30 FACTS ABOUT MPN
MPNRF’s investments in global, cutting edge, multi-center MPN research have directly advanced knowledge about the causes of these rare, chronic blood cancers, leading to the development of more and better treatments and improved quality of life for people living with an MPN. Clinical trials are an integral part of this process.
Our commitment: funding the science that results in more options for people living with myeloproliferative neoplasms, the rare chronic blood cancers known as MPNs. Our investments in global, cutting edge, multi-center research have directly advanced knowledge about the causes of MPNs, while leading to the development of new treatments and improved quality of life for MPN patients.
Be a part of the impact. Make an investment toward MPN research.B
Polycythemia vera (PV) and essential thrombocythemia (ET) are often found during routine bloodwork. It’s common for patients to have no symptoms or mild symptoms for years. Want to know more about these rare, chronic blood cancers? Visit Understanding MPNs and the subpages on our website to learn more.
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 US Food and Drug Administration (FDA). Learn more here: Drug development & clinical trials. For a personalized list of MPN clinical trials that match your diagnosis click here: Trialjectory.
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 they stopped working. Read about MPNRF-funded research here: MPN Research Impact
Anemia is a major contributor to the hallmark fatigue associated with MPNs. Read about two recently reported clinical trials: how the need for phlebotomy was reduced or eliminated in polycythemia vera patients with rusfertide; and how momelotinib met the unique needs of myelofibrosis patients with enlarged spleen and anemia. 2022 ASCO Highlights
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. Print and share a potential list of trials with your MPN care team here: Trialjectory.
Some 120 medicines are currently in clinical trial or awaiting approval by the US FDA for blood cancers, including for the rare MPNs essential thrombocythemia, polycythemia vera, and myelofibrosis. Research and trials are gaining ground every year toward new, better treatments for MPN patients. Find out what clinical trials match your diagnosis and history here: MPN Clinical Trial Finder.
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.
Current MPNs research studies and clinical trials focus on symptom relief and disease progression by understanding and targeting of biomarkers. The aim is to lead to more personalized treatments. Read here about Identifying and Validating Actionable Biomarkers in MPN
More medicines are now approved for MPNs: ruxolitinib, fedratinib and pacritinib for myelofibrosis, ruxolitinib and ropeginterferon alfa-2b for polycythemia vera. These approvals took years and the commitment of valued patients who participated in their clinical trials.
There are more clinical trials every year for people living with ET, PV, or MF. Get a personalized list of trials that match your diagnosis and medical history, then share with the medical team that manages your MPN. Get started with the MPN Clinical Trial Finder
The safety and scientific validity of a clinical study are carefully considered by a study sponsor and investigators. A listed clinical study means it is being carefully evaluated by the health authorities. Along with the benefits of participating in a clinical trial, learn the potential risks. Speak to your MPN care team.
It’s a new day in MPNs – so many promising therapies, yet there is so much work to be done toward a cure. Biopharma is a partner in the fight. Thank you to our many sponsors.
An ongoing MPNRF-funded study is testing the feasibility of comparing stem cell transplantation with a non-transplant option in patients with high-risk myelofibrosis. What is unique is that participants choose the treatment they prefer, transplant or best available non-transplant treatment. Read about this work by Dr. Vikas Gupta and other recent MPN Challenge projects in the summer 2022 MPNRF Update.
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.” 64-yr. old MF patient. Ask your doctor what MPN trial might be a good match for you.
Your participation in a clinical trial helps advance new treatments for more patients like you. Learn more about what you need to consider and what current trials match your personal medical history and diagnosis with the MPN Clinical Trial Finder
MPNRF focuses on gaps in MPN research to better understand and treat ET, PV, and MF, investing more than $16 million over the past two decades. YOU can be a part of the impact by making an investment toward MPN research today.
Hematology/Oncology doctors can play an important role in helping patients access new MPN treatments through clinical trials. More patients will consider enrolling in a trial if they are asked. So go ahead. Ask the question. And consider hosting a clinical trial. You don’t need to be at an academic institution. Contact MPNRF for more info: email@example.com
MPN patients can experience very different symptoms. If you or a loved one have an MPN and you are not satisfied with the information or course of treatment provided to you, consider finding a physician who sees many people with MPNs. Find a specialist here.
Ruxolitinib was approved in 2011 for myelofibrosis and has provided symptom relief for many patients. Because it isn’t enough for everyone, many current clinical trials are testing the effectiveness of drug combinations. One example is a phase 1/2 study of selinexor, a drug previously approved for multiple myeloma and lymphoma which, when combined with ruxolitinib, is showing significant reduction in spleen size and a rapid reduction in total symptom score (TSS). Read details here.
Clinical trials are not ONLY for patients seen at academic medical centers. Community-based practitioners can either refer patients to a site where a trial is offered, or they can consider hosting a trial at their institution. Clinical trials can offer better treatment options for ET, PV, or MF. So, communication is critically important between clinicians and their patients about potential eligibility and participation in a trial.
Research projects funded by MPNRF have led to important advances for people living with MPNs. These include discovery of the CALR mutation, understanding more about interferon and disease progression, and identifying possible new treatments. Read more about our 21-year MPN IMPACT.
MPNRF looks at critical gaps in current MPN research that may move answers from the laboratory to the clinic, faster. The foundation plays a uniquely independent role in the MPN community, convening academia, industry, patients, advocates and regulatory agencies, ultimately to improve the lives of people living with MPNs. Hear what leading MPN specialists say about the IMPACT@21
MPN research investments in the lab lead to more MPN trials in the clinic, bringing more and better treatment options to people living with ET, PV, and MF. Read how you can invest in future MPN research through planned giving.
Because symptoms can vary significantly among MPN patients, it is important to track changes in symptoms and their severity between doctor visits. Keep current on the latest MPN research updates and speak to your doctor about how changes in your symptoms may call for a change in treatment.
Several research studies previously funded by the MPNRF have led to clinical trials. Making new potential therapies available for patients is our mission. Read about our research impact through MPNRF Challenge Awards.
The U.S. Food and Drug Administration takes into account the voice of patients. The MPNRF Voice of the Patient report features 300 MPN patients and is a resource for FDA and biopharma companies to keep the patient perspective top of mind at every stage of drug development, including clinical trials and drug approval. Read the report here.
If you are considering a clinical trial, you have the right to informed consent. FDA has guidelines for sponsors and doctors to ensure participants fully comprehend potential risks and benefits, and what it will entail. Get all of your questions answered before enrolling.
Current mpn clinical trials are testing new agents alone and in combination. Examples include: fedratinib, momelotinib, parsaclisib, ruxolitinib, navitoclax, pelabresib, and navtemadlin, to name just a few. Go here to see what trials you may qualify for: TrialFinder
Researchers are learning more about why interferons sometimes work or don’t work to improve MPN symptoms. This recently published research about how our cells “talk” to each other exemplifies how work in the lab and clinic can connect to bring forward new ideas about earlier and better treatment of MPNs.
MPN research investments in the lab lead to more MPN clinical trials, bringing more and potentially better treatment options to people with MPNs. You can invest in future MPN research through planned giving.