The quest to develop a new treatment approach for amyotrophic lateral sclerosis (ALS) may soon be shortened because of 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 therapies. Insilico’s research is being conducted in collaboration with Answer ALS, a global research project focused on 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 highly reliable 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, “greatly rescue neurodegeneration through their suppression.” The findings of this study were published in Frontiers in aging neuroscience†
“The results of this collaborative research effort show what is possible when we bring human expertise together with AI tools to discover new targets for diseases that have a high unmet need,” said Alex Zhavoronkov, founder and CEO of Insilico in a statement. . “This is just the beginning.”
ALS, also known as Lou Gehrig’s disease, is a progressive neurodegenerative disease that negatively affects neurons in the brain and spinal cord. Patients with ALS quickly lose the ability to control muscle movements. This eventually leads to total paralysis and then death. It is estimated that about 12,000 to 15,000 Americans have ALS, with about 5,000 to 6,000 new cases every year.
currently available drugs, including Riluzole and Mitsubishi Tanabe’s Radicava Don’t Stop or Reverse Function Loss in ALS Patients.
Feng Ren, co-CEO and Chief Scientific Officer of Insilico, said the new therapeutic targets demonstrate the power of the PandaOmics platform.
“It’s impressive that about 70% (18 out of 28) targets identified by AI were validated in a preclinical animal model. We are working with collaborators to achieve a number of goals toward clinical trials for ALS. At the same time, we are also expanding the use of PandaOmics to discover new targets for other areas of disease, including oncology, immunology and fibrosis,” Ren said in a statement.
Insilico has not announced its plans for using the data, but there are likely a number of companies choosing to approach these potential ALS targets. Several companies, such as Amylyx Pharmaceuticals, NeuroSense and QurAlis, are working on possible 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 advisory committee to re-evaluate the drug candidate following the release of additional clinical data.