what is the core?

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.

CORE ACCOMPLISHMENTS

THE CORE has made significant progress toward better therapeutic options for ALS. These accomplishments are increasing the number and testing the feasibility of potential ALS therapies, improving possible diagnostic tools in the clinic, and expediting the transition of promising drug candidates into patient populations. Among its achievements, CORE researchers have:

  • Tested >1700 chemical compounds and FDA approved drugs for other indications in our in vitro screening unit,
  • Collaborated with >20 other academic groups and biotechs to assess their ALS compounds of interest in a range of pre-clinical models,
  • Developed a novel blood-based biomarker for ALS, which we are now validating for diagnostic and prognostic purposes,
  • Moved two potential ALS drugs, jacifusen and CK0801, to people with ALS, and
  • Developed an in-house drug, Prosetin, which can penetrate the brain through straightforward oral administration, measurably rescue stressed motor neurons in all of our ALS models, provably engage a cellular pathway of interest across neurodegeneration. 

“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

MILESTONES

2021

  • Project ALS receives permission from the US Food & Drug Administration
    to initiate Phase I clinical trial of prosetin
  • 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 
  • 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
     

2020

  • Prosetin receives Orphan Drug Designation from the US Food and Drug Administration
  • Project ALS launches research partnership with Medidata Institute targeting new, actionable insights into ALS disease progression and subtypes
  • Project ALS responds to challenges presented by the global COVID-19 pandemic and continues to move forward with new virtual fundraising efforts
ALS research

2019

  • Project ALS announces prosetin, the first investigational drug to emerge from THE CORE as a potential treatment for ALS
  • Jaci Hermstad receives first ever dose of jacifusen, a custom antisense oligonucleotide gene therapy to address Jaci’s genetic form of ALS
  • Project ALS launches a free and open to the public webinar series featuring a range of experts for accessible, conversational updates covering research, hot topics in ALS drug development, and stories of people with ALS

2018

  • Pre-Clinical Core identifies several drugs of promise for ALS
  • Project ALS celebrates 20th Anniversary. Over $90 million raised in 20 years with 89% going to research programs.
  • Researchers funded reaches 95 at 35 institutions worldwide
ALS research event fundraiser

2017

2016

  • Autophagy in ALS begins, a three-year study connecting researchers from Columbia, Cornell, UCSF, NYU and NY Genome Center
  • Salk Institute researchers show that brain cells called interneurons contribute to ALS
ALS researcher

2014

  • Don’t-Talk-a-Thon, the first Project ALS online fundraising campaign raises $600,000 and counting
  • Establish Mass General/Project ALS Internship Program
ALS awareness campaign

2013

  • 26 ALS genes now identified
ALS research

2012

  • RAB adds Nobel laureate as well as renowned experts from pharmaceutical/biotech, and neuroscience
ALS Seminar

2011

ALS Research

2010

  • Establish P2ALS, a $15 million partnership with Johns Hopkins, Dan Doctoroff and Michael Bloomberg
  • Fund first multi-lab study focused on selective vulnerability of motor neurons in ALS
Project ALS event

2009

  • Built first library of ALS patient cell lines, including those with genetic mutations
  • Develop “ALS In a Dish” – a helpful new tool for studying human ALS.
ALS disease research

2008

2007

  • Women & the Brain starts; a Project ALS-created initiative to raise awareness for brain health and support for talented neuroscientists and need-based medical students with a focus on neurology.
ALS panel

2006

  • Project ALS/Jenifer Estess Laboratory for Stem Cell Research opens, the world’s first and only privately funded lab to focus exclusively on stem cells and ALS.
  • Estess Lab team derives first functional human motor neurons from stem cells

2004

  • Launched largest-ever study in ALS genetics with Harvard Neurodiscovery Center
  • Premier of HBO Documentary Three Sisters: Searching for a Cure, an in-depth look at Jenifer Estess and Project ALS
  • Tales from the Bed, Jenifer’s memoir, becomes a national bestseller.
Tales from the Bed by Jenifer Estess

2003

2002

  • First ALS specific gene therapy experiments at the Salk Institute. Treated mice survive 33.3% longer than untreated group.
  • Original movie event Jenifer premiers on CBS and draws national attention to Project ALS
  • Jenifer Estess named a Glamour Magazine’s “Women of the Year”. Project ALS named Vanity Fair’s “It List” charity.
Jennifer Estess: 2001 Glamour Women of the Year

2001

2000

  • Jenifer Estess testifies before US Congress to the importance of federal funding for stem cell research in ALS
Stem Cell ALS Research Publicity

1999

  • Project ALS team at Mass General builds first standardized cell based-assay for rapid ALS drug testing
  • First to study stem cells and ALS. Pilot studies at Children’s Hospital Boston and Johns Hopkins.
  • Gala fundraisers in NY and LA unite entertainment and corporate communities
ALS research laboratory

1998

  • Jenifer Estess diagnosed with ALS at 35. Jenifer, her sisters, and friends start Project ALS as the first 501(c)3 nonprofit dedicated solely to ALS research.
  • Board of Directors and Research Advisory Board established
  • First $1 million raised for collaborative experiments among scientists at Harvard University, Johns Hopkins, and Columbia University.
Project ALS founding team

THE PROJECT ALS APPROACH

Twenty years ago, Project ALS changed the way scientists and doctors conducted ALS research. As a result, we are closer to the first effective treatments for ALS.

The Project ALS business model—now widely adopted–requires that leading ALS researchers work together, share data openly, and meet shared deadlines. Project ALS works collaboratively with its scientists to identify the most promising ALS research and, along with its Research Advisory Board (RAB), streamlines the research process with effective strategies. Project ALS assesses progress through regular meetings and reviews by the RAB.

Project ALS engages in the ongoing recruitment of innovators, not only from neuroscience, but diverse disciplines including stem cell biology, mathematics, computation statistics, chemistry, bioimaging, and industry, to contribute to the growing body of knowledge of what goes wrong in ALS—and how we will fix it. Project ALS provides research funding on a rolling basis, ever poised to support innovation.

RESEARCH NEWS