For the second time, the Food and Drug Administration has approved a drug treatment for patients with a specific genetic change in their tumor, regardless of the type of cancer.

At Hartford Healthcare, Laila Mnayer, division director of Molecular Pathology and Cytogenetics, had already identified the importance of these fusion mutations, called neurotrophic tropomyosin receptor kinase (NTRK). She updated the cancer genomics testing conducted at Hartford Hospital to include NTRK1, 2 and 3 fusion genes that are known to be susceptible to Vitrakvi (larotrectinib), the drug newly approved by the FDA. Now, patients here can be treated with this new drug if Dr. Mnayer’s team detects any of the NTRK fusion mutations in the tumor.

Larotrectinib was approved on Nov. 26 and added to the National Comprehensive Cancer Network guidelines for adult and pediatric patients with tumors that carry the NTRK genetic fusion mutation without a known acquired resistance mutation. NTRK mutations are uncommon but can be found in hard-to-treat metastatic cancers such as non-small cell lung carcinoma, thyroid cancer, soft tissue sarcoma and cancers of unknown origin.

“Finding novel molecular cancer drivers like NTRK fusions and then matching these with effective drugs like larotrectinib are just two of the latest examples of how scientists are chipping away at cancer morbidity and mortality,” noted Dr. Omar Eton, medical director of the Hartford HealthCare Cancer Institute’s Melanoma and Skin Cancer department. “Cancer specialists are increasingly leveraging technologies to identify driver mutations in their patients’ tumors; this is the latest advance that can help extend the duration and quality of life of cancer patients.”

This is the second time in as many years that the FDA approved a drug based specifically on a molecular target, rather than the more traditional approval based on activity in a specific cancer type. The first drug — approved May 23, 2017 — was the checkpoint antibody Keytruda (pembrolizumab), which is effective  in a high proportion of patients with tumors that have microsatellite instability, also known as mismatch repair deficiency.

“We have entered the ‘promised land’ as we always envisioned it – we can finally leverage powerful tools to identify molecular drivers of cancer, design drugs that interfere with or exploit these, and deliver effective drugs tailored to the features of an individual patient’s cancer,” Dr. Eton said. “These are exciting times, as these same tools will produce more such advances, while also solving problems of drug side effects and resistance.”

For more information on the Hartford HealthCare Cancer Institute and genetic testing for the NTRK biomarker, click here.