Key Points
- Researchers in Toronto are using focused ultrasound to open the blood-brain barrier and deliver an immunotherapy drug in patients with amyotrophic lateral sclerosis (ALS).
- The Foundation is partially funding this clinical trial.
Researchers at Sunnybrook Health Sciences Centre in Toronto have launched a new clinical trial testing focused ultrasound–induced blood-brain barrier (BBB) opening plus an immunotherapy drug in patients with amyotrophic lateral sclerosis (ALS). This trial represents a world’s first milestone, as no previous studies have used the combination treatment in this patient population.
Also known as Lou Gehrig’s disease, ALS is a condition that affects up to an estimated 30,000 people in the US. As the disease progresses, the brain stops sending messages to voluntary muscles. There is no cure.
The BBB is a protective layer of tightly joined cells that lines the blood vessels of the brain and keeps harmful substances, such as toxins and infectious agents, from entering the surrounding brain tissue. Unfortunately, this barrier also prevents certain drugs and potentially our own immune cells from reaching their targets within the brain in adequate concentrations. The BBB can make it challenging to treat conditions such as ALS because the drugs cannot reach their targets in the brain.
The six-patient trial will assess the safety, tolerability, and preliminary biological effects of using focused ultrasound to temporarily and reversibly disrupt the BBB and allow intravenous immunoglobulin (IVIg), a potential modulator of ALS-related neuroinflammation, to enter the brain. Also, for the first time, the focused ultrasound will be delivered using the Next Generation Dome Helmet, a new device that was developed by Kullervo Hynynen, PhD, senior scientist and vice president of research and innovation at Sunnybrook Research Institute.
The Focused Ultrasound Foundation is partially funding this clinical trial, which is being co-led by Agessandro Abrahao, MD, and Lorne Zinman, MD, neurologists in the Hurvitz Brain Sciences Program, as well as Nir Lipsman, MD, PhD, a neurosurgeon who is the director of the Harquail Centre for Neuromodulation and chief of the Hurvitz Brain Sciences Program.
This clinical trial builds upon the team’s earlier research published in Nature Communications in 2019 that suggested it is possible to safely use focused ultrasound to temporarily and reversibly open the BBB in patients with ALS.
“This world-first clinical trial highlights both the promise of focused ultrasound and the impact of technological innovation in medicine,” said Neal F. Kassell, MD, founder and chairman of the Focused Ultrasound Foundation. “By introducing this innovative device, we also move closer to providing accessible, personalized treatment options for ALS and other devastating brain disorders, offering new hope to patients worldwide.”
Read Sunnybrook’s Press Release
See media coverage from CTV News, The Canadian Press, and The Toronto Star.