“As it stands, the landscape for treating chronic myeloproliferative neoplasms is barren for individuals with these conditions. Currently, there is no curative treatment outside of stem cell transplantation, thus all current therapy are geared towards reducing symptoms burden,” says Peter Abdelmessieh, DO, Assistant Professor, Department of Bone Marrow Transplant and Cellular Therapies at Fox Chase Cancer Center. “Unlike acute leukemia, where a stem cell transplant is performed in setting of a complete response, if myelofibrosis patients become leukemic, transplants aren’t very effective, and patients rarely do well unless the disease is well controlled if not eradicated.”

For now, JAK2 inhibitors are the only frontline medications to alleviate symptoms. At first, patients tend to respond well to a JAK2 inhibitor such as ruxolitinib; their symptoms recede, and quality of life improves. But improvement is fleeting, and after a year or two, symptoms roar back, and a new JAK2 medication must be prescribed, and then again, until all options are depleted.

“This vicious cycle is terrible for patients and their doctors. New territory needed to be explored, so our team took on the challenge with Fox Chase researchers. Our bench scientists discovered that by adding a PARP inhibitor, a targeted drug for breast cancer, to JAK2 directed standard of care, they saw increased cell death of all mutant cell lines carrying myelofibrosis mutations. This has served as the basis for an Investigator-Initiated study which I am currently leading,” explains Abdelmessieh.

Fox Chase is the first to explore this combination of drugs and the only study in Philadelphia looking at myelofibrosis. “We want to acquire 24 patients over a two-to-three-year period, and from there, with hope of promising outcomes, we’ll take the trial further, perhaps to a durable remission, or to even better optimize patients so that they can better tolerate a stem cell transplant,” says Abdelmessieh.