Press Office
16.02.12
Organ Donation, CFT response to Welsh Government consultation
The Cystic Fibrosis Trust recently responded to the Welsh Government's consultation on changing organ and tissue donation legislation. After consulting with physicians, people with CF and their families, we offered our support for the proposals to change to a soft opt out system of organ donation. A soft opt out system means that as well as people having a chance to opt out of donating their organs during their lifetime, after death relatives are involved in the decision making process surrounding donation.'
As the only UK wide charity representing people with Cystic Fibrosis, the CF Trust has a role to play in increasing the rate of organ donation, to make sure that those patients requiring transplant (be it lung, heart-lung or liver) have the best possible chance of survival. For further detail, 
please read our full consultation response.
01.02.12
The Cystic Fibrosis Trust has welcomed US Food and Drug Administration approval of KALYDECO (Ivacaftor), from Vertex KALYDECO has been approved for use in people with CF ages 6 and older who have at least one copy of the G551D mutation. About 4 per cent of people with CF have this mutation and this first medicine to correct this genetic fault will significantly improve length and quality of life.
The CFTrust has learned that this will be available to people in the US from the end of the week. The charity now wants to ask the European Medicines Agency (EMA) to push the approval process in the EU through quicker, to allow this medicine to become available for patients with CF in the UK.
Matthew Reed, Chief Executive of the CF Trust said:
"The CF Trust is delighted that Kalydeco has been granted FDA approval. This is fantastic news for people living with CF and heralds a new era of drugs addressing the basic defect in CF. We now hope that the European Medicines Agency (EMA) will complete their own assessment as rapidly as possible so patients in the UK can have access to the same drugs as patients in the US. We will also be looking closely at the cost of providing this drug in the UK's CF Clinics."
ENDS
For all media enquiries, please contact: Louise Banks on 0208 290 7912 or email lbanks@cftrust.org.uk
Notes on KALYDECO KALYDECO targets the abnormal CFTR protein channels and opens them to allow chloride ions to move into and out of the cell, which helps thin the mucus so it can hydrate and protect the airways, and keeps them from getting clogged and then infected. Because KALYDECO targets a specific genetic mutation, a person's genotype should be known before this new medicine is prescribed. Genetic testing is widely available and FDA-cleared tests are available for people with CF whose genotype is unknown. According to the 2010 Cystic Fibrosis Foundation's Patient Registry, nearly 92 percent of people with CF have already had their CF mutations identified. More information on the drug is available from www.vrtx.com
STAYING ALIVE, DAD WITH CF BEATS THE ODDS
Cystic Fibrosis Week 29 April - 5 May
A father who suffers from Cystic Fibrosis has sent a message to other people with CF to fight on and beat CF. Tim Wotton, a keen blogger and father to four year old Felix wanted to encourage people to support the CF Trust during CF Week.
Tim has already beaten the odds, according to the latest CF Registry Data collected by the CF Trust the median survival age for someone with CF is just 41 (1), Tim who is now 41 and an ex-England Hockey player is fighting hard to stay fit and will be celebrating CF Week with his Son Felix who turns five during CF Week.
Cystic Fibrosis is caused by a defective gene that results in the internal organs, especially the lungs and digestive system, becoming clogged with a thick sticky mucus resulting in chronic infections and inflammation in the lungs and difficulty absorbing food. There is no cure for Cystic Fibrosis. People with CF cannot meet each other in case they pass on different infections making CF an isolating condition.
Tim said:
"I have been determined that CF isn't going to beat me, and even more so now that I have my lovely wife Katie and my son Felix who turns five during CF Week. I know as many others with CF do that it is a horrible disease and a daily battle to keep well. I have been using a regular blog to inspire others and to help people without CF to see what effect it has on your life.
"The CF Trust has been working to fund research in to treatments that will extend and improve the lives of people with CF, it deserves our support now and during their annual awareness raising week."
Matthew Reed, Chief Executive of the Cystic Fibrosis Trust said:
"People like Tim are an inspiration for others with CF, in particular with his regular blogs which give people an idea of what life is like for someone with CF. The CF Trust believes that everyone living with CF deserves the highest level of care and real hope for the future. This CF Week we hope that there will be an event going on in every part of the UK, in which everyone can get involved in and raise vital funds so that we can continue to strive to help people with CF live a longer and better life."
The Cystic Fibrosis Trust Week will also explore the isolation that living with CF brings, with cross-infection risks. Visit www.cfweek.org.uk for more information and to see the stories of three families affected by the condition. The site will be updated with online videos, lists of events and much more leading up to and during the start of CF Week.
ENDS
For all media inquiries and to speak to our exclusive case studies please contact:Louise Banks on 0208 290 7912 or email lbanks@cftrust.org.uk
(1) The Median predicated age of survival is taken from the UK CF Registry Annual report 2010, this is the age at which the majority of people with CF survive to but some live much longer.
Tim Wotton lives in Morden with his wife and son and works as an Internal Communications Consultant, he also writes a regular blog http://timwotton.wordpress.com about his life with CF.
Sterimar nasal spray product recall
The Medicines and Healthcare products Regulatory Agency (MHRA) has received warning of possible bacterial contamination in batches of the saline nasal spray Sterimar Isotonic 100ml. The affected batches are FE1248b and FE1249. The batch number can be located on the product packaging and on the base of the can. Anyone with CF who has been using the affected batches is advised to discontinue use and notify their CF team. For more information see the MHRA press release.
CF Trust Trustees adopt Living Longer, Living Better; Our vision for 2016.
Matthew Reed, Chief Executive, said, "The Cystic Fibrosis Trust believes that everyone living with CF deserves the highest level of care and real hope for the future.
Read our strategy; Living Longer, Living Better: Our vision for 2016
"Over the last decade significant progress has been made in a number of areas to fight CF and the median predicted survival in the UK has increased to reflect this. However, despite the opportunities there are still substantial challenges to be urgently addressed to make future hope a reality.
Living Longer, Living Better: Our vision for 2016 defines our ambition and focus of the next four years. The strategy defines the Trust's direction and focus across a number of areas with the overarching goal of ensuring that life affected by CF is not life limited by CF.
In order to reach that goal, the Cystic Fibrosis Trust will focus its work over the next four years on four key areas:
- Increasing length of life for those with CF
- Improving quality of life for those with CF and their families
- Increasing capacity of CF research and clinical care
- Expanding awareness of CF and its impact on individuals and families
By concentrating on these areas, the Cystic Fibrosis Trust, the only UK-wide CF - specific charity, will become an organisation which makes a positive impact, not one that simply focuses on output. Living Longer, Living Better will be underpinned by an ambitious and urgent research programme, transformational clinical care agenda, and stretching fundraising targets to facilitate it. This is an agenda of partnership with clinicians and other health care professionals, researchers, industry, other charities, the Department of Health, and other actors.
During the development of this strategy, the Cystic Fibrosis Trust has worked with the CF community - with adults with CF and the parents of children with CF - as well as clinicians, and research professionals. While this is the Cystic Fibrosis Trust's strategy, it is driven and inspired by them."
Standards of Care
The Cystic Fibrosis Trust has worked with its Medical Advisory Committee and with NHS Trusts through its Peer Review process to ensure that the all care provided to patients with CF is of a consistent quality. Good quality care enables children and adults with CF to have an improved quality of life and extended life expectancy.
Matthew Reed, Chief Executive of the Cystic Fibrosis Trust said:
"The CF Trust believes that everyone living with CF deserves the highest level of care and we are committed to doing all we can to make that happen. This is the second Standards of Care document that the CF Trust has published and we hope this will provide an in-depth manual of what good care provision for people with CF looks like.
"The Standards of Care will help ensure that all people with CF have access to the best quality care and treatment possible and end an unfair 'postcode lottery' for CF services."
The Standards of Care 2011 is published online and will be of use to Clinicians and allied health professionals, commissioners and other NHS services responsible for the care of people with CF. Parents and carers of children with CF and older children and adults will also find it useful to understand what level of care they should expect.
Fast track for Kalydeco
The European Medicines Agency (EMA) have now started the fast track process to review Kalydeco (VX-770) for licensing in the EU and UK. This exciting news has also been mirrored in the US where the Food and Drug Administration (FDA) have agreed to fast track Vertex's application to have Kalydeco licensed in the US. If all goes well, it is hoped that the drug will be available in 2012, the first of a new generation of CFTR modulators that address the basic defect in CF.
Jenny Agutter opens Sheffield Hospital's new CF Unit
On Monday 12 December, Patron of the CF Trust, Jenny Agutter, opened the new £2.5 million Adult CF Unit at Sheffield Hospital. The CF Trust has donated a considerable sum to this project. The other fundraising has been completed by Sheffield Hospitals Charity, which has just £20.000 left to raise to reach the target amount.
The new Ward features 12 en-suite rooms with entertainment facilities and internet access. Each room is decorated with themed muriels to take away the feeling of being in a clinical environment. The opening was featured on BBC Look North and BBC Radio Sheffield and the Yorkshire Post. Speaking about the newly open ward, Matthew Reed, CEO of CF Trust said:
"We are delighted that this new ward will open for patients with Cystic Fibrosis in Sheffield and hope that it will be a model for other hospitals around the country. The Trust is proud to have made a substantial contribution to this new ward which provides the very best facilities for adults with CF. Key to improving wellbeing is often the environment patients are living in. With en-suite facilities, gaming and the opportunity to keep in touch through the internet, going into hospital for treatment will not be as isolating as it can sometimes be."ISOLATION A BIG FACTOR FOR FAMILIES AFFECTED BY CF
The Cystic Fibrosis Trust has welcomed today's report from Contact "Family Forgotten Families - The impact of isolation on families with disabled children across the UK'.
Matthew Reed, Chief Executive of the Cystic Fibrosis Trust said:
"The findings of this survey will not be a surprise to the thousands of families coping with Cystic Fibrosis across the UK. In our own recent online consultation many families spoke of the isolation they feel, as cross infection control rules out people with Cystic Fibrosis meeting face to face.
"That is why the Cystic Fibrosis Trust has been highlighting the damaging cuts to Cystic Fibrosis services, including psychosocial support, which families say is so vital - As one mother said, 'We need more support in the community for young adults with CF. My son struggles psychologically with his condition and has now gone to University, there he has no support and is on his own'."
ENDS
· Contact a Family conducted an online survey between July and September 2011 for families with disabled children on the subject of isolation. A total of 1,148 families with disabled children responded. Contact a Family has produced a report on the back of these survey findings. Forgotten Families - The impact of isolation on families with disabled children across the UK which can be requested, as well as a full breakdown of the survey findings, from the press office on 020 7608 8741/8776.
The Cystic Fibrosis Trust supports research that can improve and lengthen the lives of people with CF. TORPEDO-CF is an important clinical trial, which looks at the best way to eradicate Pseudomonas infection, in its early stages. The trial compares intravenous with oral treatment. TORPEDO-CF is already open in 35 CF centres in the UK, with many more to follow over the coming months. If you or your child have a new Pseudomonas infection, diagnosed in your CF clinic, please ask your doctor about the option of taking part in TORPEDO-CF.
If you would like to know more about TORPEDO-CF here are some ways you can get more information:
· Visit the TORPEDO-CF website: http://www.torpedo-cf.org.uk/
· There are more details on http://www.controlled-trials.com/ISRCTN02734162/
· Contact the Clinical Trials Unit in Liverpool: Tel: 0151 282 4714 or E-mail torpedo@liverpool.ac.uk"
BBC Three film
Don't miss BBC Three documentary, "Love on the Transplant List" Monday 28 November at 9pm.
It tells the story of Kirstie Mills, 21, whose only hope of survival is a lung transplant. Given only six months to live, the documentary charts her life through her marriage and beyond.
Gene Therapy Appeal Update - 9 November 2011
Today, the EME Programme published the minutes of their board meeting held on 25 and 26 October.
The board of the EME Programme has made an "in principle" decision to fund the study (the application entitled "A randomised double-blind placebo controlled Phase 2B clinical trial of repeated application in gene therapy in patients in cystic fibrosis") subject to a number of changes being made.
The final decision will be made when the changes to the project are agreed with the GTC. The time this takes will depend on the scale and complexity of the changes. The EME programme conducts a comprehensive scientific peer review to ensure the study is further strengthened and this is all part of the standard process for EME Programme funded studies.
Matthew Reed, Chief Executive of the Cystic Fibrosis Trust said;
"We are delighted to hear that the EME Programme is keen to fund this clinical study. This is subject to some changes being made to the gene therapy application which could take some time. This is very much part of the usual process once a funding decision has been made. This is very encouraging news and we hope the changes can be agreed as soon as possible. We are in close communication with the GTC and will keep our website up to date as more information becomes available."
"In the meantime, donations to the Gene Therapy Appeal are still coming in and we continue to accept donations to the appeal online"
Questions and Answers
1. Did the GTC receive the whole grant?
The EME programme has decided to fund this clinical study in principle, subject to some changes being made in the application, as a result of the scientific peer review. The GTC and EME Programme will now go into a period of discussion to determine the details of the study and the size of the grant. Applications are managed through a robust, peer-reviewed process, which ensures scientific rigour and value to the public purse; this is all part of the standard process for EME Programme funded studies. There is no doubt that this peer review will add value and will further strengthen the study going forward.
2. How long is it likely to be before any clarifications or changes are agreed between the GTC and the EME Programme?
It is difficult to put a clear time line to this, the project team will need to discuss and implement the required changes. Once this has been done, a contract has to be drawn up and agreed between the funder and those responsible for the research. Once this contract has been signed the GTC can start the research, subject to the necessary research governance being in place.
3. If all the changes requested are made, will the trial then go ahead?
The trial has the biggest chance of going ahead once the necessary research governance has been put been in place and a contract has been signed between relevant parties. We will update our website as more information becomes available.
4. How much has the Gene Therapy Appeal (exclusive of the EME Programme grant) raised to date? And how will these funds be used?
As of 8 November 2011, the Appeal has raised more than £1.5m. We are very grateful to the CF community and all their amazing efforts to ensure this appeal is successful. We continue to fundraise for the full cost of the Gene Therapy Programme this year and the trial preparations.
Latest coverage and fundraising news from the Gene Therapy Appeal latest news.
- Watch ITV Meridian's coverage of the Appeal, featuring CF Trust Chief Executive Matthew Reed, Professor Stuart Elborn and Ollie and Nush's Ollie Dylan.
- Kirstie Tancock, nee Mills, was featured in the Mail on Sunday, 16 October.
- Watch BBC Points West, 13 October at 6.30pm. Phillip Kelly and his daughter Mary who has CF are featured in a progamme about life with CF.
- The Gene Therapy Appeal featured on featured on The One Show. The piece was presented by Alex Stobbs, the composer with CF.
- The Gene Therapy Appeal feature on STV news during the 6pm bulletin, June Ross and a family affected by CF were featured.
- BBC Oxford: 1hr long feature on the Gene Therapy Appeal - online until Sunday 2 October
- Cystic fibrosis cure under threat from £6m cash crisis in The Guardian
- Two young cystic fibrosis sufferers talk of their hope offered by gene therapy in Daily Record
- On Friday 30 September, a team of seven rowers started across the channel to raise money for the Cystic Fibrosis Trust and King's College Hospital - listen to the radio interview on BBC Sussex and donate to the team
Fundraising news
LATEST NEWS STORIES
Glasgow Warriors name CF Trust as third Charity partner
23/11/11
Glasgow Warriors are delighted to welcome the Cystic Fibrosis Trust as the third official charity partner that the club will support over the next two seasons.
Like Hearts and Balls and Yorkhill Children's Foundation, the Cystic Fibrosis Trust will be assisted with player appearances, charity events and the opportunity to fundraise directly at matches and through the Warriors' official website.
The Trust was nominated by Warriors staff as a cause that has personal resonance for them.
Glasgow Warriors chief executive Kenny Baillie commented: "We're privileged to be welcoming the Cystic Fibrosis Trust as our third official charity partner.
"The cause is close to the club's heart, as we know the devastating effect that the condition can have. With the work being done on gene therapy, there is a real prospect of advances that could have genuinely life-changing implications for thousands of people.
"The aim of the partnership is to help in some small way to raise awareness, and ultimately funds, for this incredibly valuable project."
June Ross, regional fundraising manager for the Cystic Fibrosis Trust in Scotland, said: "We are thrilled that Glasgow Warriors have chosen the Cystic Fibrosis Trust as an official charity partner.
"High-profile rugby clubs like the Warriors can really boost the public's awareness about this life-threatening condition while providing the opportunity to fundraise and make a real difference to over 9,000 youngsters across the UK.
"The CF Trust relies on fantastic opportunities like this to carry on its vital research work to find a treatment or even a cure that would prevent two people a week dying from Cystic Fibrosis. We are very proud to be working with Glasgow Warriors." Read more about the partnership.
Concerted new effort to correct the F508del mutation
Genzyme, now part of Sanofi, has announced a new programme with the US CF Foundation to develop small molecule approaches to correct the most commonly occurring mutation in CF, F508del. Genzyme, who also produce the lipid used in the gene therapy research, will be testing millions of molecules to see if the F508del mutation can be corrected through a molecular approach.
Professor Stuart Elborn, chair of the CF Trust's research advisory committee has said, "This new programme is very good news for people with CF who have the F508del mutation. It comes on the back of the breakthroughs from Vertex with Kalydeco with the less common gating mutations. We wish Genzyme all the best with their new programme." Read the press release.
Still a long way to go with Royal Brompton Hospital
Matthew Reed, Chief Executive of the Cystic Fibrosis Trust said:
"We note the outcome of the judicial review about the decision making process on proposals to close the children's heart surgery unit at the Royal Brompton. The Trust also recognises that the process still has a long way to go. The Trust's concerns remain the same and we want to ensure that there continues to be consistent and high quality services for Cystic Fibrosis across the UK.
"The proposed withdrawal of children's cardiac surgery from the Royal Brompton Hospital will have a knock-on effect and we believe that this could make this world leading children's CF service unviable. The RBH is a leading national and international CF Specialist Centre for adults and children. It is vital that paediatric anesthesia and intensive care services are retained to support high quality specialist CF care as well as the world leading research into CF that is currently conducted at the RBH.
"We are extremely concerned about the lack of adequate contingency planning to consider how the current 330 children with CF at the RBH will be treated if the current service model is forced to change. It would be an outrage if the 'Safe and Sustainable' review
of pediatric congenital cardiac surgery services made CF services for some of the UK's most vulnerable children unsafe and unsustainable.
"The CF Trust supports this parents' campaign to retain the current high quality specialist CF care provided for children at RBH, and therefore to keep all services open. We have written to the commissioners, the Secretary of State for Health, and other organisations involved in this review to ensure that the best possible outcome for families living with CF is achieved, as we have done with other CF Services across the UK when they have been threatened. Our primary concern is that safe and appropriate care for all CF patients remains of a high quality and that specialist CF services are fit for purpose."
Statement on Pharmaxis drug, Bronchitol, Inhaled Mannitol
Jo Osmond, Director of Clinical Care & Commissioning for the Cystic Fibrosis Trust has welcomed the announcement on the approval of a new medicine to treat Cystic Fibrosis, she said:
"The CHMP's (Committee for Medicinal Products for Human Use) approval of Bronchitol, developed by Pharmaxis, is very welcome for the CF adult community in Europe. We hope that ongoing trials in the younger CF population will have a similar positive outcome so that children can also enjoy the benefits of this new treatment. The CF Trust is always keen to support development of new treatments which help to improve the quality of life for people living with CF".
Named, a new drug that could improve the lives of 4 per cent of people with CF
There is good news on drug development for some people with cystic fibrosis. A new drug from US pharmaceutical company Vertex, has been named as KALYDECO™. Vertex has submitted KALYDECO™ to the Federal Drug Agency for approval.
The drug, which is designed to be taken as a pill, has the benefit of treating the underlying cause of CF by correcting the faulty CF gene mutation G551D, known as one of the 'gating' mutations where CFTR proteins are present but don't function properly. Phase III trials have shown significant improvements in lung function in people with this mutation, which affects around 4% of people in the UK.
The drug will also be submitted to the European Medicines Agency at the end of October, it is anticipated that this could be approved within six to nine months. Matthew Reed, CEO of the CF Trust said:
"The Cystic Fibrosis Trust welcomes this advance which will have the benefit of improving lung function and quality of life for some children and adults with CF living in the UK. This is the first drug that has shown to improve the underlying cause of CF."
CF Trust signs up to Ask for Evidence campaign
Helping ensure claims on new treatments are evidence based and honest. View supporters of Sense About Science here.
6 weeks to raise £6 million The CF Trust has today launched a campaign to raise £6 million in just six weeks to fund the innovative gene therapy trial. Thousands of people living with the genetic condition, Cystic Fibrosis (CF), may be given the opportunity to enjoy a better quality of life if this new innovative gene therapy trial goes ahead and proves successful.
The UK CF Gene Therapy Consortium, funded by the Cystic Fibrosis Trust, has been working for ten years to find a way of inserting a healthy gene into a CF patient's lungs to ensure that the damaging effects of the faulty CF gene are stopped in their tracks. They are now at an exciting stage and are ready to test the product on patients in a multi-dose clinical trial which aims to determine whether this new treatment works. The charity is pushing to raise the £6 million needed to fund the trial.
Emma Lake, an Expert Patient Adviser for the CF Trust and a trial participant said: "Those who have been selected for these trials have gone through lots of tests and are ready to start these exciting trials. We are hoping that the result will be a way to stop the damage to CF patients lungs. It will be so disappointing if this extra money is not raised by the end of October."
Matthew Reed, Chief Executive of the Cystic Fibrosis Trust said: "We have to date raised well over £30 million to develop this ground breaking treatment which has the potential to lengthen a CF patient's life by many many years. We are hoping that the funding for this trial will be made available to us by 31 October so that we can give thousands of CF families throughout the world hope in achieving a better and longer life."
If the money is raised the trial will go ahead in the Spring 2012. There are events across the country to raise money for the CF Trust's Gene Therapy Appeal. Donations to the appeal can be made at: http://uk.virginmoneygiving.com/CysticFibrosisGeneTherapy-1 More information about Cystic Fibrosis is available from the Cystic Fibrosis Trust's website.
TOBI Podhaler from Novartis
TOBI® Podhaler® (tobramycin inhalation powder), a fast and simple antibiotic inhaler, launches in the UK to reduce treatment burden for people with cystic fibrosis.
This new dry powder form of the established antibiotic tobramycin comes in a hand-held inhaler called the Podhaler for suppressing chronic Pseudomonas aeruginosa lung infection in people with cystic fibrosis aged 6 years and older.
Data show this new formulation is fast and convenient, significantly decreasing treatment time by 72% compared with nebulised TOBI® (tobramycin nebuliser solution).
This is the first ever antibiotic pod haler and is a development the CF Trust welcomes.
Seasonal Flu
Two people living with CF explain why it is so important to get your seasonal flu jab done as soon as possible. 
Dominic, who is 44, was diagnosed with CF at birth. He has the jab every year.
Dominic explains:"I've always had it. Last year I didn't get it at the first opportunity because I missed the first round of jabs due to not being notified at the time and then I had to have it when I wasn't well, which wasn't ideal.
"I think some people with CF who are well at the time may not feel it is necessary and then there are maybe some who are fearful at the thought that the flu jab might bring them down. But getting the flu could damage your lungs further in the long term.
"The implications of not having it don't bear thinking about. I would say have it done, regardless of a few little side effects you may or may not have." 
Linda Madden has had a heart and lung transplant in 2003, before having the transplant she had a bad reaction to the Seasonal Flu Jab. It made Linda feel like she had Flu symptoms, After the transplant she became too scared to have the jab. The last three years Linda has been battling with rejection of the transplanted organ, any serious respiratory infection could be very serious for her health.
This year, Linda thinks she must have the jab, as in December 2010, she contracted Swine Flu. She explains:
"Last December I caught Swine Flu, I thought the reaction was bad from the vaccination but it was nothing compared to this. At first I thought it was a cold but it got to the stage where I started to find breathing difficult and felt absolutely terrible, and had to go in to the Papworth Hospital and was given Tamiflu. They gave me Oxygen which helped but I didn't get home until Christmas Eve. I now have to have Oxygen at home just in case which is a set back.
"I would urge anyone with CF to get the jab done, even if you think it gives you side effects. Because of what happened to me, I will have to force myself to have it this year I can't risk any more serious infections".
Dishwashers and CF
You may have seen some articles in the press today regarding fungi in dishwashers. Virtually any moist surface will harbour germs and most bugs including Pseudomonas aeruginosa are found in environmental sites including washing machines, dishwashers, damp dishcloths etc. However although these sites harbour bacteria that does not inevitably mean they all present a serious risk of infection to someone with Cystic Fibrosis. In addition the fungi described in this paper Exophiala dermatitidis is considered to be a harmless coloniser of the airways of Cystic Fibrosis (CF) patients and there is no evidence to suggest it is pathogenic in the CF lung. Life for CF families has to be a balance between unnecessary risk of infection and quality of life.
We would advise that people with CF and their families always use their dishwasher at the highest possible temperature to ensure eradication of as many organisms as possible, and also maintain and clean their dishwasher regularly in accordance with the manufacturers advice.
To Ask an Expert a question email asktheexpert@cftrust.org.uk
Gene Therapy Trial - new appeal!
A multi-dose clinical trial for CF gene therapy is planned to start in Spring 2012. This is a crucial stage in the development of this promising therapy but we need to raise £6m to ensure the trial goes ahead. This is a huge financial challenge but we cannot afford to miss this one-in-a-lifetime opportunity.
Read more
Donate now
NHS Future Forum and Health & Social Care Bill
The Cystic Fibrosis Trust has welcomed the Coalition Government's decision to allow a greater role for specialist clinicians and patients and more investment in training. The decision came following the publication of the NHS Future Forum's report and recommendations on the Health & Social Care Bill. Read our statement.
Congratulations Rosie!
Congratulations to Rosie Barnes, former Chief Executive of the Cystic Fibrosis Trust, who has been awarded an OBE for services to healthcare in the Queen's Birthday Honours List. Rosie spent 14 years at the Cystic Fibrosis Trust and during that time the charity tripled in size and income. Rosie was directly responsible for bringing together three academic groups at Edinburgh, Oxford and London to create the UK CF Gene Therapy Consortium. She also initiated the Peer Review programme of CF Centres and Clinics which has vastly increased the quality of care delivered to CF patients across the UK and ran a successful campaign which led to the introduction of national neonatal CF screening in 2007.
Vertex G551D trial
New data from a Phase 3 study of VX-770, an investigational oral medicine in development from Vertex Pharmaceuticals for a specific type of Cystic Fibrosis (CF), were presented on Friday, June 10 at the 34th European Cystic Fibrosis Society (ECFS) Conference in Hamburg, Germany. VX-770 targets the defective protein that causes CF in approximately 4 percent of CF patients with the gene mutation known as G551D.
As reported in a top line data announcement in February, VX-770 demonstrated statistically significant profound improvements in lung function of 10.6 percent compared to placebo at week 24 - the data presented at ECFS confirm these results were sustained through week 48. Additional newly reported data showed statistically significant patient-reported outcomes, including improvement in the respiratory domain, including coughing, congestion, wheezing and other respiratory symptoms.
"Improving lung function is vitally important in the treatment of cystic fibrosis, but the disease affects many organs in a person's body," said Professor Stuart Elborn one of the study's lead investigators. "These data are significant because they are the first to show that treating the underlying cause of cystic fibrosis may have profound effects on the disease, even among people who have been living with it for decades. The remarkable reductions in sweat chloride observed in this study support the idea that VX-770 improves CFTR function thereby addressing the fundamental defect that leads to CF."
"In this study, people treated with VX-770 experienced significant improvements in lung function and reductions in sweat chloride within the first two weeks of treatment, and these benefits were sustained through the nearly year-long study," said Robert Kauffman, M.D.,
Ph.D., Senior Vice President and Chief Medical Officer for Vertex. "Given the significance of these findings, we are moving quickly to submit regulatory applications for approval in the United States and Europe in the second half of 2011."
Matthew Reed, Cystic Fibrosis Trust: "This latest data from Vertex is great news for those people with CF who have the G551D mutation, around 4% of the CF population. This is the first drug shown to treat the underlying cause of disease, rather than just treat the symptoms, and the trial has shown can increase lung function by around 10%."
Vertex F508del trial
Vertex have announced interim results from the first part of a Phase 2 study designed to evaluate a combination of VX-770 and VX-809, Vertex's medicines in development that aim to treat the defective protein that causes cystic fibrosis (CF). The data shows that a statistically significant reduction in sweat chloride was recorded in F508del patients given the oral VX-809 compound followed by dosing of a combination of the VX-770 and VX-809 compounds. Sweat chloride is the key diagnostic test for Cystic Fibrosis. Vertex intends to initiate the second part of this study in the fourth quarter of 2011 after the completion of further analyses of data from Part 1. Peter Mueller, Ph.D., Chief Scientific Officer and Executive Vice President of Global Research and Development at Vertex, said: "These data open the door to the possibility of treating people with the most common form of cystic fibrosis (F508del mutation) by using two medicines together that target the defective protein that causes the disease. We look forward to beginning the second part of this study later this year, which may help us begin to further explore the hypothesis that enhanced CFTR function may result in meaningful clinical benefits for people with cystic fibrosis."
New clinical trials centres announced
The Cystic Fibrosis Trust backed European Cystic Fibrosis Clinical Trials Network (CTN) has this week admitted two new UK centres. The Royal Brompton and Belfast will join Birmingham, Leeds and Nottingham to intensify clinical research in CF and to bring new medicines to the patients as quickly as possible. The CTN works with the US Therapeutics Development Network, European patient organisations and industry to facilitate clinical trials in Cystic Fibrosis developments within Europe.
Cystic Fibrosis on Lorraine
Check out Lorraine on ITV1 for a piece about Cystic Fibrosis.
Congratulations to the UK CF Gene Therapy Consortium 07.06.11
Last night the Consortium won not one, but two awards for their pioneering research into gene therapy for Cystic Fibrosis. The team won 'Best Translational Research Innovation' and 'Best Therapeutic Innovation in the Respiratory Innovation Awards' at the Medial Futures Innovations Awards. Click here to find out more about the awards. Read some recent coverage from the BBC and The Observer about the awards and the gene therapy appeal. Make a donation to the appeal
Volcanic Ash
We have been advised by a Specialist CF Consultant that the volcanic ash does not pose a special threat to those with Cystic Fibrosis. If people use an inhaler for asthma, they should carry it with them. For more information visit the Health Protection Agency.
CF Trust team beat MPs 6-4 at charity football match at Stamford Bridge 17.05.11
Celebrities Omid Djalili, Angus Deayton, Gus Poyet, Chris Wolstenholme, Darren Campbell, Dalton Grant, Dan Walker and Kyran Bracken beat a team of MPs 6-4 in a football match held at Stamford Bridge today to raise awareness and funds for people with Cystic Fibrosis (CF). 
Read press release.
Cystic Fibrosis Week 8-14 May 2011
Thank you for taking part in CF Week. Read more at our special CF Week website. You can still like us on Facebook and follow us on Twitter. You can read our press release about our survey into virtual friendships.
Gene Therapy Update 04.05.11
Following our announcement in March that we were reviewing our costs and commitments to the UK CF Gene Therapy Consortium, we are pleased to confirm that a multi-dose clinical trial for CF gene therapy is planned to start in Spring 2012. Please read more about our new fundraising drive for the gene therapy trials.
UK CF Registry Report 2009
The 2009 report is now available along with a summary and a set of frequently asked questions.ReportSummaryFAQs
Cystic Fibrosis Trust Medical Conference 2011
This year's Medical Conference will take place on Thursday 8 September 2011 at Conference Aston, Aston University Business School and Conference Centre, in Birmingham from 9.30 am to 4.30 pm. The conference is open to healthcare and allied professionals responsible for the care of people with Cystic Fibrosis. There will be an online booking procedure and administrative costs of £24 (£20 + VAT) will be charged on booking. Further details will be placed on our website as soon as possible.
Charity rugby match at Twickenham 21.04.11
Thanks to everyone who played in our charity rugby match at Twickenham yesterday. The CF Trust team beat the Commons and Lords team 55-12 and the match raised around £30,000. Thank you to everyone involved.
Congratulations marathon runners!
Well done to our amazing 220 London marathon runners! Thank you so much to each and every one of you for taking on this huge challenge for us.
Virgin London Marathon Sunday 17 April
Come and support our London Marathon runners! We have 220 runners taking part including 6 people with CF, 8 racing drivers, a mum hoping to beat the Guinness World Record for the fastest fairy and Matthew Reed, our Chief Executive. Come and join our cheering squad from 10am at Mile 12 (Tower Bridge Tube) and Mile 25 (Embankment Tube). Look out for our balloons and banners and you'll see us!
Nurses' Day 12 May 2011
Nurses' Day celebrates everything that's good about nurses and nursing. Share your stories about why you love nurses and show your support on this website.
Changes to Gift Aid
From 6 April Gift Aid Transitional Relief of 3% will no longer be paid to charities by HM Revenue & Customs. This means that for every donation of £1 we receive, we will only be able to claim 25p Gift Aid rather than the 28p we can claim at the moment. If you have raised funds for us recently we would very much appreciate it if you could get any donations to us before the end of March to enable us to claim the maximum amount of Gift Aid. Many thanks.
Support for devastated Cystic Fibrosis services in New Zealand: can you help?
Following the recent devastating earthquake in Christchurch, New Zealand, CF care has been severely compromised. Read more about how you can help.
VX-770 trials
The Royal Brompton Hospital and the Royal Hospital for Sick Children in Edinburgh are currently recruiting patients for a new trial of this drug. They are looking for patients 6 years and older with the G551D gene mutation and a FEV1 of over 90%. Referrals from other specialist CF centres are welcome. If you are interested please contact the Research Nurses at the Royal Brompton Sandra Scott and Lauren McCann or for the Royal Hospital for Sick Children Research Nurse Debbie Miller. More info on the Clinical Trials website.
Threat to paediatric CF service at Royal Brompton Hospital
**Updated - read this Evening Standard article about the proposed changes**
Following the recommendations of England's Joint Committee of Primary Care Trusts to cease children's cardiac care at Royal Brompton Hospital, Royal Brompton & Harefield NHS Foundation Trust is mounting a strong campaign to challenge the recommendation. Should the recommendations go ahead, the hospital could not sustain its children's intensive care or anaesthesia services - making provision for paediatric cystic fibrosis patients inadequate. This is because CF patients need operations from time to time such as bronchoscopies, placement of Port-A-Caths and gastrostomies. These are impossible without paediatric anaesthesia, and it is unacceptable for all but the most straightforward cases to be done without intensive care back-up.
The CF Trust considers the paediatric CF service at the Royal Brompton Hospital to be vitally important in terms of the standard of care provided, its strong research base which has both national and international recognition, and also for the development of guidelines for the care of children with CF which are used throughout the UK and referenced on many of the CF Trust documents, including the recently launched CF Clinical Care Pathway. In addition it is unfeasible that the 300+ patients currently cared for at RBH could be easily or readily accommodated by any of the other London based CF specialist services without significant investment and expansion, neither of which seem to be an option in the current climate.
The public consultation began on March 1 and we would urge people to make their voice on this issue heard. Visit NHS Specialised Services for more information about consultation events and how to have your say. Show your support for the Royal Brompton & Harefield NHS Trust on Facebook. Sign the petition here.
European CF Young Investigators
Calls for abstracts for young CF scientists to attend this conference are now open. Read more in our research section.
VX-770 shows promising results
New trial results from Vertex Pharmaceuticals show that VX-770 has improved lung function, reduced exacerbations and encouraged weight gain in people with CF who have the mutation G551D.
A spokesperson for the Cystic Fibrosis Trust said: "The latest results from the VX-770 trials are great news for people with CF who have the G551D mutation - around 4-5% of those living with CF in the UK."
"In people with CF, the protein called CFTR, which is made by the CF gene either doesn't work, or doesn't work enough. It controls the movement of sodium and chloride though the body's cells, which affects the level of liquid in the lungs, digestive system and other organs. This results in thick, sticky mucus clogging the airways and gut resulting in chronic infections and inflammation in the lungs and difficulty digesting food. There are over 1,500 different mutations of the CF gene in which the levels of fault can vary greatly."
"VX-770 is a new type of drug designed to correct the function of the defective CFTR protein, allowing chloride and sodium to move properly in and out of cells lining the lungs and other organs. In these latest trials, the drug has shown great promise in improving lung function and reducing the chance of a lung infection or inflammation in people with the G551D mutation. We look forward to hearing more about this drug over the coming year, which could be a great new weapon in the clinical fight against Cystic Fibrosis."
Dispatches: The Truth about Hospital Food 21.02.11Watch Dispatches: The Truth about Hospital Food on 4oD. The programme features two adults living with Cystic Fibrosis. Sign the petition about hospital food.
King's College Gala Concert - 25 February
Join us at this evening of music from King's featuring Alison Balsom and Alex Stobbs to raise funds for the CF Trust. Tickets and details are available here.
Racing drivers join Team CF! 26.01.11
We're delighted that 10 members of the British Racing Drivers' Club have chosen to run the Virgin London Marathon for the CF Trust. Some of Britain's most successful racing stars such as Andy Meyrick, James Matthews and Jonny Kane will be running on Sunday 17 April.
New research in Belfast 24.01.11
Scientists at Queen's University Belfast have begun a new research project thanks to a £1.74 million US-Ireland Research and Development Partnership grant. Read more
Have a clear out for the CF Trust
Look out for us this week on eBay at the check out! eBay have selected us as one of their charities of the week; every time a shopper pays for their item they will be asked to make a small donation to us. Also if you fancy having a January clear out you can do so and donate to the CF Trust. Simply put all your unwanted items on eBay and decide exactly what percentage of your final selling price you'd like to donate to us, and what percentage you'd like for yourself . What's better is every time you list an item for us, you'll get a fee credit on your basic insertion. So if you donate 50% of your selling price to us, eBay will waive 50% of your fees. Happy shopping and selling!
Cherry Tree House
We are delighted to announce that Cherry Tree House Adolescent Unit, part of Allen Ward at the Royal Belfast Hospital for Sick Children (RBHSC), will continue to be used for Cystic Fibrosis patients. After lengthy negotiations and consultation with parents and the CF Trust, the Belfast Health and Social Care Trust have agreed to keep two in-patient rooms in Cherry Tree House on a priority basis for teenagers with Cystic Fibrosis. 
Read our press release.
Flu Jabs
If anyone with CF has had trouble getting their flu jab this winter for whatever reason please contact us.
Tim's Countdown to 40
Tim Wotton, who has CF, has started a new blog as he starts his countdown to his 40th birthday. Read his story here.
Denufosol Update 05.04.11
Inspire Pharmaceuticals have announced disappointing results from their second Phase 3 clinical trial of the drug Denufosol.
More information is available on the Inspire Pharmaceuticals website.
Getting Nosey About CF with Oli & Nush
Our new film for children about CF is now available to view online. We also have copies available on DVD please visit this page for more information.
QVC and Honora Pearls support the CF Trust
On Wednesday 22 December the shopping channel QVC will be supporting the Cystic Fibrosis Trust. Their first ever 'QVC Cares' Day will feature top brands who have chosen to support their favourite charities throughout the day and we are thrilled that Honora Pearls are supporting the Cystic Fibrosis Trust. They will kindly donate 15% of the purchase price of all jewellery sold throughout the day to the CF Trust. Find out more on the QVC website.
Belfast Meeting Postponed 20.12.10
The parents meeting planned for today at 12.30pm at the NICVA building has been postponed due to the weather. The meeting will be rearranged for the beginning of January 2011 and we will confirm the new date as soon as possible. Members of the NI Council of the CF Trust and the Medical Team at the Children's Unit will be at the NICVA Building from 12.30pm for any parents who don't receive this message in time and will take the opportunity to hear their views. If you would like to contact someone in the meantime please call Conrad Murphy on 07756527917 or email conrad_murphy@hotmail.co.uk
Denufosol shows encouraging results
A new drug may benefit Cystic Fibrosis patients with normal to mildly impaired lung function according to the latest clinical trial results. Denufosol is a new type of drug that could delay or prevent lung disease progression in children, teenagers and young adults with Cystic Fibrosis by helping to prevent formation of sticky mucus.
Denufosol belongs to a class of drugs known as ion channel regulators. These drugs help balance the flow of ions through cell membranes, helping normalise the airway surface hydration and mucus clearance impairment present in people with Cystic Fibrosis.
The CF Trust has been following the research closely and studies to date have shown promising results in people with Cystic Fibrosis who have a good lung function. Further trials are underway but it is estimated that patients could benefit from this new drug in 3-4 years. For more information read the full press release from the American Thoracic Society or read this article from BBC News.
Cherry Tree House
The Belfast Health and Social Care Trust is planning to close Cherry Tree House Adolescent Unit at Royal Belfast Hospital for Sick Children (RBHSC). Click here to read more.
DLA Reform
The Government have announced a consultation on DLA reform. We would like to hear your views. Visit our Welfare Reform page for more details.
Getting Nosey About CF with Oli and Nush
Our new film for children about CF is now available to view online, DVDs will be available soon please visit this page for more information.
Fitness to Work tests
An independent review of the Work Capability Assessment has found that the tests need to be reformed to make them fairer and more effective. The CF Trust broadly welcomes the findings of Professor Harrington's report, and we look forward to working with the Department of Work and Pensions to ensure that work capability assessments carried out take into consideration the needs of those with Cystic Fibrosis.
BBC Horizon
A new documentary about genetic research airs on Monday 25th October at 9pm on BBC2. The programme features Sophie Longton, who has CF and Professor Eric Alton from the UK CF Gene Therapy Consortium. More info.
Equality Act 2010
New equality rights in the workplace came into force on 1 October. More info
Make sure you get your flu jab
People with CF should make sure they get their annual flu jab each autumn. To find out more contact your GP or CF centre, visit the NHS website or call NHS Direct on 0845 46 47
Vote for us!
Please vote for the Cystic Fibrosis Trust at www.Lucaskeepsrunning.co.uk so we can benefit from Lucas raising £10k for charity next year. You can vote as many times as you like and anyone can vote so please pass it on and vote often until 10 October!
A warm welcome to our new Chief Executive
Matthew Reed has been appointed as the new Chief Executive of the Cystic Fibrosis Trust following the retirement of Rosie Barnes in August. He began his new role on Monday 6 September. Read our press release.
Our response to some recent issues that have hit the headlines can be found in the Behind the Headlines section.
