Precision medicine for pediatric cancers lags behind that for adult cancers: Citing hurdles in clinical trial recruitment and drug development, researchers are exploring ways to close gaps
- Pediatric precision oncology faces challenges due to the rarity of pediatric cancers, which limits research and funding compared to adult cancers.
- Dr. Katherine Janeway highlights the need for more validated pre-clinical models and pharmaceutical interest in pediatric cancers.
- The RACE Act, effective in 2020, updates the Pediatric Research Equity Act, aiming to increase pediatric access to novel therapies by ensuring cancer drugs are studied in children when relevant.
- The FDA's draft guidance allows adolescents to enroll in adult clinical trials if they have recurrent cancers or no other treatment options.
- Significant progress has been made in immuno-oncology for pediatric leukemia, with treatments like CAR T-cell therapy and BiTEs.
- Research is ongoing for new targeted therapies, such as EZH2 inhibitors for rhabdoid tumors, and data sharing initiatives are gaining attention.
Although scientists have made great strides in applying breakthroughs in precision medicine to many adult cancers, the same cannot be said of pediatric diseases. However,researchers, advocates, and lawmakers are hoping to bridge the divide, largely by identifying more opportunities to include children, adolescents, and young adults in precision oncology clinical trials. Exactly why pediatric precision oncology has experienced fewer research gains is a varied and complicated issue, according to experts. Nonetheless, Katherine Janeway, MD, a senior physician and director of clinical genomics at the Dana-Farber Cancer Institute and Children’s Hospital in Boston, Massachusetts, points to several important drivers of the disparity. She co-authored an article in a March 2019 special edition of Science that outlined problems and possibilities in the field....
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