According to a story from Targeted Oncology, findings from a recent phase 1 trial have discovered a new potential treatment for myelofibrosis. The study combined two different drugs: panobinostat (marketed as Farydak) and ruxolitinib (marketed as Jakafi). This dose-escalating study tested the drugs in patients with myelofibrosis derived from a variety of causes.
Myelofibrosis is considered a rare type of bone marrow cancer. The disease is characterized by the excessive accumulation of abnormal stem cells in the bone marrow which trigger a process called fibrosis, or scarring. Over time, the bone marrow is replaced with scar tissue. While the exact cause of myelofibrosis is not known, genetic mutations affecting the MPL, JAK2, and CALR genes are known risk factors. Symptoms of myelofibrosis include enlarged spleen, anemia, shortness of breath, easy bruising and bleeding, greater risk of infection, bone pain, gout, fatigue, weight and appetite loss, and increased blood cell volume. As a cancer that affects stem cells, stem cell transplant can cure the disease. However, this process carries many significant risks. Other forms of treatment are symptomatic and supportive and do not alter the course of myelofibrosis. There is a dire need for safer and more effective therapies for the disease. To learn more about myelofibrosis, click here.
About The Trial
The study included patients with primary myelofibrosis, post-essential thrombocythemia related myelofibrosis, and post-polycythemia vera related myelofibrosis. A total of 15 patients participated in the trial. 67 percent of them tested positive for JAK2V617F. Ultimately, 14 patients were deemed evaluable following treatment with 40 percent of patients seeing improvement in their condition. 53 percent saw their disease activity stabilize. 83 percent of patients who saw improvement had not been treated with ruxolitinib before and were positive for JAK2V617F.
As a dose escalation study, the primary goal was to determine the maximum tolerable dose for the combination treatment. Overall the combination appeared reasonably safe and provided some evidence of clinical activity. With these results, the treatment will continue to be tested in a proceeding phase II clinical trial. Hopefully, the combination of ruxolitinib and panobinostat will prove to be effective in future studies.