NewsMar | 16 | 2023
The axeALS Foundation in partnership with the Sean M. Healey & AMG Center for ALS establishes Expanded Access Protocol (EAP) Program at the University of California, Irvine
The nonprofit’s $486,000 donation provides options for ALS patients
Irvine, CA —The axeALS Foundation is partnering with the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital to establish a new Expanded Access Protocol (EAP) program at the University of California, Irvine. Under the direction of, Namita Goyal, MD, professor of neurology, UCI School of Medicine, and director of UCI Health ALS Services, thanks to multiyear support from the axeALS Foundation, UCI will add expanded access as an option for people living with ALS who do not qualify for trials.
“We are so excited to bring this EAP program to our ALS patients at UC Irvine who do not qualify for trials and are so thankful to axeALS for providing our patients a wonderful opportunity to access ALS drugs under investigation,” said Dr. Goyal
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 thrilled to launch the next EAP center at UCI, and are grateful to Axe ALS for this award,” says Merit Cudkowicz, MD, MSc, Director of the Healey & AMG Center for ALS, and Chair of the Department of Neurology at Mass General, “The EAP program has opened doors for patients, research, and innovation in ALS care and we look forward to furthering this success at UCI. Recently, as part of the TECHvsALS initiative a new EAP center was established at the Jefferson Weinberg ALS center. We are grateful for the support of industry and foundations to broaden access to investigational therapies for people living with ALS.”
Since its launch in 2018, the Healey & AMG EAP program has expanded across a nationwide network of clinical researchers to reach more individuals with ALS. axeALS Foundation’s commitment of $486,000 will allow UCI’s EAP program to enroll up to 30 participants over three years. The program is anticipated to begin accepting participants in the spring and early summer of 2023.
“We know first-hand how difficult living with ALS can be. After my husband’s diagnosis in 2019, we have been fighting for access to new therapies and searching for solutions,” said Amanda Stevens, Executive Director of axeALS Foundation. “When we met with the Healey & AMG Center, we knew we wanted to support their mission. Knowing that our grant will enable at least 30 individuals impacted by ALS to gain access to new therapies is an exciting start to what we hope the axeALS Foundation can achieve for the ALS community in the years to come.”
The axeALS Foundation is on a mission to raise awareness and funding for ALS treatments and clinical trials to help patients and their families live as full and normal lives as possible. The foundation was a product of Eric Stevens’ original GoFundMe campaign and was officially registered as a 501c.3 organization in 2020 to help others impacted with ALS. The foundation hosts multiple charity golf tournaments every year and accepts additional contributions through its website to further its mission.
For more information about the axeALS Foundation, visit www.axeals.org. For information about axeALS Foundation’s charity golf tournament series, visit https://axealsgolftournament.org/
To read more about EAP at the Healey & AMG Center, visit Expanding Access to Therapies (massgeneral.org).
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 four FDA-approved therapies for treating ALS —Riluzole, Nuedexta, Edaravone, and Relyvrio.
About axeALS Foundation
axeALS Foundation, a 501c3 non-profit public benefit corporation, was founded by Eric and Amanda Stevens after Eric was diagnosed with ALS at just 29 years old. Their 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 the Sean M. Healey & AMG Center for ALS at Mass General
At the 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 living with ALS, and families working together to accelerate the pace of ALS therapy discovery and development.
Under the leadership of Merit Cudkowicz, MD, chair of the Department of 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, and 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.