When a clinical trial showed the targeted drug ibrutinib to be highly effective in patients with a form of lymphoma called Waldenström macroglobulinemia, the results were so compelling that the U.S. Food and Drug Administration approved the drug for all patients with the disease, even though the trial included only patients previously treated with other agents.

The cover article for the first issue of The Torch for the new year is by Dr. Shayna Sarosiek, of the Bing Center for Waldenstrom's Macroglobulinemia, Dana-Farber Cancer Institute. She discusses amyloidosis, its diagnosis, symptoms, and treatments. While it’s a relatively rare complication of a rare disease, it is prudent for us to be aware of it.

Steven P. Treon, MD, MA, PhD, Professor of Medicine, Harvard Medical School, Boston, Massachusetts, discusses preliminary clinical data from a phase 1b study of mavorixafor in combination with ibrutinib for patients with Waldenström macroglobulinemia with MYDD88 and CXCR4 mutations. These data were presented at the 2021 American Society of Hematology (ASH) Annual Meeting.

Dr. Jorge J. Castillo, lead author of the study and clinical director of the Waldenström Macroglobulinemia Program at the Dana-Farber Cancer Institute in Boston, explained to CURE® that BCL2 inhibitors like Venclexta are approved by the Food and Drug Administration for other cancers such as chronic lymphocytic leukemia and acute myeloid leukemia; however, this is the first clinical trial evaluating the drug in patients with WM. He further explained that WM has malignant BCL2 cells that “immortalize” themselves, so “they are not dying when they are supposed to.”

The International Waldenstrom’s Macroglobulinemia Forum (IWMF) recently held their annual educational forum. Jorge Castillo, MD, Clinical Director of the Bing Center for Waldenstrom Macroglobulinemia, reports on some of the important updates from the forum. Dr. Castillo discusses how bone marrow biopsies should be evaluated and the importance of genetic profiling testing. He also explains the use of BTK inhibitors and the status of certain clinical trials, including those investigating new Bcl-2 inhibitors.

In November 2021, Dr. Jorge Castillo of Dana-Farber Cancer Institute (Boston, MA, US) provided a Bing-Neel update for the IWMF International Bing-Neel Specialty Topic Support Sessions.

A drug that strips cancer cells of their quest for immortality represents an effective new option for patients with Waldenström macroglobulinemia, including those not helped by the only class of drugs currently approved to treat the disease, a clinical trial by Dana-Farber Cancer Institute investigators demonstrates.

Jorge Castillo, MD - The latest information available about potential new treatments for WM, as well as the necessity for patient participation in clinical trials to help move the pipeline along for more novel therapies.

Panelists: Drs. Stephen Ansell, Jorge Castillo, Mary McMaster, Andrew Branagan, Christian Buske, and Steven Treon. A panel of WM experts will answer, and sometimes debate, questions about Waldenstrom macroglobulinemia from the IWMF 2021 Ed Forum audience.

Dr. Treon will review the NCCN guidelines. NCCN is a not-for-profit alliance of 31 leading cancer centers devoted to patient care, research, and education. The NCCN is dedicated to improving and facilitating quality, effective, efficient, and accessible cancer care so patients can live better lives.