New Cancer Drug Saves Teen's Life

14-year-old Briana Ayala of El Paso is a budding artist but says she recently had to use her talent as a way to distract her from the pain caused by a rare tumor in her abdomen.

"It was a lot of pain for me and wondering what was happening to me," says Ayala.

She was diagnosed at age 11 with a rare form of sarcoma and started traditional treatments, like chemotherapy, however the tumor continued to grow.

Dr. Theodore Laetsch, Assistant Professor of Pediatrics at UT Southwestern and Children's Health sent her tumor for genetic testing and found that Ayala’s cancer had the TRK fusion, meaning the new drug might help.

Larotrectinib is the first cancer drug to receive FDA breakthrough therapy designation for patients with a specific fusion of two genes in the cancer cell.

Since Ayala's cancer had the TRK fusion, she was enrolled in the trial.

"It was exciting when we found out she had this mutation because this trial had just opened and we just heard about the first respondents in adult patients. Even more exciting was she came back to clinic and we could see within a couple of weeks that her stomach, which had been swollen from the cancer, was flat and the pain was much better," says Dr. Laetsch.

Most cancer drugs are targeted to specific organs or locations in the body.

Larotrectinib targets TRK fusions, which can occur in many types of cancer.

While the TRK fusions occur in only a small percentage of common adult cancers, they occur frequently in some rare pediatric cancers, such as infantile fibrosarcoma, cellular congenital mesoblastic nephroma, and papillary thyroid cancer, says Dr. Laetsch.

Nearly two years later, Ayala is excited to be cancer-free and back in school.

The TRK-fusion mutation can be present in many types of cancers, including lung, colon, thyroid, and breast cancer, as well as certain pediatric tumors.

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