According to a story from Newswise, scientists affiliated with the Huntsman Cancer Institute (HCI) of the University of Utah have found in recent lab studies that the experimental drug selinexor could play a potential role in the treatment of myelofibrosis, a rare disease. This remarkable discovery has lead to a new clinical trial being run by the institute that will test the drug as a treatment for the disease. The original study can be found here.
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.
Most patients will not survive beyond five years with the disease. Selinexor inhibits a process that myelofibrosis cells use to stay alive called nuclear cytoplasmic transport. The drug is currently not approved to treat any disease but is also being tested against certain types of blood cancer. Encouraging findings from a mouse model indicate improvements in symptoms comparable to the current standard of care for myelofibrosis, a drug called ruxolitinib. However, selinexor was also able to bring down the percentage of cancer cells, which ruxolitinib cannot do.
The selinexor clinical trial, which will test the drug in relapsed or refractory cases of myelofibrosis, is currently open at HCI and is projected to include 24 patients. Larger scale trials will commence if the early findings are encouraging.