Frequently Asked Questions

20. What new treatments for leukemia are being developed?

Researchers are studying various drugs, immunotherapies, stem cell transplantation techniques, and other types of treatments. Because leukemia is a complicated disease, researchers are also studying the effectiveness of using combinations of treatments. Following are a few examples of some areas of current research.

For example, patients receiving a stem cell transplant also undergo chemotherapy or radiation to prepare their body for the new stem cells. Also, researchers at the National Cancer Institute are looking at giving drugs to patients before and after the transplant that will stop the patient's immune system from attacking the transplanted cells.

The drug imatinib (Gleevec®) is important in the treatment of chronic myeloid leukemia. However, imatinib stops working in some people with leukemia because the cells become resistant. Fortunately, two drugs, dasatinib (Sprycel®) and nilotinib (Tasigna®), are being used to treat people who stop responding to imatinib. Both are approved by the FDA for use in patients. These drugs work against the same abnormal protein targeted by imatinib, but in different ways.

Gene therapy -- replacing, manipulating, or supplementing nonfunctional genes with healthy genes -- is being explored for treatment of leukemia. It is being studied as a way to stimulate a patient's immune system to kill leukemia cells and also to interfere with the production of proteins that cause cells to become cancerous.

A modified form of stem cell transplant, called a mini-transplant, has been developed. Compared with standard stem cell transplants, this technique requires less intensive radiation and chemotherapy before a patient is given donor cells. This approach has made stem cell transplants a treatment option for older adults and others who may not be able to tolerate high doses of radiation or chemotherapy.