Dr. Neil Shneider Honored with 2023 Richard Mayeux Award

October 12, 2023
Neil A. Shneider, MD, PhD

Neil A. Shneider, MD, PhD

Named after accomplished physician-scientist Dr. Richard Mayeux, professor and chair of the Department of Neurology in the Columbia University Vagelos College of Physicians and Surgeons (VP&S), the annual Richard Mayeux Award honors a current faculty member who has made truly notable contributions to the clinical, research, and educational mission of the department over the past academic year. A committee of peers has selected Neil Shneider, MD, PhD, Claire Tow Associate Professor of Neurology for this distinguished honor.

Dr. Shneider is a graduate of Harvard College and of the MD-PhD Program at Columbia. He is a physician-scientist with particular expertise in neuromuscular development and disease. As director of the Eleanor and Lou Gehrig ALS Center at New York-Presbyterian/Columbia University Irving Medical Center, Dr. Shneider specializes in the diagnosis and care of patients with amyotrophic lateral sclerosis (ALS) and related motor neuron diseases. Under the leadership of Dr. Shneider, the ALS Center, which was founded in 1987 by former Neurology Chair Dr. Lewis “Bud” Rowland, has developed into one of the leading comprehensive ALS care programs in the nation, serving the complex needs of ALS patients and families from the Greater New York Area and beyond.

As a principal investigator in the VP&S Motor Neuron Center at Columbia, Dr. Shneider’s laboratory focuses on the development and characterization of novel animal and cellular models of ALS for the purpose of studying disease mechanisms and testing the efficacy of candidate therapeutics. Over the last 10 years, Dr. Shneider has focused much of his research effort on rare forms of ALS caused by mutations in the gene FUS. Working with Ionis Pharmaceuticals, Dr. Shneider developed an antisense oligonucleotide (ASO) therapeutic to target FUS, and petitioned the FDA tpapprove a compassionate-use, expanded access Investigational New Drug (IND) for a​​​​​​​ young patient with a rare form of juvenile ALS caused by a de novo FUS mutation. “What began as an n-of-1 effort grew to an expanded access treatment program involving 18 ALS-FUS patients, and ultimately to a pivotal phase 3 trial of the FUS ASO that became known as jacifusen in honor of the first patient treated with this antisense therapeutic,” explains one of Dr. Shneider’s nominators. “This bench-to-beside effort by an individual clinician investigator is a model of precision medicine that will bring promising therapeutics into the clinic for patients with ALS and many disorders, both rare and common.”

Building on the FUS ASO program, Dr. Shneider worked in partnership with the n-Lorem Foundation to establish the Silence ALS Initiative, a non-profit effort to design and develop optimized and individualized ASOs for patients with ultra-rare, genetic forms of ALS, extending this promising therapeutic technology to individuals and families for whom a commercial drug development program is not possible. Silence ALS recently received funding from the Ultra-rare Gene-based Therapy (URGenT) Network of the National Institute of Neurological Disorders and Stroke (NINDS), which supports the development of state-of-the-art gene-based therapies for ultra-rare neurological diseases.

Within the ALS Center, Dr. Shneider also leads the ALS Registry and Biorepository, the Target ALS-funded Postmortem Tissue Program and ALS Longitudinal Biofluids Program at Columbia, as well as the ALS Families Project – a longitudinal study of asymptomatic carriers of rare, ALS gene mutations - all of which further the ALS Center’s efforts to enable studies of disease mechanisms in sporadic and familial forms of ALS. “Together, these programs have made patient-derived resources available to academic and industry investigators as a human model to study the pathobiology of ALS, and to develop therapeutic targets and strategies to alter the natural history of this disease,” explains a colleague.

“Building on the clinical strengths of the ALS Center, [Dr. Shneider] has worked to combine the clinical and translational research programs of the ALS Center with the basic research efforts of the Center for Motor Neuron Biology and Disease at Columbia, resulting in an integrated ALS research program focused on the development of novel therapeutics based on the understanding of the genetics and biology of ALS. For these efforts, he is a most deserving candidate for this high honor,” according to a member of the nominating committee.

The faculty and staff of the Department of Neurology could not agree more and congratulate Dr. Shneider on his many remarkable achievements!