First Treatment For TRK Fusion Cancer Approved

Two women in lab coats working in a lab.

The United States Food and Drug Administration (U.S. FDA) has approved a new treatment option for children and adults suffering from TRK fusion cancer.

The first-in-class treatment option – developed by Bayer and Loxo Oncology, Inc. – has been approved for the treatment of adult and pediatric patients with solid tumors with a neurotrophic receptor tyrosine receptor kinase (NTRK) gene fusion without a known acquired resistance mutation that are either metastatic or where surgical resection will likely result in severe morbidity, and have no satisfactory alternative treatments or have progressed following treatment.1

Which U.S. patients could this treatment be appropriate for?

After approval for U.S. patients, the treatment may be appropriate for both adults and children with tumors that have spread (metastasized) to other areas of the body, where surgery will result in severe disease or whose cancer is still present even after treatment. Overall, this type of cancer is rare, but can occur across a broad range of tumor types, including cancers of the salivary gland, thyroid, lung, colon, appendix, breast and pancreas.


What is TRK fusion cancer?

TRK fusion cancer is caused by a genomic alteration; in this case it occurs when a neurotrophic tyrosine receptor kinase (NTRK) gene fuses with another, unrelated gene. This alteration leads to an overexpression of a TRK fusion protein, which can cause the growth of a tumor. Until now, there was no standard of care for TRK fusion cancer and many patients, including children, could have potentially faced invasive treatments such as amputation or chemotherapy. The new treatment is not yet approved outside of the U.S., but Bayer and Loxo Oncology are developing the compound globally.


What are NTRK gene fusions?

NTRK gene fusions are genomic alterations that result in the production of TRK fusion proteins, which drive tumor cell spread and survival.2 The treatment, which is now approved in the U.S., is the first ever FDA-approved therapy with a primary tumor-agnostic indication. This means that the treatment isn’t indicated to treat cancer of a certain tumor type, but rather, the underlying source that's driving a patient's cancer to grow, wherever the tumor is located in the body. This is an example of how knowledge into the inner workings of cells helps us to more accurately treat cancer.


What role does genomic testing play?

By using genomic testing, it is possible to more precisely identify patients who could benefit from certain treatments. This marks a turning point of innovation in cancer care that will make a real impact across the community. Genomic testing can potentially help patients reduce the number of failed treatments they undergo by helping to identify an appropriate therapy earlier in the treatment journey that could be both effective and well-tolerated. For the first time, we can match patients whose tumors have a NTRK gene fusion with a treatment designed specifically for this genomic alteration.


Why is genomic testing important for treating TRK fusion cancer?

There is a need to incorporate widespread testing for NTRK gene fusions into clinical practice so that we can effectively treat patients with TRK fusion cancer. It is beneficial for all patients with solid tumors to undergo genomic testing to determine the underlying cause of the spread and survival of their tumors. As the cancer community continues to innovate, new treatment options are becoming available that can help address unmet patient needs and provide more tools to manage the disease. The results of genomic tests can help identify certain biomarkers for a tumor and make it possible to match the patient to the appropriate treatment option.




1 Vitrakvi® (larotrectinib) capsules and solution for oral use [Prescribing Information]. Stamford, CT: Loxo Oncology Inc.; November 2018.

Amatu A, Sartore-Bianchi A, Siena S. NTRK gene fusions as novel targets of cancer therapy across multiple tumour types. ESMO Open. 2016;1(2):e000023.