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UCI Health enrolls first patient in expanded access protocol program for ALS drug

Program is partnership with Sean M. Healey & AMG Center for ALS and axeALS Foundation

September 25, 2023
uci health als researcher and physician namita goyal standing in an office wearing black top and burgundy cardigan
Dr. Namita Goyal leads
UCI's expanded access
protocol program.

Irvine, Calif. — The University of California, Irvine (UCI) has successfully enrolled the first participant in its newly established Expanded Access Protocol (EAP) program. Under the direction of Dr. Namita Goyal, professor of neurology at the UCI School of Medicine, and director of the UCI Health ALS & Neuromuscular Center, 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 Goyal. “We are thankful for the support of axeALS in making this expanded access protocol possible.”

A pathway for patients

A continuing challenge for people living with ALS is that not everyone is eligible to participate in clinical trials due to their disease characteristics or progression. “Expanded access,” also referred to as “compassionate use,” is a pathway for patients with a serious and life-threatening disease to access an investigational product that is not yet approved by the FDA.

The EAP program brings new options to be part of research to patients by coordinating access to experimental drugs and enables the collection of safety and biomarker data in a population not studied in randomized trials. This data can help inform the next trial or help support market approval for a broader group of ALS patients than those typically included in double-blind 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.”

County's only ALS Association center of excellence

The UCI Health ALS program is the only ALS Association Certified Treatment Center of Excellence in Orange County, the nation’s sixth most populous county, and one of only nine in California. The center offers ALS clinical trials through UCI Health and the UCI Alpha Clinic.

The axeALS Foundation was founded by Southern California residents Eric and Amanda Stevens after Eric was diagnosed with ALS at just 29 years old. The foundation’s mission is to raise awareness and funding for ALS treatments and clinical trials, to help ALS patients and their families live as full and normal lives as possible, and to encourage research for the prevention, alleviation, care, treatment, and (one day) cure of ALS. 

About amyotrophic lateral sclerosis

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), Relyvrio, and Qalsody. Nuedexta is also used for the symptomatic treatment of pseudobulbar affect (PBA) in people with ALS.

Learn more about the trehalose trial ›

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.

About UCI Health

UCI Health is the clinical enterprise of the University of California, Irvine, and the only academic health system in Orange County. Patients can access UCI Health at primary and specialty care offices across Orange County and at its main campus, UCI Medical Center in Orange, Calif. The 459-bed, acute care hospital, listed among America’s Best Hospitals by U.S. News & World Report for 23 consecutive years, provides tertiary and quaternary care, ambulatory and specialty medical clinics, behavioral health and rehabilitation services. UCI Medical Center is home to Orange County’s only National Cancer Institute-designated comprehensive cancer center, high-risk perinatal/neonatal program and American College of Surgeons-verified Level I adult and Level II pediatric trauma center and regional burn center. UCI Health serves a region of nearly 4 million people in Orange County, western Riverside County and southeast Los Angeles County. Follow us on Facebook, Instagram, LinkedIn and Twitter.