VIDEO: Positioning the latest treatments for AML

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Major progress has been made in acute myeloid leukemia because of targeted therapies. For years, for about 30 years, treatment had been the same, which was chemotherapy, a combination of chemotherapy. And then by understanding better what are the mutations that cause acute myeloid leukemia or the mechanisms that cause acute myeloid leukemia, we were able to get better therapies.

There are some that are FDA-approved and that are some hopefully that will be approved soon. So among the FDA-approved therapies, we have the FLT3 inhibitors, F-L-T-3 inhibitors, and these would be the medications midostaurin (Rydapt & Tauritmo, Novartis) and gilteritinib (Xospata, Astellas Pharma) and quizartinib (Vanflyta, Daiichi Sankyo). FLT3 mutations occur in about 20 to 30% of patients with acute myeloid leukemia. And these pills are able to, for at least gilteritinib and quizartinib, lead to responses on their own when used as single agents. But currently we use them in combinations and they've improved survival in patients that have acute myeloid leukemia.

And then we have the next set of targeted therapies, which were the IDH inhibitors. So IDH1, IDH2, which are mutated roughly in about 20% of acute myeloid leukemia. And these pills have also improved outcomes for patients, especially older patients with ML, where they could be used as single agents or added in combination to azacitidine and I'm specifying FDA-approved indications.

Lastly and importantly, the medication venetoclax, which is a form of targeted therapy, this targets the apoptosis pathway and when used in combination with azacitidine, it has become the standard of care for older or unfit patients, unable to tolerate high intensity chemotherapy. So we have a few tools to use currently in acute myeloid leukemia, and they're tailored depending on the genetic composition of each patient. And the next set of targeted therapies that are hopefully gonna make it to clinic soon are menin inhibitors. And these would be for specific mutations also, NPM1 mutated acute myeloid leukemia, which is in about 30%, so the largest subset of acute myeloid leukemia, and a less common but very aggressive, or a leukemia that has resistance to treatment, KMT2A rearranged acute myeloid leukemia.