Bench to Bedside | August 2016

Childhood Cancer Advocates Find Strength in Numbers

Childhood cancer is a disease that divides. Biologically, cancer is a disease of cells dividing uncontrollably. Scientifically, genetic and molecular discoveries increasingly drive pediatric cancer researchers to divide their attention among narrower and narrower molecular subtypes of disease. And socially, families facing childhood cancer sometimes struggle against a sense of isolation if their friends and family doctors have little experience with the rare disease that now immerses their entire world.

But instead of letting cancer divide them, families, advocates, researchers, and others, are flipping that equation. An event held this summer at The Children’s Hospital of Philadelphia hosted by the Coalition Against Childhood Cancer (CAC2) was emblematic of the ways they are multiplying their efforts by coming together to defeat childhood cancer.

“It’s really helpful to have an organization that supports initiatives that promote collaboration among these different groups and provides information and educational opportunities so that people across the community have access to the same knowledge,” said Vickie Buenger, CAC2 president (pictured, left, with Peter Adamson, MD). “That’s what we do.”

Multiplying Efforts of Childhood Cancer Advocates

In the U.S., organizations focused on childhood cancer number in the hundreds, potentially as many as 1,000. Many of them are pursuing their important work in isolation, having their origins as foundations established in loving memory of a child lost to cancer or in honor of a young cancer survivor.

Within the last five years, however, approximately 130 childhood cancer organizations, independent advocates, and a handful of students, have joined forces in the CAC2. This all-volunteer nonprofit organization coalesces these stakeholders’ efforts with shared educational resources, collective support for outstanding ideas, and networking and collaborative opportunities for shared initiatives.

Representatives from dozens of these organizations convened at CHOP June 22-23 for the group’s largest and most comprehensive educational experience yet, the fourth CAC2 Summit and Annual Meeting. Members and guests heard from CHOP experts and other renowned guests from academia, government agencies, the nonprofit sector, and the pharmaceutical industry, on a variety of research, family support, and advocacy topics.

One session brought members up to date on Vice President Joe Biden’s Cancer Moonshot initiative, and another panel discussion probed psychosocial care for children with cancer and their families. CAC2 members also met for special interest breakout sessions to collaborate and share their ideas, and members toured research and clinical facilities across the CHOP campus.

A Growing Collaborative Emphasis in Childhood Cancer Research

During his opening remarks, Stephen Hunger, MD, chief of the Division of Pediatric Oncology and director of the Center for Childhood Cancer Research at CHOP, highlighted precision medicine, cancer predisposition, and immunotherapy as the biggest trends in pediatric cancer research.

Precision medicine in particular was a major area of focus for the meeting. By identifying the precise molecular mechanisms of disease and developing targeted treatments to address those mechanisms, researchers are finding newer treatments that are potentially more effective while causing fewer side effects on healthy tissue. But, too often, in these early days of precision medicine investigations, the most promising new therapies are designed to work only for narrow subgroups of rare diseases.

“It is important to find methods that work for subsets of patients, but, more importantly, we have to make it work for all patients,” Dr. Hunger said.

Two extensive panel discussions of precision medicine highlighted some of the ways that academic researchers and their partners in government and the pharmaceutical industry are working toward that goal. White House Precision Medicine Initiative (PMI) Project Manager Stephanie Devaney, PhD, spotlighted the national effort to develop targeted approaches for treatment and prevention of disease. Katherine Janeway, MD, a Boston Children’s Hospital (BCH) and Harvard investigator, highlighted several specific pediatric cancer precision medicine efforts at BCH and its affiliates. And Steven Joffe, MD, MPH, a CHOP and Penn medical ethicist and physician, led a discussion of bioethical considerations in precision medicine.

A second panel narrowed that focus in on Pediatric MATCH, a large precision medicine clinical trial to test multiple candidate drugs for multiple advanced-stage pediatric cancers according to molecular insights into the mechanisms of disease based on genetic mutations found in tumor biopsies after relapse. The trial, which is due to begin in 2017, is a collaborative effort of the multi-institution Children’s Oncology Group (COG), the National Cancer Institute (NCI), and pharmaceutical industry partners. Speakers from the NCI, Food and Drug Administration, and two pharmaceutical companies that provide investigational drugs to Pediatric MATCH (AstraZeneca and Loxo Pharmaceuticals), gave an in-depth look at this complex trial and the many biomedical, logistical, safety, regulatory, and financial challenges they are working collaboratively to overcome.

Pediatric MATCH follows a model similar to the NCI MATCH trial for adult cancers that is already underway, and in some cases it may accelerate testing of drug candidates that were not previously tested in children. The project ultimately aims to provide valuable information about multiple actionable cancer mutations affecting children with multiple rare subsets of disease, within a relatively brief multi-arm trial.

“Rare diseases or rare subsets of diseases require a nationwide infrastructure for research, and we are fortunate to have one supported by the National Cancer Institute,” said Peter Adamson, MD, a pediatric oncologist at CHOP and professor of Pediatrics at Penn who serves on the NCI’s Cancer Advisory Board and on the Blue Ribbon Panel for the National Cancer Moonshot initiative and is chair of the COG, during the CAC2 Summit’s final keynote. “For some of these studies, we are going to need a global infrastructure to get this done.”

All of the slide presentations from the CAC2 summit are available on the organization’s website.


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Produced by The Children’s Hospital of Philadelphia Research Institute.

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