A Two-Decade Quest for Targeted Cancer Treatments

What began as a revelation about the genetic basis of a rare type of childhood cancer would become a wide-ranging success story.

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“The challenge with doing childhood cancer research is that most people in the world, they don’t really know much about childhood cancer. They assume that it doesn’t really happen in kids. But it does, and it’s a very different disease than in adults.”

Pediatric oncologist Poul Sorensen has spent his career looking for new ways to target childhood cancers by understanding how they escape the immune system or resist treatment. And because these features aren’t always obvious by looking at the pathology under a microscope, Sorensen needed to look for differences down to a molecular level. He hoped these clues would lead to safer and more effective treatment strategies.

It’s an important area of research, as high-risk pediatric cancers remain the leading cause of childhood mortality. And even when successfully treated, the treatments themselves can cause lifelong harm.

“We’ve learned that there’s so much more that you can derive knowledge-wise from studying childhood cancer because the genetics are usually a lot simpler,” says Sorensen, professor of pathology and laboratory medicine at the University of British Columbia.

“By studying childhood cancer, not only can we understand how to target that specific disease, but we can learn about what’s going on in adult cancers.”

That insight seems straightforward now, but it took the scientific community many years to come around to believing it. Bayer was a key industry partner in using childhood cancer research to bring a treatment to the clinic.

“About 20 years ago, Dr. Poul Sorensen made a seminal discovery in which he identified a genetic alteration that causes a very rare form of cancer called infantile fibrosarcoma,” says Chrisoula Giannaris, Head of Medical Affairs — Hematology, Oncology & Radiology at Bayer Inc.

“Twenty years later we now have a product, or a group of products that can actually be used to treat this genetic alteration, and as a result lead to remarkable outcomes for patients.”

Through this partnership, basic research was connected to a company that held the rights to a drug that has since proven its utility in cancer treatment beyond this one rare type.

“It’s because of the work of Dr. Sorensen and the discovery of this new class of proteins that we now understand that this genetic alteration can actually happen across multiple tumour types,” adds Giannaris.

“And as a result we’re not just dealing with treating one type of rare cancer, but able to treat a multitude of cancers.”

This drug is now approved by Health Canada, the FDA in the United States, and European regulatory bodies. It’s a huge success story, and Bayer has been “nothing but supportive for all of this,” says Sorensen.

This paradigm-shifting work started by looking at a rare childhood cancer, but it grew to treat many types of cancers — even in adults — by identifying a common genetic driver. As a result, patients around the world can benefit from a more targeted treatment.

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Poul Sorensen is a board certified anatomic pathologist, specializing in the molecular pathology of pediatric cancers. He undertook his undergraduate, medical, and PhD degrees at the University of British Columbia (UBC) and McGill University, Montreal.

He completed postdoctoral training at the University of Minnesota, Minneapolis and Children’s Hospital Los Angeles, University of Southern California, after his Pathology training. He then returned to Vancouver to start his own laboratory at Children’s and Women’s Hospital in Vancouver.

Sorensen holds the Asa and Kashmir Johal Endowed Chair in Childhood Cancer Research, and he is Professor of Pathology and Laboratory Medicine at UBC.

Sorensen’s research laboratory is located in the Department of Molecular Oncology at the BC Cancer Research Centre, where he is a distinguished scientist. His research focuses on targeting aberrant signaling pathways that are activated in childhood cancers and breast carcinoma.

Sorensen’s laboratory uses a combination of genetic and biochemical approaches to identify proteins that are specifically altered in human tumours. His laboratory has discovered many novel genetic alterations in childhood cancer and breast tumours, and these discoveries have been translated into new diagnostic tests for specific tumours, and have advanced our understanding of how the involved proteins transmit signals that cause cells to become cancerous. Such information then allows for the rapid implementation of strategies to target these proteins therapeutically.

Sorensen is also the Chair of the Translational Research Committee of the Children’s Oncology Group (COG), the largest pediatric oncology clinical trials network in the world.

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