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Focused Ultrasound for Parkinson’s Disease: Results of First Clinical Trial to Deliver Therapeutics

Key Points

  • The results of an early-stage clinical trial investigating focused ultrasound to address a possible underlying cause of Parkinson’s disease were published in Movement Disorders.
  • Researchers in Toronto tested the safety and efficacy of focused ultrasound–induced blood-brain barrier opening to deliver enzyme replacement therapy.
  • The procedure was well-tolerated, and a larger study is being planned.

Excerpt from the Press Release:

The results of a groundbreaking clinical trial investigating focused ultrasound to enhance drug delivery and address a possible underlying cause of Parkinson’s disease (PD) were recently published in Movement Disorders.

Researchers from Sunnybrook Health Sciences Centre and University Health Network (UHN) in Toronto used focused ultrasound to disrupt the blood-brain barrier (BBB) in patients with early-stage PD to deliver an enzyme replacement therapy called imiglucerase – or Cerezyme®.

The BBB is a protective layer of tightly joined cells that lines the blood vessels in the brain and prevents harmful substances, such as toxins and infectious agents, from diffusing from the blood into the surrounding brain tissue. However, it can also prevent beneficial therapeutic agents from getting into the brain in high enough concentrations to be effective. Focused ultrasound has been proven to safely and temporarily disrupt this barrier, and in this study, researchers aimed to open the BBB to allow delivery of the enzyme to the putamen – a key structure in the brain related to movement disturbances. 

Some patients with PD can have a deficiency of a naturally occurring enzyme called glucocerebrosidase. Enzyme replacement therapy could be one approach to reduce or prevent neurodegeneration in PD.

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