A pilot study for brain delivery of Cerezyme® in people with Parkinson’s disease using MR-guided focused ultrasound induced opening of the blood-brain barrier

Project Name: A pilot study for brain delivery of Cerezyme® in people with Parkinson’s disease using MR-guided focused ultrasound induced opening of the blood-brain barrier
Status: Enrollment Complete
Primary Site: Sunnybrook Health Sciences Centre
Funded Date: Sep 30, 2020
Funding Period: Sep 30, 2020 - Oct 31, 2020
Funding Program: Brain Program
Category: Clinical Research
Amount Awarded: $80,000.00

Abstract

Parkinson’s disease (PD) is the second most common neurodegenerative disease. Although numerous potential disease-modifying therapeutics have been proposed, these are typically limited from entering the brain by the blood-brain barrier (BBB). To date, approaches to overcome the BBB, such as convection enhanced delivery, have relied on open surgery, and it’s inherent risks. MR-guided focused ultrasound (MRgFUS) is an incisionless, emerging approach for drug delivery that temporarily permeabilizes the BBB, the enhanced delivery of compounds delivery intravascularly. This can be achieved without surgery, and in a targeted fashion to deep parts of the brain involved in PD pathology. We are investigating whether MRgFUS induced BBB opening is safe and feasible in human PD patients, to determine it’s role as a drug delivery tool for PD.

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