February 12, 2020
Pilot program, at Columbia University, is first step in strategy to expand access to treatments for rare ALS subtypes
Washington, DC, and New York, NY, Feb. 12, 2020--The ALS Association and Project ALS today announced a joint two-year, $900,000 commitment to fund a clinical research study at Columbia University Irving Medical Center of an experimental drug for patients with a rare genetic form of ALS. The program is a key step in developing a comprehensive program to treat rare forms of ALS associated with gene mutations.
The drug, called jacifusen, is an investigational therapy for patients with mutations in a gene called FUS. Such gene mutations cause some of the most aggressive forms of ALS, including a type that begins in adolescents and young adults.
To date, three ALS patients with FUS mutations have been treated with jacifusen under FDA-approved protocols. (The protocols were supervised by Neil Shneider, MD, PhD, Director of the Eleanor and Lou Gehrig ALS Center at Columbia University Irving Medical Center.)
The new clinical research program will allow an additional eight people with similar mutations to be treated with jacifusen.
Jacifusen is named for Jaci Hermstad, the 26-year old Iowa woman who spurred its rapid development after she was diagnosed with FUS-ALS in February 2019. Hermstad lost her identical twin sister, Alex, to the same form of ALS in 2011, at age 17.
Because FUS mutations are so rare, efforts to develop treatments for more common genetic forms of ALS have not been applied to FUS-ALS patients.
"While it emerged from a tragedy, jacifusen represents teamwork at its best," said Valerie Estess, director of research for Project ALS. "Doctors, drug companies, regulators, the Hermstad family, and Project ALS came together with the same goal, and less than four months after her ALS diagnosis, Jaci received her first dose of jacifusen. We are thrilled, now, to partner with The ALS Association so that more FUS-associated ALS patients can receive jacifusen."
The ALS Association is providing $650,000 for the project and Project ALS is providing $250,000. Additional support for ongoing pre-clinical studies of jacifusen and FUS-associated ALS, led by Dr. Shneider, is provided by Ionis Pharmaceuticals.
"We want to develop simple pathways to treatments for all forms of genetic ALS including rare types, said Neil Thakur, PhD, executive vice president, mission strategy at The ALS Association. "This project is part of a larger strategy to resolve the scientific and regulatory impediments to making genetic therapies accessible and affordable for those who need them. We have been discussing this strategy with NINDS and FDA, and we look forward to carrying this work forward. We are grateful to Dr. Shneider for his leadership in piloting this approach."
Said Dr. Neil Shneider: "Following Jaci Hermstad's initial treatment, we were immediately contacted by other ALS patients with FUS mutations about jacifusen. I am grateful to The ALS Association and Project ALS for providing the funding and infrastructure that allows our Center to expand access to jacifusen humanely, responsibly, and rapidly."
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 our website at http://www.alsa.org.
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. For more information, visit projectals.org.
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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 our Center, visit our website at alscenter.cuimc.columbia.edu.