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Imerge (Imetelstat) – Clinical Trial Open to Recruitment

Posted on 08/12/2020 at 3:09 pm.

SUB-TYPE OF MDS: Low risk MDS patients
SEVERITY OF MDS: IPSS Low or Intermediate-1 risk MDS
NAME OF DRUG: Imetelstat
Aims and benefits: This phase 2/3 study aims to determine the effectiveness of Imetelstat in transfusion dependent low risk MDS patients who have either lost their response or not responded to erythropoiesis-stimulating agent (ESA) treatment. The study has 2 parts. Part 1 seeks to determine the effectiveness and safety of Imetelstat, looking more closely at improvement in blood counts and reduction in transfusion requirements. Part 2 is double-blind and randomised to compare the effectiveness of Imetelstat with placebo.
Primary outcome:To determine the percentage of patients who have not required a red cell transfusion in an 8 week period.
Secondary outcome:To determine adverse effects, red cell transfusion independence, improvement in blood counts, progression to acute myeloid leukaemia and overall survival.

Myelo’s Hope Christmas Animation

Posted on 08/12/2020 at 2:03 pm.

Christmas is just around the corner and it will look drastically different for millions of people across the globe this year. Hope and encouragement will be needed to find safe ways of enjoying the season.

Many of you will have seen Myelo's Hope the animation we released on MDS World Awareness Day which explains MDS in an accessible way and signposts patients and their loved ones to support.

Buoyed by the warm response, the animators have now released a fantastic festive version, further highlighting the issues in the original while calling for much-needed support!

A novel treatment for MDS patients in need of frequent blood transfusions

Posted on 09/11/2020 at 3:12 pm.

Written by Manuela Rotstein

The innovative drug Imetelstat has been trialed on patients with lower-risk MDS who are RBC (Red Blood Cells) transfusion dependent and have experienced relapse or not been helped by erythropoiesis-stimulating agent (ESA).

This group of patients does not have many treatment options at the moment.

In this press briefing, recorded at the 24th Congress of the European Hematology Association (EHA) 2019, held in Amsterdam, Netherlands, Pierre Fenaux of St Louis Hospital, Paris, France, presents data on the use of Imetelstat in lower risk myelodysplastic syndrome (MDS) patients.

Scientists have observed that patients with MDS have a high telomerase activity in their stem cells. Telomerase is a naturally occurring enzyme, and its activity prevents cells from dying. Telomerase is repressed in most normal cells, preventing uncontrolled proliferation. In cancer cells, however, this enzyme is active.

Imetelstat, is a "telomerase inhibitor" and targets cells with active telomerase.

The study has reported that Imetelstat shows efficacy to control the enzyme and it's safe to use. It also reports biomarker data for patients with low-risk MDS who are red blood cells transfusion dependent and who were relapsed/refractory to erythropoiesis-stimulating agents.

About the study

The study is a two-part phase II/III study.

The primary endpoint (the main result that is measured at the end of a study to see if a given treatment worked) was 8-week red blood cells transfusion independence rate.

Key secondary end points were:

24-week red blood cells transfusion independence rate
transfusion independence duration
haematologic improvement-erythroid

Of 57 patients enrolled and treated (overall population), 38 were non-del(5q) and hypomethylating agent and lenalidomide naïve (subset population).

Encouraging results from phase II of the study

A Phase II trial is still early days in terms of clinical trials – to read more about how this trial progresses – see this page at ClinicalTrials.gov

The 8- and 24-week red blood cells transfusion independence rate in the overall population were 37% and 23%, respectively, with a median transfusion independence duration of 65 weeks.
In the subset population, 8- and 24-week red blood cells transfusion independence rates were 42% and 29%, respectively, with a median transfusion independence duration of 86 weeks.
Eight-week transfusion independence rate was observed across all subgroups evaluated.
Cytogenetic and mutational data revealed a reduction of the malignant clones, suggesting disease modification activity.
The most common adverse events were cytopenias, typically reversible within 4 weeks.

In conclusion, Imetelstat treatment results in a meaningful, durable transfusion independence rate across a broad range of heavily transfused patients with Low-Risk MDS who are ineligible for or relapsed/refractory to erythropoiesis-stimulating agents. Biomarker analyses indicated effects on the mutant malignant clone.

Authors

David P. Steensma, MD¹; Pierre Fenaux, MD, PhD²; Koen Van Eygen, MD³; Azra Raza, MD⁴; Valeria Santini, MD⁵; Ulrich Germing, MD, PhD⁶; Patricia Font, MD⁷; Maria Diez-Campelo, MD, PhD⁸; Sylvain Thepot, MD⁹; Edo Vellenga, MD, PhD¹⁰; Mrinal M. Patnaik, MBBS¹¹; Jun Ho Jang, MD¹²; Helen Varsos, MS, RPh¹³; Jacqueline Bussolari, PhD¹³; Esther Rose, MD¹³; Laurie Sherman, RN¹⁴; Libo Sun, PhD¹⁴; Ying Wan, MD, PhD¹⁴; Souria Dougherty, BS, MBA¹⁴; Fei Huang, PhD¹⁴; Faye Feller, MD¹⁴; Aleksandra Rizo, MD, PhD¹⁴; and Uwe Platzbecker, MD¹⁵

¹Dana-Farber Cancer Institute, Boston, MA
²Hôpital Saint-Louis, Université Paris Diderot, Paris, France
³Algemeen Ziekenhuis Groeninge, Kortrijk, Belgium
⁴Columbia University Medical Center, New York, NY
⁵MDS Unit, AOU Careggi-University of Florence, Florence, Italy
⁶Klinik für Hämatologie, Onkologie and Klinische lmmunologie, Universitätsklinik Düsseldorf, Heinrich-Heine-Universität, Düsseldorf, Germany
⁷Department of Hematology, Hospital General Universitario Gregorio Marañon, Madrid, Spain
⁸Hematology Department, The University Hospital of Salamanca, Salamanca, Spain
⁹CHU Angers, Angers, France
¹⁰Department of Hematology, University Medical Center Groningen, University of Groningen, Groningen, the Netherlands
¹¹Division of Hematology, Department of Internal Medicine, Mayo Clinic, Rochester, MN
¹²Department of Hematology, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Republic of Korea
¹³Janssen Research & Development, Raritan, NJ
¹⁴Geron Corporation, Menlo Park, CA
¹⁵Department of Hematology and Cell Therapy, University Clinic Leipzig, Leipzig, Germany

Trial to test existing drugs against rare blood cancer

Posted on 27/10/2020 at 3:13 pm.

Written by Manuela Rotstein

A new clinical trial for MDS has just been launched.

Designed by clinical experts, using existing (repurposed) drugs in a new way, with wide PPI (Patient and Public Involvement) input and feedback by MDS UK advocates and patients, funded via Bloodcancer UK.

This trial will test existing drugs against the rare blood cancer Myelodysplastic Syndrome to improve patients' outlook and health.

It is run by UoBbiosciences & Warwick Clinical Trials Unit.

Best practice and cooperation at all levels for research benefitting MDS patients.

By making a donation to MDS UK, you are also funding our time, for the work we conducted with patients, enabling the subsequent approval of this trial.

Full details:
https://www.birminghamhealthpartners.co.uk/trial-to-test-existing-drugs-against-rare-blood-cancer/

A clinical trial designed to test existing drugs in the treatment of a rare blood cancer has been launched at the Universities of Birmingham and Warwick.

Funded by Blood Cancer UK, the trial seeks to repurpose existing drugs to improve the outlook, health and quality of life for people with Myelodysplastic Syndrome (MDS), a condition in which the patient does not have enough healthy blood cells.

Called REPAIR-MDS (Repurposed drugs to improve haematological responses in Myelodysplastic Syndrome), the trial will test a number of treatments designed to improve the formation of healthy blood cells, reducing or reversing life-limiting deficiencies in red blood cells. The trial is expected to begin recruiting patients in May 2021.

Professor Chris Bunce, in the School of Biosciences at BHP founder-member the University of Birmingham, who led the application to Blood Cancer UK and whose research contributed to the design of the trial treatments, says: “REPAIR-MDS represents a significant turning point in UK medical research. It is the first ever UK randomised trial delivered in this neglected patient group, establishing the precedent for future trials in the UK and elsewhere.”

Professor Janet Dunn, Head of Cancer Trials at Warwick Clinical Trials Unit at the University of Warwick, says: “It’s an important trial for these patients as currently treatment options are limited. We are excited to be working closely with Birmingham and the patient groups, in particular Sophie Wintrich who is CEO from the MDS UK patient support group.”

Although rare, MDS is estimated to be more prevalent in older people, over 70 years old. There is currently no cure for the disease and treatment is usually chemotherapy-based, although in some cases a stem cell transplant may be possible.

Dr Fatima Sulaiman, Head of Research for Blood Cancer UK added: “Blood Cancer UK are really excited to be supporting this trial. We believe that within the next generation, we’ll be able to beat all types of blood cancer, and this trial will take us one step closer to doing this.

“Sadly, only 31% of people diagnosed with myelodysplastic syndrome (MDS) survive 5-years, and we urgently need better treatments. Being able to repurpose existing drugs for people with MDS would mean we would be able to get new treatments to people, sooner, giving everyone the best possible chance of survival.

“Anyone worried about blood cancer can call our free and confidential helpline on +44 (0) 808 2080 888.”

The project is a national effort by the UK MDS research community and will recruit patients from across the UK. The trial was developed by the National Cancer Research Institute (NCRI) MDS clinical studies sub-group and has arisen from research carried out in the School of Biosciences and the School of Medical and Dental Sciences at the University of Birmingham led by Professor Bunce. It will be carried out in collaboration with Queen Elizabeth Hospital Birmingham.

The funding of REPAIR-MDS by Blood Cancer UK has been made possible by a donation made in open competition to the charity from the Jon Moulton Charity Trust.

New animation to explain MDS to all

Posted on 25/10/2020 at 2:09 pm.

Written by mariano

This ground-breaking animation aims to raise awareness of MDS (Myelodysplastic Syndromes) and create an accessible story for newly diagnosed patients and their loved ones, signpost them to help and support and raise funds for MDS UK.

Can you help us distribute this clip in hospitals waiting rooms?

Help us to help you and others: If your haematology waiting room has an internal TV system in place – please ask the Waiting Room Manager if this information cartoon could be featured on their TV ?

Ask the waiting room manager to contact us on

Email: admin1@mdspatientsupport.org.uk

Reference: MDS-Myelo’s Hope Infocartoon

We would aim to use donations to help us cover the costs arising from our involvement in, and contribution to MDS-related research.
MDS UK staff, committee members and expert patients are involved all year round in different aspects of research, including addressing the issue of access to MDS treatments.
We regularly provide input to Patient and Public Information (PPI) work, for both commercial and non-commercial clinical trials. We are financially compensated for some of this work, but not all.
This wider research and access work takes time, effort and energy, and therefore needs funds.
Without this work, ethics boards would not approve trials, EMA and NICE processes would be delayed, and access to drugs might be compromised. And, above all, the voice, needs and wishes of patients would not be heard or considered.
Additionally, having funded one PhD research project (at Queen’s University, Belfast), we would like to consider funding more direct research, should our funds allow us to do so in future.
If you have found this topic of interest and would like to support us in such work, we would welcome any donations, however small. If you can, please use Gift Aid to enhance the value of your donation to us by 25%, at no additional cost to yourself.

Click here to read more about how we represent the patient voice

Click here to see more about our work on access to drugs

25th of October: MDS World Awareness Day 2020 – Take Part!

Posted on 19/10/2020 at 9:17 am.

Written by Manuela Rotstein

It’s time to get your pens, brushes and camera out for MDS WAD on Sunday October 25th!

This is a crucial day for the worldwide MDS community to make a stand against this rare blood cancer and raise much needed awareness!

We would like to encourage as many people from MDS UK as possible to get involved, becoming part of a wider collective voice.

This year's theme from the MDS Alliance is "Faces of MDS", an education and awareness campaign involving sharing self-portraits and selfies with MDS facts.

Take a look at the MDS Alliance website . Download and print out the handy templates, which include MDS facts.
Using these templates, you can draw a self-portrait or ask your children, grandchildren or friends to draw your portrait. You can also take a selfie using the MDS cut-out selfie template or with an MDS WAD fact sheet
Take photos of the drawings / selfies and post them and the selfies to Facebook, Twitter and Instagram using #FacesofMDS! and #MDSWAD2020.

You can also get involved without using social media.

Simply follow steps 1-2 above and then send your creations to secretariat@scientificeducationsupport.com following the instructions on the briefing guide (see below)

Go on, it’s time to become part of the collective voice and get involved with the easy to implement MDS WAD activities!

We hope to see many of you involved!

How have you coped over the past few months of COVID-19? Please let us know

Posted on 08/10/2020 at 2:37 pm.

Written by Manuela Rotstein

This national survey is about

your perspective and experience of the COVID-19 pandemic generally
the clinical care and support you received
the information you had access to
treatment of COVID-19, if applicable

How will this data be used?

The data collected will inform clinical staff about any beneficial changes to practice they can take forward to improve the care of MDS and AML patients as well as understand what barriers and difficulties patients came across during this period.

We also hope to pass on some of this data to PHE (Public Health England), to also improve the way they provide information and services to patients and families.

We will publish results on our website as well, and use the information to improve our services to members too.

Confidentiality and GDPR

This survey is set up to be completed totally anonymously, in line with GDPR regulations for this type of work.

MDS UK members will be sent a link, but it will not be possible to trace back answers to individuals.

The link can only be used once per device.

Other surveys on COVID-19

You may have completed other surveys on the topic of Covid-19, for other organisations, but we kindly ask you to complete this one too, as it is the only survey fully dedicated to MDS, and prepared by the team of clinical experts in MDS and the NCRI sub-group on MDS.

Your MDS clinicians may also forward you a link to this particular survey.

It does not matter which link you end up using, as data will be collected together.

How long does it take to complete it?

It may take you up to 30minutes to answer all questions.

Some questions can be skipped.

We thank you for taking this time to provide these very important answers, to help us help you.

News for MDS patients from the European Registry

Posted on 08/10/2020 at 2:09 pm.

Written by Manuela Rotstein

The best way to study a typical group of MDS patients is to set up a registry. A registry involves obtaining consent from patients and collect information about their MDS and the treatments they receive at their usual clinic or Day Case Unit visits.

The European MDS Registry (EUMDS), was initiated in 2008 by a large group of European haematologists. It contains information on treatment and disease of more than 2500 MDS patients from 17 countries and it continues to include new patients.

Many of you will be familiar with the EUMDS project and some of you are registered and actively sharing your MDS data, helping researchers to improve their understanding of the disease. This helps to shape better clinical trials, and ultimately may lead to better treatments for MDS.

Which scientific studies have been performed so far by the Registry?

The information collected in the EUMDS registry forms the basis for scientific studies. In these studies MDS experts seek to answer scientific questions on MDS and the treatments available. By answering these questions scientists aim to improve the diagnosis and treatment of MDS.

The results from EUMDS scientific studies are published in scientific journals and presented on large haematological congresses.

In addition, the EUMDS publishes summaries of these scientific studies for patients and the general public, in a patient friendly format. These are published on the "Lay English Summaries": https://eumds.org/publications/summaries

See, for example, two topics which have been published under "Lay English Summaries":

Impact of red blood cell transfusion dose density on progression-free survival in lower-risk myelodysplastic syndromes patients.

Red blood cell transfusions (RBCT) are an important part of the general medical care for MDS patients. However, the overall life expectancy of patients treated with RBCT is often decreased compared to untransfused MDS patients. It is unclear whether this lower life expectancy is due to the nature of the disease (more severe illness and therefore greater need for transfusions) or whether it is due to unwanted side effects of the RBCT itself. We observed that even patients transfused with small quantities of blood transfusions have a negatively affected life expectancy. We are unsure what causes this negative association, and hypothesize that it could be related to the release of toxic iron radicals after transfusion. Future studies are needed to confirm these observations and in addition, it is necessary to perform dedicated intervention studies with the aim to overcome this negative effect.

Validation of the revised international prognostic scoring system (IPSS-R) in patients with lower-risk myelodysplastic syndromes: a report from the prospective European LeukaemiaNet MDS (EUMDS) registry

The Registry has been opened for a while, and in this timeframe the scientific community has upgraded the criteria which were used to calculate the prognosis of patients with MDS. These new criteria (IPSS-R) were validated on the first 1000 EUMDS patients who were characterized using the ‘’old’’ (IPSS) criteria. This study shows that using the new criteria, our risk assessment improved for the various MDS patient groups. Currently, both the old and the new criteria need to be reported by the physician for each patient who joins the Registry.

Visit the Lay English Summaries of articles published by the EUMDS Registry about the topics below

Early platelet count kinetics has prognostic value in lower-risk myelodysplastic syndromes
Health-related quality of life in lower-risk MDS patients compared with age- and sex-matched reference populations: a European LeukemiaNet study.
Labile Plasma Iron Levels Predict Survival In Patients With Lower-Risk Myelodysplastic Syndromes
Erythropoiesis-stimulating agents significantly delay the onset of a regular transfusion need in nontransfused patients with lower-risk myelodysplastic syndrome
Impact of treatment with iron chelation therapy in patients with lower-risk myelodysplastic syndromes participating in the European MDS registry

MDS-RIGHT

If you found the articles above of interest to you, please also read on about MDS Right.

MDS-RIGHT (Providing the right care to the right patient with MyeloDysplastic Syndrome at the right time) is a research project that has been granted 6 million Euros from the Horizon2020 programme of the European Union. In this project – which started in May 2015 and will end in 2020 - fifteen European partners have joined forces. By comparing existing health care interventions and by defining and implementing more effective and safer interventions for elderly European citizens with anaemia and/or lower-risk MDS, the project aims to lead to better treatment compliance and more (cost-)effective use of healthcare resources.

https://mds-europe.eu/right “

20for20 Gallery

Posted on 06/10/2020 at 3:16 pm.

Written by Manuela Rotstein

World Leukemia Day on 4th September

Posted on 04/09/2020 at 3:33 pm.

Written by mariano

MDS patients can be affected by leukaemia too:

10% of low-risk MDS cases do progress to Acute Myeloid Leukaemia

30% of high-risk MDS cases do progress to Acute Myeloid Leukaemia

In those MDS cases, it is then called secondary AML, as opposed to de novo AML, when someone is suddenly diagnosed with acute myeloid leukaemia.

To help raise awareness of leukaemia, you can get involved by taking spotty selfies, and sharing on social media.

Here is also some background on leukaemia, as well as a couple of videos, explaining some symptoms, which can be particularly relevant for our MDS community.

https://www.worldleukemiaday.org/what-is-leukemia-1

Take a spotty selfie:

Dig out your spotty clothes and share your ‘spotty selfie’ on social media with the hashtag #WLD20 on September 4th to show your support.

Facebook:

Show your support for World Leukemia Day by using our Facebook Frame. All you have to do is follow these easy steps…

Go to your Facebook profile and click edit on your profile picture
Click add frame
Search for World Leukemia Day
Select our Facebook frame
Click Use as Profile Picture to save
And you’re done!

Tweet:

#WLD20 #WorldLeukemiaDay

We count on you to raise awareness in leukemia on 4th September !

Imerge (Imetelstat)

Myelo's Hope: Christmas Animation

Latest News

Imetelstat has achieved meaningful and durable transfusion independence in clinical trials

How does Imetelstat work? The science behind it

About the study

Encouraging results from phase II of the study

Authors

Latest News

Cooperation to support a new clinical trial for MDS

Latest News

Can you help us distribute this clip in hospitals waiting rooms?

Please help us fund MDS UK research work:

Latest News

MDS World Awareness Day 2020: FACES OF MDS

Here's our first Face of MDS: the amazing Kate Darby!

Here’s how to take part:

MDSWAD sophie (1)

MDSWAD russell

IMG_0126 (1)

MDSWAD chrisC

25th of October is World Awareness Day

Help us raise public awareness on Myelodysplastic Syndromes!

Patients and caregivers/supporters survey on care during the COVID-19 pandemic

What does this survey cover?

How will this data be used?

Confidentiality and GDPR

Other surveys on COVID-19

How long does it take to complete it?

Technical Queries?

MDS UK team

Latest News

What is the European Myelodysplastic Syndromes Registry (EUMDS) project?

Which scientific studies have been performed so far by the Registry?

The effect of transfusions on patients quality of life

Ways to improve diagnosis and prognosis for new patients

Visit the Lay English Summaries of articles published by the EUMDS Registry about the topics below

MDS-RIGHT

Latest News

What have our wonderful participants been up to?

Today, 4th September, together with the worldwide blood cancer community, MDS UK is marking World Leukaemia Day.

Fever or night sweats?

Weak or short of breath?

Latest News