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MDS UK Patient Support Group Newsletter – June 2019

Our 10th edition of the MDS UK Newsletter is now out!

Read all about:

Patient's Stories - Living with MDS:

    • Bergit Kuhle: B12 meets MDS; Michael Bower; Bernard E Burke; Shareen Rouvray - Part 1; John and Sandra; Poetry by Kate

Research - Progress in the diagnosis and treatment of MDS:

    • Prof D Wiseman Chronic Myelomonocytic Leukaemia (CMML), European MDS Registry, Prof David Bowen; Familial/inherited MDS: rare but important to keep in mind, Professors Jude Fitzgibbon, Tom Vulliamy, Inderjeet Dokal Queen Mary University of London; A Feasibility Randomized Trial of Red Cell Transfusion Thresholds in Myelodysplasia, Professor Simon Stanworth; Online Consultations with an MDS expert, Prof David Bowen; Data – a patient’s new best friend, Dr Thomas Coats

Fundraising Successes:

    • London Marathon 2019; Turks Head 10k fun run; and a lot more!

Here to help:

    How support groups and technology can assist in communication, Dr Pavel Peter Kotoucek, FRCPath Haematology Department, Broomfield Hospital, Chelmsford; Making the most of your consultation; A a very successful Patient Information Forum in Harrogate; Regional groups update and more

Read More

Precision medicine is coming to the clinic to treat MDS

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

by Niels Jensen

What is precision medicine?

In precision medicine the basic idea is to develop a treatment for the specific cancer of individual patients based on a genetic understanding of their disease. Precision medicine has also been called personalized medicine or targetted medicine.

The basic idea is not completely new. Already in November 2013 Esquire Magazine reported the development of a treatment specifically for Stephanie Lee's colon cancer . The treatment effect had been verified on a banana fly, but the board of oncology at Mount Sinai in New York hesitated to give the experimental treatment to the patient and opted for a more conventional treatment. Read the story in Esquire

In Denmark, a collaboration between clinicians at Rigshospitalet and researchers at the Copenhagen University Biotech Research and Innovation Center (BRIC) has systematized a procedure for the development of a precision treatment for each MDS or leukemia patient who is signed up for trial, which has very few exclusion criteria, and straight forward inclusion criteria: you must have either MDS or leukemia and being treated at one of seven hematological centers in Denmark.

Read the full article: New Research Centre to improve personalized treatment for Danish patients with blood cancers

How does precision medicine work?

When a patient signs up for the trial, they are given a standard protocol treatment depending on the disease and the stage of the disease.

While the patient gets the protocol treatment' clinicians and researchers get to work: blood and bone marrow samples are collected from the patient. The patient's tumor cells are then screened in the labs against treatment effect from one or two of more than 400 already commercially available drugs.

If a drug or mixture of drugs shows a positive effect on the patient's cancer cells, then the second step is initiated. This involved growing the patient's cancer cells in the lab. Once enough cells are available they are injected into a mouse together with some of the microenvironment from the patient's bone marrow.

Then the researcher treats the mouse with the drug or drug mixture showed as having a positive effect during the laboratory screening.

If the mouse is successfully treated, then the clinicians have a precision medicine which, in Denmark, can be legally offered to the patient in the clinic.

Doctors in Denmark are allowed to use drugs off-label if they have evidence that the treatment works. This has been done for many years with EPO-like substances to improve red blood cell counts in low risk MDS patients.

Simultaneously, a whole genome next generation sequencing of the samples from the patient is performed to identify the specific mutations in the patient's cancer cells. This helps researchers to understand why a particular drug or drug mixture have a positive effect, and add the laboratory screening.

Precision Medicine - A specific drug for each genetic mutation

Professor Kirsten Grønbæk: "While we try to treat a specific patient, we also learn something for the benefit of future patients"

A scientific article published by the University of Copenhagen, Danish Research Center for Precision Medicine of Blood Cancer, explains that the program aims at improving the immediate and long-term outcome for blood cancer patients by coordinating and strengthening ongoing blood cancer research into a program pursuing research questions and integrating results from bench-to-bedside and bedside-to-bench.

The goal is to optimize the use of already approved drugs, identify new targets for therapy, develop novel therapies, test potential novel drugs in pre-clinical models, and collaborate with pharmaceutical companies on developing new drugs and test these and other novel drugs in Phase I-II clinical trials.

Professor Kirsten Grønbæk, PTH Professor and Chief Physician at Rigshospitalet, says:

Via 'drug screening' the cancer stem cells from the individual patient could be exposed to 400 different drugs.

In this way, we hope to find the medicine that accurately affects the individual's cancer stem cells, which is the cause for the cancer to return.

At the same time, we will try to find the molecular changes that indicate that this is the perfect treatment. For some patients, we will immediately find one or more drugs that work, but not for all who are in the need for treatment.

The idea is that while we try to treat a specific patient, we also learn something for the benefit of future patients.

The equipment for this trial have been financed by a grant from the Novo Nordic Foundation. A grant from the Danish Cancer Society covers the expenses for the first three years, and also access to the trial across the country. The trial was conceived by a collaboration between clinician Kirsten Grønbæk, and BRIC researchers Kristian Helin (currently part time at Memorial Sloan Kettering) and Bo Porse.

Clinical Trials open to recruitment in the UK

Support Team MDS at the Great North Run on September 8!

The Great North Run: a 13.1 mile journey from Newcastle's city centre to the coast in South Shields

Taking place on on Sunday 8 September 2019, the Great North Run is the largest half marathon in the world, taking place annually in North East England each September.

And we have an amazing team running for MDS UK Patient Support Group:

You can make a GREAT difference to MDS patients

Support the MDS Team at the Great North Run

Every donation, big or small, helps us maintain our

FREE services to MDS patients!


Kathleen Young

Angela and Shari Stirling

Russell Cook

Harmony: a European project with good news for MDS patients

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

The Harmony Alliance is a first-of-its-kind partnership of over 80 private and public organisations aiming at enabling better and faster treatments for patients with blood cancers such as MDS.

Patient Organisations have a very active role in Harmony, and we are permanently involved in the definition of outcomes for patients and the design of research projects.

At the heart of Harmony is the sharing of big amounts of data from patients with blood cancer from all over Europe.

Watch this video to understand how Big Data can help clinicians to treat blood cancer

The Harmony Alliance aims to develop treatments that are:

  • more effective
  • have fewer side-effects
  • secure a higher quality of life

However the best possible treatment may differ from one person to another. This is partly due to biological differences such as the genetic characteristics of the tumor cells.

By studying big amounts of data, researchers can learn how to predict the development of a particular disease and how certain subgroups of patients will respond to treatment.

This should result in tools that will enable clinicians and doctors to rapidly select the most promising treatment for a particular patient.

How does Harmony ensure data quality, safety and privacy?

The Harmony Alliance has developed very careful data-handling procedures to provide maximum protection of the patients’ privacy, while leaving the data rich enough for meaningful scientific analysis.

Watch this video to understand how Harmony ensures high quality and secure data that will help improve the treatment of blood cancers.

The new generation of MDS diagnostics: the Myeloid Gene Panel

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

Report from the MDS Education Forum that took place in November 2018. Interviews by Sophie Wintrich Chief Executive of MDS UK Patient Support Group.

What are Myeloid Gene Panels and how are they used in MDS?

One of the most discussed topics in the MDS Education Forum was Myeloid Gene Panels.

Myeloid Gene Panels are a set of specific genes present in MDS patients, whose mutations are thought to be driving MDS. These genes can be identified with innovative genetic tests.

As stated by Nick Cross, professor of Human Genetics at the University of Southampton:

MDS is a very complex disorder, but as with all these different types of disorders, they are fundamentally driven by abnormalities of genes that are acquired during the lifetime of an individual.

What we are looking for is the large number of genes that are involved in MDS to see whether there are specific mutations, changes in those genes, that may actually be driving MDS.

Finding these can increase the confidence that this is really a diagnosis of MDS. We also know that some particular genes are associated with a better or a worse prognosis, and that they may indicate specific types of treatment.

Take a look at what is being done in the Wessex region, through a number of services, including Myeloid Gene Panels, and why such tests and technologies are used to help scientists and physicians in their diagnosis of MDS.

Scientists Interviewed:

  • MS Sophie Laird, Clinical Scientist , Wessex Regional Genetic Laboratory
  • Dr Sally Killick , Consultant Haematologist, Bournemouth Hospital
  • Prof Nick Cross, professor of Human Genetics at the University of Southampton

The Myelod Gene Panel and how it helps MDS Patients

Dr Lynn Quek, Consultant Haematologist and Research Scientist at Oxford University Hospital, is interviewed by Sophie Wintrich.

She explains the impact that Myeloid Gene Panel data has on treatment decisions in MDS, and how the genetic information of individual patients helps to personalise and tailor their clinical care.

New service for patients: online video consultations with an MDS specialist

For MDS patients who are unable or prefer not to travel to a conventional appointment: one to one video consultations online

Professor David Bowen and Leeds Teaching Hospitals are pleased to announce a new service for patients who would like a specialist MDS consultation but who are unable to, or prefer not to travel to Leeds for a conventional clinic appointment.

This is a brand new type of NHS service opening for MDS patients, for online clinical consultations with one of the main MDS specialists in the UK, Professor David Bowen, from the MDS Centre of Excellence in Leeds.

Make an appointment!

Send an email to Professor Bowen’s secretary,

Video consultations are available, generally on Wednesdays but with some flexibility.

Who is it for?

Any MDS patients who would like a specialist MDS consultation.

This service is very similar to the standard referral request for an “Additional opinion”, or second opinion at an MDS Centre of Excellence.

Prof Bowen: "Patients should have the opportunity to see a true expert in whatever disease they have"

How can you access this?

If interested, just like any conventional referral request, speak to your GP or local haematologist, explaining you would like to have an additional opinion from an MDS expert.

MDS is a rarer disease, and often requires an MDS expert to evaluate the exact sub-type of MDS, or explore and discuss additional treatment options, including clinical trials.

Your haematologist, or GP, will then need to refer via the traditional referral system, to Professor David Bowen, at Leeds Teaching Hospital.

An appointment will be arranged after receipt of a formal referral from either the GP or local Haematologist, which should contain as much detail as possible, specifically results of bone marrow tests and details of treatment

Technical Requirements:

Patients will require an internet connection and a smartphone, tablet or laptop/desktop computer with a camera.

This video consultation service is offered via a secure portal from a professional company specialising in NHS video consultation, and confidentiality is of paramount importance.

Video communication

Video communication

How will it work?

A User Guide will be sent with the appointment notification.

The appointment will be arranged after receipt of a formal referral from either the GP or local Haematologist, which should contain as much detail as possible, specifically results of bone marrow tests and details of treatment.

The consultation will either be a one-off session, or part of an on-going care, depending on the situation and patient preference, just like the more traditional “shared care” setup.

Contact Us:

For any questions, please contact us directly at MDS UK - Tel 020 77337558

Patients outside England

NHS funding mandates that for UK patients resident outside England, the local consultant / GP must have agreed funding for the consultation from the local payers (eg Health Boards in Scotland, and equivalent in N. Ireland and Wales), prior to the formal referral to Professor Bowen.

Overseas patients

The video consultation service is available to overseas patients for a relatively small fee.

A formal referral from a local haematologist is preferred, accompanied by details of bone marrow and blood tests, and of treatments received.

These can be sent by email to Professor Bowen’s secretary,

Prior to the consultation a signed document confirming the undertaking to pay the overseas patient fee must be received by Leeds Teaching Hospital. An approximate charge for a single consultation is currently £196; this may vary.

Register Your Interest:

Become an MDS UK member and select "Video Consultation"

Prof Bowen talks about the results of MDS Right, a European study on MDS patients

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

MDS Right: a European observational study

If you are an MDS patient, it is important to find out about scientific research, and to know about ways you can contribute, as new treatments or improvements may come out of research.

Clinical trials are one way to develop new therapies, but observational studies are equally important.

Observational studies are those in which individuals are observed or certain outcomes are measured. No attempt is made to affect the outcome so no treatment changes needed, no additional decisions are made, no complex randomization and no placebo to take.

You don’t even need to be on treatment – so also Watch&Wait patients can take part.

All patients can participate, as opposed to the often complex or unrealistic eligibility criteria of drug trials, which only allow younger patients, or those without additional co-morbidities.

MDS Right aims to learn more about real MDS patients

MDS Right is an observational study, set up in May 2015, to gain access to real world data of MDS patients and elderly patients with anaemia symptoms. The hypothesis is that some of these patients might have low risk MDS. The study is running in 15 EU countries.

If you are not yet involved – please ask your haematologist about it and show them this article.

Watch this interview with Prof David Bowen, talking about the progress of the MDS Right Project and read the excerpts below.

Which improvements for patients are coming out of the Registry Programme?

Read the excerpts of David Bowen's Interview

We've got a large number of patients  (2,500) who've been followed for a few several years.

We're starting to generate a set of data that reflects what really happens to patients with MDS, how they're managed, and we can start to tease out how better to use the drugs that we have available.

We can also do research on a population of patients that reflects those that we see every day in the clinic not the super-fit patients and generally younger patients that we treat within clinical trials. So that the time has come and we've learnt quite a few things about this patient journey.

The findings around iron chelation therapy

There's been much debate about iron chelation therapy - removing iron in patients who have multiple transfusions. We're starting to generate evidence that supports the benefits of iron chelation therapy. There's been quite a bit of evidence generated in the past but not with the same degree of quality data and granularity of data.

We seem to be able to demonstrate that patients who receive iron chelation therapy are living longer than those who didn't so that's been quite valuable. It's a little bit preliminary at the moment but it supports a lot of other evidence and I think it's going to probably stack up quite well in the future.

How to use the drugs that we use for anemia, how to remove iron, these are very important aspects that we're generating, which affect the way that we manage patients.

Red cell transfusion and how to do that better

On the red cell transfusion side Simon Stanworth, my colleague, and I have worked on a trial that we've been running over the last two or three years with colleagues in Canada and in  Australia and we've basically been trying to work out whether we can allocate patients to two different types of transfusion schedule. We thought was going to be challenging and in fact we have managed to to do that: we have allocated patients to two different transfusions regimes and we see some differences in the outcome which is very exploratory at the moment but really quite exciting and our Canadian colleagues are just completing their trial. We'll see if they come up with the same findings and so we're moving on to the next phase of that shortly.

Watch Prof Simon Stanworth talking about REDDS, a study to look at transfusion patterns for low-risk MDS patients, and whether an alternative pattern of delivery might have a positive impact on quality of life

What are the drugs that we're evaluating at the moment to treat anemia in MDS?

Prof. Bowen: "The other session which is in the program on Wednesday is related to treatment of anemia. There's a new drug which is currently under consideration by the European Medicines Agency called Luspatercept and I'll be explaining exactly what the role of Luspatercept is in these patients that are the target for that, and also an idea of the various different drugs that are coming along, one or two of which could well also be useful in treating anaemia and be additional to what we've got at the moment. So we're covering several aspects of anaemia and MDS."

What is 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.

Myelodysplastic Syndromes (MDS ) are a group of chronic bone marrow malignancies, predominant in the elderly and complicated by severe anaemia (Anaemia of the Elderly, AoE). Symptoms and complications of MDS negatively impact the quality of life of patients, but in many cases MDS is not correctly diagnosed.

In elderly people anaemia is a medical condition that is often seen. It is hypothesized that 20% of all AoE cases - a total of 2 million European elderly citizens - might be explained by lower risk MDS. AoE is an increasing and complex challenge: the ageing population leads to an increasing financial burden to our healthcare systems, while insufficient awareness of the importance of a timely and right diagnosis increases the burden placed on patients and caregivers and decreases their health related quality of life (HRQoL).

The European project partners will work closely together to address this problem and to develop more (cost-)effective and safer evidence-based, tailored interventions for elderly patients with anaemia and/or lower risk MDS.

This project has received funding from the European Union's Horizon 2020 research and innovation programme under grant agreement No. 634789.

Visit MDS Europe website which covers MDS Right. It is also an amazing source of further information about MDS latest news, from a research and treatment perspective.

Whether you are an individual affected by MDS, or a patient organisation, do make a point of visiting it regularly.

Clinical Trials open to recruitment in the UK

Join Team MDS at London to Paris Bike Ride 15/07/2020 – 19/07/2020

Join Team MDS at London to Paris Bike Ride for the experience of a lifetime!

We wanted to you be the first to hear that registration is now open on our iconic London to ParisTour de France Challenge for 2020!

Taking place on 15 – 19 July 2020, this incredible adventure is our most popular cycling challenge and it's easy to see why - with the the finale of the world's greatest cycle race awaiting challengers at the finish line!

4 amazing days in the saddle

311 miles from capital to capital

You can make a GREAT difference to MDS patients

Join in and we will support you all the way

Every single fundraiser will be absolutely worshipped!


Download the full brochure here

No other interest can take precedence: a patient’s perspective on oncology drug development

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

By Bettina Ryll

My husband’s diagnosis with melanoma and our struggle to access effective therapy challenged what I had learnt about medical research. I have since founded a patient network, becoming a vocal advocate for patient- centric drug development. Herein, I discuss some of the lessons I have learnt.

I clearly remember the first lecture about evidence- based medicine I attended. I was a third- year medical student, and after 2 years of theoretical training I was now seeing my first patients.

Scientific rigour in the form of blinded, placebo-controlled randomized clinical trials seemed to be the solution to my worries: I would be able to test whether treatments were actually as effective as hoped. The structured approach would protect me from falling prey to my own biases and my patients would be safe! I was sold.

What is a Clinical Trial? Learn More

The personal experience that changed my views

In the spring of 2011, and without any warning, the little hard lump under my husband’s arm turned out to be a melanoma that had already spread to his lungs and spine. We were told not to hope for a cure and that any treatment would be palliative. Less than half an hour from returning from the appointment, my computer-savvy husband found what I hadn’t had the heart to tell him. “I won’t be here for Christmas,” he said.

Chemotherapy, the then standard-of-care therapy, was largely ineffective, as I knew since medical school. It was also the comparator in any available clinical trial. Friends referred to it as a ‘nocibo’, a non- effective treatment with adverse effects.

All this happened while, for the first time, not one but two new therapeutic classes were making tumours ‘melt’ in early phase clinical studies that were not even intended to evaluate efficacy — tumours like the one I watched growing daily in despair.

He did, however, live to see Christmas — thanks to clinical trials or, rather, thanks to us learning how to use clinical trials to obtain what he desired most: more time with his daughters.

Receiving a life- shortening diagnosis causes unbelievable suffering for patients and families. Withholding effective therapies in this setting, all in the name of ‘good science’, is inhumane.

It was an eye-opening experience to be at the receiving end of what I had once considered the solution to, not the cause of, suffering. Knowing that I could have been the person inflicting this experience on others horrifies me to this day and has become the driving force behind my advocacy efforts.

"Most European cancer patient advocacy groups welcome structured patient involvement and have started to systematically learn from strong patient communities that have been able to shape the drug development process."

This article first appeared on Read the full article

The search for new ways to design clinical trials

"In my experience, patients with melanoma join clinical trials for one of several reasons: a largely ineffective standard-of-care therapy (as is currently the case for uveal melanoma), the patient has exhausted all existing lines of
therapy, or the therapy is approved but not reimbursed in a given country. The motivation invariably is access to treatment."

"If the interest of a patient is to seek the most promising treatment, then an ethical cancer trial design needs to ensure this, ."

"Furthermore, a situation of high unmet need alters our perceptions of acceptable benefit and risk, a fact that is also increasingly acknowledged and accounted for by decision makers such as the European Medicines Agency...For a chance to survive, patients with melanoma prefer an unknown risk to a known risk with insufficient benefit, in the full understanding that the novel therapy might turn out worse."

"Most European cancer patient advocacy groups welcome structured patient involvement and have started to systematically learn from strong patient communities that have been able to shape the drug development process."

"A notable example is the community of patients with HIV or AIDS, which successfully challenged prevalent clinical trial designs by introducing novel surrogate end points, kick-started the testing of combinations of unapproved therapies and pushed for expanded access programme."

"Thanks to the Internet, nowadays patients are better educated and connected than ever before... Today’s research subjects are becoming emancipated and able to enforce their preferences. Drug development and clinical trial designs will have to follow suit... New trial designs and data sources will come with their own challenges. However, it will become increasingly hard to ignore what should have always had precedence: a patient’s best interest."

Clinical Trials open to recruitment in the UK

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MDS UK does not receive any other types of grants or assistance from Government or Trusts.

We need funds to offer patients these services free of charge:

National and local meetings for patients and families

A 1 day meeting costs about £5,000 for 100 attendees

Patient Meetings

2 newsletters per year, leaflets & information material

We send out about 15-20 information packs a month

Information Material

A telephone helpline, manned 5 days a week

We get about 10 calls a week from patients and family members

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Our website and online discussion forum

Visited by more than 32,000 people last year

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2 Facebook pages

One public, liked by 2,150 people, and one confidential, with 686 members

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