Dalhousie University professor co-authors medical breakthrough

A new gene therapy developed at Dalhousie University is bringing hope for Canadians with a rare gene mutation called Fabry disease. Paul Hollingsworth reports.

There is new hope for a Nova Scotia man thanks to groundbreaking gene therapy research from a Dalhousie University professor.

Ryan Deveau suffers from Fabry disease, a rare disorder that leaves the body unable to produce the enzyme that breaks down fatty materials.

Deveau says he’s faced a heavy load of health issues living with the disease.

“Because I don’t produce that enzyme, there is a build up around certain organs like my heart, my kidneys and potentially my brain that my body can’t break down,” said Deveau, 44, who has lived with the disease since he was young.

Fabry disease can also cause major organ damage and a shortened lifespan. Due to the health risks involved in his everyday life, Deveau usually received enzyme replacement therapy, which required routine trips to a specialist.

Then he met Dalhousie University’s Dr. Michael West, who is also a nephrologist at the QEII Health Sciences Centre in Halifax. West co-authored research on gene therapy designed to help people suffering from the disease.

“We designed the study to look at the feasibility of gene therapy for Fabry disease, and the safety of the procedure,” said West. “We were the first gene therapy trial in Fabry disease.”

The trial started in 2016 and involved five patients, including Deveau. In simple terms, researchers did not treat the gene linked to Fabry disease, but instead, they used stem cells from the patient’s bone marrow to produce a copy of a normal gene as a new way to treat the disease.

”After six years, we have evidence that the new gene is still in these patients, and it is still working,” said West.

There is no cure for Fabry disease, but Deveau is happy with the results so far.

“I feel great and I’ve got very little to no organ damage at all,” said Deveau, adding that participating in this research trial has already simplified the process of his medical treatment while improving the overall quality of his life.

“With the gene therapy, it’s a short couple of months as a patient.”

Gene therapy research will need to go through larger scale studies before it becomes a traditional medical treatment. For now, it has helped improved Ryan Deveau’s health as he continues to live with the disease.