Non-profit Partnership Spurs Phase 3 Clinical Trial for Patients With Rare Genetic Form Of ALS


ARLINGTON, Va. and NEW YORK, April 8, 2021 /PRNewswire/ -- The ALS Association and Project ALS are pleased to announce that their jointly sponsored pilot program has resulted in this week's announcement by Ionis Pharmaceuticals, Inc. of FUSION, a 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.

In February 2020, the two ALS non-profits joined forces to support a pilot clinical research program—led by Neil Shneider, MD, PhD, director of the Eleanor and Lou Gehrig ALS Center at Columbia University Irving Medical Center—which allowed several patients with this rare and aggressive form of ALS to receive treatment with jacifusen.

"The Ionis trial of ION363 is evidence that by working together at a very early stage, non-profits that are passionate about treating and curing ALS can pay forward more and better opportunities for patients," said Kuldip Dave, PhD, Vice President of Research, The ALS Association. "This pilot program with Project ALS is part of a larger strategy toward providing treatments for all genetic forms of ALS, including rare types."

The ION363 trial has its deepest roots in Valentine's Day, 2019, when Jaci Hermstad, a 25-year-old Iowa woman, was diagnosed with FUS-ALS, eight years after losing her identical twin sister to the same disease at age 17. The Hermstad family contacted Project ALS and Dr. Shneider, who had been investigating the potential of ASOs in treating patients with FUS-ALS, to ask if there were any therapies that might help Jaci.

"The Hermstads' story became an instant magnet for so many talented and caring people, from Ionis and Dr. Shneider, an expert in the FUS gene, to volunteer regulatory experts, manufacturers, and academic advisers," said Valerie Estess, Project ALS director of research. "Jaci's courage—and the teamwork she inspired—can now pay off for all patients with FUS-ALS.

Funding from the ALS Association and Project ALS made it possible for Dr. Shneider and his team at Columbia to offer jacifusen to ten additional FUS-ALS patients over the last two years while tracking safety data on the drug and disease-relevant biomarkers in parallel. "The ALS Association and Project ALS came together at a critical moment, allowing us to learn more about the safety and potential efficacy of jacifusen," said Dr. Shneider, who will lead the Phase 3 trial. "I believe that their steadfast support of this early-stage clinical research provided Ionis with the data needed to further develop jacifusen in the current pivotal trial."

"Jaci Hermstad's spirit inspired us from the start," said C. Frank Bennett, PhD, Ionis' chief scientific officer and franchise leader for neurological programs. "Ionis will take this program forward because everyone involved in this experience shares the same goal: better medicine for ALS and other devastating neurological illnesses."

About The ALS Association
The ALS Association is the largest private funder of ALS research in the world. The Association funds global research collaborations, provides assistance for people with ALS and their families through its nationwide network of chapters and certified clinical care centers, and advocates for better public policies for people with ALS. The ALS Association builds hope and enhances quality of life while urgently searching for new treatments and a cure. For more information about The ALS Association, visit

About Project ALS
Project ALS identifies and funds the most promising scientific and medical research that will lead to the first effective treatments and a cure for ALS. We recruit the world's best scientists and doctors to work together—rationally and aggressively—to develop a better understanding of the ALS disease process and, in parallel, better therapeutic strategies. To learn more about Project ALS, visit and follow on Twitter @projectALSorg.

About The Eleanor and Lou Gehrig ALS Center
The Eleanor and Lou Gehrig ALS Center at Columbia University provides comprehensive clinical care to ALS patients and families. We are an ALS Association Certified Treatment Centers of Excellence Center and an MDA/ALS Care Center. In addition, The Eleanor and Lou Gehrig ALS Center leads an integrated ALS research program at Columbia University that spans basic, translational, and clinical efforts throughout our institution. Through a greater understanding of disease mechanisms, our efforts focus on the development of novel therapeutics for individuals and families with ALS. For more information about the Center, visit

About Ionis Pharmaceuticals  
For more than 30 years, Ionis has been the leader in RNA-targeted therapy, pioneering new markets and changing standards of care with its novel antisense technology. Ionis currently has three marketed medicines and a premier late-stage pipeline highlighted by industry-leading neurological and cardiometabolic franchises. Our scientific innovation began and continues with the knowledge that sick people depend on us, which fuels our vision of becoming one of the most successful biotechnology companies. To learn more about Ionis visit and follow on Twitter @ionispharma.

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