Sean M. Healey & AMG Center for ALS and the University of California, Irvine Announce First Participant Enrolled in Expanded Access Protocol Program for Trehalose

Irvine, CA- The University of California, Irvine (UCI)  successfully enrolled the first participant in its newly established Expanded Access Protocol (EAP) program. Under the direction of Namita Goyal, MD, Professor of Neurology, UCI School of Medicine, and Director of UCI Health ALS Services, the EAP program allows people with ALS who do not qualify for trials to access investigational drugs that are not yet approved by the FDA. UCI’s EAP program is made possible by support from the axeALS Foundation in partnership with the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital.

The first participant is receiving trehalose, an investigational drug that is currently tested in the HEALEY ALS Platform Trial. In parallel to the trial, the Healey & AMG Center was awarded a grant from the National Institute of Neurological Disorders and Stroke to conduct an intermediate-size EAP of trehalose for ALS patients who do not qualify for the trial.  UCI is one of 25 platform trial centers also participating in the trehalose EAP.

“I am thrilled that ALS patients who don’t meet the  criteria to enroll in an ALS clinical trial, now have an opportunity to try cutting edge investigational therapies through this EAP program” said Dr. Goyal. “We are thankful for the support of axeALS in making this expanded access protocol possible.”

Expanded Access, also referred to as Compassionate Use, is a pathway for people with a serious and life-threatening disease to access an investigational product that is in clinical trials, but not yet approved by the FDA. The EAP program brings new options to be part of research to people by coordinating access to experimental drugs and enables the collection of safety and biomarker data in a population not studied in clinical trials. This data can help inform the next trial or help support market approval for a broader group of people with ALS than those typically included in clinical trials.

“We are excited about the successful launch of the EAP program at UCI,” said Sabrina Paganoni, MD, PhD, physician scientist at the Healey & AMG Center, Co-Director of the Neurological Clinical Research Institute at MGH and one of the Principal Investigators of the NIH-funded trehalose EAP. “This first enrollment is an important milestone, and we look forward to including many other members of the ALS community who may not be eligible for clinical trials. Programs like this help advance research and innovation in ALS.”

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), sodium phenylbutyrate/taurursodiol , and tofersen. Dextromethorphan/quinidine 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.