Gene Based Drug for Acute Myeloid Leukemia

Acute myeloid leukemia treatment with FLT3 inhibitors

/ Author:  / Reviewed by: Robert Carlson, M.D

(RxWiki News) Researchers believe that targeting the cancer gene FLT3 could cure one type of leukemia, and say results from their research explain the failure of several drugs currently used.

Scientists studying the genetic relationships involved in acute myeloid leukemia have used the genetic data produced in their research to refine the search for a new drug, and testing of one candidate is underway.

"Ask your oncologist about clinical trials available to you."

The group from the University of California in San Francisco announced that the gene FLT3 was being investigated as a possible target for treating acute myeloid leukemia, and so far they have not been discouraged by the failure of several drug therapies targeting that gene to get the expected results.

Results from further research showed that the targeting FLT3 is a valid therapy, but the drugs used so far in trials were not strong enough to completely shut off the gene.

Research on acute myeloid leukemia turned up FLT3 as a candidate for pharmaceutical therapy, but debate was strong over how important the gene was to survival of the cancer cells.

This report also backs up the initial conclusion that FLT3 is essential for the process where abnormal cells are transformed into a full blown leukemia.

The first clinical trial involved eight patients with leukemia and the experimental compound AC220.

Initially a success, remission was achieved in each case that the compound AC220 was used in, but all eight patients would later have a recurrence of their leukemia.

“These mutations are critically important for the survival of leukemia cells that harbor them,” said Neil Shah, MD/PhD, study author.

“Our results also identify drug-resistant mutations in FLT3 that represent high-value targets for future drug development, and will hopefully rekindle interest in developing potent FLT3 inhibitors for the treatment of acute myeloid leukemia.”

The study was published online in the journal Nature on April 15.

Financial relationships with Astellas Pharma, Ambit Biosciences, and Pacific Biosciences were disclosed by researchers.

Reviewed by: 
Review Date: 
April 25, 2012
Last Updated:
November 8, 2012