A new clinical trial, which can be found here at clinicaltrials.gov, is slated to begin and is currently recruiting patients. In this trial, the investigational drug pacritinib will be tested as a therapy for myelofibrosis in patients who are preparing to undergo allogenic stem cell transplantation, which is the only way to cure the disease. The trial hopes to discover whether the drug will help improve the condition of patients prior to surgery.
Myelofibrosis is a rare cancer that affects the bone marrow. This cancer is characterized by the buildup of cancerous stem cells in the bone marrow and in other areas. This causes fibrosis to occur, which is the replacement of bone marrow with scar tissue. The disease can occur on its own or can develop from another one, such as polycythemia vera. Mutations of the JAK2, MPL, and CALR genes are associated with the development of myelofibrosis. Symptoms of this cancer include an enlarged spleen, anemia, pale skin, shortness of breath, easy bruising and bleeding, bone pain, fatigue, loss of appetite, weight loss, and increased vulnerability to infection. The cancer is treated with several medications, but stem cell transplant is the only way to cure the disease. To learn more about myelofibrosis, click here.
With a stem cell transplant being the only cure for myelofibrosis at this juncture, the goal of the study is to see if pacritinib can enhance a patient’s condition before surgery in a bid to minimize potential complications and provide a better outcome for the patient. This would help maximize the effectiveness of the entire operation. Ideally, the therapy could reduce spleen size and help reduce the chance of graft-versus-host disease, a dangerous complication of stem cell transplant that can be lethal if left unchecked.
In the past, the drug ruxolitinib was tested in a similar trial. Ruxolitinib and pacritinib a both in the class of drugs known as JAK2 inhibitors. In the earlier study with ruxolitinib, the outcome was not considered ideal because of the myelosuppressive effect of the drug, which is not suitable for all patients. The drug failed to improve the outcomes of transplant surgery. However, pacritinib does not have myelosuppressive effects, so hopefully it will be a more suitable candidate for this purpose.