University of Calgary

National clinical trial comes to Calgary

UToday HomeJanuary 28, 2013

Medical geneticist Dr. Aneal Khan (left) examines patient Christopher Armstrong, one of 25 Calgarians living with Fabry disease. Photo by Paul RotzingerMedical geneticist Dr. Aneal Khan (left) examines patient Christopher Armstrong, one of 25 Calgarians living with Fabry disease. Photo by Paul RotzingerResearchers at the University of Calgary have launched the first gene therapy clinical trial in the world for Fabry disease, a rare inherited enzyme deficiency that can shorten the lifespan of people who have it by as much as 40 years.

The clinical trial has been prompted by promising gene therapy results in mice performed in the laboratory of Dr. Jeffrey Medin at the University Health Network in Toronto. Medin is the principal investigator of the pan-Canada team grant that is supporting this trial.

“We hope this will one day become a form of treatment that effectively cures Fabry disease,” says Dr. Aneal Khan, a researcher at the University of Calgary Alberta Children’s Hospital Research Institute, who is leading the Calgary segment of the national project.

“It could also help establish a platform on which we can create gene therapies for other illnesses and establish Calgary as a national leader in this experimental field of interventional genetics.”

Researchers will first remove a quantity of stem cells from a Fabry patient’s blood. Next, a working copy of a new gene will be inserted into the stem cells using a specially engineered virus. During the final phase of the trial, researchers hope to transplant these stem cells back into the donor patient and the new, working copy of the gene will make the missing enzyme.

Although several gene therapies have been used in Canada for cancer, this study will be the first in the country to test a gene therapy for an inherited metabolic disorder.

Although the project is headquartered in Toronto, physicians and scientists in Calgary will play a major role in the clinical trial. In particular, the lab at Foothills Medical Centre in Calgary has specialized expertise in the stem cell filtering process that will be used for the clinical trial.

“The trigger that’s making all this possible is Calgary’s expertise in isolating the specific blood stem cells we need,” says Medin. “Without a sufficient quantity of those stem cells to work with, our likelihood of success would be greatly reduced.”

Once the pre-clinical experimental results have satisfied the regulatory requirements of Health Canada, the team aims to treat the first human Fabry disease patient, which they estimate will be within two years.

The project is being funded by the Canadian Institutes of Health Research (CIHR) and the Kidney Foundation of Canada.