Unprecedented therapy found effective for blood cancer patients with no treatment options

Unprecedented therapy found effective for blood cancer patients with no treatment options

Researchers have found a new type of therapy to be effective for patients with a particular type of bone marrow cancer that is resistant to several standard therapies.

This trial tested selinexor with dexamethasone, a combination that significantly knocked down the cancer in more than a quarter of patients, including two patients who went into complete remission.

Proteins and messenger RNAs play an important part of cancer cell growth, and selinexor has an unprecedented mechanism that blocks the export of protein and messenger RNAs from the nucleus of the cancer cell to the cytoplasm, causing the cancer cell to die.

This therapy caused at least a minimal response in almost 40 percent of patients who had multiple myeloma, a cancer of a type of white blood cell called a plasma cell.

Ajai Chari, Dan T. Vogl, Maria Gavriatopoulou, Ajay K. Nooka, Andrew J. Yee, Carol A. Huff, Philippe Moreau, David Dingli, Craig Cole, Sagar Lonial, Meletios Dimopoulos, A. Keith Stewart, Joshua Richter, Ravi Vij, Sascha Tuchman, Marc S. Raab, Katja C. Weisel, Michel Delforge, Robert F. Cornell, David Kaminetzky, James E. Hoffman, Luciano J. Costa, Terri L. Parker, Moshe Levy, Martin Schreder, Nathalie Meuleman, Laurent Frenzel, Mohamad Mohty, Sylvain Choquet, Gary Schiller, Raymond L. Comenzo, Monika Engelhardt, Thomas Illmer, Philip Vlummens, Chantal Doyen, Thierry Facon, Lionel Karlin, Aurore Perrot, Klaus Podar, Michael G. Kauffman, Sharon Shacham, Lingling Li, Shijie Tang, Carla Picklesimer, Jean-Richard Saint-Martin, Marsha Crochiere, Hua Chang, Samir Parekh, Yosef Landesman, Jatin Shah, Paul G. Richardson, Sundar Jagannath. Oral Selinexor–Dexamethasone for Triple-Class Refractory Multiple Myeloma. New England Journal of Medicine, 2019; 381 (8): 727 DOI: 10.1056/NEJMoa1903455