BOSTON -- The Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital in conjunction with Seelos Therapeutics and NINDS have announced the first patient-dosing in the NINDS-funded Expanded Access Protocol (EAP) for Trehalose.

In the Fall of 2022, the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital was awarded a grant from NINDS to conduct an intermediate size EAP in people living with Amyotrophic Lateral Sclerosis (ALS).

The grant is supported by the ACT for ALS (Accelerating Access to Critical Therapies for ALS Act), and this study is the very first EAP made possible by the bill. This EAP is led by Healey Center faculty, Drs. Suma Babu, James Berry, and Sabrina Paganoni in collaboration with Seelos Therapeutics.

“We are thrilled that the trehalose EAP made possible by the NINDS grant is getting underway” said Sabrina Paganoni, MD, PhD, Physician Scientist at the Healey & AMG Center and Co-Director of the Neurological Clinical Research Institute at MGH. “This program will provide real-world data about the role of trehalose in ALS and will supplement clinical trial data. We are excited that this grant mechanism allowed us to design and launch a scientifically relevant and patient-centric expanded access study.”

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 Trial  .

"The beginning of this expanded access program represents the culmination of collaborative work from many advocates and people living with ALS, Congress, the NIH, Seelos, and dedicated study teams at Mass General and ALS centers across the country” said James Berry, MD, MPH, Winthrop Family Scholar in ALS Sciences, and Director of the Neurological Clinical Research Institute at MGH.

The trehalose ALS EAP allows for ALS individuals who are otherwise not be eligible for ongoing clinical trials 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 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. 

“This EAP made possible by the historical ACT-for-ALS ushers in a new era of much needed access opportunities for many ALS individuals who otherwise would not be able to participate in therapeutic development research” said Suma Babu, MBBS, MPH physician investigator at the Healey & AMG Center for ALS at MGH. “The first participant’s dosing in this study marks an important moment for the ALS patient and research community!”

“ALS is a tragic neurological disorder that exacts a devastating toll on individuals and their families. This study may provide some insights into the mechanisms underlying this disorder and may help researchers identify biomarkers that would help track disease progression,” said Lumy Sawaki-Adams, M.D., Ph.D., program director at NINDS. 

 This study is supported by NIH grant 1UF1NS131791-01.

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 few FDA therapies approved for treating ALS—riluzole, edaravone (IV and oral formulation), and Relyvrio. Nuedexta is also used for the symptomatic treatment of pseudobulbar affect (PBA) in people with ALS.

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, people with ALS and families working together to accelerate the pace of ALS therapy discovery and development.

Under the leadership of Merit Cudkowicz, MD 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.