ALS, also known as Lou Gehrig’s disease, is a neurodegenerative disease that is closely related to Alzheimer’s, Parkinson’s, and Huntington’s. In ALS, as motor neurons die, a person progressively loses the ability to walk, speak, swallow, and breathe.
Project ALS is working to develop the first effective treatments.
Finding Cures at the Core
The Project ALS Therapeutics Core at Columbia (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. Our goal is to develop the first effective treatments, and ultimately, a cure for ALS.
Closing in On A Cure
Since 1998, Project ALS has led research across the fields of stem cell biology, genetics, drug screening, and clinical trials at over 30 leading academic institutions.
Having received permission from the US Food & Drug Administration to initiate a Phase I clinical trial of prosetin in 2021, the first people are dosed at Worldwide Clinical Trials in Texas. In collaboration with Medical Excellence Capital, Project ALS launches ProJenX, a clinical-stage biotechnology company developing novel, brain-penetrant therapies targeting biologically-defined pathways for the treatment of ALS and other debilitating brain diseases.
Project ALS announces new Research Advisory Board representing a range of experience and expertise across the fields of neuroscience, clinical research, drug development, and technology
Project ALS sponsored pilot program results in initiation of Phase 3 clinical trial of ION363 (also known as jacifusen)—a novel antisense oligonucleotide (ASO) for ALS patients with a mutation in the fused in sarcoma (FUS) gene—by Ionis Pharmaceuticals. In 2019, Jaci Hermstad received the first ever dose of jacifusen, a custom antisense oligonucleotide gene therapy to address Jaci’s genetic form of ALS. Read the full press release here.
Project ALS launches research partnership with Medidata Institute, targeting new, actionable insights into ALS disease progression and subtypes. This multidisciplinary research effort brings together leading academic researchers with Medidata’s cutting-edge technology toward understanding, and successfully treating ALS.
Having begun as the Pre-Clinical Core in 2017, the Project ALS Therapeutics Core at Columbia University (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. 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. Read the full press release here.
Project ALS kicked off the ALS Living Library at Massachusetts General Hospital. Dr. James Berry directs the efforts to catalog, characterize and share with researchers, data from samples donated by ALS patients. Learn more here.
Autophagy in ALS begins, a three-year study connecting researchers from Columbia, Cornell, UCSF, NYU and NY Genome Center. Drugs identified by the Autophagy Team are fed in to the Pre-Clinical Core for ALS Drug Testing at the Motor Neuron Center (Columbia). Learn more about their progress here.
Project ALS and the Robert Packard Center for ALS Research at Johns Hopkins University announced a $15 million partnership Dan Doctoroff and Michael Bloomberg to to advance ALS research exponentially over the next three years. Read the full press release here.
The Project ALS/Jenifer Estess Laboratory for Stem Cell Research opens. It is the world’s first and only privately funded lab to focus exclusively on stem cells and ALS. In this same year, the Estess Lab team derives the first functional human motor neurons from stem cells.
Scientists at Columbia University differentiate mouse stem cells into functional motor neurons, the very brain cells destroyed in ALS.
The Project ALS team at Mass General Hospital builds the first standardized cell based-assay for rapid ALS drug testing. Pilot studies in stems cells and ALS also begin at Children’s Hospital Boston and Johns Hopkins.
The Deep Dive
Want to learn more about Project ALS research? Catch up on episodes of Project ALS Research LIVE where we talk to special guests about critical ALS research.
Project ALS Research Live: ALS Drug Candidates, from Jaci Hermstad to Phase III Clinical Trial
Erin and Valerie are joined by special guests Dr. Becky Crean—Executive Director, Clinical Development at Ionis Pharmaceuticals—and Lori Hermstad—mother and ALS advocate—for a conversation on the power of advocacy and community in the drug development process.
Project ALS Research Live: An Update from THE CORE
Erin and Valerie welcome back Emily Lowry, PhD, Director of Internal Operations at the Project ALS Therapeutics Core at Columbia (THE CORE) for up-to-the-minute developments from THE CORE, and to answer your questions about how we utilize units of THE CORE to get better drugs to clinical trial for people with ALS.
Project ALS Research Live: Next Steps for Prosetin
Erin and Valerie are joined by ALS clinical and clinical trial specialist, Jinsy Andrews, MD, MSc, & regulatory consultant, Kevin Phelan, PhD, to discuss prosetin moving to Phase I clinical trial.
Serge Przedborski, MD, PhD
Neil Shneider, MD, PhD
Hynek Wichterle, PhD
Director of Operations
Emily Lowry, PhD
Jinsy Andrews, MD, MSc
Estela Area Gomez, PhD
Susan Brenner Morton
Francesco Lotti, PhD
Emily Lowry, PhD
George Z. Mentis, PhD
Director of Research
Associate Director of Research Operations