Prize4Life, a nonprofit organization whose mission is to accelerate the discovery of treatments and a cure for ALS (Amyotrophic Lateral Sclerosis, also known as Lou Gehrig’s disease), today announced the winner of its $1M Avi Kremer ALS Treatment Prize. The winners are the team of Drs. Martine Barkats and Maria-Grazia Biferi, from the Institute of Myology U974 INSERM UMPC in Paris, France. They have developed a gene therapy approach targeted at one of the most common known causes of inherited ALS. This approach significantly extends survival and slows disease progression beyond any results reported to date in the most frequently used ALS mouse model.
The ALS Treatment Prize was launched with the aim to accelerate discovery of a treatment for ALS by encouraging researchers from around the globe to test new candidate therapies. The prize criteria sought a treatment candidate that increased lifespan of SOD1 ALS mouse models by at least 25 percent (under specific experimental conditions) – significantly more than had ever been done before. SOD1, or superoxide dismutase, was the first ALS-causing gene to be identified, more than 20 years ago, and is mutated in approximately two percent of all ALS cases. The winning team’s approach to shutting down expression of SOD1 not only extended survival of treated adult mice by more than 50 percent (far above the 25 percent prize requirement), but also significantly improved the motor function of the mice even after they began to show symptoms. While SOD1 gene silencing therapies have been the focus of many research efforts in the past and present, the winning team’s approach is unique in its design, the mode of administration and its improved efficacy. The ALS Treatment prize was also designed to increase the likelihood of generating reproducible results between labs, a known challenge in biomedical research. At the close of the challenge, a repeated independent study conducted by the ALS Therapy Development Institute in Cambridge, Mass. confirmed the winning team’s results.
“We launched this prize with the goal of creating momentum around testing of new treatments for ALS, and are enthusiastic about the potential of the winning team’s solution. It far exceeds the survival threshold we laid out, and is beyond anything that’s been done before in this mouse model. We are hopeful these results will ultimately translate to humans,” said Dr. Nicole Szlezak, Chair of Prize4Life’s Board of Directors.