How can targeted therapy treat chronic myeloid leukemia?

Dr. Mehmet Oz, MD
Cardiology (Cardiovascular Disease)

A well-known molecular target was developed for chronic myeloid leukemia (CML), a rare blood cancer. It was the first cancer to have a drug target intentionally designed to pull the plug on a cancer-fueling enzyme that has great growth-signaling powers. CML is a type of blood cancer caused by too many inefficient white blood cells in the bone marrow and blood, a potentially fatal scenario. But these badly behaving white blood cells have some unique molecular characteristics, making them ideal for targeted therapy.

Somehow, on CML-causing white blood cells, a region on chromosome 22 swaps with a region on chromosome 9, and this new arrangement produces the abnormal chromosome called the Philadelphia chromosome (named for the city where the discoverers lived). This abnormality is the key to the disease. Unfortunately the mutated Philadelphia chromosome makes an enzyme that keeps a cell’s divide signal in the "always on" position. And when the cells containing the Philadelphia chromosome flourish, they crowd out healthy cells, and the incompetent white blood cells grow unchecked.

Since almost all people with CML are Philadelphia positive (Ph+), Dr. Brian Druker, an oncologist at Oregon Health and Sciences University, proposed developing a drug to disable this enzyme. As a result many people with Ph+ CML, who once had few good treatment options, now have a chance at a disease-free life.

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