Integrating Genomics into Pediatric Cancer Care

Study provides proof that a child's genetic makeup can provide actionable information to alter cancer treatment regimens.

Researchers at the University of Michigan have evaluated the impact of integrating clinical sequencing into clinical practice when treating children and young adults with relapsed, refractory, or rare cancer. The study found that clinical sequencing, while feasible, revealed actionable findings in nearly half of the patients, and resulted in treatment change as well as genetic counseling in a small percentage of patients. The results, published in JAMA, suggest that extensive genetic analysis can present clinical alternatives.

In a single site observational study, 102 children and young adults with a median age of 11.5 years underwent exome and transcriptome sequencing and genetic counseling. A precision medicine tumor board evaluated the results and advised the families and their physicians.

Forty two of the 102 patients had actionable findings that resulted in an alternate care regimen; this was true for 15 of 28 (54%) patients with hematological malignancies and in 27 of 63 (43%) patients with solid tumors. The result: treatment change in 15% of patients and genetic counseling in 10% of patients to discuss future risks. These personalized interventions resulted in 10% of patients with partial clinical remission of 8 to 16 months or sustained complete clinical remission of 6 to 21 months. All the patients in remission and their families agreed to genetic counseling and screening, the authors wrote.

The lead researcher on the study, Rajen Mody, MBBS, a pediatric oncologist at the University of Michigan, stressed that one needs to bear in mind that the cases evaluated in the study are the hardest to treat. So the results “offer some hope that we’ll be able to treat these children in a more personalized way,” he said. This approach will now open up avenues for the most resistant cases of pediatric cancer that currently do not have targeted drugs.