New drug for leukemia shows promising results in early phase trial

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Written By: Shraddha Rupavate | Updated : December 9, 2014 10:26 AM IST

As per recent findings of a study presented at the 56th annual meeting of the American Society of Hematology, a new cancer therapy has showed promising results in treating patients suffering from advanced leukaemia.

AG-221 -- New cancer therapy

About 15 percent of patients having acute myeloid leukemia carry a mutated form of the IDH2 gene. This mutation prevents the development of immature white blood cells into healthy, mature immune cells that fight infection effectively. As a result, abnormally growing cells crowd out normal cells, leading to the development of acute leukaemia. (Read: Soon, leukemia could be treated with artificial bone marrow developed in Germany!)

Researchers found that the investigational drug AG-221 could block this mutation in IDH2 protein. Interestingly, the drug works by targeting a gene that can transform cells to become healthy again instead of inhibiting the IDH2 mutation that leads to cancerous growth. The drug allows the immature white blood cells to grow normally.

Dramatic overall response rate observed

For the study, 45 patients suffering from leukemia and having IDH2-positive leukaemia or haematologic malignancies were included. These patients had advanced disease with a prior relapse or those who were earlier unresponsive to prior therapy. In the early phase trial, the patients were administered 150 mg or 200 mg of AG-221 once or twice daily in 28-day cycles. (Read:9 blood cancer signs and symptoms you should know)

The overall results showed dramatic response rate. Overall 56 percent response rate was observed. with 33 percent (15 patients) achieved complete remission and 10 patients (22 percent) partial remission. 17 patients (38 percent) reached a stable stage.

What next?

According to lead author of the study Eytan M. Stein, medical oncologist at the Memorial Sloan Kettering Cancer Center in the US, the investigational drug has the potential to transform the treatment of leukemia.

'We 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 malignancies,' Stein concluded.

Image source: Getty images


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