Stronger ACT for ALS Act Would Foster New Treatments, ALS Association Says

After working for months behind the scenes, the ALS Association is calling a strengthened Accelerating Access to Critical Therapies for ALS Act (ACT) a key step toward swifter development of new therapies.

The revised bill (HR 8662/S. 4867) calls for $100 million over five years for amyotrophic lateral sclerosis (ALS) research and establishment of the first federal body specifically charged with coming up with treatments for neurodegenerative diseases.

ACT for ALS was reintroduced Oct. 29 in the U.S. House of Representatives by Jeff Fortenberry (R-Nebraska) and Mike Quigley (D-Illinois), and in the Senate by Chris Coons (D-Delaware) and Lisa Murkowski (R-Alaska).

“Together, these revisions will ensure the ACT for ALS Act will benefit the entire ALS community and bring promising treatments to people with ALS,” the ALS Association stated in a press release. “We look forward to working with congressional champions, committees of jurisdiction, and the ALS community as the bill is refined throughout the legislative process to ensure the bill becomes law as soon as possible.”

The organization worked with ALS community stakeholders to incorporate its recommendations into the bill, which would create a new framework specifically for getting potential new treatments to patients. It also would create a new U.S. Food and Drug Administration (FDA) research grant program for rare neurodegenerative disorders.

A Collaborative for Rare Neurodegenerative Diseases would be established at the U.S. Department of Health and Human Services (HHS) to coordinate federal efforts to develop and approve new therapies and cures.

The $100 million in funding, which must be appropriated by Congress, would be allocated among the three programs.

The HHS collaborative would advise on program grants, and establish an advisory panel including patients who would review the three programs’ designs and implementation. The panel also would report its findings to Congress.

In addition, the entity would produce and make public an action plan outlining government efforts to make program enhancements, and to develop policy and regulatory science initiatives. It would facilitate pharmaceutical companies’ use of the FDA guidance on ALS treatment development, and define or develop regulatory or translational pathways for treatment candidates. (Translational research emphasizes improved health outcomes.)

Finally, the collaborative would develop and implement a mechanism for sharing feedback about research, as well as strategies for providing accessible care services.

The ALS Association is asking bill supporters to urge lawmakers to cosponsor the ACT for ALS Act. The measure was referred to the House Committee on Energy and Commerce.