US Pharm. 2018;43(5)(Specialty&Oncology suppl):16-18.
A first-of-its-kind drug targeting a fused gene found in many types of cancer was effective in 93% of pediatric patients tested, researchers at the University of Texas (UT) Southwestern’s Simmons Cancer Center announced.
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, no matter what cancer type. The research appears in The Lancet Oncology.
“In some cancers, a part of the TRK gene has become attached to another gene, which is called a fusion. When this occurs, it leads to the TRK gene being turned on when it’s not supposed to be, and that causes the cells to grow uncontrollably. What’s unique about the drug is it is very selective; it only blocks TRK receptors,” said lead author Dr. Ted Laetsch, assistant professor of pediatrics and with the Harold C. Simmons Comprehensive Cancer Center.
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, said Dr. Laetsch, who leads the Experimental Therapeutics Program (ETP) at the Pauline Allen Gill Center for Cancer and Blood Disorders at Children’s Health in Dallas.
“Every patient with a TRK fusion-positive solid tumor treated on this study had their tumor shrink. The nearly universal response rate seen with larotrectinib is unprecedented,” Dr. Laetsch said.
