Hope for AML patients with rare gene mutation

Time of India

5 hours ago

Pune: New and emerging therapies, which cause less collateral damage to normal blood and target specific mutations, are a promising alternative to treat Acute Myeloid Leukemia (AML), several oncologists have said.
June is AML awareness month.
Specialists have begun using ivosidenib, one such drug, that targets AML with IDH mutation, after Central Drugs Standard Control Organisation approved it in May.
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India accounts for the highest number of AML cases after the US and China as per statistics from 2021.
Dr Sameer Melinkeri, clinical haematologist at Pune's Deenanath Mangeshkar Hospital & Research Centre, said new therapies such as ivosidenib, azacitidine and venetoclax, help precisely target individual mutations in AML.
"Ivosidenib is the first targeted therapy developed specifically for AML patients who carry the rare IDH1 gene mutation," said Dr Melinkeri.
"It blocks a faulty enzyme—mutant IDH1—that causes the buildup of a harmful substance called 2-hydroxyglutarate in the body. This substance stops blood cells from maturing and spreads cancer cells," the doctor added.
He said ivosidenib turns off the cancer-causing switch (mutation) in patients with IDH, a specific gene mutation.
By stopping the enzyme, ivosidenib helps blood cells mature normally, reducing cancerous cells and allowing healthy cells to grow.
Dr Reshma Puranik, a medical and haemato-oncologist with MOC, Cancer Care and Research Centre said that ivosidenib is a first-in-class, oral, small-molecule inhibitor of the mutant IDH1 enzyme and works well in brain tumors with IDH mutation.
In trials, ivosidenib and azacitidine achieved superior outcomes with similar toxicity compared to azacitidine with newly-diagnosed IDH1-mutated AML.
"Patients on ivosidenib need to monitor blood counts along with renal and liver function tests, serum electrolytes and QT interval on ECG. Some patients get a minor rash, feel fatigued, have oral mucositis (ulcers) and are at risk of differentiation syndrome," Dr Puranik added.
While the new targeted therapies are expensive, more awareness and distributors in the market will bring the cost down, experts say.
Dr Shilpa Gupta, consultant haemato-oncologist in a Mumbai-based cancer centre, said, "I have started ivosidenib for a patient and will wait for the results."