MDA $500K research grant will support HEALEY ALS platform trial
Award to Mass General researcher will help fund program for 3 years
The Muscular Dystrophy Association (MDA) has awarded a $500,000 research grant to a Massachusetts General Hospital scientist to support the HEALEY ALS platform trial — a pioneering trial that’s seeking to find treatments for amyotrophic lateral sclerosis (ALS) in a faster timeframe.
The three-year clinical research grant was given to Sabrina Paganoni, MD, PhD, a co-principal investigator at HEALEY ALS. It builds on a past $500,000 grant awarded to Paganoni that supported the platform trial from early 2021 through March of this year. This new MDA research grant will help fund the platform trial through 2027.
“We are incredibly grateful for Muscular Dystrophy Association’s generous support of the HEALEY ALS Platform Trial,” said Paganoni, who also co-directs Mass General’s Neurological Clinical Research Institute, said in a MDA press release.
HEALEY ALS (NCT04297683), led by the Sean M. Healey & AMG Center for ALS at Mass General, is designed to test multiple candidate ALS treatments simultaneously against a shared placebo group, with the aim of faster and better testing of potential therapies. New treatments are added as they become available, and joining the platform helps shorten their development timeline.
New MDA research grant brings project funding to $1M
Since its launch in 2020, seven therapies have been selected for Phase 2 testing in the platform trial. Patients enrolling in the trial were randomly assigned to one of the arms that was enrolling at the time they joined.
In each treatment arm, three-quarters of participants are given the investigational drug while the remaining individuals receive a placebo. Because each treatment arm uses the same eligibility criteria, data from those given active treatment in each arm can be compared with data from patients given a placebo across all arms of the study.
To date, three candidate therapies — zilucoplan, verdiperstat, and SLS-005 — have had their development discontinued due to lack of efficacy compared with the pooled placebo group. But two therapies — CNM-Au8 and pridopridine — are moving forward to Phase 3 clinical testing based on positive efficacy signals seen in HEALEY.
The final two trial arms, which have completed patient enrollment but are still ongoing, are testing ABBV-CLS-7262 and DNL343.
With the new grant, researchers will explore new biomarkers that could help determine how well the candidate treatments are working. The funding also may improve efforts to reach and enroll more participants — which would make the platform trial more representative of all people who live with ALS.
Additionally, the grant monies will provide the resources needed for the researchers to engage with the U.S. Food and Drug Administration and discuss how to adjust their protocols based on past experience. That is expected to help to ensure alignment on the path for potential approval of new treatments.
The MDA is deeply committed to advancing research that brings us closer to life-changing treatments and, ultimately, a cure for ALS.
“This funding will enable us to expedite the testing of promising therapies and move us closer to our ultimate goal of finding effective treatments for people living with ALS,” Paganoni said. “Collaborations like this are essential to driving innovation and making a meaningful difference in the lives of patients and their families.”
Since the 1950s, the MDA has awarded more than $176 million in total for research into ALS, including for the HEALEY ALS platform trial.
“The MDA is deeply committed to advancing research that brings us closer to life-changing treatments and, ultimately, a cure for ALS,” said Sharon Hesterlee, PhD, MDA’s chief research officer. “Our continued funding of the Healey ALS Platform Trial is a significant step forward in our ongoing mission to support cutting-edge research, enhance patient care, and advocate for the ALS community.”
For Merit Cudkowicz, MD, the principal investigator and sponsor of HEALEY ALS and director of the Sean Healey Center, “the support of MDA is instrumental in driving forward our quest for a cure.” Cudkowicz added that “together, we are making significant strides in the fight against ALS.”
About the Author
