ancer is the leading disease-related cause of death in children in the United States. Yet pediatric cancer is often left behind when it comes to funding research and developing new drugs. Not only does this give short shrift to children with cancer, but it also threatens to rob us of advances that could benefit cancer patients of all ages.
That’s what I and fellow members of the Coalition for Pediatric Medical Research told the staff of Vice President Joe Biden, who is leading the cancer “moonshot.” We recently met in Biden’s Washington office to make the case that childhood cancer must be represented as the government considers ways to propel cancer research.
Six decades ago, the first major breakthrough in the treatment of cancer came when Dr. Sidney Farber used an experimental drug to treat leukemia in children. Some went into remission — a medical first. Chemotherapy was born, revolutionizing the care of both children and adults with cancer.
Since then, overall cure rates for childhood cancers have risen to 80 percent. Some pediatric cancers post cure rates of 90 percent, while others remain difficult or nearly impossible to cure. At the same time, a majority of childhood cancer survivors experience harmful, lifelong complications of the treatments that saved them. Such late effects can be far more devastating to the survivor diagnosed at age 5 than the survivor diagnosed at age 65.
Focus on children
We are learning that cancer research is a two-way street. Just as breakthroughs in adult cancer research can help children, breakthroughs in pediatric cancer can also benefit adults.
The leading edge of cancer research and treatment is toppling two old silos: cancer’s location in the body and patient age. A targeted therapy aimed at adult skin cancer is also helping children with brain tumors who share the same mutation. And research into the relatively pristine genomic landscape of pediatric cancers — children have accumulated far fewer genetic mutations than adults — is providing new insights into childhood cancers that may also translate into advances for adults.
The path forward, as noted in the coalition’s Recommended Pediatric Priorities for the Cancer Moonshot Initiative, contains several challenges. The biology and types of childhood cancers differ from those that affect adults and require distinct investigation and therapies.
Although there are more than 150 types of childhood cancer, pediatric cancer receives only a small fraction of National Cancer Institute and National Institutes of Health funding. Research and development of new therapies largely depend on federal and philanthropic resources because industry is focusing its oncology efforts on the vastly larger potential market for adult cancer drugs.
The coalition believes that these four areas should be part of the moonshot’s pediatric agenda:
Gene activity. Genetic abnormalities seen in pediatric cancers often involve the regulation of gene activity — instructions that tell genes to turn on or off. Such abnormalities are also important in a wide range of adult cancers. Deviations in gene regulation are currently difficult to treat with drugs. We need to develop novel avenues of research to accelerate the journey from bench to bedside of drugs aimed at gene regulation.
Gene-targeted therapy. In the clinical revolution called precision medicine, childhood cancers have lagged behind adult cancers. Recent research suggests that children could benefit from more extensive genetic sequencing of their cancers. The National Cancer Institute, which is already investigating precision medicine for adult cancers, is expected to open a pediatric Molecular Analysis for Therapy Choice (MATCH) trial later this year. This and other studies into applications of genetically targeted therapies for pediatric cancers will push forward this promising approach.
Federal efforts. The federal government can play a critical role by requiring and encouraging pharmaceutical companies to develop drugs for children with cancer. Recently enacted incentives that extend patent periods or speed the approval process for drug applications are important steps in this direction. The federal government needs to ensure that clinical trials in children are started earlier in the drug approval process. It should also make sure that guidelines for pediatric dosing are created for potentially beneficial drugs, and that new drugs are made in the liquid formulation needed for young children who can’t swallow pills.
Collaborative efforts. Because childhood cancer is less common than adult cancer, progress is possible only with collaboration across institutions. The National Pediatric Research Network, which was recently approved by Congress, is an important new tool. It should be funded and implemented without further delay.
The decades of productive life awaiting boys and girls cured of cancer should be reward enough for ensuring that children have a place in the cancer moonshot. The promise of years ahead, too, for older individuals cured of cancer makes finding room for pediatric cancer in the moonshot that much more critical.
David A. Williams, MD, is president of the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center.