ALS is a complex neurodegenerative disease, but the causes remain poorly understood and there are few effective therapies available. There is an urgent need for large-scale, collaborative efforts to identify useful biofluid biomarkers that can guide researchers about its onset, progression, and response to effective therapies.
To achieve these goals, the NIH has established a transformative national ALS clinical research consortium called Access for All in ALS (ALL ALS). This new consortium is a multi-institutional effort and aims to disrupt the ALS clinical research landscape in the U.S. It will operationalize recommendations of the Accelerating Access to Critical Therapies for ALS Act (ACT for ALS) Public Private Partnership. This grant is made possible by the Accelerating Access to Critical Therapies for ALS Act, which was signed into law in December 2021.
The consortium will be led by principal investigators at the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital (MGH) (Drs. Suma Babu, James Berry, and Sabrina Paganoni); the Barrow Neurological Institute (Drs. Bob Bowser and Jeremy Shefner); Columbia University (Drs. Jinsy Andrews, Matthew Harms, Neil Shneider, Hemali Phatnani, and Frank Provenzano); and Georgetown University (Dr. Brent Harris).
ALL ALS will include two clinical coordinating centers: the ALL ALS East Clinical Coordinating Center, located at the Neurological Clinical Research Institute (NCRI) of MGH, in Boston, MA, and the ALL ALS West Clinical Coordinating Center at the Barrow Neurological Institute in Phoenix, AZ. The clinical coordinating centers will oversee 34 clinical trial sites across the United States and Puerto Rico. Data collection for both clinical coordinating centers will be managed by the MGH NCRI under the leadership of Alexander Sherman, Director of the Center for Innovation & Bioinformatics.
The MGH NCRI brings extensive experience functioning as a multicenter research coordination center and leading scores of high-quality ALS biobanking studies and clinical trials, in partnership with industry, academic investigators, and not-for-profit research groups.
The Barrow Neurological Institute is a leading center for ALS biobanking with extensive experience in biomarker studies and is a national leader in managing national and international ALS clinical trials and leads the Target ALS longitudinal biofluids consortium.
Columbia University Irving Medical Center is a leader in developing novel, personalized medicines for people with rare genetic forms of ALS and in diagnosing and treating patients with presymptomatic ALS mutations.
The ALL ALS consortium will collect clinical and biomarker data from people with ALS symptoms, presymptomatic individuals at risk for ALS , and healthy controls. To foster wide representation across populations, data will be collected from both remote and in-person visits, including many clinical sites located in geographical areas with underrepresented minorities. Data will be collected and harmonized in a single central data portal to enable information sharing and dissemination with researchers worldwide.
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.
For more information on the Sean M. Healey & AMG Center for ALS and the Neurological Clinical Research Institute, please visit our website.
About Barrow Neurological Institute Gregory W. Fulton ALS and Neuromuscular Disease Center
Barrow Neurological Institute at Dignity Health St. Joseph's Hospital and Medical Center in Phoenix, Arizona is internationally recognized as a leader in neurosurgery, neurology, neurological research and patient care. Consistently voted among the top facilities in the United States for neurological care, Barrow treats patients with a wide range of neurological conditions, including ALS, brain and spinal tumors, cerebrovascular conditions, concussions and neuromuscular disorders. Its surgeons perform more neurosurgeries annually than any hospital in the nation. Barrow is proud to be the home of the Gregory W. Fulton ALS and Neuromuscular Disease Center, the largest ALS clinic in the southwest. The Barrow is a leader in biobanking efforts for ALS, performs clinical trial training and monitoring at sites throughout the country and internationally, and leads Target ALS funded natural history and tissue banking programs in ALS. For more information, please visit barrowneuro.org.