New Partnership Will Advance Focused Ultrasound in ALS Diagnosis and Treatment


Key Points

  • The Foundation has partnered with The ALS Association to advance transformational treatments for people with amyotrophic lateral sclerosis (ALS).
  • Also known as Lou Gehrig’s disease, ALS is a progressive neurodegenerative disease for which there is no cure.
  • A formal request for applications (RFA) will be issued later this year, with $1 million in research funding available.

The Focused Ultrasound Foundation has partnered with The ALS Association to advance transformational treatments for people with amyotrophic lateral sclerosis (ALS). Leveraging a $500,000 ALS Association Partnership Grant and $500,000 of its own funds, the Foundation will support research into diagnosing and treating ALS using focused ultrasound technology.

Also known as Lou Gehrig’s disease, ALS is a progressive neurodegenerative disease that affects more than 30,000 people in the US. The disease damages nerve cells in the brain and spinal cord, causing them to degenerate and die over time. Ultimately, people with ALS lose the ability to speak, eat, move, and breathe on their own. There is no cure for ALS, and most people with the disease die within two to five years of diagnosis.

“We believe that focused ultrasound could be a valuable tool in advancing the diagnosis and treatment of ALS,” said Foundation Chairman Neal F. Kassell, MD. “There is a clear unmet clinical need for these patients, and this partnership will provide critical ALS-specific expertise to the focused ultrasound community, exploring a totally new approach to facilitate drug, gene, and stem cell delivery. Moreover, it is our goal that this funding will lead to promising preliminary data that can be leveraged to secure larger investments necessary to propel the field toward clinical translation and adoption of promising therapies to make ALS a livable disease.”

For more than 15 years, the Foundation has been dedicated to advancing the development and adoption of focused ultrasound technology. Since its inception, one of the Foundation’s priorities has been brain research, and it funded, in part, an initial clinical trial that proved it is possible to safely use magnetic resonance-guided focused ultrasound to temporarily, and reversibly, open the blood-brain barrier (BBB) in people with ALS. This groundbreaking study took place at Sunnybrook Health Sciences Centre in Toronto and was published in Nature Communications in 2019.

The Foundation and The ALS Association intend to build on this research by collaborating to extend up to $1 million in research funding to develop innovative treatment platforms for ALS. Novel strategies that will be considered for this funding opportunity include – but are not limited to – employing focused ultrasound–initiated BBB opening and targeted drug delivery to enhance delivery of gene therapies, viral vectors, and stem cells. Projects involving the use of BBB opening to enable liquid biopsy to help diagnose and monitor disease progression are also of interest.

“There is an urgent need for new ways to diagnose and treat ALS. We are excited to partner with the Focused Ultrasound Foundation at this early stage to learn more about how this novel technology could ultimately transform the experience of ALS,” said Kuldip Dave, PhD, senior vice president for research at The ALS Association. “Our research program is built around de-risking innovative ideas to attract follow-on funding that can move the work forward past our initial grant. By joining forces with the Focused Ultrasound Foundation, we have an even greater opportunity to pay forward more and better chances of improving the lives of people with ALS.”

The ALS Association is the largest philanthropic funder of ALS research in the world, working to make ALS a livable disease while urgently searching for new treatments and a cure. It 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.

A formal request for applications (RFA) will be issued in the coming months. Research projects will be vetted by a designated advisory panel.

Help Support Similar Neurodegenerative Projects
The Focused Ultrasound Foundation has developed a Neurodegenerative Program to help advance focused ultrasound for a wide variety of disorders, such as Alzheimer’s disease, Parkinson’s disease, ALS, dystonia, essential tremor, Huntington’s disease, and more. Last year, our generous donors succeeded in fulfilling a matching gift challenge, raising a total of more than $5.5 million for the program. This funding will support a pipeline of more than 20 focused ultrasound projects including this one with The ALS Association, but there are still immense unmet needs for patients with these disorders. If you are interested in supporting our neurodegenerative program, please contact the Foundation’s Managing Director, Jessica Lukens.