U.S. announces schedule of trial for linking targeted cancer drugs to gene mutations

The U.S. government will launch what it called a "groundbreaking" precision medicine trial in July to determine whether targeted therapies based on cancer patients' specific gene mutations, rather than their cancer type, will be effective.

The U.S. National Cancer Institute (NCI) announced Monday in a statement that more than 20 different study drugs or drug combinations, each targeting a specific gene mutation, will be incorporated in the trial, known as NCI-MATCH.

Overall, about 3,000 patients will have their DNA sequenced to detect genetic abnormalities that may be driving tumor growth. About 1,000 patients whose gene mutations might be targeted by one of the study drugs or drug combinations will be enrolled into the various treatment arms in the trial.

NCI acting director Doug Lowy called NCI-MATCH "a unique, ground-breaking trial" and said it "holds the potential to transform cancer care."

"It is the first study in oncology that incorporates all of the tenets of precision medicine," said Lowy. "There are no other cancer clinical trials of this size and scope that truly bring the promise of targeted treatment to patients whose cancers have specific genetic abnormalities."

For the new trial, those eligible will be adults 18 years of age and older with solid tumors or lymphomas that have advanced following at least one line of standard systemic therapy, or with tumors for which there is no standard treatment. It also calls for at least 25 percent of the 1,000 patients enrolled to involve people with rare types of cancer.

The cancer treatment drugs being used in the trial will include both U.S. Food and Drug Administration approved drugs as well as investigational agents that are being contributed by a number of pharmaceutical companies, such as Pfizer and Roche.

The primary goal for the trial will be whether or not there is a tumor response to treatment, defined as tumor shrinkage by at least 30 percent in one dimension such as thickness, width, or length. The secondary goal is six-month progression-free survival, which is a measure of whether a patient's disease remains stable.

"For our purposes, a response rate of 5 percent or less in a molecularly-defined population will not be considered promising, whereas a response rate of 16 percent to 25 percent will be encouraging," said NCI study co-chair Barbara Conley, associate director of the NCI's Cancer Diagnosis Program. "After starting treatment in NCI-MATCH, a 6-month progression-free survival of 15 percent will not be considered promising, whereas a progression- free survival at six months of 35 percent will indicate that we would want to develop that treatment further."

Most medical treatments have been designed for the "average patient." As a result of this "one-size-fits-all-approach," treatments can be very successful for some patients but not for others.

Earlier this year, U.S. President Barack Obama announced plans for a "Precision Medicine Initiative," which refers to the use of a person's cancer genetic information to diagnose or treat their disease. The initiative was touted by the Obama administration as having the ability to revolutionize the way people improve health and treat disease. Endite