Approximately 295,000 Americans have one of a rare group of blood cancers — polycythemia vera, essential thrombocythemia, or myelofibrosis — collectively known as myeloproliferative neoplasms (MPN), which are characterized by an increase in red blood cells, white blood cells, or platelets.
In polycythemia vera (PV), there are too many red blood cells made in the bone marrow. Essential thrombocythemia (ET) occurs when the bone marrow produces too many platelets. Both PV and ET may put the patient at risk for heart attack, stroke, or pulmonary embolism. People with primary myelofibrosis (PMF) have “fibrosis,” a buildup of scar tissue in the bone marrow, causing problems such as an enlarged spleen or liver.
On a mission
While MPNs are chronic and there are a few therapies available, there isn’t a cure. But one group — the MPN Research Foundation (MPNRF) — is on a mission to fund research towards discovering new treatments, improving patients’ quality of life, and, ultimately, finding a cure.
The research foundation, which has been working for 21 years to fund global innovative approaches for prevention and new therapies, has awarded over $16 million for MPN blood cancer research.
“MPNs, specifically new treatment options, have become a hot topic. The promising news is we now have four treatments available for some MPN patients. The hopeful news is also the several treatments in late-stage development that could become available in the next 18-24 months,” says Kapila Viges, CEO of MPN Research Foundation. “Doctors are eager to learn about and gain experience with these drugs to have more options to offer and help patients through their journey.”
Symptoms and diagnosis
Symptoms of MPNs, such as fatigue, bleeding or bruising, nights sweats, and itchy skin, often start months or years before diagnosis. However, symptoms are often overlooked since they may overlap with another diagnosis.
Diagnosis often occurs after abnormal bloodwork, such as high or low blood counts. MPNs are typically diagnosed in men and women who are over age of 50, with most being in their 60s. With proper treatment, many people with MPN live normal life spans. However, others may experience complications including the clots and abnormal bleeding.
“In terms of prognosis, some MPN patients may progress very quickly, in a matter of a few years,” says Richard Winneker, Ph.D., head of scientific strategies for MPNRF. “Yet many patients can go decades maintaining stable health, with the uncertainty of if or when they might get worse. It’s this concept of MPN disease progression that MPNRF is focused on to better understand it.”
Patient voices
MPNRF held an externally-led patient-focused drug development meeting with 135 in-person and web-based participants, which gave the MPN patient community an opportunity to educate Food and Drug Administration (FDA) representatives, biopharmaceutical companies, and academic researchers about the challenges of living with an MPN.
Out of that meeting, MPNRF released the “Voice of the Patient” report. For example, David A., a 64-year-old PV patient, says his disease progression was marked a decade after diagnosis with increasing fatigue, muscle pain, and lack of concentration. One of his worst symptoms was itching.
“It was nearly body-wide pain worse than any burn I’ve ever had,” he explains, concluding, “It was so bad I was unable to fix a glass of ice water, which I thought helped, or say more than three words.”
Another PV patient, Diane R., reports, “I’m very worried about having another TIA or stroke. To quote my neurologist, ‘It’s not if but when.’” MPNRF is committed to raising awareness among all stakeholders, including patients and providers. Their website is a comprehensive resource: http://www.mpnresearchfoundation.org/