1

PKD

A magazine for PKD patients, Foundation supporters, health professionals and researchers.

Progress2 Letter from the

Chairman of the Board

4 finding treatments: Patient PartiCiPation is Key

10 WaLK for PKd: one steP at a time

13 eLevating the Patients’ voiCe

14 PKd Community CeLeBrates aWareness day

Fall 2013

Margot Troutt Keys and daughter Lena share their story on Page 3

Letter from the Chairman of the Board

Thanksgiving is a time when we get together with family and friends, enjoy a uniquely American holiday, and reflect on all the gifts that we have received in this life. As someone who has lived with PKD, I consider it one of my gifts and an honor to lead the Board of Trustees as we work to realize our vision that “one day, no one will suffer the full effects of polycystic kidney disease.”

This Thanksgiving, I am thankful for the many people who make a difference in quest of our vision. These include:• Researchers who work tirelessly in pursuing

treatments, making steps forward with every study and trial, despite challenges that come their way;

• ThosewithPKD,theirlovedonesandfriends who remain steadfast in their hope of one day having therapies to improve their lives, and those of their children and grandchildren;

• Patients who take part in clinical studies to advance research as quickly as possible (read page three to learn about a mother and daughter committed to doing their part);

• Volunteers who give their time and energy to host successful Walks and events, and provide a community of support for those with PKD;

• The more than 10,000 people who participate in the Walk for PKD every year;

• Donors who provide financial support and hold fundraisers to further the Foundation’s work;

• Thestaff who are dedicated to providing the best service and resources possible to the PKD community;

• And my fellow Board members, who devote time, skills and personal resources to keep the Foundation focused on its critical mission.

The path to ending PKD is a journey that takes many hands and many people. It is one of miles not inches; years not days. I am grateful for the individuals in the PKD community who keep us moving forward, and to be on this journey with you. Please continue reading this issue that offers more insight into clinical studies, and how you can become more actively involved in making a difference. Thank you for all you do!

Warm regards,

Frank CondellaBoard of Trustees Chair

M e ssage

2

3

But what really shook Margot was when her daughter Lena was diagnosed at age nine. She is now 15, a teenager trying to lead a normal life.

“I felt terrible when I found out Lena had PKD,” Margot said. “She was checked in utero and had been cleared, but when her later hypertension revealed she had it, I just burst into tears. I don’t think any parent feels good about passing on a life-threatening illness to their child, and I was no exception.”

Lena took it well for a nine-year-old. “I knew my mom had it and she seemed okay, so I wasn’t too worried.”

But PKD takes a toll on both mother and daughter. Margot works hard to watch her diet and exercise, and feels pretty good physically, but emotionally and logistically PKD has dramatically changed her lifestyle. Lena hasn’t told most of her friends at school for fear they will treat her differently. It is important to Margot to be a role model for Lena about how to take care of herself and live a fulfilled life with PKD.

One way the Keys family works to be their own best advocates is to connect with the PKD Foundation. Margot and Lena attended United on the Hill (the PKD Foundation’s advocacy event) in June and participated in

the Philadelphia Walk for PKD in September. Bill, Margot’s husband, ran in the Marine Corps Marathon in October as part of the Run for PKD program (runforpkd.org).

The Keys also arm themselves with as much information as possible. One way they do this is by taking part in clinical studies. “I highly recommend participating in clinical studies because you find out a lot about yourself and your own disease,” Margot said. “I participate to learn about what I can do to take care of myself, such as exercising, getting support and managing stress. Without being armed with information, you are just sitting and waiting when you may be able to change the outcome.”

Margot is in an observational study at the Hospital of the University of Pennsylvania. The goal of the study is to follow PKD patients over time to understand more clearly how the disease progresses, and if there are any notable trends over time. She goes in often for routine appointments for blood work, MRIs of her kidneys and physical examinations.

Lena is in the LEAPP Program through the Children’s Hospital of Philadelphia. LEAPP stands for Longitudinal Experience to

Appreciate Patient Perspectives. The program involves two first-year medical students being assigned to patients with serious chronic illnesses, who they will keep in contact with for several years to understand how a chronic illness can affect a patient’s life, work and family. In this program, Lena will be the teacher and the medical students will learn from her.

Margot encourages others with PKD to get involved with clinical studies. “It is important to participate in studies because it helps all of us and future generations to knowmoreaboutPKD,” Margot said. “It is a generational disease. If you won’t do it for yourself, do it for your kids. Until there is a cure, it helps to address the disease and find out more about how to live our best lives.” n

Keys family Works to Change the outcome of PKd

It is important to Margot Troutt Keys to be a role model for her daughter Lena abouthow to take care of herself and live afulfilled life with PKD. (Shown left to right: Margot, Lena and Bill)

Margot troutt Keys found out her father had PKd when she was only eight years old, after he passed away from a cerebral hemorrhage. Margot and her teenage siblings were tested at that time for PKd, but they were cleared. at the age of 25, Margot started having terrible side pain and blood in her urine. she soon learned that she had PKd after all, despite the tests performed earlier in her life.

r e se a rch

4

What is a CliniCal study?

A clinical study involves research using human

volunteers (participants) that is intended to add to

medical knowledge. There are two types.

ObservatiOnal studies

CliniCal trials

A drug is given or a device is used to alter the course of the disease.

leading tO:yOu might: • Write in a journal• Talk with a doctor• Keep a food diary• Have your blood

pressure taken

example:Consortium for radiologic imaging studies of PKd (CrisP) treatments

yOu might:• Take a pill/drug• Have a procedure

done• Use a device• Change your diet

Data is collected but there is no attempt to alter the disease progression.

example:tolvaptan temPo ¾ trial

Provides clinical information about disease progression

Evaluates effectiveness of an intervention to slow or stop a disease

5

The PKD Foundation believes empowered patients are the key to accelerating the development of a robust treatment pipeline. Without participation in research, medical breakthroughs cannot

happen and new therapies cannot be approved for patient use.

Progress to find treatments for polycystic kidney disease is partially driven by patient involvement in clinical studies. According to clinicaltrials.gov, “A clinical study involves research using human volunteers that is intended to add to medical knowledge.” There are two types of studies and patients can participate in many ways. These include:

• ObservationalStudies monitor and record various clinical and/or subjective factors over a period of time, like blood pressure or quality of life, to better understand how the disease progresses. Involvement includes activities such as having conversations with researchers and physicians, or recording daily events in journals. No drugs are given or other interventions* are made to alter the course of the disease. An example of an observational study is the Consortium for Radiologic Imaging Studies of Polycystic Kidney Disease (CRISP).

• Clinical trials monitor and record various clinical and/or subjective parameters by testing experimental treatments, devices or combinations of drugs that may alter the course of the disease. Participants receive specific interventions* which may be medical products such as drugs or devices; procedures; or changes to participants’ behavior such as diet. The purpose of a clinical trial is to test a new drug or other intervention for safety and effectiveness in treating the disease in question before it can be prescribed for patients. An example of a clinical trial is the Tolvaptan TEMPO ¾ Trial.

*According to clinicaltrials.gov, an intervention is a process or action that is the focus of a clinical study. This can include giving participants drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches such as surveys, education and interviews.

What to Know about Participating in Clinical studiesParticipating in a clinical study can be a very satisfying and worthwhile experience. The results can make a difference in the care of future patients by providing information about the benefits and risks of therapeutic, preventative, or diagnostic products or interventions.

By participating in a clinical study, you can:• Play a more active role in your own health care.• Have access to new treatments before they are

available to the public.• Contribute to the development of treatments for PKD.• Make things better for the next generation of those

with PKD.

It is important that any participation should be done in consultation with your health care provider. To participate in a clinical study, you will need to have a formal diagnosis of PKD made by a physician.

Anyone interested in participating in a study should know as much as possible about the study and feel comfortable asking the research team questions. There are processes in place to protect patients, such as the informed consent process. It involves researchers communicating with potential and enrolled patients about a clinical study, providing all the important information about the study, ensuring potential participants understand the risks and benefits and stress that enrollment is strictly voluntary. All important information about a study must be given to the potential participant in a written document that is easily understood. Typically, a person must sign an informed consent form to participate. For more information on this process and a list of sample questions to ask, visit clinicaltrials.gov.

With thousands of people living with PKD in the U.S. alone, clinical study participation can provide help and hope to those affected by PKD.

finding treatments: Patient Participation is Key

Many studies are actively recruiting.

You can find specific information about these

at clinicaltrials.gov.

r e se a rch

6

awareness and drop-outs the Biggest Challenges for successful studiesawareness The greatest hurdles for successful studies are low recruitment due to lack of awareness, and participants dropping out before the study is complete. 

According to a report from the National Institutes of Health (nih.gov/health/clinicaltrials/providers/awareness.htm) that compiled several studies:• 85% of patients were either unaware or unsure that

participation in a clinical trial was an option at the time of diagnosis.

• 75% of these patients said they would have been willing to enroll had they known it was possible.

Today, approximately 80 percent of all clinical trials fail to recruit enough volunteers within planned timelines. Under-enrollment is potentially one of the most significant problems facing PKD drug development.

In 2010, to address this awareness challenge, the PKD Foundation launched the ClinicalTrialsAwarenessProgram(CTAP), an aggressive program to help create awareness amongst patients and families and speed up clinical trial recruitment. The program is focused on educating patients about current studies so they can make educated decisions about participating. The goal is to simplify the process of finding clinical studies for PKD patients in their geographic area by sending Accelerating Clinical Trials (ACT) Alert emails about studies that are being conducted.

Traditionally, the pharmaceutical industry leverages mass media, like television, radio and print advertising,

to inform patients about clinical trials. To reach the national, widely dispersed PKD community in this way is expensive and not an effective use of funds. This makes it difficult for PKD patients to be made aware of opportunities to consider clinical trial participation.

In 2012, ACT Alerts focused on two clinical trials that were enrolling ADPKD patients. Thirty-sixthousandemailsweresentoutsixtimesoverathree-monthperiodwithanaverageof14percentofrecipientsopening the emails. The organization conducting the trials used a questionnaire to gain insights from participating patients, and nearly 80 percent of the 416respondentslearnedaboutthetrialfromtheFoundation’sACTAlert.

Participation and dropping OutWhen designing a clinical study, the study research team determines the number of participants needed for enrollment in order to measure a significant effect of the intervention. While drop outs can’t always be avoided, it does impact the results if enrollment numbers drop below what is needed for study significance. Participants should discuss study requirements with their health care provider and members of the research team before enrolling to ensure expectations are understood and can be met.

FDA’s Drug ApprovAl process2.Preclinical animal testing to gather information on safety and effectiveness.

3.an investigational new drug (ind) application outlines what the sponsor of a new drug proposes for human testing in clinical trials.

4.Phase 1 studies (typically involve 20 to 80 people). this phase emphasizes safety.

1. drug compound developed by drug sponsor.

ToreceiveACTAlerts,pleasevisitpkdcure.org/email-preferences, enter your information and check the ‘ClinicalTrialsandResearch’button.

7

Phases of a Clinical TrialThe U.S. Food and Drug Administration (FDA) is the federal agency charged with oversight of all the clinical trials going on in the U.S. at any time. A new drug to treat PKD must move through each stage before it can be reviewed for approval by the FDA. The FDA defines the phases for the clinical trials.• PhaseI– The new drug is tested for safety and side

effects in a small number of healthy volunteers with PKD.

• PhaseII – The new drug is tested for safety, dose ranging and preliminary effectiveness in a small number of volunteers.

• PhaseIII – The new drug is tested in a large number of volunteers with PKD to establish effectiveness, monitor side effects and compare results with current treatments.

• The data collected during the clinical trial is analyzed and then submitted to the FDA for regulatory review, which can take one to three years. If there is an intent to apply for an NDA (new drug application), it is communicated to the FDA and shapes the design of the study. Once it is approved, the new drug can be prescribed by physicians to treat PKD. (See Page 8 about the tolvaptan NDA).

• PhaseIV – These studies are done after the drug has been approved by the FDA and it is in use (considered a post-marketing study). Additional information about risks, benefits and optimal use is collected and analyzed.

clinicAl stuDies For pKD treAtmentsin the u.s., there are more than 40 clinical studies related to PKD. twenty-six are currently active, 17 of which are recruiting for participation. for more information on these studies, visit pkdcure.org/clinicalstudies. Clinical studies are critical and a required step to the development of new therapies.

5.Phase 2 studies (typically involve a few dozen to about 300 people). this phase emphasizes effectiveness.

6.Phase 3 studies (typically involve several hundred to about 3,000 people). this phase gathers further information about safety and effectiveness through different populations and dosages.

7.the pre-nda period, just before a new drug application (nda) is submitted. a common time for the Fda and drug sponsors to meet.

Phase 2 and 3 studies are conducted on patients with the disease that is being studied.

8

Tolvaptan Updatein late august, otsuka Pharmaceutical development & Commercialization, inc. received a Complete response Letter (CrL) from the u.s. food and drug administration (fda) regarding tolvaptan to treat adult patients with adPKd. the fda issues CrLs to convey that their initial review of a new drug application (nda) is complete and they cannot approve it in its present form. according to otsuka, the fda requested they provide additional information. the next follow-up steps are being determined by otsuka.

Like you, we were hoping for approval and were disappointed to hear this news. We know the PKd community is anxious for a treatment to become available and we share your frustration. We aren’t giving up on our mission, and we don’t want you to either.

While we were disappointed, some positive breakthroughs occurred because of the tolvaptan clinical trial. • a path has been opened up that has never been charted

for pharmaceutical companies to pursue developing PKd treatments. the foundation is committed to continuing this momentum, and continuing its critical work in the accelerating treatments to Patients program (atP).

• there are currently two drug candidates being screened through the foundation’s drug repurposing program.

• to continue moving translation research forward, this summer the foundation accepted research grant applications. in early 2014, funding of grants focused on the development of PKd therapies, as well as the basic science of PKd, will be announced.

it also became clear that the voices of PKd patients are being heard. more than ever, it is time for individuals to share their stories to impact decision-makers. dialysis, transplantation and homeopathic living should not be the only options for people with PKd.

tolvaptan is just one piece of the treatment puzzle, and a first step. We couldn’t have reached the place where a candidate was even introduced if it weren’t for those with PKd who took part in the clinical trial and those who testified and submitted letters to the fda’s Cardiovascular and renal drugs advisory Committee meeting. We thank everyone who had a part in these efforts. now it is time for all of those in the PKd community to do their part by taking part in studies and trials, and advocating for treatments. Please keep reading for more information about how you can make a difference (see page 13).

special Webinar A special webinar was held by Dr. Arlene Chapman, who played a critical part in the tolvaptan clinical trial, and testified at the FDA’s Cardiovascular and Renal Drugs Advisory Committee meeting. She provided an explanation about the tolvaptan response, and discussed other clinical trials that are currently underway. View the webinar at pkdcure.org/tolvaptanwebinar.

To receive timely information about updates such as tolvaptan and other important re-search developments, sign-up or update your email today by visiting pkdcure.org/email-preferences.

11.the Fda reviews information that goes on a drug’s professional labeling (information on how to use the drug).

10.if the Fda files the nda, an Fda review team is assigned to evaluate the sponsor’s research on the drug’s safety and effectiveness.

9.after an nda is received, the Fda has 60 days to decide whether to file it so it can be reviewed.

8.submission of an nda is the formal step asking the Fda to consider a drug for marketing approval.

r e se a rch

9

research grants funding resumed

In June, the PKD Foundation once again released a Request for Applications (RFA) for research grant proposals

focused on developing PKD therapies, as well as the basic science of PKD. Applications focused on PKD in children were also encouraged.

The Foundation previously funded research grants from 1984-2009, spending more than $30 million to fund more than 500 grants. Past funding has led to two clinical trials, a recent study by the University of Kansas Medical Center Kidney Institute suggesting that Vitamin B3 holds promise for treating PKD, and early preclinical animal testing of a precursor of tolvaptan.

“The research grant program is important because it allows the Foundation to re-establish its efforts to move PKD science forward, facilitating the development of therapies to treat this devastating disease,” said Interim Chief Scientific Officer Lorrie Rome. Awarded grants will be announced in early 2014. The Foundation received 51 proposals by the September 2013 deadline. Rome anticipates that 12, two-year grants will be awarded for $80,000 per year (per grant). n

13.Fda reviewers will approve the application or issue a complete response letter (Crl). see the first paragraph in the update on page 8 for more information about Crls.

12.the Fda inspects the facilities where the drug will be manufactured as part of the approval process.

9

14.Once the Fda approves a drug, the post-marketing monitoring stage begins to detect serious unexpected events and take action when needed. drug can now be prescribed to patients.

Source: fda.gov

10

The Walk for PKD signifies a unified group, moving forward toward treatments, and ultimately a cure

for PKD. The Walk for PKD is the signature fundraising and awareness event for the PKD Foundation and the largest gathering of PKD patients, family, friends and supporters; nearly 10,000 strong.

Forty-nineWalkstookplacethisfall nationwide and raised more than $1.5 million (at the time of printing). Thank you for putting in the time, miles and money to unite to fight PKD!

If you’ve participated in a Walk for PKD, you know the real story is the emotion behind every step of those walking to end this devastating disease. The sense of togetherness and support can be seen and felt in every family walking for a loved one. It’s there behind the tears of those who walk in memory of a beloved friend or family member who lost their battle to PKD. Those who have passed live on in the hearts of those who will keep walking until treatments are found.

MikePhelps,Lenexa,Kan., has walked twice for PKD since his wife Ashley was diagnosed with PKD in 2010. The Phelps family walks to support the PKD Foundation’s funding of the research that’s so important to Ashley.

“The Foundation provides support, and funds new developments in PKD research,” says Mike. “I believe it’s a lifeline to many people who have PKD. Without the Foundation, we would not have advanced to where we are today with PKD research.”

For Ashley, the chance to meet others experiencing the same challenges she faces is one of the best parts of taking part in the Walks.

“I got involved because you meet so many people who are just like you or know someone like you,” she says. “We have met so many amazing people who have helped me through my journey with PKD.

“Walking gives me hope that the money we raise will help find a treatment, or even a cure, for this horrible disease.”

Since PKD is a genetic disease, it can affect entire families. Many walkers, like TimWhitecotton of Denver, walk with the hope that one day there will be more options for treatment of kidney failure than dialysis or transplantation.

“This is my first year doing the Walk for PKD,” he says. “It’s so important for me to do this in order to help find a cure for this disease that has

so significantly impacted my family and other families over multiple generations.

“The PKD Foundation is doing life-saving work,” Tim continues. “Every step I take to help raise money for research brings us closer to a cure and hope for greater quality of life.”

This is also the first year that AmyKnox,Denver, has participated in a Walk. Both her grandmother and father had PKD. Amy was fortunate enough to receive a “Ferrari of a kidney” from her sister on January 30, 2013.

Amy says she will be carrying some very special people in her thoughts as she takes part in her first Walk for PKD.

“My father and I were both blessed to receive kidneys from siblings,” says Amy. “His lasted 18 years before he passed away.

one step at a timeWalkers across the country unite to fight PKD at annual Walk events.

e duc at ion a nd su PP ort

The sense of togetherness is shown by the Mehl family. To hear Sue Mehl’s story, visit pkdcure.org/voicesofpkd.

continued next page…

Photo credit: Randy Braley

11

“I’m doing very well, and I thank my sister every day,” she shares. “I’m walking in honor of my father who was my hero, those like me who are out there fighting this disease, and our beloved kidney donors who have given us all a second chance at life.”

The PKD Foundation encourages all Walk participants, teams, donors and supporters to remember fundraising continues through Dec. 31. So please sustain your

fundraising efforts to make the very most of Walk season.

The Walk for PKD is the Foundation’s largest signature fundraising and public awareness event and has raised more than $20 million in the fight to end PKD since 2000. For information about Walks being held in spring 2014, go to walkforpkd.org. n

gifts of securities a Win-WinOne of the easiest ways to make a gift to the PKD Foundation is to donate securities (stocks, bonds or mutual fund shares) that you have owned for more than a year and that have increased in value. Giving securities offers tax advantages not available with other types of contributions.

For JimWoods, giving to the PKD Foundation is a priority. “My health right now is a thumbs up. I am not on dialysis or in need of a transplant for my PKD. However, I will do anything I can for my three kids. It really hits home when you know this disease can

affect your family, particularly your children and the next generation. Any progress made in research now can benefit my children and their children.”

As Jim found out, giving a gift of stock is a win-win. “With stock, it is less costly than an outright cash gift. The appreciation can be felt by the PKD Foundation and I can make a difference, but my investment is actually less, and I receive tax and financial benefits.

Every little bit counts, and the PKD Foundation makes it really simple to donate this way.”

How It WorksAs stock prices increase, so do the taxes you owe on the long-term capital gain. These taxes are at the highest rates since 1997. But when you donate publicly traded stock you’ve owned for more than one year to a qualified charitable organization such as the PKD Foundation, you enjoy two major tax benefits:

1. You will be exempt from paying capital gains taxes on any appreciation (increase in value)—taxes you would pay if you had otherwise sold the securities.

2. You are entitled to a federal income tax deduction based on the current fair market value of the securities, regardless of their original cost.

The income tax deduction for long-term capital gain property is limited to 30 percent of your adjusted gross income in the year you make the gift, but your excess deduction is deductible for up to five additional years.If you own stock you wish to keep, consider donating that and using the cash you were going to use to make a charitable contribution to buy the same stock through your broker. This way, you’ll have a new, higher cost basis in the newly acquired stock.

Pleasenote: The steps above are for donating publicly traded stocks you have owned for more than one year that are worth more than when you first purchased them. If you have stock losses:

1. Sell the stock yourself to realize the loss and take any allowed deduction for tax purposes.

2. Then generate a charitable deduction by donating the cash proceeds of the sale to the PKD Foundation.

If you wish to transfer stock electronically, please instruct your broker that the PKD Foundation UMB Bank brokerage account number is 087-124052 and the DTC number is 0226. If you hold your stock certificates, please contact the PKD Foundation at 800.753.2873, ext. 187. n

11

12

Are you passionate about finding treatments and a cure for PKD? The DIY (Do It Yourself) for PKD

fundraising program allows you - the PKD community - to take the fight against this disease into your own hands by organizing a local fundraiser. Through DIYforPKD, you can do just about anything to raise dollars for PKD – host a lemonade stand, neighborhood garage sale, bowl-a-thon, work jeans day or dinner party for PKD.  The PKD Foundation has online tools and tips to make fundraising easy and rewarding.

Zac Haas threw his daughter Anabel a first birthday party to raise money for the PKD Foundation.

“My daughter Anabel was diagnosed with PKD around 20 weeks gestation. We were taken aback regarding the possibility of a serious, life-altering condition. Many emotions went through our minds and affected my entire family emotionally and physically. Anabel is now one year old and thriving.

For her first birthday, we invited 50 friends and family members

for those who don’t have a best case scenario like us. We want the PKD Foundation to be able to do research to find treatments and a cure.”

For more inspiration, visit pkdcure.org/voicesofpkd and read about other DIY for PKD events, such as SeanKenny plunging into freezing waters and CyrillaHaverkamp hiking 500 miles.

If you are interested in planning a fundraiser, please visit pkdcure.org/DIYforPKD for more information or contact Sara Brown at sarab@pkdcure.org n

diy for PKdDIY for PKD: Fundraise Your Way

to celebrate with us. Instead of bringing gifts, we asked our guests for donations to the PKD Foundation. We wanted to encourage a gift that could change the lives of many people forever, instead of opening gifts that would only last a short period. Anabel already has an abundance of toys and clothes from her older sister and grandparents who spoil her!

Weraisednearly$2,000forthePKDFoundation. We are fortunate to be able to have the best case scenario with one normal kidney and one affected by PKD. It is important to us to raise money

The Haas family threw a first birthday party for Anabel to raise money for the PKD Foundation.

The Organ Procurement and Transplantation Network (OPTN)/United Network for Organ Sharing (UNOS)

Board of Directors approved substantial amendments to the OPTN policy for deceased donor

The PKD Foundation submitted a statement and recommendation during the call for comments.  This was submitted by Board Member Dr. Benjamin Cowley, (Scientific Advisory Committee

significant revisions to deceased donor Kidney allocation Policy

kidney allocation at their meeting in late June. The changes seek to improve outcomes for kidney transplant recipients, increase the years recipients have a functioning transplant and increase the use of available kidneys. The changes are expected to occur in late 2014.

e duc at ion a nd su PP ort

continued next page…

12

In September, Rep. Leonard Lance (NJ-7) reintroduced the MODDERN Cures act in the House of Representatives. One of the PKD Foundation’s legislative priorities, this act aims to advance the

development of new treatments for chronic and rare diseases, and is critical in the search for treatments for PKD.

Thebillwill:• Encourage research on treatments, which have been

abandoned in the lab, but hold promise for treating diseases like PKD that have unmet medical needs.

• Encourage rapid development of new, safe and effective medicines.

• Provide a pathway to bring promising new compounds to market.

• Remove barriers that limit medical innovation.• Provide incentives for researchers to develop new

diagnostics.• Ensure timely and appropriate reimbursement

for new tests and treatments so that patients have access to the latest medical technology as soon as possible.

• Establish a predictable timeline for the introduction of generic equivalents.

• Advance creative solutions for developing companion diagnostic tests.

• Create a system that rewards efficiency and effectiveness to benefit all people with chronic diseases.

Patients advocating for treatmentsMODDERN Cures Act ReintroducedModernizing Our Drug and Diagnostic Evaluation and Regulatory Network

a dvo c ac y

The bill was developed in partnership with the National Health Council (NHC), of which the PKD Foundation is a member. The NHC provides a united voice for people living with chronic diseases and disabilities and their family caregivers. Read the bill at pkdcure.org/AdvocacyActionCenter.

Whatyoucando: Visit the PKD Advocacy Action Center for letter templates and other tools to assist you in asking your representative to co-sponsor the MODDERN Cures Act. A list of co-sponsors is also available at pkdcure.org/AdvocacyActionCenter.

Advocacy Action Center UpdatedNow more than ever, it is critical for patients to advocate on behalf of themselves, families, friends and loved ones with PKD. To help you do this, we have updated our Advocacy Action Center and Toolkit where you’ll find:• Updates about current legislation efforts• PKD-related legislative priorities• Sample emails/letters that you can personalize• Social media messages• Talking points• Twitter widget• Links to find local legislators• Tips on how to connect

Sign up to be a PKD Advocate and receive alerts by emailing pkdcure@pkdcure.org n

13

(SAC) Chair at the time of submission). The comment included that while the amendments are beneficial to younger patients with ARPKD, they may negatively impact patients who need transplants later in life. “The anticipated shift in the highest quality organs away from older recipients may adversely affect ADPKD patients disproportionately.” 

Visit pkdcure.org/kidneyallocation to learn more about the changes and read the Foundation’s full statement submitted during the call for comments. n

14

Sept. 4, 2013 was a special day to the PKD Foundation and the PKD community. Thanks to Senate Resolution 201, the

day was declared National Polycystic Kidney Disease (PKD) Awareness Day and aimed to raise awareness and understanding of PKD. The day was chosen to kick off the Walk for PKD season, which started that next weekend. The resolution was sponsored by Sen. Roy Blunt and co-sponsored by Sens. Benjamin Cardin, Barbara Mikulski, Marco Rubio and Charles Schumer. We thank them for their efforts and a big thanks to all our PKD advocates who helped us by contacting their local senators.

We turned the day into a social media campaign by calling it #PKDAwarenessDay and devoted the entire day to raising awareness of this life-threatening, genetic disease and the critical need for treatments and a cure. We asked for your help to raise awareness – and you did not disappoint! We were inspired and moved by your enthusiasm and support. Withyourhelp,wewere able to spread the word and reach more than 150,000 people.

The special day proved the importance of raising awareness and sharing your PKD story. The more people know about PKD, the closer we can get to treatments and a cure. Even though #PKDAwarenessDay is over, every day is an opportunity to create awareness about PKD! Share your story and help put a face and a voice to a PKD. For more information on Voices of PKD, read the next page. n

PKd Community Celebrates awareness day

awa r e n e ss

Our daughter who passed away from ARPKD at 28 hrs old.

ASPARTOF#PKDAWAReneSSDAY,we posted a question on Facebook to ask why you were raising awareness. Wereceivednearly300responses,andbelow is a sampling of your answers.

To garner more support for the promising research for treatment and a cure that is taking place....and to promote the living donor programs so that those of us afflicted by PKD may have a chance to live another day.

For me. Fortunately I am the last living member of my family who has PKD. So I also raise awareness for everyone who is affected by this disease.

My grandmother, my aunt, my uncle, my mother, my brother, and my three sisters. I love them all, and I don’t want to see my siblings have a transplant like my grandmother and mother had to.

For a grandfather I never knew and his son, my father.

For my grandmother that I never had the chance to meet, my mom, my uncle who went home this year, my sister, my aunt, my cousins and myself. Praying it never affects my daughter or my nieces.

My granddaugher, who passed away from PKD at 4 weeks old.

My mother-in-law (deceased due to PKD) my husband (received a transplant Dec. 2012) and my daughter, in early stages.

For my grandmother and my father, may they be with us in spirit forever. I also share this with myself. I was given a kidney on January 30 by my sister. Also for my son who I hope is spared this disease.

15

voices of PKdThe PKD Foundation launched the Voices of PKD, a collection of stories, testimonials, photos and video to tell the story of PKD through the eyes of the PKD community. By sharing your story, you can help put a voice and a face to a widely unknown disease. Your experience can paint a more powerful picture of what it is like to live with PKD.

Visit pkdcure.org/voicesofpkd to share your story, read about others’ experiences or download a flyer to share with family and friends. You are the voices of PKD! n

16

Please send your contact information updates or notify us of duplicate mailings of this publication by contacting donate@pkdcure.org or 800-753-2873, ext. 187.

BoardofTrustees(2013-2014term)Frank Condella (Chair)Blaise Hazelwood (Treasurer)Jerome Lienhard, II (Secretary)Sen. Robert BennettPaul ConwayBenjamin Cowley, Jr., M.D.Scott GoodmanLisa Guay-Woodford, M.D.Klee KleberBrock NelsonRichard NelsonDwight OdlandJulia RobertsJeffrey RonaAnne RyanTerry Watnick, M.D.Hilary Wolfe

The mission of the PKD Foundation is to promote programs of research, advocacy,education, support and awareness in order to discover treatments and a cure forpolycystic kidney disease and improve the lives of all it affects.

PKD Foundationpkdcure.orgpkdcure@pkdcure.org 816.931.2600800.PKd.CurefaX 816.931.8655

ScientificAdvisoryCommitteeTerry Watnick, M.D. (Chair)Stefan Somlo, M.D. (Vice Chair)John Bissler, M.D.Benjamin Cowley, Jr., M.D. Iain Drummond, Ph.D.Vincent Gattone II, Ph.D.Benjamin Margolis, M.D.York Pei, M.D.Ronald Perrone, M.D.Richard Sandford, Ph.D., FRCPDarren Wallace, Ph.D.Angela Wandinger-Ness, Ph.D.

LeadershipTeamMichelle Davis, Interim CEO and Chief Development Officer

Angela Connelly, Chief Marketing Officer

Ray Smith, CPA, Chief Financial Officer

Lorrie Rome, Interim Chief Scientific Officer

Connect with us! facebook.com/pkdfoundation twitter.com/pkdfoundation

8330 Ward Parkway, suite 510Kansas City, missouri 64114