The Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital has been awarded a grant from the National Institute of Neurological Disorders and Stroke (NINDS) to conduct an intermediate size Expanded Access Protocol (EAP) in Amyotrophic Lateral Sclerosis (ALS). The grant is supported by the ACT for ALS (Accelerating Access to Critical Therapies for ALS Act). This EAP will evaluate the benefits of the investigational drug trehalose in individuals with ALS and will be led by Healey Center faculty, Drs. Suma Babu, James Berry, and Sabrina Paganoni.

Expanded Access, also referred to as Compassionate Use, is an FDA-regulated pathway that allows patients with a serious and life-threatening disease to access an investigational drug that is not yet approved by the FDA. Trehalose (SLS-005, Seelos Therapeutics) is observed to have protective effects in ALS mouse models by activating autophagy, a mechanism that is impaired in ALS.

The safety and efficacy of trehalose are currently being tested in the HEALEY ALS Platform  . The trehalose ALS EAP made possible by this award will allow ALS individuals who are otherwise not be eligible for the platform trial to access this investigational drug. Additionally, this EAP will provide real world safety, biomarker, and clinical data in ALS individuals treated with long duration of trehalose.

This study is the very first EAP made possible by the historic “ACT for ALS” bill. This EAP will be conducted at up to 25 platform trial centers across the US, bringing access to experimental trehalose closer to home for many ALS patients. The researchers at the Healey & AMG Center for ALS are hopeful that this study will pave the way for a new era of patient-centric and scientifically relevant EAPs to be designed and conducted for the ALS community in the US for years to come. Such EAPs would run in parallel to late phase randomized controlled clinical trials and contribute safety, biomarker, and clinical data to supplement clinical development programs of investigational products in ALS.

“I am excited that the Healey & AMG Center is the recipient of the first ACT for ALS funding opportunity and look forward to launching the trehalose EAP,” said  Merit Cudkowicz, MD, MSc, 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 thrilled to bring broader access to this experimental therapy as we conduct the ongoing testing of trehalose in the HEALEY ALS Platform trial.”

 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 progressive muscle weakness and atrophy. There are currently three FDA therapies approved specifically for treating ALS symptoms—riluzole, nuedexta and edaravone. But there is no cure.

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 on a quest to discover life-saving therapies for all individuals affected by ALS. Launched in November 2018, the Healey Center leverages a global network of scientists, physicians, nurses, caregivers, patients and families working together to accelerate the pace of ALS therapy discovery and development.

Under the leadership of Merit Cudkowicz, MD, chief of the Department Neurology, and a Science Advisory Council of international experts, we are reimagining how to develop and test the most effective therapies to treat the disease, identify cures and, ultimately, prevent it.

The key to our success is our tightly integrated research and clinical efforts, encouraging opportunities to bring the challenges our patients face every day into our laboratories, focusing investigations on finding solutions that will make a meaningful difference to our patients without delay. Our collaborative efforts are designing more efficient and effective clinical trials while broadening access to these trials for people with ALS.