified voice and advocate on various public policy issues for individuals affected with PSP, CBD and other atypical Parkinsonian disorders . CurePSP works in coordination with private non-profit organizations, pharmaceutical companies and the public sector to encourage accelerating biotech innovation for rare disease treatments through science-driven public policy . CurePSP helps educate the public and governmental leaders about research and an improved quality of life for people living with PSP, CBD and other atypical Parkinsonian disorders . CurePSP works in partnership with major patient advocacy groups, such as the Parkinson Action Network and the National Organization for Rare Diseases, on behalf of those with atypical Parkinsonian disorders . CurePSP works in coordination with federal agencies, such as the National Institutes of Health, Federal Drug Administration and the Social Security Administration, which impact research and services for its constituents . CurePSP is part of the continuing struggle to achieve research freedom for human embryonic stem cell research .

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This is my last address to you, as I retired from the Board of Directors on June 30, 2013, after also serving nearly five years as its Chair .

As I reflect on the last few years at CurePSP, I can report huge improvements in the educational offerings . This has been helped by technological advances that allow us, for example, to offer educational webinars and the ability to post our “how to” video vignette series on YouTube . The information families need to get through these diseases physically and emotionally is all available . With the support groups and volunteers to just talk to people, no one now needs to go through this difficult journey alone .

I believe we need two major things going forward…and these both need your assistance .

The first is more volunteers to lead support groups, join committees and even consider serving on our Board .

The next is to kick the research efforts into high gear . All of us that have dealt with this disease want desperately to get a cure and some better treatments . Your donations and solicitation efforts are our major source of funding for this . We all can do more by using the new tools available from CurePSP to request donations from our family, friends and colleagues . When we reach out to this broader community, our funds raised, and opportunity to impact education and research, can expand exponentially .

I will continue to stay involved by facilitating my three support groups and conducting my annual fundraising email solicitation campaign to my friends and family . Please join me in doing all we can to support each other through these diseases and fund research for a cure .

From the Former Chair of the Board of DirectorsJanet Edmunson, MEd

Janet Edmunson presenting at the 2013 Northeastern Family Conference in Boston, MA.

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From the Current Chair of the Board of DirectorsJohn Burhoe

Succeeding an extraordinary leader is always a challenge, as is my task of following Janet Edmunson’s four year stewardship as Chair of CurePSP . Under her leadership, Programs and Education have expanded and flourished with Research maintaining a steady growth . For her accomplishments, Janet has been recognized by the Board with Honorary Board membership, a title only granted to a very few, and most richly deserved . Thank you, Janet, for your leadership and setting the bar high .

Our Board is driven by firsthand experience in the importance of CurePSP’s support services for patients and their families . Personally, I lost my wife, Mary Lou, to PSP in 2008 . This is a

common experience of nearly every board member .

These are also very exciting times for our research program . The past year has brought further evidence of a potential critical link between the cause of PSP/CBD and those of Alzheimer’s, Parkinson’s, ALS, frontotemporal dementia (PSP, CBD, etc .) and chronic traumatic encephalopathy . Consider the possibilities and implications . We have known for many years that finding a cure for one “tauopathy” such as PSP, CBD or Alzheimer’s could provide a cure for the others . These new discoveries broaden that common ground of research to other neurodegenerative disorders where the protein that aggregates abnormally is one other than tau . More simply stated, a discovery sponsored by CurePSP could have an impact on all of its sister neurodegenerative diseases and vice versa . CurePSP now has the opportunity, indeed the responsibility, to utilize its relationships with many preeminent research organizations (University of California San Francisco, University of Pennsylvania, Tau Consortium, etc .) to be a leader in these efforts . This is our goal!

To address these opportunities, CurePSP established a new Research Road Map at our June Board Meeting . CurePSP has also constructed an ambitious grant funding mechanism, “Special Initiative Grants,” to support the work of preeminent scientists and medical researchers .

We are at a critical point where CurePSP can continue as one of many worthy organizations pursuing a common goal, or, we can lead this effort; utilizing our motivation, passion, talent and associations to find the cure not only for PSP; but, potentially all neurodegenerative diseases . Please join us on this journey .

John Burhoe, Nobel Laureate Dr. Stanley Prusiner and Dr. Richard Zyne.

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On behalf of the staff and board of CurePSP, I am pleased to present the Annual Report for 2013 (representing the fiscal year July 1, 2012 through June 30, 2013) to our patients, caregivers, healthcare professionals and supporters . To all of those with whom we serve and work, this report is dedicated to our continuing vision of a world free of PSP, CBD and related brain diseases . I thank our Board of Directors, which has moved the Foundation to higher levels of service and commitment in finding a cure for these rare brain disorders and for improving patient quality of life .

2013 was a highly productive year and we continue to build upon the strength of our staff, volunteers and scientific advisors to expand our programs in research, patient and caregiver support, medical education, patient advocacy, and public awareness .

Here are some of the highlights of 2013:

RESEARCH

Since the inception of our research program in 1997, CurePSP has funded 144 grants valued at $11 million . During FY 2013, CurePSP:

• Completed nine research projects in general research, genetics, and Brain Bank support valued at approximately $1 .15 million (see the Research section of this report for details on the results of these projects)

• Awarded seven new research grants in general research, genetics, and student fellowships totaling $928,000• Continued the work of the Charles D. Peebler, Jr. PSP and CBD Genetics Program through the PSP whole exome sequencing

project through funding by the Morton and Marcine Friedman Foundation and the Peebler PSP Research Foundation• Helped maintain and fund the Eloise H. Troxel Memorial Brain Bank in support of investigator-initiated projects, clinical trials

and genetics research - the Brain Bank has been essential in the work of the CurePSP Genetics Consortium and the Charles D. Peebler, Jr. PSP and CBD Genetics Program, providing hundreds of vital brain tissue specimens for DNA assessment

• Initiated new work with CBD Solutions to develop a clinical rating scale for corticobasal degeneration to determine disease progression and natural history, which are essential to outcome measures in therapeutic trials

• Provided eleven brain tissue harvesting grants to patients and families . Since 2007, CurePSP has awarded 119 grants worth over $66,000

• Conducted the annual International Research Symposium in November 2013 in Baltimore, Maryland with eleven presentations representing research in protein misfolding, the prion hypothesis, genetics, treatment, and clinical research - over forty scientists, students and guests attended the meeting

• Continued a strong working relationship with the Tau Consortium (Bruce Miller, MD, UCSF, et .al .) and provided continued funding for special projects

• Initiated the Urso Student Fellowship Program for PSP Research• Funded the first CurePSP special initiative grant to Nobel Laureate Stanley Prusiner, MD for the development of transgenic rats

expressing wild-type and mutant human tau• Initiated the Research Road Map - a comprehensive, multi-year, $15 million program focusing on PSP, CBD and related disorders

From the President-CEORichard Gordon Zyne, DMin

Dr. Michael DeTure presenting at the 2013 Research Symposium.

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PATIENT AND CAREGIVER SUPPORT, EDUCATION AND ADVOCACY

CurePSP, at its origin, has always been about serving those who were suffering with these diseases and how to provide them with resources, education, moral support, and especially hope . It has been one of our major goals to increase our services to these people through our outreach and to establish support systems to help them during their trying multi-year journey . During FY 2013, CurePSP:

• Developed and inaugurated a new content-rich website for patients, caregivers, researchers, and the general public• Held a major national patient/caregiver conference in the Boston area• Received a 500% increase in requests for information• Increased the number of support groups by 23% to 44 throughout the United States and Canada• Updated and revised all educational materials for patients, caregivers and healthcare professionals• Promoted the development of support groups in Canada and actively began exploration of the development of a Canadian

affiliate• Established a Physician Education Committee• Continued to develop education, training and resources for neurologists, nurses, physical therapists, occupational therapists,

and speech-language pathologists• Successfully worked with the Social Security Administration to expand the Compassionate Allowance Program to include PSP,

CBD, MSA• Continued to work in strong partnership with NORD (National Organization for Rare Diseases) and PAN (Parkinson’s Action

Network) on behalf of patients and caregivers and to support federal legislation to accelerated access to treatments and improve regulation of services

DEVELOPMENT AND FINANCES

CurePSP uses donated funds appropriately and our foundation continues to meet generally accepted standards in the areas of governance, financial management, programmatic standards and donor relations . I am proud to report that the Foundation meets all standards of accountability—with a perfect rating—for a non-profit organizations through the Better Business Bureau . During FY 2013, CurePSP:

• Maintained an 81% to 19% ten-year ratio of “program services” to “support services”

• Maintained a strong and sound financial position• Received 8,700 gifts worth more than $1 .7 million from 7,200

donors for all purposes• Raised more than $560,000 in restricted gifts for research • Raised a total of $1,960,773 from 1,110 donors for the Vision

for the Cure Campaign since its inception in 2011• Received $67,400 from bequests and planned gifts• Raised over $249,000 through special events and activities

I continue to be encouraged by the work and success of the Foundation . As we enter our 24th year of service, we look forward with a renewed commitment to our mission and an abiding gratitude for our partners in this caring endeavor . On behalf of our board and staff, I thank you for your generous and active participation in CurePSP .

Rolly Miles Golf Tournament for CurePSP participants.

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The Strategic Vision FY 2014 establishes the criteria for long-range planning, annual planning, and budgeting for the new fiscal year that begins July 1, 2013 through June 30, 2014 . It is founded upon the vision and mission of CurePSP and serves as the basis for strategic decision-making .

RESEARCH

STRATEGIC VISION STATEMENT

CurePSP will advance the understanding of progressive supranuclear palsy (PSP), corticobasal degeneration (CBD) and closely related brain diseases by funding research focusing on: identifying the causes and risk factors for these disorders; developing early stage diagnostic tests and developing treatments or interventions to ameliorate symptoms and to prevent, slow, halt or reverse disease progression .

OBJECTIVES FY 2014

RESEARCH ROAD MAPCurePSP initiated the Research Road Map at the end of FY 2013 as a framework for a comprehensive array of projects they may eventually lead to treatments, therapies and even a cure for PSP, CBD and related disorders . The Foundation recognizes that the fight against these diseases can now take a new direction, capitalizing on recent discoveries, exciting ongoing work and new hypotheses . We propose to fund research initiatives in five areas that in many cases can proceed in parallel - genes, prions, proteins, models, and markers (see Research Report section for more details) .

RESEARCH PROJECTSIncrease support for investigator-initiated research projects in studies focused on epidemiology, pathology, pathophysiology, and genetics and projects focused on developing diagnostic tests, interventions or treatments for these disorders .

INTERNATIONAL RESEARCH SYMPOSIUMContinue to conduct and fund the annual International Research Symposium, which allows recent CurePSP grantees to present their results publicly, encourages networking among interested researchers and scientists, and fosters interest from the National Institutes of Health and other major funding organizations .

TRAINING RESEARCH FELLOWSHIPSContinue the annual Urso Summer Student Program in PSP research, and study the possibility of a career development award for early–stage academic researchers .



Serve the needs of patients and caregivers managing with PSP, CBD, MSA and related disorders in order to maintain well-being and quality of life . Also, serve as the core and link to a network of support, ensuring accessibility of information, education and counsel for each patient and caregiver .

OBJECTIVES FY 2014

PATIENT AND CAREGIVER RESOURCESContinue to develop educational materials for patients, caregivers, and healthcare professionals that is accurate, comprehensive and current . It is necessary for educational resources to be written in lay language and produced in a variety of formats to meet the various needs of patients and caregivers .

SUPPORT NETWORKS Continue to expand support networks in major metropolitan and other areas where there are volunteers and online support groups . Also, continue to facilitate the development of an affiliate group, support groups and other resources in Canada .

VOLUNTEER RECRUITMENT, DEVELOPMENT AND TRAININGContinue to develop new leadership within the volunteer network and conduct ongoing assessments of the needs of support group leaders and general volunteers .

PEER-ORGANIZATION COLLABORATIONSCollaborate and exchange information with peer organizations, especially those whose disease symptoms are closely related to PSP, CBD, MSA and related disorders .

NATIONAL CONFERENCESDevelop and promote national conferences which will provide opportunities for bringing together patients, caregivers, volunteers, and healthcare professionals for concentrated education and support .

EDUCATIONAL RESOURCES ON WEBSITEOffer webinars on educational topics of need for patients and caregivers . Use social media outlets, when appropriate, for patient support and education .

INTERNATIONAL LEADERSHIP AND PROGRAM DEVELOPMENTContinue to collaborate and exchange information with its international partners in the UK, Europe, Canada and elsewhere .

Strategic Vision FY 2014

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CurePSP will provide scientific and clinical information/resources regarding the specific nature of PSP, CBD, MSA and related disorders—including their diagnoses, symptoms, and treatments—to neurologists, physical therapists, occupational therapists, speech-language pathologists and nurses .

OBJECTIVES FY 2014

GENERAL NEUROLOGISTS AND MOVEMENT DISORDER SPECIALISTS Implement a Physician Education Committee, composed of neurologists and other physicians, to explore ways to promote PSP, CBD, MSA and related disorders and provide educational resources for professionals .

PHYSICAL THERAPISTS, OCCUPATIONAL THERAPISTS, SPEECH- LANGUAGE PATHOLOGISTS, NURSES, SOCIAL WORKERSFacilitate opportunities for education and training for various types of therapists and healthcare professionals . Educational materials and learning opportunities will be developed specifically for these disciplines .

ADVOCACY AND GOVERNMENTAL RELATIONS


Serve as a unified voice to appropriate governmental entities, including the Congress of the United States, and advocate on various public policy issues for our constituents . Continue to help educate the public and governmental leaders about research and an improved quality of life for our constituents .

OBJECTIVES FY 2014

MAINTAIN AND STRENGTHEN ACTIVE RELATIONSHIPS WITH PATIENT ADVOCACY ORGANIZATIONS Continue to work with the Parkinson’s Action Network (PAN), the National Organization for Rare Diseases (NORD), and the Rare Disease Legislative Advocates on matters concerning patient advocacy, education of public leaders, and governmental relations on behalf of our constituents with PSP, CBD, MSA and related brain disorders .

MAINTAIN AND DEVELOP RELATIONSHIPS WITH FEDERAL AGENCIES AFFECTING THE NEEDS OF CUREPSP PATIENTSContinue to communicate and promote the needs of patients and the opportunities for research with the National Institutes of Health (NIH), the National Institute of Neurological Diseases and Stroke (NINDS), the NIH Office of Rare Diseases Research, the FDA Office of Orphan Products Development (OOPD), and the Social Security Administration on behalf of patients with PSP, CBD, MSA and related disorders .

DEVELOPMENT OF PATIENT REGISTRIES Continue to support the development of a patient registry for progressive supranuclear palsy and corticobasal degeneration and work with Rutgers Robert Wood Johnson Medical School on this project .

PUBLIC AWARENESS AND COMMUNICATIONS


Expand the general public’s awareness of PSP, CBD, MSA and related brain diseases through use of various print and web-based media, providing education and general information . Also, continue to enhance CurePSP’s corporate image, identity, branding, and position in the not-for-profit health field .

OBJECTIVES FY 2014

WEBSITE, SOCIAL MEDIA, AND GENERAL PUBLIC INFORMATIONContinue to explore and implement new technologies that will aid in expanding the reach of the organization’s message, expand and improve the quality of its social media presence, and refine printed materials on an ongoing basis in order to maximize both the impact and effectiveness those materials will have on the general public .

Strategic Vision FY 2014

Staff member Chantel Stokes exhibiting at the 3rd annual World Parkinson Congress.

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Leadership and Staff

OFFICERS

CHAIRJohn T . BurhoeDallas, Texas

VICE CHAIREverett R . CookNew York, New York

TREASURERGeorge S . Jankiewicz, CPA, CFP, MBATowson, Maryland

SECRETARYDavid J . PeeblerMontclair, New Jersey

IMMEDIATE PAST CHAIRJanet M . Edmunson, MEdSouth Portland, Maine

PRESIDENT-CEORichard Gordon Zyne, DMin (Ex Officio)Timonium, Maryland

BOARD MEMBERS

Burton Benjamin, MDStamford, Connecticut

Yvette M . Bordelon, MD, PhDLos Angeles, California

Amy BranchNew York, New York

Heather J . Cianci, PT, MS, GCSPhiladelphia, Pennsylvania

Brendan M . DixonPasadena, California

Jeffrey S . Friedman, MD, PhDSan Diego, California

Lawrence I . Golbe, MDNew Brunswick, New Jersey

Stephen S . GoldmanDerwood, Maryland

Rachel G . Gross, MDPhiladelphia, Pennsylvania

Daniel JohnsonSwift Current, Saskatchewan,Canada

James G . McClellan, CPA/PFSSugar Land, Texas

William R . McFarlandDallas, Texas

John A . H . Porter, MD, FAANAdvance, North Carolina

Patricia RichardsonLos Angeles, California

James J . SheaSherwood, Wisconsin

Ileen J . WatsonAnnapolis, Maryland

HONORARY AND EMERITUS MEMBERSHONORARY CHAIRJohn C . Steele, MD, FRCPTamuning, Guam

CHAIR EMERITUSStephen G . Reich, MDBaltimore, Maryland

Joanne ArmstrongSykesville, Maryland

Janet M . Edmunson, MEdSouth Portland, Maine

Paul H . Freeman, JDMiami Beach, Florida

Murray Goldstein, DO, MPHBethesda, Maryland

James E . KoehnleinTimonium, Maryland

CHAIRLawrence I . Golbe, MD Rutgers Robert Wood Johnson

Medical SchoolNew Brunswick, New Jersey

Lester I . Binder, PhDNorthwestern University,

Feinberg School of MedicineChicago, Illinois

Ted M . Dawson, MD, PhDJohns Hopkins University,

School of MedicineBaltimore, Maryland

Dennis W . Dickson, MDMayo Clinic, College of MedicineJacksonville, Florida

Etienne C . Hirsch, PhDHospital de la SalpetriereParis, France

Günter U . Höglinger, MDTechnical University Munich,

German Center for Neurodegenerative Disease

Munich, Germany

Virginia M .-Y . Lee, PhDUniversity of Pennsylvania,

School of MedicinePhiladelphia, Pennsylvania

Irene Litvan, MDUniversity of California, San

DiegoLa Jolla, California

Christiane Richter-Landsberg, PhD

University of OldenburgOldenburg, Germany

Huw R . Morris, PhD, FRCPCardiff UniversityCardiff, Wales, United Kingdom

David E . Riley, MDUniversity Hospitals

Neurological InstituteSouth Euclid, Ohio

Gerard D . Schellenberg, PhDUniversity of Pennsylvania,


David Williams, MBBS, PhD, FRACP

Alfred Hospital, NeurosciencesMelbourne, Australia

Richard Gordon Zyne, DMin (Ex Officio)

President-CEO, CurePSPTimonium, Maryland

SCIENTIFIC ADVISORY BOARD

CORPORATE LEADERSHIP AND BOARD OF DIRECTORS

SCIENTIFIC AND MEDICAL LEADERSHIP

GENETICS CONSORTIUM AND THE CHARLES D. PEEBLER, JR. PSP AND CBD GENETICS PROGRAM

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Leadership and Staff

PRINCIPAL INVESTIGATORGerard D . Schellenberg, PhDUniversity of Pennsylvania,


Bernie Devlin, PhDUniversity of Pittsburgh,

School of MedicinePittsburgh, Pennsylvania

Dennis Dickson, MDMayo Clinic, College of

MedicineJacksonville, Florida

Rohan de Silva, DPhil University College London,

Institute of NeurologyLondon, United Kingdom

Matthew J . Farrer, PhDMayo Clinic, College of


Hakon Hakonarson, PhDChildrens Hospital of

Philadelphia, Center for Applied Genomics

Philadelphia, Pennsylvania

John Hardy, PhDUniversity College London,


Günter U . Höglinger, MDTechnical University Munich,

German Center for Neurodegenerative Disease

Munich, Germany

Andrew Lees, MD, FRCP University College London,


Ulrich Müller, MD, PhDUniversity Hospital Giessen

and MarburgGiessen, Germany

Rosa Rademakers, PhDMayo Clinic, College of


Chang-En Yu, PhDUniversity of Washington,

Seattle Veterans Affairs Medical Center

Seattle, Washington

CHAIRHeather Cianci, PT, MS, GCS Dan Aaron Parkinson’s

Rehabilitation CenterPhiladelphia, Pennsylvania

Ellen Belle, MA, PTColorado Neurological

InstituteEnglewood, Colorado

Diane Breslow, MSW, LCSWNorthwestern University,

Feinberg School of MedicineChicago, Illinois

Becky Dunlop, RN, BSNJohns Hopkins Parkinson’s

Disease & Movement Center

Baltimore, Maryland

Christine Robertson Roxberry, OTR/L

Dan Aaron Parkinson’s Rehabilitation Center

Philadelphia, Pennsylvania

Laura Purcell Verdun, MA, CCC-SLP

VoiceTrainer, LLCFairfax, Virginia

PRESIDENT-CEORichard Gordon Zyne, DMin

VP, DEVELOPMENT & DONOR RELATIONSKathleen Matarazzo Speca

VP, PROGRAMS & EDUCATIONTrish Caruana, MSW

CHIEF FINANCIAL OFFICERJohn Fiedler, CPA

DIRECTOR, RESEARCH & CLINICAL AFFAIRSLawrence I . Golbe, MD

ADMINISTRATIVE MANAGERKarin Martin

DIRECTOR, COMMUNICATIONS & MARKETINGBruce M . Janele

ACCOUNTING MANAGER & RESEARCH ADMINISTRATORAdrienne Bantum

DEVELOPMENT SERVICES MANAGERWenona Hill

PROGRAM ASSISTANT & VOLUNTEER COORDINATOR Chantel Stokes

DEVELOPMENT ASSISTANTKelly Saunders

ADMINISTRATIVE VOLUNTEERSSara GiffenBen LovettLuke LovettJoey Minch Julie Seeberger

Paul H . Freeman, JDMajor GiftsMiami Beach, Florida

Steve GarciasPrograms & EducationRingwood, New Jersey

Robert Hamill, MDResearchUnderhill, Vermont

Dan HeinsPrograms & EducationLouisville, Kentucky

Adam Kleeman, CPA FinanceBaltimore, Maryland

Irene Litvan, MD ResearchLa Jolla, California

Cindy MacDonald Programs & EducationNaples, Florida

Jane Oppenlander, PhD ResearchBurnt Hills, New York

Gary Rose Programs & EducationLongview, Washington

Shawn Smith, MDPhysician EducationElkridge, Maryland

Russell Swerdlow, MD ResearchKansas City, Kansas

MEDICAL PROFESSIONAL ADVISORY COMMITTEE

CORPORATE MANAGEMENT AND STAFF

NON-BOARD COMMITTEE MEMBERS

14

STATEMENTS OF FINANCIAL POSITIONJUNE 30, 2013 AND 2012

ASSETS 2013 2012 ___________ ___________CURRENT ASSETS: Cash and Cash Equivalents $ 861,143 $ 892,503 Cash and Cash Equivalents – Temporarily Restricted 943,747 1,595,538 Accounts Receivable - Credit Cards 6,409 17,542 Pledges Receivable 1,000 1,100 Pledges Receivable - Temporarily Restricted 13,850 41,450 Prepaid Expenses 26,636 22,931 ___________ ___________

Total Current Assets 1,852,785 2,571,064 ___________ ___________

PROPERTY AND EQUIPMENT - AT COST: Office Equipment 99,406 95,267 Furniture and Fixtures 42,700 42,700 Software – Database 54,033 54,033 Website 31,253 31,253 ___________ ___________ 227,392 223,253 Less Accumulated Depreciation 205,223 196,963 ___________ ___________

Net Value of Property and Equipment 22,169 26,290 ___________ ___________

LONG TERM ASSETS: Cash and Cash Equivalents – Permanently Restricted — 4,060 Marketable Securities – Permanent Endowment 351,244 321,309 Deposits 3,983 3,983 ___________ ___________ Total Other Assets 355,227 329,352 ___________ ___________

TOTAL ASSETS $ 2,230,181 $ 2,926,706 ___________ ___________ ___________ ___________

LIABILITIES AND NET ASSETSCURRENT LIABILITIES: Accounts Payable and Accrued Expenses $ 76,403 $ 101,848 Grants Payable – Due Within One Year 368,570 677,440 ___________ ___________ Total Current Liabilities 444,973 779,288 LONG TERM LIABILITIES: Grants Payable – Due After One Year 43,750 125,070 ___________ ___________ Total Liabilities 488,723 904,358 ___________ ___________ NET ASSETS: Unrestricted Undesignated 786,077 753,194 Board Designated – Research — 100,000 Board Designated – Programs and Education 8,935 8,935 ___________ ___________

Total Unrestricted 795,012 862,129 ___________ ___________

Temporarily Restricted 595,277 834,850 Permanently Restricted 351,169 325,369 ___________ ___________

Total Restricted 946,446 1,160,219 ___________ ___________

Total Net Assets 1,741,458 2,022,348 ___________ ___________

TOTAL LIABILITIES AND NET ASSETS $ 2,230,181 $ 2,926,706 ___________ ___________ ___________ ___________

15

STATEMENT OF ACTIVITIES AND CHANGE IN NET ASSETS FOR THE YEAR ENDED JUNE 30, 2013 AND 2012

2013 Temporarily Permanently Unrestricted Restricted Restricted Total ___________ ___________ ___________ ___________REVENUES AND OTHER SUPPORT: Contributions $ 936,720 $ 518,125 $ — $ 1,454,845 Special Events (Net of $21,183 in Expenses) 206,888 41,766 — 248,654 Interest and Dividend Income 2,932 5,486 — 8,418 Realized and Unrealized Gains on Investments — — 29,537 29,537 Grant Adjustment — 125,000 — 125,000 Net Assets Released from Restrictions 933,687 (929,950 (3,737) — ___________ ___________ ___________ ___________

Total Revenues and Other Support 2,080,227 (239,573 25,800 1,866,454 ___________ ___________ ___________ ___________ EXPENSES: Program Services: Research 1,235,276 — — 1,235,276 Programs and Education 383,804 — — 383,804 Communications and Public Awareness 148,510 — — 148,510

Support Services: Management and General 104,345 — — 104,345 Board 38,724 — — 38,724 Fundraising 236,685 — — 236,685 ___________ ___________ ___________ ___________

Total Program and Support Services 2,147,344 — — 2,147,344 ___________ ___________ ___________ ___________ CHANGE IN NET ASSETS (67,117 (239,573 25,800 (280,890

NET ASSETS AT BEGINNING OF YEAR 862,129 834,850 325,369 2,022,348 ___________ ___________ ___________ ___________

NET ASSETS AT END OF YEAR $ 795,012 $ 595,277 $ 351,169 $ 1,741,458 ___________ ___________ ___________ ___________ ___________ ___________ ___________ ___________

2012 Temporarily Permanently Unrestricted Restricted Restricted Total ___________ ___________ ___________ ___________REVENUES AND OTHER SUPPORT: Contributions $ 1,051,251 $ 1,151,025 $ — $ 2,202,276 Special Events (Net of $11,519 in Expenses) 158,238 77 — 158,315 Research Management Fee 36,082 — — 36,082 Interest and Dividend Income 6,528 4,915 — 11,443 Realized and Unrealized Gains on Investments — — (12,480 (12,480 Grant Adjustment — 1,529 — 1,529 Net Assets Released from Restrictions 683,450 (680,135 (3,315) — ___________ ___________ ___________ ___________

Total Revenues and Other Support 1,935,549 477,411 (15,795 2,397,165 ___________ ___________ ___________ ___________ EXPENSES: Program Services: Research 845,683 — — 845,683 Programs and Education 397,258 — — 397,258 Communications and Public Awareness 132,903 — — 132,903

Support Services: Management and General 92,615 — — 92,615 Board 38,608 — — 38,608 Fundraising 216,272 — — 216,272 ___________ ___________ ___________ ___________

Total Program and Support Services 1,723,339 — — 1,723,339 ___________ ___________ ___________ ___________ CHANGE IN NET ASSETS 212,210 477,411 (15,795 673,826

NET ASSETS AT BEGINNING OF YEAR 649,919 357,439 341,164 1,348,522 ___________ ___________ ___________ ___________

NET ASSETS AT END OF YEAR $ 862,129 $ 834,850 $ 325,369 $ 2,022,348 ___________ ___________ ___________ ___________ ___________ ___________ ___________ ___________

)

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16

HONOR ROLL OF DONORS For the Period July 1, 2012 through June 30, 2013

THANK YOU ALL

CurePSP is indebted to the many donors who have so graciously generated support for programs and services . Gifts acknowledged in this report are for the 12 month period beginning July 1, 2012 through June 30, 2013 . In addition, we will be including a complete honor roll of donors on our website, www .curepsp .org . Due to space limitations, we will only be including donors who have given gifts of at least $1,000 in the Honor Roll seen in this report . A list of all donors may be seen in the Honor Roll on our website .

While every effort has been made to ensure the accuracy of our donor lists, errors and omissions in this report may have occurred . Our supporters are assured of our appreciation for their generosity, and we apologize for any inaccuracies in this report . We would kindly appreciate having them brought to our attention and we will make every effort to present corrections in subsequent publications .

During FY 2013, (July 1, 2012 through June 30, 2013) CurePSP received more than 8,700 gifts from 7,200 donors totaling more than $1,722,000 .

HOW YOUR GIFTS WERE USED – FY 2013

82% FOR PROGRAM SERVICES | 18% FOR SUPPORT SERVICES

RESEARCH

FUNDRAISING

MANAGEMENT

AWARENESS

PROGRAMS & EDUCATION

57%

18%

7%

7%

11%

http://www.curepsp.org

17

The Legacy Society

CurePSP offers donors many giving opportunities to support various services of the Foundation, including research, outreach, education, and general operating . All contributions are gratefully appreciated, acknowledged, and honored for their generosity .

The Legacy Society is a distinct group of major benefactors who have responded to the call of long-term commitment and leadership by contributing cumulative donations exceeding $10,000 . Contributions by Legacy Society Benefactors have been made through direct and current gifts of cash or property but may also be made through planned gifts such as bequests, life insurances, or trusts . Legacy Society Benefactors have a strong commitment to the services and programs provided by CurePSP and are recognized by this special presentation in the Annual Report and Honor Roll of Donors .

Since 1994, Legacy Society benefactors have contributed more than $15,940,000 to CurePSP .

TRANSFORMATIONAL BENEFACTORS

DIAMOND BENEFACTORSCUMULATIVE GIFTS OF $1,000,000 AND ABOVE

Transformational Benefactors are those institutions, entities or individuals who have made a major and consistent impact on the Foundation through their contributions and their services over the years .

Mr . Jay C . Troxel - 2006

Mr . Charles D . Peebler, Jr . and the Peebler PSP Research Foundation - 2008

Mr . Abe Pollin & Mrs . Irene Pollin - 2009

Peebler PSP Research Foundation, Inc .

Mr . Abe Pollin & Mrs . Irene Pollin

Mr . Jay C . Troxel

PLATINUM BENEFACTORSCUMULATIVE GIFTS OF $500,000 TO $999,999

Morton and Marcine Friedman Foundation

Mr . & Mrs . Steve Poizner

GOLD BENEFACTORSCUMULATIVE GIFTS OF $100,000 TO $499,999

Anonymous Hazel Reed Baumeister TrustThe Karen and Everett Cook FoundationMs . Joyce EstesMr . Justin Franco, IIIMr . Paul H . FreemanMr . Donald E . Graham

Mr . Jack HedrickMs . Eva HowarthElayne & Benno Hurwitz Philanthropic

FundMr . Peter A . LundMr . Brett MilgrimDudley Moore Research Fund

Lyndon Selter Parker TrustThe Fred and Mabel R . Parks FoundationPerot FoundationRainwater Charitable FoundationEstate of Hannah RubinEstate of Ruth Tarlow UrsoFrank W . Weymouth Living Trust

Cumulative gifts and commitments, July 1994 through June 2013 .

LEGACY SOCIETY BENEFACTORS

18

SILVER BENEFACTORSCUMULATIVE GIFTS OF $50,000 TO $99,999

BRONZE BENEFACTORSCUMULATIVE GIFTS OF $10,000 TO $49,999

AnonymousMrs . Sarita AgrawalAkers Foundation, Inc .The Shana Alexander Charitable

FoundationMr . & Mrs . Dennis AlterThe Altus One Fund, Inc .L & N Andreas FoundationEdward H . Andrews FoundationThe Annenberg FoundationMr . & Mrs . Donald BakerBall CorporationBank of America Foundation, Inc .Dr . & Mrs . Bruce BarnettMr . James C . BarronMr . Charles F . BarryMs . Gloria A . BartasThe Gary Dean Beck Family FoundationMr . David A . BellMs . Deborah BergerDr . Allen H . BeznerBridgemill FoundationAlex Brown & Sons IncorporatedMr . William BudgeMr . Charles Burlingham, Jr .Mrs . Mary K . CallawayMrs . D . Wayne CallowayMr . & Mrs . John CampbellMr . & Mrs . Richard M . CashinMrs . Beverly Ann ClarkMrs . Janice Clements

Club Assist c/o Alan W . BradshawCMGRP, Inc .Collinwood Middle SchoolMr . & Mrs . Curt ConoverThe Conover Foundation, Inc .Crestar FoundationMr . Charles CumminsMrs . Elizabeth A . DaftMr . Dee C . DanielsMr . & Mrs . Delmont DavisMr . Peel DillardDIRECTVDIRECTV Matching Gift CenterMr . & Mrs . Douglas A . DonahueMrs . Ann H . DunwodyMrs . Janet M . EdmunsonMs . Barbara FedorMr . & Mrs . Ronald J . FerrisFloyd Charitable Lead UnitrustMs . C . Rae FraneyMr . & Mrs . Andrew M . FreemanMr . Alan C . FreemanMr . & Mrs . Joe A . FriedmanMr . Richard Fuld, Jr .William M . Fuller FoundationMr . W . R . FulljamesCol . Grady D . Gafford, Ret .Mr . & Mrs . Robert GalloisGeller & Co . LLCGive With Liberty / Liberty Mutual

Foundation Match

Mr . Peter T . GrauerMr . Stewart GreenebaumMs . Victoria L . GreenleafDr . George L . GriceThe Estate of Oliver GrotelueschenMr . & Mrs . Martin D . GrussMr . Brack HaleHerbert Hamburger EstateMr . Stephen HamerMr . & Mrs . Timothy HanleyThe Hearst CorporationMr . & Mrs . William Herrman, IIHoag Family FoundationMr . & Mrs . Harry G . HohnMr . Sanford HorowitzHorsley Bridge PartnersMr . Sydney HuffnagleMs . Carol M . HutchinsonInter Public GroupJackson Charitable FoundationThe Nathan P . Jacobs FoundationMr . Richard Janney & Ms . Joanne E .

ArmstrongMr . Levi JohnsonMr . & Mrs . David KaminVernice Kaphgst Charitable Remainder

TrustMr . & Mrs . John B . Kelley, Jr .Mr . Donald W . KeltonMr . & Mrs . Thomas KempnerMr . & Mrs . Peter Kimmelman

The Legacy Society

AnonymousMr . Klaus AlthammerMr . & Mrs . Douglas BloomCAP FoundationMr . & Mrs . E . Paul CaseyMr . & Mrs . Brendan M . DixonMrs . Barbara FoxBurt Harkins Family Foundation

Mr . William H . HerrmanMrs . Mary V . KirkeyMr . Frederick KoallickLight of Day Foundation, Inc .Mr . James E . McGookeyMrs . Edith B . ParkerMs . Patricia C . RichardsonEstate of Blanche Ruppert

Mr . & Mrs . William A . SchreyerMr . Jack SchussMrs . Julia S . ShacklefordIrvin & Ruth Swartzberg Family

FoundationClaude D . Vannoy EstateMr . David WallaceThe Wallis Foundation

19

Mr . & Mrs . James E . KoehnleinMr . & Mrs . Christopher KorvesThe Lapin FoundationMrs . Deloris A . LaRueMr . & Mrs . Bruce R . LauritzenMrs . Lillian LeachMr . Laurence C . Leeds, Jr .Mr . Bruce LiimatainenMr . Sidney LindenbaumLeon Lowenstein Foundation, Inc .MCJ FoundationMr . & Mrs . H . F . MaassabMs . Anne MarionMartine Avenue Productions, Inc .Mr . Colin McCaffertyDr . & Mrs . Kenneth A . McKusickMr . & Mrs . Sean McLaughlinMr . Dave McNaughton and Nexen

Petroleum U .S .A ., Inc .Mr . & Mrs . Paul McNultyMeredith CorporationMerrill Lynch & Co . Inc .The Minneapolis FoundationModern Woodmen of AmericaWm . A . & Elizabeth B . Moncrief FoundationMr . Philip Harry MooreMr . & Mrs . John A . MoranMr . Matthew NastukMr . & Mrs . Denny NeagleMr . Richard Nye and Ms . Francesca

StantillMrs . Ellen J . OleanMr . John D . OlsonMr . Joseph C . OppenlanderMr . Stanley C . PaceMr . & Mrs . Daniel PaduanoMrs . Esther A . PalumboThe Esther A . Palumbo Family FundMrs . Susan D . ParkerPatton Family FoundationMrs . Phyllis R . PerreaultMr . & Mrs . Duane PhillipsPhoenix World Wide EnterprisesMs . Mari-Anne PisarriMr . & Mrs . Frank PizzitolaMs . Sharon PrattMs . Carol Prugh

Qualcomm, Inc .Quanex FoundationMs . Karen W . RainwaterMr . John B . Ricker, IIIMr . John B . Ricker, Jr .Mr . & Mrs . Brent T . RiderThe Estate of A . John RidgeMrs . Sandra L . RileyMrs . Clarice RosenMr . Haim SabanMr . & Mrs . Paul C . Schorr, IIIMr . Roy R . SchwartzMr . Ernest Segundo, Sr .Alice Shaver FoundationMrs . Ruth Eleanor SingerEdward W . Smith, Jr . FoundationMs . Gail SmithMr . & Mrs . Richard W . SmithMr . & Mrs . Thomas SmithSony Computer Entertainment America,

Inc .Sports TutorMr . & Mrs . John SpragueMr . & Mrs . Michael SteinhardtMs . Eleanor R . StewartMr . & Mrs . Lane StokesMs . Emily H . SusskindMr . Richard J . SussmanMr . Gerald TeelMr . & Mrs . J . Liener Temerlin

Janis Temple Bequest Mr . & Mrs . Nathan ThorneMr . Oakleigh ThorneMr . & Mrs . Donald ToberMr . Daniel P . TullyMrs . Lesley J . UndercoflerUnited Way of Southern PennsylvaniaUnited Way of Tri-StateMrs . Kathleen VeehVehicle Donation Processing Center, Inc .Mr . John R . VoigtMr . & Mrs . Eugene M . Waldron, Jr .Mr . William S . Walsh, Jr .Edgar B . & Jane Wyatt Ward FundEstate of Joan WarkickMr . David Weinberg and Ms . Michele

SmithThe Sidney J . Weinberg FoundationPerelson Weiner, LLPMrs . Beverly G . WeiserMr . Eric S . WesolekMrs . Dorothy WhitakerMr . & Mrs . John C . WhiteheadMrs . Marguerita A . WhitneyMs . Mary WilhelmWillkie, Farr & Gallagher, LLPMr . John H . WilsonMrs . Lenore WinsbergMrs . Marie K . ZimmermanZyne Family Trust

The Legacy Society

Board members Amy Branch and Everett Cook with Paula and Richard Zyne.

20

Funding Research to Find a Cure - Major Gift Campaigns

CAMPAIGN FOR GENETICS: 2007 - 2010

The Charles D. Peebler, Jr. PSP and CBD Genetics Program continues its work to help find a cure or prevention for PSP and CBD . Phase One of the program was completed in 2010 and publication of the data was in the July 2011 issue of Nature Genetics . The CurePSP Major Gifts Committee raised $1 .5 million for research projects directly related to the work of the Peebler Genetics Program .

VISION FOR A CURE CAMPAIGN: 2011 - 2013

In January 2011, CurePSP launched its second major gifts campaign to raise funds for a broad range of research projects . Each of these programs is essential for CurePSP to achieve its ultimate goal of a world free of PSP, CBD and related diseases .

• The Charles D . Peebler, Jr ., PSP and CBD Genetics Program• Investigator-Initiated Research Projects• The CurePSP – Eloise H . Troxel Memorial Brain Bank at the Mayo Clinic, Jacksonville, Florida

On June 30, 2013 the campaign came to an end . A total of $1,960,773 was raised from over 1,100 donors .

Everett R . Cook, ChairNew York, New York

Amy BranchNew York, New York

John T . BurhoeDallas, Texas

Brendan M . DixonPasadena, California

Lawrence I . Golbe, MDNew Brunswick, New Jersey

Paul H . Freeman, JDMiami Beach, Florida

James McClellan, CPASugar Land, Texas

Adam M . MurphyAtlanta, Georgia

David J . PeeblerNew York, New York

Patricia RichardsonLos Angeles, California

Richard Gordon Zyne, DMin Timonium, Maryland

MAJOR GIFTS COMMITTEE - AS OF JULY 2013

RESEARCH ROAD MAP 2013

CurePSP initiated the Research Road Map at the end of FY 2013 as a framework for a comprehensive array of projects that may eventually lead to treatments, therapies and even a cure for PSP, CBD and related neurodegenerative brain disorders . The Foundation recognizes that the fight against PSP can now take a new direction, capitalizing on recent discoveries, exciting ongoing work and new hypotheses . We propose to fund research initiatives in five areas that in many cases can proceed in parallel . These include:

• Genes• Prions• Proteins• Models• Markers

See the Research section of this report for details on this campaign .

21

The Vision for a Cure Campaign - Gifts and Pledges

$500,000 AND ABOVEMorton and Marcine Friedman Foundation

$100,000 TO $499,999The Karen and Everett Cook Foundation The Fred and Mabel R . Parks Foundation Peebler PSP Research Foundation, Inc . Perot Foundation

$50,000 TO $99,999Mr . Donald Graham

$25,000 TO $49,999Anonymous Foundation Mr . & Mrs . Richard Cashin Mr . Paul Freeman Mr . Martin Gruss

$10,000 TO $24,999Ms . Gloria BartasMr . Peel DillardMr . & Mrs . Douglas Donahue and

the Donahue Family FundMr . Thomas KempnerMr . Peter Kimmelman and the

Irene Diamond Fund

Mr . Frederick KoallickMr . Lawrence Leeds and the

Leeds Family FoundationNexen Petroleum U .S .A ., Inc .Mr . Richard NyeMr . & Mrs . Richard SmithMr . & Mrs . John Sprague

Mr . Michael Steinhardt and the Steinhardt Foundation

Mr . Richard SussmanMr . Oakleigh Thorne and the

Oakleigh L . Thorne FoundationMr . & Mrs . Nathan ThorneMr . & Mrs . Donald Tober

Mrs . Lesley UndercoflerMr . David WeinbergMrs . Dorothy WhitakerMr . & Mrs . John WhiteheadZyne Family Trust

$5,000 TO $9,999AnonymousMs . Karen BechtelMr . Daniele Bodini and the

Alexander Bodini FoundationMr . Thomas Edelman and the

Thomas J . Edelman FoundationMr . & Mrs . Thomas FlexnerMr . & Mrs . Alex Greenberg

Mr . David Hathaway and the Hathaway Family Foundation

Mr . Bo HongMr . & Mrs . Christopher KorvesMr . & Mrs . Peter Kraus and the

Kraus Family FoundationMs . Kari KuglinMr . Philip Mactaggart

Mr . & Mrs . Richard Menschel and the Charina Foundation, Inc .

Mr . & Mrs . Jack MeyerMs . Jamie MuesingMr . & Mrs . Stuart OranMr . & Mrs . William QuinnEdward W . Smith Jr . FoundationMr . & Mrs . James W . Tozer, Jr .

Mr . & Mrs . Willem de Vogel and the Marion and Willem de Vogel Foundation

Mr . W . Grant Williams, III and the Sayers Foundation, Inc .

$1,000 TO $4,999Mrs . Laurel AndertonArchway Insurance Brokers, LLCAtlas Family FoundationMr . Zack Bacon, IIIMr . David BattMr . Burton BenjaminMrs . Deanne BrowningMr . & Mrs . R . Duke BuchanMr . & Mrs . Rob CernakMrs . Susan ChevinsMr . Russell ConserMr . Dee C . Daniels

Mr . & Mrs . Delmont Davis and the Ball Corporation, Inc .

Mr . Massimo FerragamoMs . Leona FitzsimondsMr . & Mrs . Robert GalloisGE FoundationNomi Ghez FoundationMr . Stephen HamerMr . & Mrs . Frederick HamiltonMr . Kirk HenckelsMr . Robert G . Hottensen

Elayne & Benno Hurwitz Philanthropic Fund

Mr . & Mrs . David KaminEwing M . Kauffman FoundationMr . & Mrs . Benjamin LeCompteMrs . Melissa LeeMrs . Lexi LeopoldMs . Jada LewisMAASSAB FoundationMiami University Honors ProgramMr . Matthew NastukMr . & Mrs . Carl Navarre

Ms . Shirley PriceMr . David ReganMr . & Mrs . Steven SabovikMrs . Karen SeabergMs . Diana SpencerMs . Eleanor StewartMs . Kara ThiemeMr . & Mrs . Frank Torres and the

Google Matching Gifts ProgramMr . Leslie TurnerMr . Spence Wilson

$1 TO $9991110 Gifts

TOTAL: $1,960,7731,174 Total Gifts

22

Named Funds

$1,000,000 AND ABOVEPeebler PSP Research Foundation Irene and Abe Pollin CBD Research Fund Eloise H . Troxel Memorial Fund

$500,000 TO $999,999The Karen and Everett Cook Foundation Morton and Marcine Friedman Foundation Edwin & Pearl Poizner Memorial Fund

$300,000 TO $499,999Eva Freeman Memorial Fund

$100,000 TO $299,999Elayne and Benno Hurwitz

Philanthropic Fund Robert T . Kirkey Memorial Fund Theresa and Peter Lund Research Fund

Dudley Moore Research FundThe Ruth T . Urso Endowment

$20,000 TO $99,999Ken Jennings Legacy FundDavid Livernois Memorial FundCarol J . Major Legacy Fund

Norma Oppenlander Memorial FundLyndon Selter Parker Trust FundMargaret Parker Memorial Fund

Laurence B . Richardson Memorial FundRoberta Schenker Memorial FundZyne Family Trust

$10,000 TO $19,999Colette M . Bednarczyk Memorial Fund Charles R . Edmunson Memorial FundStevens Frink Family Fund

Robert P . Hanrahan Memorial Fund Devon D . Huffnagle Memorial FundVandana Juneja Memorial Fund

Jackie Myers Family Fund

$5,000 TO $9,999Joel H . Gilbert Memorial Fund Nancy Newell Fund

$1,000 TO $4,999Dr . Joseph Cavallaro Memorial FundJoe Dean Memorial FundSylvia Guido FundSelma Aronowitz Klass Memorial Fund

Pansey C . Littles Memorial FundDorothy MacDonald Memorial FundSid Millman Memorial FundDavid S . Olander Memorial Fund

Lucille C . Parrilli Memorial FundAnthony Spare Memorial FundSami and Annie Totah Family Fund

A CurePSP Named Fund provides donors with the opportunity to make gifts that will have a lasting impact while recognizing their family, or honoring or memorializing a loved one . Individuals, groups or businesses can establish a named fund which publicizes their commitment to the mission of CurePSP . An annual donation of at least $1,000 is required to keep a named fund active and recognized .

Named funds of $1,000 or more receive recognition in the CurePSP Annual Report, and may also receive recognition at a family conference, or webinar, in printed materials, or through a research grant depending on the amount raised .

Generous contributions have been made to the named funds below .

23

Planned Gifts and Bequests

PLANNED GIFTS OR BEQUESTS RECEIVED IN FY 2013

Ms . Billie M . ClarkMr . Philip H . Moore

Ms . Blanche RuppertMs . Mary E . Wilhelm

Planned giving is a method of supporting CurePSP that enables philanthropic donors to make larger gifts than they could make from their income . A planned gift is any major gift, such as a bequest, charitable remainder trust, or other instrument, made in lifetime or at death as part of a donor’s overall financial and/or estate planning . During fiscal year 2013, CurePSP received four planned gifts or bequests valued at approximately $67,000 .

Special events are vital to CurePSP’s programs and services and serve as a strong educational tool by creating awareness throughout local communities . Special events also help reach new families affected by atypical Parkinsonian disorders . Thanks to the kindness and generosity of our CurePSP volunteers, 73 special events and/or fundraising activities occurred this fiscal year, throughout 24 different states, which raised $248,654 to support our mission . The following provides a list of events and their locations:

Special Events and Fundraising

Annapolis, MD Terry Watson Memorial Golf Tournament Nationwide CurePSP Wristband FundraisersAtlanta, GA Dining Out - Miracle for Mom Nationwide eBay/Mission FishAtlanta, GA Atlanta Hawks Basketball Game – Miracle for Mom Nationwide Good SearchAtlanta, GA Atlanta Marathon Nationwide Safeway Good NeighborAustin, TX Ironman Triathlon Nationwide Vehicle Donation CenterBaltimore, MD Obrycki’s Restaurant Fundraiser Nationwide Wedding Gift FundraisersBelmont, NY Marylyn’s Spring Stroll Nationwide Writing CampaignsBoston, MA Conference Giveaway Fundraiser New London, CT Amica MarathonBoston, MA Boston Marathon New York, NY Karen’s Hope Writing CampaignBoyds, MD Cure the Pain Walkathon New York, NY New York MarathonBrooklyn, NY Bearduary Event Oneonta, NY Silpada Jewelry Party Chicago, IL Fedor Family Fundraising Event for CurePSP Oneonta, NY Madison Handbag ShowCinnaminson, NJ Charity Denim Day Oxnard, CA Dinner Benefit Denver, CO Denver Triathlon PA (various counties) Gupta GraduationDyersburg, TN Sara Coles Wedding Patrick, SC Earl Walton WalkEnterprise, FL 5K& 15K Relay Run Philadelphia, PA Philadelphia MarathonGreeley, CO Judi Ericson Walk Philadelphia, PA Philly Support Group Ruby Tuesday Dine Out NightsHarlem, NY Bikram Yoga Philadelphia, PA Philadelphia Support Group – Bake SaleHayworth, NJ Andiamo Benefit Pittsburgh, PA Pittsburgh Annual Wine TastingHuron, MI CurePSP Memorial Golf Outing Pittsburgh, PA Montour Trail Half MarathonKenmore and Seattle, WA Barney’s Hummingbird Cards Plainview, NY Abigail’s Ice Cream FundraiserKnoxville, TN National Bridge Club Tournament in honor of Thomas Timm Port Huron, MI CurePSP Memorial Golf TournamentLas Vegas, NV Maura’s Birthday Fundraiser Raleigh, NC 50 Wonderful Things Birthday CelebrationLincoln, CA Newell Family Note Card Fundraiser Silver Creek, GA Cory’s Challenge Madison, WI to Chicago, IL Marathon South Portland, ME Charles R . Edmunson Writing CampaignMarco Island, FL CurePSP Awareness & Memorial Walk Syracuse, IN Ken Jennings Putt for PSP Golf TournamentNantucket, MA Nantucket Triathlon Tampa, FL Fashion ShowNationwide Butterfly Note cards Tempe, AZ Ann Ludwig Dance RecitalNationwide Capital One – CurePSP Credit Card Washington, DC Rock and Roll MarathonNationwide CurePSP Hope Bracelet Fundraisers Wausau, WI Sorenson Soccer FundraiserNationwide CurePSP iPad Giveaway West Babylon, NJ NovemBeardNationwide CurePSP Merchandise (Café Press) White Marsh, MD Boscov’s FundraiserNationwide CurePSP Ribbon Magnet Fundraisers Windsor Lock, CT Turkey Cook Off

24

VISIONARIES FOR A CURE$10,000 AND ABOVE

AnonymousMr . & Mrs . John CampbellEstate of Billie M . ClarkThe Karen and Everett Cook FoundationMr . Brendan M . DixonMr . Paul H . FreemanMorton & Marcine Friedman FoundationMs . Victoria L . GreenleafMr . Jack Hedrick

Mr . Richard Janney & Ms . Joanne E . Armstrong

Light of Day Foundation, Inc .Mr . Dave McNaughtonThe Esther A . Palumbo Family FundMrs . Edith B . ParkerThe Fred and Mabel R . Parks FoundationPerot FoundationMs . Karen W . Rainwater

Ms . Patricia C . RichardsonEstate of Blanche RuppertAlice Shaver FoundationMr . Oakleigh ThorneMrs . Lesley J . UndercoflerMrs . Dorothy WhitakerMs . Mary Wilhelm

Report of Gifts $1,000 and Above

Gifts and commitments, July 1, 2012 through June 30, 2013 .

SEEKERS OF HOPE$5,000 TO $9,999

AnonymousMr . Klaus AlthammerMr . & Mrs . Robert BoucaiMr . & Mrs . E . Paul CaseyMr . & Mrs . Joseph A . FriedmanGive With Liberty /Liberty Mutual

Foundation Match

Mr . & Mrs . Alex GreenbergDr . George L . GriceBurt Harkins Family FoundationMr . William H . HerrmanThe Nathan P . Jacobs FoundationMr . Frederick KoallickMs . Jamie L . Muesing

Mr . Joseph C . OppenlanderPeebler PSP Research Foundation, Inc .Penelope T Charity ShowMr . Ernest Segundo, Sr .Mrs . Julia S . ShacklefordMrs . Lenore Winsberg

ADVOCATES FOR CARE$2,500 TO $4,999

The Conover Foundation IncMrs . Dale FerrisMr . Kirk HenckelsMr . & Mrs . Christopher KorvesThe Lapin Foundation

Ms . Lisa MeyererModern Woodmen of AmericaMrs . Phyllis R . PerreaultEdward W . Smith Jr . FoundationMr . Richard J . Sussman

Mr . Gerald TeelThrivent Financial for LutheransMrs . Sarah C . WardeZyne Family Trust

HEALTH BUILDERS$1,000 TO $2,499

The Jack and Carol Adam Family FundMr . & Mrs . Mark AgeeMs . Swati AgrawalMr . & Mrs . Roger AllenDr . & Mrs . Greg AmayaAmerica’s CharitiesMrs . Delysia Ashwood-McNairBank of America Foundation, Inc .Dr . & Mrs . Bruce BarnettMr . Charles F . BarryMr . David B . BattMr . W . W . BednarczykDr . Burton Benjamin

Mr . Tobias J . BermantMrs . Elizabeth Shackleford Bernardi & Mr .

Ronald BernardiMr . Robert BoccafolaDr . Yvette Bordelon and Dr . Carlos

Portera-CailliauMr . Frederic A . BrossyMrs . Deanne BrowningMrs . Doreen BryantMrs . Ann E . BullockMr . Charles Burlingham, Jr . Cafe PressMrs . Mary K . Callaway

Capital One CorporationMs . Cheri R . CernakMr . & Mrs . Rob CernakCherbec Advancement FoundationMrs . Susan ChevinsMr . & Mrs . Gary L . ColemanMr . Russell J . ConserMr . Charles CumminsMr . Dee C . DanielsMr . & Mrs . Delmont A . DavisMrs . Ann H . DunwodyMr . & Mrs . Drew DurkinEchelon, LLC

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Unfortunately, space does not permit us to recognize all of our generous donors . For a complete list of donors who gave $1 - $999, please reference the 2013 Honor Roll of Donors on our website .

MISSION SUPPORTERS$100 TO $999

2,451 Donors, Total Gifts of $454,327

HELPING PARTNERS$1 TO $99

4,468 Donors, Total Gifts of $164,222

Mrs . Janet M . EdmunsonEnterprise Holdings FoundationEwing M . Kauffman FoundationMrs . Dorothy FalcinellaMr . David FalsoneFifth Group Restaurants, LLCFinal Mile Race Management, LLCMr . Charles FoltzThe Family of Robert & Geneva FosterMs . C . Rae FraneyJ .A . Frate, IncThe Frate GroupMr . Alan C . FreemanDr . Jeffrey S . FriedmanMr . & Mrs . Robert GalloisMr . Bill GaneyGoogle Matching Gifts ProgramMr . and Mrs . Timothy HanleyMs . Donna H . HooperElayne & Benno Hurwitz Philanthropic

FundMr . & Mrs . Jack D . JenningsMr . & Mrs . David KaminMrs . Mary V . KirkeyThe Klaeser FamilyMr . Armin A . KruegerMr . Benjamin LeCompte, IIIMrs . Melissa Nachatelo LeeMrs . Lexi LeopoldMr . Sanford R . LindenbaumRalph A . Loveys Family Charitable

FoundationMr . & Mrs . Eric MarxMr . & Mrs . John McCareyMr . & Mrs . Wilson McElhinnyMr . Brian P . McMillanMr . & Mrs . Paul McNulty

Dr . Faisal MerchantMr . & Mrs . David L . MillerMr . & Mrs . Ralph G . MillerMr . Gaurie MittalMr . Matthew NastukMrs . Marjorie G . NeuwirthDr . Jane E . OppenlanderLyndon Selter Parker TrustParagon GamingDr . Roger PorterMs . Shirley PriceRainier Investment Management, Inc .Mr . & Mrs . Richard RawsonMrs . Sandy RileyMr . Jeff RingRiver Cree Resort & CasinoMrs . Clarice RosenMr . & Mrs . Steven G . SabovikMr . Jack SchussMr . Roy R . SchwartzMr . & Mrs . Tom ScorzaMr . & Mrs . Ladd SeabergMs . Phyllis A . SeitzMr . Norman A . Sensinger, Jr .Mrs . Ruth Eleanor SingerMrs . Phyllis SladeMs . Melanie SmithMs . Linda SpearsJohn Staurulakis, Inc .Dr . C . R . StevesMs . Eleanor R . StewartMr . & Mrs . James M . StewartMr . Steven StraussIrvin & Ruth Swartzberg Family

FoundationMr . Jack C . TaylorMs . Kara Thieme

TLC FoundationMr . Frank TorresTrigon International CorporationUnited Way of Central & Northeastern

ConnecticutUnited Way of New York CityMr . & Mrs . Andrew P . VaterVehicle Donation Processing Center, Inc .Ms . Wendy WalchMr . Frank WangWells Fargo Community Support

CampaignMr . Eric S . WesolekMrs . Marguerita A . WhitneyWhitney FoundationMr . Spence WilsonMr . Brian Yocum

Report of Gifts $1,000 and Above

Staff member Trish Caruana and Board member Dr. Yvette Bordelon.

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Programs & Education Report 2013

As we approach the end of this year I find myself in awe over the accomplishments of the CurePSP organization as a whole, as well as the Programs and Education (P&E) Committee . Several months ago, when I was approached to consider volunteering as a member, there was no hesitation . During my husband’s battle with PSP, the resources that were made available through the Foundation provided many tools to assist me as a caregiver and provided hope for both myself and my husband . After participating as a member of the P&E Committee, I soon learned that many of the resources made available to users like myself were the result of much planning and development by this committee . Soon after I became a member, I was invited to consider chairing the committee and again I accepted . So many people depend on the many

resources defined by this committee, CurePSP and their volunteers .

We are excited about the prospects of the many programs we are offering in the coming year to continue our support of those people diagnosed with PSP, CBD, MSA and other atypical Parkinsonian disorders, as well as their caregivers and families . It is with pride that I am met with this challenge and am grateful for our committee members who possess so much knowledge and history of our past and ongoing mission . Our members have very diversified backgrounds and bring to the table a variety of experience and knowledge .

Additionally, we depend wholly on the CurePSP staff for orchestrating and developing the many resources available through the website, webinars, educational material, conferences and programs to increase the level of awareness about the diseases . The many hours of research, preparation and implementation have contributed to a variety of programs and material used to support patients, caregivers and their families .

I look forward to this coming year and working with our committee members and the CurePSP support staff in the continued efforts to provide the highest quality support in our mission . This being said, the following report contains the major accomplishments for this year and a synopsis of our vision for the future .

From the Chair of the Programs and Education CommitteeIleen J. Watson

Dr. Aaron Haug presenting at the 2013 Rocky Mountain Family Conference.

Staff member Chantel Stokes at CurePSP’s exhibiting booth at the 3rd annual World Parkinson Congress.

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While research has and will continue to be our prime mission focus we are always concerned for our patients with PSP, CBD and related disorders and their caregivers . The Foundation, at its origin, has always been about serving those who were suffering with these diseases and how to provide them with resources, education, moral support, and especially hope . It has been one of our major goals to increase our services to these people through our outreach and to establish support systems to help them during their trying multi-year journey . Here are some of the highlights of our programs and education during 2013 .

RESOURCES

While our service area includes the United States and Canada, CurePSP has had much success in getting its message out to the entire world through publications, newsletters, conferences, website, webinars, social media and especially through the personal touch on the telephone .

CurePSP continually provides materials to patients and caregivers in various forms using a multitude of media tools . As technology improves and becomes more easily available, patients and families may receive their resources from the many on-line and digital productions . Every year we update materials, inform patients about new and additional services, discuss potential breakthroughs in research, and encourage people to participate in clinical trials when they occur . Certainly one of the most creative and useful ways to communicate with patients, caregivers and healthcare professionals is through new technology and especially through our website and the vast array of social media outlets . While many of our patients still do not use a computer because of age or illness, their caregivers are continually viewing our site and communicating on Facebook, Twitter, YouTube, and several other sites . In addition, we keep our constituents and the general public apprised of our work and the achievements in research through public press releases and email blasts . During 2013, CurePSP:

• Completely redesigned the website• Worked with media outlets to facilitate the creation of numerous public relations and awareness pieces, such as NBC Dallas-

Fort Worth ahead of the Dallas conference, CBS Atlanta for Adam Murphy’s fundraisers and The Doctors TV Show on CBS for appearances by Patricia Richardson and Carlos Portera-Calliau, MD

SUPPORT NETWORKS AND VOLUNTEERS

People often experience healing and hope through their interaction with other people . The establishment of support groups and support networks is essential to this process . During 2013, CurePSP:

• Maintained 44 active face-to-face support groups and 10 online support groups• Online groups increased by 29% in FY2013• New face-to-face support groups increased overall by 23% in FY2013• Support group leader trainings increased by 400% - 41 trainings in FY 2013 up from 10 in FY 2012 - increase in trainings and

refinement of training module and materials has led to increased participation of volunteers• Recruited 40 new volunteers, 22 of whom received training during this past fiscal year

PEER ORGANIZATION COLLABORATIONS

CurePSP doesn’t function in a vacuum and we are constantly working with various healthcare organizations and peer organizations to help promote our mission and to make contact with people who may have PSP, CBD or other atypical Parkinsonian disorders . Frequently, the best way to get our message out is to work with support centers, such as Parkinson’s Centers of Excellence, where we may participate in conferences, seminars and training activities for healthcare professionals . It’s also one of the best ways to get to know movement disorder specialists who may have patients with PSP and CBD . Because our disorders are very similar in many ways to other disorders, we coordinate activities from such organizations as the Association for Frontotemporal Degeneration, the MSA Coalition and sister organizations in the UK . During 2013, CurePSP:

• Presented at the annual PSP conference at the Struthers’s Center in Minnesota


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• Attended the Genetic Alliance Bio Bank training in Washington, DC• Conducted live in-services for healthcare staff at Sanctuary of Holy Cross in Silver Spring, Maryland • Met with Movement Disorder Specialists at SUNY Downstate and Sinai Hospitals in New York City to increase awareness of

CurePSP and to develop collaborations• Attended and exhibited at the University of Pennsylvania’s Annual Caregiver Conference for Atypical Parkinson’s Disease and

Dementia

NATIONAL CONFERENCE

One way to reach a lot of people all at one time is through a national conference . These big meetings are opportunities to meet face-to-face with many people at various stages of disease progression . It’s an opportunity for clinicians to answer many questions and also for patients and families to meet others to discuss their issues and concerns . It may also be the first time a newly diagnosed patient meets another with PSP or CBD . This can be a difficult experience, but it can also be an experience where others may provide emotional support, advice, and hope . During 2013, CurePSP:

• Held 2013 Northeastern Family Conference in Peabody, Massachusetts in April

INTERNATIONAL LEADERSHIP

The need for services and the enthusiasm of Canadian volunteers lends well to the possibility of developing a Canadian affiliate . In North America, CurePSP is the only organization focused on PSP, CBD and other atypical Parkinsonian disorders and the demand for our services in Canada is especially high to the extent that we must proceed to help build a Canadian affiliate during 2014 . CurePSP is the premier organization in the world for PSP research, but we learn a lot from our sister organizations, especially in the UK, where socialized medicine has enabled a more structured system of care for those with neurodegenerative brain diseases . During 2013, CurePSP:

• Met with movement disorder staff at Toronto Western Hospital, the Markham Center and Kingston Hospital and held a total of three meetings to recruit Canadian volunteers

• Developed 2 new face-to-face support groups in Ontario and 1 new online group led by Canadian volunteers• Promoted new Canadian support groups in coordination with the Parkinson Society Canada to assist with networking and

promotion of new support groups


GENERAL NEUROLOGISTS AND MOVEMENT DISORDER SPECIALISTS Helping medical and healthcare professionals learn about PSP, CBD and related diseases is an essential part of CurePSP’s mission . Many primary care physicians and even many general neurologists have never seen a patient with PSP let alone CBD . Lack of familiarity with our patients’ disorders may often lead to misdiagnosis or inappropriate therapies which are obviously of little help or may actually exacerbate symptoms . During 2013, CurePSP:

• Established a Physician Education Committee• Worked on developing a lecture series for residents and general neurologists• Worked on developing a video training series for physicians related to identification of symptoms in PSP/CBD/MSA• Worked on developing reference tools for neurologists for identifying common symptoms of the diseases

PHYSICAL THERAPISTS, OCCUPATIONAL THERAPISTS, AND SPEECH-LANGUAGE PATHOLOGISTS

Very often, the professional who can do the most for the PSP patient and the family are the therapists (physical, occupational, and speech-language) who do so much to improve the quality of life for people who are managing and struggling with their symptoms and their daily tasks . The therapist needs to know what these disorders are all about so they can develop plans of service to meet the needs of individual patients and their families . CurePSP and its Medical Professional Advisory Committee continually help to develop


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resources and provide opportunities for therapists to meet to discuss the needs of patients with PSP, CBD and related disorders .During 2013, CurePSP:

• Developed collaboration with Emory University in Atlanta, Georgia, and facilitated training for PT/OT/SLPs• Exhibited at the annual physical therapist conference in Salt Lake City, Utah

NURSES

Just as therapists may provide some of the most useful services to PSP patients, we often find that nurses, such as those in nursing homes and assisted living facilities, have frequent involvement with our patients . Many nurses who may be familiar with Alzheimer’s disease, Parkinson’s disease, or general dementia may not be familiar with PSP or CBD but know that there are similarities and differences . It is important that they understand these differences and be able to respond with personal care that reflects the special needs of these patients . During 2013, CurePSP:

• Participated in national nursing conferences and also supported the work of a nursing project at Johns Hopkins Medical Center, Baltimore, Maryland, which focuses on PSP

• Exhibited at the National Gerontological Nurses Association Conference in Baltimore, Maryland• Discussed internship options for nurses at the Pennsylvania Comprehensive Neuroscience Center, University of Pennsylvania,

School of Nursing• Presented at a School of Nursing event at the Johns Hopkins Hospital to increase awareness of CurePSP• Supervised Master’s degree nursing student in a clinical care setting, a web and phone-based project supported through Towson

University in Baltimore, Maryland• Maintained and supported the activities of the Medical Professional Advisory Committee

PATIENT ADVOCACY

CurePSP is actively involved in promoting our cause with the rare disease community through advocacy and governmental relations . Since we are not alone in the business of rare diseases, of which there are nearly 7,000, CurePSP works very closely with umbrella organizations such as the Parkinson’s Action Network (PAN) and the National Organization for Rare Disorders (NORD) to help promote the cause and mission of rare disease research and support for patients . CurePSP regularly attends PAN and NORD functions and learns from other rare disease organizations about what is effective . During 2013, CurePSP:

• Attended the Parkinson’s Action Network (PAN) Environmental Science and Parkinson’s Briefing in Washington, DC• Attended the annual Parkinson’s Action Network Forum in February 2013 and participated in lobbying activities on Capitol Hill

GOVERNMENTAL RELATIONS

It is essential that CurePSP make its case on Capitol Hill for more support for research through the National Institutes of Health and the National Institute of Neurological Disorders and Stroke and the National Institute on Aging . We also try to help educate governmental employees about our disorders and work with professionals at the Food and Drug Administration to help promote drug development and therapies . Over the past couple of years, CurePSP provided encouragement and leadership to the Social Security Administration and was instrumental in getting compassionate allowances for PSP, CBD, MSA and ALS/PDC . During 2013, CurePSP:

• Attended the annual NIH/NINDS Forum in Bethesda, Maryland• Participated in the release of the Social Security Compassionate Allowance video• Attended the Social Security Administration’s awards ceremony in December 2012 and received the Commissioner’s

Appreciation Award for work done on the Compassionate Allowance Program• Attended the annual NIH conference for Rare Disease Week in February 2013


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CurePSP’s Research Committee provides oversight of the Foundation’s research and training programs including their planning, operations and evaluation . Members of the Committee include an impressive and highly accomplished cohort of scientists and clinicians who have helped to make great advances in CurePSP’s research objectives over the recent years . Dr . Lawrence Golbe, the Foundation’s Director of Research and Clinical Affairs, along with the Scientific Advisory Board that he chairs, play integral roles in assisting and guiding the Research Committee . The Research Committee in turn reports to the Board of Directors to implement their recommendations .

This year has been amazingly successful with great advances being made in the CurePSP Genetics Program, in addition to several investigator-initiated grants being completed and results presented at our International Research Symposium, the accomplishments of which are described in detail in the Research Report . We also had our first Urso Summer Student Program in PSP Research recipient, Emily Beisser, present the results of her project at this meeting as well . The Research Committee continues to advocate for additional funding to fuel the Foundation’s mission and to keep up this strong momentum .

The Research Road Map developed this year by the many visionary leaders within CurePSP sets the stage for accelerating the pace of research . Several recent extremely productive research projects funded by our organization lead to the genesis of this strategic vision . We feel strongly that future efforts supported within our Research Road Map will have an even greater impact on the field and get us that much closer to finding meaningful and effective therapies . Our research program, subserved by this Road Map, consists of investigator-initiated research projects by which financial support is provided to basic scientists and clinician scientists who are exploring the frontiers of knowledge about these disorders . It also includes large collaborative research efforts addressing specific problems . In addition, the program continues to support national resources needed by the scientific community, specifically the Eloise H. Troxel Memorial Brain Bank at Mayo Clinic, Jacksonville . The Urso Summer Student Program in PSP Research works to support up to three trainees per year; efforts that will encourage our next generation of scientists and clinicians to pursue research careers focused on our disorders . A new effort started this year is the SIG program . SIGs or Special Initiative Grants are designated to support innovative and high-risk research utilizing a novel funding method .

It has been a productive year and the next year promises even greater success . The Research Committee continues to identify research priorities, recruitment of the best scientific talent to address these priorities and training of the next generation of gifted young scientists to continue these efforts .

From the Chair of the Research CommitteeYvette M. Bordelon, MD, PhD


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Dr. Michael DeTure presenting at CurePSP’s 2013 International Research Symposium.

Urso Summer Student Fellowship recipient Emily Beisser presenting at CurePSP’s 2013 International Research Symposium.

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Lawrence I. Golbe, MDDirector of Research and Clinical AffairsChair, Scientific Advisory BoardCurePSP

Director, Division of Movement DisordersRutgers Robert Wood Johnson Medical School

The past year has brought so many exciting developments in CurePSP’s research program that it’s hard to know where to start . So, as I taught my kids, let’s start with a map .

RESEARCH ROAD MAP

Over the past year, CurePSP has created and started to implement a “Research Road Map .” It lays out three short lists:

• Three broad scientific ideas about how PSP and CBD occur: genetics, prions and proteins

• Two experimental tools that we need to get the job done: models and markers

• Five practical goals: drug target identification, screening of candidate drugs, testing in animal models, small-scale human testing and large-scale testing in patients

To help you understand our thinking in offering this Road Map, consider the following:

• The Road Map is offered to researchers and funders as a general guide, not a blueprint

• It need not proceed in any specific chronological sequence• While it is generated by our Foundation, it does not

dictate scientific ideas or experimental methods, which are always best if left to the expertise and creativity of researchers themselves

• It is broad but not complete, as we recognize that valuable contributions may lie outside this specific route

• It is tentative, not immutable; at each year’s annual PSP/CBD Research Symposium, we will schedule a block of time for the assembled experts to take stock of where we are on the Road Map and, importantly, whether the route needs to be changed

The Research Road Map is laid out in greater detail later on in this report .

SPECIAL INITIATIVE GRANTS

The Research Road Map has emboldened CurePSP to undertake an ambitious new research grants program, our Special Initiative Grants (SIGs) .

Our traditional investigator-initiated grants program offers grants of up to $100,000 (increased this year from $75,000) to be spent in one or two years . Those funds must be on hand at CurePSP at the time the award decision is made and payments to the researcher’s institution are guaranteed pending satisfactory progress reports . The scientific idea is generated by the applicant without previous negotiation with CurePSP . Even the most junior researchers are eligible . The topic may or may not fit well into our Research Road Map .

The SIG program is very different . It offers amounts from $200,000 to $1 million to be spent over two to five years . The applicants must be senior, highly productive and accomplished researchers . The topics will fit well into our Research Road Map . Importantly, the funds are not on hand at the time of the decision and will be raised over time . The researcher is expected to assist directly in fundraising and CurePSP will take advantage of the reputation of the researcher in appealing to major donors . Both the traditional grants and the SIGs must pass review by CurePSP’s Scientific Advisory Board .

So far, we have awarded one SIG, to Stanley B . Prusiner, MD of the University of California San Francisco and winner of the Nobel Prize in 1997 for his discovery of prions . Dr . Prusiner will use his grant to develop a new animal model for testing treatments for PSP and CBD, a “tau rat .”

CBD SOLUTIONS COLLABORATION

Earlier this year, CurePSP was approached by CBD Solutions to help support research into that disorder . The company is a philanthropy based in Sweden and founded by Sten Mortstedt, a businessman whose wife has the disorder . So far, CurePSP

Regular Grants Special InitiativeTotal Amount Up to $100,000 $200,000 - $1milDuration 1 - 2 Years 2 - 5 YearsResearcher Seniority Any SeniorFits into Road Map Yes or No YesFunds on Hand Yes NoResearcher Fundraises No YesMust Pass SAB Review Yes Yes

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has proposed a number of projects for support . One that has now been funded is development of a CBD clinical rating scale . The co-principal investigators will be Irene Litvan, MD of the University of California San Diego and myself, with about 20 collaborative sites to be recruited . CurePSP will serve as the administrative center .

TAU CONSORTIUM COLLABORATION

Another philanthropically-supported research effort is the Tau Consortium . This group of 15 investigators is supported by the Rainwater Charitable Foundation, founded in 2009 by Texas businessman Richard Rainwater, who suffers from PSP . Its scientific director is Bruce L . Miller, MD, a behavioral neurologist at University of California San Francisco . Its goal, like that of CurePSP, is to find causes and treatments for PSP . CurePSP will provide $100,000 for Tau Consortium members to investigate the relationship to PSP of the rare tau disorder “lytico-bodig,” which occurs only on Guam and nearby islands . The principal investigator will be Michael Geschwind, MD PhD, a neuroscientist at UCSF . Lytico-bodig, fortunately but mysteriously, is dying out . If the reasons can be found, the thinking goes, perhaps the same fate can be arranged for PSP and CBD .

COMPLETED INVESTIGATOR-INITIATED PROJECTS

At our annual Research Symposium in Baltimore in November, six researchers presented the results of their CurePSP-supported work that concluded over the past year .

Gerard D . Schellenberg, PhD of the University of Pennsylvania presented preliminary results of his whole-exome sequencing analysis . Supported by the Friedman Family and Peebler Family Foundations via CurePSP, this project compared the specific DNA sequences of a number of people with PSP with those of a similar number of healthy individuals . The results could point the way to new ideas for how brain cells are damaged in all PSP, even in the majority without a family history of a similar disorder . This will point to new drug targets, consistent with the overall plan in our Research Road Map .

Ulrich Müller, MD, PhD of University Hospital Giessen and Marburg presented his epigenetic analysis of PSP . Collaborating in this project was Günter Höglinger, MD of the same institution . Epigenetics is the study of chemical alterations to the genome other than actual changes in

the code sequence itself . This may explain environmental exposures as contributing causes of PSP and provides new drug targets as part of our Road Map .

T . Chris Gamblin, PhD (pictured above) of the University of Kansas presented work creating multiple tau defects in the same experimental model at the same time, shedding light on the question of just which of the multiple tau protein abnormalities present in PSP and CBD are responsible for the damage . He used two common lab models, the roundworm C . elegans and the mold Aspergillus . This reinforces the “proteins” area of the Road Map .

Jose A . Santiago, a graduate student at Rosalind Franklin University of Medicine and Science in Philadelphia, presented work performed with mentor Judith Potashkin, PhD . They found that certain variants of RNA in blood can distinguish PSP from healthy persons, those with Parkinson’s disease and those with multiple system atrophy . This will allow us to more effectively screen subjects for clinical trials, permitting those projects to proceed more quickly and less expensively . This fits well into the “markers” rubric of our Road Map .

Michael DeTure, PhD of the Mayo Clinic presented his discovery that a set of proteins involved in disposal of abnormal proteins called “heat-shock proteins” (HSPs) hold onto abnormal (or overabundant) tau protein and that this combination induces formation of antibodies that may be found useful as treatment . His initial attempts to treat a “tau mouse” with such antibodies are promising but not yet conclusive .

Jose Abisambra, PhD, of the University of Kentucky, but at the University of South Florida when he did the work, presented evidence that abnormalities in tau of the sort found in PSP and CBD interfere with the “unfolded protein response .” The

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UFR is an important mechanism in brain cells for disposing of defective proteins . Importantly, one of the genes found to be closely related to PSP in our 2011 “whole-genome analysis” was PERK, a protein that is part of the UFR . This also fits well with our Road Map and opens the door to new drug targets .

A GREAT NEW IDEA, A WORD OF CAUTION AND THE PATH FORWARD

The newest widely-accepted scientific thinking on PSP posits that it and many of the other neurodegenerative diseases, including Alzheimer’s and Parkinson’s, all rely on a “prion-like” or “protein templating” mechanism . That’s where a normal brain protein misfolds for whatever reason and then induces its normally folded brothers to similarly misfold . The misfolded protein molecules then aggregate into toxic clumps, killing the cell and releasing its contents to be taken up by neighboring cells, producing a chain reaction that spreads through the brain . If the better-funded research into Alzheimer’s or Parkinson’s finds a key in that pathway, it may fit the locks on PSP and CBD as well . But if it does not (and things are rarely so simple) we’re still on our own . That’s why our research program must stay strong and why we have to invest in both the road-tested senior researchers and in their successors, the young investigators who need just that one grant to launch a career and to create a new, robust dedication to curing PSP and CBD .

For the last quarter of 2013, we have received eight grant applications, mostly from young investigators . That’s an excellent crop, but our budget will only allow us to fund two or three . With your help, we can keep more researchers involved in PSP and CBD and prevent them from fleeing to areas with better chances of funding . The scientific ideas are very exciting right now and our organization, with your help, is well-positioned to turn them into a cure .

RESEARCH ROAD MAP

A NEW DIRECTION

CurePSP recognizes that the fight against PSP can now take a new direction, capitalizing on recent discoveries, exciting ongoing work and new hypotheses . We propose to fund research initiatives in five areas that in many cases can proceed in parallel . They can take place in academia or industry and would be awarded via a competitive, peer-reviewed process . These areas are genes, prions, proteins, models and markers . The first three are new hypotheses . The last two are new experimental tools .

GENES

PSP research can now follow up on the discoveries of our CurePSP Genetics Consortium, funded by our Charles D. Peebler, Jr. PSP Genetics Program . In 2011, the Consortium published the results of its genome-wide association study (GWAS), which used “snip chips” to find genes not previously known to be associated with PSP . It found three such genes with very strong associations - STX6, EIF2AK3 and MOBP . It also confirmed the known association of one variant in MAPT (the tau gene) and found a second, new tau gene variant . This work has now been extended, via funding from the Morton and Marcine Friedman Foundation, using a technique called whole-exome sequencing analysis . Already, exome sequencing has identified additional PSP-associated genes that escaped the earlier analysis .

Plan: Once the whole-exome sequencing study is completed in the first half of 2014, CurePSP will move on to supporting biochemical studies of these genes to determine their normal function and to understand how the PSP-associated mutations damage brain cells .

Cost: We will study the 10-15 most promising genes at $200,000 per gene or $2 million to $3 million .

PRIONS

Work in the 1970s showed that the rapidly progressive neurodegenerative disorder Creutzfeldt-Jakob disease and a few other diseases were caused by a newly discovered brain protein that assumed an abnormal folding pattern that was toxic to the cells . This misfolded “prion protein” then induced nearby normal prion protein molecules to similarly misfold, creating a chain reaction that spread throughout the brain . Evidence over the past five years now suggests that a Dr. Richard Zyne with neurologist and researcher Dr. John Steele.

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similar mechanism, albeit operating more slowly and affecting different proteins, is at work in other neurodegenerative diseases, possibly including PSP .

Plan: Results of ongoing research on treatment of prion disorders can be used to jumpstart a similar effort focused on PSP . Initial steps along this research pathway include creation of cell culture and animal models of PSP that show characteristics of prion disorders such as cell-to-cell spreading

of misfolded protein . Subsequent steps will focus on identifying drugs or other interventions that interfere with the misfolding/propagation process .

Cost: Initial model development to test the prion disorder mechanism using five cell culture/animal models at $200,000 to $1 million; followed by characterization of 10-15 treatment targets at $200,000 per target or $2 million to $3 million .

PROTEINS Defects in how brain cells handle various proteins, even in the absence of a prion-like mechanism, may be central to the development of PSP and the other neurodegenerative diseases . Examples of such defects in protein handling are

overproduction, inappropriate folding, and deficiencies in disposal of worn, defective or excess proteins . Many potential drug targets exist in the complicated protein handling systems in brain cells . Plan: Identify treatment targets by studying protein handling mechanisms in PSP . As part of this, we will use new PSP-specific laboratory models (see below) to evaluate drugs modifying protein production and turnover that are already

under development for other disorders such as cystic fibrosis .

Cost: 10-15 targets at $200,000 per target or $2 million .

MODELS

Once drug candidates are identified, they must be tested in animal models . There are several laboratory models for PSP, ranging from brain cells in a “dish” to roundworms, fruit flies, zebrafish and mice . Most of these models rely on insertion of a tau gene with a single mutation that occurs in some people with a different tau disorder . We need models that are created by methods similar to what is causing human PSP itself . The new genetic discoveries mentioned above, and advanced in preparing patient-derived stem cells may provide that opportunity .

Plan: Create models in established lab animals and in cell lines using genes,

alone or in combination, that have been found to be associated with PSP . Alternatively, make induced pluripotent stem cell lines (iPS) directly from patient samples with specific defined mutations associated with PSP . Then characterize these models to determine their utility in evaluating treatments .

Cost: Five models at various phylogenetic levels at $200,000 per model or $1 million .

MARKERS Drug candidates that prove safe and effective in animal models are then tested in humans . These would be drugs to slow or stop progression, not merely to blunt symptoms . Initiating

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Genes:Work out the cellular pathways controlled by the top 10 genes from CurePSP’s 2011 WGA and 2013 WESPrions:Work out steps in tau misfolding, templating, and cell-to-cell transmissionProteins:Identify defects in production, handling and disposal of tau and other proteins in PSP/CBD

Models:Create new cellular and animal lab models more faithful to human PSP/CBD

Markers:Devise better tests for diagnosis and measurement of PSP/CBD and its treatment response in humans

New Experimental Tools

Scientific Hypotheses

Procedures

Identify top 30-50 drug targets

Perform high-throughput screens in molecular or cellular models

Test drugs in vertebrate preclinical models

Early-phase clinical trials by industry or academia

Late-phase clinical trials by industry

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treatment at the earliest possible stage of the disease would be most effective . Furthermore, we need more sensitive ways of measuring effectiveness of such drugs in PSP in order to minimize the necessary size, duration, and therefore cost of drug trials .

Plan: To find one effective principal outcome measure for neuroprotection trials in PSP, we will have to evaluate multiple ideas . These may be tests of tau or other proteins in the spinal fluid or blood, imaging procedures using novel MRI techniques or radiotracer ligands, physiological test batteries, or ideas yet to be developed .

Cost: Evaluate ten candidate markers at $500,000 per marker or $5 million .

COST AND TIME

It is estimated that the cost to complete the projects in the Research Road Map will be $13 million to $15 million over a seven-year period .

RESULTS

We expect that of the 30-45 drug (or other treatment technique) candidates tested, perhaps five or six will demonstrate potential value for commercial development . At that point, the pharmaceutical industry may take over or partner with academia, using its resources for the very expensive Phase 2 and 3 clinical trials .

CurePSP consistently looks for opportunities to achieve its vision of a world free of PSP, CBD and related brain diseases through good science, technology and human talent . We are on the verge of significant breakthroughs that will not only clarify the causes of these devastating illnesses but identify much-needed treatments that will, in the end, cure PSP, CBD and other atypical Parkinsonian disorders . The timing has never been better to unlock the mysteries of neurodegenerative brain disease .

CUREPSP GENETICS PROGRAM AND THE WHOLE EXOME SEQUENCING PROJECT

Jeffrey S. Friedman, MD, PhD

MemberCurePSP Board of Directors

Adjunct ProfessorThe Scripps Research Institute

FounderFriedman Bioventure

In 2011, using a method called ‘genome-wide association’, or GWAS for short, a large group of cooperating researchers supported by CurePSP identified 4 genes that increase the risk of getting PSP . However, these new genes do not guarantee that a carrier will develop the disease . The genes identified in this landmark study were:

MAPT — This is the gene the makes (encodes) the tau protein . Mutations in tau had previously been shown to increase the risk of PSP and the related disorder corticobasal degeneration (CBD) . Accumulation of tau (aggregates) in specific brain regions is a constant feature of PSP, and it is believed that the process of tau aggregation represents an important aspect of disease progression . The GWAS study confirmed prior work linking tau to PSP, and identified new variants of tau associated with PSP .

EIF2AK3 — Cells can be thought of as little factories for making all of the proteins that are encoded by our genes . Like any factory, there has to be a system for monitoring the quality of the products being made, and EIF2AK3 is a part of the cellular quality control system . When EIF2AK3 senses misfolded proteins (a quality control problem), it puts the brakes on protein production .

MOBP — This gene encodes a protein component of myelin, the insulating sheath that covers the axons (wires) of nerve cells in the central nervous system .

STX6 — This gene encodes a protein involved in regulating the transport of proteins from the site of synthesis (endoplasmic reticulum) to various locations in or outside of cells .

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VARIANTS IN THESE FOUR GENES AND THEIR POSSIBLE LINK TO PSP

We have some general ideas of how alteration in the activity of these genes might contribute to development of PSP . Plus, our reporting of these genes 2 years ago has stimulated a new wave of research to help directly answer this question! For MAPT (the gene encoding the tau protein), EIF2AK3 and possibly STX6, we think that the PSP-associated variants affect production or removal of the tau protein . In fact, the process of protein homeostasis (that is, the balance between production and removal of a protein from cells) seems to go awry in several neurodegenerative diseases — PSP, CBD, Huntington’s, Alzheimer’s and Parkinson’s being examples . Several research groups are actively working on drugs that attempt to modulate protein homeostasis, and some of these efforts are targeting turnover of the tau protein . As noted earlier in this report, CurePSP’s Research Road Map includes funding for biochemical studies to understand functional differences between ‘normal’ and disease-associated variants of these genes, and more generally to study the role of alterations in protein homeostasis as a risk factor for PSP .

ADDITIONAL GENES INVOLVED IN PSP THAT HAVEN’T YET BEEN IDENTIFIED

The 4 genes identified in the GWAS study represent the

most common variants that increase risk of getting PSP . However, only a small percentage of all individuals with PSP, perhaps 5-10% of cases, actually carry one of these variants . This strongly suggests that there are many additional gene mutations, present at a low frequency in the population, that contribute to development of PSP . CurePSP, recognizing that additional research was needed to better define the genetics of PSP, started the ‘exome’ sequencing study using the infrastructure and team that was already in place for the GWAS study .

THE ‘EXOME’ STUDY, AND PSP GENETICS

When the GWAS study began in 2008, it was the best technology available for discovery of disease-associated genes . The first human genome sequence had been completed in 2003 at a total cost of about $3 billion, representing an effort that took more than 10 years . Today, we can sequence a genome in a few days for about $10,000 and we are still improving speed and reducing cost . Technology has radically changed, making direct sequencing of individual patient samples both technically feasible and achievable with a relatively modest budget .

The exome study takes advantage of the reduction in cost and improvement in sequencing speed and capacity to look directly at the DNA sequence of those regions of the genome

Researcher Naomi Kouri presenting at CurePSP’s 2013 International Research Symposium.

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that are responsible for making proteins . The name ‘exome’ comes from ‘exon,’ the term for any gene region which codes for a protein . This represents comprehensive sequencing of approximately 3% of the human genome . Importantly, this 3% of the genome is believed to contain upwards of 95% of disease-causing mutations — making exome analysis a cost-effective tool to identify the vast majority of disease- associated changes in DNA .

CurePSP has funded the Genetics Consortium, under the direction of Dr . Gerard Schellenberg of the University of Pennsylvania, to sequence exomes from 750 autopsy-verified cases of PSP . As of September 2013, the sequencing work itself was essentially complete, and the first stages of analysis of the data began . Already, a preliminary analysis of the first 270 samples has found at least 2 new genes with mutations associated with PSP . Importantly, mutations in these genes have been found in more than one PSP sample, strengthening the association with disease . While this analysis is preliminary, it gives CurePSP confidence that results from the exome study will expand the number of genes associated with development of PSP . Importantly, the exome results are more informative than the results of the GWAS study in that they identify the specific mutation in a particular individual that is disease-associated . While we currently have 4 genes associated with PSP from the GWAS study, at the conclusion of the exome study, we expect to identify an additional 20 or 30 new genes with PSP-associated mutations .

RESULTS FROM THE EXOME STUDY

A preliminary analysis of data from the exome study was presented at the CurePSP Research Symposium in November, 2013 . We expect analysis of the data to be completed by mid-2014, with results to be published shortly thereafter .

Every gene and mutation we identify as being associated with development of PSP brings us a step closer to understanding the cause or causes of this disease, and provides us with a potential target for development of drugs to slow, stop or prevent the disease . Over the past year, CurePSP has worked on a long-term research plan that we call the Research Road Map . The Road Map is an ambitious, multi-year, modular program with multiple components, but is primarily focused upon moving advances in basic science toward the development of drugs to treat PSP patients .

Stages on the path to an effective drug include:

• Identification of relevant genes • Understanding how specific mutations in those genes alter

their function• Screening for new drugs that correct or complement the

altered gene function• Development of early diagnostic tests to identify patients

who will go on to develop PSP • Creation of animal models to facilitate early stages of drug

testing • Supporting early stages of human testing of promising

drugs

The first stage of the Research Road Map is the identification of genes and specific mutations that are associated with PSP — this will be the output of the exome study, plus the 4 genes previously identified in the GWAS study . Because we expect multiple new genes to be identified, one of the initial tasks of the research community will be to prioritize which genes to work on first! Of course, we want to choose those targets that will include the largest number of patients with PSP . The Road Map envisions supporting work on the top 10-15 most promising target genes, depending upon availability of funds .

We also expect that an analysis of the functions of genes identified in the exome study will help us refine our ideas about the validity of a prion-like hypothesis, a protein turnover hypothesis, or lead to generation of new hypotheses to explain what causes PSP . Identification of new genes involved in development of PSP will also lead directly to the creation of new animal models of the disease, and will facilitate the development of new diagnostic tests for PSP —both of which are integral parts of the CurePSP Research Road Map .

These are very exciting times at CurePSP . We are fortunate to have some of the best scientists in the world working on PSP genetics . Over the next year, the results from ongoing genetic studies will, for the first time, identify the targets we must pursue in order to develop drugs to alter the course of PSP . While the funding requirements for these studies is significant and represents a quantum leap for CurePSP, the quality of the scientific foundation upon which the Road Map is built is among the best in the world . We believe that the strategy is sound and the path ahead is clear . The speed at which we traverse this path for patients and families affected with PSP is dependent upon our ability to procure and wisely distribute the funds required to move along the Research Road Map .

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PRUSINER TRANSGENIC RAT MODEL

CurePSP is honored to recognize Nobel Laureate and National Academy of Sciences member Stanley B . Prusiner, MD as its first grant recipient under CurePSP’s new Research Road Map initiative . Dr . Prusiner is Director of the Institute for Neurodegenerative Diseases (IND) and Professor of Neurology at the University of California

San Francisco (UCSF) . He received his undergraduate and medical school training at the University of Pennsylvania and his postgraduate clinical training at UCSF . Dr . Prusiner’s contributions to scientific research have been internationally recognized and he was awarded the Nobel Prize in Medicine in 1997 .

During 2013, CurePSP provided funding for Dr . Prusiner’s proposal entitled, Transgenic Rats Expressing Wild-Type and Mutant Human Tau . Dr . Prusiner and his researchers at IND intend to develop a transgenic rat with the human tau protein and a luciferase reporter . An animal model of this type is critical to the advancement of our understanding and treatment of prion disorders . Dr . Prusiner and his team at IND have successfully created a similar animal model in mice, however, rats would be far superior to mice as animal models . Compared to mice, the frontal lobes of rat brains more closely resemble those of humans .

A rat model of this type will enable more effective testing for drugs and early detection for prion-like tau-related diseases which include progressive supranuclear palsy (PSP), corticobasal degeneration (CBD), frontotemporal dementias (FTDs) and Alzheimer’s disease . Altogether, these diseases affect an estimated population of 5 .5 to 6 million people in North America . Chronic traumatic encephalopathy (CTE), another tau-related disease affecting many military personnel and athletes who have suffered high impact/traumatic brain injuries, may also benefit from this research initiative .

ELOISE H. TROXEL MEMORIAL BRAIN BANK In 1998, CurePSP established a centralized PSP/CBD brain bank—the Eloise H. Troxel Memorial Brain Bank at the Mayo Clinic in Jacksonville, Florida—the largest in the world for

this purpose and a resource for PSP and CBD investigators worldwide . The Brain Bank is under the direction of Dennis W . Dickson, MD, world-renowned neuropathologist and a member of CurePSP’s Scientific Advisory Board . The Brain Bank accepts donations of brains from patients with known or suspected PSP, CBD or MSA . An autopsy report establishing an authoritative diagnosis is provided to the family generally in several months . CurePSP covers the cost of brain tissue removal and shipping for families who are unable to afford that expense . The Brain Bank maintains a collection of brain samples both frozen (for chemical analysis) and formalin-fixed (for anatomical analysis) that it makes available to researchers worldwide at no cost .

The Brain Bank also provides diagnostic confirmation for patients enrolled in research programs funded by CurePSP where such information is part of the research results .

2013 INTERNATIONAL RESEARCH SYMPOSIUM CurePSP’s annual International Research Symposium was held in Baltimore on November 23 . More than 40 attendees were able to compare ideas, form collaborations and receive inspiration and motivation .

In addition to presentations by the six CurePSP-funded investigators mentioned previously in Dr . Golbe’s report, three invited speakers who are world leaders in their fields discussed their work . The featured keynote speaker was Kurt R . Brunden, PhD of the University of Pennsylvania, who discussed his work in developing a new class of drug for PSP and other tau-based brain disorders . Dennis W . Dickson, MD of the Mayo Clinic Jacksonville and Director of CurePSP’s Eloise

Brain Bank Director Dr. Dennis Dickson at CurePSP’s 2013 International Research Symposium.

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H. Troxel Memorial Brain Bank, updated the audience on the many projects that the brain tissue and DNA samples from the Brain Bank have made possible .

Three presentations of research were submitted for presentation as posters . One was presented by Christof Wiessner, PhD, a scientist at Asceneuron, a Swiss biotech firm that is developing inhibitors of an enzyme called “O-linked β-N-acetylglucosaminidase” or “O-GlcNAcase,” which breaks down a chemical that enhances abnormal tau activity and may enter human trials in 2015 . Another poster, presented

by Emily B . Beisser, an undergraduate at Bucknell University, discussed the first validated PSP Staging System, developed with Lawrence I . Golbe, MD of Rutgers Robert Wood Johnson Medical School . The third poster, presented by Naomi Kouri, a graduate student at Mayo Clinic Jacksonville who collaborated with Garret Wilson, Gerard Schellenberg and Dennis Dickson, demonstrated how a new digital microscopic image analyzer can be used to quantify tau abnormalities in brain tissue that can then be correlated with genetic analysis in the same individuals .

PRESENTATION TITLE PRESENTEREpigenetic Analysis in PSP Ulrich Müller, MD, PhD

University Hospital Giessen and MarburgUpdate on PSP/CBD Genetics and Preliminary Results of a Whole-Exome Analysis

Gerard D . Schellenberg, PhD University of Pennsylvania

Models to Determine the Toxicity of Tau Aggregates T . Chris Gamblin, PhD University of Kansas

Risk Markers for PSP José Santiago, MS (for Judith Potashkin, PhD) Rosalind Franklin University of Medicine and Science

HSP-Peptide Complexes Mediate Immunization in a Mouse Model of Tauopathy

Michael DeTure, PhDMayo Clinic, Jacksonville

Mechanisms of Tau and ER Stress for Novel PSP and CBD Therapeutics José F . Abisambra, PhD University of Kentucky

Development of New Microtubule Stabilizers for Tauopathies Kurt R . Brunden, PhDUniversity of Pennsylvania

Brain Bank Research Update Dennis Dickson, MD Mayo Clinic, Jacksonville

Interactive CurePSP Research Road Map Workshop: Are We on the Right Track?

Lawrence I . Golbe, MD Rutgers Robert Wood Johnson Medical School

2013 International Research Symposium attendees.

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Splice Variant Markers for PSP (507)

Judith Potashkin, PhDRosalind Franklin University of Medicine and SciencePhiladelphia, Pennsylvania

Type of Project: (1) Tau: Genetics, Biochemistry and Treatment Target

Differential diagnosis of progressive supranuclear palsy (PSP) from Parkinson’s disease (PD) relies mostly on clinical observation and the patient’s response to drug treatment . Unfortunately, this often leads to a high rate of misdiagnosis early in the disease . Therefore, it would be useful to identify markers that could be used to diagnose PSP patients, since early detection could improve disease management . Currently, there are very few biomarkers for PSP available and none have been fully validated . The ideal biomarkers would come from a readily obtained source such as blood and should show a high degree of accuracy . We have recently identified 6 risk markers in blood that accurately distinguishes PD from atypical Parkinsonian disorders (APD), including PSP and multiple system atrophy (MSA) patients .

In this study, we will evaluate the potential of the 6 risk markers for improving the diagnosis of PSP patients . In addition, we will determine whether additional candidate markers identified in a previous study may be useful for the diagnosis of PSP . These studies are expected to substantially increase the number of PSP markers currently available . Biomarkers identified in this study could help identify individuals at risk for developing PSP and for monitoring disease progression and treatment .

2013 Funded Research Grants

PSP Whole Exome Study - Phase Two (508)

Gerard Schellenberg, PhDUniversity of Pennsylvania, School of MedicinePhiladelphia, Pennsylvania


We are attempting to completely understand the role of inheritance in Progressive Supranuclear Palsy (PSP) . The reason for pursuing additional genetic factors is that genes which cause or increase susceptibility to PSP will provide insight into the basic biochemical processes that cause the disease . The better we understand these processes, the greater the chance of designing therapies to treat or prevent PSP . The work we are performing is called whole exome sequencing, an approach that is designed to detect rare inheritable genetic changes that cause or increase susceptibility to developing PSP .

Whole exome sequencing - In our experiment, we are determining the DNA sequence of all the exomes for all genes in the human genome for subjects with PSP . This is called whole exome sequencing . Our project is to perform whole exome sequencing on 750 – 1,000 subjects with autopsy-documented PSP .

PSP genetics - Presently, we know of two different types of inherited changes that contribute to PSP . The first is a genetic variation which increases an individual’s susceptibility to developing PSP . With this variation, other factors, such as environmental factors, must be present for the person to develop PSP . The second type is another genetic variation consisting of rare mutations that cause PSP . If a person inherits this mutation, they will definitely get PSP in mid-to-late life .

Progress - Our goal is to perform whole exome sequencing on 750 – 1,000 subjects with PSP and to compare these sequences to data from 1,000 subjects without neurological disease . We performed pilot work to maximize sequencing efficiency followed by a production phase .

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Tau Dimerization: A Mechanism of Tau Function and Dysfunction? (509)

Stuart Feinstein, PhDUniversity of California, Santa BarbaraSanta Barbara, California


The protein tau is critical for the maintenance of the nervous system . It is also a key contributor to many neurodegenerative diseases, including PSP and CBD . While we understand many functions performed by tau, our understanding of how it actually performs these functions remains primitive . One half of tau (the “C-terminal half”) can associate with microtubules and regulate their essential behaviors . However, the ability of this region of tau to perform these critical functions drops dramatically without the other half of the protein (the “N-terminal half”), which lacks the ability to bind and regulate microtubules . Unfortunately, the mechanism(s) by which the N-terminal half exerts its potent influence(s) upon the C-terminal region remain completely enigmatic .

The relevance of normal tau action to pathological tau action in PSP and CBD is that a fragment of tau, derived from the N-terminal half of the protein, has been shown to accumulate in PSP and CBD affected brains and to be neurotoxic, but the mechanism of its neurotoxicity is unknown . However, we and our collaborators have recently reported biochemical and biophysical evidence indicating that the N-terminal region of tau promotes dimerization and that this dimerization is necessary for normal tau function . This leads to the hypothesis that tau mediated neuronal cell death and dementia in PSP and CBD is mediated by N-terminal tau fragment mediated inhibition of normal tau action, leading to a “loss-of-function” effect resulting in neuronal cell death and dementia . Our work supporting this dimerization hypothesis is based presently solely on in-vitro data . The goal of this proposal is to test the hypothesis that tau dimerization mediated by the N-terminal region occurs in neurons and is required for tau action in neurons . By better understanding normal tau action, we will gain completely novel insights into pathological tau action in PSP and CBD .


A Review and Proposal for New Research Studies on Guam and in Umatac Village, 2013: Assessment of Guam ALS/PDC in 2013-2014 (510)

Michael D . Geschwind, PhDUniversity of California, San FranciscoSan Francisco, California

John C . Steele, MDGuam Memorial HospitalTamuning, Guam

Type of Project: (5) Toxins and Epidemiology

The ALS/ Parkinsonism-dementia complex (ALS/PDC) is a neurodegeneration of indigenous Chamorros of the Mariana islands which has caused illness in successive generations offamilies for more than 2 centuries . Since the mid-1950s,the ALS/PDC incidence has declined by its phenotypes (ALS first, then parkinsonism, and now dementia), and the age of onset has steadily increased .

We will assess the current epidemiology and genetic susceptibility to this disease in Umatac, a small village of approximately 887 residents in southern Guam, and the epicenter of ALS/PDC . We will begin to assess the cellular and genetic underpinnings, as well as possible transmissibility by examining whether disruption of autophagy and related protein degradation pathways are responsible for this enigmatic disorder .

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Transgenic Rats Expressing Wild-Type and Mutant Human Tau (511)

Stanley Prusiner, MDUniversity of California, San FranciscoSan Francisco, California


The primary tauopathies, often referred to as the frontotemporal dementias (FTDs), include Pick’s disease,progressive supranuclear palsy (PSP) and corticobasal degeneration (CBD) . The tau protein plays a central role in the pathogenesis of these neurodegenerative disorders . Tau aggregates can accumulate alone, as in the primary tauopathies, or in concert with other peptide aggregates in other diseases . Tau aggregates have been found with amyloid β (Aβ) in Alzheimer’s disease (AD) and also with PrPSc in familial Creutzfeldt-Jakob disease (CJD) caused by an insertion mutation and in Gerstmann-Sträussler-Scheinker (GSS) caused by an amino acid substitution . After the centrality of tau was established in Pick’s disease, PSP and CBD, a series of incisive studies demonstrated that tau proteins can undergo transformation into prions that are self-propagating . Tau prions spread throughout the brain, analogous to PrPSc and Aβ prions that cause CJD and AD, respectively . The ability to quantify tau prions offers new paradigms for early diagnosis and effective therapeutic intervention in all of the tauopathies . We propose to develop bigenic (double transgenic) rats that express either wild-type or mutant human tau along with luciferase under control of the glial fibrillary acidic protein gene regulator or promoter . These bigenic rats will provide a second genetically-engineered species (mice being the first) to assess the efficacy of lead compounds that are being developed as therapeutic interventions for the tauopathies . In addition, the bigenic rats will provide a much improved tauopathy model for assessing the efficacy of PET ligands for neuroimaging; such ligands (reporters) are crucial for the early diagnosis of the tauopathies so as to initiate treatment prior to the occurrence of irreversible brain damage .


Mechanisms and Therapy for Tauopathy based on RNA Binding Proteins (512)

Benjamin Wolozin, MD, PhDBoston University School of MedicineBoston, Massachusetts

Type of Project: (6) Clinical and Laboratory Treatment-Oriented Research

The accumulation of misfolded tau characterizes the pathology of progressive supranuclear palsy (PSP) and many other dementias . We have discovered that a protein, termed TIA-1 (a RNA binding protein), can induce tau misfolding upon co-expression with tau . RNA binding proteins, such as TIA-1, form a particular type of RNA-protein complex, termed the stress granules (SG) . SGs identify a novel type of pathology that we have shown is prevalent throughout the brains of people with dementias involving the tau protein . This type of “molecularpathology” has not been studied previously, yet could be vitally important for understanding the causes of dementia .

Development of therapeutics for PSP has been stymied by an inability to rapidly and efficiently induce aggregation of full length tau protein (other than expressing it in transgenic mice and waiting many months) . We have discovered that TIA-1 rapidly and efficiently induces misfolding of tau, in a manner that appears to be similar to that seen in PSP . This discovery potentially represents a profound advance for the field .In this proposal, we will screen for chemicals that can inhibit the formation of misfolded tau, such as occurs in PSP, and characterize the effects of TIA-1 on tau aggregation in the brain, using a mouse model of tau-mediated disease . We will then work with the best compounds to identify the compounds that appear to have characteristics most amenable to becoming a potential treatment for PSP .

This work has two objectives: 1) characterize the process of TIA-1 induced tau misfolding, and 2) develop novel compounds able to prevent and/or reverse tau misfolding . Future work willexamine the efficacy in-vivo using mouse models of tau pathology . We anticipate that development of tau-misfolding inhibitory compounds could become or lead to novel therapies for PSP and possibly provide a long sought treatment .

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Urso Student Fellowship - A Clinical Staging System for Progressive Supranuclear Palsy (513)

Emily BeisserBucknell UniversityLewisburg, Pennsylvania

Lawrence Golbe, MDRutgers Robert Wood Johnson Medical SchoolNew Brunswick, New Jersey


The only widely-used clinical rating scale for PSP was published in 2007 by Lawrence Golbe, MD and Pamela Ohman-Strickland, PhD [Golbe and Ohman-Strickland 2007] and comprises 28 items and requires about 10 minutes to administer . However, this scale requires specialized training in neurology and even 10 minutes can be a burden in some clinical settings . A staging system requiring minimal training and only a few seconds to apply would be useful in communicating a distribution of disease severity in a group of patients or in providing a rough indication of degree of disability in an individual patient .

The Hoehn-Yahr Scale for Parkinson’s disease can meet the need of a rapid staging system, as its results can be assessed in a few seconds, usually by history alone . Despite some drawbacks, it has achieved universal use and will serve as a rough model for the present PSP Staging System .

Using the large amount of PSP patient data amassed by Dr . Golbe via use of the PSP Rating Scale (PSPRS), this work will attempt to fit the observed PSPRS progression to a 10-point scale, which has face validity as a reflection of the progression of gait and balance disability in PSP . The scale considers only gait/balance and swallowing difficulty, the two most important aspects of PSP with regard to morbidity and mortality .


The Eloise H. Troxel Memorial Brain Bank (514)

Dennis Dickson, MDMayo ClinicJacksonville, Florida

Type of Project: (8) Brain Bank

In the approximately six-month period from January 2013 to June 12, 2013, the Eloise H. Troxel Memorial Brain Bank received 46 brains, including 25 men and 21 women (all were Caucasian) with clinical diagnoses of PSP (n=31), CBD (n=11) and MSA (n=4) . The average age at death was 74 ± 8 years . The brains came from 23 different states . There were three or more from Illinois (n=4), Texas (n=3), California (n=3), Minnesota (n=3), Pennsylvania (n=3), Florida (n=3), and Georgia (n=3) . All cases came with frozen tissue as well as fixed tissue . A diagnostic report was issued in 10 ± 3 weeks, and a copy was sent to the next-of-kin along with a cover letter describing the findings in lay language . The major pathologic diagnoses were PSP (77%), CBD (9%), and MSA (6%) . Uncommon diagnoses included 1 case of vascular PSP, a rare case of multisystem tauopathy and a patient with idiopathic olivopontocerebellar degeneration . The latter was thought to have MSA-C clinically, but the pathologic findings did not support this diagnosis .

The tissue continues to be used for research . In a recently published study, we compared cases of PSP with a family history of Parkinsonism or PSP to patients with no family history (Fujioka et al ., 2013) . We had genetic data on a subset of cases through the CurePSP GWAS and were able to compare genetic differences between the two groups . None were found . We have also reported on an unusual presentation of CBD that is some cases is clinically mistaken for PSP (Kouri et al . 2013) . Samples have been provided to Dr . Nilufer Taner at the Mayo Clinic for her recently CurePSP-funded research on gene expression using RNA-seq to correlate with pathologic endophenotypes . We have recently received a request from Dr . Schellenberg for over 200 PSP samples for ongoing genetic studies . This request will be fulfilled in the ensuing weeks .

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UNIVERSITY OR GENERAL AREA OF PSP NO . GRANT TITLE RESEARCHER INSTITUTION YEAR AMOUNT AND CBD RESEARCH

001 Presence and Amount of Glycation & Massimo Tabaton, MD University of Genoa, 1997 $ 10,000 (2) Non-Tau Based Pathologies: Oxidation Markers in PSP Department of Mitochondrial, Radicals, Cell Neurosciences Death Genoa, Italy

002 Genetics and Environmental Studies John C . Steele, MD Guam Memorial 1997 $ 10,000 (5) Toxins and Epidemiology of Bodig and Lytico in Villages of Hospital (7) Clinical, Non-Treatment- Southern Guam Tamuning, Guam Oriented Research

003 Ferritin is Associated with the Aberrant Jesus Avila, PhD Centro De Biologica 1997 $ 9,700 (1) Tau: Genetics, Biochemistry Tau Filaments Present in PSP Molecular and Treatment Target (b . Madrid, Spain Biochemistry of tau and

tangles)

005 Linkage Analysis in Familial PSP (To Justo Garcia de Universidad 1997 $ 10,000 (3) Non-Tau Based Genetic Perform Linkage Analysis in a Large Yebenes, MD, PhD Autonoma de Madrid Studies Spanish Family with PSP) Madrid, Spain

006 Reaction Time and Acoustic Startle in Josep Valls-Sole, MD Hospital Clinic 1997 $ 10,000 (7) Clinical, Non-Treatment- Patients with PSP, Multi-System Atrophy, Barcelona, Spain Oriented Research and Parkinson’s Disease

007 The History of PSP Adolfo Brusa, MD Corso A . Saffi 1997 $ 1,200 (5) Toxins and Epidemiology Genoa, Italy (7) Clinical, Non-Treatment- Oriented Research

008 Haplotype Relative Risk Analysis in Lawrence I . Golbe, MD, UMDNJ Robert Wood 1997 $ 20,000 (1) Tau: Genetics, Biochemistry PSP A . M . Lazzarini, PhD Johnson Medical and Treatment Target (a . The School tau gene) New Brunswick, NJ

101 Establishment of a PSP Brain Bank Dennis Dickson, MD Mayo Clinic 1998 $ 20,000 (8) Brain Bank Jacksonville, FL

102 Trial of Donepezil HCL in PSP Patients Irene Litvan, MD Henry M . Jackson 1998 $ 29,200 (6) Clinical and Laboratory Foundation Treatment-Oriented Research Bethesda, MD

103 Potential Role of Mitochondrial Defects M . Flint Beal, MD Cornell University, 1998 $ 20,000 (2) Non-Tau Based Pathologies: in PSP Weill Medical College Mitochondrial, Radicals, Cell New York, NY Death

104 Molecular Studies of the Tau Gene in William G . Johnson, MD UMDNJ Robert Wood 1998 $ 20,000 (1) Tau: Genetics, Biochemistry PSP Lawrence I . Golbe, MD Johnson Medical and Treatment Target (a . The School tau gene) New Brunswick, NJ

105 Oxidative Mechanisms in PSP David S . Albers, PhD Massachusetts 1998 $ 20,000 (2) Non-Tau Based Pathologies: General Hospital, Mitochondrial, Radicals, Cell Neurology Dept . Death Boston, MA

106 Tau Gene Mutations in PSP Joseph J . Higgins, MD Laboratory of Clinical 1998 $ 20,000 (1) Tau: Genetics, Biochemistry Neurogenetics, and Treatment Target (a . The Wadsworth Center tau gene) Albany, NY

107 Mitochondria in PSP Russell Swerdlow, MD University of Virginia, 1998 $ 17,000 (2) Non-Tau Based Pathologies: School of Medicine Mitochondrial, Radicals, Cell Charlottesville, VA Death (3) Non-Tau Based Genetic Studies

108 Neuroanatomical Basis for PSP Eyelid Mark S . LeDoux, MD, University of 1998 $ 20,000 (4) Anatomic and Motor Dysfunction PhD Tennessee Health Histopathological Surveys Science Center Memphis, TN

109 Neuropathological Grading Scale for Mark W . Becher, MD University of New 1998 $ 10,000 4) Anatomic and PSP Mexico, Health Histopathological Surveys Sciences Center Albuquerque, NM

Comprehensive Inventory of Research Grants 1997 — 2013

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201 Synaptic Protein Loss and Alterations in Elizabeth B . Newcastle University 1999 $ 25,000 (4) Anatomic and Growth Inhibitory Factors as a Biological Mukaetova-Ladinska, Newcastle upon Tyne, Histopathological Surveys Foundation of Behavioural Changes & MD United Kingdom (7) Clinical, Non-Treatment- Cognitive Decline in PSP Oriented Research

202 Mechanisms of Neurofibrillary Tangle Nancy A . Muma, PhD Loyola University 1999 $ 20,000 (1) Tau: Genetics, Biochemistry Formation in PSP Chicago and Treatment Target (b . Maywood, IL Biochemistry of tau and tangles)

203 Is Brain Oxidative Stress & Damage Stephen J . Kish, PhD Centre for Addiction 1999 $ 25,000 (2) Non-Tau Based Pathologies: Characteristic of PSP? & Mental Health Mitochondrial, Radicals, Cell Toronto, ON Death

204 Role of the Thalmus in PSP and Jasmine Henderson, Prince of Wales 1999 $ 25,000 (4) Anatomic and Parkinson’s Disease PhD Medical Research Histopathological Surveys Institute Randwick, Australia

206 Identification of the PSP Gene Parvoneh Navas, PhD University of 1999 $ 25,000 (1) Tau: Genetics, Biochemistry Washington and Treatment Target (a . The Seattle, WA tau gene)

207 Problems of Cell Death in PSP Kurt A . Jellinger, MD University of Vienna, 1999 $ 18,000 (2) Non-Tau Based Pathologies: School of Medicine Mitochondrial, Radicals, Cell Vienna, Austria Death

208 Activity and Expression of Antioxidant Sarah Jane Augood, Massachusetts 1999 $ 20,000 (2) Non-Tau Based Pathologies: Enzymes in the PSP Brain PhD General Hospital Mitochondrial, Radicals, Cell Boston, MA Death

209 Are Impairments of Energy Metabolism M . Flint Beal, MD Cornell University, 1999 $ 19,929 (2) Non-Tau Based Pathologies: Contributory in PSP? Weill Medical College Mitochondrial, Radicals, Cell New York, NY Death

210 Ultrastructural and Biochemical Hanna Ksiezak-Reding, Mount Sinai School 1999 $ 18,000 (1) Tau: Genetics, Biochemistry Hetergeneity of Paired Helical PhD of Medicine and Treatment Target (b . Filaments in PSP New York, NY Biochemistry of tau and tangles)

211 Mutational Analysis of the Tau Gene Joseph J . Higgins, MD New York State 1999 $ 20,000 (1) Tau: Genetics, Biochemistry in PSP Dept . of Health, and Treatment Target (a . The New York, NY tau gene)

300 Environmental Factors and Rosemary S . Waring, University of 2000 $ 20,000 (6) Clinical and Laboratory Detoxification Mechanisms in PSP MA, PhD Birmingham Treatment-Oriented Research Birmingham, United Kingdom

301 Regulation of Human Tau Gene Jane Wu, PhD Washington 2000 $ 20,000 (1) Tau: Genetics, Biochemistry Expression and its Role in PSP University, School and Treatment Target (a . The of Medicine tau gene) St . Louis, MO

303 Are Matrix Metalloproteinases Involved David S . Albers, PhD Cornell University, 2000 $ 20,000 (2) Non-Tau Based Pathologies: in the Pathogenesis of PSP? Weill Medical College Mitochondrial, Radicals, Cell New York, NY Death

304 Effect of Lipoperxidation on cdk5 Massimo Tabaton, MD University of Genoa, 2000 $ 20,000 (1) Tau: Genetics, Biochemistry Activity and Tau Protein Aggregation: Department of and Treatment Target (b A Model of PSP Pathogenesis Neurosciences Biochemistry of tau and Genoa, Italy tangles (2) Non-Tau Based Pathologies: Mitochondrial, Radicals, Cell Death

400 Dopa-resistant Parkinsonism in Dominique University Hospital 2001 $ 50,000 (5) Toxins and Epidemiology Guadeloupe: Evaluation of Caparros-Lefebvre, MD Guadeloupe, France Isoquinolines Derivates and Acetogenines Toxicity in Rats


46


401 mRNA Profiling in the Postmortem PSP Sarah Jane Augood, Massachusetts 2001 $ 47,500 (4) Anatomic and Brain: Identifying Abnormal Signaling PhD General Hospital Histopathological Surveys Pathways Boston, MA

402 Comparison of Region Specific mRNA Vincenzo Bonifati, MD Erasmus University 2001 $ 45,000 (4) Anatomic and Expression Profiles of PSP Brains with Rotterdam, Histopathological Surveys those of Alzheimer, FTDP-17, Pick The Netherlands Disease & Non Affected Brains, Using DNA Microarray Technology

403 Mechanisms Regulating Neurofibrillary Nancy A . Muma, PhD University of 2001 $ 42,406 (1) Tau: Genetics, Biochemistry Tangle Formation in PSP Washington and Treatment Target (b . Seattle, WA Biochemistry of tau and tangles)

404 Tau Auto-Antibody Production in PSP James W . Tetrud, MD The Parkinson’s 2001 $ 38,976 (1) Tau: Genetics, Biochemistry Institute, Research and Treatment Target (b . and Treatment Biochemistry of tau and Center tangles) Sunnyvale, CA

405 Regulation of Human Tau Gene Jane Wu, PhD Washington 2000 $ 25,794 (1) Tau: Genetics, Biochemistry Expression and its Role in PSP University, School and Treatment Target (a . The of Medicine tau gene) St . Louis, MO

406 Characterization of the Molecular Justo Garcia de Fundacion Jimenez 2001 $ 45,000 (1) Tau: Genetics, Biochemistry Mechanisms Leading to PSP Yebenes, MD, PhD Diaz and Treatment Target (b . Madrid, Spain Biochemistry of tau and tangles)

407 Interaction of Parkin Protein with Paul S . Fishman, MD University of 2001 $ 40,000 (1) Tau: Genetics, Biochemistry Abnormal Tau Maryland, School of and Treatment Target (b . Medicine Biochemistry of tau and Baltimore, MD tangles) (3) Non-Tau Based Genetic Studies

408 Finding the Cause and Effect of a David S . Albers, PhD Cornell University, 2001 $ 49,775 (2) Non-Tau Based Pathologies: Bioenergetic Defect in PSP Weill Medical College Mitochondrial, Radicals, Cell New York, NY Death

409 Eloise H . Troxel Memorial Brain Bank Dennis Dickson, MD Mayo Clinic 2001 $ 60,000 (8) Brain Bank Jacksonville, FL

410 Development of a Measure of Health- Anette Schrag, MD, University College 2002 $ 46,711 (7) Clinical, Non-Treatment- Related Quality of Life PSP PhD London Oriented Research London, United Kingdom

411 Glial Tau Aggregates in PSP and Hanna Ksiezak-Reding, Mount Sinai School 2002 $ 50,000 (1) Tau: Genetics, Biochemistry Human Cultured Cells PhD of Medicine, and Treatment Target (b . Department of Biochemistry of tau and Psychiatry tangles) New York, NY

412 Cortial and Striatal Cholinergic David J . Burn, MD Newcastle General 2002 $ 46,314 (4) Anatomic and Receptor Subtypes in PSP, Alzheimer’s Hospital, Regional Histopathological Surveys Disease and Dementia with Lewy Neuroscience Bodies Centre Newcastle upon Tyne, United Kingdom

413 Analysis of the 17q21 Region in PSP, Eduardo Tolosa, MD, Hospital Clinic, 2002 $ 36,000 (1) Tau: Genetics, Biochemistry Tau Gene Analysis in Parkinson’s Disease PhD Neurology Service and Treatment Target (a . The Dementia and in Other Atypical Barcelona, Spain tau gene) Parkinsonisms


47


414 Screening of Tau Mutation in a Unique Rong Chen, MD, PhD The Parkinson’s 2002 $ 14,250 (1) Tau: Genetics, Biochemistry PSP Family Institute, Research and Treatment Target (a . The & Treatment Center tau gene) Sunnyvale, CA

415 Characterization of Tau Auto- James W . Tetrud, MD The Parkinson’s 2002 $ 30,010 (1) Tau: Genetics, Biochemistry Antibodies in PSP Institute, Research and Treatment Target (b . & Treatment Center Biochemistry of tau and Sunnyvale, CA tangles)

416 Cross-Linking of Tau in PSP Nancy A . Muma, PhD Loyola University 2002 $ 49,998 (1) Tau: Genetics, Biochemistry Neurofibrillary Chicago and Treatment Target (b . Maywood, IL Biochemistry of tau and tangles)

417 Prehistory of PSP: Bibliographic Adolfo Brusa, MD Ospedalia Galliera 2002 $ 3,000 (7) Clinical, Non-Treatment- Search and Copying Genoa, Italy Oriented Research

418 The Relationship of Guamanian John C . Steele, MD Guam Memorial 2002 $ 48,300 (7) Clinical, Non-Treatment- Pigmentary Retinopathy to ALS/PDC Hospital Oriented Research of Guam Tamuning, Guam

419 Efficacy of Environmental on a Mouse Jada Lewis, PhD Mayo Clinic 2002 $ 49,987 (1) Tau: Genetics, Biochemistry Models of Tauopathy Jacksonville, FL and Treatment Target (b . Biochemistry of tau and tangles)

420 A Novel Approach for Neuroprotection Irene Litvan, MD Henry M . Jackson 2002 $ 45,460 (6) Clinical and Laboratory in PSP Foundation Treatment-Oriented Research Bethesda, MD

421 Diagnostic Protein Biomarker Lap Ho, PhD Washington 2003 $ 50,000 (4) Anatomic and Discovery in PSP University, School Histopathological Surveys of Medicine (7) Clinical, Non-Treatment- St . Louis, MO Oriented Research

422 mRNA Profiling in the Postmortem PSP Sarah Jane Augood, Massachusetts 2003 $ 50,000 (4) Anatomic and Brain: Target Identification PhD General Hospital Histopathological Surveys Boston, MA

423 Identification of Cis-Elements that Jianhua Zhou, PhD University of 2003 $ 46,540 (1) Tau: Genetics, Biochemistry Regulate Exon 10 Splicing in the Tau Massachusetts and Treatment Target (a . The Gene Medical School tau gene, b . Biochemistry of Worcester, MA tau and tangles)

424 Hyperphosphorylation, Tau Filaments Maria Grazia University of 2003 $ 50,000 (1) Tau: Genetics, Biochemistry and Neurodegeneration in a Transgenic Spillantini, PhD Cambridge and Treatment Target (b . Mouse Model of a Human Tauopathy Cambridge, Biochemistry of tau and United Kingdom tangles)

425 Genetic Analysis of 17q21 Region Alison M . Goate, Washington 2003 $ 25,000 (1) Tau: Genetics, Biochemistry Sporadic Tauopathies DPhil University, School and Treatment Target (a . The of Medicine tau gene) St . Louis, MO

426 Development and Characterization Etienne C . Hirsch, PhD Hospital de la 2003 $ 48,700 (5) Toxins and Epidemiology of a Novel Experimental Model of PSP Saltpetriere Paris, France

427 Aging Effects and Gene Therapy in a Ronald L . Klein, MD Louisiana State 2003 $ 50,000 (6) Clinical and Laboratory Novel Nigrostriatal Degeneration Model University, Health Treatment-Oriented Research Sciences Center Shreveport, LA

428 Refining the Genetic and Functional Matt Farrer, PhD Mayo Clinic 2003 $ 50,000 (1) Tau: Genetics, Biochemistry Role of the Tau H1 Haplotype in Jacksonville, FL and Treatment Target (a . The Neurodegeneration tau gene)

429 Formation Filamentous Tau Inclusions Shu-Hui Yen, PhD Mayo Clinic 2003 $ 50,000 (1) Tau: Genetics, Biochemistry in Human Cells with Inducible Jacksonville, FL and Treatment Target (b . Expression of Tau Proteins Biochemistry of tau and tangles)


48


430 Cystamine, a Transglutaminase Nancy A . Muma, PhD University of 2003 $ 44,000 (1) Tau: Genetics, Biochemistry Inhibitor, for the Treatment of Washington and Treatment Target (c . Tau Tauopathies, Especially in PSP Seattle, WA as a treatment target) (6) Clinical and Laboratory Treatment-Oriented Research

431 Discovery of Tau Phosphorylation Kenneth S . Kosik, MD Brigham and 2003 $ 44,000 (1) Tau: Genetics, Biochemistry Inhibitors for the Treatment of PSP Women’s Hospital and Treatment Target (c . Tau Boston, MA as a treatment target) (6) Clinical and Laboratory Treatment-Oriented Research

432 Correlation of Clinical Severity, Brain David J . Brooks, MD Imperial College 2003 $ 44,000 (7) Clinical, Non-Treatment- Inflammatory Changes & Apparent School of Medicine Oriented Research Water Diffusion Coefficients in PSP & London, Idiopathic Parkinson’s Disease United Kingdom

433 Parkin Mutations in a Mouse Model Parvoneh Navas, PhD University of 2003 $ 44,000 (1) Tau: Genetics, Biochemistry of PSP Washington and Treatment Target (b . Seattle, WA Biochemistry of tau and tangles) (3) Non-Tau Based Genetic Studies


435 Assesment of Tau Protein Isoform Profile Rohan de Silva, DPhil University College 2004 $ 36,898 (1) Tau: Genetics, Biochemistry in Cerebrospinal Fluid of Tauopathy London and Treatment Target (b . Patients as a Potential Diagnostic London, Biochemistry of tau and Biomarker United Kingdom tangles) (7) Clinical, Non-Treatment Oriented Research

436 Proteomic Analysis of a Transgenic Shu-Hui Yen, PhD Mayo Clinic 2004 $ 46,000 (1) Tau: Genetics, Biochemistry Mouse Model of Tauopathy Jacksonville, FL and Treatment Target (d . Tau modulation of other cell processes)

437 Heat Shock Proteins as Inhibitors of Christine University of 2004 $ 46,000 (1) Tau: Genetics, Biochemistry Tau Aggregation in Oligodendrocytes Richter-Landsberg, MD Oldenburg and Treatment Target (c . Tau Oldenburg, Germany as a treatment target)

438 Proteomis Analysis of Post Mortem Benoit Giasson, PhD University of 2004 $ 46,000 (4) Anatomic and PSP Brain Pennsylvania Histopathological Surveys Philadelphia, PA

439 Identification of Compounds that Jianhua Zhou, PhD University of 2004 $ 40,000 (1) Tau: Genetics, Biochemistry Modulate Exon 10 Splicing in the Massachusetts and Treatment Target (c . Tau Tau Gene Medical School as a treatment target) Worcester, MA (6) Clinical and Laboratory Treatment-Oriented Research

440 PSP and a Failing Ubiquitin-Proteasome F . W . van Leeuwen, Netherlands 2004 $ 45,000 (4) Anatomic and System PhD Institute for Brain Histopathological Surveys Research Amsterdam, The Netherlands

441 A Zebrafish Model of Tauopathy Edward Burton, MD University of 2005 $ 49,941 (1) Tau: Genetics, Biochemistry Pittsburgh and Treatment Target (c . Tau Pittsburgh, PA as a treatment target)

442 Proteomics Analysis of a Novel Murine Mark S . Forman, MD, University of 2005 $ 50,000 (1) Tau: Genetics, Biochemistry Model of Astrocytic Tau Pathology PhD Pennsylvania and Treatment Target (d . Tau in PSP Philadelphia, PA modulation of other cell processes)

444 Effect of Coenzyme Q10 in PSP: Diana Apetauerova, Lahey Clinic 2005 $ 50,000 (6) Clinical and Laboratory A Randomized, Multicenter, Placebo- MD Burlington, MA Treatment-Oriented Research Controlled, Double Blind Study


49


445 Do PSP-Associated TAU Hana N . Dawson, PhD Duke University 2005 $ 50,000 (1) Tau: Genetics, Biochemistry Polymorphisms Alter the Expression Medical Center and Treatment Target (a . The of the TAU Microtubule Binding Domain? Durham, NC tau gene

446 Inhibiting Transglutaminase Splice Nancy A . Muma, PhD Loyola University 2005 $ 50,000 (1) Tau: Genetics, Biochemistry Variants for Treatment of PSP Chicago and Treatment Target (c . Tau Maywood, IL as a treatment target) (6) Clinical and Laboratory Treatment-Oriented Research


448 AKT-Dependent Signaling in PSP and Hanna Ksiezak-Reding, Mount Sinai School 2005 $ 50,000 (1) Tau: Genetics, Biochemistry Transgenic Mouse Model of Tauopathy PhD of Medicine and Treatment Target (b . New York, NY Biochemistry of tau and tangles, d . Tau modulation of other cell processes)

449 Studies of PSA Neuroprotective Role Stanislav L . Karsten, University of 2006 $ 50,000 (1) Tau: Genetics, Biochemistry Using Transgenic Mouse Models PhD California, Los and Treatment Target (c . Tau Angeles as a treatment target) Los Angeles, CA (6) Clinical and Laboratory Treatment-Oriented Research

450 Hypothesis-Driven Gene Profiling in an Ronald L . Klein, MD Louisiana State 2006 $ 50,000 (1) Tau: Genetics, Biochemistry Animal Model of PSP University Health and Treatment Target (b . Sciences Center Biochemistry of tau and Shreveport, LA tangles, d . Tau modulation of other cell processes)

451 Interaction of Neuronal and Glial Tau Mel B . Feany, MD, PhD Brigham and 2006 $ 50,000 (1) Tau: Genetics, Biochemistry in a Drosophila Model of Tauopathy Women’s Hospital and Treatment Target (b . Boston, MA Biochemistry of tau and tangles, d . Tau modulation of other cell processes)

452 Finemapping of Risk Loci for PSP Michael Hutton, PhD Mayo Clinic 2006 $ 50,000 (3) Non-Tau Based Genetic Identified in a Genome-Wide Scan Jacksonville, FL Studies

453 Parkin and Tau Mutational Effects on Parvoneh Navas, PhD University of 2006 $ 50,000 (1) Tau: Genetics, Biochemistry Tangle Formation in PSP Washington and Treatment Target (b . Seattle, WA Biochemistry of tau and tangles)

454 Tau Aggregation in Oligodendrocytes Christine University of 2007 $ 104,000 (1) Tau: Genetics, Biochemistry and the Role of Thrombin Signaling Richter-Landsberg, MD Oldenburg and Treatment Target Oldenburg, Germany

455 Strength Training Patients with PSP Christine Sapienza, PhD University of Florida 2007 $ 94,327 (6) Clinical and Laboratory for Dysphagia Huber Fernandez, MD Gainesville, FL Treatment-Oriented Research

456 Inhibition of Tau Pathology in Transgenic Hanno M . Roder, PhD Mayo Clinic 2007 $ 250,000 (6) Clinical and Laboratory Mouse Models with an Optimized Michael L . Hutton, PhD Jacksonville, FL Treatment-Oriented Research Orally Active Tau Kinase Inhibitor

457 Genome-Wide Association Study in Ulrich Müller, MD, PhD University Hospital 2007 $ 200,000 (3) Non-Tau Based Genetic PSP Günter Höglinger, MD Giessen, Germany Studies

458 Unraveling Multi-Protein Chaperone Chad Dickey, PhD University of Oxford 2007 $ 150,000 (2) Non-Tau Based Pathologies: Complexes in PSP and Other Oxford, Mitochondrial, Radicals, Cell Tauopathies United Kingdom Death

459 Haplotype Regulation of Alternative Richard Wade-Martins, University of Oxford 2007 $ 150,000 (1) Tau: Genetics, Biochemistry Splicing at the MAPT Locus MA, DPhil Oxford, and Treatment Target (b . United Kingdom Biochemistry of tau and tangles, d . Tau modulation of other cell processes)

460 Longitudinal Prospective PSP Study Irene Litvan, MD University of Louisville 2007 $ 100,000 (5) Toxins and Epidemiology Louisville, KY


50


461 Genome-Wide Association Study in Gerard Schellenberg, Geriatric Research 2007 $ 96,700 (3) Non-Tau Based Genetic PSP PhD Education and Studies Chang-En Yu, PhD Clinical Center Seattle, WA

462 Genome-Wide Association Study in Bernie Devlin, PhD University of 2007 $ 20,000 (3) Non-Tau Based Genetic PSP Pittsburgh Studies Pittsburgh, PA

463 Genome-Wide Association Study in Hank Hakonarson, PhD Children’s Hospital of 2007 $ 580,000 (3) Non-Tau Based Genetic PSP Philadelphia, Center Studies for Applied Genomics Philadelphia, PA

464 Genome-Wide Association Study in Ulrich Müller, MD, PhD University Hospital 2007 $ 70,700 (3) Non-Tau Based Genetic PSP Günter Höglinger, MD Giessen, Germany Studies

465 Genome-Wide Association Study in Rohan de Silva, DPhil University College 2007 $ 20,000 (3) Non-Tau Based Genetic PSP John Hardy, PhD London Studies Andrew Lees, MD, FRCP London, United Kingdom

466 Genome-Wide Association Study in Dennis Dickson, MD Mayo Clinic 2007 $ 29,700 (3) Non-Tau Based Genetic PSP Matthew Farrer, MD Jacksonville, FL Studies Rosa Rademakers, PhD

467 Mapping Progressive Neurodegeneration David Williams, PhD, Monash University 2008 $ 50,000 (7) Clinical, Non-Treatment- in PSP using Transcranial Magnetic FRACP Melbourne, Australia Oriented Research Stimulation and CSF-Tau

468 Phenotypic Characterization of Edward Burton, MD University of 2008 $ 50,000 (1) Tau: Genetics, Biochemistry Tauopathy Zebrafish Pittsburgh and Treatment Target (b . Pittsburgh, PA Biochemistry of tau and tangles, d . Tau modulation of other cell processes)

469 Toward a Molecular Understanding Martin Margittai, PhD University of 2008 $ 100,000 (1) Tau: Genetics, Biochemistry of Tau Misfolding Denver and Treatment Target (b . Denver, CO Biochemistry of tau and tangles, d . Tau modulation of other cell processes)

470 Noninvasive Cortical Stimulation for Allen Wu, MD University of 2008 $ 100,000 (6) Clinical and Laboratory Motor and Non-Motor Features of PSP California, San Treatment-Oriented Research and CBD Francisco San Francisco, CA

471 Unilateral Pedunculopontine Deep Elena Moro, MD University of 2008 $ 100,000 (6) Clinical and Laboratory Brain Stimulation in PSP Toronto Treatment-Oriented Research Toronto, ON


473 The Role of Microglial-meditated Kiran Bhaskar, PhD Cleveland Clinic 2009 $ 150,000 (2) Non-Tau Based Pathologies: Neuroinflamation in Fronto-Temporal Florida Mitochondrial, Radicals, Cell Dementia Tau Pathology Tampa, FL Death

474 Targeting Hsp70 as a Therapeutic Chad Dickey, PhD University of 2009 $ 250,000 (6) Clinical and Laboratory Strategy for CBD and Other Taupathies South Florida Treatment-Oriented Research Tampa, FL

475 The Effects of CBD-Associated Tau Hana N . Dawson, PhD Duke University 2009 $ 250,000 (1) Tau: Genetics, Biochemistry Gene HI Haplotype on Tau Taupathies Medical Center and Treatment Target Durham, NC

476 Polyphenols from Grape Seeds as a Giulio Maria Pasinetti, Mount Sinai School 2009 $ 250,000 (6) Clinical and Laboratory Potential Therapeutic Agent in MD, PhD of Medicine Treatment-Oriented Research Taupathies New York, NY

477 A Pilot Clinical Trial of HAP (AL-108) for Adam Boxer, MD, PhD University of 2009 $ 250,000 (6) Clinical and Laboratory CBD and Frontotemporal Labor California, San Treatment-Oriented Research Degeneration with Predicted CBD Francisco Pathology San Francisco, CA


51


478 Genome-Wide Identification of Rohan de Silva, DPhil University College 2009 $ 209,119 (1) Tau: Genetics, Biochemistry Transregulators of Tau Gene John Hardy, PhD London and Treatment Target Expression & Splicing London, United Kingdom

479 HSP-Peptide Complex Mediated Michael DeTure, PhD Mayo Clinic 2009 $ $210,000 (8) Molecular and Cellular Immunization in a Mouse Model of Jacksonville, FL Abnormalities Tauopathy

480 In Vivo Identification of Mel B . Feany, MD, PhD Brigham and 2009 $ 217,500 (6) Clinical and Laboratory Pharmacological Treatments for Women’s Hospital Treatment-Oriented Research Tauopathy Boston, MA

481 Tau-Mediated Effects Upon Stuart Feinstein, PhD University of 2009 $ 226,493 (8) Molecular and Cellular Microtubule Structure and Kinesin California, San Abnormalities Translocation Francisco San Francisco, CA

482 Development of Activators of Stanislav L . Karsten, Los Angeles 2009 $ 217,483 (6) Clinical and Laboratory Puromycin Sensitive Aminopeptidase PhD Biomedical Treatment-Oriented Research to Treat CBD Research Institute Los Angeles, CA

483 Comparative Proteomics of CBD & Jayanarayan Mayo Clinic 2009 $ 156,205 (8) Molecular and Cellular PSP Brain to Identify Biomakers Kulathingal, PhD Jacksonville, FL Abnormalities

484 Structural Polymorphism of Tau Martin Margittai, PhD University of 2009 $ 52,200 (8) Molecular and Cellular Filaments in CBD Denver Abnormalities Denver, CO

485 MicroRNA Regulation of MAPT in CBD Rosa Rademakers, PhD Mayo Clinic 2009 $ 217,500 (1) Tau: Genetics, Biochemistry and Related Tuaopathies Jacksonville, FL and Treatment Target

486 Inhibition of Tau Pathology in Hanno M . Roder, PhD TauTaTis, Inc . 2009 $ 126,000 (6) Clinical and Laboratory Transgenic Mouse Models with an Michael L . Hutton, PhD Jacksonville, FL Treatment-Oriented Research Optimized Orally Active Tau Kinase Inhibitor

487 Modeling Haplotype-Specific Gene Richard Wade-Martins, University of Oxford 2009 $ 217,500 (1) Tau: Genetics, Biochemistry Function at the MAPT Locus in 4R MA, DPhil Oxford, and Treatment Target Tauopathy United Kingdom

488 Autophagic Modulation and Clearance W . Haung (Ho) Yu, PhD Columbia University 2010 $ 75,000 (1) Tau: Genetics, Biochemistry of Tau Aggregates Medical Center and Treatment Target New York, NY

489 Tau and its Pathology in Neuorons Maria Grazia University of 2010 $ 75,000 (1) Tau: Genetics, Biochemistry Derived from Induced Pluripotent Stem Spillantini, PhD Cambridge and Treatment Target Cells of FTDP-17T Patients with Cambridge, PSP-Like Tau Pathology United Kingdom


491 PSP/CBD Genome-Wide Association Gerard Schellenberg, University of 2010 $ 137,257 (1) Tau: Genetics, Biochemistry Analysis Follow-up and Replication PhD Pennsylvania and Treatment Target Chang-En Yu, PhD Philadelphia, PA

492 Pathogenetic Mechanisms of Gerard Schellenberg, University of 2010 $ 250,000 (1) Tau: Genetics, Biochemistry Progressive Supranuclear Palsy and PhD Pennsylvania and Treatment Target Corticobasal Degeneration Chang-En Yu, PhD Philadelphia, PA

493 A Pilot Clinical Trial of HAP (AL-108) for Adam Boxer, MD, PhD University of 2010 (see #501) (6) Clinical and Laboratory CBD and FTLD Degeneration with California Treatment-Oriented Research Predicted CBD Pathology (see #501) San Francisco, CA

494 Manipulating the Substrate Specificity Chad Dickey, PhD University of South 2011 $ 75,000 (1) Tau: Genetics, Biochemistry Program in the Chaperone System to Florida and Treatment Target Remove Tau in PSP and CBD Tampa, FL

495 CHIP-Mediated Regulation of Hsp90 Leonard Petrucelli, PhD Mayo Clinic 2011 $ 50,000 (1) Tau: Genetics, Biochemistry High Affinity Complex Jacksonville, FL and Treatment Target


52



496 Epigenetic Modifications in PSP Ulrich Müller, MD, PhD University Hospital 2011 $ 213,281 (3) Non-Tau Based Genetic CBD and Frontotemporal Labor Günter Höglinger, MD Giessen & Marburg Studies Degeneration with Predicted CBD Giessen, Germany Pathology

497 Evaluating Reduced Tau Levels as a Timothy M . Miller, MD, Washington 2011 $ 75,000 (1) Tau: Genetics, Biochemistry Therapy fo PSP and CBD PhD University School of and Treatment Target Medicine St . Louis, MO

498 Models to Determine the Toxicity of T . Chris Gamblin, PhD University of Kansas 2011 $ $75,000 (4) Anatomic and Tau Aggregates Lawrence, KS Histopathological Surveys


500 PSP Whole Exome Study - Phase One Gerard Schellenberg, University of 2012 $ 250,000 (1) Tau: Genetics, Biochemistry PhD Pennsylvania and Treatment Target Philadelphia, PA

501 An Exploratory, Randomized, Double- Adam Boxer, MD, PhD University of 2012 $ 125,000 (6) Clinical and Laboratory Blind, Placebo-Controlled, Parallel California Treatment-Oriented Research Study to the AL-108-231 of San Francisco, CA Davunetide

502 Mechanisms of Tau and ER Stress for Jose F . Abisambra, PhD University of South 2012 $ 75,000 (6) Clinical and Laboratory Novel PSP and CBD Therapeutics Florida Treatment-Oriented Research Tampa, FL

503 Gene Expression and Neuropathology Nilufer Ertekin-Taner, Mayo Clinic 2012 $ 75,000 (2) Non-Tau Based Pathologies, Endophenotypes for Gene Discovery MD, PhD Jacksonville, FL Mitochondrial, Radicals, Cell in PSP Death

504 Identifying Functional Genetic Risk Nilufer Ertekin-Taner, Mayo Clinic 2012 $ 75,000 (3) Non-Tau Based Genetic Studies Factors for PSP by RNA-seq MD, PhD Jacksonville, FL

505 Tracking Progressive Neurodegeneration Massimo Filippi, MD Vita-Salute San 2012 $ 62,000 (4) Anatomic and in PSP Using Diffusion Tensor MRI Raffaele University Histopathological Surveys Milan, Italy


507 Splice Variant Markers for PSP Judith Potashkin, PhD Rosalind Franklin 2012 $ 75,000 (1) Tau: Genetics, Biochemistry Novel PSP and CBD Therapeutics University of Medicine and Treatment Target and Science Chicago, IL

508 PSP Whole Exome Study Phase Two Gerard Schellenberg, PhD University of 2012 $ 500,000 (1) Tau: Genetics, Biochemistry Pennsylvania and Treatment Target Philadelphia, PA

509 Tau Dimerization: A Mechanism of Tau Stuart Feinstein, PhD University of 2013 $ 75,000 (1) Tau: Genetics, Biochemistry Function and Dysfunction? California and Treatment Target Santa Barbara, CA

510 Assessment of Guam ALS/PDC in 2013-14 Michael Geschwind, PhD University of 2013 $ 100,000 (5) Toxins and Epidemiology John Steele, MD California & Tau Consortium

511 Transgenic Rats Expressing Wild-Type and Stanley Prusiner, MD University of 2013 $ 100,000 (1) Tau: Genetics, Biochemistry Mutant Human Tau California and Treatment Target San Francisco, CA

512 Mechanisms and Therapy for Tauopathy Benjamin Wolozin, MD, Boston University 2013 $ 75,000 (6) Clinical and Laboratory Based on RNA Binding Proteins PhD Boston, MA Treatment-Oriented Research

513 Urso Student Fellowship Emily Beisser Rutgers Robert Wood 2013 $ 3,000 (6) Clinical and Laboratory Johnson Medical School Treatment-Oriented Research New Brunswick, NJ


144 RESEARCH GRANTS FUNDED THROUGH JUNE 30, 2013 $ 10,933,554

53

Methods of Supporting CurePSP

The method by which you contribute to CurePSP can determine your tax benefits . Every person’s situation is unique . The Foundation’s Office of Development and Donor Relations will be happy to discuss with you various methods of giving and provide detailed opportunities for support . All inquiries will be held in the strictest confidence . Commitments made today ensure that CurePSP will maintain its role as a not-for-profit health service and research organization of high quality and standards .

Listed below are methods of how you may support the programs and services of CurePSP, which include general support, research, patient and caregiver support, advocacy, and education .

Through the Annual Fund – General appeal mailings and emails are regularly sent to CurePSP families several times each year and are the primary way for supporting our programs and services .

Through Special Events – CurePSP recruits volunteers to support fundraising efforts . Events include golf outings, walkathons, cycling, wine tastings, dances, dinner parties, and much more . In addition, CurePSP encourages constituents who are avid runners to participate in community marathons by piggybacking on an established event in honor of or in memory of a loved one . Piggybacking on community events removes the burden of planning and implementing an event and allows participants to participate for a specific charity .

Through Major Gifts – Donors are encouraged to make major gifts of at least $5,000 for research grants and other designated purposes . Donors may restrict their gifts for these particular uses . Donors that contribute at least $50,000 for research purposes may also have a special “named research grant” in their honor or in memory of a loved one .

Through Named Funds – A CurePSP Named Fund provides donors with the opportunity to make gifts that will have a lasting impact while recognizing their family, or honoring or memorializing a loved one . Named Funds may be established through special events or a special family writing campaign . An annual donation of at least $1,000 is required to keep a Named Fund active and recognized . Named Funds of $1,000 or more receive recognition in the CurePSP Annual Report and may also receive recognition at a family conference, webinar, or on printed materials .

Through Planned Gifts – The following provides samples of planned giving options: • Bequests• Gifts of Life Insurance• Charitable Remainder Trusts• Charitable Lead Trusts

If you are interested in establishing a planned gift to support the programs and services of CurePSP, please contact your financial advisor for further information on the best option for you .

Through the Legacy Society – Donors who give total cumulative gifts exceeding $10,000 are permanently acknowledged as members of the Legacy Society and receive recognition in the Annual Report and Honor Roll of Donors .

For more information on all of the above “methods of supporting CurePSP,” please contact Kathleen Matarazzo Speca, Vice President of Development & Donor Relations via email at speca@curepsp .org or at 800-457-4777, x5672 .

Foundation for PSP | CBD and Related Brain Diseases30 E . Padonia Road, Suite 201

Timonium, MD 21093

USA: 800-457-4777 Canada: 866-457-4777

Web: www .curepsp .orgEmail: info@curepsp .org

54

100 Women in Hedge Funds Annual GalaUS Industry Leadership Award Presented to Karen Cook

November 13, 2013Introduction by Michael Steinhardt

Founder, Steinhardt Management Company

“I had the pleasure of knowing Karen Cook since 1975, nearly 38 years . Longer, in fact, than her husband Everett . As an early hedge fund manager who actively traded large blocks of stock, I naturally turned to Goldman Sachs as my primary firm for execution . They were the leading trading desk, even more so at that time with fixed commissions giving way to negotiated commissions and with block trading coming into its heyday .

Back then it was unheard of to entrust large block trades to a female trader . There were none, at least until Karen came along . When I started doing business with Karen, I did not give it a second thought that she was a woman . Goldman obviously trusted Karen - and, from the beginning, Karen earned my trust . She was smart, decisive, capable of committing large sums of capital, and took initiative to provide insight and guidance to me . She was a true professional . She was there every day applying her intelligence, her judgment, and her talent for the benefit of the firm and its clients .

I now realize, in hindsight, how exceptional it was for Karen to be on that desk . Here she was, all of 23 years old, when, as a floor clerk at the NYSE, she walked onto the Goldman Sachs trading floor, resume in hand . She refused to be deflected by a receptionist whose instinct it was to turn her away, politely offering to give her resume to Human Resources . Instead, spotting someone whom to her had executive bearing, she walked over and introduced herself . It was Bob Rubin who would one day run Goldman and later become US Treasury Secretary . Bob conducted an interview on the spot . Eleven interviews and two weeks later, Karen had a job as the first female trader at Goldman .

To fully understand the situation, you would have to place yourself on that trading floor back then . It was dirty, crowded, loud and crass . All the traders were men, many smoking cigarettes or cigars, shouting orders, epithets, or four letter words .

There was Karen sitting at the top of a u-shaped desk with 3 partners . A beautiful redheaded female with no role models . No books on how to dress for success . No mentors to tell her what to say or what not to say . She was surrounded by a sea of barbarians . As her Goldman colleague Richard Perry recently said, “Karen was the original science experiment, dropped into this Petri dish . She was the alpha female .”

So, how did she do in this Darwinian environment? Simply stated, Karen thrived . She knew you had to grow, develop, create, and adapt . And like everyone else on that desk, she went toe-to-toe in terms of raw intelligence, speed, perseverance, hunger and a combination of brains and guts .

She was dealing with titans of industry, CEOs, terribly aggressive and tough hedge fund managers; I being one of them .

So why did these people want to do business with Karen? Why did I? In a word, TRUST—We trusted her and she trusted us .

This was a world where your word was your bond, and Karen

delivered commitment and confidence every day . To put it succinctly, Karen had excellent judgment and the highest standards of integrity .

On the softer side, Karen smiled, laughed, she had style, she was beautiful - she broke down barriers with charm and consistency . What better combination to become this trailblazer for women on Wall Street and be a role model for so many women .

There came a time in Karen’s life, in 1988 when, with two young children, two dogs, a cat, and a husband, that she felt a strong maternal pull to be with her family . Again, there were no role models to whom she could turn to resolve the conflict so many women faced between raising a family and going to work every day . There were no flexible work hours nor family leave programs .

So, following 2 years of being a full-time mom sitting on park benches with other mothers who put their professional careers on hold, Karen established Alternatrak, a firm that found part-time professional work for mothers who had left Wall Street but still wanted to be in the game . Goldman also hired her on a consulting basis to develop their personnel policies relating to mothers . They did not want to waste the talent of so many capable women . Today, the flexible programs she pioneered have been implemented throughout the industry and are making a difference for millions of women . Goldman was a pioneer in making these changes, and Karen was its trailblazer .

After she sold Alternatrak to her partner, Karen came to work at Steinhardt Partners as my Head of Investor Relations in 1990 . Six months later, I decided to quit the hedge fund business . I no longer needed a Head of Investor Relations . Karen, having just begun, was effectively already out of a job . Judy, my wife, said to me, “I know you have to let a lot of people go, but you must keep Karen .” Karen reinvented herself . She adapted . She became my Chief Investment Officer . She started a fund–of–funds called Nepeta Partners and attracted outside capital . She even forced me to finish my autobiography, NO BULL, taking my oral renditions, writing them up, submitting them back to me for refinement, and then reworking them . She would not accept anything I submitted unless it was up to her standards . And her standards were high . She would have it no other way, and she knew I would not either . During the course of writing NO BULL, I once said to her, “I hope you are not this tough on Everett .” She smiled and said, “I am . My boys too!”

Karen left us too soon . She made a big difference in the lives of many . She showed us that you could raise a family and have a career . And be successful at both! Karen was a success at Goldman, with Alternatrak and at Steinhardt . She was a success at home . She had a beautiful marriage with Everett and raised two champion sons, Everett and Conor . Because of her efforts and her example, she left this world a better place . We are better women and men for it .

I stand before you tonight with great pride in being able to call Karen not only my colleague, but also my close friend . And it is with great pride that I present the 100 Women in Hedge Funds US Industry Leadership Award to Everett, her husband, on her behalf . In my opinion, no one could deserve it more .

Thank you .”

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curepsp 2013 annual report

Documents

research report

research committee

report of gifts

funding research

director of research

curepsp annual report

education report

wife karen cook