New Leukemia Drug AG-221 Re-kindles Hope Even in Advanced Cases

Early clinical trials in patients with advanced leukemia show that AG-221, a new drug  developed, has shown dramatic results when administered among the patients.

The new drug AG-221 works by targeting a gene that can transform cells into becoming healthy again instead of stopping a mutation that leads to cancer cell growth.

Approximately 15 percent of acute myeloid leukemia patients have a mutated form of the IDH2 gene, which prevents immature white blood cells from developing into healthy, infection-fighting cells which accumulate, crowd out normal cells, and lead to the development of acute leukaemia.

AG-221 is an investigational drug under trial that blocks the mutated IDH2 protein, effectively allowing these immature white blood cells to develop normally. “This drug has the potential to transform the treatment of leukemia,” said lead study author Eytan M. Stein, medical oncologist at the Memorial Sloan Kettering Cancer Center in the US.

“llWe have not yet reached the maximum tolerated dose and patients are responding dramatically. More research is needed, but I am optimistic that this drug will fundamentally alter the natural history of IDH2-mutant leukemia and other hematologic┬áll


In their study, 45 patients with IDH2-positive leukaemia or previous history of malignancies or advanced phase of the disease or with relapsed cases or unresponsive to prior therapy — were given the drug AG-221 .

The trial case patients were given up to 150 mg or 200 mg of AG-221 once or twice daily for 28 days and when evaluated, the overall response rate was 56 percent as 15 patients or 33 percent of the sample patients achieved complete remission and 10 patients or 22 percent of them achieved partial remission.

The condition of 17 patients or 38 percent became stable and there were no treatment-related deaths. The findings were presented at the 56th annual meeting of the American Society of Hematology.

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