‘World first’ Parkinson’s treatment undergoing clinical trial in Toronto

Sunnybrook's Dr. Nir Lipsman is co-leading a study with Dr. Lorraine Kalia and Dr. Suneil Kalia of University Health Network on a new treatment for Parkinson's . Sunnybrook Health Sciences Centre

Researchers in Toronto are in the early stages of developing what’s being called a “world-first” treatment for Parkinson’s disease.

Currently in its first phase of clinical trials, the ultimate goal of the new treatment would be to treat symptoms of Parkinson’s, prevent further decline in patients and reduce the amount of medications that people need to take for the illness, according to Dr. Nir Lipsman, the study’s co-principal investigator and a neurosurgeon at Sunnybrook Health Sciences Centre.

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“That’s ultimately the goal — to change both the day-to-day activities of patients but also the course of their illness,” he said. “We’re still, again, (in) early days, but this is a critical first step.”

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Parkinson’s is a neurodegenerative disorder where patients experience tremors, muscle rigidity and have difficulty with balance, among other symptoms.

More than 100,000 people in Canada have been diagnosed, according to Parkinson Canada, and there is no cure.

The Toronto researchers, who are based at Sunnybrook as well as University Health Network, are using a focused ultrasound technology to deliver a treatment directly to the regions in the brain that are affected by Parkinson’s.

This is done by non-invasively opening a passage in the blood-brain barrier — the physical obstacle in the body that prevents compounds, including potentially useful therapies, from gaining access to the brain, Lipsman said.

“What an ultrasound allows us to do is create a kind of temporary window in that blood-brain barrier to allow the delivery of therapies to the brain that ordinarily cannot get in,” he added.

“Now what we can do is open the blood-brain barrier entirely non-invasively with the patient in the MRI scanner, so they don’t have to have skin incision or holes in the skull.”

Researchers are looking at whether delivering an enzyme called Glucocerebrosidase to the brain will help prevent the build-up of a protein that is associated with Parkinson’s called alpha-synuclein.

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The goal for this phase of the trial is to have six people undergo three rounds of treatment, with follow up for at least six months. Three patients are already signed up.

Pat Wilson, 56, of Cookstown, Ont., is the first person to participate in the study. While she hasn’t had improvement so far, she has had to adjust her medication because she’s seen a change in how her body reacts to it.

Wilson was diagnosed with Parkinson’s in 2013 and her father also had the disease. She wanted to participate in the trial to try to help others.

“It’s important because we need better treatments, longer-lasting treatments, maybe. Anything to help people in the future who might get it.”

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Over time, as the Toronto researchers gain experience and conduct additional trials, Lipsman said they hope they can demonstrate that they’re moving the dial when it comes to preventing neurodegeneration from taking place.

“If we can do that, then we can prevent disability, we can improve quality of life and we can prolong life as well,” he said.

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