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Amyotrophic lateral sclerosis (ALS, Lou Gehrig’s disease) is a neurological disease with limited treatment options. Only recently have major advances in ALS science led to a rich pipeline of promising therapeutics. There is a critical need to develop innovative clinical trial approaches to more rapidly test treatments, in the right people and at the right time, and improve access to experimental treatments for people with ALS. The Healey Center for ALS at Mass General has assembled a team of experts to dramatically rethink how early phase trials in ALS are performed and to accelerate a path to effective treatments.

Borrowing from successes in cancer drug development, the Sean M. Healey & AMG Center for ALS at Mass General, in partnership with TackleALS, are leading the first Platform Trial initiative for ALS. Traditionally, each trial evaluates only one drug at a time, and requires lengthy start-up and execution times. Platform trials, instead, are trials where multiple drugs are tested at the same time, using specialized statistical tools. New regimens (drugs) can be added as they become available thereby decreasing or eliminating the gap in time from identification of a rationale therapy to testing. Thus, the focus is on the disease, rather than any individual experimental agent, and the platform remains open long-term until successful cures are found. The HEALEY ALS Platform Trial will test multiple promising experimental therapeutics using an efficient and informative early-phase design with increased access for people with ALS.

On March 20, 2019, we issued the first call for proposals for the initial drugs for the HEALEY ALS Platform Trial and we received an outstanding response with almost 30 applicants from 10 countries. Our Therapy Evaluation Committee, a group of expert ALS scientists and members of the Healey Center Science Advisory Committee chose the top five therapeutic ideas ready to enter the Platform Trial. Industry partners from these first five companies are working closely with the Healey Center ALS Trial Design Team, Northeast ALS Consortium (NEALS) and Berry Consultants to tailor the arm of the Platform Trial to their experimental treatment.

We fully anticipate that more treatments will be added to the HEALEY ALS Platform Trial, supported by pharma, foundation partners, philanthropy, federal and other fundraising initiatives. We encourage the academic and industry community to engage with the Healey Center ALS Trial Design Team at any time to work together on ALS therapy development. Nominations of therapies for inclusion in the platform trial will continue on an ongoing, rolling basis.

For Industry and Academic Investigators interested in adding your treatment idea to the HEALEY ALS Platform TRIAL or in contacting the Healey Center ALS Trial Design Team, please contact HealeyCenterforALS@mgh.harvard.edu.