Potential Treatment for ALS and Frontotemporal Dementia DiscoveredDisney Scripps Florida

Potential Treatment for ALS and Frontotemporal Dementia Discovered

Matthew Disney earned a PhD in biophysical chemistry from the University of Rochester. Currently a full professor at the Scripps Research Institute in Jupiter, Florida, Matthew Disney serves in its chemistry department and has received grants for projects such as identifying and studying RNA loop-small molecule interactions and designing small, modular molecules targeting defective RNA. Since joining Scripps Florida, Matthew Disney received the American Chemical Society’s Eli Lilly Award in Biological Chemistry, among others.

Potential Treatment for ALS and Frontotemporal Dementia Discovered

His team recently published work in collaboration with a Mayo Clinic team on a specific therapeutic strategy targeting amyotrophic lateral sclerosis (ALS) and a type of frontotemporal dementia, as well as a set of biomarkers to determine the therapy’s effectiveness. A mutation in the gene C90RF72 causes a longer-than-usual genetic sequence and results in the creation of abnormal RNA and toxic proteins. A pair of small-molecule drug compounds were able to target the abnormal RNA, disrupting its interactions with other key proteins and decreasing the toxic proteins. The success of the therapy can be measured in the level of toxic proteins in the patient’s cerebrospinal fluid.