CHICAGO (Reuters) - The kidney cancer pill Nexavar has shown promise in treating a small number of people with a type of blood cancer known as acute myeloid leukemia or AML, U.S. researchers said on Tuesday.
They said the Bayer AG and Onyx Pharmaceuticals Inc drug Nexavar, also known as sorafenib, dramatically reduced the percentage of leukemia cells circulating in the blood of 16 people whose cancer is linked with a mutation of the FLT3 gene. This mutation occurs in about one third of all AML patients.
“AML patients with this mutation have a particularly poor prognosis, so this highly targeted drug appears to be a significant step forward in leukemia therapy,” said Dr. Michael Andreeff of M.D. Anderson Cancer Center at the University of Texas.
AML is a type of cancer in which the bone marrow makes abnormal blood cells. It is the most common type of acute leukemia in adults.
The percentage of cancer cells circulating in the blood of patients in the study dropped to 7.5 percent, from 81 percent. In two patients, leukemia cells circulating in the blood dropped to zero, the researchers reported in the Journal of the National Cancer Institute.
People in the study had failed other treatments.
But Andreeff said in a statement the drug alone would not be able to defeat the disease, which attacks patients in several ways. He said Nexavar would need to be combined with other treatments.
So far, there have been no major side effects. Andreeff said the drug has little effect on AML patients who do not have the genetic defect.
A study last week in the journal Lancet Oncology, however, found the drug significantly raises the risk of high blood pressure, and researchers said people taking the drug should be monitored and treated for that risk.
Nexavar was recently approved to treat liver cancer in the United States and Europe. It is currently being tested for use against several other types of cancer, including non-small cell lung cancer and breast cancer.
About 14,000 cases of AML are diagnosed each year in the United States. The disease kills about 9,000 people a year. Patients are typically treated with a combination of drugs, with the hope of bringing about remission.
Editing by Maggie Fox and Alan Elsner