Latest MDS research on the 57th Annual Meeting of the American Society of Haematology (ASH)

Dr Mike Dennis

by Dr Mike Dennis,  
Consultant Haematologist, The Christie, Manchester

The 57th meeting was held in Orlando, Florida (USA) in December 2015 and was attended by more than 20,000 professionals interested in blood disorders.  Here is a summary of Dr Mike Dennis report. Read the full report here.

Biology of MDS

An increasing number of specific gene mutations are being identified in patients with MDS.

An international collaboration of more than 3000 patients’ samples with MDS was presented by Rafael Bejar, Assistant Professor, University of California, San Diego (USA). This provided a wealth of further knowledge on the types of mutations that occur, their potential prognostic impact and potential therapeutic targets.

Lee presented work on spliceosomal mutations, demonstrating their importance in the generation of MDS and how they can be modulated by therapy, therefore creating a new therapeutic strategy for future clinical trials.

In the interim, there are relatively simple pathology tests (such as P53 staining) that can be very informative in clinical decision-making for clinicians.

Developments in MDS therapy

For me, the main themes explored from a therapeutic perspective were
1. Treatment options for low risk patients no longer responding to Erythropoiesis-Stimulating Agent (ESA). 

Luspatercept (previously known as ACE-536)  is a protein that causes a rise in haemoglobin in healthy volunteers.  Professor Aristoteles Giagounidis MD, Head of the Department of Oncology, Haematology and Palliative Care, Marien Hospital, Düsseldorf (Germany) presented results of a study in Germany where 58 patients with low risk MDS have been treated, if they’ve failed or are unlikely to respond to ESA. The majority went on to an extension study where the response rate was nearly 70%, with most responses being long lasting and the treatment well tolerated.  An international trial ‘MEDALIST’ is now being planned.

Other similar agents, such as Sotatercept (ACE-011), are also in clinical development.

2. Telomerase 

Most cancers have high levels of telomerase, including the more aggressive forms of MDS. Imetelstat is a strong inhibitor of telomerase which, in clinical trials for myelofibrosis, was found to have a high response rate in patients with specific mutations also common in MDS (SF3B1).

Aref Al-Kali MD (Assistant Professor of Medicine, Mayo Clinic, USA or Tel-Aviv Sourasky Medical Center, Israel) presented the initial findings of a trial in MDS patients with low risk disease, suggesting a good response rate with a reduction in transfusion requirements and good tolerability – again, there are trials planned with the agent in the UK in the near future.

3. Treatment options to improve the response rate with azacitidine 

There were a large number of studies looking at combination therapy with the current standard azacitidine to improve response rates and therefore survival whilst remaining well tolerated and preserving life quality for patients.

Eltrombopag (EPAG) is an oral medication which can stimulate platelet production. Professor Moshe Mittelman MD, Tel-Aviv Sourasky Medical Center, Tel-Aviv, Israel presented the findings of the ASPIRE study where EPAG was used in patients with advanced forms of MDS and low platelet counts, indicating it appears safe and well tolerated with some activity. Again, it was noted that it can stimulate neutrophil and red cell production. EPAG is currently undergoing trials in the UK in combination with azacitidine (ELASTIC trial).

4. Transplantation 

Randomised trials in stem cell transplantation are a rare event partly due to the complexity and the clinician experience with their approach to various clinical transplant situations. The MAvRIC trial was presented by Scott - 272 patients with MDS/AML who were transplanted across 32 transplant centres in the US.

The findings largely supported the already widely held belief that full intensity stem cell transplants are more toxic but have a lower relapse rate when compared to the more frequent approach of reduced intensity conditioning.


From a clinician’s perspective, patients with MDS are all too often a significant challenge with relatively few truly effective therapies available.

Our increasing understanding of the biology of the disease is creating more informed treatment decision-making and further therapeutic opportunities which can be explored. Progress in getting new treatments available for patients with MDS has, at times, been frustratingly slow and we are now looking at a future where further licensed therapies in MDS will not be far away.

MDS patients and carers attend parliamentary reception for Cancer52

5th May 2016

On 20th April a group of seven MDS (Myelodysplastic Syndromes, a rare blood cancer) patients and carers went to Westminster and attended a parliamentary reception organised by Cancer52, a coalition of more than 80 charities representing rare and less common cancers. The reception was hosted and addressed by Chairman of the All-Party Parliamentary Group on Cancer (APPGC), John Baron MP.

The purpose of the reception was to raise awareness of these cancers and highlight the need for earlier diagnosis.

Rare and less common cancers together account for more than half of all UK cancer deaths – more than the ‘big four’ (breast, prostate, lung and bowel cancer together) – and on average have poorer earlier diagnosis rates.

These seven patients and carers were invited because they are members of MDS UK Patient Support Group, one of the member charities of Cancer52:

Prof Rodney Taylor is Chairman of MDS UK, an MDS patient himself, has experienced a number of MDS treatments (supportive care, transfusions, an MDS specific drug) over the course of 5-6 years. A year ago, when he stopped responding to a life extending drug azacitidine, he had to resort to a stem cell transplant at age 71. His wife and carer, Dr Janet Taylor accompanied him. They met with their MP Dr Tania Mathias.

Reverend Kes Grant, was diagnosed with MDS 16 years ago, at age 35. Following a gradual but serious deterioration of her health and quality of life, she too is now facing the prospect of a stem cell transplant. A treatment she is not looking forward to, as outcomes vary greatly and carry a good deal of uncertainty. Kes recently had to give up her work as a school chaplain, due to MDS, despite trying very hard to still work part-time. This has been devastating for her. Kes attended with her partner and carer Maggie Buckley. They met with their MP Matthew Pennycock.

Leanne Tilbrook, a very young MDS patients (typical age at diagnosis is 71yrs old), needs a transplant soon. She attended with her father Steve Tilbrook – who has been supporting MDS UK for a long time.
Neil Harrap very sadly lost his wife Julie to MDS two years ago, following complications of her stem cell transplant. He has now vowed to help raise awareness of the disease with MDS UK.

MDS UK members and their MP’s were briefed on the number of people diagnosed with rare and less common cancers in their area.

Jane Lyons, CEO of Cancer52, said, “It was fantastic to see so many people affected by rare and less common cancers at the reception raising awareness of these diseases with their MPs. For many the speed of diagnosis is critical as rare and less common cancers are more likely than the common cancers to be diagnosed late, often in A&E, and this has an impact on one year survival rates."

Rodney Taylor said, “It was a pleasure to join Cancer52 at their reception and discuss such an important issue. We need to be doing far better at diagnosing, referring and treating cancers earlier, particularly rare and less common cancers. Access to innovative treatments need to be much improved too.”

Dr Tania Mathias MP said “A privilege to meet patient-expert and constituent Prof Taylor: excellent action points concerning rare cancers.”

Useful links:
For more information about MDS UK please visit
For more information about Cancer52 please visit:

The All-Party Parliamentary Group on Cancer is the voice of the cancer community in Parliament, raising cancer at every opportunity, including on the Floor of the House and in meetings with Ministers and others.
The Cancer Strategy (Achieving world-class cancer outcomes: a strategy for England 2015-2020) was published in July 2015

Nurses’ Day – 12 May 2016 – Kes Grant’s Thank You

17th May 2016

Nurses Day

Nurses’ Day takes place on 12th of May each year, on the anniversary of Florence Nightingale’s birth.

It’s a chance to celebrate the remarkable difference nursing staff around the world make every day. It’s also a time for patients to thank nursing staff for their dedication and care.

This year we've taken the opportunity to thank all fantastic haematology nurses and CNS's through our Facebook page. Patients also shared their story and say thank you for the support the nurses provide, often in very difficult circumstances, on the Royal College of Nurses website. One of these moving stories was Kes's.

Kes Grants's Thank You



"16 years ago I was diagnosed with a rare blood cancer called Myelodysplastic Syndrome or MDS. MDS is incredibly debilitating – I live in almost constant pain, and part of the disease causes my joints to swell up and my hair to fall out."

"Despite the challenges I face every day, I still feel inspired to talk about my MDS, to share experiences with newly diagnosed patients and to let them know that life is worth living.

"Part of the reason for this is my Clinical Nurse Specialist (CNS), Geke. She has been a rock from the very beginning.

"Three times a year we visit a patient support group at King’s College Hospital in London. At times we deliver talks together on coping with fatigue and the demands of a fluctuating condition.

"I hope our unique double act encourages others to seek help, and in particular the support of a CNS, because Geke is truly my safety net.

"While MDS affects me every day, I know the nature of the condition makes Geke’s job difficult too. Patients get very poorly and die but Geke still invests her time and energy to make every single person feel special.

"I know there are challenges ahead for me. I’m losing my mobility and with it, my independence, and I’ll need a transplant soon. But no matter what MDS throws at me, I know Geke will be there to reassure me.

"My message to Geke this Nurses’ Day can be summed up in a single quote:

"What we have done for ourselves dies with us.

What we have done for others and the world remains and is immortal."


You can still share your story and say Thank You to your haematology nurse ‪#‎thankanurse‬ on our Facebook page!

Apply to review Cancer Research UK research centres


Cancer Research UK are recruiting a patient panel to review their research centres and you can apply to be a member.

Cancer Research UK funds 18 experimental cancer medicine centres and gives support to 15 more research centres across the UK. These centres are essential to beating cancer sooner.

All of these centres will apply for five years funding. The applications are going to be reviewed by an expert committee. But they also want people affected by cancer to provide feedback on the centres, so they are creating a Patient Panel.

If you apply to be in the Patient Panel:

  • You will review applications and send your comments
  • You will be attending a Patient Panel meeting in London.
  • You will be discussing and scoring each application together with the other members of the Panel.

Do you have a keen interest in research?
Are you willing to read information to develop your understanding the work of Cancer Research UK and the four UK Health Departments?
If you are please apply to take part in the panel.

To apply please email to request an application form and return it before 17th June 2016

Read the full article here.


New MDS Patient Handbook

17th May 2016

Being diagnosed with myelodysplastic syndrome (MDS) can be a shock, particularly when you may never have heard of it.

MDS UK has now produced a brand new MDS patient booklet, created in cooperation with our colleagues from Leukaemia Care and Bloodwise.
It has been written to help you understand more about MDS. It describes what they are, how they are diagnosed and treated and also the expected outcome (prognosis). It also provides information on coping with the emotional impact of an MDS diagnosis.

Whilst the covers are different, the content is the same.
All 3 organisations will work together to ensure this booklet is available in all hospitals.

At MDS UK, we felt there was an urgent need to revise and replace the existing information material.
We also wanted to ensure that as many patients as possible would benefit from this in-depth booklet, and hence asked those two great organisations to join us in this project. It is highly necessary and beneficial to all patient groups to work together whenever possible, and save valuable funds that way.

We sincerely thank:
- our colleagues – Leukaemia Care and Bloodwise
- the contributing authors for the different chapters – Dr Sally Killick, Dr Dominic Culligan, Philip Alexander, nurses Janet Hayden and Geke Ong
- our peer reviewer – Prof David Bowen
- the patient reviewers Chris Dugmore and Claudia Richards

If you wish to obtain a copy of the booklet please click here or contact us by email or telephone: or 020 7337558

A stock of booklets will also be available at all of our local group meetings.

Understanding Mutations to Treat MDS

The most dramatic development in MDS research over the past few years has been the identification of the gene mutations that cause MDS. These mutations are not inherited, but are acquired in blood stem cells during the lifespan of a person.

Benjamin Ebert is researching these mutations. A major focus of his research is to determine how this genetic information can be used to improve the diagnosis of MDS, and to find the best therapy for each MDS patient.

Lenalidomide, for instance, is a powerful treatment for MDS patients with deletions of Chromosome 5q, a specific genetic mutation.

In the future the research could predict response to azacitidine and decitabine, only available in trials in Europe, and it might help to find out who is best suited for a transplant.

Some of these findings are only preliminary, and Ebert and his team are working to study larger groups of patients to make definitive predictions.

Not a cure but a lot of progress - all of the time, thanks to national and international researchers, and many clinical trials.

Read the full article here:

Take Part In This Survey And Help To Create Better Tools For Patients

Thanks to the latest improvements in cancer treatment many more cancer patients are now able to live with their condition.

As a chronic illness, cancer requires long-term watch, maintenance and treatment and patients and their families need to manage their own care.

The iManageCancer project is creating intelligent, fun new ways to allow patients to manage their lives from their mobile phones.

Now they are asking for your help.

This month, they have launched a survey aimed at healthcare professionals, patients and the general public to find out what they'd like to see if they used a personal health record online.

The survey is available to take by clicking here

Everyone can take part, and the more, the better; whether you are comfortable with technology, or not so much.

The results will help to create better tools to help people with cancer, so please to share this with everyone you know!


Evie McClean Starts an Exciting New YouTube Channel

Evie does You Tube – and she is a real pro!!! Go Evie! Have a look at her first video clip.

Evie is a member of MDS UK Patients Support. Here is Evie’s video clip with 10 facts about her. We hope she will do a great clip for MDS UK to talk about MDS and transplants.

For her birthday Evie received an unexpected personal greeting from non other than David Beckham! Watch the video of David Beckham congratulating Evie

Thank you so much to David Beckham and everyone for supporting Evie! MDS is a poorly understood blood cancer disease, leading to feeling isolated. It means a lot to all patients to be encouraged this way.

Evie is very active on social media. On top of her YouTube Channel, Evie also has a Facebook group.

Like our MDS-UK-Patient-Support-Group Facebook Page and you’ll get more updates on Evie and other MDS stories

Evie McClean

Evie McClean

Urgent – CDF solution for Blood Cancer drugs

nhs england - logoThe Cancer Drug Fund (CDF)

de-listing further drugs


Many blood cancer patients are about to face more severe issues of access to treatment, following the announcement that the CDF is de-listing further drugs in order to balance its budget.

Twelve of these are blood cancer drugs.
None of the cuts affect MDS patients, but the principle is at stake – and the future funding of all cancer drugs for all cancer patients.

The Blood Cancers Alliance, an informal group of all blood cancer charities, has now written to David Cameron and Jeremy Hunt to urge them to find a suitable solution to this wholly unacceptable situation.
The open letter is copied here and will be published in the Times newspaper today Wedn 4th Nov 2015.
Please share it widely, including your MP.
This letter is also appearing on all websites of the Blood Cancers Alliance members.

Open letter to Rt Hon David Cameron MP and Rt Hon Jeremy Hunt MP:

Blood cancer charities urge Government for Cancer Drugs Fund solution

As an alliance of blood cancer charities, and on behalf of the 27,000 blood cancer patients and their families who have signed the petition against the delisting of life-saving drugs from the Cancer Drugs Fund (CDF), we are writing to express our concern regarding the Government’s failure on the CDF.

The Government is aware of the flawed nature of the CDF, but due to politics, has let it progress to the situation where clinically effective treatments are today being removed without a long-term solution to access.

The CDF is majorly overspent and no data has been collected on whether or not any CDF-funded treatments have actually worked. As a result of these failures 12 blood cancer drug indications, previously deemed clinically effective, are set to be removed from the Fund with no guarantee of future access.

While the CDF has improved access to cancer drugs not routinely available in NHS England, it was always intended to be a temporary solution while a long-term pricing mechanism was worked out. The proposed consultation on the CDF has taken too long to materialise, and as the new CDF system is set to be in place from April 2016, the time is rapidly decreasing for stakeholders to shape a system that is fit for purpose.

Through the revised CDF and Accelerated Access Review, there is a real opportunity to put patients at the heart of the system and ensure they are able to access the most innovative medicines. This is an opportunity that the Government can no longer afford to miss.

Yours sincerely

Blood Cancers Alliance

Eric Low OBE
Chief Executive – Myeloma UK

Sophie Wintrich
Chief Executive – MDS UK

Sandy Craine
Chief Executive – CML Support

Roger Brown
Chair – WMUK

David Innes
Chair – CLL Support Association

Monica Izmajlowicz
Chief Executive – Leukaemia CARE

Jonathan Pearce
Chief Executive – Lymphoma Association

Cathy Gilman
Chief Executive – Bloodwise



The petition can still be signed by following this link – Don’t cut life saving blood cancer drugs
It is a 38 degrees action – which we highly recommend as an organisation.


About the Blood Cancers Alliance

About blood cancers

  • Every year around 34,000 patients are diagnosed with a blood cancer
  • The main blood cancer groups are leukaemia, lymphoma and myeloma, although there are over 130 types of blood cancer
  • Blood cancers account for around 1 in 10 cancer[1] diagnoses in the UK


Blood cancer drugs identified for delisting:

Drug Cancer
1 Bendamustine Relapsed mantle cell lymphoma
2 Bendamustine Relapsed chronic lymphatic leukaemia
3 Bosutinib Accelerated phase chronic myeloid leukaemia
4 Bosutinib Chronic phase chronic myeloid leukaemia (restricted to subgroup of patients significantly (grade 3 or 4) intolerant to nilotinib and dasatinib)
5 Brentuximab Relapsed anaplastic large cell lymphoma
6 Brentuximab Relapsed Hodgkin’s lymphoma
7 Dasatinib Philadelphia chromosome positive (Ph+) acute lymphoblastic leukaemia
8 Ibrutinib Relapsed mantle cell lymphoma
9 Ibrutinib Relapsed or refractory chronic lymphatic leukaemia (except where patients are contraindicated to rituximab and idelaisib or significantly intolerant to idelaisib)
10 Idelalisib plus rituximab Relapsed chronic lymphatic leukaemia
11 Lenalidomide Relapsed myeloma
12 Pomalidomide Relapsed myeloma

[1]NCIN Data briefing. 2013. Registrations for Blood Cancers in England.

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More background information about the Cancer Drug Fund

We have published several News Posts regarding the CDF on our website over the last 2-3 years:


Here is also the official NHS website information – via the NHS England – CDF website:

“On 1 April 2013, NHS England took on responsibility for the operational management of the Cancer Drugs Fund (CDF). The NHS spends approximately £1.3 billion annually on the provision of cancer drugs within routine commissioning. The CDF was established as an additional funding source to this.

The CDF has provided an additional £200m each year since then to enable patients to access drugs that would not otherwise have been routinely available from the NHS. NHS England recently pledged an additional £160m over the next two years to strengthen the fund. It was established in 2010 and will run until the end of March 2016.

There is a single, national list of drugs and indications that the CDF will routinely fund and standard operating procedures for administration of the fund.”

MDS World Awareness Day – 25th of October 2015

2015 MDS World Awareness Day: Video Stories

Some of our true champions for MDS awareness have shared their stories with us. Watch their video clips and share them in support of MDS World Awareness Day 2015!

Emma Paine: What happens when you need a second transplant?

Emma is an MDS patient.  She had a first stem cell transplant in 2011 – and recovered well.  Until now.

She is currently in hospital having chemotherapy and urgently needs a second transplant.
BUT – funding for second transplants is being revised in the UK – due to “cost-effectiveness” issues.
18 months ago, Emma would have received her potentially life-saving transplant without a problem.
Given the current NHS financial crisis, and new clinical data on the success of 2nd transplants after a relapse, the Dept of Health has tightened the rules relating to this procedure – and hospitals all over the UK need to seek special funding requests for those patients (IFR=Individual Funding Requests)

To date it is not certain whether Emma will receive the transplant or not.
Click here to follow Emma blog about her experience and her battle to be granted a further chance to live: Emma’s Fight against MDS

Raising awareness of mds day #mdsworldawarenessday #mdsukpatientsupportgroup

Posted by Emma Paine on Sunday, 25 October 2015

Olivia, Tilly and Ellis Hepburn make an appeal

Olivia, Tilly and Ellis, MDS super heroes and children of Lisa and Gavin Hepburn, are making an appeal for people to donate blood and stem cells to save lives of people like their Dad, who has MDS.

Lisa told us: “Today is MDS Awareness Day. Three years ago we hadn’t even heard of this disease. That’s why today is so important to make people aware. We are so grateful to the person that gave Gavin his donated bone marrow, and to the endless people that donate blood. To everyone that has helped, supported and been a shoulder to cry on these past years we are so grateful to you too…”

Their wonderful clip has already been viewed over 12,000 times on Facebook. Please keep sharing it! Thank you – on behalf of the entire MDS community.

Today is MDS Awareness Day. Three years ago we hadn’t even heard of this disease. That’s why today is so important to make people aware . We are so grateful to the person that gave Gavin his donated Bone Marrow, and to the endless people that donate blood. To everyone that has helped, supported and been a shoulder to cry on these past years we are so grateful to you too …

Posted by Lisa Hepburn on Sunday, 25 October 2015




5 ways you can help to raise awareness about MDS

We are asking all patients, families, clinical staff, supporters to post photos or short video clips of themselves on all social media platforms, to raise awareness of this rare blood cancer MYELODYSPLASTIC SYNDROME.



wad poster 2015


All our colleagues and friends worldwide will be doing the same and will post messages online on the 25th October. See our Facebook page for more clips, and photos and the MDS Alliance website for links to all international groups marking MDS Day.

1. Record and share a video clip. You can use the message below as an example:

Hello – my name is ……. I am an MDS patient.
(or I am an MDS Supporter – son/daughter/spouse/brother/sister/parent/friend/colleague/neighbour of an MDS patient).
Please share my message in support of the rare blood cancer MyeloDysplastic Syndromes – and the MDS World Awareness Day on 25th October.
Please support the MDS UK Patient Support Group and the international MDS Alliance.
Help to register more stem cell donors and blood donors.
Be a star.
Thank you.

2. Share these messages of MDS patients and supporters with your family and friends

It’s #MDSWorldAwarenessDay tomorrow. Please click on my video to see how you can help! #MDSUKPatientSupportGroup

Posted by Jenny Milne on Saturday, 24 October 2015



3. Share all photos and videos using the hashtag #MDSworldawarenessday



4. Organise a coffee morning or tea afternoon to show your support and use the posters below

PDF poster MDS WAD POSTER A4 Oct2014

PDF version: WAD2015

Take your photo with the posters clearly visible!


5. Share your photos with the poster. Some lovely ones below



WP_20151002_054web   WP_20151002_052web











World Awareness Day 2015


World Awareness Day 2015










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