Matthew Disney and his team at Scripps Research Institute recently published work identifying a potential treatment for amyotrophic lateral sclerosis (ALS) and frontotemporal dementia. They discovered a pair of small molecule drug compounds that target abnormal RNA caused by a mutation in the C90RF72 gene, disrupting its interactions and decreasing toxic proteins. The therapy's effectiveness can be measured by levels of toxic proteins in cerebrospinal fluid.

1. Potential Treatment for
ALS and Frontotemporal
Dementia Discovered
Disney Scripps Florida

2. Potential
Treatment for
ALS and
Frontotempor
al 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.

3. Potential
Treatment for
ALS and
Frontotempor
al 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.