Opening the Blood-Brain Barrier: New Treatment for Parkinson’s in Phase 1 Trial

Photo of Lorraine Kalia, a lead investigator into a first-of-its-kind therapy for Canadians living with Parkinson’s

A new Phase 1 clinical trial has been in the news this month offering hope and enthusiasm in the field of Parkinson’s research. This project led by Canadian researchers at Toronto’s Sunnybrook Health Sciences Centre and University Health Network would be a first-of-its-kind therapy for Canadians living with Parkinson’s. 

Lorraine Kalia, one of the lead investigators and a member of Parkinson Canada’s Scientific Advisory Council and Julie Wysocki, Director of Research Program and Partnerships share their reaction to this meaningful trial and its early results. 

Parkinson Canada directly supports research into potential new treatments for Parkinson’s and monitors the research landscape for new developments with potential application for Canadians with Parkinson’s. 

“New treatment options for Parkinson’s are always welcome.  The addition of a non-invasive treatment that can temporarily open the blood-brain barrier to deliver medications is very exciting news for the Parkinson’s community. Current treatments, including medications and deep-brain stimulation, help manage the symptoms, but none yet reverses or stops the progression of the disease, says Julie Wysocki, Director of Research Program and Partnerships with Parkinson Canada, “That would definitely be the holy grail,” she adds. 

What is the study?

Excerpt taken from Sunnybrook Media Release:

A team of researchers from Sunnybrook Health Sciences Centre and University Health Network are using focused ultrasound technology to deliver a therapeutic directly to affected brain regions in patients with Parkinson’s.

Focused ultrasound harnesses the power of ultrasound waves to reach deep brain regions without the need for scalpels or cutting. In this study, researchers are using low intensity MRI-guided focused ultrasound to open the blood-brain barrier, which protects the brain from toxins but can also block potentially helpful medications.

“The goal of this Phase I trial is to examine the safety of temporarily opening the blood-brain barrier in key motor regions known to be implicated in Parkinson’s disease as well as delivering promising therapeutics directly to these areas of the brain,” says Dr. Nir Lipsman, the study’s co-principal investigator and Director of Sunnybrook’ Harquail Centre for Neuromodulation.

A key hallmark of Parkinson’s is the abnormal accumulation of a protein called alpha-synuclein in the brain which leads to unhealthy brain cells and neurodegeneration. Promising treatments to reduce alpha-synuclein accumulation, however, are limited by their inability to cross the blood-brain barrier.

Study researchers are investigating the delivery of an enzyme called glucocerebrosidase to the putamen, a key structure in the brain related to movement. Glucocerebrosidase helps prevent buildup of alpha-synuclein but in Parkinson’s the enzyme can be defective, leading to Parkinson’s symptoms. Enzyme replacement therapy is one potential strategy to reduce or prevent neurodegeneration in Parkinson’s.

In the study, low-intensity ultrasound waves target the putamen, a critical motor structure. When ultrasound interacts with tiny-microscopic bubbles injected in the bloodstream before the treatment, the bubbles rapidly vibrate, causing a temporary opening in the blood-brain barrier. This opening, which closes within hours of the procedure, allows the direct delivery of an enzyme replacement therapy, administered simultaneously with focused ultrasound.

Watch the video below to learn more, and meet Pat Wilson, the first patient involved in the trial: 

What does it mean?

Although this trial is in phase 1 and it maybe years until it is approved widely, we are hopeful that this trial will lead to a new treatment and to improving the lives of People Living with Parkinson’s. This trial is an exciting first step in the use of focused ultrasound and its potential to help deliver medications more effectively through opening the blood brain barrier.  

It’s important to remember we are still in the very early stages of this clinical trial, and while there is currently no cure for Parkinson’s, we are investigating whether using MRI-guided focused ultrasound to allow for enzyme replacement in the brain could be a promising approach to reduce or stop neurodegeneration in Parkinson’s disease,” explains Dr. Lorraine Kalia, co-principal investigator on the trial and a neurologist and scientist at the Krembil Brain Institute, part of University Health Network (UHN).

“I think we’re really at the beginning of a very exciting time in terms of discovering and developing treatments for people with Parkinson’s disease. This is going to be the first step of hopefully a large amount of research looking into different drugs that we could deliver in this way. I am cautious but also optimistic that this will be a path to lead us to a cure for Parkinson’s disease.”

Editor’s Note:

Dr. Lorraine Kalia is a member of the Parkinson Canada Scientific Advisory Council and is a co-lead investigator in this study, along with her husband Dr. Suneil Kalia and Dr. Nir Lipsman. Both Lorraine and Suneil Kalia have received grants funded by the Parkinson Canada Research Program. 

Suneil Kalia: Eliminating ‘bad chaperone’ proteins to find a cure for Parkinson’s 

Lorraine Kalia: Misbehaving Proteins: Alpha-synuclein in LRRK2-related Parkinson’s Disease

6 thoughts on “Opening the Blood-Brain Barrier: New Treatment for Parkinson’s in Phase 1 Trial”

    • Awesome a person with frozen gait for four years I am very interested in what you do for your patients.plexase keep me informed. Thanks.wally Boyer

  1. Very impressive methodology. Multi steps to create a temporary state of open access through the blood brain barrier; access that self reversed with time.
    Also promising possibility you can create access to enzymes as well as therapeutic agents. How is the procedure for selecting candidates working?
    Thank you for telling this research story in its conceptual fullness.
    It makes it possible to more clearly see success in treatment.


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