The US Food and Drug Administration (FDA) and the US National Institutes of Health (NIH) have unveiled a public-private partnership (PPP) to understand and develop treatments for neurodegenerative diseases.

Named the Critical Path for Rare Neurodegenerative Diseases (CP-RND), the partnership will be convened by the Critical Path Institute (C-Path).

It will focus on developing therapies for amyotrophic lateral sclerosis (ALS) and various other rare neurodegenerative diseases. 

The alliance will bring together specialists in rare neurodegenerative ailments, including advocacy groups, private entities and patients. 

The partnership will focus on developing patient-focused drugs as well as using the Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP), funded by the FDA, to combine scientific evidence on rare neurodegenerative diseases and aid in characterising the ailments and their natural history.

The platform will also be used to detect neurodegenerative disease molecular targets and enhance the predictability, efficiency and productivity of the clinical development of treatments.

Using the shared capabilities of the partners, the alliance intends to create actionable solutions that will expedite the development of drugs for rare neurodegenerative diseases. 

Last December, US President Joe Biden signed the Accelerating Access to Critical Therapies for ALS Act (Act for ALS) into law.  

It mandated that the US Department of Health and Human Services, through the FDA and NIH, would form a PPP. 

FDA chief medical officer Hilary Marston 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 today will leverage the shared expertise of all participants to create a path towards new breakthroughs in treating these diseases.”