There is an urgent need for new and improved therapies for ALS, as there is still no cure. To help expand the drug development pipeline, we are proud to support the preclinical assessment of emerging ALS therapies through The Lawrence and Isabel Barnett Drug Development Program.
This program, previously known as TREAT ALS, was renamed The Lawrence and Isabel Barnett Drug Development Program in 2017 to honor the prominent entertainment executive and television and theater actress for their numerous contributions to the Association and their impact on ALS research. Carrying on their parents’ legacy, the Barnett children and the Barnett Family Foundation continue to invest in this innovative program that leverages academic scientific expertise with industry know-how to drive novel therapeutics into clinical trials for ALS and related neurological disorders
Awardees receive up to $500,000 over two years to support the preclinical assessment of new and repurposed ALS therapies in preparation for clinical testing. This program prioritizes projects that have a high probability of reaching the clinic within three years.
Impact of the Program
Investment at the preclinical stage drives the crucial transition from academic research project to industry drug development effort. This stage in the drug development pipeline is often underfunded because it is too late for traditional academic funding mechanisms and too early for most industry funding mechanisms. By funding projects at this stage, The Barnett Drug Development Program helps fill this gap and has contributed to the expansion of companies and therapeutics entering ALS clinical trials.
Between 2016 and 2021, we committed more than $10 million through The Barnett Drug Development Program to support 32 projects. Twenty-one of these projects have either attracted new partners to help advance the program or have received follow-on funding from larger funders, such as the National Institutes of Health, Department of Defense ALS Research Program, pharmaceutical/biotech companies or venture capitalists.
Success Stories
- Ionis Pharmaceuticals and Biogen in collaboration with researchers at University of California San Diego and Johns Hopkins University developed antisense therapy against SOD1 and C9orf72, the two most common genetic causes of ALS. Tofersen, which emerged from this research to treat SOD1 ALS, is scheduled to be evaluated by the FDA in spring 2023.
- Dr. John Landers at the University of Massachusetts Chan Medical School conducted key preclinical studies of pridopidine that showed this drug prevented neuronal cell death and strengthened connections between neurons. Pridopidine is now in phase 2/3 clinical testing through the HEALEY ALS Platform Trial.
- Research by Dr. John Ravits at the University of California San Diego demonstrated the potential of a genetic modifier called ataxin-2 as a treatment target for ALS. Dr. Ravits is now helping test this approach as part of Biogen’s phase 1/2 clinical trials of the drug BIIB105.
- Dr. Justin Ichida at the University of Southern California completed preclinical investigations into using PIKfyve inhibitors to treat ALS caused by mutations in the C9orf72 gene. This laid the foundation for a new class of treatments being developed by companies like Acurastem, Verge Genomics and AI Therapeutics.
- Researchers at the University of Oxford, Structural Genomics Consortium at the Montreal Neurological Institute at McGill University, and the Karolinska Institute joined together to create an open-access research antibody validation pipeline, providing the ALS research community with the highest quality antibodies for ALS genes.
