Clinical trial offers a novel treatment for blood cancer patients

The Cure Leukaemia blood cancer charity have launched a new clinical trial called the PROMise, offering a novel treatment option for patients with the blood cancer myelofibrosis (MF).

The trial is funded by the charity’s Trials Acceleration Programme, and will involve patients aged 16 or over at 15 NHS centres; including Oxford's Churchill Hospital, University Hospital of Wales, and The Christie NHS FT. It 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.

Cure Leukaemia made a three-year commitment of £3m to fund the national Trials Acceleration Programme in January 2020. The network consists of specialist research nurses at blood cancer centres across the UK and a facilitatory Hub, based at the Centre for Clinical Haematology at the Queen Elizabeth Hospital in Birmingham.

Professor Adam Mead, Chief Investigator, at Oxford University, 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 improve their symptoms, and 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."

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, being just two years for patients with high-risk disease.

Stem cell transplant is the only curative therapy for MF; however, this is only suitable for a small minority of younger patients without any other conditions. Ruxolitnib treatment is the current NHS standard of care for those who aren’t eligible for stem cell transplant. However, many patients do not achieve an adequate response to this drug, and significant residual symptoms remain. There is, therefore, an urgent demand to improve the therapeutic approaches for MF patients.

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 a 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."

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

•Establish a safe and tolerable dose of PLX2853, administered in combination with ruxolitinib.

•Assess the efficacy of the combination of PLX2853 and ruxolitinib, to reduce the symptoms associated with MF.

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