New kind of targeted drug shows promise for leukemia

11 December 2014

A new kind of cancer therapy has shown promising results for patients suffering from advanced leukemia. 

The early-phase clinical trial, led by Dr Eytan Stein, a medical oncologist at Memorial Sloan Kettering Cancer Center, found that more than half of patients involved (56 per cent) achieved complete remission after the therapy.

Around 15 per cent of patients with acute myeloid leukemia patients have a mutated form of the IDH2 gene, which is normally key for cell metabolism. However, when mutated, the gene triggers an increased production of 2-hydroxyglutarate, which prevents white blood cells from becoming infection-fighting cells. These immature cells accumulate and lead to the development of acute leukemia.

In the new study, a drug was developed to block the mutated IDH2 protein, allowing these immature white blood cells to develop normally. 

Dr Stein said traditional forms of cancer therapy kill diseased cells but have major side effects, as they can also damage healthy cells, making targeted therapies much better. He added that the new drug is "especially unique" as it targets a gene that transforms cells instead of the mutation that leads to cancer cell growth.

Overall, the study found that an additional 22 per cent of participants achieved partial remission. and 38 per cent had stable disease.

Posted by Edward Bartel


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