Dr. Treon on Breakthroughs in Waldenström's Macroglobulinemia

Steven Treon, MD, PhD, Director, Bing Center for Waldenström's Macroglobulinemia and physician at Dana-Farber Cancer Institute, discusses the game changing discoveries in Waldenström’s Macroglobulinemia (WM) that led to the approval of ibrutinib for treatment of the disease.

Until recently, researchers did not understand the genetic basis of WM and had to rely on therapies designed to treat lymphoma and myeloma.

Three years ago, researched discovered that a mutation in the MYD88 gene was present in over 90% of the patients with WM. Researchers were then able to identify the important signaling pathways that are triggered by this mutation.

The discovery of the CR4 mutation, which is present in about one-third of patients with WM, provided further understanding the disease. These two breakthroughs paved the way for the approval of ibrutinib, which Treon says is a ‘game changer’ for the disease.

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