A prioritization algorithm has identified subgroups of pediatric oncology patients who can benefit from molecularly matched targeted therapies. This approach extended the time until disease progression by 3 months for a small group of poor prognosis pediatric patients with very high-priority targets.
Children with refractory, relapsed, and progressive high-risk malignancies have a poor survival of less than 20 percent and 9.5 months median survival. The new study evaluated the clinical potential of precision oncology in children by identifying molecular targets for off-label treatments and potential biomarkers for enrollment and development of clinical trials, as well as providing additional diagnostic precision.
The INdividualized Therapy FOr Relapsed Malignancies in Childhood (INFORM) study “gives children a chance to benefit from off-label therapy, sometimes from adult oncology, targeted drugs, and enrollment in biomarker-driven clinical trials. This is the first time clinical outcome in a real-world setting of a large-scale, multinational personalized pediatric oncology platform was assessed for clinical benefit,” said lead author Cornelis van Tilburg, MD, PhD, a pediatric oncologist at Hopp Children’s Cancer Center Heidelberg. He presented the study’s findings during the virtual scientific program of the ASCO 2020 Annual Meeting (Abstract LBA10503).
While current treatment of childhood cancers results in high overall cure rates, relapsed high-risk disease is associated with a poor prognosis. For the most part, precision oncology has yet to be applied in pediatric cancer care, unlike in adult cancer where it has improved outcomes.
“For pediatric patients, if the cancer has relapsed, the prognosis is poor and there are few new innovative treatments,” said van Tilburg. “Compare this to adult oncology, where there are many new trials, many new biomarkers, and many new drugs. Pediatric oncology is really lagging behind when it comes to precision medicine and the development of new drugs.”
Source: Matching High Risk Pediatric Cancer Patients to Targeted Therapies
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