Promising Treatments for Myeloproliferative Neoplasm

When businessman Robert Rosen was diagnosed with a Myeloproliferative Neoplasm (MPN) back in 1997, he discovered treatment options were quite limited for this rare and progressive type of blood cancer. In fact, few people had ever heard of MPN, which actually refers to three related disorders, Polycythemia Vera (PV), Essential Thrombocythemia (ET), and Primary Myelofibrosis (PMF).

These conditions affect blood counts and platelet levels, and can also lead to blood clots and an enlarged spleen, resulting in an array of debilitating symptoms. In many cases PV and ET will progress to Myelofibrosis, the most serious form of MPN for which the only cure is a bone marrow transplant.

Yet despite the severity of MPN, Rosen noticed that it wasn't on the research community's radar screen. In fact, he couldn't even find an accurate estimate of how many people were affected by MPN.

Raising Awareness (and Money) for Myeloproliferative Neoplasm Research

"I'm not a scientist by trade but I've always been interested in science," he says. "I was reading about the human genome project and I asked my doctor who was doing research on MPN and he told me very few people were. I said, 'Someone has to step up.'"

Rosen quickly put his corporate experience to work trying to change things. He pulled together a team to form the MPN Research Foundation in 1999. From there, things just seemed to flow. "We raised money, bought software, and awarded our first grant in about a year," he says. Now, more than a decade later, the foundation has awarded close to $9 million in grants for MPN research.

Developing New Treatment Approaches for Myeloproliferative Neoplasm

Although to date no cure has been found, the foundation has been able to help support the development of several new and exciting treatment options.

"Most of the treatments in development now are focused on inhibiting a genetic mutation that seems to be implicated in MPN in some way," Rosen says. In laymen's terms, what this means for patients is that the latest class of medicines, which are referred to as Janus Kinase 2 inhibitors, commonly called JAK2 (the name of the gene that's affected), can help relieve some of the swelling of the spleen and related symptoms and improve their quality of life.

Improving Longevity for People With Myeloproliferative Neoplasm

The next question for scientists to answer is whether the new class of drugs will improve longevity, Rosen says. So far, it looks like it may, but the jury is still out until more research is conducted over a longer time period. Currently only one treatment is approved for myelofibrosis, while several others are moving through the drug development pipeline. Hopes are running high that the future will bring more, and even better, treatments and outcomes for patients and their families.

Recognizing the Signs of Myeloproliferative Neoplasm

While this is all promising, Rosen points out people with MPN need to get a proper diagnosis and familiarize themselves with treatment options. All too often doctors don't recognize the signs of MPN, which can mimic a host of other conditions. Some of the common symptoms may include:

  • Fatigue
  • Easy bruising and bleeding
  • Susceptibility to infection
  • Joint pain
  • Fever
  • Feeling of fullness

If you or one of your family members is experiencing one or more of these symptoms, it's important to check with your doctor to rule out anything serious.

To learn more about MPN, you can visit the foundation's website at You can also make a tax-deductible donation to the foundation to help further efforts to some day find a cure for MPN.

Bob Rosen and Animesh Pardanani, MBBS, PhD, reviewed this article.


Sources: "Advancing Research, Empowering Patients." MPN Research Foundation. N.d. Web. 1 June 2013.