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In the past decade alone, new drugs and new uses for existing drugs have greatly improved rates of cure or remission for people with blood cancer of all ages. Newer "targeted therapies" and "risk-adapted therapies" have resulted in higher overall response rates and decreased side effects. More than 50 drugs of different types are now being used singularly or in combination to treat blood cancer.

Blood cancer treatment often includes one or a combination of:

Goals of drug therapy

The goal of drug therapy is to eliminate cancer cells so that:

  • There's no sign of illness
  • Normal cells are restored (remission)

Cancer cells can grow too fast or fail to die quickly. Drug therapy can speed up cancer cell death. 

Types of drug therapies

Various types of anticancer medications kill cancer cells in different ways. The type of drug your doctor chooses to treat you can depend on your age, the type and stage of disease, your response to previous treatments and other factors.

Some blood cancers are treated effectively with a single drug. However, many conventional and investigational drug therapies combine drugs that attack cancer cells at different points in their growth cycles. This approach often makes therapy more effective and reduces the chance that the cancer cells will become resistant to a particular drug. As a result, more patients are achieving long-term remissions or cures.

Sometimes cancer cells may be resistant to the initial drugs used or can become resistant to the drugs after a period. In this case, your doctor may prescribe different drugs to target and kill the cancerous cells.

Note: A drug that is not FDA-approved for a specific diagnosis can sometimes be used as “off-label” treatment. “Off-label” prescribing is when a doctor gives a drug that is not FDA-approved to treat a patient’s condition, but the drug is FDA-approved for another condition. An off-label drug may be used if the doctor feels it will benefit the patient. This is a common practice. 

Learn more about these drug therapies below. For more information, visit our cancer drug listings. Open each section below to learn more.

Antimetabolites mimic the building blocks of DNA or RNA that cancer cells need to survive and grow. When the cancer cell uses an antimetabolite instead of the natural substances, it can't produce normal DNA or RNA and the cell dies.

Antimitotics damage cancer cells by blocking a process called mitosis (cell division), which prevents cancer cells from dividing and multiplying.

Antitumor antibiotics prevent cell division by either binding to DNA to prevent the cells from duplicating or inhibiting RNA synthesis.

Some enzymes can prevent cancer cells from surviving. 

Bisphosphonates are used to treat high levels of calcium in the blood caused by certain cancers, including myeloma. Bisphosphonates won't slow or stop the spread of cancer, but they can slow bone breakdown, increase bone thickness and reduce bone pain and fracture risk. 

Chemotherapy is the use of potent drugs or chemicals, often in combinations or intervals, to kill or damage cancer cells in the body. Chemotherapy drugs are often called anticancer agents. The drugs must be toxic enough to kill leukemic cells, which is why chemotherapy can be hard on your body—the drugs' toxicity can harm your healthy cells as well. However, successful chemotherapy depends on the fact that cancerous cells are more sensitive to the chemicals in the drug than normal cells are.

Learn more about chemotherapy.

DNA-damaging agents (antineoplastics) and alkylating agents react with DNA to change it chemically and keep it from allowing cell growth. 

DNA-repair enzyme inhibitors attack the cancer cell proteins (enzymes) that normally repair damage to DNA. DNA repair is a normal and vital process within the cell. Without this repair process, the cancer cell is much more susceptible to damage and cannot grow.

Histone deacetylase inhibitors attack cancer cells by targeting the proteins that support DNA in the cell nucleus. 

Certain hormones (corticosteroids) can kill lymphocytes. They're believed to work by blocking cell metabolism through their effect on specific genes. In high doses, these synthetic hormones—relatives of the natural hormone cortisol—can kill malignant lymphocytes. 

Hypomethylating (demethylating) agents interfere with cancer cell duplication by slowing or reversing hypermethylation. Methylation is a critical part of cell growth and replication. This process sometimes speeds up in cancer cells. 

Immunomodulators influence the immune system function by suppressing or stimulating immune response.

JAK inhibitors block the enzymes JAK1, JAK2, JAK3 and tyrosine kinase 2, which play a role in the cell-signaling process that leads to the inflammatory and immune responses seen in certain diseases. JAK inhibitors interrupt the signaling pathway. 

Monoclonal antibodies are laboratory-produced proteins that target specific antigens on the cancer cell's surface to interfere with the cell's function and destroy it. Some monoclonal antibodies are combined with a toxin or radioactive substance. 

PI3K (phospho inositide 3 kinases) inhibitors are a group of closely related kinase proteins. They act like switches in the cell – turning on other proteins. Switching on PI3Ks may make cells grow and multiply, or trigger the development of blood vessels, or help cells to move around. In some cancers PI3K is permanently switched on, which means that the cancer cells grow uncontrollably. Researchers are developing new treatments that block (inhibit) PI3K. They hope this will stop the cancer cells growing and make them die. 

Proteasome inhibitors are designed to limit the effects of a cell structure called a proteasome. When a proteasome doesn't function properly, the cell dies. Cancer cells may be more susceptible to the effects of proteasome inhibition than normal cells. 

Compounds that block the export of tumor suppressor proteins so that they stay in the nucleus and stop tumor growth, leading to cell death.

Tyrosine kinase inhibitors block the action of a specific, abnormal protein that gives cancer cells the signal to grow. 

For more information, visit our cancer drug listing.  

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Philadelphia-positive acute lymphoblastic leukemia (Ph+ ALL)

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acute lymphoblastic leukemia (ALL)

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acute lymphoblastic T-cell leukemia (ALL)

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T-cell acute lymphoblastic leukemia (T-ALL)

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Hodgkin lymphoma (HL)

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