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Gene 'driver' of Waldenström's Macroglobulinemia in up to one-third of patients identified

  • Drug that targets the driver mutation proves effective in animal studies

    Steven Treon, MD, PhD

    In nearly one-third of patients with Waldenström's Macroglobulinemia, a specific genetic mutation switches on the disease, and a new drug that blocks the defective gene can arrest the disease in animal models, researchers at Dana-Farber Cancer Institute and allied institutions will report at the 2013 annual meeting of the American Society of Hematology (ASH). The finding may open the way to clinical trials of the drug in Waldenström's patients whose tumor cells carry the mutation.

    Waldenström's is a form of non-Hodgkin lymphoma diagnosed in 2,000 to 3,000 people in the United States each year. In 2011, researchers led by Steven Treon, MD, PhD, director of the Bing Center for Waldenström's Macroglobulinemia at Dana-Farber, and Zachary Hunter, showed that tumor cells in 90 percent of Waldenström's patients carry a specific gene mutation — the first time the disease had been traced to a particular genetic flaw. More recently, Treon reported 29 percent of a small group of Waldenström's patients had mutations in the gene CXCR4.

    In a study to be presented at the ASH meeting, Irene Ghobrial, MD, director of the Michele & Stephen Kirsch Laboratory at Dana-Farber, and Aldo Roccaro, MD, PhD, also of Dana-Farber, and their colleagues examined samples of Waldenström's tumor cells from a far larger group of patients — 250 in all — and found that CXCR4 was indeed mutated in about 30 percent of them.

Posted on December 09, 2013

  • Steven P. Treon, MD, PhD
  • Research
  • Waldenström’s Macroglobulinemia
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