DEVELOPING NEW THERAPIES TO FIGHT BRAIN CANCER

Today’s clinics tailor cancer therapies to individual patients, and Dr. Kevin Petrecca, Associate Professor of Neurology and Neurosurgery, and his fellow researchers aim to refine treatments further, thanks to two complementary donor-supported initiatives, Next Generation and TARGiT.

“Compelling evidence suggests that stem cells lie at the origin of brain cancer, so in Next Generation we’re focusing on understanding how a normal brain stem cell transforms to a cancer cell,” says Petrecca.

Next Generation, so named as it represents the new generation of research strategies, involves a huge sequencing initiative that draws on stem cells isolated from 150 brain cancers.

“We have one of the largest live stem cell banks in the world, all derived from our patients,” says Petrecca. This stem cell bank supports a comprehensive research effort. “As cancer develops from multiple causes, studying one cell line gives us only a small snapshot, but studying a large cohort enables us to determine patterns,” he says.

TARGiT offers a discovery validation platform for approaches developed in Next Generation. “TARGiT is that step just before the clinic, where we confirm that whatever drug we are testing, or whatever pathway we are disrupting, actually works in a model that represents the human disease,” explains Dr. Petrecca.

Researchers take a mouse with no immune system, and implant a specific patient’s brain cancer cells in it. “This is the best model for brain cancer,” he says. “In this model, the cancer’s response to a medicine very closely mimics the human disease. If our discoveries work in this preclinical model of disease, then they can go on to clinical testing.”

TARGiT was launched in 2011 with support from the family and friends of Dr. Francis Boulva, and has led to the development of two medicines. The earliest phase of Next Generation started informally about two years ago, and is now growing thanks to the funds raised by the volunteer-led fundraising gala A Brilliant Night and a generous donation from the Trottier Family Foundation.

“We’re just now making Next Generation a strategic priority,” explains Petrecca. “Stem cell work is expensive, and the role of philanthropy is critical. Classical funding agencies want proof of principle prior to providing support and this can stifle innovation. Philanthropy allows us to test new approaches and achieve proof of principle.”