How can we make personalized therapy for childhood cancer a reality?

For some pediatric cancers, such as acute lymphoblastic leukemia, older forms of therapy — and older ways of defining who receives which therapy — have served well over the last few decades. But that approach is no longer sufficient. Revolutionary gains have been made in adult oncology using personalized genomic therapy — therapy based on matching treatments to the genetic makeup of a patient’s tumor. The time has come to take them to the pediatric space.

But how will pediatric oncology get there? A panel discussion at Boston Children’s Hospital’s Global Pediatric Innovation Summit about personalized cancer genomics — moderated by Bloomberg News’s John Lauerman and featuring Katherine Janeway, MD, clinical director of the Solid Tumor Center at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center — took on this question. Panelists raised four overarching concepts to consider: numbers, sharing, collaboration and incentives.

Pediatric oncology is rife with small numbers:

  • Small numbers of patients, because childhood tumors are rare.
  • Small numbers of mutations. As panelist Melinda Merchant, MD, PhD, of AstraZeneca noted, pediatric tumors are distinctly quiet from a genomic point of view.
  • Small amounts of data. “Out of about 17,000 genomes or exomes available in public databases for study, less than 2 percent are pediatric,” said Merchant.
  • Small numbers of trials. The pharmaceutical industry is developing relatively few compounds for pediatrics, according to Hubert Caron, MD, PhD, a panelist from Roche Pharmaceuticals.

The way around the numbers problems, the panelists agreed, is collaboration and sharing. Janeway made an impassioned plea for greater sharing of genomic data, noting that it takes hundreds if not thousands of tumor genomes to make meaningful insights.

“Genomic data sharing is an objective of pretty much everybody who sequences childhood tumors,” she said. “Every sequence of every childhood tumor is important to us making progress, not only in precision medicine but in understanding the underpinnings of childhood cancer.”

Once collaboration, sharing and incentives come together, genomic therapy for childhood cancers should reach a tipping point.Taking up the baton, Caron called for “very strong collaboration between academia, pharmaceutical companies, health authorities and parents and patients.” Parents can play a particular strong role in driving collaboration, he noted.

Panelist Nancy Goodman of the advocacy group Kids v Cancer put that to the research community directly. “Parents really do have the burning desire to move things as fast as possible,” she said. “The pediatric cancer advocacy community is really focused on finding ways to make all of you more efficient and help you achieve your dreams, because your dreams are our dreams.”

Getting collaboration to flourish and drugs to enter the pipeline takes incentives, Caron added. “If there is no stick but also no carrot, companies will not spontaneously develop in very small markets.” Goodman pointed out examples that her organization has helped create or refine: the Creating Hope Act (a carrot that provides vouchers for companies that develop FDA-approved pediatric drugs) and the Pediatric Research Equity Act (a stick that obliges companies to study existing drugs in children).

Once collaboration, sharing and incentives come together, genomic therapy for childhood cancers should reach a tipping point. Crystal Mackall, MD, chief of National Cancer Institute’s Pediatric Oncology Branch, described that moment for cancer immunotherapy, cell therapy and other cancer innovations: “If enough people invest and you get the brain trust behind it and the financial resources thrown at it, the chances for success grow exponentially.”

See more posts and videos from the Global Pediatric Innovation Summit.