• Recent advances in research have resulted in new treatments for myeloproliferative neoplasms (MPNs).
• Some studies investigate treatments that may prove more effective than currently approved therapies.
• Talking to your doctor about clinical trials can help you determine whether joining a study is a good option for you.

Myeloproliferative neoplasms are a group of rare blood cancers that include primary myelofibrosis, polycythemia vera, and essential thrombocythemia. In MPNs, the bone marrow overproduces red blood cells, white blood cells, or platelets. This results in symptoms such as fatigue, pain, night sweats, itching, dizziness, weight loss, and trouble sleeping.

In most cases, MPNs are chronic conditions that can impact your quality of life. If your condition is considered low risk for progression, your doctor may recommend monitoring your condition instead of immediate treatment.

The main goals of treating MPNs are to control disease progression, prevent complications, and improve quality of life. In some cases, intensive therapies such as stem cell transplantation (also known as a bone marrow transplant) may offer a cure.

Clinical research over the past two decades has increased the number of therapies available for people with MPNs. Newer treatments often provide people living with MPNs more options for controlling their symptoms and improving their quality of life.

Recent Developments in MPN Research

Like all cancers, MPNs develop as a result of genetic mutations (permanent changes to the DNA) in cells. The discovery of specific mutations that play a role in the development of MPNs has had a major impact on how the condition is staged and treated.

One mutation that may lead to MPNs occurs in a gene called Janus kinase 2, more commonly called JAK2. The JAK2 gene is involved in producing a protein that helps cells grow and divide. Knowledge about the JAK2 gene mutations led directly to the development of new targeted treatments.

• The JAK1/JAK2 inhibitor ruxolitinib (Jakafi) is used to treat intermediate or high-risk myelofibrosis in adults. It’s also used to treat polycythemia vera in adults who can’t tolerate the chemotherapy drug hydroxyurea.
• The JAK2 inhibitor fedratinib (Inrebic) may be prescribed for adults with intermediate or high-risk myelofibrosis.

Knowing which genetic mutations are present in your cancer cells helps doctors predict your prognosis (how your MPN will progress over time) and whether you have a higher risk for advanced-stage disease.

Because knowing about genetic mutations can provide so much information, screening for the known MPN-related genetic mutations is now recommended whenever a case of MPN is suspected.

To improve the quality of life of individuals living with MPN, clinical research is focusing on the following goals:

• Identifying additional genetic mutations to target with new treatments
• Better understanding MPN symptoms to provide the best treatment options
• Developing more effective treatments for MPNs
• Finding out whether more people with MPNs — including those with lower-risk disease — may benefit from starting treatment

Why Consider Participating in a Clinical Study?

New treatments for MPNs are available because people diagnosed with MPNs participated in research. Research studies advance medical knowledge about MPNs and can contribute to improvements in the way MPNs are treated now and in the future.

Apart from contributing to medical knowledge and helping others with MPNs, there are many reasons why some people choose to participate in clinical trials.

New Treatments

Some clinical studies offer new treatments before they become widely available. Research is a necessary process before treatments can be approved by the U.S. Food and Drug Administration (FDA). As a participant in a clinical trial, you may have access to a newer treatment.

You are also contributing to research that may make effective treatments more widely available for individuals with MPNs in the future.

Alternative Treatment Considerations

Some people with blood cancers, like MPNs, leukemia, or lymphoma, consider participating in a clinical trial if their condition progresses while they’re taking standard treatments. For example, in rare cases, MPNs can develop into acute myeloid leukemia, which is called blast phase MPN. After this transition, an individual with MPN often has a worse prognosis. Some people facing blast phase MPN, or those with a high risk for progression to blast phase, may choose to join a clinical trial — depending on the risks and benefits.

Increasing Diversity

Wider participation in clinical trials can help increase diversity among the study population. When a greater variety of people participate in trials, it improves scientists’ and doctors’ understanding of how differences in age, gender, and racial and ethnic backgrounds affect an individual’s response to new potential treatments.

Concerns About Research Studies

It’s normal to have concerns about joining a clinical study and potentially receiving an investigative treatment. You may wonder how likely it is to benefit you or worry about the risks for side effects. Some worries may be related to common misunderstandings about how clinical trials work.

Read about the six things you should know about clinical trials for MPNs.

Factors to Consider Before Joining a Clinical Study

Before participating in research, there are many factors to consider. Whether a clinical study is right for you may depend on when you were diagnosed, whether you have already tried one or more treatments for your MPN, and your response to treatment.

Age, gender, overall health condition, and specific characteristics of your MPN may also be factors in your eligibility for a specific clinical trial. You may want to discuss options with your doctor and your family members before joining a clinical trial.

Talking to Your Doctor About Clinical Trials for MPNs

Clinical trials provide additional treatment options for people living with MPNs today and hope for new, more effective therapies in the future. If you’re curious about whether a clinical trial may be a good option for you, talking to your doctor is the best place to start.

Your doctor can help you identify clinical studies for which you may be eligible and talk you through the potential risks and benefits of participating. Talking to your doctor about clinical trial participation can help you start the conversation.

Talk With Others Who Understand

On myMPNteam, the social network for people with MPNs and their loved ones, members come together to ask questions, give advice, and share their stories with others who understand life with MPNs.

Are you living with MPN? Have you considered a clinical trial? Share your experience in the comments below, or start a conversation by posting on your Activities page.