There is an urgent need for new and improved therapies for ALS, as there is still no cure. To help accelerate drug development and get promising treatments one step closer to regulatory evaluation, the ALS Association has committed nearly $4 million to support four early-stage clinical trials through our Hoffman ALS Clinical Trial Awards.
While the number of ALS clinical trials has greatly expanded over the last five years, the number of people living with ALS participating in this research remains low and highly variable across trial sites. Our five 2023 Trial Capacity Award winners are working hard to eliminate barriers to trial participation.
In order to make ALS a livable disease and ultimately find a cure, we need to increase the number of clinical trials and make it easier for more people to participate in those trials. No one knows this better than Dr. Merit Cudkowicz, Director of the Sean M. Healey & AMG Center for ALS and Chief of Neurology at Massachusetts General Hospital.
Dr. Laura Ranum, director of the University of Florida’s Center for NeuroGenetics and the Kitzman Family Professor of Molecular Genetics and Microbiology, is currently conducting a phase 2 trial for C9orf72-linked familial ALS with support from the Association’s Clinical Trial Awards Program.
In March 2022, after several months of multiple doctor visits and many tests, Jacob Harper was diagnosed with P525L FUS ALS—a nano-rare version of ALS only found in people under the age of 25. Jacob is on the Jacifusen clinical trial trying to slow progression, as he shares his testimony with people from all over the world.
The Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital and the Northeast ALS (NEALS) Consortium announced topline results from Regimen D of the HEALEY ALS Platform Trial, which is evaluating pridopidine versus placebo in adults with ALS.
The ALS Association has awarded $4.9 million to help speed the testing of ALS therapies through clinical trials. The Association’s inaugural Trial Capacity Awards will support efforts at 13 established and emerging ALS clinical trial sites to increase the number and diversity of people living with the disease who have the opportunity to participate and improve the efficiency and pace at which these studies are conducted.
We talked with Dr. Stephen Johnson, postdoctoral fellow from Massachusetts General Hospital, to learn more about his research focused on ways to accelerate discovery of efficacious therapeutics through identification and validation of novel outcome measures using readily available technology.