In continuation of my update on LOXO-101
When a baby's life was threatened by a rare pediatric cancer that would not respond to surgery or chemotherapy, doctors at Nemours Children's Hospital rapidly, successfully shrank the tumor by 90 percent using an experimental treatment, according to a new study published online in Pediatric Blood and Cancer. The now-20-month-old girl achieved the remarkable improvement by receiving a drug called LOXO-101 that was being tested on adults, researchers reported.
"Most infants and children with infantile fibrosarcoma (IFS) can be cured through surgery and chemotherapy. When our patient's disease progressed in spite of these treatments, we had to investigate new options that could target the disease," said Dr. Ramamoorthy Nagasubramanian M.D., lead author of the manuscript, division chief of pediatric hematology-oncology at Nemours Children's Hospital. "The dramatic reduction in tumor size shows early but promising evidence of the potential for LOXO-101 to provide significant benefit for pediatric patients with NTRK gene fusions."
Nemours' oncology team operated on the girl at 6 months to remove a large tumor located in the neck and face. The tumor did not respond to standard chemotherapy and relapsed after extensive surgery. Genetic testing confirmed an ETV6-NTRK3 gene fusion, which is frequently found in IFS. At the time, LOXO-101 was in a Phase 1 multi-center basket trial in adults. Working with Nemours, Loxo Oncology, Inc., a biopharmaceutical company developing highly selective medicines for patients with genetically defined cancers, was able to expand the trial to children and enroll her.
