Three quarters of patients, both adults and children, with a variety of advanced cancers occurring in different sites of the body responded to larotrectinib, a novel therapy that targets a specific genetic mutation. Results of this multisite phase 1/2 trial have been published in the New England Journal of Medicine on February 22, 2018. Unlike most cancer therapies, this oral treatment is based on the genetic traits of the tumor and not the organ where the cancer originated.
An acquired genetic defect, TRK fusions accelerate cancer cell growth. Larotrectinib is highly selective for inhibiting this process. Fifty-five patients, ranging from 4 months to 76 years of age, with 17 unique tumor types, were treated with larotrectinib. Three quarters of patients enrolled responded to therapy and 86% of responding patients remain on study or have undergone curative surgery. No patients discontinued treatment due to drug-related side effects.
Several pediatric patients that enrolled in the study had infantile fibrosarcoma, a type of cancer that harbors a TRK fusion and is difficult to treat since it responds poorly to chemotherapy. Radiation therapy is also not a good option since it has devastating long-term effects for young patients.
“This is truly a magic
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