BOSTON – The HEALEY ALS Platform trial led by the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital in collaboration with the Northeast ALS Consortium (NEALS) is paving the way for rapid testing of several potential treatments for people with amyotrophic lateral sclerosis (ALS), a neurodegenerative disease affecting the brain and spinal cord.

Enrollment is now complete for Regimen F evaluating ABBV-CLS-7262, an investigational product being developed by Calico Life Sciences LLC (Calico) in collaboration with AbbVie.

The HEALEY ALS Platform Trial is designed to evaluate multiple investigational products simultaneously, thus accelerating the development of effective and breakthrough treatments for people living with ALS. The trial leverages a shared placebo group and central infrastructure to make efficient use of resources. The central infrastructure allows the study team to learn from each regimen and continue to refine and adapt the trial. The Sean M. Healey & AMG Center for ALS works closely with NEALS and industry partners to tailor the platform trial regimen to their investigational product.

“We are thrilled to have reached this critical milestone for this investigational product in the Platform Trial” says Merit Cudkowicz, MD, MSc, principal investigator and sponsor of the HEALEY ALS Platform Trial, director of the Sean M. Healey & AMG Center for ALS, chief of the Department of Neurology at MGH, and the Julieanne Dorn Professor of Neurology at Harvard Medical School. “We are grateful for the partnership and participation of people with ALS and their families that allow trials like this to be possible.”

Investigational products that enter the platform trial are selected by a group of expert ALS scientists and members of the Platform Trial Therapy Evaluation Committee.

ABBV-CLS-7262 targets eIF2B, a key regulator of the integrated stress response (ISR), a pathway activated in people with ALS. In neurons exposed to cellular stressors, inhibition of the ISR by ABBV-CLS-7262 restores protein synthesis and dissolves pre-formed TDP-43 containing stress granules. This effect of ABBV-CLS-7262 is of clinical interest because TDP-43 containing stress granules are thought to lead to TDP-43 inclusions, a hallmark of ALS pathology.

Regimen F is co-led by Senda Ajroud-Driss, MD, Neurologist at Northwestern University, Feinberg School of Medicine in Chicago, Illinois. “I am really inspired by this incredibly efficient enrollment, a testament to the hard work and commitment of the Healey ALS Platform Trial sites and the dedication of the ALS patients and their families” said Dr. Ajroud-Driss.

ABBV-CLS-7262 is not yet approved for use in any country. Calico is a research and development company focused on understanding the biology that controls human aging, and founded by Alphabet and Arthur D. Levinson, Ph.D. Together with AbbVie, they are collaborating to discover and develop potential new therapies for patients with age-related diseases, including neurodegenerative disorders.

For updates on the trial, please join our weekly Healey ALS Platform Trial webinars.

Watch this video from Calico for more information on the mechanism of action behind their investigational product, ABBV-CLS-7262.

Background on ALS

Amyotrophic lateral sclerosis (ALS) is the most prevalent adult-onset progressive motor neuron disease, affecting approximately 30,000 people in the U.S. and an estimated 500,000 people worldwide. ALS causes the progressive degeneration of motor neurons, resulting in muscle weakness and atrophy. There is an urgent need to understand the biology of ALS and to develop effective therapies.

About the Sean M. Healey & AMG Center for ALS at Mass General

At the Sean M. Healey & AMG Center for ALS at Mass General, we are committed to bringing together a global network of scientists, physicians, nurses, foundations, federal agencies, and people living with ALS, their loved ones, and caregivers to accelerate the pace of ALS therapy discovery and development.

Launched in November 2018, the Healey & AMG Center, under the leadership of Merit Cudkowicz, MD and a Science Advisory Council of international experts, is reimagining how to develop and test the most promising therapies to treat the disease, identify cures and ultimately prevent it.

With dozens of active clinical trials and lab-based research studies in progress right now, we are ushering in a new phase of ALS treatment and care. Together, we will find the cures.

About the Massachusetts General Hospital
Massachusetts General Hospital, founded in 1811, is the original and largest teaching hospital of Harvard Medical School. The Mass General Research Institute conducts the largest hospital-based research program in the nation, with annual research operations of more than $1 billion and comprises more than 9,500 researchers working across more than 30 institutes, centers and departments. In July 2022, Mass General was named #8 in the U.S. News & World Report list of "America’s Best Hospitals." MGH is a founding member of the Mass General Brigham healthcare system.