A drug currently used to treat kidney cancer has shown promising results in treating the most common and deadly form of adult leukemia, according to a US study released Tuesday.
The drug sorafenib attacks a genetic mutation active in about a third of patients with acute myeloid leukemia (AML).
"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 senior author Michael Andreeff of the University of Texas M. D. Anderson Cancer Center in Houston.
In a Phase I clinical trial, sorafenib reduced the median percentage of leukemia cells circulating in the blood from 81 percent to 7.5 percent and in the bone marrow from 75.5 percent to 34 percent among AML patients with the genetic mutation.
Two of the 16 patients had circulating leukemia cells, or blasts, drop to zero.
There have been no major side effects in the clinical trial to date, so no maximum tolerated dose has been reached, Andreeff said.
The drug has little effect on cells with normal versions of the gene and does not interfere with normal blood cell formation.
Andreeff's group has already opened a Phase II trial that combines sorafenib with the standard of care chemotherapy combination for acute myeloid leukemia.
As safety and dose escalation research progress, sorafenib could assume a role in frontline therapy, he said.
About 14,000 new cases of AML are diagnosed annually in the United States and the disease kills about 9,000 people each year.
The study was published in the Journal of the National Cancer Institute.