Leukemia

Microscopic image of infected blood

Leukemia is a devastating disease. Until recently, the treatment options could be as devastating as the diagnosis itself. But due to innovative research underway in the scientific community, treatments are becoming easier to tolerate and more effective. As a result, survival rates and quality of life have greatly improved for those diagnosed with this disease.

What is Leukemia?
Leukemia is a cancer of the blood and bone marrow. This cancer causes the formation of tissues with millions of abnormal white blood cells, which crowd out normal cells. The result leaves the body weakened, unable to fight infections.

Leukemia is either acute (spreads rapidly) or chronic (gets worse gradually). It affects two different types of blood cells: lymphoid or myeloid. The four major types of Leukemia are: Acute Lymphocytic, Chronic Lymphocytic, Acute Myelogenous, and Chronic Myelogenous.  The most common forms of leukemia in adults are Acute Myelogenous Leukemia (AML) with an estimated 12,330 new cases each year, and Chronic Lymphocytic Leukemia (CLL) with an estimated 14,990 new case each year.^[i]

What are the warning signs?
The early warning signs of leukemia include paleness, fatigue, weight loss, a tendency to bruise easily, repeated infections, and heavy or uncontrolled bleeding, such as nosebleeds. Any of these symptoms can appear suddenly in acute leukemia. Normally, a person with acute leukemia will feel sick due to these symptoms and see their doctor. The progress of chronic leukemia can be slow and produce no symptoms for years. Chronic leukemia is often detected by a doctor during a routine checkup, before the patient is aware of any symptoms.

Leukemia affects people of all ages
This year more than 43,050 people in the United States will be diagnosed with leukemia, and an estimated 21,840 people will die of the disease. Although most cases occur in adults over the age of 60, leukemia does not discriminate between the young and the old. It is the leading cause of cancer-related deaths among children and young adults under the age of 20, however the death rate for leukemia in children ages 0 to 14 in the United States decreased 76 percent from 1969 to 2007.^[ii]

New drugs offer new hope

Until recently, the only treatment options available for leukemia patients were invasive bone marrow transplants, debilitating radiation, chemotherapy, or a combination of all three. These therapies work in very different ways, but share a common goal of eliminating or achieving remission of leukemia cells and restoring normal blood cell production.

In the past decade, several important new drugs have been developed, and new uses have been discovered for existing drugs. These drugs have greatly improved cure rates and remission periods for patients with leukemia. Many are more effective and better tolerated than older treatments and have fewer adverse effects on normal tissues.

The five-year survival rate for patients suffering from leukemia has more than quadrupled in the past 49 years. In the 1960s, a patient with leukemia had a 14% chance of living five years. By 1999-2006, the overall relative survival rate had increased to 55.3%.^[iii]

Two examples of the drugs used today to treat leukemia are arsenic trioxide and all-trans retinoic acid (ATRA). These treatments kill leukemia cells, stop the leukemia cells from dividing, or help the leukemia cells mature into normal white blood cells.

Gene-based therapies, such as Gleevec® (imatinib mesylate), represent a major advance in treating leukemia. When Gleevec was approved by the Food and Drug Administration in 2001 for certain patients with Chronic Myelogenous Leukemia, Dr. Harmon Eyre of the American Cancer Society called it, "a huge breakthrough... a great drug, a great new discovery."  Read more about the discovery of Gleevec

Additional Resources

We invite you to explore Innovation.org to learn more about this disease, new medicines in development to treat it, and to read the real-life stories of patients battling with leukemia and other diseases.

Recent FDA Approvals

• Istodax^® - romidepsin (approved 11/5/2009)
  For the treatment of cutaneous T-cell lymphoma (CTCL).
• Arzerra™ - ofatumumab (approved 10/26/2009)
  For the treatment of refractory chronic lymphocytic leukemia.
• Folotyn™ – pralatrexate injection (approved 9/24/2009)
  For the treatment of relapsed or refractory peripheral T-cell lymphoma.
• Treanda^® - bendamustin hydrochloride (approved 3/20/2008)
  For treatment of chronic lymphocytic leukemia.
• Tasigna^® - nilotinib (approved 10/29/2007)
  For the treatment of chronic phase and accelerated phase Philadelphia chromosome-positive chronic myelogenous leukemia in adults resistant to or intolerant to prior therapy that included Gleevec.
• Sprycel™ - dasatinib (approved 6/29/2006)
  For the treatment of chronic myeloid leukemia in patients who are no longer responding to, or cannot tolerate, imatinib (Gleevec); treatment of Philadelphia chromosome-positive acute lymphoblastic leukemia in patients who are no longer responding to, or cannot tolerate, other therapies.
• Arranon^® - nelarabine (approved 10/28/2005)
  For the treatment of leukemia.

Patient Perspectives

Progress Against Cancer

Overall, life expectancy has increased and more patients are able to defeat their cancer. Read more about progress in the war on cancer.

Medicines in Development Database
Visit our Medicines in Development Database for more information about drugs currently in clinical trials or at FDA for review in breast cancer and other diseases.   

Clinical Trials Resources