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Gene Discovery May Offer Breakthrough for Rare Leukemia
Date:5/9/2013

By Barbara Bronson Gray
HealthDay Reporter

THURSDAY, May 9 (HealthDay News) -- In the war against cancer, it looks like matchmaking -- between genes and drugs -- could be an important tool, according to new research into the genetic underpinnings of two rare forms of leukemia.

By matching a patient's genetic mutation responsible for a rare, rapidly progressing form of leukemia with a drug that specifically targets the problem the mutation creates, researchers report that one patient is experiencing fast, marked improvement.

The new findings shed light on how many forms of cancer may be tackled in the near future. Scientists are discovering how to differentiate between mutations that are driving the proliferation of cancer cells and those that are merely passengers in the process.

"If your car breaks down, you have to open up the hood to see what part has broken," said study author Jeffrey Tyner, an assistant professor at the Knight Cancer Institute at Oregon Health & Science University. "Here we have to open up the tumor cells to see what part is broken to understand what to do."

After first identifying the genetic drivers of a specific type of cancer, scientists then match those mutant genes to drugs that will specifically target them.

According to Julia Maxson, study first author and a postdoctoral fellow at the Knight Cancer Institute, "The move in the field is to take the individual broken genes and say which of these really promote cell growth. Then, you can find the right drugs."

The research, published May 9 in the New England Journal of Medicine, identified "driver" gene mutations related to two rare forms of blood cancers: chronic neutrophilic leukemia (CNL) and chronic myeloid leukemia (CML). The scientists identified particular mutations in the gene responsible for what is called "colony-stimulating factor 3," which signals kinases, a type of cell enzyme.

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