Cancer Covered Blog

FLT3 mutations in AML: From Trivia to Triumph

Here’s an aggravating fact about medical education–a large chunk of what’s taught in medical school is useless trivia:

  • The muscle that elevates your palate is called the tensor veli palatini…
  • Here’s the best way to treat leprosy…
  • Adults whose acute leukemia cells carry an internal tandem mutation of the FLT3 surface tyrosine kinase have a worse prognosis…

I and other cancer specialists have known that last little nugget for about ten years, but haven’t been able to do squat with it:

“So should I use a different induction chemotherapy for FLT3-mutants?” (No)

“Should all FLT3 mutants get a stem cell transplant?” (Not necessarily)

“Is there anything I can do differently for these patients?” (Silence)

Thanks. Thanks a fricken’ lot.

It’s a little unfair to rant, I know. There’s a long lag time between discovery and new treatments for cancer -but the wait is hard, especially with new leukemia patients coming in the door every month.

But last week finally– finally–a drug targeting this specific molecular defect was approved by the FDA.

See, FLT3 is like an “on” switch for cell division. It’s supposed to only be on when it gets a particular environmental signal, and not every cell even carries the switch. But about 35% of acute myeloid leukemia patients have FLT3 abnormally expressed in their cancers – and the switch is stuck in the “on” position, driving cancer cell growth.

Happily, the new FLT3-inhibiting drug midostaurin (given as a twice-daily pill) markedly improved response and survival rates in AML when added to the standard chemotherapy cocktail. The benefits seemed most pronounced among people who ultimately went on to receive a stem cell transplant – but a higher percentage of patients were able to achieve the remission usually required as a first step before transplant.

So now I can re-categorize the FLT3 information from “trivia” to “critical information” in my brain, as I impatiently wait for more breakthroughs.

FDA Press Release 4/28/17: click here

Dr. Mitch Winkler signature
J. Mitchell Winkler, MD

Mitch has boundless energy and an unyielding approach to finding the most suitable solutions for each of his patients.

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