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Sunnybrook research helps take future treatment of ALS patients to a new level

September 26, 2019

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Peer-reviewed study has shown the safety of using focused ultrasound in opening the blood-brain barrier in patients with ALS

Sunnybrook researchers are setting the stage for new possibilities in the future treatment of amyotrophic lateral sclerosis (ALS) after a groundbreaking peer-reviewed study published in Nature Communications has shown the safety of using focused ultrasound (FUS) in opening the blood-brain barrier (BBB) in patients with ALS.

“ALS is a devastating and terminal neurological disease,” says Dr. Lorne Zinman, study senior author and director of Sunnybrook’s ALS Clinic. “The successful results of this world-first trial provides an innovative way to directly and non-invasively access the brain to test the most promising ALS therapeutics to slow and one day stop disease progression.”

FUS harnesses the power of up to a thousand sound waves and in this study, was used at a low frequency to target a specific area of the brain. The ultrasound causes micro-bubbles that have been injected into the bloodstream to vibrate, which gently and temporarily, opens the BBB.

The BBB is a structure of cells around the brain that protects it from toxins or bacteria in the bloodstream. However, it also blocks potentially helpful medication and therapeutics from reaching the brain.

“Our research team accomplished another world-first in this study by also safely targeting the motor cortex, a part of the brain affected by ALS, with MR-guided FUS,” says Dr. Agessandro Abrahao, the study’s first author and investigator at Sunnybrook’s Harquail Centre for Neuromodulation. “The technology enabled us to open the BBB in this part of the brain that controls the body’s voluntary movements.”

“FUS is leading edge, non-invasive technology that is a game-changer in terms of accessing areas of the brain to help treat patients like never before,” says Dr. Nir Lipsman, co-author of the study and director of Sunnybrook’s Harquail Centre for Neuromodulation. “While this is an early first step, our goal is to investigate novel, direct-to-brain interventions for the most challenging brain conditions, like ALS. In the coming months, we will begin the next phase of the study which will involve the delivery of an ALS therapeutic.”

Also known as Lou Gehrig’s disease, ALS affects about 3,000 people in Canada. It is a degenerative motor neuron disease in which the brain gradually stops sending messages to muscles. Over time, patients can experience paralysis and the inability to talk, eat and breathe. There is no cure for ALS. Sunnybrook has the largest ALS Clinic in Canada, which sees 250 new patients diagnosed every year.

“The Sunnybrook research team is unsurpassed at pioneering new approaches for focused ultrasound to treat a variety of challenging brain disorders,” says Neal F. Kassell, MD, Chairman of the Focused Ultrasound Foundation. “This report documents the first step in a path of clinical trials that could lead to a novel treatment for certain ALS patients, and it is leading the way for this technology to help enhance future treatment options for many patients. The Focused Ultrasound Foundation is pleased to have had the opportunity to support this trial and looks forward to participating in future research in this area.”

For nearly 20 years, Dr. Kullervo Hynynen, Director of Physical Sciences at Sunnybrook Research institute worked with INSIGHTEC, a global medical technology innovator of incisionless brain surgery, to develop the FUS technology.

Philanthropic investment has been a major driver behind Sunnybrook’s innovative research in FUS. This trial was made possible with leading support from the Temerty Foundation, the Harquail Family, ALS Society of Canada and Focused Ultrasound Foundation.

For more information about FUS at Sunnybrook Health Sciences Centre, visit sunnybrook.ca/focusedultrasound


Media contact:

Jennifer Palisoc
Communications Advisor, Sunnybrook Health Sciences Centre
416-480-4040
jennifer.palisoc@sunnybrook.ca

About the Focused Ultrasound Foundation

The Focused Ultrasound Foundation was created to improve the lives of millions of people worldwide by accelerating the development of focused ultrasound, an early-stage, noninvasive, therapeutic technology with the potential to transform the treatment of many serious medical disorders. The Foundation works to clear the path to global adoption by coordinating and funding research, fostering collaboration, and building awareness among patients and professionals. It is dedicated to ensuring that focused ultrasound finds its place as a mainstream therapy for a range of conditions within years, not decades. Since its establishment in 2006, the Foundation has become the largest non-governmental source of funding for focused ultrasound research. More information can be found at fusfoundation.org.

About INSIGHTEC

INSIGHTEC® is a global medical technology innovator transforming patient lives through incisionless brain surgery with MR-guided focused ultrasound. Research for future applications in the neuroscience space is underway in partnership with leading academic and medical institutions. INSIGHTEC is headquartered in Haifa, Israel, and Miami, with offices in Dallas, Tokyo and Shanghai.

About Sunnybrook

Sunnybrook Health Sciences Centre is inventing the future of health care for the 1.3 million patients the hospital cares for each year through the dedication of its more than 10,000 staff and volunteers. An internationally recognized leader in research and education and a full affiliation with the University of Toronto distinguishes Sunnybrook as one of Canada’s premier academic health sciences centres. Sunnybrook specializes in caring for high-risk pregnancies, critically-ill newborns and adults, offering specialized rehabilitation and treating and preventing cancer, cardiovascular disease, neurological and psychiatric disorders, orthopaedic and arthritic conditions and traumatic injuries. The Hospital also has a unique and national leading program for the care of Canada’s war veterans.