Expanding the Understanding of ALS With New Methods: Brian Wainger, MD, PhD


The assistant professor of neurology and anesthesiology at Harvard Medical School discussed non-traditional ways ALS research is changing and where the focus should continue to be.

"All of a sudden there’s more powerful tools in the hands of more people. Some of these tools are things that can be adapted and brought into clinic quickly. Overall, it’s a really exciting time for translational medicine in neuroscience and degenerative diseases in particular.”

Ezogabine, once FDA-approved for the treatment of adjunctive treatment for partial-onset seizures, was found in a recent study to decrease cortical and spinal motor neuron (MN) excitability in patients with amyotrophic lateral sclerosis (ALS). Strong clinical evidence that supported hyperexcitability as a prominent phenotype in both familial and sporadic ALS helped investigators move directly from pluripotent stem cell (iPSC) modeling to a clinical trial using neurophysiological metrics of MN excitability as pharmacodynamic biomarkers.

The uniqueness of going from iPSC modeling straight to a phase 2 clinical trial raises questions about the boundaries of researching ALS, and the outside-the-box thinking needed to counteract a disease that has a limited number of treatment options. Lead author of the trial Brian Wainger, MD, PhD, feels as though researchers need to find creative ways to try to better understand ALS, while keeping in mind the lengthy process that encompasses traditional drug development.

Wainger, assistant professor of neurology and anesthesiology, Harvard Medical School, sat down with NeurologyLive to detail how the study came about, and the types of strategies needed to grow the understanding of ALS and how it progresses.

Wainger BJ, Macklin EA, Vucic S, et al. Effect of ezogabine on cortical and spinal motor neuron excitability in amyotrophic lateral sclerosis: a randomized clinical trial. JAMA Neurol. Published online November 23, 2020. doi: 10.1001/jamaneurol.2020.4300
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