New ‘life-saving’ drug brings higher survival rate to patients with acute myeloid leukemia

MELBOURNE, Australia — Acute myeloid leukemia (AML) is a particularly aggressive and fast-spreading form of leukemia. It starts in the bone marrow but moves quickly to the blood stream where it can spread to other areas like the lymph nodes, liver, and central nervous system. Physicians consider AML one of the hardest forms of leukemia to treat, but the results of a recent clinical trial indicate a new drug may change all that. Researchers from Monash University say global trials of the drug CC-486 reveals a 30-percent improvement in survival rates among patients.

Notably, the drug seems to be especially helpful for AML patients over the age of 55. Considering that the disease usually grows worse as a patient ages, and most older AML patients pass away within two years of diagnosis, finding a treatment is of the utmost importance.

The trial’s leader, Professor Andrew Wei from the Monash University Australian Centre for Blood Diseases and a hematologist at Alfred Health, says his team focused on older patients because “of an unmet need to identify new agents able to improve outcome in patients after completing chemotherapy.”

“After intensive chemotherapy, the risk of AML relapse is high. Many older patients are not eligible to receive a stem cell transplant and so a less toxic option to reduce disease recurrence is desirable, rather than just being monitored and waiting for the disease to come back. Based on the results of the QUAZAR study, it is very exciting to think that, by taking a tablet that is relatively well-tolerated, we can help reduce relapse risk and improve survival,” Wei explains in a university release.

Prolonging survival without needing hospitalization for AML patients

Researchers examined a total of 472 acute myeloid leukemia patients in the trial. Each person either received CC-486 or a placebo. Those given the real drug had an average survival from remission of nearly 25 months. The average survival time of patients given a placebo was under 15 months.

It’s also worth mentioning that CC-486 comes in pill form, meaning patients can take it at their convenience from the comfort of home. A few months ago, the U.S. Food and Drug Administration fast-tracked CC-486 for approval. So it may not be that long until it’s available for American cancer patients.

Researchers conducted the trial in 148 different locations across 23 countries. Professor Wei believes CC-486 will revolutionize care and treatment among older adults with acute myeloid leukemia.

“Our findings show that CC-486 significantly delays recurrence of the disease, thereby prolonging survival and without impacting on quality of life,” he concludes. “This is a very significant advance because the drug is easy to administer and means that adults with AML don’t have to spend extra time in hospital.”

The study is published in the New England Journal of Medicine.