A new drug called AG-221 is being used in the treatment of patients with acute myelogenous leukemia (AML), a disorder which affects the blood cells in bone marrow. While the drug is still in Phase I of its trials, doctors seem optimistic about the results. Instead of destroying the leukemic cells, the drug forces them to mature, leading in some cases to complete remission within six months. The results have lead researchers who worked on AG-221 to being studying new drugs for other forms of leukemia and cancer.

Understanding leukemia

The Mayo Clinic defines leukemia as “cancer of the body’s blood-forming tissues, including the bone marrow and the lymphatic system.” For reasons that aren’t quite clear, leukemic cells fail to mature; instead, they keep multiplying in number until the healthy, mature cells are forced out.

Acute myelogenous leukemia can develop when the DNA in a person’s bone marrow is damaged in some way. It affects around 50,000 people each year, and the fatality rate hovers around 75%. In approximately 15% of patients with AML, a gene which produces an enzyme (called IDH-2) that helps cells generate energy mutates, causing the cells to multiple rapidly.

What makes AG-221 different from other treatment options

AG-221 targets that specific gene mutation by forcing the cells to mature as opposed to trying to kill them off. One of the biggest problems with most forms of cancer treatment is that the medications kill off the healthy cells as well as the cancerous ones. The chemotherapy treatments for AML use a two-drug mix: one drug can cause damage to the heart muscle, and the other causes damage to the nervous system. In particular, the cerebellum is at risk. Statistically, most patients die before the drugs can be effective – and those who don’t are severely weakened by the effects. In comparison, AG-221 may cause a loss of appetite and some mild nausea.

What this means for the future of cancer treatments

The company which produces AG-221 is Agios, and they’re already working on a drug which targets a different mutated gene. If the second drug proves successful in AML patients, they may begin trials for people with glioma (a kind of brain tumor) and people suffering with cancer of the cartilage.

While all of this news is exciting, it’s crucial to remember that this new drug is still in the experimental stage. Most patients with AML die within five years; AG-221 has only been in trials for a few months. For now, a cautious optimism might be the best way to think about this new drug.

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