Calgary man first in the world to receive experimental gene therapy
Researchers will be monitoring Darren Bidluka for the next five years to see if his condition improves
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A Calgary man has become the first patient in the world to be treated with gene therapy for a disease other than cancer.
Darren Bidulka has Fabry disease, a rare inherited metabolic disorder that creates a specific enzyme deficiency that can cause damage to major organs, chronic pain, and gastrointestinal difficulties.
Approximately 420 Canadians are affected by the disease.
“My hope is that this is a long-term solution for people with Fabry disease and other genetic disorders,” Bidluka said, describing the treatment process as “absolutely fascinating.”
Those with Fabry disease have a gene known as ‘GLA’ that malfunctions, meaning they can’t produce the correct enzyme to break down a fat called Gb3.
If Gb3 builds up in the body, it can cause problems in the kidneys, brain, and heart.
Calgary researchers conducted an experimental trial by taking Bidulka’s own blood stem cells and manipulated them with a specially engineered virus, augmenting the cells with fully-functioning copies of the GLA.
These modified stem cells were transplanted back into Bidulka in January.
“Now that I’m on the road to recovery I’m super excited that this could be the leading edge of some big developments in the medical community,” Bidluka said.
Dr. Aneal Khan, medical geneticist for Alberta Health Services (AHS) and member of the Alberta Children’s Hospital Research Institute with the Cumming School of Medicine at the University of Calgary, led the trial.
“It’s exciting, I’m in a field of medicine where we often don’t have any effective treatment or hope for patients to recover when they get diagnosed,” Dr. Khan said.
He hopes the therapy can be used to treat similar conditions, but cautioned that the trial is only experimental.
“We will be monitoring (Bidluka) over the next five years to see what improvements, if any, take place,” Dr. Khan said.
“We’re still a long way from this being a standard process.”
The treatment has been approved for experimental purposes only by Health Canada.