Accelerating Access to Critical Therapies for Amyotrophic Lateral Sclerosis Act

Amyotrophic Lateral Sclerosis, or ALS, is a progressive and fatal neurodegenerative disorder, affecting around 30,000 people in the United States. Although some cases of ALS are inherited, the majority are sporadic, meaning that they occur with no clear family history. ALS is characterized by the degeneration and death of motor neurons in the brain and spinal cord, which control voluntary muscle movement and breathing, resulting in difficulty chewing, swallowing, speaking, and breathing. There are currently no therapies that reverse damage to motor neurons or cure ALS. The goals of NINDS’s ALS research are to understand the cellular mechanisms involved in the development and progression of the disease, investigate the influence of genetics and other potential risk factors, identify biomarkers, and develop new treatments. Building on these efforts, the Accelerating Access to Critical Therapies for ALS Act (ACT for ALS; P.L. 117-79), signed into law on December 23, 2021, directed the NIH to establish a research grant program that leverages data from the Food and Drug Administration (FDA)’s expanded access to experimental drugs pathway for individuals who are not eligible for ALS clinical trials. The ACT for ALS also called for the establishment of a public private partnership (PPP) to foster the development of treatments for ALS and other rare neurodegenerative diseases. People with lived experience of ALS (PWLE) are integral partners in implementing the ACT for ALS. Their perspectives, included throughout all outputs of the Act, help shape research priorities to ensure real-world needs and patient-centered outcomes are addressed. ACT for ALS is supplementary to NIH- and NINDS-funded investigator-initiated ALS research