01/28/2026
Childhood cancer is not simply a smaller version of adult cancer. These are different diseases from the start, shaped by biology that follows its own rules. Many childhood cancers arise when normal developmental programs shift off course. This difference from adult cancers influences how researchers identify targets and develop therapies that can truly benefit young patients.
For years, children received treatments originally designed for adults, even though the underlying mechanisms of their diseases differ. More than half of the genetic mutations that drive childhood cancers are not found in adult cancers.
“In children, the mutational burden is much lower; their tumors form because a small number of mutations cause normal development to go off track,” said Charles Roberts, MD, PhD, executive vice president and St. Jude Comprehensive Cancer Center director. This gap helps explain why adult targeted therapies have often fallen short for children who urgently need new options.
Investigators at St. Jude are working to change this reality and research has shown how studying pediatric tumors can uncover insights that reach far beyond childhood disease.
The need for treatments built around pediatric biology has never been clearer. New approaches such as targeted protein degradation, CAR T-cell therapy, RNA based therapeutics and epigenetic reprogramming are beginning to redefine what is possible.
St. Jude invests in areas where traditional drug development often falls short. By supporting early-stage projects and creating a pathway for discoveries to reach clinical testing, St. Jude helps ensure that biological insights can become real options for children who need them most.
