Let’s be honest, for the most part, experts have failed to deliver potent drugs to clinical trial in ALS. Part of the reason is we haven’t known enough about the genes contributing to ALS…the cellular pathways involved in ALS…or when or how to intervene effectively in the disease process, to truly make a difference. That has all changed.
The newly minted Project ALS Pre-Clinical Core at Columbia University draws on decades and up-to-the-minute ALS breakthroughs throughout genetics, medicinal chemistry, drug screening platforms, disease modeling, drug delivery to the brain, and basic neuroscience research, to identify promising ALS therapies before they ever reach clinical trial.
The Core, as it is known, has been functioning as a virtual drug-screening effort for nearly eight years. Now it’s official—the Project ALS Pre-Clinical Core at Columbia’s Motor Neuron Center, is currently the best available opportunity for drug companies and academic laboratories to test potential ALS drug candidates thoroughly and comprehensively, under one roof. The mission of the Core is to deliver its top performers toclinical trial at the Columbia ALS Clinic, and beyond.
Directed by Neil Shneider, MD, PhD, Serge Przedborski, MD, PhD, and Hynek Wichterle, PhD, the Core comprises five interrelated units: in vitro testing, in vivo testing, custom antibody and viral vectors, neurolipidomics, and a clinical research nurse. Core operations are co-directed by Erin Fleming and Emily Rhodes Lowry, PhD.
The Core is unique among ALS drug screening efforts for its ability to assess drugs in all stages of pre-clinical development. For example, the molecule kenpaullone, a molecule whose therapeutic promise has been known to academic researchers for years, is finally getting its work-over for ALS. Originally deemed a slow track project for the Core, kenpaullone has emerged as a strong finisher…and a clue of intense interest. The Core has also assessed four FDA approved drugs for other indications that may also be useful in ALS. Project ALS and the Core will continue to work with drug companies and academic laboratories to identify additional FDA drug candidates for systematic Core assessment. Finally, the Core is developing novel therapeutics for ALS. Initial efforts to develop all-new approaches look encouraging.
Project ALS is grateful to neurodegenerative disease research, patients, and the growing family of supporters that will beat ALS, for allowing us to build a systematic approach to ALS drug screening based on decades of love, loss, teamwork, and breakthroughs.
The current issue of Neuroscience journal is dedicated to the staggering contributions to science made by longtime Project ALS Research Advisory Board Chair Thomas M. Jessell, PhD, before his death from the rare neurodegenerative disease progressive supranuclear palsy on April 28, 2019, at 67.