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Targeted Therapy for Leukemia May Prove a Breakthrough

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WebMD Health News

April 4, 2001 -- Cindy Littrell of Salem, Ore., has leukemia -- chronic myeloid leukemia, or CML, to be exact. Diagnosed in February 1993 at age 45 by a blood test taken during a routine physical exam, she later endured a bone marrow transplant in an attempt to rid her body of the disease.

After the bone marrow transplant, she was hospitalized and treated with full-body radiation and chemotherapy.

"I was in a 'bubble room,'" she tells WebMD. "I could not have anyone come in the room unless they were totally sanitized and properly attired. I was alone. It was a very lonely feeling. I was in there for 30 days. You're exhausted from the chemotherapy and radiation. [The treatment] destroys your taste buds for a while, and you don't feel like eating, although they want you to eat. You're fed protein and that sort of thing intravenously. You try and exercise and move to build your strength up."

Littrell was back at work in a record six months, but five years later the leukemia was back, discovered again through a routine blood test. Standard treatments either didn't work or made her too sick. Her doctors considered giving her a second bone marrow transplant, even though they knew it would be even harder on her this time and less likely to work. While making plans for a second transplant, they found that Littrell had moved from the early or chronic stage of the disease to the more aggressive, accelerated phase called the blastic phase.

Until recently, a diagnosis of accelerated or acute CML was practically a death sentence, but for Littrell this was good news. It meant she was eligible to participate in a clinical trial of an experimental drug being conducted by Brian Druker, MD, a professor of medicine at Oregon Health Sciences University in Portland. Druker helped develop the drug called STI571.

STI571 represents the latest phase in cancer research and therapy where drugs are designed to target the specific molecular abnormalities that cause disease, and it is being hailed as a breakthrough for CML.

CML, a cancer of the blood cells, accounts for 15-20% of all leukemia cases. An estimated 5,000 new cases of CML are diagnosed in the U.S. every year. The disease is usually first detected in individuals aged 50-60 and is relatively mild in its early stages. Late-stage CML, however, is deadly.

Current treatments of CML are not adequate. Bone marrow transplantation, the most effective therapy, is feasible in only about one-third of patients and is so toxic that some people die from the procedure itself. Other drugs for CML include interferon and hydroxyurea, both of which make patients very ill and are not very effective in all cases.

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