Myelofibrosis is a rare blood cancer that develops when a disruption occurs in blood stem cells, resulting in excessive scar tissue formation (fibrosis) in the bone marrow, where most blood cells are produced. To help compensate for abnormal blood counts, splenomegaly (enlargement of the spleen) occurs in most patients, which can cause pain below left side of the ribs and fullness. Other symptoms experienced by patients with myelofibrosis includes feeling tired or weak, bruising or bleeding easily and severe anemia, which is a lack of healthy red blood cells. If you are one of the thousands of people living with myelofibrosis in the U.S. today, you may be experiencing first-hand how these symptoms can be debilitating and greatly impact your quality of life. Myelofibrosis can occur in both men and women and is more likely to be diagnosed in people over age 50.
While there have been significant advances in the understanding and treatment of myelofibrosis, more remains to be done. Current treatments can help relieve symptoms or improve blood cell counts, but there are no treatments that address the underlying cause of disease. The good news is that research is underway to study this rare, difficult-to-treat blood cancer. To find out more about myelofibrosis research and potential eligibility to participate in ongoing studies, please visit www.myelofibrosisresearch.com/PAMR.
New treatments are investigated through clinical research studies (also called clinical trials) which are scientific studies that evaluate an investigational medication in a certain condition. All medications must be tested in clinical research studies before they can be approved and prescribed to patients.
Research is ongoing to evaluate a new, potential option called navitoclax. Navitoclax is being studied in clinical trials to evaluate its safety and efficacy in the treatment of myelofibrosis. It is an investigational, oral BCL-XL/BCL-2 inhibitor. The BCL-2 family of proteins are known regulators of the apoptosis (which means “cell death”) pathway. People with myelofibrosis often have a high expression of BCL-XL and BCL-2, and the BCL-2 family of proteins prevent apoptosis from occurring.
Navitoclax has not been approved by the U.S. Food and Drug Administration (FDA) or other regulatory health authorities. Safety and efficacy are under evaluation.
If you are over 18 years old with a diagnosis of myelofibrosis, you may be eligible to participate in one of these clinical trials.
How to get involved
Participating in a clinical trial is a personal decision. As with any new treatment under investigation, it may affect you differently from your current medication. You may also need more doctor visits which would necessitate more time and travel.
And finally, you could play a role in helping to advance the field of myelofibrosis research. Your participation in the study is voluntary – you can stop participating at any time for any reason. The investigational drug will be provided at no cost to you, and you will not be responsible for study procedures that are not part of the routine treatment for your disease. You may also be eligible for travel expense reimbursement.
To find out more about myelofibrosis research and potential eligibility for the studies, please visit www.myelofibrosisresearch.com/PAMR.
 Harrison et al. Health-related quality of life and symptoms in patients with myelofibrosis treated with ruxolitinib versus best available therapy. Br J Haematol. 2013 Jul;162(2):229-39
 Harrison C, Garcia J, Somervaille T, et al. Addition of Navitoclax to Ongoing Ruxolitinib Therapy for Patients With Myelofibrosis With Progression or Suboptimal Response: Phase II Safety and Efficacy. J Clin Oncol. 2022; JCO2102188.