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What to Know About Targeted Therapies for Leukemia

Targeted therapy is a newer type of treatment for some types of leukemia. It's designed to attack a certain change in a cancer cell. This is a change that makes the cancer grow, divide, repair, or "talk" with other cells. These drugs destroy or slow the growth of cancer cells. But they avoid most normal, healthy cells. That means this treatment tends to cause fewer and less severe side effects than other kinds of treatment. These three main types of targeted therapy can treat leukemia:

  • Monoclonal antibody therapy

  • Small molecule targeted therapy

  • All-trans retinoic acid

Each is described below.

Monoclonal antibody therapy

This treatment targets molecules on the outside of certain leukemia cells. It uses lab-created antibodies that are similar to those your immune system makes. The antibodies attach to specific substances on cancer cells. Then they kill the cancer cells or block their growth.

Small molecule targeted therapy

Some types of targeted therapies use small molecules to attach to abnormal proteins on cancer cells. This kind of treatment is used for chronic myelogenous leukemia (CML). People with CML have a faulty chromosome called the Philadelphia chromosome. The problem is that two genes have fused. For some reason, this makes the leukemia cells grow. Compounds used to treat CML attack the cells that have this "bad" chromosome.

All-trans retinoic acid (ATRA)

ATRA can kill some leukemia cells or stop the cells from dividing. It can also help leukemia cells mature into white blood cells. ATRA is used for a rare type of acute myelogenous leukemia (AML) called acute promyelocytic leukemia (APL).

How you take targeted therapy for leukemia

How you get targeted therapy depends on the kind you get:

  • Monoclonal antibody therapy. You get monoclonal antibody therapy by intravenous (IV) infusion. You won't need to stay in a hospital overnight. How long each treatment lasts depends on the dose you get. It also depends on whether you have any reactions to the treatment. It may take about two hours.

  • Small molecules. You will likely take small molecules as a pill by mouth once or twice a day. If you start taking the drug early in the course of the disease, you may take it for many years.

  • ATRA.  You may take ATRA during more than one phase of treatment. It may be during your first treatment or later for a long period of time. It's usually combined with other chemotherapy drugs to put your leukemia into remission. You take it in a pill form. 

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