New Therapies May Soon Appear for Myelofibrosis

According to a story from Medpage Today, the Society of Hematologic Oncology recently held its virtual meeting on September 9-12. During the event, Dr. Srdan Verstovsek of the MD Anderson Cancer Center at the University of Texas recently claimed that as many as four new therapies for myelofibrosis could be approved in the next three to five years. These treatments will utilize different mechanisms that will give physicians greater flexibility and versatility in treating the disease.

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.

New Approaches to Treatment

Inhibitors of the Janus kinase are the most common medications used to treat this cancer. Dr. Verstovsek says that the next generation of treatments will focus on alternative approaches:

“It is no longer about the JAK-STAT pathway. There are many other epigenetic abnormalities.”

His predictions about the potential appearance of new treatments relatively soon is rooted primarily in the number of phase 3 trials that are or will soon be evaluating potential therapies for myelofibrosis. Some of these therapies include selinexor (marketed as Xpovio), which is an inhibitor of nuclear export, and anti-PD-1 antibodies. There are also a wide variety of experimental therapies being investigated as well.

Other studies are investigating combination treatments that include JAK inhibitors alongside already approved cancer drugs or experimental drugs. Dr. Verstovsek says that some particular therapies that have shown great potential so far include:

  • Luspatercept
  • CPI-0610
  • Navitoclax
  • Imetelstat

 

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