this is archive.php

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 .

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:

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

A New Edition of the MDS Patient Handbook is out!

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 new edition of the MDS patient booklet, created in cooperation with our colleagues from Leukaemia Care and Bloodwise.

At MDS UK, we felt there was an urgent need to revise the existing information material. We 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.

This booklet has been written by Dr Sally Killick, Consultant Haematologist; Dr Dominic Culligan, Consultant Haematologist; Philip Alexander, Counsellor and Cognitive Behaviour Psychotherapist; Geke Ong and Janet Hayden, Clinical Nurse Specialists; and peer reviewed by Professor David Bowen, Honorary Professor of Myeloid Leukaemia Studies and Consultant Haematologist, St James’s Institute of Oncology. The booklet has also been reviewed by patients and we are grateful to Chris Dugmore and Claudia Richards for their valuable contribution.

The booklet 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.

New Edition! Learn more about MDS with Our MDS Patient Handbook

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 is also available at all of our local group meetings.

Vidaza / Azacitidine: Know more about this drug

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

Are Vidaza and Azacitidine the same drug? Why does it have two names?

Vidaza and Azacitidine are the same drug

Azacitidine is the official generic and non-proprietary name given to the drug active ingredient while Vidaza is its trade name.

The drug is widely available in the UK for the treatment of MDS since its approval in February 2011 by NICE - the National Institute for Health and Clinical Excellence (NICE). MDS UK Patient Support Group was in consultation with NICE during the approval process and campaigned for the approval of drug.

Is Azacitidine (Vidaza) a chemotherapy?

Azacitidine is a chemotherapy drug, however, it is a "hypomethylating agent". Hypomethylating agents are considered a non-intensive treatment.

They are aimed at slowing the progression of the disease with as few side effects as possible, maintaining a good quality of life. They will not cure MDS but may ‘modify’ it.

How does Azacitidine work?

Azacitidine works at the DNA level, "switching on" genes that stop the cancer cells growing and dividing. This reduces the number of abnormal blood cells and helps to control cell growth.

It is recommended as a treatment option for adults who are not eligible for stem cell transplantation and have intermediate-2 and high-risk myelodysplastic syndromes according to the International Prognostic Scoring System (IPSS) (See more at Nice Website).

How is Azacitidine administered?

You have azacitidine as an injection just under your skin (subcutaneously) given by a doctor or nurse. This can be in your upper arm, leg, buttock or stomach. You usually have it as an outpatient but there have been successful experiences with home administration.

You will have azacitidine as a course of several cycles of treatment. You can a treatment each day for a week and then 3 weeks with no treatment. This makes up a treatment cycle. You normally have at least 6 cycles and the treatment continues for as long as it is working.

You can also have it every day, for 5 days. Then have 2 days off and have azacitidine again for 2 days at the start of the next week.

The Treatment Involves Several Courses

The Treatment with Vidaza/Azacitidine Involves Several Courses
Joseph Bella: MDS patient experience of Azacitidine and Bone Marrow Transplant

Joseph Vella: MDS patient experience of azacitidine and stem cell transplant

"I hope and trust that my little story could encourage other MDS sufferers to decide to go through with the treatment"

Azacitidine (Vidaza): Latest research

Azacitidine is an important drug for the treatment of MDS but not all patients will respond to the treatment. Some will progress to leukaemia, others won't have an adequate response in terms of their blood count.

At the 14TH INTERNATIONAL SYMPOSIUM ON MYELODYSPLASTIC SYNDROMES, that took place in Valencia in May 2017, one of the topics discussed was the latest research around this important drug. Read more about how researchers are trying to establish which patients will have a good outcome with Azacitidine and whether the use of Azacitidine together with other drugs can make the treatment more effective.

Learn more about the latest MDS research: Download the full MDS Symposium Patient Summary

Predicting Good Outcomes with Vidaza/Azacitidine Treatment

Researchers are exploring which factors might be useful for predicting a good outcome in patients with MDS.

Certain treatment decisions for patients with MDS are based on cytogenetics, i.e. the study of the chromosomes. Azacitidine is appropriate for patients with higher risk MDS who often have mutations in chromosome 7 or three or more abnormalities in their chromosomes.

Dr. Raphael Itzykson, Université Paris Diderot, France, presented a study showing that if the platelet count after one cycle of the drug doubles, this is a good sign for an overall success of the treatment. However this happens in only a small proportion of patients.

Several ongoing efforts, including the HARMONY study, have a good chance of identifying more factors that predict azacitidine outcomes in MDS and other blood cancers and of predicting the effects of treatment on the patient's quality of life, healthcare costs, and care strategies.

The Best Partner for Vidaza/Azacitidine in Higher-Risk MDS

Dr. Mikkael Sekeres (Cleveland Clinic, Ohio) focused his presentation on combinations of Azacitidine and other drugs for higher-risk MDS.

The combination of Azacitidine and vorinostat seemed promising. In a phase II clinical trial, about 70% of patients with untreated higher-risk MDS, CMML, or AML responded to the treatment, which was about double the expected rate for azacitidine alone. These responses lasted an average of 16 months.

Similarly, response rates and duration of response were promising in a phase I-II clinical trial of the combination of lenalidomide and azacitidine for higher-risk MDS.

Dr. Fenaux added that studies are also evaluating combinations of Azacitidine with other treatments, such as valproic acid, venetoclax, immune checkpoint inhibitors, and idarubicin for higher-risk MDS or CMML.

Establishing The Correct Dose

Other research is assessing more intensive hypomethylating treatments or lower doses for longer use. Studies are testing different drugs, including venetoclax, cenersen, and a 10-day decitabine cycle (another hypomethylating agent) for MDS with TP53 mutations.

Dr. Sekeres concluded that azacitidine alone is still the standard treatment for higher-risk MDS. But some evidence
hints at better and more long-lasting responses for combination treatments if patients stay on them long enough. The hypomethylating drug “partners” under investigation might become options for higher-risk MDS in some patients.

Clinical Trials open to recruitment in the UK

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

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

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 the start of 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 will be a brand new type of NHS service opening for MDS patients, in April 2019, 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.

Starting date for this service:

Video consultations will be available from April 2nd 2019, generally on Wednesdays but with some flexibility.

Who is it for?

Any MDS patients who would like a specialist MDS consultation but who are unable to, or prefer not to travel to Leeds, or another Centre of Excellence for a conventional clinic appointment.
This service will be 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 feel free to contact us directly at MDS UK - - Tel 020 77337558

Or send an email to Professor Bowen’s secretary,

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

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"

What is CAR-T cell therapy? Learn how this immunotherapy works in blood cancer

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

Car-t cell therapy aims to boost the immune system to attack tumor cells

Cell therapies, sometimes called “living therapies", are an especially promising and rapidly growing area of cancer research.

One approach that’s been pioneered by Memorial Sloan Kettering researchers, led by investigator Michel Sadelain, is called CAR-T cell immunotherapy. This type of targeted immunotherapy aims to boost the immune system by giving immune cells the information they need to better recognize tumor cells as foreign and attack them.

How does Car-t therapy work in blood cancer?

The technique involves filtering white blood cells called T cells from a patient’s blood and introducing a new gene into those cells. A disabled virus called a vector is used to carry this new gene inside the T cells and insert it into the cells’ genomes.

The gene programs the T cells to make a chimeric antigen receptor (CAR), which enables them to recognize a specific protein that’s present in cancer cells. These CAR-T cells are then grown in the laboratory and infused back into the patient, where they seek out and destroy the cancer.

CAR T cell therapy is currently being evaluated in the clinic at MSK for certain types of leukemia and lymphoma.

In this approach, T cells are genetically engineered to recognize a protein called CD19, which is found on the surface of blood cells called B cells.

In the largest study reported so far, for adult patients with B cell acute lymphoblastic leukemia — a rapidly progressing form of blood cancer — a report published by MSK researchers last year found that 88 percent of patients responded to the therapy.

In late 2014, the US Food and Drug Administration granted MSK Breakthrough Therapy Designation for its CD19 CAR therapy. In August 2017 the FDA approved a Novartis Car-t therapy, called Kymriah (tisagenlecleucel), for children and adults up to age 25 with B cell who have not responded to conventional therapy or who have relapsed. In October 2017 it approved Yescarta (Axicabtagene Ciloleucel), another cell-based gene therapy, to treat adult patients with certain types of large B-cell lymphoma who have not responded to or who have relapsed after at least two other kinds of treatment. Yescarta, a CAR-T cell therapy, was the second gene therapy approved by the FDA.

The science behind it

  • A chimeric antigen receptor (CAR) helps T cells identify tumors.
  • These T cells then recognize blood cancer cells as foreign and attack them.
  • CAR T cell therapy is being used to treat leukemia and other cancers.

Learn More:
Science: Aug. 30, 2017
Modified T cells that attack leukemia become first gene therapy approved in the United States
FDA: Oct. 17, 2017
FDA approves CAR-T cell therapy to treat adults with certain types of large B-cell lymphoma
Cancerworld: Nov. 26, 2018
The Car T cell revolution: what does it offer, and can we afford it?
Science Daily: Dec. 3, 2018
New strategies may improve CAR-T cell therapy

Take a look at current MDS clinical trials

MDS UK is recruiting!

Would you like to be part of our team?

MDS UK Patient Support Group is looking for two new employees, to assist with the running of the charity.

We are a small national charity, offering assistance to patients and families who are diagnosed with the rare blood cancer Myelodysplastic Syndromes (MDS).

We are based at King’s College Hospital, which is one of the main expert hospitals for this disease. King’s College Hospital is located in South London, in Camberwell.

We are a well-known organization in the field of haematology, and we work with all main clinical experts working in MDS.

This is a small team of hard-working individuals, working closely with local volunteers from all walks of life, and varying degrees of IT competence.

Patience, understanding, a caring attitude and a sense of humour would help. The work is rewarding, and offers opportunities to expand the roles in future.

The main position offered is for an Office Manager – who would also be looking after our registrations, member’s database and general needs of our local groups. This is a part-time role, for 3 days/week, offered on a freelance basis.

The second post offered is for an Administrative Assistant, to help out with the Membership request and processes, as well as administration of our local groups. This is a part-time role, for 2 or 3 days/week, offered on a freelance basis. Previous experience of working within the charity area, or within a medical environment would be helpful, but not essential.

The main requirements for these posts are:

  • Excellent computer skills, excellent organizational skills, good IT skills
  • Ability to prioritise workload and work proactively, with minimal supervision
  • Excellent communication skills, written and oral
  • Attention to detail essential
  • Clear understanding for absolute patient confidentiality
  • An interest in the field of health, cancer, patient support is a prerequisite

To download a full job description, please click here: Office Manager or Admin Assistant

Salary will depend on previous experience and skills

Office Manager: £25,350.00- £29,250.00 pro-rata, dependent on experience and skills
Admin Assistant: £19,500.00 - £20,475.00 pro-rata, dependent on experience and skills

These posts are available from mid-February 2019 – and will be available long-term.
For general enquiries, please call 020 77337558

To apply, please send a CV and covering letter, stating why you would like to work for our charity to

PANTHER: Clinical Trial Open to Recruitment

Research FOR Patients
-For an informed and empowered opinion-

All the trials listed in our site have been properly vetted for scientific accuracy. Many thanks to Dr Simone Green – Hull and East Yorkshire Hospitals NHS Trust - for the continuous work in updating the listing.


  1. SUB-TYPE OF MDS:Higher risk MDS, CMML, low blast count AML
  2. SEVERITY OF MDS: Intermediate, high risk or very high risk MDS as defined by the World Health Organization (WHO) criteria or Revised International Prognostic Scoring System (IPSS-R)
  3. NAME OF DRUG: Azacitidine, Pevonedistat
  4. Aims and benefits: Pevonedistat prevents the activity of a specific enzyme (Nedd8 activating enzyme) and thus may result in the inhibition of tumour cell growth and survival. This is a phase 3 study to determine if combining Pevonedistat with Azacitidine improves survival when compared with single agent Azacitidine.

Read More

BRIGHT: Clinical Trial Open to Recruitment

Research FOR Patients
-For an informed and empowered opinion-

All the trials listed in our site have been properly vetted for scientific accuracy. Many thanks to Dr Simone Green – Hull and East Yorkshire Hospitals NHS Trust - for the continuous work in updating the listing.


  1. SUB-TYPE OF MDS:Previously untreated higher-risk MDS, AML or CMML
  2. SEVERITY OF MDS: Intermediate, high risk or very high risk MDS as defined by the World Health Organization (WHO) criteria or Revised International Prognostic Scoring System (IPSS-R)
  3. NAME OF DRUG: Glasdegib, Azacitidine
  4. Aims and benefits: PGlasdegib disrupts cancer stem cell survival. This has the potential to reduce the development of drug resistance and prevent relapse. This is a Phase 1b study aimed at determining the safety and effectiveness of Glasdegib in combination with Azacitidine in this specific patient group.

Read More

Free donations by shopping