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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, jayne.croft@nhs.net

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 info@mdspatientsupport.org.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, jayne.croft@nhs.net

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!

WITH YOUR HELP, WE CAN HELP

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 Nature.com. 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


I want to raise money for MDS UK

Why fundraising for MDS? Watch the video:

If you'd like to fundraise for us, please fill in this form!

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

Contact Us

Our website and online discussion forum

Visited by more than 32,000 people last year

Visit the Forum

2 Facebook pages

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

Visit our Facebook page

A national survey project

Assessing the needs of UK MDS patients amongst 450 MDS patients

Complete our survey

You can make a significant difference to our small charity.

Maybe you have a particular talent or skill, or you know someone with useful contacts? Here is an A to Z list of Amazing Fundraising Ideas

Every single fundraiser will be absolutely worshipped!

With your help – we can help.

Many thanks from the MDS UK Team


Missed the Harrogate MDS Patients & Family Forum? Catch Up Online!

Patients and their families heard about the latest in MDS research and treatment

The latest MDS UK Patient and Family Forum took place in Harrogate on Friday 22nd March.

It was an invaluable opportunity for those affected by MDS to hear about the latest developments in MDS research and treatment.

The meeting was open to MDS patients and their guests, as well as clinical staff. Patients and their families had the opportunity to participate in informal discussion regarding their quality-of-life issues, new clinical trials, new diagnostic methods, therapies and treatment options.

The day also included presentations by MDS experts, guest speakers and patients. The list of speakers included Prof David Bowen, Consultant Haematologist, St. James's Institute of Oncology, Leeds, Dr Simone Green, Castle Hill Hospital, Cottingham, Hull and Dr Anjum Khan, St. James's Institute of Oncology, Leeds, among others.

Read The 2019 Forum Presentations with the Latest News on MDS

New advances in MDS & emerging treatment options by Dr Catherine Cargo

  • Advances in understanding MDS genetic biology
  • How can this help in the diagnosis, risk assessment & treatment?

Download PDF

Clinical Trials in MDS by Dr Simone Green

Which clinical trials are open to recruitment in the UK? What are their aims?

Download PDF

What are Myelodysplastic Syndromes and what are their current treatment options by Prof. D. Bowen

  • What is MDS?
  • What are the goals for treatment?
  • How do we treat MDS in the UK in 2019?

Download PDF

The European MDS Registry - by Dr Catherine Langton

What is a Registry Trial? How can it improve the lives of patients?

Download PDF

Following our Harrogate Patient Forum, we had several attendees interested in helping us start a local MDS group for Yorkshire.

If you are interested in joining this group, and contributing, please contact us on info@mdspatientsupport.org.uk .

We also had requests, and interest to start a local group for Newcastle, which we will look into as well.

Again – please contact us if interested!

Don't miss our next Patient and Family Forum! Contact us to get invited. Telephone: 020 7733 7558 Email: info@mdspatientsupport.org.uk


Survey for AML patients to help measure the effects of new drugs for AML

Currently, several new drugs are being tested in clinical trials, aiming at improving the treatment of haematologic malignancies (HM).

These trials, however, measure different results - also called treatment response, or outcomes - which makes it difficult to compare and combine all this information.

Lars Bullinger (Charité Hospital in Berlin) makes the following observations:

“For instance, researchers may use a variety of parameters to describe response to treatment: complete remission, complete remission with incomplete hematologic recovery; complete remission and MRD negative; partial remission; or stable disease.

In addition, the majority of clinical trials do not assess long-term side effects and their influence on the patients’ quality of life.

A core outcome set (COS) may offer a solution to these problems.”

This survey asks AML patients about which specific results they think are important to measure in AML clinical trials based on their experiences and establish a core outcome set.

With of help of AML patients The HARMONY Alliance will ensure that in future clinical trials appropriate outcomes are measured, those that are important not only to health care professionals but also to patients.

To maximize participation please feel free to share this invitation.

Following the start of this pilot, further disease-specific core outcome set studies will be planned for each of the 7 haematological cancers.

Learn more about AML DELPHI - Core Outcome Set Survey

Download the full document

Would you like to take part in the survey?

Register now and you'll receive a registration email with login-details

The results of these surveys will be used to select what is important to measure (the outcomes) in all future clinical trials - based on patient experiences and preferences.

This is a REALLY important piece of work and you can have an impact on its results!


Are new cancer drugs been developed and recommended to patients?

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

The Institute of Cancer Research, London, one of the world’s most influential cancer research organisations, has published an in-depth analysis about the discovery and access of innovative cancer drugs.

Read the full report

Good news for haematological cancers

Haematological cancers have seen dramatic advances in treatment over the last decade as cancer patients reap the rewards from progress in research.

Over one-third of all authorisations for new drugs were for haematological cancers (leukaemia, lymphoma and myeloma), with many individual drugs receiving authorisations for multiple indications which, in part, may reflect the diversity of this group of diseases.

In total, the European Medicines Agency, the EMA, licensed 34 new drugs for blood cancers. Among those drugs authorised for MDS are Vidaza and Revlimid and Epoetin

How long does it take for patients to get new cancer drugs once they have been patented?

The report also examines the data to find out how quickly patented discoveries for cancer are moving through the drug development pipeline, EMA authorisation and the NICE appraisal process.

Some findings were not so encouraging. The time it is taking to evaluate drugs in clinical trials and make them available for NHS patients is increasing. And the average time from the start of a phase I trial to EMA authorisation is increasing – going up from 7.8 years between 2000 to 2008 to 9.1 years from 2009 to 2016

On the positive side, since 2009, NICE has successfully reduced the lag time between EMA authorisation and the beginning of its appraisal from a mean of 21 months to 6.5 months. However of the drugs NICE has assessed, only two-thirds have been recommended by NICE for NHS use. Highly innovative drugs, which attack cancers in new ways, were less likely to have been approved by NICE for use in the NHS.

Report Conclusions

Taking into consideration all types of cancer, and the access to medicines among different populations, the report shows evidence that:

  1. Many cancer patients are missing out on new drugs
  2. More drugs are needed for children’s cancers
  3. It is taking far too long to deliver new cancer drugs to patients
  4. We need to do more to encourage radical innovation

Professor Raj Chopra Head of the Division of Cancer Therapeutics and Director of the Cancer Research UK Cancer Therapeutics Unit at the ICR:

“Big leaps forward in cancer survival are achievable, but only if we find ways to better harness our increased scientific knowledge of cancer and accelerate the delivery of innovative new drugs to patients.”

Clinical Trials open to recruitment in the UK


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