NICE committees recruiting

NICE needs new patient/carers/clinical staff members for their guideline committee.
This is a good opportunity to help shape future haematology services in the UK.

Please contact NICE directly to apply and for specific queries – but please feel free to call us if you have general questions about NICE and how MDS UK has participated in appraisals to date.

Please see information from NICE below:

Re:  NICE Improving Outcomes in Haematological Cancers, service guidance (update)

As you may already be aware, NICE is updating ‘The Improving Outcomes in Haematological Cancers – service guidance’ which was published in 2003 (see http://www.nice.org.uk/guidance/csgho/evidence/improving-outcomes-in-haematological-cancers-the-manual2).  The National Collaborating Centre for Cancer (NCC-C) has been asked to carry out this work.

It is very important that we recruit appropriately skilled people to become members of the guideline committee (GC) and we will be advertising through a variety of professional and other channels, inviting people to apply to join the group. We would then select the most appropriate people on the basis of their experience and background.

I would be very grateful if you could circulate this e-mail to anyone within your organisation who may be interested in applying. 

We are looking for representatives from the following professions:

• Clinical Nurse Specialist x 2
• Haematologists x 3
• Paediatric Haematologist x 1
• Medical Oncologist with an interest in lymphoma
• Specialised Commissioner
• Specialist Integrated Haematological Malignancy Diagnostic Service (SIHMDS) lead
• Histopathologist
• Radiologist with an interest in haematological malignancy
• Consultant Clinical scientist with an interest in diagnosis of haematological malignancy
• Haemato-Oncology Pharmacist
• Patient / carer representatives x 2

The advert and application form for healthcare professional membership of the GDG (including the job description and person specification) can be viewed on the NICE website at: Member: Haematological cancers service guidance (update) committee group | Join a committee | Get Involved | NICE

The closing date for receipt of applications for healthcare professionals is 5.00pm on Thursday 30^th April 2015.  Interviews will be held by telephone on Friday 8^th May 2015.

We are also looking for 2 patient/carer members, the advert and application form (including the job description and person specification) can be viewed on the NICE website at: Lay member: Haematological (blood) cancer, service guidance update guideline committee | Join a committee | Get Involved | NICE

The closing date for receipt of applications for patient/carer members is 5.00pm on Thursday 30^th April 2015

A copy of the draft scope for this guideline is available on the NICE website Haematological cancers – improving outcomes (update) | Guidance and guidelines | NICE

If you should need further information or clarification please do not hesitate to get in touch.

Lianne Gwillim

Project Manager/ Rheolwr Prosiect
National Collaborating Centre for Cancer / Canolfan Genedlaethol Cydweithredu dros Ganser
2nd Floor / 2il Llawr
Park House / Tŷ Parc
Greyfriars Road / Heol y Brodyr Llwydion
Cardiff / Caerdydd
Wales / Cymru
CF10 3AF

Tel/Ffôn – 029 2040 2917
Fax/Ffacs – 029 2040 2911
E-mail/E-bost – Lianne.Gwillim@wales.nhs.uk

MDS UK running vests have arrived!

To celebrate our record number of brave marathon and other sporty events, we decided to have these running vests made – for MDS to stand out in the crowds.

This year we are so lucky to have 7 marathon runners – in Brighton and London:
Jodie Cooper, Caitlin Limmer Moni Lau in Brighton
Kevin Dwyer, Daniel Holah, Romit Basu and Georgina Cook in London

We then have many more energetic participants for the Isle of Wight Challenge:

Paul and Nessy Ungi, Andy Hadrick, Mark Bowen, Andrew Marples, Andrew Ross, Cate Skeat, Hazel Hutt, Susan Bunyan, Sarah Beardon, Verity Penzer, Shaun Hicks, Katia Pothecary

And for the Grand Union Challenge – Andy Houghton!

You can read details about each participant in our Fundraising events column – and sponsor them too.

https://mdspatientsupport.org.uk/events-1/fundraising-events-upcoming/

Many thanks to all these fantastic people giving up their time and energy to support our charity and the patients and families we assist.

We could not do it without you.

Aplastic anaemia (AA) is a rare but serious acquired bone marrow failure disorder of considerable clinical similarity to the hypoplastic type of MDS.  In same cases AA  may also progress to MDS.

Given the link between the two conditions we often collaborate with the the support group for AA patients – Aplastic Anaemia Trust. Together we organise joint fundraising and awareness raising events e.g. annual Windsor Walk in May. We also run our local Patient Support Group meetings at King’s College Hospital in London in collaboration.

Click here for details of our next AA/MDS/AML meeting at Kings.

We support AAT’s efforts in raising awareness of this very rare blood disorder and promote the Aplastic Anaemia Awareness Day on their behalf. 

Please read the AAT’s message to all its supporters below:

“On 4th March we will be holding the first Aplastic Anaemia Awareness Day in the UK when we are asking our patients, their families and supporters to help raise awareness of aplastic anaemia and the vital work of the Aplastic Anaemia Trust. The date has been chosen to coincide with the Aplastic Anemia & MDS Awareness Week in the USA.

It’s as easy as arranging a dress down day at work, holding a cake sale or encouraging friends and work colleagues to put their spare change in one of our cardboard collecting boxes. You can show your support by wearing an AAT wristband, pin badge or ribbon.

Perhaps you can educate others about aplastic anaemia by sharing your AA story in a local newspaper or radio station or use social media such as Facebook and Twitter to tell your friends about AA and the work of The AAT. The more people know about aplastic anaemia the better!

Contact Bryony at Patient Support for collecting boxes and posters. Pin badges, wristbands, ribbons and other AAT merchandise are available from our online shop or from the AAT office.

You can help us make a difference!”

Bryony Partridge, Patient Support, Aplastic Anaemia Trust

Contact details:
The Aplastic Anaemia Trust
PO Box 289
Bexhill-on-Sea
TN40 1EJ

Tel: 0870 487 0099
Email: support@theaat.org.uk

Please show your support by marking Aplastic Anaemia Day!

For further information on Aplastic Anaemia please visit the website of the Aplastic Anaemia Trust – www.theaat.org.uk

Daisy Turner and her MDS experience

We are pleased to share the story of a very brave and inspirational young MDS patient – Daisy Turner.

Daisy was diagnosed with MDS as a teenager and represents one of the uncommonly occurring cases of MDS in young people. However, her young age isn’t the only thing that makes her story unique. After being successfully treated with Bone Marrow Transplant few years ago, Daisy made a decision to dedicate her career to support other young people suffering from cancer. She now works as Fundraising Manager for CLIC Sargent – UK’s leading cancer charity for children and young people, and their families.

Her enthusiasm and commitment were recently recognised by Platform505 – an on-line site providing inspiration, thought and discussion for women who take an active part in life and have something to say.

An interview conducted by Platmorm505 with Diasy was published on their website last month – please click here to see the article.

The interview can also be found on our Patient stories page – please click here to see the stories of our members: Joseph Vella, Christina Fowler, Jayne Snell, Maynel Vessey and Daisy Turner.

More stories will be added to this page in future – to help other patients and their families to learn to live with MDS and share experiences.

Please get in touch if you too would like to share your MDS story –  whether you are a patient, family member or friend.
Email it to mds-uk@mds-foundation.org, or mail it to our main office.
You may talk about any aspect of the condition, or how your experience has affected or changed you.
Feel free to add photos as well.

World Cancer Day is a truly global event to unite the world’s population in the fight against cancer. Its aim is to raise awareness that there is much that can be done at an individual, community and governmental level, to harness and mobilise the solutions and catalyse positive change.

World Cancer Day is an initiative of the Union for International Cancer Control (UICC), a leading international non-governmental organisation that unites the cancer community to reduce the global cancer burden, to promote greater equity, and to integrate cancer control into the world health and development agenda. Please read more about UICC and World Cancer Day here.

MDS UK supports this important initiative and calls all our supporters to join in to mark World Cancer Day – and unite in MDS.

All you need to do is write #wewillunite onyour hand – MDS – and maybe the name of the person with MDS you are supporting.
Then please post this to us on mds-uk@mds-foundation.org
We will upload all photos to this webpage as a mark of support to all those battling MDS – those who survived MDS or those who sadly lost their life to MDS.

Below you can see some of the photos we’ve go already.
Lets – all together make a giant statement to mark this important day – and show how many people struggle with this almost invisible – but very difficult disease.
Show your support to one another – together we are stronger.
Thank you!

Cancer Drug Fund – 20/01/15

The CDF team was recently forced to remove 20 drugs from the list of drugs they fund – in order to balance the budget allocated to them by the Department of Health.
The CDF was never supposed to become a permanent solution to the problem of funding cancer drugs.
Instead, NHS England was supposed to set in place an improved system, via Value Based Pricing, and via a reform of NICE.
To date, and in prime pre-election time, these changes have not yet been implemented, and therefore changes had to be made to how the funds for the CDF were going to be used.

In the article below, Peter Clark explains the decision and the broader issues of cancer drug funding.
He also talks about the difficulties of obtaining data that is “robust” enough for NICE – which is often a significant problem when assessing drugs for rarer cancers – as patient numbers are low.

MDS UK is glad to hear that NICE is considering changing their threshold, to reflect how the public views cancer.
It is clear that the public views cancer as a top priority – affecting people across all ages.

As a separate point on the CDF, MDS UK was slightly disappointed to hear how much importance is accorded to Quality of Life (QOL) issues – in relation to other data.  Furthermore, the CDF insists on QOL data collected during trials only – and peer reviewed data.
This is particularly frustrating for support groups who don’t necessarily have this type of data – but are able to supply plenty of QOL patient evidence – especially when such QOL of data is missing from clinical trials.
This decision to only accept a standard and established type of QOL date seems at odds with the CDF’s suggestion they would accept to fund those drugs where the evidence is still “uncertain”.

As a reminder – no MDS drugs were removed from the CDF list.
Both MDS licensed drugs are recommended by NICE:

• azacitidine (Vidaza) – for Intermediate and high-risk MDS, AML and CMML
• lenalidomide (Revlimid) – for isolated del 5q MDS

Only Del 5q with 1 other chromosomal abnormality is currently funded by the CDF.

Further article in the Pharmaceutical Journal – 20/01/15:

“Cancer Drugs Fund has undermined NICE, its chair admits”

Comments from its Chair, Peter Clark and NICE Deputy Chief Executive, Gillian Leng

http://www.pharmaceutical-journal.com/news-and-analysis/news/cancer-drugs-fund-has-undermined-nice-its-chair-admits/20067634.article

“The Cancer Drugs Fund (CDF) has undermined the National Institute for Health and Care Excellence (NICE) and has provided a “get out of jail card” for pharmaceutical companies, says Peter Clark, the chair of the CDF.

In an address to the Westminster Health Forum on 15 January 2015, Clark, a practising oncologist, said he regrets that the CDF has undermined the NICE approval process and admitted that the system for commissioning chemotherapy in England is “broken”.

“CDF has stacked up drug indications with no long-term decisions about the future of [the fund],” said Clark.

The CDF budget for 2014–2015 is £280m but it is set to overspend by £110m, he said. In response to the overspend, a number of drugs and indications will be removed from the fund. Clark said the move, announced on 12 January 2015, was “inevitable” and that there would be further prioritisation of the drugs that are funded through the CDF.

To be eligible for CDF funding, cancer drugs are now assessed on clinical benefit versus cost but Clark conceded that the simple evaluation used by the CDF was “in no way a cost-effectiveness analysis or doing what NICE does”. “This is all we can do,” he said.

Clark wants the CDF to fund drugs that offer significant benefits to patients while they are appraised by NICE and to facilitate further data collection and evaluation.

In Clark’s vision, a positive decision from NICE would see the drug funded through “baseline commissioning” but a negative decision could lead to one of two things happening. Drugs that received a negative decision based on solid long-term data would be removed from the CDF. Where there was uncertainty about the drug’s effectiveness, further outcome data based on its use within the NHS should be collected. In turn, these data would be shared with the drug’s manufacturer, NICE and the CDF, at which point the drug would be reappraised by NICE, but this time with “mature data”.

“My view is that with this maturity-of-data process there will be many more NICE [approvals],” said Clark.

Clark believes that cancer has a special place in people’s psyche, which influences their willingness to pay for treatment. Gillian Leng, deputy chief executive of NICE, suggested that NICE may be asked to up the cost effectiveness threshold for cancer medicines. “If cancer is deemed a priority, then we’ll make it a priority,” she said.

A report by the National Audit Office was released on the same day as the Westminster Health Forum event. It reveals that cancer survival in the UK has improved since 2011 but remains about 10% lower than the European average.”

Citation: The Pharmaceutical Journal, 31 January 2015, Vol 294, No 7847, online | URI: 20067634

Leukaemia and Lymphoma Grant for research into Familial MDS

MDS is not considered a hereditary disease – but there is a very small percentage of cases where 2 or 3 members of the same family are affected.

This is why Leukaemia & Lymphoma Research have granted the Barts Cancer Institute £1.4 Mio pounds for research into this really important area.  This grant will help researchers get closer to understanding why this happens – and help families at risk.

MDS UK looks forward to working with Barts and LLR to support MDS families affected by this diagnosis.

From the Barts Cancer Institute website:
http://www.bci.qmul.ac.uk/news/grants-a-awards/item/llr-grant-fml-bci-blizard

What are the research aims?

The programme begins in April 2015 and aims to uncover the cause(s) of familial myeloid leukaemia (FML) that include principally acute myeloid leukaemia (AML) or myelodysplastic syndrome (MDS) and to provide the necessary companion diagnostics to see improvements in diagnosis and management of these groups of patients.
Current challenges for researchers and clinicians working on FML include:

•     It is an under-reported condition
•     Patients themselves may be unaware of their familial predisposition
•     The genetic basis is known in fewer than half of affected families
•     Large variation in the age of onset, disease manifestation and transmission via otherwise healthy mutation carriers
•     Absence of customised diagnostic techniques to meet the clinical needs of this patient group and their families.
•     Few clear clinical guidelines
•     Little known regarding “clinical heterogeneity” (differences in symptoms), or the nature of secondary mutations impacting on disease outcome

Professor Dokal said:

“This research award represents the most comprehensive and integrated programme to date internationally. We aim to change our understanding of this group of diseases as well as clinical management and outcomes. The research will be a collaboration, drawing on expertise across the medical school and we look forward to getting underway.”

The project’s aims include:

• Identify and characterise new cases of leukaemia and MDS to build our understanding of this group of life threatening disorders.
• Determine the genetic variability of the disease including what governs its progression from mild to severe.
• Find and study novel disease-related genes, as many patients remain uncharacterised at the genetic level.
• Improve clinical management by establishing centralised genetic testing of patients with known or suspected of having an inherited   component and the development of specific guidelines and a patient support group.

Professor Fitzgibbon and Dr Vulliamy summarised:

“Studying larger series of familial cases will provide an unique insight into these diseases compared with  looking at sporadic cases alone; when every case has the same starter mutation we hope to gain an understanding of disease latency (time taken for the onset of overt disease) and offer explanations for the heterogeneity in outcome seen both within and between families”

Professor Dokal went on to say:

“Collectively these studies will enable us to determine the causes of leukaemia and familial myelodysplasia (MDS). From this we hope to develop new diagnostic genetic tests for these patients, and more accurately predict the number of patients with these disorders to facilitate better planning of health services. Together, this will significantly improve clinical outcomes for patients.”

Patient Support

Part of this project is focused on helping patients and their physicians correctly manage current and future treatments. For example, while we would often select people’s siblings as bone marrow donors if the need arises, a sibling with no symptoms could not donate for MDS, as they might also have mutations despite not experiencing the disease themselves.

As well as the medical practicalities, there is the need for personal support. The internet has provided useful spaces for patients with rare diseases to share their experience and discuss challenges in understanding and helpful environments – together with patients, our researchers will help set up such groups.

Researchers awarded a £1.4million from the charity Leukaemia and Lymphoma Research

12 January 2015
http://blizard.qmul.ac.uk/about/news/1053-researchers-awarded-a-1-4million-from-the-charity-leukaemia-and-lymphoma-research.html

A team of researchers from the Blizard Institute (Professor Inderjeet Dokal and Dr Tom Vulliamy) and Barts Cancer Institute (Professor Jude Fitzgibbon) have been awarded a Specialist Programme Grant of £1.4million from the charity Leukaemia & Lymphoma Research. This award constitutes a five-year programme entitled ‘The biology and management of familial myelodysplasia and leukaemia’ and will begin in April 2015.

The team aims to identify and characterise new cases of familial myelodysplasia (MDS) and leukaemia, determine the genetic variability of the disease including progression from mild to severe disease, and improve clinical management by developing specific clinical guidelines and establishing a patient support group.

This research award represents the most comprehensive and integrated programme to date internationally, as it aims to provide a step change in the basic understanding of this group of diseases as well as in clinical management and outcomes.

TED talk on MDS/AML research by expert Dr Azra Raza.

Published on 15 Jan 2015

Cancer research relies heavily on testing drugs in mice — yet it’s clear that for effective treatment, we need to be testing in humans. In an informative talk, oncologist Azra Raza walks us through the challenges of personalized cancer research and offers a potential solution, based on decades of sample collection.

Oncologist Azra Raza is the director of the MDS Center at Columbia, where she moves the latest cancer therapies from theory to practice.

This talk was given on Saturday, November 1, 2014 at TEDxNewYork. The theme was “Grand, Central.” For more information, visit http://tedxnewyork.com or follow http://twitter.com/tedxnewyork.

More news to on fundraising event set up to help research on MDS in the USA:

The Columbia University Medical Center has benefited from a great fundraising concert, to help fund further research into MDS.
Read more here:

http://www.nearnessofyouconcert.org/

“Cancer has either directly or indirectly touched practically everyone. Since the sequencing of the human genome, a host of rapidly evolving technological advances have thrown open exciting new vistas for therapy driven research, with the aim ofcuring several cancers. Thanks to the work of hundreds of thousands of investigators over the last several decades, we are finally witnessing encouraging developments in several types of cancers already.

Columbia University Medical Center (CUMC) finds itself at an auspicious moment with the opportunity to dramatically change the course of Myelodysplastic Syndrome (MDS) research, treatment and patient care. Every element needed to discover a cure for MDS is here—our ability to perform research with the largest group of patients and tissue samples; the technology and facilities offered by the Herbert Irving Comprehensive Cancer Center (HICCC); the capacity to approach every area of patient care; and our access to the best clinical staff in the field. CUMC is eager to capitalize on this rare opportunity to become the leader in MDS care in the country, and the world.”

Performers and Line up:

Hosted by Deborra-Lee Furness and Hugh Jackman. The Nearness of You Concert in honor of Michael Brecker. Special performers included Paul Simon, James Taylor, Dianne Reeves, Randy Brecker, and Bobby McFerrin.

This further article provided more information about Michael Brecker’s battle with MDS:
http://www.guitarworld.com/acoustic-nation-paul-simon-and-james-taylor-headline-concert-benefit-cancer-research

“On Tuesday, January 20, 2015, Paul Simon, James Taylor, Bobby McFerrin, and other prominent musicians will honor the memory of their friend and colleague, 15-time Grammy-winning saxophonist Michael Brecker, at “The Nearness of You” benefit concert in New York City.

Hugh Jackman and Deborra-lee Furness are the benefit chairs, and Meredith Viera will serve as master of ceremonies.

Proceeds from the concert will support cancer research at Columbia University Medical Center (CUMC) and the work of Azra Raza, MD, and Siddhartha Mukherjee, MD, PhD. Dr. Raza is director of the Myelodisplastic Syndromes (MDS) Center at CUMC.

Dr. Mukherjee is a physician and researcher at the MDS Center. In 2011, he won a Pulitzer Prize for The Emperor of All Maladies: A Biography of Cancer.

In 2004, Brecker was diagnosed with MDS. In need of a bone marrow transplant, he searched in the International Bone Marrow Registry for a match. He failed to find a matching donor, and in 2007 he died at age 57.

As a result of the tens of thousands of people worldwide who registered in an effort to save Brecker’s life, 54 lives were saved when new registrants were perfect matches for others in need.

“When I learned that a cure for MDS will likely provide the key to curing leukemia, I realized there was no better way to honor my husband’s legacy than by raising important funds for research through this benefit concert,” said Brecker’s widow, event producer Susan Brecker.

Partial support of the “The Nearness of You” concert has been provided by Novartis. The concert will be held in The Appel Room at Jazz at Lincoln Center’s Frederick P. Rose Hall. General tickets, starting at $200, may be purchased as of Monday, November 17, at The Jazz at Lincoln Center box office, located on the ground floor of the Time Warner Center (Broadway and 60th Street), by Center Charge phone at 212-721-6500, or online at www.jazz.org/events/all/”

This concert helped raise $1.2million dollars – which will help this particular research center to work further on MDS and AML.
Together with all the work done in all international haematology experts centres – research is getting closer to a cure for MDS and AML.