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In 1998, not much was known about ALS basics. Project ALS set out to learn the basics—how and why ALS happens—so that we could go after ALS with better drugs. For two decades, Project ALS invested in hardcore basic research that led to the identification of 50 new ALS genes…better drug screening technologies…and relevant therapeutic targets to hit with drugs. We worked hard, built an impressive list of firsts, and opened the black box of ALS
Today the Project ALS Therapeutics Core (the Core), a network of academic and industry researchers, is translating the ALS basics into potential therapies for patients. For example, based on our growing knowledge of certain types of cellular stress involved ALS, the Core has generated new candidate therapies to hit specific cell stress pathways. If we hit the target, we may slow down. The Project ALS Therapeutics Core has been productive. The Core’s first in house drug, prosetin, developed specifically for the treatment of ALS, is currently in ALS clinical trial. The Core is building on the promise of prosetin to develop screening tools and effective therapies (link to Core progress and current projects)
Project ALS is a family affair. What started off as an effort among sisters has grown to the hundreds of thousands, to include mothers and fathers, scientists and neurologists, Nobel Laureates and gifted young researchers, children and friends—we are all in this together.
The all-new drug developed by the Project ALS Therapeutics Core for the treatment of ALS, is now moving into Phase 1 trial for people with ALS in Canada.
For more information on the trial, please contact [email protected]
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.
Researchers at the Project ALS/Jenifer Estess Laboratory for Stem Cell Research, Columbia University, and Harvard University derive motor neurons from ALS patient skin. This achievement is later named Time magazine’s Medical Breakthrough of the Year.
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 all-new drug developed by the Project ALS Therapeutics Core for the treatment of ALS, is now moving into Phase 1 trial for people with ALS in Canada.
For more information on the trial, please contact [email protected]
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.
Researchers at the Project ALS/Jenifer Estess Laboratory for Stem Cell Research, Columbia University, and Harvard University derive motor neurons from ALS patient skin. This achievement is later named Time magazine’s Medical Breakthrough of the Year.
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.
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