PPP3CB

The quest to develop a new treatment approach to amyotrophic lateral sclerosis (ALS) may soon be shortened due to the discovery of new potential therapeutic targets by Insilico Medicine. Using a proprietary AI-driven target discovery engine called PandaOmics, New York-based Insilico found genes that could serve as potential targets for new therapeutics. Insilico’s research is being conducted in collaboration with Answer ALS, a global research project aimed at finding new therapies and a potential cure for ALS. The AI-driven discovery engine analyzed expression profiles of central nervous system samples from public datasets and direct iPSC-derived motor neurons (diMN) from Answer ALS. As a result, the study identified 17 high-confidence and 11 novel therapeutic targets. These targets were further validated in a model that mimics the most common genetic cause of ALS. Insilico said eight unreported genes, including KCNB2, KCNS3, ADRA2B, NR3C1, P2RY14, PPP3CB, PTPRC and RARA strongly “rescue neurodegeneration through their suppression.” The findings from this study were published in Frontiers in Aging Neuroscience. “The results of this collaborative research effort show what is possible when we bring together human expertise with AI tools to discover new targets for diseases where there is a high unmet need,” Alex Zhavoronkov, founder and CEO of Insilico said in a statement. “This is only the beginning.” ALS, also known as Lou Gehrig’s disease, is a progressive neurodegenerative disease that negatively affects neurons in the brain and the spinal cord. Patients with ALS rapidly lose the ability to control muscle movement. That eventually leads to total paralysis and then death. It is estimated that approximately 12,000 to 15,000 Americans have ALS, with about 5,000 to 6,000 new cases diagnosed annually. Currently available drugs, including Riluzole and Mitsubishi Tanabe’s Radicava, do not halt or reverse the loss of function in ALS patients. Feng Ren, co-CEO and chief scientific officer of Insilico, said the new targets for therapeutics demonstrate the power of the PandaOmics platform. “It is impressive that around 70% (18 out of 28) targets identified by AI were validated in a preclinical animal model. We are working with collaborators to progress some targets toward clinical trials for ALS. At the same time, we are also further expanding the utilization of PandaOmics to discover novel targets for other disease areas including oncology, immunology and fibrosis,” Ren said in a statement. Insilico did not announce its plans for the data’s use, but there are likely a number of companies that may opt to approach these potential ALS targets. Several companies, such as Amylyx Pharmaceuticals, NeuroSense and QurAlis are working on potential treatments for this devastating disease. Amylyx's ALS asset, AMX0035, authorized for use in Canada, will be revisited by a U.S. Food and Drug Administration Advistory Committee to re-evaluate the drug candidate following the publication of additional clinical data.