This group of related blood cancers can occur in anyone at any age, so it is important to understand the symptoms and treatments of these diseases.
Myeloproliferative neoplasms (MPNs) are a closely related group of progressive blood cancers in which the bone marrow typically overproduces one of the mature blood elements. Other shared features include tendencies toward blood clotting and bleeding, organ enlargement, bone marrow scarring (fibrosis), and a possibility of transformation. Although MPNs can strike anyone at any age, most patients are afflicted in the sixth decade of life or later. Polycythemia vera and essential thrombocythemia are often found during routine bloodwork. It’s common for patients to have no symptoms or mild symptoms for years.
Stem cell transplants may offer a cure for some myelofibrosis sufferers, but there are no known cures for most MPNs. However, patients can experience few or no symptoms for extended periods of time, and many people who suffer from MPNs can enjoy longevity with proper monitoring and treatment. The identification of the JAK2 gene marker in 2005 and the CALR gene marker in 2013 have led to significant advances in the diagnosis, understanding of disease processes, and treatment of MPNs.
Bringing new treatments to MPN patients takes many years, from discovery in the lab to pre-clinical research that tests for safety and effectiveness. Successful pre-clinical therapeutic candidates move to human clinical trials and ultimately require approval by a regulatory agency, such as the U.S. Food and Drug Administration.
With research advancing so rapidly, many people living with an MPN find clinical trials offer a better treatment option for their disease symptoms, either after current therapies failed or were not fully effective. Talk to your doctor about MPN trials you might qualify for, then ask questions and consider your personal options.
MPN clinical trials have gone beyond JAK inhibitors and are now looking at new targets and therapeutic pathways that will expand the universe of options for patients. This is especially promising for patients who could not tolerate JAK inhibitors or stopped working.
Because MPNs are rare, the challenge of finding enough people with ET, PV, and MF who are willing and able to participate in a clinical trial can slow new drug approvals. “My life-altering symptoms have nearly disappeared since starting a drug combination in a clinical trial. The drugs I’m on would not be available to me otherwise.” a 64-year-old MF patient said. Ask your doctor what MPN trial might be a good match for you.
MPN research is rapidly accelerating our understanding of what causes these chronic diseases and how to treat them. While only a few MPN drug treatments are now approved, hundreds of clinical trials are underway across the globe, studying potential new and better treatment options. An MPN specialist can help you explore your eligibility to participate in one or more trials.