Midostaurin in AML: First Targeted Agent Improves Survival

Midostaurin in AML: First Targeted Agent Improves Survival

I have intentionally left out the majority of the findings as they are a bit too heavy in terms and statistics and tone for the regular guy. The points here are HUGE . It has been thirty years since a breakthrough occurred in AML. Also this breakthrough, as long  predicted ,is not yet for all AML patients but ones with a specific cellular target. If present, every question you ask about survival ,remission ,toxicity, and duration of good news is dramatically in favor of this new agent Talk to your Oncologist |

ORLANDO, Florida — There hasn’t been a new drug approved for the treatment of acute myeloid leukemia (AML) since 1990, but one may be coming soon, after results show that the first targeted agent for the disease, midostaurin (under development by Novartis), has shown an improvement in survival in a subset of patients.

For the last 30 years, the cornerstone of treatment of acute myeloid leukemia (AML) has been an anthracycline such as daunorubicin together with cytarabine (DepoCyt, Sigma Tau), given in a “3+7” schedule, and nothing added to this regimen has improved outcomes much. Even midostaurin, when it was first tested in AML, failed.

It was only the realization that this is a targeted agent and the restriction of this drug to patients with the FMS-like tyrosine kinase 3 (FLT3) mutation that produced the results showing an overall-survival benefit. That took 10 years and an unusual academia-industry collaboration, which resulted in the CALGB 10603 (RATIFY) study. The study was conducted by the Alliance for Clinical Trials in Oncology, with the drug and funding for the North American portion provided by Novartis.



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