Our foundation is dedicated to supporting groundbreaking research in ALS, a cause that was deeply important to Dick. We are honored to share updates on the promising work being done at the Sean M. Healey & AMG Center for ALS at Mass General Brigham, where Dr. Brian Wainger, MD, PhD, and his team are making strides in identifying new treatment possibilities.
According to Alexandra Van Strien, who works with Dr. Wainger:
"ALS and Frontotemporal Disorder are likely caused by multiple pathological processes occurring at different locations on motor neurons. In particular, evidence suggests that distinct mechanisms underlie neuronal toxicity in the cell body, the control center of the neuron, and the axons that project outward from the cell body.
Sean M. Healey & AMG Center for ALS at Mass General Brigham researcher Brian Wainger, MD, PhD, developed screens using human neurons to identify drugs that target multiple molecular mechanisms of ALS at the same time. Two independent screens identified a family of compounds that includes an FDA-approved drug that can be evaluated further for repurposing.
The Wainger research group is studying this FDA-approved drug to better understand the mechanistic target of the drug’s benefit in ALS/FTD and reduce toxicity in animal models. The team is using genetic and pharmacological approaches, and this work will also open the door to antisense oligonucleotide and other genetic-based treatment modalities based on the drug’s mechanism of action.
The team is also working with a medicinal chemistry team to optimize the CNS entry of the compound. We already have identified core structural components of the molecule necessary for its activity, including one compound with efficacy at only 1 nM. The goal will be to develop an improved molecule with greater CNS entry and stronger potency.
If successful, we will pursue both the opportunity to repurpose the drug directly into human clinical studies and the development of new compounds with improved properties for drug development."
We are incredibly grateful for the work being done by Dr. Wainger and his team, and we look forward to sharing more updates as this research progresses. If you would like to learn more or support this critical research, please visit https://www.massgeneral.org/neurology/als