Research Matters.

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.

Project ALS Therapeutics Core at Columbia Logo

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.

Feb 2022
Prosetin Clinical Trial Begins
Prosetin Clinical Trial Begins

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.

Oct 2021
Research Advisory Board
New RAB Announced

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

Apr 2021
Jacifusen Clinical Trial Announced
Jacifusen Phase III Clinical Trial

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.

Mar 2020
Medidata Partnership
Project ALS and Medidata Institute

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.

Apr 2019
The Core Announced
Project ALS Therapeutics Core

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.

Nov 2017
Mass Gen Living Library
Project ALS Establishes Living Library At Mass General Hospital

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.

2016
Autophagy in ALS
Autophagy Team Breaks Through

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.

Jan 2010
P2ALS Begins
Partnership with Packard Center, Doctoroff & Bloomberg

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.

2008
Patient-Derived Motor Neurons
Motor Neurons from ALS Patient Skin

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.

2006
Jenifer Estess Stem Cell Lab Opens
Project ALS/Jenifer Estess Laboratory for Stem Cell Research opens

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.

2001
Stem Cells Discovery
Mouse Stem Cells to Functional Motor Neurons

Scientists at Columbia University differentiate mouse stem cells into functional motor neurons, the very brain cells destroyed in ALS.

1999
Pivotal Breakthroughs
Assays and Stem Cell Studies

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.

Latest News from the Core

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.

10/20/2022

Project ALS Research Live: A Family Effort

Valerie was joined by Kathy and Richard, members of the Weber family (NextGen ALS)—which has been afflicted by a genetic form of ALS (CHCHD10) for generations—and Drs. Giovanni Manfredi and Hibiki Kawamata Fujita from Weill Cornell Medicine to talk about our effort to develop therapies for genetic ALS and what this could mean for all forms of the disease.

09/20/2022

Project ALS Research Live: An Update On Prosetin​

Erin and Valerie are back to give an update on prosetin—the first potential new drug under investigation for the treatment of ALS to emerge from the Project ALS Therapeutics Core at Columbia—that Project ALS moved to Phase I of FDA clinical trials earlier this year. They are joined by Dr. Hynek Wichterle, scientific co-director of the Core, and Stan Abel, president and CEO of ProJenX.

11/02/2021

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.

Help Us Fuel the Core Research Engine

Groundbreaking ALS Research Is Happening Right Now Because of You.

Core Leadership

Scientific Codirectors

Serge Przedborski, MD, PhD
Neil Shneider, MD, PhD
Hynek Wichterle, PhD

Director of Operations

Emily Lowry, PhD

Unit Directors

Jinsy Andrews, MD, MSc
Estela Area Gomez, PhD
Susan Brenner Morton
Francesco Lotti, PhD
Emily Lowry, PhD
George Z. Mentis, PhD

Director of Research

Valerie Estess

Associate Director of Research Operations

Margot Shanahan

Research Consultant

Erin Fleming

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