Larotrectinib: a ‘precision drug’ that treats multiple cancers

The drug targets a rare genetic mutation that can be found in a variety of cancers.

DNA in a test tube

Updated on April 22, 2024.

In recent years, researchers have been developing new drugs that may rapidly improve the ability to treat certain types of cancer. These medications are designed to target specific genetic traits of cancers, rather than specific types of tumors. By pinpointing genetic mutations, they can attack tumors regardless of where they grow in the body.

One such drug, called Vitrakvi (larotrectinib), was approved by the United States Food and Drug Administration (FDA) in 2018. It doesn’t target a particular cancer, such as breast or colon cancer. Instead, it treats tumors that carry a genetic feature, or biomarker, known as neurotrophic receptor tyrosine kinase (NTRK) gene fusion.

When they function normally, NTRK genes lead to the formation in the body of a type of protein called TRK proteins. But in abnormal cases, NTRK genes may become fused to other unrelated genes, which could trigger the growth of tumors. These mutations are uncommon, but they can occur in some rare cancers, including a very rare cancer affecting children called infantile fibrosarcoma. They may also appear in a very small percentage of common cancers, in solid tumors that arise in various parts of the body, including the lungs, thyroid gland, and colon.

Vitrakvi, developed by Loxo Oncology, was approved under the FDA’s accelerated approval program, which enables the agency to “green light” drugs for serious conditions to fill an unmet medical need based on promising initial clinical trials.

The drug was studied in three trials, involving 55 children and adult patients diagnosed with solid tumors with NTRK gene fusion that had spread and did not respond to any other treatments. These patients’ tumors could also not be safely removed through surgery.

Overall, the drug had a 75 percent overall response rate. Of the 55 patients included in the trial, 49 percent had a partial response, in which they experienced either a reduction in the size of their tumors or tumors that stopped growing entirely. Meanwhile, 25 percent had a complete response, or their cancer was no longer detectable. Of these responses, 63 percent lasted for at least 12 months while 49 percent lasted for 24 months or more, the FDA reported.

Common side effects of the drug include fatigue, nausea, cough, constipation, diarrhea, dizziness, vomiting, and liver damage. The drug should not be taken by pregnant or breastfeeding people because it could harm a developing fetus or newborn.

More clinical trials are needed to confirm Vitrakvi’s long-term safety and effectiveness. But Vitrakvi’s approval was important for a couple of reasons. For one, it was a significant step forward for precision medicine, which challenges a one-size-fits-all approach to cancer treatment. What’s more, it’s a single drug that could help treat many tumor types.

The population of people affected by NTRK fusion is small. But for these people, the benefits could be dramatic. Until Vitrakvi’s approval, there were no drugs for certain cancers that often carry this mutation. Vitrakvi is available as an oral capsule or a liquid formulation for both adults and children.

Article sources open article sources

U.S. Food & Drug Administration. “FDA approves an oncology drug that targets a key genetic driver of cancer, rather than a specific type of tumor.” November 26, 2018.
National Cancer Institute. “Targeted Therapy Larotrectinib Shows Promise in Early Trials, Regardless of Cancer Type.” March 9, 2018.
Cision/PR Newswire. “FDA Approves Vitrakvi (larotrectinib), the First Ever TRK Inhibitor, for Patients with Advanced Solid Tumors Harboring an NTRK Gene Fusion(1,2).” November 26, 2018.
U.S. Food & Drug Administration. “FDA approves larotrectinib for solid tumors with NTRK gene fusions.” December 14, 2018. Accessed August 11, 2021.
A Amatu, A Sartore-Bianchi, et al. “Tropomyosin receptor kinase (TRK) biology and the role of NTRK gene fusions in cancer.” Annals of Oncology: Official Journal of the European Society for Medical Oncology. 30(Suppl_8), viii5–viii15.
E Cocco, M Scaltriti, A Drilon. “NTRK fusion-positive cancers and TRK inhibitor therapy.” Nature Reviews. Clinical Oncology, 15(12), 731–747.
KJ Caldwell, E De La Cuesta, et al. “A newborn with a large NTRK fusion positive infantile fibrosarcoma successfully treated with larotrectinib.” Pediatric Blood & Cancer. May 26, 2020. Volume 67, Issue 9.
JP Solomon, R Benayed, R, et al. “Identifying patients with NTRK fusion cancer.” Annals of Oncology : Official Journal of the European Society for Medical Oncology. 30(Suppl_8), viii16–viii22.
Vitrakvi.com. Important Safety Information and Indication. 2021.
Vitrakvi.com. How Vitrakvi Can Help (Patient Info on Effectiveness). Updated September 2023. 

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