The new treatment has demonstrated impressive results in patients with the common form of leukemia in the initial phase of clinical trials. These studies, conducted under the direction of Dr. Eytan M. Stein of the Cancer Center Memorial Sloan Kettering, was presented at the 56th Annual Meeting of the American Society of Hematology.
Scientists have tested the new drug for the treatment of leukemia
Approximately 15% of patients with acute myeloid leukemia have mutations in the gene IDH2. IDH2 gene involved in the production of a protein that plays an important role in cell metabolism. When a mutation of this gene increases the production of abnormal protein 2-hydroxyglutarate, which inhibits differentiation of progenitors of white blood cells - myeloblasts to mature forms, are responsible for fighting infections. These immature cells accumulate displace normal cells and lead to the development of acute leukemia.
The new drug - AG-221, blocks the mutated protein IDH2, thereby helping immature white blood cells to differentiate into neutrophils. "Current treatments for leukemia (chemotherapy, radiation therapy, stem cell transplantation) to kill cancer cells. However, they have severe side effects because they kill healthy cells of the hematopoietic system. Therefore, targeting therapy is the most safe and effective at the same time ", - says Dr. Stein.
In the study, 45 patients with acute myeloid leukemia mutation IDH2 were able to complete the first stage of treatment. All patients had the same stage of the disease, which is not applied to modern methods of treatment. Patients received 150 mg or 200 mg AG-221 in one or two steps per day for 28 days. The maximum tolerated dose was not reached.
Efficacy was 56 percent; 15 patients (33%) achieved a complete clinical remission, and 10 patients (22%) partial remission. In addition, 17 patients (38%) showed a decrease of disease symptoms. Remission lasted for eight months and is still ongoing. During the study there was no deaths, treatment-related.
"This drug has the potential to use it as a drug against leukemia. We have not yet reached the maximum tolerated dose. More research is needed to accurately assess the efficacy and safety of this drug in the treatment of IDH2 positive leukemia and other hematologic malignancies, "- says Dr. Stein.
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