The US Food and Drug Administration (FDA) and the National Institutes of Health (NIH) have launched a public-private partnership aimed at advancing the understanding of neurodegenerative diseases, the organisations announced.
The goal of the partnership – the Critical Path for Rare Neurodegenerative Diseases (CP-RND) – is to generate ‘actionable solutions that can tangibly accelerate’ drug development for amyotrophic lateral sclerosis (ALS) and other rare neurodegenerative diseases, the FDA said in a statement.
Hilary Marston, FDA chief medical officer, said: “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. Collaboration across public and private sectors can accelerate the progress to address this urgent need.
“The partnership we are announcing will leverage the shared expertise of all participants to create a path towards new breakthroughs in treating these diseases.”
The FDA and NIH have selected the Critical Path institute (C-Path) as the convener of the partnership, which the organisations hope will bring together experts in rare neurodegenerative diseases, including patient communities, advocacy organisations and private entities.
Areas of focus will include patient-focused drug development, and the utilisation of the FDA-funded Rare Disease Cures Accelerator-Data and Analytics Platform External Link Disclaimer (RDCA-DAP) to bring together scientific data on rare neurodegenerative diseases.
The FDA outlined that this will ‘facilitate the characterisation of neurodegenerative diseases and their natural history, the identification of molecular targets for neurodegenerative disease, and increased efficiency, predictability, and productivity of clinical development of therapies’.
The partnership follows President Biden signing the Accelerating Access to Critical Therapies for ALS Act into law on 23 December 2021, requiring the US Department of Health and Human Services (HHS) through the FDA and NIH to establish and implement a public-private partnership not later than one year after enactment.
ALS alone affects as many as 30,000 people in the US, with 5,000 new cases diagnosed each year. Characterised by a progressive degeneration of motor nerve cells in the brain and spinal cord, it is estimated that ALS is responsible for approximately five of every 100,000 deaths in people aged 20 years and older.
“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 progression of ALS,” said Walter Koroshetz, director of the National Institute of Neurological Disorders and Stroke (NINDS), part of the NIH.