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 It’s time for ALS drugs that work

We’ve come a long way in our understanding of ALS—but there are still no treatments that make a meaningful difference in this devastating disease.

The Core leverages the best of non-profit advocacy and world-class academic research toward better ALS drug development. Founded and funded by Project ALS, The Core integrates world-leading neuroscience expertise at Columbia’s Motor Neuron Center with patient-driven clinical research at the Eleanor and Lou Gehrig ALS Center to develop new ALS therapies, evaluate promising drug candidates comprehensively and efficiently, and move top performers to people as quickly as possible.

The Core begins and ends with ALS patients. We rely on blood samples to develop better disease biomarkers and cellular models for drug screening—and we will deliver more humane and informative clinical trials back to people with ALS.

“For the first time, ALS patients can directly participate in research that will move us toward therapies that actually work…The Core provides an immensely exciting opportunity to capitalize on decades of ALS advances and translate them into meaningful treatments now.”

– Neil Shneider, MD, PhD, Director of the Eleanor and Lou Gehrig ALS Center at Columbia

Structure

Clinical Research Unit
Director: Jinsy Andrews, MD, MSc

Access to customizable source of patient samples for preclinical studies
Development and execution of early-phase clinical studies in carefully characterized ALS patient population

Electrophysiology Unit
Director: George Z. Mentis, PhD

Characterize physiological, synaptic,
and motor circuit defects in the Core’s
ALS animal models

In Vivo Evaluation Unit
Director: Emily Lowry, PhD

Non-regulatory evaluation of safety, solubility, stability, CNS penetrance, PK and toxicity of candidate therapies
Optimization of compounds exhibiting strong neuroprotection but insufficient CNS penetrance

In Vitro Screening Unit
Director: Emily Lowry, PhD

Robust human iPSC-derived motor neuron screening for high-throughput screening for potential ALS drugs with neuroprotective effects
Analysis includes quantification of motor neuron survival, morphology, cell body size, neurite outgrowth, and other parameters

Viral Vector Unit
Director: Francesco Lotti, PhD

Viral vector production for novel genetic targets in ALS
Development of virally-mediated constructs for relevant animal models of ALS for use as positive controls

Antibody Unit
Director: Susan Brenner Morton

Novel antibody development for research and post-mortem sample examination
Custom antibody resource for pharmacodynamic target validation

Neurolipidomics Unit
Director: Estela Area Gomez, PhD

Profile lipid signature in ALS patient biofluids to assess value as potential diagnostic, prognostic, and therapeutic ALS biomarker, and implement parallel lipidomic assessments for ALS efficacy studies

 THE TEAM

 World-Leading ALS Research

Columbia is an epicenter of ALS discovery—from basic neuroscience to cutting-edge clinical research.

The Core unites Columbia’s ALS experts—scientists and doctors who are attacking the disease from all angles—to focus their efforts on establishing better therapeutic options for people with ALS.

 Built for Collaboration

Our goal is that anyone interested in ALS drug development can easily gain access to The Core’s ALS expertise and novel disease models.

The Core has a flexible, cost-efficient collaboration model for industry and academic partners. Learn more about working with us here.