Since the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital launched the HEALEY ALS Platform Trial in 2020, Merit Cudkowicz, MD, MSc, executive director of the Mass General Brigham Neuroscience Institute and director of the Healey Center, and Sabrina Paganoni, MD, PhD, co-director of Mass General’s Neurological Clinical Research Institute, have undertaken a groundbreaking effort to support and empower the amyotrophic lateral sclerosis (ALS) community and accelerate drug development by testing multiple treatments simultaneously.
Dr. Paganoni recently received a new boost in her quest: a new $500,000, three-year grant from the Muscular Dystrophy Association (MDA) to support the trial. With these funds, Dr. Paganoni and her team can conduct additional analyses on patient biofluid samples they have collected during the trial — 60,000 in total — and further investigate effective treatments for ALS, a disease that currently has no cure.
“This grant comes at a critical time in our research,” Dr. Paganoni says. “We now have this wealth of resources, both in terms of samples and data, and we can leverage these funds to advance our research.”
“The HEALEY ALS Platform Trial represents a paradigm shift, significantly enhancing the speed and efficiency of testing potential treatments,” says Sharon Hesterlee, PhD, chief research officer at the Muscular Dystrophy Association. “The platform trial stands out for its bold and collaborative approach to ALS research. The focus on innovative trial design not only prioritizes scientific rigor but also ensures that ALS patients have greater access to experimental therapies.”
“Dr. Paganoni is not only a trailblazer in ALS research but also a deeply compassionate clinician who prioritizes her patients’ quality of life. Her dual roles as a researcher and care provider enable her to bridge the gap between the lab and the clinic, ensuring that her work directly benefits the individuals who need it most.”
A First for ALS Trials
Led by Dr. Cudkowicz, the HEALEY initiative is the first-ever ALS platform trial. In this format, instead of testing a single drug, clinical researchers can evaluate the performance of multiple drugs at the same time and keep testing new treatments as they are discovered. A platform trial approach translates to more opportunities to find effective therapies in a shorter timeframe.
The first four years have focused on development of an efficient, robust, patient-centric trial. Drs. Cudkowicz and Paganoni and their team collaborated with various industry partners to test treatments and, along the way, collected the additional biomarker samples. Now, they are positioned to capitalize on knowledge and data accumulated through the trial so far, share their data and resources with the ALS community and those researching other neurological diseases, and adapt the trial for even greater efficiency. As MDA’s grant is not tied to research into a specific treatment, the funding is essential in supporting these next steps.
“Our ability to conduct additional biomarker analyses and share placebo data and samples with the research community is only possible thanks to philanthropy,” Dr. Paganoni says. “The MDA grant gives us the opportunity to research these samples and continue to advance the science. This way, the trial’s mission continues to expand beyond testing individual drugs and impacts global research in ALS.”
A Legacy of Steadfast Support
MDA’s support for ALS research dates back to the 1950s, when Eleanor Gehrig became MDA’s campaign chairman, inspired by her late husband Lou Gehrig’s battle with the disease. Over the decades, MDA has helped fund pivotal studies and establish multidisciplinary care centers to improve patient care and outcomes. The organization has invested more than $176 million in research alone and helped launch more than 150 MDA Care Centers nationwide, including three at Mass General: the MDA Care Center at Mass General Neurology, the MDA/ALS Care Center at Mass General’s Wang Ambulatory Care Center; and the MDA Care Center at Mass General Pediatric Care.
“Mass General is a leader in medical innovation, known for its emphasis on interdisciplinary collaboration and patient-centered care,” Dr. Hesterlee says. “The institution’s commitment to fostering groundbreaking initiatives like the HEALEY ALS Platform Trial demonstrates its dedication to transforming how we approach treatment and care for devastating diseases.”
“Dr. Paganoni is not only a trailblazer in ALS research but also a deeply compassionate clinician who prioritizes her patients’ quality of life,” Dr. Hesterlee adds. “Her dual roles as a researcher and care provider enable her to bridge the gap between the lab and the clinic, ensuring that her work directly benefits the individuals who need it most. Her vision and leadership have set a new standard for how clinical trials are designed and conducted, making her a vital partner in Muscular Dystrophy Association’s mission to end ALS.”
To learn how you can support the Healey & AMG Center for ALS, contact us.
