Children With Cancer Have Complete Responses in a COG Phase 1 Trial: Pills Zero in On Abnormal Genes That Drive Specific Cancers, Children’s Hospital of Philadelphia Study

A pill designed to zero in on abnormal genes that drive specific cancers has produced encouraging early results in children with an uncommon but aggressive type of lymphoma, as well as in children with a rare form of neuroblastoma. A phase 1 clinical trial of the drug crizotinib achieved remissions, with minimal side effects, for 10 of the children participating in a clinical study carried out by the multicenter Children’s Oncology Group (COG). The results were “an exciting proof-of-principle” for the targeted treatment, said the study leader, Yaël P. Mossé, M.D., a pediatric oncologist at The Children’s Hospital of Philadelphia.

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