Patients who are diagnosed with myelofibrosis will experience constitutional symptoms, splenomegaly, and patients with higher risk disease are at an increased risk of transformation to acute myeloid leukemia (AML), and a shortened life expectancy. As an acquired clonal Philadelphia chromosome negative myeloproliferative neoplasm, myelofibrosis can present in a number of ways. Patients may be diagnosed with primary myelofibrosis or secondary myelofibrosis following progression of polycythemia vera (PV) to post-PV myelofibrosis, or essential thrombocythemia (ET) to post-ET myelofibrosis. Other than hematopoietic stem cell transplantation (HSCT), that is a potentially curative therapy, currently available therapies are unable to control all of the clinical manifestations of myelofibrosis. Our research is focusing on potential new treatment options, including combination strategies with ruxolitinib as the backbone treatment.

We are conducting three research studies to evaluate an investigational agent called navitoclax in patients with myelofibrosis. The primary objective of this program is to evaluate the effect of navitoclax (in most cases, in combination with ruxolitinib) on reducing spleen volume in patients with myelofibrosis. Patients may continue on the study treatment as long as they are receiving benefit. We need help from the local medical community to help us identify qualified study participants around the world.

Navitoclax is under clinical development and is not approved by regulatory health authorities. Safety and efficacy have not been established.