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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:
admin1@mdspatientsupport.org.uk 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


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

Now have a look at this graphic story, courtesy of the CLL Society:

Take a look at current MDS clinical trials


MDS UK grows its team to increase its support to patients

The MDS UK Committee is pleased to welcome two new members to the charity team: Lizzie Marshall, and Chris Fryer.

Both Lizzie and Chris have experience in the Non-Profit and Charity Sector.

Lizzie is a trainee psychotherapist with a background in the performing arts. She works supporting vulnerable people with learning difficulties alongside her new role at MDS UK.

Working with Database Manager Chris Fryer, Lizzie provides administrative and database assistance to the charity. She often is the one to send out information packs to new members, to answer questions about navigating the website and online forums, and send out the booklets and leaflets you see in hospital waiting rooms.

Chris has spent most of my career working in the non-profit sector. He takes pride in applying his administrative and technological understanding into helping support families and help spread awareness of MDS.

The addition of these two new members will allow us to keep providing our services to an increasing number of patients and their families.

Welcome on board Lizzie and Chris!


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.

PANTHER

  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.

BRIGHT

  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


CPDR001X2105: 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.

CPDR001X2105

  1. SUB-TYPE OF MDS:High risk MDS and AML, not suitable for induction chemotherapy or haemopoietic stem cell transplant
  2. SEVERITY OF MDS: High Risk MDS
  3. NAME OF DRUG: PDR001, MBG453, Decitabine
  4. Aims and benefits: PDR001 is a monoclonal antibody that functions to block the activity of PD-1 (programmed cell death protein 1) thus allowing T-cells to recognise and target cancer cells. MBG453 is a monoclonal antibody that binds to a specific protein found on the surface of T-cells thus allowing the T-cell to function normally against cancer cells by reducing tumour growth. This is a phase 1b study aimed at determining the safety and tolerability of the study drugs alone and in combination with decitabine. The study also aims to determine the recommended doses of each of these drugs.

Read More


RFUSIN2AML2: Clinical Trial Open to Recruitment

Research FOR Patients
-For an informed and empowered opinion-

All the trials listed on 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.

RFUSIN2AML2

  1. SUB-TYPE OF MDS: High Risk MDS RAEB-2 and Acute Myeloid Leukaemia Patients Unsuitable for Allogeneic Haematological Stem Cell Transplant
  2. SEVERITY OF MDS: High Risk MDS
  3. NAME OF DRUG: B7.1 (CD80)/IL-2 Immune Gene Therapy
  4. Aims and benefits: This is a phase I study intended to identify the safety and tolerability of an "AML Cell Vaccine" given to eligible MDS and AML patients who have achieved a best response of complete remission or partial remission following their first or second course of standard induction chemotherapy.

Read More


FT-2102: Clinical Trial Open to Recruitment

Research FOR Patients
-For an informed and empowered opinion-

All the trials listed on 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.

FT-2102

  1. SUB-TYPE OF MDS: Patients with Acute Myeloid Leukaemia or Myelodysplastic Syndrome with an IDH1 R132 mutation
  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: FT-2102
  4. Aims and benefits: FT-2102 is an inhibitor of the mutation IDH1, which is seen in 7-14% of
    This is a phase 1/2 study of FT-2102 as a single agent and in combination with azacitidine or low dose cytarabine. The study aims to determine the safety and effectiveness of FT-2102 in this specific patient group.

Read More


URGENT: A father of 7, recently diagnosed with MDS needs your help

Damian, a father of 7, needs an urgent stem cell transplant

This is an urgent appeal for stem cell donors, for a Dad who was diagnosed in July 2018 with a high-risk form of MDS, Myelodysplastic Syndromes, a rare form of blood cancer, that can progress to leukaemia.

Damian is a father of 7 lovely children – aged 4 to 27.  Damian was told that without treatment, he only had 6 months to live, as his type of MDS is severe and progressing fast. (Read more in The Scarborough News)

End of 2018, he was given intensive chemotherapy to try and reduce the severity of the disease, with a view to do a bone marrow transplant as soon as possible.

This treatment worked to some extent, but sadly, right at the beginning of 2019, the disease flared up again.

He is now looking at another cycle of chemotherapy - but of a different kind, in the hope to get him into remission, and then go through with the stem cell transplant.

If there was a matched donor available, this would be a fairly straight-forward sequence of events. But currently, there is no perfect match for Damian.

Overall, there are multiple options for stem cell transplants (or bone marrow transplants)

  • Often, a sibling can be a good match.
  • Or an unrelated matched donor can be found.
  • Sometimes stem cells from cord blood is an option.  This is when the stem cells contained in the umbilical cord of a new born baby are used.  Two umbilical cords are necessary to provide enough stem cells for 1 adult patient.

For Damian, the cord blood transplant is being considered, but staff only have 1 lot of umbilical stem cells. Another lot of umbilical cord stem cells are needed to go ahead with the transplant.

And this is where pregnant ladies and the general public can help!

There are 5 hospitals in the UK that have the facilities to collect and process the donated umbilical cord blood and stem cells.

Those are:

  • Barnet
  • Luton and Dunstable
  • Watford
  • St George’s, Tooting, London
  • UCL, London

As well as 4 Anthony Nolan collection points:

  • Saint Mary's Hospital, Manchester
  • King's College Hospital, London
  • Leicester Royal Infirmary
  • Leicester General Hospital

All details as to how to register to donate your baby’s umbilical cord after the birth can be found here:

https://www.nhsbt.nhs.uk/cord-blood-bank/donate/where-can-i-donate/

Your action is needed:

We ask all readers to share this post and information with any pregnant ladies you may know – especially in the geographical areas listed above.

If a 2nd lot of these stem cells proves to be a match for Damian Butcher, the transplant can go ahead.

The option 2: a transplant with cells donated by his brother – who is a half-match.  This is called a Haplo transplant.  Possible – but not the preferred option so far.

Alternatively, there could be another option, an adult unrelated donor – if a match could be found. We therefore ask new donors to come forward urgently to get registered. This can be done via Anthony Nolan (for those aged 16-30)  Or DKMS (for those aged 18-55)

You can read more on Damian on the Anthony Nolan site and the DKMS site.

Registration is extremely easy. Nowadays the donation part is almost pain-free and not much worse than a blood donation.

Get informed, speak to staff, consider it very seriously.

Your actions could save Damian's life.

Thank you

#donor4MDSdad

How to donate bone marrow

If you’re between 16 – 30 and in good health, sign up to Anthony Nolan's register and you could be a lifesaving match for someone with blood cancer

You Can Become a Donor!

Even if you’re older than 30 you can become a blood donor if you're in good health. Check your eligibility at the dkms site

How to Join the Register? All it takes is a bit of spit or a swab!

Your stem cells could end up saving the lives of someone facing a desperate situation anywhere in the world.

If you are called to donate, there are two simple and easy ways to give stem cells.

One method takes just 3 - 4 hours and is not dissimilar to giving blood. If a patient needs a bone marrow transplant then the consultant will be checking the worldwide registers for a suitable match.

However, it's important to encourage family and friends to join!

What happens if you are a match? Bone Marrow Donation Procedure

Your animated guide to becoming a bone marrow donor. Find out everything you ever wanted to know about donation, from joining the register to what happens if you are a match.

What are stem cells? How can they be used for medical benefit?

A part of your bones called “bone marrow” makes blood cells. Marrow is the soft, spongy tissue inside bones. It contains cells called “hematopoietic” stem cells (pronounced he-mah-tuh-poy-ET-ick). These cells can turn into several other types of cells. They can turn into more bone marrow cells. Or they can turn into any type of blood cell. Here is a short educational film by the Irish Stem Cell Foundation


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