The ALS Therapy Development Institute (ALS TDI) and the Gladstone Institutes formed a research collaboration to speed the discovery of potential treatments for ALS.
About 30,000 people in the United States live with amyotrophic lateral sclerosis (ALS), or Lou Gehrig's disease, today and the global population of ALS patients is approximately 400,000. About 5,000 new cases of ALS are diagnosed in the United States each year with the average patient survives only two to five years following their diagnosis.
Under this new agreement, Gladstone will evaluate potential pharmaceutical compounds using a human model of ALS. Gladstone generated the model by transforming skin cells from ALS patients into induced pluripotent stem cells and then programming them into neurons. The technique builds on a discovery for which Shinya Yamanaka, M.D., Ph.D., a Gladstone senior investigator, won the 2012 Nobel Prize in Physiology or Medicine.
This particular iPS-based ALS model includes a gene mutation that produces TDP-43, a protein commonly found in most forms of ALS. Promising drug compounds that pass the initial evaluation process at Gladstone will be fast-tracked for preclinical testing at ALS TDI, which will assess the compounds for activity and efficacy in various mouse models of human neurodegeneration.
"The strong evidence that abnormal TDP-43 protein is involved in the development of ALS, coupled with models that may replicate ALS more faithfully than other tools, may speed development of therapies for the thousands of individuals diagnosed with this devastating disease," said Steve Finkbeiner, M.D., Ph.D., senior investigator at Gladstone and professor of neurology and physiology at the University of California, San Francisco.
Earlier this year, ALS TDI entered collaborations with Neurotune and Regenesance to investigate potential treatments for ALS. Biogen Idec, UCB, Aestus Therapeutics, MDA, and RGK Foundation also have partnerships with ALS TDI.
Gladstone is an independent and nonprofit biomedical-research organization with a mission to prevent, treat, and cure cardiovascular, viral, and neurological diseases. Gladstone is affiliated with the University of California, San Francisco.