Research into myelofibrosis

Myelofibrosis is a rare blood cancer. It causes scarring of the bone marrow which makes it more difficult to produce blood cells. 

Researchers around the world are looking at better ways to treat myelofibrosis and manage treatment side effects. Go to Cancer Research UK’s clinical trials database if you are looking for a trial for myelofibrosis in the UK. You need to talk to your specialist if there are any trials that you think you might be able to take part in.

Some of the trials on this page have now stopped recruiting people. It takes time before the results are available. This is because the trial team follow the patients for a period of time and collect and analyse the results. We have included this ongoing research to give examples of the type of research being carried out in myelofibrosis.

Research and clinical trials

All cancer treatments must be fully researched before they can be used for everyone. This is so we can be sure that:

  • they work
  • they work better than the treatments already available
  • they are safe

Myeloproliferative neoplasms

Myelofibrosis is one of a group of conditions called myeloproliferative neoplasms (MPN). They also include:

  • essential thrombocythaemia
  • polycythaemia vera

JAK2 gene

Researchers are looking at ways to improve the diagnosis and treatment of all MPNs.

Some of this research is suggesting that it may be better to group these cancers depending on whether they are:

  • JAK2 positive or
  • JAK2 negative

The JAK2 gene makes a protein that controls how many blood cells the stem cells make. A fault with your JAK2 gene means the stem cells can start producing blood cells when they're not meant to. Scientists hope that improving the diagnosis this way might help doctors choose targeted treatments in the future.

How do myeloproliferative neoplasms develop?

Researchers are looking to try to explain how myeloproliferative neoplasms develop. And to use this information to develop new treatments in the future.

Scientists think that MPNs might happen because proteins called tyrosine kinases act as growth factors Open a glossary item and tell cells to keep on dividing. Researchers are looking at white blood cells in samples from people taking part in a study. They are looking for tyrosine kinases and for any features or damage which may make them keep on dividing.

Research into symptoms of myelofibrosis

People with myelofibrosis can have a range of symptoms. These symptoms can affect different parts of their body. Researchers are looking at ways to collect information about the symptoms people have.

Research into treatment for myelofibrosis


Ruxolitinib is a cancer growth blocker Open a glossary item. It is already used to treat the symptoms of myelofibrosis. It's called a JAK2 inhibitor.

Researchers want to know if this drug can be used in other ways to treat myelofibrosis. 

Trial teams are also looking at whether ruxolitinib can be combined with other drugs to improve treatment. These other drugs include:

  • azacitidine - a chemotherapy drug

  • PLX 2853 a targeted drug

  • navitoclax

  • INCB057643 - a targeted drug

Other drugs

Scientists are looking at a targeted cancer drug called KRT-232. The study team are trying to find out if this drug helps people when JAK inhibitors, such as ruxolitinib, are no longer helping.

Researchers are also looking at:

  • fedratinib

  • momelotinib

  • givinostat

Treatment to prevent GvHD after a stem cell transplant 

A team of doctors is comparing standard treatment Open a glossary item with 2 newer combinations of treatment to prevent graft versus host disease (GvHD). 

Graft versus host disease (GvHD) is a possible complication of a stem cell transplant Open a glossary item from another person. GvHD happens when particular types of white blood cells (T cells) in the donated stem cells or bone marrow attack your own body cells. This is because the donated cells (the graft) see your body cells (the host) as foreign and attack them.

We know from research that some newer combinations of treatment may help to reduce the risk of GvHD. But they haven’t been compared to the standard treatments used in the UK. So the team is running this trial to find out more. 

Cancer Research UK Clinical Trials Database

You can find a clinical trial looking at myelofibrosis on our clinical trials database. Click on the ‘recruiting’, ‘closed’ and ‘results’ tabs to make sure you see all the trials.

  • Cancer Research UK Clinical Trials Database

    Accessed November 2023 

  • website

    Accessed November 2023

  • Myelofibrosis: Clinicopathologic Features, Prognosis, and Management

    J M O’Sullivan and C N Harrison

    Clinical Advances in Hematology and Oncology, 2018. Volume 16, Issue 2.

  • Targeted therapies for myeloproliferative neoplasms

    B Li, R K Rampal and Z Xiao

    Biomarker Research, 2019. Volume 7.

  • Ruxolitinib for treating disease-related splenomegaly or symptoms in adults with myelofibrosis

    National Institute for Health and Care Excellence, March 2016.

  • Addition of Navitoclax to Ongoing Ruxolitinib Therapy for Patients With Myelofibrosis With Progression or Suboptimal Response: Phase II Safety and Efficacy
    C Harrison and others 
    Journal of Clinical Oncology. 2022. Volume 40, Issue 15, Pages 1671-1680.

Last reviewed: 
20 Nov 2023
Next review due: 
20 Nov 2026

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