Genetic analysis of tumours provides targeted therapy for pediatric cancer

Pediatric patients with hard to treat cancer are getting another chance at life through personalized care. The Terry Fox PROFYLE (PRecision Oncology For Young peopLE) research project is transforming the way they treat children with cancer by using genetic analysis of tumour samples to identify specific molecular targets of therapy. And the participants enrolled in the study are helping future children by understanding the causes of cancer resistant to treatment.

Pediatric oncologist Lucie Pecheux is the principal investigator for the University of Alberta and is the co-clinical site lead of Terry Fox PROFYLE’s Adolescent and Young Adults Node. It is open in 33 sites across Canada and involves many collaborations. “Exploring the tumour’s genetics helps figure out what are the early steps of cancer development, which is critical to identify a specific therapeutic target to fix or at least control molecular drivers of cancer,” says Pecheux, assistant professor in the Division of iHOPE.

Each year, on average, they diagnose 880 children under the age of 15 with cancer, and 150 die from the disease in Canada, according to the Canadian Cancer Registry. This unique pan-Canadian initiative allows for any pediatric patients with hard to treat cancer to have access to a network of expertise, and to advanced genetic science techniques that are not currently available in clinical laboratories.

“What is amazing with this study is that it brings together research and personalized care for these children from anywhere in Canada. The project connects experts from the east to the west coast, enabling the patient’s care team to send samples for next generation evaluation. Collaborative expert committees analyze the results and then the treating oncologist receives management recommendations,” says Pecheux.

Over 80 per cent of clinicians report that results from PROFYLE have the potential to inform their patient management, whether by suggesting a change in a treatment plan, identifying a mutation in a patient’s genes, or even by modifying or refining the diagnosis. When a mutation is identified, it means other family members may have this cancer predisposition and they are sent for a consultation with a geneticist.

Precision analysis of hard to treat cancer across Canada involves the collaboration and cooperation of top cancer researchers, including Pecheux. Each researcher has a specialty, including genomics, biomarker analysis, pathology, cancer modelling, drug development, and bioethics. Pecheux is fascinated by looking at pediatric cancer from a genetic point of view. “The amount of comprehensive genomic information generated from these patients’ tumours and analysis of all of the cells is amazing. Beyond the clinical help for each of these children, it identifies new molecular drivers of pediatric cancer. This opens multiple other research pathways, hopefully leading to developing targeted therapy for these diseases with a terrible prognostic,” she explains.

Patients and families taking part in Terry Fox PROFYLE have not only access to personalized clinical care but also contribute with researchers like Pecheux to comprehend the genetic characteristics of pediatric cancer and discover new therapeutic options.

Study management support for this study is provided by the Women and Children's Health Research Institute, through funding from the Stollery Children's Hospital Foundation.