ALS Association Invests $3M in Platform Trial to Accelerate Development of Treatments

To help accelerate development of effective treatments for amyotrophic lateral sclerosis (ALS), the ALS Association is investing $3 million in the disease’s first platform trial.

The award is $1 million annually for three years to support the platform investigation, a type of clinical study that evaluates the efficacy of multiple treatments simultaneously. The trial launched last September at the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital.

“This platform trial will significantly speed efficacy trials for ALS research, and is a major advancement for ALS research,” Calaneet Balas, president and CEO of the ALS Association, said in a press release. “In addition to supporting the HEALEY ALS Platform Trial, we will be working to ensure the rest of the research pipeline is moving fast enough to keep up.”

The funding will go toward Massachusetts General clinical and data coordination, the trial’s biomarker development endpoint engine, and the Monitoring and Outcome Measures Core at Barrow Neurological Institute. The ALS Association will join the committee responsible for advising on study progress, data sharing, budget issues, fundraising, patient recruitment and retention, and other matters. In addition, trial participants will be enrolled at the nonprofit’s Certified Treatment Centers of Excellence and affiliated centers.

“Working closely together, we will bring new therapies forward faster for people with ALS,” said Merit Cudkowicz, MD, Healey Center director and Massachusetts General chief of neurology. “The platform trial builds on previous ALS Association-supported research on many new ALS targets and trial innovations.”

“The HEALEY ALS platform trial initiative can only succeed with the support and involvement of the entire ALS community,” added Cudkowicz. “We look forward to ongoing partnerships with people and families with ALS, all ALS foundations, our colleagues in the NEALS Consortium, industry partners and regulatory agencies.”

After announcing it was seeking the best ALS treatments to start clinical studies, the Healey Center opened its trial with five candidate therapies from among 30 applications from 10 countries.

Chosen by a group of leading researchers and members of the Healey Center Science Advisory Committee, the experimental treatments include Biohaven Pharmaceuticals’ verdiperstat, Ra Pharma’s zilucoplan, Clene Nanomedicine’s CNM-Au8, Prilenia’s pridopidine, and Implicit Bioscience’s IC14 immunotherapy. The platform trial will start with three of these therapies, followed soon by the remaining two.

In addition to assessing the different treatments, the platform trial will include multiple promising biomarkers and outcome measures targeted at developing new tools to measure the therapies’ effectiveness.

The trial can add newly developed clinical therapies to reduce the time it takes to identify a prospective treatment and to test it. The trial’s design also increases access to clinical trials, and decreases costs by quickly determining treatment effectiveness. Additionally, the results from all subjects in placebo groups can be combined, ultimately leading to stronger data.

The Healey trial was designed by partners of the five selected companies, in collaboration with the Healey Center Trial Design Team and the international nonprofit Northeast ALS Consortium (NEALS). The model has been successful in identifying treatments for certain kinds of cancer.

Since 2010, the ALS Association has committed more than $9 million to ALS research at Massachusetts General. The organization is the world’s largest private funder of ALS research.

The Healey Center opened in 2018 to discover life-saving therapies for ALS patients.

For more information on the HEALEY trial, write to [email protected].