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Top 5 Wellness Tips

Photo by Brett Jordan on Unsplash  

Just a reminder from us here at MDS UK, to get in tune with a few tips to increase and maintain a balanced and healthy lifestyle. Remember to tailor solutions to your needs and seek medical advice from your CNS/GP to fit your individual requirements. 


Most people have their favourite dish which they could easily have a few times a week, whether it’s a very green salad or a super cheesy lasagne, the key is balance. Remember to limit processed and sugary food and drinks.  

Keep fruit, nuts, and seeds as part of your intake, as well as whole grains, healthy fats, vegetables and water. It’s also important be mindful of portion sizes. 

Watch out! During the colder months, we see a variety of new hot drinks and snacks brought to the menus of our favourite places. As amazing as a “Sweet Caramel Crunch Cake and Vanilla, Hazelnut and Chocolate Wonder Latte” sounds, be mindful of the sugar in the powders and syrups – they can be sneaky! Try to opt for more herbal teas than sugar- filled temptations.  

Photo by Taylor Kiser on Unsplash  


We’re not saying to run uphill every day, but it’s important to get moving! To support a healthy brain and improve bone and muscle strength, incorporate some movement into your daily routine. This can include walking, stretching, swimming, cycling, or badminton. It’s key to keep up with having good circulation, coordination, concentration, improved mood, and reflexes. 

Photo by Lucas van Oort on Unsplash  


Not getting enough sleep? Make it a priority to adjust your routine before bedtime. Although it is tempting and very easy to end up watching 2 seasons back-to-back, when you’re done, it could be 4am. Yikes!  

Consider setting a wind-down alarm to remind you to start getting ready for bed. Let’s say you want to be in bed for 9pm, try to start your night routine at 7:30/8pm to give yourself enough time. During this time, instead of watching television, maybe read a few pages of a book and have tea instead of sugary snacks. 

Sleep helps with improving your memory, weight management, creativity, concentration, and the feeling of being energised.  

Quality of sleep is important too, not just the eight hours. For example, drinking too many liquids before bed could have you heading to the bathroom multiple times throughout the night! 

Photo by Rehina Sultanova on Unsplash  


Seeing as the body is made up of approximately 60% water, it makes sense that we make sure to get enough of it. You don’t need to be severely dehydrated in order to feel the effects. 

Constipation, brain health and levels of energy are all affected by water, try to have one litre of water a day – but avoid having a bulk of water in the evening to save you from the midnight trips to the bathroom! 

As the festive season approaches there may be more booze available at dinner than usual, but still remember that you need adequate water. 

Photo by Kobu Agency on Unsplash  


This is a great way to relax at any time of day, meet new people and develop a skill. Hobbies can help you to avoid the feeling of being bored or engaging in habits that don’t support you having healthy, balanced life.  

You can search for clubs and groups in your local area to meet others with similar interests, such as chess or painting classes. You could try puzzles, model building collecting items, dance class or hiking adventures.

Photo by Rifqi Ali Ridho on Unsplash  

UK MDS Forum Education Day 2021

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UK MDS Forum Education Day 2021

This report is written by Chris Dugmore and Claudia Richards who attended the UK MDS Forum event virtually.

Having attended in person in previous years, and the event being cancelled last year, the offer of a Covid-secure attendance was very appealing! We were able to put questions to the presenters via a Q&A Chat facility. We both enjoyed the day and were impressed by the professional presentation of this hybrid event.

As ever, we left with our heads full of acronyms and bunches of letters and numbers representing the vast and ever-growing array of genetic mutations which are now known to have huge significance for people with MDS. We hope we have managed to capture the key themes from the day.

The notes from the Education Day, which can be downloaded here have been checked for accuracy by the individual presenters.

We hope you will find them both interesting and accessible.

Repair MDS: A new approach to treat lower-risk MDS patients

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Repair-MDS: a new clinical trial for lower risk MDS patients

Called REPAIR-MDS (Repurposed drugs to improve haematological responses in Myelodysplastic Syndrome), the trial will test a number of treatments designed to improve the formation of healthy blood cells, reducing or reversing life-limiting deficiencies in red blood cells.

The project is a national effort by the UK MDS research community and will recruit patients from across the UK.

The trial was developed by the National Cancer Research Institute (NCRI) MDS clinical studies sub-group and has arisen from research carried out in the School of Biosciences and the School of Medical and Dental Sciences at the University of Birmingham led by Professor Chris Bunce. It will be carried out in collaboration with Queen Elizabeth Hospital Birmingham.

The funding of REPAIR-MDS by Blood Cancer UK has been made possible by a donation made in open competition to the charity from the Jon Moulton Charity Trust.

Comments by MDS UK:

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.

The details of the trial and the compounds tested (courtesy of Prof Bunce and BCUK):

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 Patient and Public Involvement (PPI) Work - led by MDS UK

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

MDS Patients

The REPAIR-MDS trial will start recruitment soon!

We will launch a formal recruitment call soon, with all details of the hospitals enrolling patients, and the exact inclusion and exclusion criteria

You will have to speak to your haematologist to be enrolled

Keep an eye on the website and your emails for further details

A new therapy in early stages of development could help MDS patients to control iron levels

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Thalassemia, Myelodysplastic Syndrome (MDS), and Iron Overload

Thalassemia and MDS are both rare diseases that prevent a person from producing enough healthy red blood cells. Low levels of healthy red blood cells, known as anemia, result in less oxygen being delivered to different parts of the body. This can cause symptoms such as excessive tiredness and weakness. It can also lead to other serious health problems, such as heart disease.

People living with thalassemia or MDS can also store too much iron in their bodies, leading to a phenomenon called ‘iron overload’, which damages organs such as the heart and liver. Both conditions are typically treated with regular blood transfusions, which add to the problem of iron overload.

Iron chelation therapy removes excess iron from the body using special medicines. While it helps reduce the amount of iron in the blood for people with thalassemia or MDS, it does not treat the underlying cause of the condition or stop it from progressing. There is, therefore, a need for therapies that directly address the biological drivers of disease.

About SLN124 and the GEMINI II study

SLN124 is a gene ‘silencing’ therapy – one that is designed to temporarily block a specific gene’s message that would otherwise trigger an unwanted effect.

In this case, SLN124 aims to temporarily ‘silence’ TMPRSS6, a gene that prevents the liver from producing a particular hormone that controls iron levels in the body – hepcidin. As hepcidin increases, it is hoped that iron levels in the blood will decrease, which could in turn allow more healthy red blood cells to be produced, thereby improving anemia.

In preclinical studies, SLN124 has shown positive effects on improving levels of red blood cells and reducing harmful iron levels.
SLN124 is now being studied in the GEMINI clinical trial program. GEMINI II is a phase 1 study to investigate the effects of SLN124 in people with thalassemia or myelodysplastic syndrome (MDS), whose bodies produce fewer healthy red blood cells than normal and who can store too much iron in their bodies.

Silence Therapeutics is developing a new generation of medicines by harnessing the body's natural mechanism of RNA interference, or RNAi, to inhibit the expression of specific target genes thought to play a role in the pathology of diseases with significant unmet need.

Silence Therapeutics has announced positive data from GEMINI Phase 1 Study of SLN124 in healthy volunteers

  • Data showed SLN124, an siRNA which targets TMPRSS6, was safe and effective in reducing plasma iron levels and had a long duration of action.
  • Data support ongoing phase 1 study of SLN124 in patients with thalassemia and myelodysplastic syndrome (MDS)
  • First clinical data from Silence’s proprietary mRNAi GOLD™ platform
  • Two more clinical data readouts anticipated this year

SLN124, an siRNA which targets TMPRSS6, is in development for the treatment of iron-loading anemia conditions, thalassemia and myelodysplastic syndrome (MDS).

The GEMINI phase 1, randomized, double-blind, placebo-controlled, single-ascending dose study evaluated the safety and tolerability of SLN124 (1.0, 3.0 and 4.5 mg/kg doses) in 24 healthy volunteers (18 active and 6 placebo). Pharmacokinetic parameters and pharmacodynamic biomarkers of iron metabolism were also measured to assess reduction in iron.

Initial data from the study showed all doses of SLN124 were generally well-tolerated with no serious or severe treatment emergent adverse events (TEAEs) or TEAEs leading to withdrawal. TEAEs did not appear to be dose dependent and the majority were mild, including transient injection site reactions which resolved without intervention.

Notably, up to an approximate four-fold increase in average hepcidin and 50% reduction in plasma iron levels were also observed after a single dose of SLN124. Effects on hepcidin and iron appear to be dose dependent and were still observed at the end of the 8-week study at all dose levels, indicating a sustained and long duration of action.

These clinical data support preclinical findings which demonstrated SLN124 effectively improved red blood cell production and reduced anemia by increasing levels of hepcidin – a key natural regulator of iron balance and distribution in the body.

Silence Therapeutics expects to measure red blood cell production and effects on anemia in the ongoing GEMINI II phase 1 study of SLN124 in people with thalassemia and MDS, who unlike healthy volunteers have significantly elevated iron levels.

Mark Rothera, President and CEO of Silence Therapeutics, said:

“These data represent the first clinical data from our mRNAi GOLD™ platform and underscore the promising potential for our technology to deliver precision medicines. We look forward to further data in patients anticipated from both of our wholly owned clinical programs later this year – the GEMINI II study of SLN124 for iron-loading anemia conditions and the APOLLO study of SLN360 for cardiovascular disease due to high lipoprotein(a).”

Giles Campion, M.D., EVP, Chief Medical Officer and Head of Research & Development of Silence Therapeutics, said:

“Today’s results confirm the strong preclinical profile of SLN124 in humans – we observed excellent safety, robust gene knockdown expressed by up to an approximate four-fold increase in average hepcidin along with a 50% reduction in serum iron levels and a durable effect which lasted throughout the study. We are encouraged by these data in healthy volunteers and the opportunity for SLN124 to potentially address iron-loading anemia conditions such as thalassemia and MDS.”

John Porter, M.D., Prof. and Consultant Haematologist, University of College London Hospitals, says

“Despite advances in our understanding of thalassemia and MDS, there are no existing treatments that specifically target the underlying mechanisms of these conditions as a way to improve the degree of anemia. There is a major unmet need for a therapy that can provide safe and continuous control of iron balance and distribution as a way to improve the efficiency of red cell production. I’m encouraged by data from the SLN124 study in healthy volunteers and look forward to further clinical testing.”

What is the latest on MDS UK funded research?

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First update on our PhD research project

MDS UK has great pleasure to provide the first update on the PhD research post we started funding in January 2020.

This post was made possible, thanks to legacies and donations received by our charity.

This update introduces the PhD student, William Walker. A team of experts at Queen’s University Belfast is providing experienced support and guidance.

In MDS patients some blood cells DNA is not repaired properly

Dr Kienan Savage, Senior Lecturer at School of Medicine, Dentistry and Biomedical Sciences at the Queen's University of Belfast, Centre for Cancer Research, explains the science behind the project:

“Some patients with myelodysplastic syndrome (MDS) can go on to develop the more aggressive acute myeloid leukaemia (AML). These diseases occur in the older population and have poor outcome mainly due to low level of tolerance of intensive chemotherapy.

In order to develop MDS, and indeed for this to progress to AML, some of a patient’s blood cells must acquire damage to their DNA that is not repaired properly, resulting in mutations that allow these cells to transform into cancer cells.

We know that in some cancers, particularly solid tumours like breast and ovarian tumours, the cancers develop due to a defect in their ability to repair damaged DNA, thus allowing them to accumulate more cancer initiating mutations.

Intriguingly this DNA repair defect often makes these cancers more susceptible to specific types of chemotherapies and newer targeted therapies, with reduced side-effects."

A DNA repair defect often makes a specific cancer more susceptible to certain chemotherapies

We aim to examine the ability to treat patients with DNA repair deficient MDS with drugs with more limited side effects

"By adapting a test designed to identify DNA repair deficient breast cancers, we have found that a large proportion of MDS patients, particularly those that progress to AML, have similar DNA repair deficiencies.

This project aims to use a combination of laboratory experiments, computational biology and clinical samples from patients to examine the ability to therapeutically target patients with DNA repair deficient MDS with specific drugs with more limited side effects than those currently used and therefore allow us to effectively treat more patients.

We are gaining a greater understanding into the mechanism causing MDS

William Walker said “I would first like to express my sincere gratitude to MDS UK for their donation. I would also like to show my greatest appreciation to the families whom have contributed to this funding.

We have entered a new era of MDS research thanks to advancements in genomics and many other computational technologies - this allows us to gain further understanding into the most intricate mechanisms causing MDS. Therefore, this new technology accelerates our progress and guides us in developing more effective and more specific MDS therapies.

I am incredibly excited to embark on this PhD programme, as I will be building on the current success at Queen’s University, Belfast. I feel very privileged to have joined such a strong team of world-leading MDS researchers and I believe this project will produce very promising insights into the targeting of DNA repair deficiencies in MDS. It’s a pleasure to wake up every day and know that I will be contributing to MDS research on behalf of MDS UK.”

MDS UK note: This piece of research now comes at a difficult time, due the inevitable drop in donations from Covid-19. The funds for the PhD have been ear-marked of course. However, without additional funds, our charity will struggle to keep up the full range of services to patients.
A further piece of research is also completely out of question, despite an interesting query about a complex sub-type of MDS.
We need to ensure the future of the charity first of all, before embarking into a further research project.

Take part in the Delphi survey: an URGENT call to patients and carers – Deadline: February 8th

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Take part in this survey to influence how MDS clinical trials are run

An URGENT reminder about a slightly different type of questionnaire, run by the European big data project Harmony, which is doing research in haematological diseases, including MDS.

The Harmony project WILL provide answers to complex research questions.

This is a very interesting and slightly complex European survey. It was supposed to run last autumn, but progress was slow, as Covid took precedent.

The project has a now a strict deadline of the 8th February 2021.

It is of extreme importance that many patients, carers and advocates complete this questionnaire, as it will influence how MDS clinical trials are run in future and what outcomes are deemed important to concentrate on.

An opportunity to influence the information collected in clinical trials for MDS

The Harmony MDS Delphi survey is about finding out what OUTCOMES are most important to measure, when running MDS clinical trials.

Several OUTCOMES can be measured, but doctors or researchers or patients will have different opinions on what is really important to them.

The Delphi survey will find ALL the OUTCOMES which doctors, researchers and patients all agree with.

That result is then called the CORE OUTCOME SET (in other words the main group of important outcomes, or results), and will be used in clinical trials, to measure efficacy and benefit of new drugs.

Here is also a 3min video which explains the definition of a CORE OUTCOME SET:

Have a say! A call to patients, carers and advocates

This survey WILL make a difference in how future clinical trials in MDS will be conducted, and what information will be collected.

Remember – you are the patients, carers and advocates. Only you can provide the patient voice for preferences in MDS care and treatments.

If you do not say what is important for YOU, other people will decide for you, but it may not quite reflect your wishes and preferences.

Take part here:

You will need to register first, then answer questions for about 20min.

Here is also more information on Harmony:

David P. Steensma, MD, looks for a novel therapy to treat TP53-Mutant MDS

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TP53 gene mutations or chromosome 17 loss which results in TP53 deletion is present in up to 20% of MDS patients. Therapies for these patients have been relatively unsuccessful in delaying the progression of the disease.

Emerging treatments for patients with MDS are frequently based on cytogenetics, i.e. the study of the chromosomes. For this reason it's becoming more important for haematologists to check for mutations, and for patients to know those results.

Ask your haematologist for the results of your molecular test, to find out if you have any mutations, and which ones they are. This information also helps when reading news about future MDS treatments.

In this excellent, very clear interview with USA MDS expert Prof David Steensma, he lists some positive news for MDS patients with the TP53 mutation. It involves a combination of drugs, eprenetapopt  (APR-246) and azacitidine. Combining treatments seems increasingly promising generally in MDS.

Click here to read the full article on Onclive
Prof David Steensma

David P. Steensma, MD, is the clinical director of the Center for Prevention of Progression at Dana-Farber Cancer Institute and an associate professor of medicine at Harvard Medical School both in Boston, Massachusetts.


OncLive: Historically, how have TP53 mutations been targeted, and how effective have these therapeutic strategies been?

Steensma: TP53-mutant disease is one of the biggest unmet needs in MDS. When patients with TP53 mutations receive allogeneic hematopoietic cell transplantation, they tend to do more poorly than those without TP53 mutations, including frequent early relapses. We know that TP53 is enriched in therapy-related MDS, so people who have had treatment for some other type of non-myeloid cancer many years later may develop MDS as a result of the treatment, and when that happens, it is often TP53-mutant disease.

TP53 mutations are associated with short survival, especially when both copies of TP53 are lost. Clinically, the TP53-mutant population is a very high risk group that is often treated with one of the DNA hypomethylating agents, either azacitidine or decitabine, and those lead to a high rate of response. But the response duration is often pretty short, with a median of 8 to 9 months, so the overall survival for these patients tends to be less than 1 year.

How do recent data support the use of this agent in the treatment of TP53-mutant MDS?

One of the exciting things is that on phase 1 and 2 trial eprenetapopt (APR-246) was associated with quite a high overall and complete response rate, certainly higher than we would typically expect to see when looking at historical controls of azacitidine monotherapy. Anytime patients are enrolled in uncontrolled clinical trials, though, we wonder, “Are they just healthier than the general population? Were they destined to do well otherwise?” So the uncontrolled data are difficult to interpret but are encouraging. Still, in MDS, we’ve been burned many times by phase 2 trials in which interventions looked like they had a high response rate, were then taken into phase 3, and showed no improvement.

Some examples are the combination of azacitidine with histone deacetylase inhibitors, such as vorinostat [Zolinza] or entinostat. More than 4 randomized trials of azacitidine with deacetylase inhibitors showed no benefit compared to azacitidine monotherapy, just more adverse events with the combination. Azacitidine plus lenalidomide [Revlimid] also looked pretty good in a phase 2 study but didn’t pan out in the phase 3 investigation [NCT01522976]. The phase 3 trial that is testing APR-246 plus azacitidine versus azacitidine alone [NCT03745716] has now completed accrual and hopefully will report out by the end of the year. It is a really important study for trying to understand if this is a viable strategy.

UPDATE FROM MDS UK - 20-01-2021:

A second phase II study to evaluate if it is possible to improve outcomes for patients with TP53-mutant myelodysplastic syndromes (MDS) and oligoblastic acute myeloid leukemia, who had poor outcomes with standard-of-care azacitidine therapy, produced again encouraging results.

This second study was conducted by David A. Sallman, MD; Amy E. DeZern, MD; Guillermo Garcia-Manero, MD; David P. Steensma, MD and others.

Read the full study in the Journal of Clinical Oncology

The combination of azacitidine and eprenetapopt (APR-246) was well-tolerated in patients with TP53-mutant MDS or acute myeloid leukemia.

Azacitidine and eprenetapopt resulted in a 71% overall response rate and 44% complete remission rate in MDS patients, comparing favourably with a therapy of azacitidine alone.

These results support the ongoing pivotal phase III, multicenter, randomised study of eprenetapopt in combination with azacitidine versus azacitidine alone in patients with TP53-mutant MDS.

Please explain the rationale for combining eprenetapopt and azacitidine.

Both azacitidine and decitabine are important standards of care for patients with MDS. Those are the backbones of therapy, and azacitidine is the only agent that’s been shown to improve overall survival for higher-risk patients in a randomized trial—so patients deserve to have exposure to this drug.

The reason for combining azacitidine with APR-246 is that reactivating TP53 alone only may partially sensitize the cell to dying if it is an abnormal cell. Normally if there’s DNA injury detected in a cell, that cell would be programmed to die, and that program would be mediated by p53. If you restore a p53 function in an abnormal cell but there’s not enough DNA damage to the cell, then the cell may still survive. If you combine APR-246 with an agent that causes DNA damage or cellular stress like azacitidine, then that intact p53 may finally be able to sound the alarm and kill the cell, so to speak.

In MDS, we’ve seen a number of trials where there was no logic behind combining agents with azacitidine. Previously, it’s been, “Oh, our drug has a little bit of effectiveness, or even no effectiveness, but maybe it will be better if we combine it with azacitidine,” and that’s kind of a desperate move. Here, as with venetoclax—which also sensitizes cells to death but doesn’t have a lot of independent activity on its own in myeloid disorders—there’s a real rationale for combination.

What do we know about the safety profile of eprenetapopt (APR-246)?

From what we’ve seen so far, APR-246 has a favorable safety profile. The recurrent toxicity that we’ve seen is a peculiar form of neuropathy where patients may feel altered sensation, such as their skin feeling especially sensitive. They may feel like ants are crawling on their skin or other peculiar sensations, and this usually gets better with prochlorperazine, which is a widely used antinausea medicine. So, for the most part, that adverse event has been manageable.

I was impressed in the phase 1/2 experience by just how well patients did with the drug. Overall, APR-246 didn’t seem to cause a lot of trouble, other than the neuropathy. We will see in the randomized comparison how the toxicity patterns compare to azacitidine alone, and that will provide more information. My suspicion is that we’ll see more neuropathy with the combination group, but probably not a lot of other differences in terms of adverse events.

What challenges remain in other subtypes of MDS?

There are a number of unmet needs for patients with MDS outside of TP53-mutant disease. In the higher-risk population, for the majority of patients, hypomethylating agents will either stop working within 1 to 2 years or they weren’t working in the first place. We don’t have a second-line therapy that’s useful and has been shown to improve survival or delay disease progression, so that’s a big unmet need. In fact, the long-awaited, randomized trial [INSPIRE; NCT02562443] of rigosertib just reported out in that post hypomethylating agent setting, and data showed that rigosertib failed to improve survival compared with physician’s choice of alternate therapy.

The second big unmet need is for the lower-risk patients, whose main problem is low blood counts that could cause them to be transfusion dependent on a regular basis, which is quite inconvenient for them. We had luspatercept [Reblozyl] approved for MDS with ring sideroblasts and SF3B1 mutations in April, which was the first new drug approved for an MDS indication in 14 years. However, luspatercept only helps a subset of patients, and the approval was only for those with ring sideroblasts, which is 15% to 20% of patients at most. There are a lot of other patients with low blood counts that either aren’t good candidates for luspatercept or have been failed by luspatercept, and the question becomes, “How can we improve counts for them?”

These are probably the 2 biggest needs, but there are others. For the transplant population, how to prevent relapse after transplant is also a key question.


  1. Aprea Therapeutics receives FDA breakthrough therapy designation for APR-246 in combination with azacitidine for the treatment of myelodysplastic syndromes (MDS) with a TP53 mutation. News release. Aprea Therapeutics January 30, 2020. Accessed August 25, 2020.
  2. Sallman DA, DeZern AE, Garcia-Manero G, et al. Phase 2 results of APR-246 and azacitidine (AZA) in patients with TP53 mutant myelodysplastic syndromes (MDS) and oligoblastic acute myeloid leukemia (AML). Blood. 2019;134(suppl 1):676. doi:10.1182/blood-2019-131055

Trial to test existing drugs against rare blood cancer

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Cooperation to support a new clinical trial for MDS

A new clinical trial for MDS has just been launched.

Designed by clinical experts, using existing (repurposed) drugs in a new way, with wide PPI (Patient and Public Involvement) input and feedback by MDS UK advocates and patients, funded via Bloodcancer UK.

This trial will test existing drugs against the rare blood cancer Myelodysplastic Syndrome to improve patients' outlook and health.

It is run by UoBbiosciences & Warwick Clinical Trials Unit.

Best practice and cooperation at all levels for research benefitting MDS patients.

By making a donation to MDS UK, you are also funding our time, for the work we conducted with patients, enabling the subsequent approval of this trial.

Full details:

A clinical trial designed to test existing drugs in the treatment of a rare blood cancer has been launched at the Universities of Birmingham and Warwick.

Funded by Blood Cancer UK, the trial seeks to repurpose existing drugs to improve the outlook, health and quality of life for people with Myelodysplastic Syndrome (MDS), a condition in which the patient does not have enough healthy blood cells.

Called REPAIR-MDS (Repurposed drugs to improve haematological responses in Myelodysplastic Syndrome), the trial will test a number of treatments designed to improve the formation of healthy blood cells, reducing or reversing life-limiting deficiencies in red blood cells. The trial is expected to begin recruiting patients in May 2021.

Professor Chris Bunce, in the School of Biosciences at BHP founder-member the University of Birmingham, who led the application to Blood Cancer UK and whose research contributed to the design of the trial treatments, says: “REPAIR-MDS represents a significant turning point in UK medical research. It is the first ever UK randomised trial delivered in this neglected patient group, establishing the precedent for future trials in the UK and elsewhere.”

Professor Janet Dunn, Head of Cancer Trials at Warwick Clinical Trials Unit at the University of Warwick, says: “It’s an important trial for these patients as currently treatment options are limited. We are excited to be working closely with Birmingham and the patient groups, in particular Sophie Wintrich who is CEO from the MDS UK patient support group.”

Although rare, MDS is estimated to be more prevalent in older people, over 70 years old. There is currently no cure for the disease and treatment is usually chemotherapy-based, although in some cases a stem cell transplant may be possible.

Dr Fatima Sulaiman, Head of Research for Blood Cancer UK added: “Blood Cancer UK are really excited to be supporting this trial. We believe that within the next generation, we’ll be able to beat all types of blood cancer, and this trial will take us one step closer to doing this.

“Sadly, only 31% of people diagnosed with myelodysplastic syndrome (MDS) survive 5-years, and we urgently need better treatments. Being able to repurpose existing drugs for people with MDS would mean we would be able to get new treatments to people, sooner, giving everyone the best possible chance of survival.

“Anyone worried about blood cancer can call our free and confidential helpline on +44 (0) 808 2080 888.”

The project is a national effort by the UK MDS research community and will recruit patients from across the UK. The trial was developed by the National Cancer Research Institute (NCRI) MDS clinical studies sub-group and has arisen from research carried out in the School of Biosciences and the School of Medical and Dental Sciences at the University of Birmingham led by Professor Bunce. It will be carried out in collaboration with Queen Elizabeth Hospital Birmingham.

The funding of REPAIR-MDS by Blood Cancer UK has been made possible by a donation made in open competition to the charity from the Jon Moulton Charity Trust.

How have you coped over the past few months of COVID-19? Please let us know

Patients and caregivers/supporters survey on care during the COVID-19 pandemic

MDS UK has teamed up with the NCRI MDS subgroup (National Cancer Research Institute), the UK MDS Forum (National MDS clinical experts group), and created a survey for MDS, CMML and AML patients and caregivers.

What does this survey cover?

This national survey is about

  • your perspective and experience of the COVID-19 pandemic generally
  • the clinical care and support you received
  • the information you had access to
  • treatment of COVID-19, if applicable

How will this data be used?

The data collected will inform clinical staff about any beneficial changes to practice they can take forward to improve the care of MDS and AML patients as well as understand what barriers and difficulties patients came across during this period.

We also hope to pass on some of this data to PHE (Public Health England), to also improve the way they provide information and services to patients and families.

We will publish results on our website as well, and use the information to improve our services to members too.

Confidentiality and GDPR

This survey is set up to be completed totally anonymously, in line with GDPR regulations for this type of work.

MDS UK members will be sent a link, but it will not be possible to trace back answers to individuals.

The link can only be used once per device.

Other surveys on COVID-19

You may have completed other surveys on the topic of Covid-19, for other organisations, but we kindly ask you to complete this one too, as it is the only survey fully dedicated to MDS, and prepared by the team of clinical experts in MDS and the NCRI sub-group on MDS.

Your MDS clinicians may also forward you a link to this particular survey.

It does not matter which link you end up using, as data will be collected together.

How long does it take to complete it?

It may take you up to 30minutes to answer all questions.

Some questions can be skipped.

We thank you for taking this time to provide these very important answers, to help us help you.

Technical Queries?

Please email

MDS UK team

Many thanks to the clinical staff and MDS UK team involved in this survey:

NHS staff: Dr P Krishnamurthy, Dr S Killick, Nurses J Hayden and M Kenyon, Dr J Chadwick, Analyst A Jackson

MDS UK staff and volunteers: C Dugmore, C Richards, IT expert E Fuste, S Wintrich, C McGovern

News for MDS patients from the European Registry

Research FOR Patients
-For an informed and empowered opinion-
Have you made your clinical paper accessible yet?

What is the European Myelodysplastic Syndromes Registry (EUMDS) project?

The best way to study a typical group of MDS patients is to set up a registry. A registry involves obtaining consent from patients and collect information about their MDS and the treatments they receive at their usual clinic or Day Case Unit visits.

The European MDS Registry (EUMDS), was initiated in 2008 by a large group of European haematologists. It contains information on treatment and disease of more than 2500 MDS patients from 17 countries and it continues to include new patients.

Many of you will be familiar with the EUMDS project and some of you are registered and actively sharing your MDS data, helping researchers to improve their understanding of the disease. This helps to shape better clinical trials, and ultimately may lead to better treatments for MDS.

Which scientific studies have been performed so far by the Registry?

The information collected in the EUMDS registry forms the basis for scientific studies. In these studies MDS experts seek to answer scientific questions on MDS and the treatments available. By answering these questions scientists aim to improve the diagnosis and treatment of MDS.

The results from EUMDS scientific studies are published in scientific journals and presented on large haematological congresses.

In addition, the EUMDS publishes summaries of these scientific studies for patients and the general public, in a patient friendly format. These are published on the "Lay English Summaries":

See, for example, two topics which have been published under "Lay English Summaries":

The effect of transfusions on patients quality of life

Impact of red blood cell transfusion dose density on progression-free survival in lower-risk myelodysplastic syndromes patients.

Red blood cell transfusions (RBCT) are an important part of the general medical care for MDS patients. However, the overall life expectancy of patients treated with RBCT is often decreased compared to untransfused MDS patients. It is unclear whether this lower life expectancy is due to the nature of the disease (more severe illness and therefore greater need for transfusions) or whether it is due to unwanted side effects of the RBCT itself. We observed that even patients transfused with small quantities of blood transfusions have a negatively affected life expectancy. We are unsure what causes this negative association, and hypothesize that it could be related to the release of toxic iron radicals after transfusion. Future studies are needed to confirm these observations and in addition, it is necessary to perform dedicated intervention studies with the aim to overcome this negative effect.

Ways to improve diagnosis and prognosis for new patients

Validation of the revised international prognostic scoring system (IPSS-R) in patients with lower-risk myelodysplastic syndromes: a report from the prospective European LeukaemiaNet MDS (EUMDS) registry

The Registry has been opened for a while, and in this timeframe the scientific community has upgraded the criteria which were used to calculate the prognosis of patients with MDS. These new criteria (IPSS-R) were validated on the first 1000 EUMDS patients who were characterized using the ‘’old’’ (IPSS) criteria. This study shows that using the new criteria, our risk assessment improved for the various MDS patient groups. Currently, both the old and the new criteria need to be reported by the physician for each patient who joins the Registry.

Visit the Lay English Summaries of articles published by the EUMDS Registry about the topics below


  • Early platelet count kinetics has prognostic value in lower-risk myelodysplastic syndromes
  • Health-related quality of life in lower-risk MDS patients compared with age- and sex-matched reference populations: a European LeukemiaNet study.
  • Labile Plasma Iron Levels Predict Survival In Patients With Lower-Risk Myelodysplastic Syndromes
  • Erythropoiesis-stimulating agents significantly delay the onset of a regular transfusion need in nontransfused patients with lower-risk myelodysplastic syndrome
  • Impact of treatment with iron chelation therapy in patients with lower-risk myelodysplastic syndromes participating in the European MDS registry


If you found the articles above of interest to you, please also read on about MDS Right.

MDS-RIGHT (Providing the right care to the right patient with MyeloDysplastic Syndrome at the right time) is a research project that has been granted 6 million Euros from the Horizon2020 programme of the European Union. In this project – which started in May 2015 and will end in 2020 - fifteen European partners have joined forces. By comparing existing health care interventions and by defining and implementing more effective and safer interventions for elderly European citizens with anaemia and/or lower-risk MDS, the project aims to lead to better treatment compliance and more (cost-)effective use of healthcare resources. “

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