According to a story from Cancer Network, the treatment landscape for myelofibrosis, a cancer of the bone marrow, still has substantial unmet need. An important component of treatment are JAK inhibitors, but patients still face problematic symptoms, such as thrombocytopenia and anemia. Another consideration is variations in the disease course, as some patients have a more proliferative course of excess cells instead of insufficient cells. 

About Myelofibrosis

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.

A Game Changer Approach is Still Needed

There are some approaches that can improve anemia and there are therapies in clinical trials and other stages of development, such as newer JAK inhibitors and therapies targeting the TGFB pathway, that could further treat this symptom. The biggest challenge of myelofibrosis treatment is the limited options for altering the disease course or a cure. A stem cell transplant can save lives, but it carries massive risks.

There are treatments that can greatly improve quality of life, and that can make a big difference for a lot of patients; but returns tend to diminish over time. Some treatments in development, such as parsaclisib, target a pathway parallel to JAK-STAT and have had a decent showing in trials so far. Overall, there are still serious areas of unmet need in myelofibrosis, but the development stage for this disease is busy and there are several combination therapies that are showing promise. Hopefully, outcomes for this disease will improve in the near future with a new generation of medications.