Researching Focused Ultrasound to Ease Delivery of Gene Therapy to the Brain

At a Glance

  • Studying focused ultrasound’s ability to open up the blood-brain barrier to deliver gene therapy agents deep within the brain
  • Potential to treat conditions including Parkinson’s and Alzheimer’s disease

"This new technology provides a 21st century solution to a problem that’s been around for a long time and continues the trend in neurosurgery toward offering less invasive therapies for a variety of brain disorders."

— Dr. Michael G. Kaplitt

Focused ultrasound has the potential to transform treatment of tremors by gaining access deep within the brain without harming healthy tissue. It also makes possible the reversible opening of the blood-brain barrier to deliver therapeutic agents to targeted diseased areas.

Dr. Michael Kaplitt became the first physician in New York to use high intensity focused ultrasound (HIFU) treatment in the brain as part of the clinical trial in July 2016. During that procedure, he watched a trial participant’s tremors disappear in a matter of minutes. “This new technology provides a 21st century solution to a problem that’s been around for a long time and continues the trend in neurosurgery toward offering less invasive therapies for a variety of brain disorders,” said Dr. Kaplitt, who is an associate professor of neuroscience and neurological surgery at Weill Cornell Medicine and a neurosurgeon at NewYork-Presbyterian/Weill Cornell Medical Center.

New device being studied in animal models

Weill Cornell researchers are now studying in animal models a device that uses the same type of ultrasound energy, but at a much lower level. The device does not destroy part of the brain, but rather opens up the blood-brain barrier to allow gene therapy agents and other treatments to pass through into the brain tissue itself.

Instead of injecting the viruses that harbor these genes directly into the brain as is done in human studies and previous animal studies, the researchers have been able to do a simple intravenous injection of the gene therapy agents and show that the area targeted with the ultrasound will take up the gene therapy agents – all performed very precisely and very efficiently.

Eventually, he said, scientists could study the technique’s application to a variety of neurological disorders ranging from Alzheimer’s disease to addiction. His laboratory has also been investigating ways to use HIFU to non-invasively deliver gene therapies to specific brain regions.

Dr. Kaplitt previously had developed techniques for performing gene therapy in the brain using invasive injections, and performed the first human clinical trial of gene therapy for Parkinson’s disease at Weill Cornell.

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