Project ALS And Columbia University Announce New Screening Platform For ALS Therapeutics

Seven-Unit Core Positioned to Push Most Promising Drug Candidates Directly to Patient Trial

New York, NY /PRNewswire/ - Columbia University and Project ALS today announced the Project ALS Therapeutics Core at Columbia, a 3-year, $6.3M initiative toward the first meaningful therapies for ALS. The Core is the world's first and only partnership between a world-class academic institution and a leading nonprofit organization dedicated to a full-spectrum approach to ALS drug development, preclinical evaluation, and human clinical trials.

Core scientific directors Serge Przedborski, MD, PhD, Neil Shneider, MD, PhD, and Hynek Wichterle, PhD (Columbia), will coordinate the seven-unit effort to integrate drug candidates emanating from academic laboratories and industry with clinical research at the Eleanor and Lou Gehrig ALS Center. The goal is better clinical trials—and the first effective treatments for people with ALS, a uniformly fatal neurodegenerative disease closely related to Alzheimer's, Parkinson's, and Huntington's diseases.

The Core unites world-leading researchers and physicians to set a new standard for ALS therapy development. By applying recent discoveries in ALS genetics and biology, novel stem cell techniques, and cutting-edge technologies toward a comprehensive, efficient approach to identifying and evaluating potential ALS drugs, the Core will advance in-house therapeutic candidates and partner with industry to test rational drugs for ALS—and move validated therapies to people as quickly as possible. Already, the Core has yielded a novel drug, and evaluated dozens of commercial compounds in partnership with pharmaceutical companies.

The Core builds on two decades of Project ALS-funded breakthroughs at world-leading research institutions. "It's time for drugs that work," said Valerie Estess, Director of Research at Project ALS. "For twenty years, Project ALS researchers have built tools that allow us to screen potential drugs more efficiently. The Core is the first attempt to use all the validated tools under one roof, at Columbia, which is arguably the epicenter of neuroscience. This is our best shot to make a big difference right now."

The Core begins and ends with ALS patients—it will utilize patient blood samples toward drug screening, biomarker discovery, and genetics studies, and deliver better therapeutic options back to the clinic, to patients who have participated at the start of the process.

"For the first time, ALS patients can directly participate in research that will move us toward therapies that actually work," said Neil Shneider, MD, PhD, Director of the Eleanor and Lou Gehrig ALS Center at Columbia. "The Core provides an immensely exciting opportunity to capitalize on decades of ALS advances and translate them into meaningful treatments now."

About The Project ALS Therapeutics Core at Columbia
The Project ALS Therapeutics Core at Columbia aims to bring the first meaningful treatments for ALS from the lab bench to patients, rationally and efficiently. Under the scientific leadership of Drs. Serge Przedborski, Neil Shneider, and Hynek Wichterle, the Core integrates seven research groups—clinical research; lipidomics; gene therapy; antibody development; in vitro screening; in vivo evaluation; and in vivo electrophysiology—at Columbia's Motor Neuron Center and Eleanor and Lou Gehrig ALS Center in a world-class, patient-focused approach to ALS therapy development. For more information about the Core, contact Erin Fleming: erin@projectals.org.

About Project ALS
Jenifer Estess, her sisters and friends, started Project ALS in 1998, when Jenifer was diagnosed with ALS. Project ALS shifted the paradigm of ALS research, requiring its funded researchers and doctors to work together in small teams, toward a new standard of results-oriented accountability. In twenty years, Project ALS has overseen productive research collaborations among 25 leading institutions leading to the discovery of over 60 ALS genes, the development of the world's first patient-based models of ALS, and now, the acceleration of ALS drug testing and clinical trials. For more information, visit projectals.org.

About the Motor Neuron Center
The Motor Neuron Center, based at Columbia University Medical Center, brings together top scientific minds to understand what happens in ALS and related motor neuron diseases. By taking new leads and tools from motor neuron biology, and testing and applying them to animal models of disease, as well as to clinical research in people, the Motor Neuron Center facilitates the speedy transfer of research and preclinical findings to patients in the hope of finding effective therapy. For more information, visit http://www.columbiamnc.org/.

About the Eleanor and Lou Gehrig ALS Center
The Eleanor and Lou Gehrig ALS Center provides comprehensive care to individuals and families with ALS, PLS and related forms of motor neuron disease (MND). The Center's multi-disciplinary team of ALS physicians and associated healthcare professionals offers compassionate care to ALS/MND patients and families, and provides the resources necessary to meet the evolving challenges of living with motor neuron disease. Research activities— including genome analysis and clinical trials— are integrated into the ALS Center clinics to give individuals the opportunity to participate in the fight against ALS/MND. For more information, visit http://www.columbianeurology.org/patient-care/als-and-related-motor-neuron-diseases.