The ALS Association and Focused Ultrasound Foundation have partnered to support a small pilot clinical study that will leverage focused ultrasound technology as part of a new ALS treatment approach. The study will be led by Agessandro Abrahao, M.D., an assistant professor of neurology at the University of Toronto and an associate scientist at Sunnybrook Research Institute.

“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.”

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. An initial clinical trial showed how focused ultrasound can be used to temporarily, and reversibly, open the blood-brain barrier (BBB) in people with ALS. The BBB helps protect the nervous system by tightly controlling what can pass from the blood to the brain and spinal cord, often making it difficult for potential ALS therapies to get to the motor neurons being damaged by the disease.

Dr. Abrahao’s study will build on this research by using focused ultrasound to help deliver a potential new treatment recently identified by University of Toronto researchers to the brains of four people living with ALS. The new molecule, called JRMS-22, may help clear toxic clumps of a protein called TDP-43 that are found in the neurons of most people affected by the disease 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 are pleased to partner with the ALS Association on this important project." 

The funded study is supported in part by a $500,000 ALS Association Partnership Grant awarded to the Focused Ultrasound Foundation in 2023. These grants help partner organizations grow, scale, or augment their own research funding programs, building co-funded collaborations that are larger or more impactful than if partners were pursuing them alone.

"In a complex disease like ALS, no organization, entity, or individual can really solve this challenge alone. Collaboration and coordination are vital to accelerating the development of new treatments and cure—getting us further faster," said Dr. Dave.

Click here to learn more about focused ultrasound and Dr. Abrahao’s research.