ACT for ALS: Collaboration

ACT for ALS was created to not only expand access to clinical research participation, but to accelerate ALS research by fostering collaboration and funding for neurodegenerative disease research.

Before the ACT for ALS, there was very little interaction between the regulators at FDA and the researchers at NIH, over neurological diseases in general, and ALS specifically. Today, FDA and NIH are coordinating actions across disciplines on ALS. They hold regular check-in calls to monitor and share information about the research landscape. These conversations are breaking down barriers to collaboration critical to making progress on ALS treatments and cures.

Click on each section below to learn more about that area of collaboration achieved through ACT for ALS.

AMP ALS

Funded by the ACT for ALS, the Accelerating Medicines Partnership ALS (AMP ALS) is an initiative that accelerates turning scientific discoveries into new ALS therapies. It brings together the NIH, FDA, drug sponsors, nonprofits, and academia to generate shared, open-access datasets. By facilitating data exchange, AMP ALS deepens and accelerates our understanding of ALS. AMP ALS has already made significant progress, analyzing over 2,000 ALS patient samples to identify molecular subtypes, biomarkers, and therapeutic targets. Its data and findings are openly accessible through their platform, the AMP ALS Knowledge Portal.

CRITICAL PATH FOR RARE NEURODEGENERATIVE DISEASES

Established under ACT for ALS, the Critical Path Institute (C-Path)’s Critical Path for Rare Neurodegenerative Diseases (CP-RND) initiative develops essential drug development tools for ALS. CP-RND fosters collaboration among federal agencies, industry, academia, patients, and nonprofits to advance standards used to approve therapies like biomarkers, clinical outcome assessments, and AI/trial simulation tools. A key achievement is the RDCA-DAP platform for secure, pre-competitive data sharing, integrating patient data from different studies.

ACCESS FOR ALL IN ALS (ALL ALS)

Access for ALL in ALS (ALL ALS) is an NIH-funded consortium that conducts natural history studies of ALS and comprises two coordination centers and currently 32 active research sites. So far 737 participants have enrolled in their two studies: PREVENT and ASSESS. * These studies aim to enhance our understanding of ALS, paving the way for more targeted and personalized drug development.

Despite knowing of its existence for 160 years, one of the challenges facing researchers is the lack of a “natural history study” for ALS. Natural history studies describe the course of a disease from before symptom onset through progression to final outcomes without intervention and are critical for research and drug development, providing comprehensive, real-world data that drive scientific understanding, clinical innovation, and regulatory approval pathways.

PREVENT ALL ALS focuses on understanding the earliest manifestations of ALS in asymptomatic gene carriers through a natural history study.

ASSESS ALL ALS is a longitudinal natural history study and biomarker collection aims to understand a wide spectrum of ALS by recruiting 1600 individuals currently living with ALS and a control cohort of 450 individuals without ALS and no genetic risk. They make research more accessible by leveraging new technology for remote participation, overcoming the lack of geographic access to ALS research sites. Their biorepository, BioSEND, stores biofluid samples for sharing with the broader research community.

* https://www.all-als.org/ As of 7/22/25