RESEARCH WE HAVE FUNDED
As a member of ALS United and with the help of generous local support we contribute funds in research every year, in locations across the globe. We collaborate with a network of top-notch researchers and professionals to fund projects across the research pipeline.
Researchers identify a mechanism that can reverse abnormal RNA processing and shortcut the development process leading to novel therapeutics for people living with ALS
AUTTX, LLC. is focused on developing small molecule and ASO therapeutics for neurological disease resulting from abnormal gene splicing - working to develop novel therapeutics for people living with ALS.
Led by Isabelle Draper, Ph.D., and Alan S. Kopin, M.D., their project will study the abnormal processing of RNA molecules which provide instruction to neurons - a hallmark of amyotrophic lateral sclerosis (ALS).
“Our work is critical in helping expedite the development of targeted drug therapies that can reverse abnormal molecule processing, ” Dr. Kopin said. “We are grateful to the funders and supporting community.”
AUTTX will build on research that has identified a mechanism that can reverse abnormal RNA processing. AUTTX already discovered ASOs that stimulate this repair mechanism. To expedite the development of drugs targeting this pathway, AUTTX will screen a selected library of small molecules. Such compounds will shortcut the development process leading to novel therapeutics for people living with ALS.
Sporadic ALS – Understanding it’s Cause and the Development of Potential Therapies
Amprion is a global leader advancing diagnosis of neurodegenerative disorders through seed amplification testing. The San Diego-based diagnostics company seeks to understand the cause of sporadic ALS and actively assists in the development of potential therapies by supporting clinical and laboratory research.
Led by Richard Smith, MD, State Director of the Center for Neurologic Study and Russ Liebovitz, MD, PhD, co-founder of Amprion, the project has already convened leading physicians and scientists who published a landmark paper in the European Journal of Neurology earlier this year. The study found that approximately 15% of patients with a sporadic form of the disease had Lewy Body co-pathology as measured by Seed Amplification Assay (SAA), a test developed by Amprion that detects aggregates associated with misfolded alpha-synuclein which has been shown to correlate with Lewy Body pathology at autopsy.
“Dr. Smith and the Amprion team have already unearthed a subgroup of ALS patients in which misfolded alpha-synuclein is detected and likely contributes to disease symptoms and possibly progression, ” Lebovitz said. “This grant will fund additional work as we seek to correlate the contribution of a-synuclein to clinical features such as rapid progression, Parkinson's symptoms, sleep disorders, or cognitive decline.”
According to Smith, “a confirmatory study would be a compelling basis for developing a treatment strategy for this variant of ALS.”
Additional work spearheaded by Amprion will include the development of laboratory tests for the detection of other misfolding proteins associated with ALS pathology. Noteworthy will be the inclusion of neurologists who will determine if the clinical features of ALS are different in patients where a-synuclein and other misfolded proteins are found.