Kootenai Medical Center is one of a handful of sites in the U.S. studying a new drug aimed at treating a rare form of cancer, and the Coeur dAlene hospital says its seeking to position itself as a regional center in that type of research.


The experimental drug thats being investigated at KMC, called Yondelis, originally was derived from a compound in a tiny marine organism, commonly called a sea squirt, says Dr. Brian Samuels, who is heading the research at KMC. The compound now is being produced synthetically by Madrid, Spain-based PharmaMar, in partnership with Raritan, N.J.-based Johnson & Johnson Pharmaceutical Research & Development LLC, Samuels says. Studies have shown Yondelis to be effective in shrinking soft-tissue sarcoma tumors and preventing the progression of the disease.


Soft-tissue sarcoma is a rare form of cancerabout 5,000 people are diagnosed with it in the U.S. each yearthat occurs in muscles, fat, tendons, and nerves, and can be difficult to treat, Samuels says. Until recently, such sarcomas have been treated with chemotherapy drugs designed to be used to treat other types of cancers, he says.


Samuels has conducted two Yondelis-related studies so far at KMC, is preparing to launch another soon, and plans to do several others in the next few years. Meanwhile, KMC has been treating more than a dozen soft-tissue sarcoma patients from the region with Yondelis on a restricted basis until the drug gets approved by the U.S. Food and Drug Administration, he says.


Were hoping to put KMC on the map for sarcoma research, Samuels says. We want to develop research here so that at least we can be a resource even if patients dont get their care here.


KMC became a site for Yondelis studies in 2004, when Samuels moved to Coeur dAlene from Chicago, where he ran the sarcoma research program at the University of Illinois. While in Chicago, Samuels enrolled about 10 patients in a study that evaluated the effectiveness of different doses of Yondelis in treating soft-tissue sarcoma. After arriving in Coeur dAlene, he added a patient at KMC to that study, which closed last year and the results of which have been submitted to the FDA for approval.


The most recent study at KMC, which started in October 2005 and was completed last June, involved treating soft-tissue sarcoma patients with a combination of Yondelis and another chemotherapy drug, called adriamycin, and evaluating effective dosage levels of that combination, Samuels says. Two patients at KMC were involved in the study, which included a total of 41 patients with advanced sarcoma and was conducted at about six research centers in the U.S. and two in France, he says. As a result of the treatment, tumors shrinked or stabilized in roughly 40 percent of the patients in the study, whose tumors prior to the treatment had been resistant to other chemotherapy drugs, he says.


For patients with advanced sarcoma, this is a good outcome, Samuels says. Its pretty unprecedented.


Currently, researchers are discussing whether to study a larger group of patients using the combination of the two drugs, since the FDA doesnt approve drugs based on studies as small as the one that was completed recently, he says. Samuels says he hopes, though, that Yondelis will be approved for soft-tissue sarcoma treatment in the U.S. within the next year. It would be the first drug approved by the FDA specifically for treating the disease, he says.


In a similar drug-pairing study, Samuels hopes to get approval soon from KMCs institutional review board, which oversees projects involving human subjects at the hospital, to start enrolling patients in an experiment involving Yondelis and a drug called doxil thats used to treat advanced ovarian cancer. Samuels also hopes to enroll patients at KMC in a study that PharmaMar and Johnson & Johnson are considering launching to examine Yondelis effectiveness in treating prostate cancer.


Meanwhile, KMC is set to become one of two sites in the U.S. to conduct a study on Yondelis effectiveness in treating lyposarcoma, which is a soft-tissue sarcoma found in fat cells, in an experiment that will start in about four months, he says. Researchers are looking to enroll about 50 patients in that study worldwide, and Samuels says he hopes to study about three patients at KMC.


Were looking at a small subset of a subset of a rare disease, so two or three patients here would be a major contribution, he says.


Because of KMCs involvement in Yondelis research, the hospital has been approved to administer the drug to certain patients through an FDA program called expanded access protocol, which makes investigational drugs that have been shown to be effective against specific cancers available to patients before the FDA approval process has been completed, Samuels says. Through that program, KMC so far has treated about 15 patients from around the Northwest who have soft-tissue sarcoma thats been resistant to standard treatments. The hospital is one of three sites on the West Coast where patients can receive Yondelis treatments on that basis, he says.


If we can offer clinical trials along the way without patients having to go to major centers on the East or West Coast, that would be a major plus for patients, Samuels says.


Contact Emily Brandler at (509) 344-1265 or via e-mail at [email protected].