Vancouver, BC - Two BC Cancer Agency scientists are members of an international science team whose breakthrough gene therapy project is likely to have applications to future cancer treatment.

The project, aimed at correcting sickle cell disease in mice, may not only suggest future therapies for sickle cell disease in humans, it may also provide a basis for delivering therapeutic genes to human cells for more effective cancer treatments.

"While this is a big breakthrough for a disease that is not cancer, it fits in very well with the Agency's commitment to develop gene-targeted strategies for cancer control," explains Dr. Connie Eaves, deputy director with the Terry Fox Laboratory at the Agency, and Professor of Medical Genetics at the University of British Columbia.

The therapy counteracts the faulty gene that causes red blood cells to deform, or "sickle," by using a viral delivery system to transfer an anti-sickling variant of the gene to bone marrow stem cells. After these corrected stem cells are transplanted back into the host, the anti-sickling gene becomes active when the stem cells mature into red blood cells.

Treating sickle cell disease is considered the "Mount Everest" of challenges among gene therapy researchers.  The gene "replacement parts" needed are relatively large on a genetic scale, which makes it difficult to transport the genetic material into the genome of blood stem cells.

To overcome this obstacle, the research team combined recent discoveries in their own labs with advancements from other labs to help choose the best gene replacement approach, and a delivery system that would be effective.

"We've worked over the years to improve these delivery methods," says Dr. Keith Humphries, senior scientist with the Terry Fox Lab of the Agency. "We many tests in different labs, as well as repeating each others work, to ensure the results could be reproduced anywhere."

Drs. Eaves and Humphries have been involved in this research for 10 years in a collaboration between the Agency, MIT in Boston and The Albert Einstein College of Medicine in New York. The outcome of the study appears in the prestigious Science magazine's Dec. 14 issue, released today.

For more information, please contact:
Nicole Adams
Public Relations Officer
BC Cancer Agency
(604) 877-6272

nadams@bccancer.bc.ca