New cancer drug shows promise in pediatric patients with tumor-specific gene mutations

ORLANDO, Fla. (March 29, 2018) – A new cancer drug has proven safe and effective for pediatric patients with a rare tumor gene mutation, according to a study published today in The Lancet. The study tested the safety and dose of larotrectinib (LOXO-195) in pediatric patients with a mutation known as tropomyosin receptor kinases (TRK) that can occur in a variety of tumor types.

“This research highlights a paradigm shift in cancer care where we are identifying and treating tumors based on their genetic mutations, rather than just where they exist in the body, said Ramamoorthy Nagasubramanian, MD, an author of the study and division chief of pediatric hematology-oncology at Nemours Children’s Hospital. “Additionally, this study represents a win for the field of pediatric research because the drug is being tested in children almost simultaneously with adults, when typically, pediatric research is years behind.”

The multicenter, open-label, phase 1/2 study was conducted at eight sites in the U.S. and enrolled infants, children, and adolescents between the ages of 1 month and 21 years, with an average age of 4-5 years. The drug was administered orally among 24 patients with varying increasing dose concentrations adjusted for age and bodyweight. Of


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