Despite advances in pediatric cancer treatment, limited funding and access challenges continue to threaten progress and children’s long-term outcomes.
Doug Graham, M.D., Ph.D.
Past President, American Society of Pediatric Hematology/Oncology; Chief, Aflac Cancer and Blood Disorders Center, Children’s Healthcare of Atlanta; Division Chief, Pediatric Hematology/Oncology/BMT, Professor of Pediatrics, Emory University
Pediatric cancer remains the leading cause of death from disease in children and adolescents. Nearly 16,000 children and adolescents in the United States are diagnosed with cancer each year. Despite remarkable progress in recent years, 1 in 5 children diagnosed with cancer will not survive. Further, survivors of childhood cancer face significant late effects from their cancer or its treatment. Studies have shown that by age 50, pediatric cancer survivors have a 99% cumulative incidence of developing at least one chronic health condition (i.e., lung disease, heart disease, hearing loss, endocrine conditions, neurocognitive impairment, secondary cancers, liver damage, kidney damage, and infertility).
Breakthroughs in pediatric cancer treatment
Pediatric oncologists are committed to finding better therapy options for the 20% of children we are not yet able to cure and are striving to find more targeted and less toxic therapies with fewer late effects. Recent breakthroughs in precision medicine and immunotherapy are helping us come closer to achieving these goals.
Technological advances, such as genome sequencing, allow physicians to rapidly evaluate approximately 20,000 genes in cancer cells within a few weeks and determine if treatment can be customized to improve outcomes. Sequencing is also helping us identify children who are at increased risk of developing cancer (including siblings of children with cancer), allowing us to monitor and intervene early.
In addition to the role of precision medicine in enhancing outcomes, we have made remarkable strides in harnessing the immune system to help combat cancer. Medicines like PD-1 inhibitors help activate the immune system and prevent cancer cells from evading detection. Additionally, cellular therapy, such as CAR-T cell therapy, has been especially impactful for children with relapsed and refractory acute lymphoblastic leukemia, the most common type of childhood cancer. CAR-T cell therapy involves collecting T cells from the patient’s blood and genetically modifying these immune cells to seek out and destroy the leukemia cells. Remarkably, many children with leukemia who have failed all other treatments have achieved sustained remissions with this new therapy.
The importance of continual funding
Despite these advances, pediatric cancer research remains woefully underfunded, creating barriers to realizing the potential of our recent medical discoveries. In fact, only about 4% of the National Cancer Institute’s annual budget is dedicated to childhood cancers. As past president of the American Society of Pediatric Hematology/Oncology, I had a front-row seat to the critical importance of the additional research funding needed to move forward with these promising new treatment approaches. Funding from cancer foundations and philanthropic donations also enables new ideas to be explored to achieve further medical advances. Without these funds, we also risk not being able to support pediatric cancer research as a viable career path for our trainees and junior faculty.
Even with exciting advancements in diagnostics and therapeutics, a question we must contend with is access, particularly for children dependent on Medicaid. Medicaid and the Children’s Health Insurance Program cover nearly 50% of all children nationwide, and that percentage is much higher in certain geographical locations, especially rural communities. Medicaid provides vital access to pediatric oncology specialists, clinical trials, cutting-edge cancer drugs and treatments, bone marrow transplants, and cancer survivorship programs. Evidence shows that pediatric patients who experience disruptions in Medicaid coverage have worse survival rates than patients without disruptions. Ensuring continuous Medicaid coverage for children could make a dramatic difference in cancer outcomes, as could simplifying enrollment processes and removing barriers for access to out-of-state specialized cancer care.
September is Childhood Cancer Awareness Month. While cancer awareness is important, it must be accompanied by action to address crucial issues. Protect Medicaid and simplify enrollment. Fund pediatric cancer research. Enable cancer providers and researchers to discover and advance more treatment options for children with cancer. Maintain a pipeline for the next generation to pursue a career in pediatric cancer research. Give every child with cancer the opportunity for a lifetime.
