This is REALLY big treatment news for patients – for many reasons:

• A new clinical trial option for lower-risk MDS patients
• Aim: to help the body create more red blood cells – reducing, or even avoiding need for transfusions – possibly more benefits
• A trial open to MDS patients truly representative of the condition, including typical age group of around 75 yrs old
• It uses known drugs, which have been used for years in other conditions – therefore deemed safe
• Oral formulation – much appreciated by patients who are already regularly pricked by needles for transfusions and blood tests
• There is no placebo (sugar pill) arm to this trial – meaning all patients will be receiving a treatment that could help (2 versions are being tested)
• It may turn low-risk MDS into a more manageable form of the condition, improving quality of life – a very important factor for older patients especially, in current absence of a safe enough curative option.

‘Previous research has shown that the drugs bezafibrate (a cholesterol-lowering drug) and medroxyprogesterone acetate (a type of contraception), “BaP” when put together, benefit people with AML who are unable to receive chemotherapy.

However, when given to people who were elderly and more frail, the drug combination could only be given at a low dose, otherwise it caused unwanted side effects. As the treatment had to be given at such a low dose, it was not as good as destroying cancer cells. In comparison, this drug combination has been shown to be well tolerated and successful at destroying cancer cells in children when they receive a higher dose. In a recent study, Professor Chris Bunce, Dr Farhat Khanim and their team looked at the effect of adding an additional drug to the BaP treatment and looked at what this did to AML cells in the lab. They found that adding valproic acid, a common drug used for people with epilepsy, could increase the anti-cancer effect of the other drugs, without having to increase the dose of BaP.

They now want to test this treatment, known as VBaP, in elderly people with AML to see how much benefit they get from the treatment.

The team also want to trial this drug combination in people with myelodysplastic syndrome (MDS), a blood cancer that can progress onto AML.

They think the treatment may improve effects of the disease for people with MDS and may also reduce the number of people who go on to develop AML.’

The MDS UK patient community provided PPI, Patient and Public Involvement work, for this trial.

In 2020, MDS UK, working with the research teams in Birmingham and Warwick, designed a PPI methodology and sequence of events, making use of 4 of our regional MDS patient groups across the country.

We presented the concept of this treatment option to the 4 groups, in a focus group setting, guided by the respective clinical expert in each area.

Each group was given a description of the trial design, the rationale, the compounds tested, the potential side-effects and the expected effect in MDS.

Patients were asked about understanding, acceptance, concerns, opinions of the whole concept.

They all had opportunities to ask any questions.

Each group and each person were then asked a set of specific questions, to also collate their opinions numerically.

The groups participating were representative of the typical MDS patient, in terms of age and experience of the condition.

Some patients had only recently joined our support group, whilst others had been members for a long time.

We concentrated on opinions and answers from the lower-risk MDS patients who would be potential candidates for this trial.

The comments, answers to specific questions and queries were then used by the research team to shape the trial protocol and start to construct the patient information materials.

The PIS (Patient Information Sheets) were then further revised by our expert patient advocates, to ensure readability, clarity, and inclusion of answers to all potential queries.

Several iterations were required to ensure a good end product, which satisfied everyone’s requirements, and resulted in much improved documents.

The hard work paid off, as the PPI team was commended by the Ethics board for the robust PPI work completed, and the fact that a PPI representative was present at the Ethics meeting, alongside a scientist and a clinician.

As a small charity, we are so very proud of our patient community and grateful to them for their time, dedication and efforts connected to this work, and PPI work in general.

As a charity, our mission is to not only support patients and caregivers, but also to educate and empower, giving them the option to become advocates for their condition.

As a rarer cancer, unknown to the general population, and poorly understood by GP’s, this is an absolute necessity to strengthen the voice of this group of people, where the median age at diagnosis is 75 years old.

This work, collaboration and efforts are ensuring true patient voice, proper attention to Quality of Life, and clearest, most accessible patient information, made with relevant patients, for patients.

Ultimately, such work may lead to faster access to the most helpful treatments.

As ever, we thank our scientific board of clinical experts for advice and guidance on this work.

A further, more detailed update on this PPI work will follow. Stay tuned!

Thank you to all involved in making this trial a reality for MDS patients