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ACE-536-MDS-002 (Luspatercept)

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.

ACE-536-MDS-002 (Luspatercept)

  1. SUB-TYPE OF MDS:Lower Risk MDS patients who require red cell transfusions but have not received Erythropoietin
  2. SEVERITY OF MDS: IPSS-R Very Low, Low or Intermediate Risk MDS
  3. NAME OF DRUG: Luspatercept
  4. Aims and benefits: To compare the safety and efficacy of Luspatercept versus epoetin alfa for treating anaemia in patients who require red cell transfusions.
    Luspatercept promotes red blood cell formation by regulating the growth of red blood cells during the late-stage of their development. It works differently to erythropoietin.
    This is a Phase 3 Randomized Study between Luspatercept and Epoetin alfa.

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FG-4592-082 (Roxadustat) 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.

FG-4592-082 (Roxadustat)

  1. SUB-TYPE OF MDS:Lower Risk MDS With Low Red Blood Cell Transfusion Burden
  2. SEVERITY OF MDS: IPSS-R classification very low, low or intermediate risk with <5% blasts
  3. NAME OF DRUG: Roxadustat
  4. Aims and benefits: To determine whether Roxadustat is safe and effective in treating anaemia in patients with Primary Lower Risk Myelodysplastic Syndrome and Low Red Blood Cell Transfusion Burden.
    Roxadustat is an oral preparation that stimulates erythropoiesis (production of red cells) by increasing the body’s production of the hormone erythropoietin and it regulates the way in which the body uses iron. This study is a Phase 3 Randomized Double-Blind Placebo-Controlled Study in which there is a treatment period of 52 weeks and a 4 week end of treatment assessment.

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Spotted in London this morning: a mysterious figure raising awareness of MDS & blood cancer

Mystery red figure in London Train

Mystery figure spotted next to a surprised Londoner on the train

Exclusive photos given anonymously to MDS UK have captured the moment a mysterious red creature seemed to walk on the streets of London raising awareness of MDS on Blood Cancer Awareness Month.

In one the photos, the red figure appears reading the newspaper on the train, while next to it a surprised Londoner makes desperate attempts to ignore it by looking at her phone.

Red figure and passersby at London during Blood Cancer Awareness Month

Passersby Amused!

Passersby on the train station appeared amused by the creature and were not afraid of sharing a ride on the escalators with the seemingly supernatural being.

In many images, the red figure can be seen displaying a sign attracting attention to MDS UK Patient Support Group!

Taxi drivers and tourists at London main attractions were today reminded by the outlandish creature that September is Blood Cancer Awareness Month, and many people next to them may suffer from this “invisible” disease.

Red figure in London during Blood Cancer Awareness Month

Have you seen the red mystery figure?

According to members of the charity, the supernatural being has done an amazing job at raising awareness of MDS.

The CEO of MDS UK, Sophie Wintrich, said: "We are looking for the mystery red figure to thank it immensely on behalf of MDS patients and their families."

Online users have however questioned whether the photos are real or not.

September: Blood Cancer Awareness Month

Make Blood Cancer Visible 2019

September is #BloodCancerAwarenessMonth.

MDS UK Patient Support Group, together with colleagues from Anthony Nolan, Bloodwise, CLL Support Association, CML Support, Leukaemia Care, Lymphoma Action, Myeloma UK, and Waldenstrom's Macroglobulinemia (WMUK), joined Make Blood Cancer Visible, an awareness campaign sponsored by Janssen UK.

This year, the theme is “Connecting the dots”, aimed at encouraging people to identify the many symptoms that can be experienced by people with blood cancer.

Additionally, throughout the month, MDS UK will posting poems on the theme of blood cancer written by our talented MDS UK member, KateD and other MDS patients,  starting with her M.D.S. poem we published in our June Newsletter.

Blood cancer is the third biggest cancer killer in the UK, claiming the lives of more than 15,000 people each year – more than breast cancer or prostate cancer. We believe that making blood cancer more visible will help people identify symptoms earlier, strengthen the community of people affected by blood cancer and help us to fund lifesaving research.

Help us raise awareness with the general public this September by sharing Kate's MDS Poems, or sending your own, and spreading the facts on this "Connecting the dots" infographic.

M.D.S. A Poem by Kate D.

My Day Starts.
I Must Do Something,
but then remember;
I can’t always do what I used to.
It Mostly Doesn’t Show
to those who don’t know.

Most Days Show how life used to be
before my Many Daily Struggles began
and Mild Depression Set in.
My Dog Sits on my shoulder.
But it Mostly Doesn’t Show
to those who don’t know.

Most Doctors Strive to do
what is best for the patient;
Many Don’t Spot the tell - tale signs,
are confused and perfunctory because
there are Multiple Different Symptoms
that confuse and delay.

My Marrow’s Diagnosis’ State is not
a good one; in fact,
My Diagnosis Stinks, but
it Mostly Doesn’t Show
to those who don’t know.

Medicinal Drugs Start.
I May Die Slowly or stay like this
for years yet as it
Mostly Doesn’t Show
to those who don’t know.

There are More Daily Struggles
for people like me
who have this fatigue,
these aching joints and bones,
this marrow that doesn’t work,
these days of despair.

Many Don’t Survive.
But I am not the worst and
for those in the know,
it Mostly Does Show
bravery, honesty, resilience.

Many Days Sort themselves out
in hospital waiting rooms on
hard plastic chairs,

Most Doctors Smile and ask how I am,
and continue to Make Some Diagnosis
if they know and can show
me that My Daily Struggle,
my MDS is Myelodysplastic Syndrome.

Make Blood Cancer Visible 2019 Infographic: Connecting the dots

Make Blood Cancer Visible 2019 Infographic: Connecting the dots

Watch Laurence Llewelyn-Bowen's story for #MakeBloodCancerVisible

Laurence Llewelyn-Bowen is the official ambassador for Make Blood Cancer Visible 2019.

We thank Janssen UK for sponsoring this work and making this awareness campaign possible. Without their help and organization, this type of work would not have been possible.

Familial/inherited MDS: rare but important to keep in mind

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

Is MDS likely to be passed down from parent to child?

Written by Prof Jude Fitzgibbon, Prof Tom Vulliamy and Prof Inderjeet Dokal, Queen Mary University of London

When a patient is first diagnosed with myelodysplastic syndrome (MDS), one of the most frequent questions posed is whether the disease is likely to be passed down from parent to child, and if other family members could conceivably develop this malignancy too.

Conventionally, heritable (i.e. passed from parent to child) forms of MDS are thought to be rare and are typically, NOT considered to run in families.

Our research group at Queen Mary University of London (QMUL), with funding from the charity Bloodwise, have been collecting and storing blood and bone marrow samples from these rare patients and their families in order to better understand the nature of the faulty genes responsible for inherited MDS. This research is important for the individual families, as it provides valuable
information for treatment of the disease, assessing risk and genetic counselling, but it also offers a unique opportunity to identify the critical early genetic events that give rise or predispose patients to MDS.

Jude Fitzgibbon talks with Sophie Wintrich about familial MDS. Watch the video

Since 2016, inherited forms of myeloid malignancies, including MDS, have been included as a separate disease entity in the World Health Organisation (WHO) classification of haematological cancers.

This is leading to a greater awareness on behalf of haematologists regarding the existence of these forms of disease, enabling more tailored management of this group of at-risk individuals.

This is important, as patients with ‘familial MDS’ (i.e. with a predisposing mutation present in every cell) tend to develop symptoms at a much younger age compared to people with ‘sporadic disease’ (i.e. mutations are restricted to the MDS only).

We also appreciate that MDS can arise as part of a wider syndrome, with many patients/families initially exhibiting bone marrow failure syndromes such as Fanconi anemia, dyskeratosis congenita, and Shwachman–Diamond syndrome which often subsequently lead to MDS.

Why is it important to identify patients with familial MDS?

Our research is demonstrating that there isn’t a singly mutated gene responsible for familial MDS but many different genes,
some of which are also mutated in sporadic forms of the disease (RUNX1, GATA2). In comparison other mutations (in the germline) appear enriched or exclusive to inherited forms of MDS (DDX41, SAMD9) and this is offering researchers novel insights into the causes of MDS and the prospect of developing better treatments for all MDS patients.

It is, therefore, important that, when a new patient is diagnosed with MDS in the clinic, steps are taken to determine whether the disease has a significant genetic-inherited component. This is crucial, as in some subtypes of inherited/familial MDS subsequent therapy must be modified.

  • For example, if a patient has MDS associated with an underlying telomerase mutation then the chemotherapy conditioning regimen performed prior to a bone marrow transplant has to be reduced.
  • Equally, a haematologist would want to ensure that, in selecting bone marrow/stem cell donors, an asymptomatic family member with the same genetic defect is not used as the donor as the recipient could go on to develop MDS again at a later time point./li>

Germline vs Somatic (Sporadic) Mutation

Which services does the research group provide?

In order to facilitate identification and genetic categorization of inherited/familial MDS our research group (in collaboration with the Genetics Laboratory at Birmingham and support from Bloodwise) provides genetic testing in such cases. This means if a clinician suspects that their patient may have a significant genetic component they can send blood samples directly to our laboratory.

Indeed, in the future, our expectation is that every MDS patient will have a molecular profile performed as part of their overall management, to identify the specific mutations that are exclusive to their MDS and to assess if there is a significant inherited component, linked to their disease, where a mutation is present in all cells in the patient’s body.

We are also able to provide specific advice on the management of patients if a genetic defect is found in one of the many genes that are associated with inherited/familial MDS by contacting us directly (email

Furthermore, if a genetic defect is not found in one of the known familial MDS genes and there is a strong clinical suspicion for familial MDS (for example, if there is a history of multiple MDS cases in the family) then these samples are put forward for research studies aimed at new gene discovery.

In summary:

  • Familial/inherited MDS is a very heterogeneous and complicated disorder. It is thought to be rare but the precise figures on its incidence and prevalence are not known.
  • Over the last 20 years many genes have been identified that are responsible for familial/inherited predisposition to MDS and they have highlighted the importance of making specific modifications to therapy to achieve optimal outcomes.
  • Our ongoing research programme at QMUL provides genetic testing for all of these genes as well as a strong focus on identifying new disease genes where current genetic tests fail to identify a defect in at-risk families.

MDS UK Patient Support Group Newsletter – June 2019

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

Read all about:

Patient's Stories - Living with MDS:

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

Research - Progress in the diagnosis and treatment of MDS:

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

Fundraising Successes:

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

Here to help:

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

Read More

Precision medicine is coming to the clinic to treat MDS

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

by Niels Jensen

What is precision medicine?

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

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

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

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

How does precision medicine work?

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

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

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

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

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

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

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

Precision Medicine - A specific drug for each genetic mutation

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

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

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

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

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

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

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

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

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

Clinical Trials open to recruitment in the UK

Team MDS at the Great North Run on September 8 2019

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

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

An amazing team of 11 run for MDS UK Patient Support Group:

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Harmony: a European project with good news for MDS patients

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

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

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

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

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

The Harmony Alliance aims to develop treatments that are:

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

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

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

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

How does Harmony ensure data quality, safety and privacy?

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

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

The new generation of MDS diagnostics: the Myeloid Gene Panel

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

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

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

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

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

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

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

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

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

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

Scientists Interviewed:

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

The Myelod Gene Panel and how it helps MDS Patients

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

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

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