FDA Expands Use of Targeted Therapeutic for Lung Cancer

Targeted therapeutic is the first treatment approved by the FDA for patients with lung cancer positive for mutations in the ROS-1 gene.

The U.S. Food and Drug Administration (FDA) recently announced that it had approved the use of crizotinib (Xalkori) for treating patients with advanced non-small cell lung cancer (NSCLC) that tests positive for a mutation in the ROS-1 gene. This is the first time the FDA has approved a treatment for this group of patients.

NSCLC is the most common form of lung cancer diagnosed in the United States; researchers estimate the disease will account for about 83 percent of the 224,390 new cases of lung cancer expected to be diagnosed in 2016. About 5 percent of NSCLC cases are fueled by aberrant activity of a protein called ALK, which is very closely related to the ROS-1 protein. Crizotinib was approved for treating patients with ALK-positive NSCLC in August 2011 after it was shown that 50 percent of the patients treated with the ALK-targeted therapeutic experienced complete or partial cancer shrinkage.

Given the similarities between ALK and ROS-1 and that in vitro studies showed that crizotinib could potently inhibit both kinases, researchers decided to investigate whether crizotinib might benefit the approximately 1 percent of patients with NSCLC whose tumors have aberrantly active ROS-1 as a result of mutations in the ROS-1 gene.

According to results of a phase I clinical trial published in late 2014 in The New England Journal of Medicine, the answer was yes ? 72 percent of patients with advanced NSCLC whose cancer tested positive for ROS-1 mutations had complete or partial shrinkage of their tumors, and these responses lasted for a median of 17.6 months.

With the five-year survival rate for lung cancer still at just 17 percent, advances like this are urgently needed.

The FDA approval was rendered on Mar. 11, 2016.