ALS Association Funding Earmarked to Improve Clinical Trial Infrastructure

The ALS Association has committed $652,543 over the next three years to support efforts to improve the quality and efficiency of clinical trials for amyotrophic lateral sclerosis (ALS), and to promote the development of treatments and research partnerships.

The efforts are a collaboration of the Northeast ALS Consortium (NEALS), the Barrow Neurological Institute in Arizona, and the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital, where activities will take place.

“An important part of our commitment to ALS research is to invest in processes and procedures that can be institutionalized across the research community to create efficiencies, to facilitate global collaboration and expedite impact,” said Kuldip Dave, PhD, ALS Association vice president of research, in a press release.

The support seeks to improve trial quality and efficiency by training scientists in clinical trial conduct and outcome measures used to assess treatment efficacy. The training is particularly timely because of trial conduct challenges caused by the COVID-19 pandemic.

“One of the most important initiatives NEALS has developed over the years is to train current and upcoming investigators, coordinators, and evaluators both on the activities necessary to perform high-quality trials, and to direct the trials of the future,” said Jeremy Shefner, MD, PhD, neurology department chair at Barrow and senior vice president.

“The funding we receive from the ALS Association enables us to continue and expand these educational activities and to make sure that voices from patients and advocates are heard and attended to as our field expands,” Shefner said.

Coordinated efforts to accelerate therapy development will focus on increased distribution of human biological samples to researchers and pharmaceutical companies to find biomarkers that can help identify therapeutic targets.

NEALS, the Barrow Neurological Institute and the Massachusetts General Hospital Neurological Clinical Research Institute (NCRI) have a repository of samples from NEALS and NCRI ALS studies. The repository is partially funded by the ALS Association. ALS researchers may visit this site for access to samples.

“The funding will build on prior Association support for the NEALS biorepository to provide ALS research investigators globally with quick and broad access to biofluid samples,” said Merit Cudkowicz, MD, director of the Healey Center and chief of neurology at Massachusetts General. “Biomarkers can accelerate diagnosis and screening of treatments.”

The organization also intends to promote partnerships with ALS patients, families, and trial participants to further improve patient-centered trial designs and patient access opportunities. The plan includes recruiting new scientists in clinical trial design and conduct.

In January, the ALS Association pledged $3 million over three years to support the first ALS platform investigation, which will take place at the Healey Center in collaboration with NEALS and the Barrow Neurological Institute. Launched in September, the platform is a type of clinical study that evaluates the effectiveness of multiple therapies simultaneously.

The $3 million supports Massachusetts General clinical and data coordination, the study’s biomarker development endpoint engine, and the Monitoring and Outcome Measures Core at Barrow. The nonprofit has joined the committee responsible for advising on matters including study progress, data sharing, budget issues, fundraising, patient recruitment, and retention. In addition, trial participants will be enrolled at the organization’s Certified Treatment Centers of Excellence and affiliated centers.

The U.S. Food and Drug Administration in January authorized the evaluation of the platform trial’s first three ALS treatment candidates: Ra Pharma’s zilucoplan, Clene Nanomedicine’s CNM-Au8, and Biohaven Pharmaceuticals’ verdiperstat.

In the United States, about 5,000 individuals are diagnosed with ALS annually.