Missed the Birmingham MDS Patients & Family Forum? Catch Up Online

The latest MDS UK Patient and Family Forum took place in Birmingham on the 4th of February.

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

MDS Families and Patients Forum 4th February

Those attending the forum took part in a variety of workshops and sessions delivered by experts and were invited to ask questions on the most pressing issues, from how to manage fatigue to newly available treatments.

The list of speakers included Peter James, Clinical Nurse Specialist at Worcestershire Acute Hospitals NHS Trust, Susan Smith, Research Nurse at the Kings Mill Hospital, Sutton in Ashfield and Dr Justin Loke, Haematologist, QE at Birmingham, among others.

Take a look at the full agenda and the photos of the event here

Read The Forum Presentations

MDS Patients Forum: A Run-through

What happened in Birmingham? Everything you need to know, from the welcome notes to the credits.

Download PDF

Clinical Trials: Potential Benefits and More

Why entering a Clinical Trial? Get to know more about the process and potential outcomes

Download PDF

Myelodysplastic Syndromes and their Treatment: an Update

Refresh your knowledge on MDS and get the latest on treatment options

Download PDF

Don't miss our next forum: contact us to get invited. Telephone: 020 7733 7558 Email: mds-uk@mds-foundation.org


Quality of Life is an Important Treatment Goal in Patients with MDS

In this presentation from the 2016 "European Focus on Myeloproliferative Neoplasms and Myelodysplastic Syndromes", Dr. Fabio Efficace discusses the importance of quality of life as a treatment goal in myelodysplastic syndromes (MDS)

Dr. F. Efficace discusses importance of Quality of Life as Treatment Goal in MDS

When latest genetic analysis and therapeutic strategies are incorporated into MDS treatment the outcomes and quality of life in patients with MDS improves.

Recent research trials have incorporated the opinion coming from MDS patients themselves about their own quality of life to improve the assessment of several treatment options.

These changes to refine and strengthen diagnostic and risk-assessment models, along with the rapid evolution of disease management strategies that use standard of care and novel therapeutic agents, will likely further enhance clinical outcomes in patients with MDS and improve their quality of life.

Read Latest Research


Researchers find new way to target blood stem cell cancers

Researchers designed an antibody that recognises and destroys CD99-covered leukemia cells while sparing normal blood stem cells

Leukaemia Cell

This is a microscopic image of a leukemia cancer stem cell (Credit: Montreh Tavakkoli) with normal DNA coloured in blue. CD99, those green-coloured spots, is a protein-sugar molecule, which occurs more frequently than normal on stem cells responsible for blood cancers, including acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS).

Building on this discovery, researchers from NYU Langone Medical Center and Memorial Sloan Kettering Cancer Center designed an antibody that recognises and destroys CD99-covered leukemia cells while sparing normal blood stem cells, a finding confirmed by experiments in human cells and in mice with AML cells.

Antibodies are immune system proteins that stick to a specific target. In recent years, researchers have become capable of engineering antibodies so that they target disease-related molecules.

"Our findings not only identify a new molecule expressed on stem cells that drive these human malignancies, but we show that antibodies against this target can directly kill human AML stem cells," says corresponding study author, Christopher Y. Park, MD, PhD, associate professor in the Department of Pathology at NYU Langone and its Perlmutter Cancer Center.

"While we still have important details to work out, CD99 is likely to be an exploitable therapeutic target for most AML and MDS patients, and we are working urgently to finalize a therapy for human testing," says Park.

Direct Cell Killing

Acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS) arise from abnormal stem cells that build up in bone marrow until they interfere with normal blood cell production. Patients struggle with anemia, increased risk for infection, and bleeding.

In AML, a small group of leukemic stem cells become incapable of maturing into red or white blood cells as intended. Most leukemias respond initially to standard treatment, but relapse is common as standard treatments fail to kill leukemia stem cells, which continue to multiply.

The researchers examined stem cells from 79 AML and 24 MDS patients and they found that approximately 85% of stem cells in both groups had high levels of CD99. The levels were so high that diseased stem cells could be cleanly separated from related, normal stem cells in AML patients.

The research team then made several CD99 antibodies, and chose to focus on the one that most effectively killed those cells. Researchers found that when the study antibody attaches itself to CD99 on the surface of a cancer stem cell, it causes leukemia stem cells to die.

"With the appropriate support, we believe we can rapidly determine the best antibodies for use in patients, produce them at the quality needed to verify our results, and apply for permission to begin clinical trials," says Park.

Read More

More information: "CD99 is a therapeutic target on disease stem cells in myeloid malignancies," Science Translational Medicine stm.sciencemag.org/lookup/doi/10.1126/scitranslmed.aaj2025

Provided by: New York University School of Medicine

Read Latest Research


How successfull can a clinical trial be? By Shirley O’Brien

How a clinical trial gave me my life back after MDS and AML

Five years ago, my spouse and I had settled into our dreamed-of retirement. But on Feb. 6, 2012, I was diagnosed with myelodysplastic syndrome (MDS). Because I was in my early 70s, a bone marrow transplant wasn’t my best option.

Shirley O’Brien

What happened when the chemo stopped working

I received chemo infusions for seven days every 28 days to improve my bone marrow and blood cell function. But after nearly 3 1/2 years of this, I learned the chemo was no longer working.

A subsequent bone marrow biopsy demonstrated progression of my MDS, with the identification of an IDH1 mutation.

I sought a second opinion and got a grim prognosis. The oncologist gave me only five to seven months to live. He said I needed to find a clinical trial soon.

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.

Choosing a clinical trial at MD Anderson

During my search, I learned about a Phase II clinical trial at MD Anderson using an experimental drug called AG120. About a week after I applied, Courtney DiNardo, M.D., asked me to travel from my home in Tucson for testing.

Between MD Anderson’s huge campus and the battery of medical tests, our first visit was overwhelming. Yet, when Dr. DiNardo entered the room, she immediately made us feel like we were long-time patients or even friends. She was so cool, young and confident.

Only 24 hours after my spouse and I returned home, Dr. DiNardo called and asked us to return right away. We canceled our holiday plans, packed our motorcoach and arrived in Houston on Dec. 12, 2015.

At MD Anderson, we learned that my MDS had progressed to acute myeloid leukemia (AML). This was shocking, but I felt a strong sense of hope. We were right where we needed to be. People come to MD Anderson from all over the world, and I was grateful to be there with so many other AML patients.

On December 23, I took my first pills for the clinical trial. Then came endless EKGs and every-other-day blood tests to check my blood cell counts.

My amazing AG120 results

Two weeks into the clinical trial, my white cell count was higher than it had been in two years. My spouse and I were amazed.

But the biggest surprise was my blast count. When I’d arrived at MD Anderson, it was at 30% — extremely high. At the end of the first 28-day cycle, it was just 2%, which is normal.

Unlike chemo, which tries to kill the blasts and everything else in the bone marrow, AG120 blocks the mutant IDH1 protein that caused my AML. It allows the blasts to mature properly into normal white cells of the immune system. The bone marrow is no longer crowded out by AML, and the normal red cells and platelets return, too.

I’m now beginning my 12th cycle of AG120, and my blood values, red blood cells, white blood cells and platelets have all reached normal range. I’m in complete remission, but I will continue to take AG120 indefinitely. Whenever Dr. DiNardo’s team asks about side effects, I can’t come up with anything.

I am so grateful and praise God every day for giving me my life back through the AG120 clinical trial.

I used to always say you have to be your own advocate because no one else will. But I was unable to take charge of my cancer until I met Dr. DiNardo. I’ll always remember what she said the day before I enrolled in the clinical trial: “You are in the right place at the right time with the right mutation.”

Take a look at current MDS clinical trials


ASH 2016: Promising developments for MDS patients

Dr. Garcia Manero, from The University of Texas MD Anderson Cancer Center, highlights some promising clinical data presented at the 58th Annual Meeting of the American Society of Haematology (ASH)


Young patients denied second chance for life by NHS England

Blood Cancer – the issues around the highly complex topic of 2nd stem cell transplants.

Early in 2016 a MDS patient was denied a potentially life- saving 2nd bone marrow transplant despite showing a good chance of being cured by this 2nd treatment.

After 4 months of waiting, spent in and out of hospital, while NHS England turned down the treatment again, following an appeal, this patient contracted a severe infection and died.

He was only 39 years old – and a father of 3 young children.

End of 2016:

A mum with 2 young children is finding herself in a similar situation. She needs a 2nd transplant after a relapse of her blood cancer, Acute Myeloid Leukaemia.

The request for funding was denied just recently – meaning she has 2 choices: find the £100k herself to pay for the transplant or accept she may only have months left to live.

What is the background behind these appalling situations?

At the end of 2013, NHS England took the decision to stop funding allogeneic stem cell transplants (allo HSCT) for blood cancer patients who suffered a relapsed of their disease – after an initial successful transplant.
Expert clinicians requested a review of this decision, which did happen.

But in July 2016 NHS England came to a decision and stated they would no longer routinely fund 2nd allo HSCT’s – based on the evidence and success rates. These treatments were no longer deemed to be “cost-effective” for the current financial situation of the NHS.

It is true that overall, outcomes of 2nd transplants are not great – on average.
But transplants, types of blood cancer, sub-types, patients are very unique and these situations are rarely comparable.
Not only is the need for a 2nd HSCT rare, but not many patients would be so eager to undergo a 2nd transplant. Nor would haematologists be willing to perform one if there was not some good chance it would save someone’s life.
Clinicians and patients understand the issue of quantity versus quality of life.

Ever since 2013, haematologists deciding their patients would benefit from a 2nd HSCT, would have to make a compelling case for it – via an IFR (Individual Funding Request) which would then be submitted to an NHS England IFR Panel.
The IFR panel would then discuss whether the case was exceptional enough to agree the funding.

What does that mean in practice and in actual figures?¹

A bone marrow transplant costs about £100k per patient on average.
Between April 2013 and June 2016, only 19 requests for a 2 nd HSCT were made in all of England:

5 were declined by the IFR Screening team (the case was never seen by the IFR Panel)
5 were declined by the IFR panel
3 had “other outcome” (sic - as per NHS FOI answer)
6 were approved – this means just 2 cases per year. Total cost of approx £200k.

The 2 recent patients mentioned have been denied this chance – to save NHS England £200k.

It is crystal clear that the NHS cannot afford to treat everyone whatever the cost – so let’s set aside the
emotional and humanity argument.

It is the money side and management of NHS funds we take issue with.

NHS England refuses to consider, or key budget holders seem unable to take into account the cost of keeping these patients alive with supportive care – or palliative care.
The patient who died ended up costing the NHS closer to £250k – just to keep him alive for 5-6 months before his death – between frequent hospital stays, tons of antibiotics, expensive new medication, frequent complex blood and platelet transfusions.

A saving of £100k = an NHS bill of £250k + a death
This is not tax payer’s money well spent

Furthermore – we have uncovered that despite NHS England’s explanations, IFR cases are not assessed by transplant specialists, but by a mix of “trained” IFR staff, pharmacists and public health consultants or another random types of physicians.
There is no process forcing IFR staff to submit highly complex transplant IFR’s to relevant experts in that field.
There is an ongoing NHS England consultation on this very topic closing on 15/01/17.

Anthony Nolan, together with the Blood Cancer Alliance community and leading experts, published an open letter to the Secretary of State for Health (Times, July 2016) and led a significant campaign – but to no avail:

www.anthonynolan.org/news/2016/07/14/our-letter-following-nhs-england-announcement-funding-second-transplants

“We believe this is simply wrong. If a stem cell transplant is the best option for a patient, and a donor can be found, we believe the treatment should be available. We know that there are many people alive today, leading fulfilling and active lives with their families, because they had a second transplant.”
(Anthony Nolan campaign extract).

¹ Figures obtained through Freedom of Information Act enquiries – and from medical notes shared by the family

Below are the full stories of both families. 

This makes for very difficult reading.

We urge the government to review this nonsensical and inhumane situation.

There are cases where patients do stand a good chance to have a successful  2nd transplant – but these decisions must be made by expert haematologists – not public health physicians, let alone non-clinical staff – however well trained.

The savings are a false economy, as evidenced in this case.
Supportive care ends up costing twice as much, patients occupy badly needed hospital beds, families lose a bread-winner and government a tax-payer.

This makes no sense.

Sasha Jones – who urgently needs a transplant – but NHS England says NO

Dec 2016

I am a fun loving 34 year old nail artist from Greenwich, London. I am the wife of my childhood sweetheart Lloyd Williams and
Mother to our 13 year old daughter Katia and 8 year old Son Lj Williams. I am fighting for a second bone marrow transplant. I am
Sasha Jones (Williams) and I deserve to be alive just like you.

After a routine blood test on the morning of March 11 th 2015 due to me feeling unwell, I was sadly given the devastating news that
the tests suggested I was suffering from Acute Myeloid Leukaemia and was admitted within hours.

On Sunday 15 th March I started my chemotherapy treatment and a bone marrow aspirate revealed that the only chance I had of keeping the disease at bay was to have a bone marrow transplant. So the search begun to find a donor and my treatment commenced.

After my first round of chemo it was revealed the amazing news that I was in complete remission and had 3 donor matches!
3 rounds of chemo over a 5 month period later and I was admitted to Kings College Hospital for my graft. On 26th August 2016 and after 1 week of high intensity chemotherapy treatment I was given the bone marrow transplant I so desperately needed in the hope my leukaemia would stay in remission.

After 18 days in hospital, lots of blood treatment, pain management and rest I was relieved to seem well enough to go home to my husband and children. However, this was short lived when I was re-admitted 3 days later after Kings discovering the activation of a virus passed on through my new donor. I then spent another grueling 14 days in hospital trying to get rid of the reactivated virus only to be released home again to start my recovery.

But, yet again after another 2 days at home I was crucifying readmitted once more for yet another 14 days due to another virus called BK 2000 causing haemorrhagic cystitis. This left me with months and months of grumbling bladder pain (which I still have too this very day every time I urinate) and incontinence which made the recovery process a very uncomfortable, embarrassing and painful process.

By the beginning of October I returned home and managed to then stay home with medication and 2 visits a week to Kings College Hospital to monitor my progress. On top of all this my donor and I did not have the same blood type which also caused problems up until as recent as August 2016 when my blood changed to a type A+ from my original O+.

During 2016 I visited the hospital on a weekly basis and it started to look like everything was going right.
My recovery was long and hard but I never let it get me down, I had to do it for the children!
I could see the light at the end of the tunnel with my 1 year bone marrow milestone approaching on 26th August 2016!
Sadly my happiness was short lived...

On Tuesday 9th August 2016 after a routine blood test again I got the devastating news that my new bone marrow was now producing leukaemia cells and that the transplant had failed.
This was confirmed by a bone marrow aspirate result on August 23rd 2016, I had relapsed, the dreaded cancer had returned and I needed to start my battle all over again beginning with chemotherapy followed by an infusion of donor cells once in remission.
I was to be put forward for new clinical trials using donor cells and a vaccine to give me any hope of surviving without a second transplant as this option had been stripped from patients due to NHS cut backs. NHS England had announced that it would not routinely fund second stem cell transplants for patients with blood cancer or blood disorders who have relapsed.

I was then hit by the news that not only was NHS England denying patients these life-saving 2nd transplants, but that also my donor was no longer available to offer an infusion of cells or to assist with the completion of the trial.

I have therefore been left with no alternative than to have a 2nd bone marrow transplant and I’m currently waiting on a decision from NHS England as to whether they are going to provide me with the funding I so desperately need to save my life.
Given that they cannot offer me an alternative end to my treatment as my donor has been removed from the register due to circumstances unbeknownst to me, it is believed that under these exceptional circumstances (seeing as my life itself isn’t important enough) that I SHOULD be entitled to another bone marrow transplant to give me a chance of fighting the disease and beating it with a better matched donor that I have waiting for me right now.

I am young, in the prime of my life, have everything going for me and everything to live for, not to mention my 2 children who deserve the right to grow up with their mother. They have also the right to the love, support, care and stability that only a mother’s love can give.
We as a society and nation try only too hard every day to promote and push - only to take that chance away from 2 children for a mere £120k by not handing their mum a life line that they have such easy access to......

Another 2 children’s lives do not need to be destroyed, cancer already takes so many of us.
Why are we now giving up on us....
When did we stop trying to save lives over money but at the same time we put money into research to save lives that you then later let die...

#fingercrossedforsasha  #rideforsasha
https://www.gofundme.com/rvg4m54m

MDS UK update: Sasha has been refused the 2nd transplant by NHS England.

Thanks to your help her private fundraising has reached about £110k and the funding of stem-cell transplants was discussed in Parliament! This is a huge step in the right direction. Please keep sharing her story and sign the petitions to make Sacha the last patient that needs to pay for a 2nd transplant.

Family of Gavin – RIP

This is a summary of events – from diagnosis in 2012 – through to Gavin’s passing on the 6th May 2016.
Written by Lisa, his wife.

Nov 2012: Gavin gets his MDS diagnosis. Devastated and confused we are told he will need a bone marrow transplant in the new year.

June 2013: Gavin receives his first transplant and is home in little over 2 weeks. We are all elated and pray this is the new beginning for us all.

Oct 2013: Gavin is taken back into Kings for an infection that is destroying his red blood cells. Extremely ill his life is quite literally in the balance. But he fights it and is home and well within 3 weeks.
In between the above and September 2014 Gavin has top up transfusions of red blood cells and platelets (but rarely at this point).
The doctors are pleased and allow us all to travel to the USA for a month’s holiday.
Gavin was the healthiest he had been in a long time and we had a wonderful time.

Sept 2014: Gavin becomes unwell. A Bone Marrow Biopsy suggests that he may have relapsed.
A course of Chemotherapy is advised. We are all devastated but remain positive.
Gavin's condition is very up and down between September and December and he is taken into Kings before Christmas with another infection.
The Rollercoaster that is MDS is truly devastating to us all.

2015: Not great - highs and lows, lots of infections and stays at Kings and more Chemo, that had to be stopped.

June 2015: Gavin is needing regular red blood cell and platelets transfusions. A planned holiday to Menorca is cancelled due to Gavin's condition. We head to Devon for 3 weeks, but the holiday is full of interruptions as Gavin needs regular transfusions at Plymouth Hospital. He remains positive though, but we are dreading the thought that another transplant would be needed.

November 2015: We receive the devastating news that Gavin will require another transplant. His condition at this point is very up and down with infections putting him back into Kings at regular intervals, again, in the run up to Christmas. At this point he is also living his life going backwards and forwards for transfusions at our local hospital twice a week. This is becoming very draining on him and us as a family. But we remain positive.

Feb 2016: We receive the devastating news that Gavin's funding for transplant has been refused.
How? Why?
We arrange a meeting with our MP Nicholas Soames. He is very sympathetic and agrees to bring up Gavin's case with parliament.
Ironically that evening an ambulance is called as Gavin is taken ill with another infection.
We vow to raise the money, but are advised that another application for transplant will be put forward.
We feel like we are not doing anything to get this ball rolling - this is his life and we are desperate.Gavin's condition at this point is up and down, and he is struggling to remain positive.

April 2016:  After a wonderful day at the Isle of Wight, Gavin gets rushed back into hospital, East Surrey, our local hospital. It's the start of the 3 day doctor’s strike. We are all scared / devastated that this is happening again. On arrival there, we are told he will get the best care . How wrong were we. Gavin waits 8 hours for a simple bag of blood, he is climbing the walls in pain.
The doctors diagnose Pleurisy. He is put onto morphine for the pain. Gavin stays there for 3 days, and is not put onto the Kings Care plan until that 3rd day (when the doctors return back to work). At our total insistence as a family we get him transferred to Kings.
Dr Raj advises he needs this transplant but he needs to get well first. But he won't be leaving without it (hope again).
Gavin's condition deteriorates. He has a Lung Wash to clear the infection, the worst thing I've had to sit and listen too, I just wanted to run in and make them stop.
He improves a little and seems brighter. The morphine though has made him confused about things (very upsetting for myself and the children). It has also made him very constipated and blocked.
He can barely eat or drink.
It just feels like we are going 1 step forward 10 steps back.
The children just want their fun loving Daddy home and I my husband.

1st May 2016: Gavin gets told that there is nothing more they can do... we both can't believe it and won't believe it.
We put ourselves in a bubble and choose to ignore. Go to Costa and talk about anything but THAT.

2nd May 2016: Take the children to see him, very upsetting. He could barely be around them for no more than 15 mins.
Not like Gavin at all. He begs me not to leave that night.

Tuesday 3rd May 2016: I'm met by the palliative team at Kings. There is nothing more that can be done but to make him comfortable. I feel like my whole world has been blown up in front of me. My children, how do I tell them? We call all our family and friends. There is a constant flow of people for days.

Wednesday 4th May 2016: The children arrive and I tell them the news. They all react differently. Lots of tears, screaming the disbelief. I can safely say it was the hardest thing ever, but actually worst was to come.
We spend days and nights sitting and talking to him. The girls are amazing with him. Nursing him, holding his hand, cuddling him. Quite amazing for a 11 and 9 year old.

Friday 6th May 2016: My world is changed forever, our world. Gavin passes at 6.09am the exact time he was born on 17th Sept 1976. My mum brings the children up, I tell them there's another star in the sky.
They are destroyed - I am destroyed.

This should NEVER have happened. My gorgeous loving husband who had a zest for life and wanted to live was gone.
Myself and the children, 8 months on, are still devastated.
We receive regular counselling to get us through our grief, the pain and trying to understand WHY ?
The NHS failed Gavin when he needed it most...

Give Sasha a Chance!

Add your signature to Sacha's page on Change.org

A publicly funded NHS

The NHS must remain free & never be privatised

MDS UK Note:

We are regularly in touch with the family – but Lisa and the children are still understandably struggling heavily with the loss of Gavin – and therefore all enquiries should be directed to MDS UK. Thank you for your understanding.
We also sincerely thank the whole extended family, their friends, colleagues, employer for their ongoing support, participation, fundraising and awareness raising of MDS.
We have the deepest of admiration for Gavin’s two daughters especially, who have displayed an incredible courage, maturity and strength over the last 3 years.
They have spoken up about MDS, in school assembly, with friends, their athletics club, written to the press, participated in MDS UK activities, swam and baked for fundraisers, filmed an amazing awareness clip with their little brother – and even cut their hair off to make wigs for children with cancer.

There is hardly anyone they have not engaged around them- and still do so.

Please look up all of the above on our website www.mdspatientsupport.org.uk
We are immensely proud of them-and will continue to fight this issue in memory of Gavin – and for his family.
Their wish now is simply that no family should ever need to go through what they are experiencing – this is why they agreed to share these details now.

Gavin's Family Video to Raise Awareness on MDS


How to involve patients in their own care

Support patients to manage their condition better

Evidence tells us that when patients are actively involved in their own care, treatment and support can improve outcomes and experience for patients.

How to get involved

Everyone involved in care should advocate for the experience, voice and presence of patients, carers and the public to make a real difference to research in the NHS in order to improve health and well-being.

We all know importance of maintaining a healthy lifestyle through exercise and a healthy diet, but what else can be done?

Help and get actively involved in the work of the NIHR, the National Institute for Health and Research

In their website you'll find out about research that has taken place, you can take part in a free online course and contribute with questions and ideas for research.

The National Health Service (NHS) states:

The NHS is there to improve our health and wellbeing, supporting us to keep mentally and physically well, to get better when we are ill and, when we cannot fully recover, to stay as well as we can to the end of our lives. It works at the limits of science – bringing the highest levels of human knowledge and skill to save lives and improve health. It touches our lives at times of basic human need, when care and compassion are what matter most.

The NHS is founded on a common set of principles and values that bind together the communities and people it serves – patients and public – and the staff who work for it.

The Constitution Handbook adds:

Research is a core part of the NHS. Research enables the NHS to improve the current and future health of the people it serves. The NHS will do all it can to ensure that patients, from every part of England, are made aware of research that is of particular relevance to them.

Going the Extra Mile

The NIHR in England in their Going the Extra Mile report provides the strategic vision and direction for our work in engaging and actively involving patients, carers and the public in all aspects of research. It sets the following goals:

By 2025 we expect all people using health and social care, and increasing numbers of the public, to be aware of and choosing to contribute to research by:
- Identifying future research priorities and research questions
- Informing the design and development of innovations
- Participating in research studies
- Advocating for the adoption and implementation of research in the NHS

Both health workers and patients should ensure that they contribute to INVOLVE and ensure that information held on their website about where they work or receive care is accurate.

What's happening elsewhere?

We do need to consider what is happening elsewhere by exploring, for example: the European Patients‘ Academy on Therapeutic Innovation (EUPATI), the Irish Platform for Patient Organisations, Science and industry (IPPOSI). Further afield there is the Canadian Institutes for Health Research and their Strategy for Patient Orientated Research. You may also choose to look at the developments of Involving People in Research in Western Australia and the Patient Centred Outcomes Research Institute (PCORI) in the USA. These cover many of the leading economies and we risk deepening the health inequalities unless we also look at what is happening in there countries.

The great work of charities

There are many charities who focus on health research and others who carry out research themselves. Many are now actively involving patients and the public in the research that they fund. This ranges from priority setting, improving study design and sitting on funding decision making panels.

The Shared Learning Group on Involvement brings together a number of people working in charities doing patient, public involvement and engagement. They also have a ‘research sub-group’. Parkinson's recently produced an excellent guide for their researchers on patient and public involvement. The Association of Medical Research Charities (AMRC) is a further good source of information.

The role of Universities

There is a significant amount of public involvement taking place across all academic institutions. This is partly driven by the Research Excellence Framework which requires research work to have ‘reach’ and ‘significance’. It is worth finding out about the Academic Health Sciences Network (AHSN) and the local Collaborations for Leadership in Applied Health Research and Care (CLAHRC)

Take a look at current MDS clinical trials


What is CAR T cell therapy? Learn about how this immunotherapy works

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 it work?

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 the 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. The 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.

The science behind it

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

Take a look at current MDS clinical trials


The European MDS Registry: learning about the ‘real’ MDS patients

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

What is a clinical Registry?

In order to develop new drugs to treat MDS, clinical trials are mandatory, typically testing the new drug against the treatment that is considered to be the standard at that time. However patients in clinical trials are often different to those that we see every week in our clinics because the clinical trial will try to treat a group of patients who seem quite similar to each other and that means leaving out the patients with other diseases in addition to their MDS, or those too infirm to travel to and from the trial centre for example.

The best way to study a typical group of MDS patients without any restrictions is to set up a Registry. This involves obtaining consent from patients to collect information about their MDS and the treatments that they receive at their usual clinic or Day Case Unit visits. They may be asked to complete some questionnaires and sometimes to volunteer occasional extra blood samples but essentially the idea is simple; to systematically capture the usual care of all MDS patients over time into one large database.

European MDS Registry

The European MDS Registry (EUMDS) started on April 1st 2008, recording information from patients with ‘low-risk’ MDS in 10 European countries. Now the Registry has expanded to include 145 individual hospitals in 17 countries. The Registry has gathered information on more than 2000 low-risk MDS patients. 

Patients start in the registry within 3 months from their diagnosis and are followed for their lifetime with information recorded every 6 months. This information includes any treatment that they received, how they perceive their quality of life and giving a small ‘serum’ blood sample. Patients in the registry now have information from an average of four 6-monthly visits recorded. 

The UK has a prominent role in the project. We have registered 327 UK patients so far, the second highest number registered per country after France. The UK hospitals that are participating in order of number of patients recruited are:

Leeds, Aberdeen, Blackpool, Worcester, Airedale, Northampton, Mid Yorkshire, Bradford, Bournemouth, Harrogate, Truro, York, Oxford, Cambridge, Nottingham, Newcastle, Glasgow, Huddersfield, Manchester Christie, Birmingham Queen Elizabeth.

We have collected half of all of the blood ‘serum’ samples for the entire project, which are being used for interesting research studies (701 UK samples out of a total of 1211 samples analysed in EUMDS). Our patients have completed the quality of life questionnaires with a high completion rate (298/327 patients; better than most European countries). The UK is playing a prominent role in the organisational and strategic aspects of the EUMDS programme, which is led overall by Professor Theo de Witte from The Netherlands. Professor David Bowen leads the UK arm and is also Co-Chair of the Steering Committee for the EUMDS programme. The University of York Health Sciences Unit (Dr. Alex Smith and colleagues, https://www.york.ac.uk/healthsciences/research/ecsg/) hosts the database, provides the informational technology support and analyses the data.

We are building a picture of how low-risk MDS is cared for generally in Europe and also in different countries with some interesting patterns emerging. As well as the general information collected and analysed, there are more detailed research projects evolving within the registry, for example looking at the patients who have received blood transfusions, studying those that have received a blood stimulating drug like erythropoietin (EPO), or the possible importance (or not) of overloading with iron for patients receiving blood transfusions.

We are also studying the bone marrow samples using modern diagnostic tests like next-generation sequencing [NGS] which gives us more detail about the different biology of each patient’s type of MDS. We will then be able to look at how this new detailed information could help to predict how patients will fare generally (for example life expectancy/survival and the chances of MDS changing to more aggressive leukaemia) and maybe better predict who might respond to which treatments.

We are now moving onto the next phase of the programme, including higher-risk MDS patients in the registry and engaging new sponsors and new funding streams.

We are deeply grateful to those patients who are helping with this important research initiative.

MDS-RIGHT: a platform for research, for patients, and for other stakeholders

Although technically it maybe an ‘offshoot’ of EUMDS, the European Union funded 5-year MDS-RIGHT project is a wide ranging, ambitious programme with the potential to lead to an integrated European network for:

  1. MDS research
  2. patients
  3. influencing important stakeholders such as the regulatory agencies, the payers (Department of Health in UK) and policymakers.

Almost all key opinion leaders for MDS are centrally involved in MDS-RIGHT giving this prestigious programme high credibility and huge potential. Again the University of York is an important partner with Health Economics (cost effectiveness, led by Professor Manca, https://www.york.ac.uk/che/staff/research/andrea-manca) now added to the goals.

MDS-Europe

The European MDS Registry is supported by an unrestricted educational grant from Novartis Oncology Europe.
MDS-RIGHT is funded by the European Union (a programme called Horizon 2020 research and innovation under grant agreement No. 634789).
Key websites:
https://mds-europe.eu
http://www.eumds.org

MDS UK – Note to patients

MDS UK Note: Interested in taking part as a patient?
If you are newly diagnosed – please ask your haematologist about it.
Show them this article and state you’d like to volunteer your blood and biopsy samples – and contribute to this important research.

For any further information, please contact MDS UK:
Email: Mds-uk@mds-foundation.org or Tel: 020 733 7558

Please quote the following information if you wish to use our ResearchFORPatients article:

Source: www.mdspatientsupport.org.uk / ResearchFORpatients

Original reference paper:
Registry Nov 2016 Author: David Bowen, Honorary Professor of Myeloid Leukaemia Studies and Consultant Haematologist, St James's Institute of Oncology, Leeds


Clinical data on new medicines is now open to patients and researchers

Researchers and patients can access thousands of pages submitted by pharmaceutical companies

As of the 20th of October, the European Medicines Agency (EMA) gives open access to clinical reports for new medicines authorised in the European Union.
For every new medicine, citizens, including researchers and academics, will be able to directly access thousands of pages from clinical reports submitted by pharmaceutical companies. Clinical reports give information on the methods used and results of clinical trials conducted on medicines.
Vytenis Andriukaitis, European Commissioner for Health and Food Safety, said

"Transparency is an essential component in clinical research. Its outcome – whether positive or negative – should be made publicly available."

Learning from the experience of others

With EMA’s proactive approach to providing access to the data, patients and healthcare professionals will be able to find out more information about the data underpinning the approval of medicines they are taking or prescribing.

It will also facilitate the independent re-analysis of data by academics and researchers after a medicine has been approved. This will increase scientific knowledge, and potentially further inform regulatory decision making in the future.

Increased transparency will also benefit innovation. The shared knowledge about a medicine helps developers learn from the experience of others and can lead to more efficient medicine development programmes by reducing duplication of research and de-risking some new developments.

Protecting personal data

This will be a learning curve for the Agency and all its stakeholders, as they start to apply the policy for the first time.

While the policy gives an unprecedented proactive access to clinical data, it also demands the highest standard of protection of patients’ personal data. The process will evolve over time as more experience is gained and may lead to adaptations of EMA’s guidance.

Take a look at current MDS clinical trials


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