Research into myelofibrosis

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.

Myeloproliferative neoplasms

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

  • essential thrombocythaemia
  • polycythaemia vera

JAK2 gene

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

Some of this research is suggesting that it may be better to group these cancers depending on whether they are JAK2 positive or 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 myeloproliferative neoplasms 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 that can affect different parts of their body. Researchers are looking at different ways to collect information about the symptoms people have.

Research into treatment for myelofibrosis

Ruxolitinib

Ruxolitinib is a cancer growth blocker Open a glossary itemwhich 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. 

For example, researchers are looking at whether ruxolitinib would benefit people with earlier stage disease. These people might not have any significant symptoms and would not normally start treatment. Researchers think that ruxolitinib could delay or lower the chances of early myelofibrosis becoming more severe in people at high risk.

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

  • BKM120 - this blocks a group of proteins called PI3K
  • azacitidine - a chemotherapy drug
  • sonidegib - a targeted cancer drug

Other drugs

Scientists are looking at a targeted cancer drug called KRT-232 for people with myelofibrosis. 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
  • luspatercept

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.

Last reviewed: 
29 Jul 2020
Next review due: 
29 Jul 2023
  • Cancer Research UK Clinical Trials Database

    Accessed June 2020

  • ClinicialTrials.gov website

    Accessed June 2020

  • 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

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

    National Institute for Health and Care Excellence, March 2016

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