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To Cure Cancer, Targeted Therapies Best Future Bet

8251_49784674_hr“Cancer medicine does not ask us to do precision medicine as a side note; cancer medicine ask us to do precision medicine as a basis,” said Siddhartha Mukherjee, Pulitzer Prize-winning author of The Emperor of All Maladies: A Biography of Cancer, at the opening keynote of the Boston Children’s Hospital Global Pediatric Innovation Summit on Monday.

This year, physicians and innovators gathered at the Seaport World Trade Center in Boston to discuss precision medicine in pediatric care. Cancer, which Mukherjee describes in his book as “a shape-shifting disease of colossal diversity” especially calls for targeted therapies.

In his talk, Mukherjee discussed the future of cancer treatment as moving away from the standard cell-kill model. “You can’t just attack the mutations themselves, you also have to attack the entire network of genetic signals and environmental/microenvironmental signals that is keeping the cancer cells alive in the human body in the context of normal physiology,” he explained. “The idea here, of course, is to use the entire pharmacopoeia that’s available to us—broadly speaking— to try shut off these signals.”

“People have likened this to going into a network and shutting off its critical nodes. We began, if we could, by shutting off one little switch, and the question is can we take multiple of these switches and shut them off and would that finally bring the cancer cell to it’s end.”

Pediatric cancer patients have been incredibly important to our current understanding of cancer and its treatment, partly because of their 80 to 90 percent enrollment rate in clinical trials. In Ken Burns’ documentary Cancer: The Emperor of All Maladies, which is based on Mukherjee’s book, Burns recounts the story of Emily Whitehead, who at age five was diagnosed with acute lymphoblastic leukemia. After two relapses, Emily’s parents enrolled her in a clinical trial run by Dr. Carl June of the Children’s Hospital of Philadelphia, where she became the first pediatric patient to be treated with T-cells. Emily’s T-cells were collected, “reprogrammed” to attack cancer cells and released back into her blood stream. Although the treatment almost killed her— a last minute administration of tocilizumab ameliorated lethally high IL-6 levels—it proved successful. Emily has been in remission for three years, and her story has reinvigorated interest in T-cell therapy.

“Emily Whitehead’s example is one of moving beyond simply killing cells using a cytotoxic compound,” explained Mukherjee. “In her case, her immune system was activated—it was an immunological process that was activated—what we were really focusing on in that situation was not the cancer cell itself, but the immune environment of the cancer cell. Could you activate the immune system and thereby send her cancer into remission?”

Mukherjee ended his keynote with a somber question. “If you look at the 1950s—if you go back to the Jimmy Fund Clinic— there was a kind of desperation, an urgency about the treatment of children’s cancer,” he noted. “There was a desperation and urgency and a kind of fire that was lit at places like Boston Children’s Hospital that allowed an entire community coalesce around the idea of treating cancer. The question that I’ve thrown out to this audience—to many audiences— is, have we lost some of that urgency?”

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