What’s it going to take before an effective treatment gets approved by the FDA for acute and aggressive mastocytosis? Well, hopefully the mastocytosis community won’t have to wait too long!
Midostaurin is now approved for treating adults who’ve recently been diagnosed with AML, which stands for acute myeloid leukemia and test positive for the mutated FLT3 gene. AML is a potentially life-threatening disease. This breakthrough drug is administered orally and inhibits the multi-targeted growth of kinase. Doctors in the study found that patients who received PKC412 treatment lived on average six years compared to just two years for those who receive the placebo! No significant adverse hematologic side effects were reported, although I’m unsure about other adverse events. If you’d like to read the article, you can read it here.
But what’s also REALLY exciting—is that the same drug, is also being tested to determine if it’s effective against mastocytosis—specifically, the very aggressive, systemic form of mastocytosis, which is also known as mast cell leukemia. The drug’s maker Novartis, which is headquartered in Basel, Switzerland, has joined forces with Invivoscribe Technologies, Inc. in the development to identify a FLT3 mutation in patients.
Will Novartis rock the house again? Stay tuned to see if PKC412 treatment works well for mastocytosis!
Do you have AML or mastocytosis and did or are you participating in a clinical trial? If so, how well did you tolerate the drug? Has it helped you? Thanks for your support to get the word out! Please share!