Hamilton researcher set to manufacture potential treatment for genetic diseases
A leading McMaster researcher has set up a biotech company in Hamilton and is looking to start manufacturing a potential treatment for genetic diseases.
Dr. Mark Tarnopolsky plans to establish a manufacturing facility in Ontario — preferably in Hamilton — and is in talks with pharmaceutical and biotech companies to also make the treatment for mitochondrial disease, muscular dystrophy and a wide variety of genetic disorders in the United States.
"We're in negotiations to license our technology," said Tarnopolsky, who is the only physician in Canada to focus the majority of his practice on mitochondrial diseases http://www.hamiltonhealthsciences.ca/documents/Patient%20Education/MitochondrialDiseaseUnderstanding-lw.pdf. "We're also designing a facility to manufacture."
The potential of a new treatment comes as advocacy and support group MitoCanada http://www.mitocanada.org/ holds a major fundraising event Saturday at Confederation Park from 11 a.m. to 4 p.m. to fund research like Tarnopolsky's.
"We keep hoping and hoping with the money that gets raised, they'll find a cure," said Chuck Gibson, whose 68-year-old wife, Betty Gibson, has been debilitated by a hereditary mitochondrial disease that his children and grandchildren could also get. "It's like cancer but it doesn't have the notoriety of that disease."
His wife was told by doctors that she was "just aging" when she complained of hearing loss, lack of energy and weight loss. But it became clear something was very wrong during a trip to New York City in August 2011.
"We had a bus trip through the city and through the whole trip she stared straight ahead and didn't comment to me about anything," said Gibson.
He immediately drove his wife straight back to Hamilton General Hospital where she was at first misdiagnosed as having a stroke. Despite Tarnopolsky working out of McMaster University and McMaster Children's Hospital, it took months before a doctor recognized he should be called in.
"It was like someone breathed some life into us," said Gibson about Tarnopolsky coming straight to her hospital bedside on his way home from a trip to Africa. "She laid there disappearing before my eyes. She was down to 79 pounds. I was at my wit's end."
Treatments researched by Tarnopolsky have helped the Gibsons go home and live a quality life. But there is currently no cure. She tires quickly and has difficulty with memory and communication.
Tarnopolsky's company, Exerkine Corporation, is hoping to dramatically change the prognosis for those with genetic diseases. The company, which has ties to McMaster University and is located there, is preparing to start clinical trials on the new treatment once the manufacturing facilities are in place.
Early results in the lab have been "spectacular" said Tarnopolsky.
His work is promising enough to win a 2015 Canadian Institutes of Health Research Foundation Grant, which provides the nation's top researchers with stable, long-term funding for ambitious, high-impact studies.
Tarnopolsky would like to "give back" to the community where he's lived and worked by setting up the manufacturing facility in Hamilton but the details have yet to be worked out.
It will be at least five years before the treatment has been proven in clinical trials, approved by Health Canada and made available to patients.
In the meantime, families like the Gibsons will come from all over Ontario to meet at Confederation Park for the all-day event that will inform about the latest advancements, inspire with speakers and a film, raise around $25,000 with a walk-a-thon and spread awareness by creating a human ribbon that will be photographed from the sky.
"It's important for us to start to educate the public that this is an important disorder to pay attention to," said Maureen Latocki, executive director of MitoCanada, which is based in Brantford. "Mitochondrial dysfunction has been connected closely to many other known diseases."
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