Clinical Trials & Research for MPN

If you are thinking about clinical trials, it is likely that you are not satisfied with the current treatment alternatives. The only curative treatment for MPN is the hematopoietic (blood) stem cell transplant, often referred to as SCT.  Because of its inherent risks, stem cell transplants are offered only to myelofibrosis patients with severe symptoms and appropriate score on the DIPSS+ risk assessment tool. There is more on this topic on the Stem Cell Transplant page.

There is a lot of progress in the understanding of the pathogenesis of MPN which leads to innovative treatments. This is good news because many of the standard treatments are old-line.  New treatments cannot come to market unless eligible patients are willing to participate in clinical trials.

There are several types of clinical treatment trials:

  • Study new drug compared to currently used drug or no drug
  • Study combinations of existing drug treatments
  • Study drugs approved to treat MF in the treatment of PV or ET

There are also Quality of Life Trials:

  • Study impact of complementary techniques (e.g., yoga, exercise) on MPN symptoms
  • Study patient health over time with standard treatment

1st Things First:     Learn the basics about Clinical Trials.  The Patient Advocate Foundation provides a good overview here as part of it’s “Guide to Clinical Trials” They also offer case workers to assist patients.

Each trial has a detailed protocol and no two are identical. Before you enroll in a clinical trial, think through these questions:

  1.   Why do I want to take part in this clinical trial?
  2.   What are my treatment options and what is likely to happen to my health if I do not do the trial?
  3.   What are the risks and potential benefits of participating in the trial?
  4.   What will the trial require of me — travel to a trial center, additional labs and doctor visits, costs for travel, costs for things not covered by   my insurance and the trial?
  5.   What if I have to drop out of the trial for another health issue but the trial drug is helping me?
  6.   How do they help patients who must drop out of a trial transition to standard care?
  7.   What expenses will the trial cover? What will my insurance cover?  What are the estimated gaps and are there resources to help?
  8.    If the treatment works for me, can I keep getting it at no cost after the study ends?
  9.    If the treatment harms me, what treatment will be provided?
  10.   Is long-term follow-up care part of the study?

MPN Research Foundation has a link to all trials for MPN on their website.

Medline Plus

   The NIH site MedlinePlus also has lots of great information:

    Every cancer treatment/research center lists the clinical trials in which they participate on their websites.

ALL clinical trials at:

   Check the sites periodically, as new studies are added throughout the year.

The NIH (National Institutes of Health) National Cancer Institute (NCI) lists NCI-supported trials at

Approved drugs and Investigational drugs

U.S. Food and Drug Administration (FDA)

The FDA is responsible for protecting the public health by assuring that foods (except for meat from livestock, poultry and some egg products which are regulated by the U.S. Department of Agriculture) are safe, wholesome, sanitary and properly labeled; ensuring that human and veterinary drugs, and vaccines and other biological products and medical devices intended for human use are safe and effective.

There are times we or a loved one need access to investigational drugs. These are meds that are promising but not yet approved by the FDA.  Commonly known as compassionate use of meds, the FDA provides a path to help acquire the drugs under its “Expanded access” program. Here are two helpful sites, one laying out the ground-rules, the other providing the necessary application information.

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