The State of Pediatric Cancer

Each month, I hold a special gathering for patients and their families at St. Jude Children's Research Hospital to hear about their experiences and how we can make their time at our institution the best it can be.

Families come to St. Jude from across the country and around the world. For most of them, the journey starts with the words, "Your child has cancer." The news is frightening, overwhelming and life altering.

Their questions run the gamut from "Why my child?" to "What treatments are needed?" to "How do we get through this?" But all families make the same appeal: "I want the best for my child."

A childhood cancer diagnosis today is far different than it was when St. Jude opened in 1962. At that time, leukemia was almost always fatal. But pediatric cancer treatment is one of the great success stories of modern cancer medicine. About 80 percent of children in the U.S. and other developed countries now survive their disease.

The way we treat cancer is also improving. Fifty years ago, the focus was solely on saving the child. Today, we've become much better at tailoring therapies that maximize cures while minimizing long-term complications.

At specialized pediatric cancer centers, doctors and researchers work together to improve care and learn from patients enrolled on clinical trials. Such studies have played an essential role in the progress we've made against childhood cancer.

Treatment through clinical trials increases collaboration across institutions through multidisciplinary care teams. Not only do these studies help us fine-tune therapies, but also introduce new therapies and test them against current standards of care.

At St. Jude, the only National Cancer Institute-designated Comprehensive Cancer Center devoted solely to children, our investigations range from new therapies to approaches that optimize supportive care.

Our long-term follow-up studies define the complications that can result from therapies used to cure children of cancer and how those issues can be managed using interventional approaches.

While we've made strides against many pediatric cancers, significant work remains. This year, nearly 16,000 U.S. children will be diagnosed with cancer.

The number of pediatric cancers is only 1 percent of the total number of new cancer cases diagnosed each year in this nation. Yet, cancer remains the leading cause of death due to disease in pediatric patients between the ages of 1 and 15.

One in 5 children with cancer in the U.S. will die from their disease. Despite this sobering statistic, only 4 percent of federal funding for cancer research is dedicated to childhood cancer.

This summer, the Cancer Moonshot Blue Ribbon Panel released a series of recommendations aimed at accelerating progress against cancer. I served as co-chair of the panel's Pediatric Cancer Working Group.

We outlined opportunities for a nationally coordinated effort to advance our knowledge of the unique driver mutations that underlie some of the most common childhood cancers. We want to use that information to accelerate the development of targeted therapeutics against these cancers.

Our recommendations were built on the significant recent progress made in our understanding of pediatric cancer. This progress has arisen from collaborative genomic efforts conducted over the last five years.

For example, St. Jude, in collaboration with Washington University School of Medicine in St. Louis, launched the Pediatric Cancer Genome Project (PCGP), a first-of-its-kind gene sequencing initiative to understand why cancers arise, spread and resist treatment. The project has unearthed a treasure trove of information that served as a foundation for the moonshot initiative.

From the PCGP, we learned that nearly 1 in 10 children with cancer is born with genetic changes that increase cancer risk. This finding marks a turning point in our understanding of cancer risk and will likely change how patients are evaluated. The genetic changes can be used to help guide treatment and advance precision medicine.

In some cases, entire families may benefit from genetic testing and counseling. St. Jude is now offering comprehensive genetic testing to pediatric cancer patients, their parents and siblings as part of a clinical research study called Genomes for Kids. We hope the program will serve as a model to extend genome sequencing to every childhood cancer patient in the country--and help doctors better tailor individual treatments for better outcomes.

Childhood cancer treatment is a marathon, not a sprint. Treatment can last years, and its burden is more than physical. The emotional and financial impact alone can challenge the strongest of families.

While the road to cure is arduous, increased collaboration, new technologies and enhanced data sharing offer new hope that I believe one day will ensure the best future for every child with cancer.