ACT for ALS
Accelerate treatment development and protect expanded access.
ACT for ALS is bipartisan federal legislation that passed in 2021. It created a practical, patient-centered approach to accelerate ALS treatment development by building a shared data infrastructure to move promising therapies toward approval faster and by expanding access to investigational treatments for people who cannot enroll in clinical trials—while collecting critical evidence along the way.
What ACT for ALS Does
- Open data and research infrastructure ACT for ALS built the collaboration and data backbone that ALS research has long lacked—creating connected platforms and a competitive grant program that make it easier to find targets, run studies, and move therapies toward approval faster.
- Evidence-generating expanded access programs Most people living with ALS never enroll in a traditional clinical trial, often due to disease progression, complex medical needs, or restrictive eligibility criteria. ACT for ALS addressed this gap by funding research-based Expanded Access Programs (EAPs) designed to provide access while pairing that access with structured learning.
Open Data and Research Infrastructure
ACT for ALS established a partnership with the Foundation for the NIH to create AMP-ALS, aligning federal agencies, academic scientists, companies, and nonprofits around shared data and shared priorities. A core output is the ALS Knowledge Portal, which provides open access to existing clinical datasets and integrates new data and biosamples as they are generated.
CP-RND and FDA are developing common data standards and fit-for-purpose tools to help evaluate ALS treatments more efficiently—including biomarkers, modeling platforms, and clinical outcome assessments. Biomarkers can help characterize disease biology, track progression, monitor response, and inform target selection.
Using ACT for ALS funding, NIH launched the ALL ALS Consortium, conducting a large natural history study with 1,200+ participants across dozens of sites and collecting tens of thousands of samples. Those samples and associated data are deposited into the ALS Knowledge Portal, creating the largest open natural history resource in ALS and helping make participation and resulting datasets more representative.
ACT for ALS established a competitive Rare Disease Grant Program at NIH to support research approaches, tools, and infrastructure relevant to ALS and other rare neurodegenerative diseases—including data collection methods, biomarker development, and clinical research capabilities.
Evidence-Generating Expanded Access Programs (EAPs)
ACT for ALS funds research-based Expanded Access Programs (EAPs) at NIH. These programs are designed to do more than provide access—they intentionally pair access with structured learning, generating data on patients who are routinely excluded from trials and helping the field understand how investigational therapies perform in real-world ALS populations.
Impact to Date: NIH has launched five EAPs nationwide, providing over 700 people living with ALS access to investigational therapies they otherwise could not receive. Early experience also shows promise for tele-EAP models that can reach patients facing geographic or mobility barriers. Lessons from these programs are informing the next stages of development—two therapeutic candidates are advancing to Phase 3 trials.
Why Reauthorization Matters
Without reauthorization, EAPs providing access to hundreds of people living with ALS will wind down, ALL ALS will lose support, and the shared data and collaboration infrastructure accelerating therapy development will be disrupted.
Reauthorization will:
- Sustain evidence generation from the full ALS population, including people typically excluded from trials.
- Keep the national ALS research ecosystem coordinated across NIH, FDA, researchers, industry, and advocates.
- Protect national data and biospecimen resources so discoveries build on prior contributions instead of restarting efforts.