FDA and NIH Open Initiative to Spur ALS Research, Potential Treatments
Critical Path Institute leading public-private partnership's work
The U.S. Food and Drug Administration (FDA) and the National Institutes of Health (NIH) have forged a partnership that seeks to advance both the understanding of rare neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS) and the development of potential treatments.
The public-private collaboration, called the Critical Path for Rare Neurodegenerative Diseases (CP-RND), is funded by an FDA grant and will be led by the Critical Path Institute (C-Path), an independent partnership created by the FDA in 2005 to serve as a multi-stakeholder infrastructure working to accelerate possible therapies.
ALS research initiative to include patient communities, advocacy groups
“There is a crucial need to develop new treatments that can improve and extend the lives of people diagnosed with rare neurodegenerative diseases, including ALS,” Hilary Marston, MD, the FDA’s chief medical officer, said in an agency press release.
“Collaboration across public and private sectors can accelerate the progress to address this urgent need,” Marston added. “The partnership we are announcing today will leverage the shared expertise of all participants to create a path towards new breakthroughs in treating these diseases. We look forward to working with NIH, C-Path, and other public and private partners to carry out this important effort.”
C-Path will bring together experts in these rare diseases, including members of patient communities, private entities, and advocacy organizations, who will work to determine the effort’s direction and priorities.
“These innovative and collaborative approaches are needed to accelerate medical product development for the benefit of individuals living with these rare diseases, and their families,” Wainwright Fishburn, C-Path’s board chair, said in separate press release from the institute. “I am thrilled about this partnership that will strengthen collaborations between C-Path, FDA, and NIH, and bring together a diverse group of stakeholders.”
Focus areas will include work into treatments focused on the patients’ needs and use of the FDA-funded Rare Disease Cures Accelerator-Data and Analytics Platform to pull together disease research data. The platform aims to help characterize these rare disorders and their natural histories, identify molecular targets that could result in treatments, and accelerate clinical development of identified potential therapies.
The partnership’s overarching goal is to use participants’ shared expertise to speed treatment development for neurodegenerative conditions.
“This public-private partnership will convene the entire ALS community to develop novel strategies and approaches to therapy development and clinical testing with the goal to finally produce a treatment that stops the tragic progressions of ALS,” said Walter Koroshetz, MD, director of the National Institute of Neurological Disorders and Stroke, which is part of the NIH.
President Joe Biden signed the Accelerating Access to Critical Therapies for Amyotrophic Lateral Sclerosis Act into law in December, requiring the U.S. Department of Health and Human Services to create and implement a public-private partnership within a year following enactment. The legislation frees up $100 million annually through fiscal year 2026 to improve the prevention, diagnosis, and treatment of ALS and similar disorders.
The research partnership is a key part of the FDA’s five-year action plan, announced in June, that seeks to advance the development of treatments for ALS and other rare neurodegenerative diseases, as well as access to them.
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