Drug for Kidney Cancer Offers Hope in Treating Deadly Leukemia
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.
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