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Research into the causes, diagnosis, and treatment of leukemia is going on at many medical centers, hospitals, and other places.

Genetics of leukemia

Scientists are making great progress in learning how changes in the DNA in normal bone marrow cells can change them into leukemia cells. Doctors are now learning how to use these changes to help them predict a person’s outlook and figure out whether they need more or less intense treatment. In the future, this information may be used to develop newer targeted treatments against acute myeloid leukemia (AML).

Finding “hidden” disease (minimal residual disease)

Progress has also been made in finding leukemia cells after treatment, when there are so few leukemia cells that they can’t be found by routine bone marrow tests. A test called PCR (polymerase chain reaction) can find even very small numbers of leukemia cells. This is helpful in seeing how well the chemo has destroyed the leukemia and whether it is likely that the cancer will come back (relapse).

Doctors are also trying to figure out what effect hidden disease has on a patient’s outlook, and how this might affect the need for more treatment.

Better chemo

Studies are going on to find the best combination of chemo drugs, with fewer side effects, and to figure out which patients will benefit the most from different types of treatment.

Sometimes chemo may not work very well because the leukemia cells become resistant to it. Researchers are now looking at ways to prevent or reverse this resistance by using other drugs along with chemo.

Stem cell transplants

Studies are also being done to improve the stem cell transplant process and to predict which patients are most likely to be helped by this treatment. Many studies are also trying to help figure exactly which type of transplants might be best.

Targeted therapies

Chemo drugs can help many people with AML, but these drugs don’t always cure the disease. New targeted drugs that attack some of the genetic changes seen in AML are now being developed.


The goal of immunotherapy is to boost the body’s immune system to help destroy cancer cells.

Monoclonal antibodies are man-made versions of immune system proteins (antibodies) that are designed to attach to certain targets, such as substances on the surface of leukemia cells. Some of these antibodies have radioactive chemicals or cell poisons attached to them so that when they are injected into the patient, they lock onto the cancer cells and kill them. One such antibody (gemtuzumab ozogamicin or Mylotarg®) was at one time was approved to treat AML in older patients. Although it was taken off the market because it didn’t seem very helpful, it is again showing promise in certain patients in clinical trials.

Vaccine therapy: A study of an experimental vaccine had promising results. For this vaccine, white blood cells (cells of the immune system) are removed from the patient's blood and exposed to a protein found on many AML cells. These cells are then given back to the patient into a vein (IV). In the body, the cells cause other immune system cells to attack the patient's leukemia.

CAR T-cell therapy: This is a promising new way to get the immune system to fight leukemia. For this technique, immune cells called T cells are removed from the patient’s blood and altered in the lab so they have specific substances (called chimeric antigen receptors, or CARs) that will help them attach to leukemia cells. The T cells are then grown in the lab and infused back into the patient’s blood, where they can now seek out the leukemia cells and attack them.

This technique has shown very promising results in early studies against some hard-to-treat types of lymphocytic leukemias. Although it’s not yet clear if it will work against AML, this is now being studied.

Last Medical Review: 04/21/2015
Last Revised: 02/22/2016