Clinical data reported in New England Journal of Medicine
MORGANTOWN, W.Va. – The WVU Cancer Institute is one of several sites nationwide that participated in a groundbreaking clinical research study of a novel drug proven very effective in the treatment of pediatric and adult cancers that carry a specific genetic mutation.
Larotrectinib, developed by biopharmaceutical company Loxo Oncology, was tested in three clinical research studies at cancer centers nationwide. Patients ranged in age from four months to 76 years and had a total of 12 different tumor types, including common and rare cancers, but all had the genetic mutation in the NTRK gene in common.
Seventy-five to 80 percent of the 55 adult and pediatric patients treated with larotrectinib experienced significant tumor reductions with only mild side effects, regardless of patients’ age and specific tumor types. One patient with infantile fibrosarcoma responded so well to the test drug that limb amputation surgery was no longer necessary as part of the child’s treatment plan.
“This study demonstrated the principle of genomically targeted therapy and represents a possible paradigm change in treatment for these patients,” Patrick Ma, M.D., principal investigator of the WVU Cancer Institute study, said. “Larotrectnib works by seeking out the NTRK cancer gene that is activated by a form of gene fusion in these cancers and prevents its ability to promote cancer growth.”
Patients enrolled in the study had TRK fusion cancers caused by NTRK gene mutations that were detected through molecular profiling, a technology that allows clinicians to determine the genomic make-up and origin of cancer and its specific drug responsiveness to inform cancer treatment.
“Importantly, TRK fusion cancers occur in less than one percent of cancers but can be found in more than 20 cancer types,” Dr. Ma said. “Identifying these unique, genomically altered orphan cancers becomes crucially important with highly effective targeted therapy available that can make a real difference in treatment outcomes. With the help of comprehensive molecular profiling, we not only can identify and match patients to optimal therapies, but can also better understand changes in tumor cells throughout treatment and offer patients more personalized therapy.”
Ma is director of the Clinical Lung Cancer Program and co-leader of the Sara Crile Allen and James Frederick Allen Lung Cancer Program at the WVU Cancer Institute. He is co-author of the article "Efficacy of larotrectinib in TRK fusion-positive adult and pediatric cancers," which was published in the Feb. 22 issue of the New England Journal of Medicine.