Pediatric cancers are killers that often elude approved therapies, but the advent of precision medicine is creating a renaissance in pediatric oncology, as illustrated by two studies selected for the ASCO preview program.

The first, a status report from the National Cancer Institute-Children’s Oncology Group Pediatric Molecular Analysis for Therapy Choice (NCI-COG Pediatric MATCH), which was launched in 2017, is the story of a good idea that appears to be working better than expected. The concept: tumor sequencing of tumor samples from children and young adults whose cancers do not respond to treatment could identify genetic targets that could then be matched to investigational, targeted therapies.

Will Parsons, MD, PhD, of Baylor College of Medicine in Houston said the expectation was that 10% of the tumor samples would yield matches. Thus far, the program has collected 400 tumor specimens, completed DNA sequencing on more than 350, and found eligible matches for 24% of those samples, and 10% of the patients who had tumors sequenced and matched are now receiving targeted therapy.

The second pediatric precision medicine report involved entrectinib — not one of the 10 targeted therapies in the NCI-MATCH. Giles W. Robinson, MD, a pediatric neuro-oncologist from St. Jude Children’s Research Hospital in Memphis discussed very early findings (I/IB) from a study that included 29 patients age 4.9 months to 20 years. The patients had a variety of central nervous system tumors, neuroblastomas, or other solid tumors, and responses were observed in 12 of 28 evaluable patients. “Our results show that children with life-threatening cancers can benefit greatly even after other conventional therapies have not worked – we’ve seen some rapid and durable responses, which is very gratifying,” Robinson said.

Entrectinib is given orally and targets TrkA/B/C, ROS1, and ALK tyrosine kinases. Robinson said the results “were striking and fast in all tumors that harbored NTRK1/2/3, ROS1 or ALK fusions.”

Abstract 10011: Identification of targetable molecular alterations in the NCI-COG Pediatric MATCH trial.

Abstract 10009: Phase 1/1B trial to assess the activity of entrectinib in children and adolescents with recurrent or refractory solid tumors including central nervous system (CNS) tumors.

The ASCO meeting, which kicks off May 31 in Chicago, will feature 2,400 abstracts, and another 3,200 submitted abstracts will be published online. Follow BreakingMED coverage at Physicians’ Weekly.

Read the full ASCO 2019 RoundUp

Peggy Peck, Editor-in-Chief at BreakingMED.org, a service of @Point of Care, LLC, which provides daily medical news reports curated to serve the unique needs of busy physicians and other healthcare professionals.