Charlottesville, VA, May 21, 2024 — The Focused Ultrasound Foundation and ALS Association are proud to announce their joint collaboration to fund a groundbreaking new research project exploring the role of focused ultrasound in amyotrophic lateral sclerosis (ALS) treatment. Also known as Lou Gehrig’s disease, this neurodegenerative disease leads to a loss of communication between the brain and muscles, causing progressive paralysis. The typical life expectancy for those diagnosed with ALS is two to five years. With more than 30,000 Americans affected by ALS and no known cure, new treatment approaches are urgently needed.
Focused ultrasound is a noninvasive therapeutic technology with the potential to transform the treatment of many medical disorders by using ultrasonic energy to target tissue deep in the body without the need for incisions or radiation. It is approved for nine indications in the U.S.—and more than 30 worldwide—and is in various stages of research and development for the treatment of nearly 180 serious diseases and conditions, including ALS.
The study, led by Agessandro Abrahao, M.D., M.Sc., assistant professor of neurology at the University of Toronto and an associate scientist at Sunnybrook Research Institute, will investigate whether focused ultrasound can help open the blood–brain barrier to allow a potential new treatment recently identified by University of Toronto researchers more easily reach motor neurons in the brain. The new molecule, called JRMS-22, may help clear the toxic clumps of a protein called TDP-43 that are found in the neurons of most people living with ALS and potentially prevent them from forming in the first place.
“Focused ultrasound is a versatile technology with several mechanisms of action, including drug delivery and gene therapy, which hold the promise of monitoring progression, alleviating ALS symptoms, and even halting the progression of the disease,” said Emily White, M.D., managing director of operations and FUS partners at the Focused Ultrasound Foundation. “Advancing research on neurodegenerative conditions like ALS is pivotal to the Foundation, and we arepleased to partner with the ALS Association on this important project. We are also grateful to have the support of donors passionate about neurodegenerative research, including the Eddie and Jo Allison Smith Family Foundation and the Red Gates Foundation; their commitment fuels our ability to pursue such promising treatments and make a meaningful difference in the lives of those battling ALS.”
The funded study is supported in part by a $500,000 ALS Association Partnership Grant awarded to the Foundation in 2023.
“We urgently need more and better treatments that can improve and extend the lives of people with ALS,” said Kuldip Dave, Ph.D., senior vice president of research at the ALS Association. “We are proud to partner with the Focused Ultrasound Foundation to support this innovative research project and learn more about how this novel technology could be used to help make ALS a livable disease.”
The Foundation has a long history of supporting research on neurodegenerative diseases, including a landmark study at Sunnybrook Health Sciences Centre in Toronto. This 2019 study, published in Nature Communications, demonstrated the safety and potential of focused ultrasound to temporarily open the blood–brain barrier in people living with ALS.
This collaborative effort underscores the importance of innovative, noninvasive approaches in treating challenging neurodegenerative diseases, offering renewed hope for those living with ALS and their families.
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About the Focused Ultrasound Foundation
The Focused Ultrasound Foundation was created in 2006 to improve the lives of millions of people worldwide in the shortest time possible by accelerating the development of noninvasive focused ultrasound technology. Based in Charlottesville, Virginia, the Foundation is a tax-exempt, high-performance, entrepreneurial service organization working to clear the path to global adoption of focused ultrasound by organizing and funding research, fostering collaboration, and building awareness among patients and professionals. The Foundation was recognized in 2018 as one of “America’s 10 Best Medical Research Organizations” by Charity Navigator, and is the largest nongovernmental source of funding for focused ultrasound research.
About the ALS Association
The ALS Association is the largest philanthropic funder of ALS research in the world. The Association funds global research collaborations, assists people with ALS and their families through its nationwide network of care and certified clinical care centers, and advocates for better public policies for people with ALS. The ALS Association is working to make ALS a livable disease while urgently searching for new treatments and a cure. For more information about The ALS Association, visit our website at als.org.