Project ALS and Columbia University are launching a new platform with a goal of discovering more useful treatments for amyotrophic lateral sclerosis (ALS) patients, and pushing the most promising candidates to clinical trials.
Core, as it is called (officially, the Project ALS Therapeutics Core at Columbia), is a three-year, $6.3 million initiative that hopes to promote “better trials” and to find “the first meaningful therapies for ALS,” the two partner institutions announced in a press release.
“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,” she said. “This is our best shot to make a big difference right now.”
The initiative is the first joining an academic institution and a nonprofit organization and will span the full process of ALS therapy development, from discovery to preclinical evaluation and clinical trials in patients. The seven-unit platform will test in-house therapeutic candidates and team up with pharmaceutical companies to test potential treatments.
Most promising and core-validated candidates will be evaluated in clinical trials, with the help of the Eleanor and Lou Gehrig ALS Center, a clinic dedicated to motor neuron diseases at Columbia’s Department of Neurology.
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The Core brings together world-leading researchers and physicians “to set a new standard for ALS therapy development,” the release said. It builds upon recent discoveries in ALS genetics and biology, and makes use of novel technologies, including stem cell techniques, to identify and evaluate potential therapies. It also integrates seven research groups: clinical research, lipidomics (the large-scale study of lipids, or fatty acids), gene therapy, antibody development, in vitro (lab dish) screening, in vivo (in a living body) evaluation, and in vivo electrophysiology.
It all “begins and ends with ALS patients,” the two institutions said, noting that patient blood samples will be used to screen for treatments, look for novel biomarkers, and perform genetic studies.
“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, and the center’s director. “The Core provides an immensely exciting opportunity to capitalize on decades of ALS advances and translate them into meaningful treatments now.”
Core scientific directors, all from Columbia University, are Serge Przedborski, MD, PhD; Hynek Wichterle, PhD; and Shneider.