The US Food and Drug Administration (FDA) has approved the use of a two-drug combination developed by researchers at Rational Therapeutics Institute (Long Beach, Calif) for the treatment of ovarian cancer. This new approval covers the use of gemcitabine(Drug information on gemcitabine) (Gemzar) in combination with carboplatin(Drug information on carboplatin) to treat women who've suffered an ovarian cancer relapse at least 6 months after initial treatment.

Expanded Indication

Before this expanded indication, gemcitabine was FDA-approved to treat breast cancer, lung cancer, and pancreatic cancer. The activity for cisplatin(Drug information on cisplatin) and its derivative carboplatin plus gemcitabine was first recognized as a treatment for ovarian cancer by Robert Nagourney, MD, medical director of Rational Therapeutics and The Todd Cancer Institute at Long Beach Memorial Medical Center. His research team used a human tumor microspheroid platform (an ex vivo assay trademarked as EVA) based on drug-induced programmed cell death.

In November 1995, Rational Therapeutics received emergency approval from the FDA to use the platinum-plus-gemcitabine combination to treat a late-stage ovarian cancer patient who had failed all conventional therapies. Dr. Nagourney used the results of the EVA analysis to identify the combination for this patient, resulting in a remission that lasted several years.

Dr. Nagourney's discovery of the two-drug combination for treating ovarian cancer led to a phase II clinical trial (reported in Gynecologic Oncology, January 2003), which was followed by a national trial conducted by the Gynecologic Oncology Group (reported in Gynecologic Oncology, April 2006).

Assay-Directed Therapy

"We are delighted that our laboratory observation based on tumor-tissue profiling has given rise to a new treatment option for advanced ovarian cancer," said Dr. Nagourney. "FDA approval of the carboplatin-plus-gemcitabine combination affirms the utility of ex vivo drug induced cell death analysis as a tool to guide drug development for other difficult-to-treat cancers." Results from Rational Therapeutics' EVA assay have been shown in published studies to correlate with patient response, time to progression, and survival.

Dr. Nagourney added, "Clinically validated drug testing assays hold much promise as a means to guide drugs through the phase I-phase II transition, which is an especially costly and time-consuming part of the drug development process."

Ovarian cancer recurs in 90% percent of women who are diagnosed and treated. According to the American Cancer Society, there will be an estimated 20,180 new cases of ovarian cancer in the United States this year.