"We now have with sorafenib (Nexavar, Bayer/Onyx) something that works," said Joseph Llovet, director of research at the Mount Sinai School of Medicine in New York. "This is the first time we've had an effective systemic treatment for liver cancer."

Reporting to the 43rd annual meeting of the American Society of Clinical Oncology, Llovet said that when compared to placebo, patients who received sorafenib tablets lived an average of 10.7 months. The patients on placebo survived an average of 7.9 months.

Although modest, the 2.8 month survival advantage for patients on the drugs did reach statistical significance, he said.

Sorafenib, a tablet taken orally, is approved in the United States for treatment of kidney cancer, and is being tested in other cancers as well. About 19,000 people in the United States contract liver cancer each year and more than 16,000 people die of the disease each year, according to statistic from the American Cancer Society.

"This really is a big deal," said William Blackstock, professor of radiation oncology at Wake Forest University in Winston-Salem, N.C., who moderated a news briefing at which the results of the trial was discussed.

"We really didn't have anything to treat advanced liver cancer, and 40 percent of patients in the United States with liver cancer are diagnosed in an advanced state," Blackstock told United Press International. "This will give us a platform upon which we can perform clinical trials."

He said that conducting a placebo-controlled study would be nearly impossible in the United States because most patients would refuse to enter a trial in which one group would use sham medication.

With the ability to offer a medicine that can extend survival as a comparator drug, new medications can be tested against sorafenib and could lead to further improvements in survival, Blackstock suggested.

"Because there are no therapies that significantly improve survival for the thousands of patients with liver cancer, these findings demonstrate the compelling study results of Nexavar as the new reference standard of care for the first-line treatment of hepatocellular cancer," Llovet said.

In the Sorafenib Hepatocellular Carcinoma Assessment Randomized Protocol, or SHARP, Trial, Llovet and colleagues recruited 602 patients. The doctors assigned 299 patients to receive 400 mg of sorafenib and 303 patients to receive placebo. The trial was truncated because early review indicated that patients were doing better with sorafenib than with placebo.

"We are looking at a true increment in survival," Len Lichtenfeld, deputy chief medical officer for the Atlanta-based American Cancer Society, told UPI. "While primary liver cancer is not a huge disease concern in the United States, it is a serious problem worldwide. In the United States, its incidence is increasing."

Lichtenfeld said one of the advantages with sorafenib in treating liver cancer is that the drug does not appear to cause a lot of toxicity. "In the study, the toxicity was similar to that of placebo," he noted

Blackstock told UPI that he expects doctors who have patients with liver cancer to start using the drug as soon as possible. The question of whether the drug will be reimbursed by third party payers is in the air. Blackstock said the drug costs $5,000 a month.

Llovet said some insurers are reimbursing patients for treatment with sorafenib, even though the drug has yet to be approved for liver cancer.