New trial PROMises hope for patients affected by myelofibrosis

A new clinical trial has been launched to offering a novel treatment option for patients with the blood cancer myelofibrosis (MF). 

The PROMise, launched by the Cure Leukaemia-funded Trials Acceleration Programme, will involve patients aged 16 or over at 15 NHS centres, including Oxford's Churchill Hospital, and its Chief Investigator is University of Oxford Professor of Haematology, Adam Mead.

PROMise, co-ordinated via the TAP hub at the Cancer Research UK Clinical Trials Unit (CRCTU) and sponsored by the University of Birmingham, will recruit MF patients over the next two years, providing them with the opportunity to take part in a trial that offers a promising alternative to the current standard of care.

Each year in the UK over 300 patients are diagnosed with MF, a condition associated with debilitating symptoms including extreme fatigue, pain, weakness and shortness of breath.  Between 10 and 20% of MF patients go on to develop acute myeloid leukaemia (AML) and consequently, a diagnosis of MF has a huge impact on both length and quality of life, with median survival from the time of diagnosis just two years for patients with high-risk disease.

The only curative therapy for MF is stem cell transplant; however, this is only suitable for a small minority of younger patients who do not have other conditions.

The current NHS standard of care for those unsuitable for stem cell transplant is treatment with ruxolitnib. However, many patients do not achieve an adequate response to this drug, and significant residual symptoms remain.

There is, therefore, an urgent need for an improvement in therapeutic approaches for MF patients.

Professor Mead said: "The PROMise study is a really exciting study that is introducing a new treatment called PLX2853, in combination with ruxolitininb, for patients with MF. The impact on patients for this combination of treatments, I hope, will be improvement of their symptoms, improvement of their quality of life, without causing them side effects.

"The funding from Cure Leukaemia is really important for the day-to-day running of this study as Cure Leukaemia are providing nursing support in each of the centres."

Up to 60 patients will take part in this phase 1 clinical trial. It aims to:

• establish the dose of PLX2853, administered in combination with ruxolitinib, that is safe and tolerable
• assess the efficacy of the combination of PLX2853 and ruxolitinib for reduction of symptoms associated with MF
• assess the safety of the combination of PLX2853 and ruxolitinib

CEO of Cure Leukaemia, James McLaughlin said: "Without Cure Leukaemia's funds, this study would not be currently recruiting patients and giving them access to potentially life-changing treatment. This highlights why this network is so vital and why funds raised for Cure Leukaemia make a direct impact on the lives of the 38,000 people diagnosed with a form of blood cancer in the UK each year."

Patients wishing to take part in this study should speak directly to their consultant.

Watch a video relating to this story, featuring Professor Mead.