We have compiled some answers to some questions we are frequently asked about Cystic Fibrosis and the Cystic Fibrosis Trust. You may also find our Ask the Expert section useful.

Cystic Fibrosis (CF)
What is CF?
How common is CF?
What causes CF?
Can you catch CF?
Is Cystic Fibrosis known by any other name?
How are people diagnosed with CF?
What are the symptoms of CF?
Are there different types of CF?
Are men as likely to have CF as women?
Does CF affect ethnic minorities?
What is the life expectancy of someone with CF?
How does CF affect daily life?
Is there anything that children with CF can't do?
Do people with CF require a special diet?

Treatment of Cystic Fibrosis
How is CF treated?
Does treatment start as soon as a baby is diagnosed?
Where do people with CF get their care?
Where is care best for people with CF?
What kind of transplant might people with CF need?

Transplants
Will everyone with CF need a transplant?
What is the survival rate for someone who has a transplant?
Does the CF Trust support presumed consent?
Can someone with CF be an organ donor?

About the CF Trust
What does the CF Trust do?
What progress is being made in research?
Does the CF Trust receive any money from the UK Government, Scottish Parliament or Welsh Assembly?
Does the CF Trust work with other CF organisations around the world?
Does the Trust receive funding from pharmaceutical companies?

Gene therapy and other research
What is gene therapy?
Is gene therapy a cure for CF?
When will gene therapy be available?
How much does it cost?
Will gene therapy only benefit newborn babies or will older children and adults benefit too?
Is stem cell therapy an option for treating CF?
Does the CF Trust fund research involving animals?
Does the CF Trust fund research involving human and hybrid human embryos?
How can someone with CF get on clinical trials for gene therapy and other new treatments?

Screening for CF
Can you tell if a baby has CF before it is born?
How will I know if I am a carrier of the gene that causes CF?
Are babies screened for CF at birth?
What is PGD and PGH?
Why isn't the CF Trust campaigning for everyone to be screened to see if they are a carrier?

Other
Can people with CF have children?
Can people with CF socialise with each other?
Do people with CF have to pay prescription charges?
Are people with CF entitled to benefits?
Does CF restrict employment opportunities?

CYSTIC FIBROSIS

What is CF?
Cystic Fibrosis (CF) is one of the UK's most common life-threatening inherited diseases. Cystic Fibrosis is caused by a single defective gene. As a result, the internal organs, especially the lungs and digestive system, become clogged with thick sticky mucus resulting in chronic infections and inflammation in the lungs and difficulty digesting food.

How common is CF?
Around 8,500 people in the UK have CF, that's one in every 2,500 babies born. CF affects around
100,000 people around the world.

What causes CF?
CF is a genetic condition. 1 person in 25 carries the faulty CF gene usually without knowing - over two million people in the UK. If two carriers have a baby, the child has a 1 in 4 chance of having Cystic Fibrosis

Can you catch CF?
No, CF is not contagious it can only be caused by inheriting two copies of the faulty gene.

Is Cystic Fibrosis known by any other name?
Cystic Fibrosis is also known as CF, mucovoidosis, or mucoviscidosis.

How are people diagnosed with CF?
All newborn babies in the UK are now screened for Cystic Fibrosis at birth using the heel prick blood test. This tests for the most common mutations of the gene that causes CF.

Around 1 in 10 children with Cystic Fibrosis are diagnosed prenatally, at birth, or shortly after birth due to a condition called meconium ileus where the gut becomes blocked with meconium - a thick, dark, sticky substance which is made in all babies' intestines before being born. Urgent surgery may be needed to relieve the blockage.

Some children born earlier than 2007 who were not screened at birth, or those with more unusual mutations of the CF gene, may be diagnosed later after they have become unwell and developed symptoms.

What are the symptoms of CF?
Symptoms usually first develop within the first year of life, but in a minority of cases may not appear until later in childhood. The severity of symptoms can vary and not all people with CF will have all the symptoms. In those with Cystic Fibrosis, the lungs make thicker sputum (mucus) than normal. This can trap bacteria in the small airways and lead to infection. Symptoms which typically develop include persistent cough, wheezing, shortness of breath and breathing difficulties and repeated chest infections.

In people with Cystic Fibrosis, thickened secretions block the normal flow of the digestive juices from the pancreas. This results in food not being digested or absorbed properly, in particular fatty foods and fat-soluble vitamins (vitamins A, D, E and K). This can cause malnutrition leading to poor growth and poor weight gain, bloated abdomen and tummy aches and prolonged diarrhoea.
Other symptoms can include sinus infections, constipation and nasal polyps. Some adults with CF may also get CF related diabetes, arthritis, osteoporosis and liver problems.

Are there different types of CF?
Cystic Fibrosis is a very complex condition affecting people in different ways. Some people suffer more with their digestive system than the lungs. Others are pancreatic sufficient. There are over 1,500 identified mutations of the CF gene.

Are men as likely to have CF as women?
Yes, CF is as common in both sexes.

Does CF affect ethnic minorities?
Yes CF affects people of many ethnic backgrounds, although it is more common in the Caucasian population.

What is the life expectancy of someone with CF?
It is misleading to suggest that there is an 'average life expectancy' that can be applied to everyone with Cystic Fibrosis. Life expectancy is likely to be different for people of different ages - and this is before taking into account the fact that CF can also affect people differently. The median predicted survival for someone with CF currently stands at 35 years old. This means that according to the statistics on our clinical CF registry, currently half of those with CF will live to over 35 years old, although a baby born today can be expected to live longer.

How does CF affect daily life?
Cystic Fibrosis involves a rigorous daily treatment regime including physiotherapy, oral, nebulised and occasionally intravenous antibiotics, and taking enzyme tablets with food. Some people with CF will have a feeding tube overnight.

For those who are very ill with Cystic Fibrosis and with a severely impaired lung function, daily life can be a struggle as basic tasks can leave them breathless. Some patients use a wheelchair to get around, and use oxygen to help them breathe.

Is there anything that children with CF can't do?
Children with CF should be encouraged to do everything that ordinary children do, although there are a few things that should be avoided because they may harbour particular bugs which can cause problems for people with CF. Read an article about this.

Do people with CF require a special diet?
In most people with CF the small channels that carry the digestive juices from the pancreas to the intestine become clogged with thick, sticky mucus. As a result people with CF do not absorb and digest their food easily.

As well as taking enzyme pills to help digest food most people with CF require 20-50% more calories each day than people without CF, however some may need considerably more. People with CF are encouraged to eat cakes, crisps, chocolate and other foods often considered 'unhealthy'. It is important for those with CF to maintain a healthy weight as it helps the body deal more effectively with chest infections or weight loss caused by illness.

TREATMENT OF CYSTIC FIBROSIS

How is CF treated?
Those with CF have to undergo a rigorous daily regime of treatments to stay healthy. This can include taking inhaled and injected drugs to clear mucus and fight infections, taking dozens of enzyme pills to digest food and having physiotherapy morning and night.

Does treatment start as soon as a baby is diagnosed?
Yes. Most babies will CF will need to start taking digestive enzymes straight away and will also begin physiotherapy to keep the lungs clear of mucus.

Where do people with CF get their care?
The majority of adults with CF go to one of the Specialist CF Centres in the UK. Children may have a shared care arrangement, where they have some of their care at a local hospital and some at the nearest Specialist CF Centre, though the centre takes responsibility for their health. The CF Trust recommends all people with CF receive care at a recognised Specialist CF Centre.

Where is care best for people with CF?
It is vitally important that those with Cystic Fibrosis receive appropriate healthcare to ensure a better quality and length of life. Therefore, the Cystic Fibrosis Trust strongly recommends that people with CF receive care from the multidisciplinary team of specialist doctors, nurses and allied health professionals at a recognised Specialist CF Centre. This includes specialist CF dietetic care and physiotherapy and should also provide psychosocial support.

TRANSPLANTS

What kind of transplant might people with CF need?
Most of the transplants done on people with CF are lung transplants, as usually these are the organs most affected by Cystic Fibrosis. However some people with CF will have problems with their liver or kidneys and may need these organs transplanted.

Will everyone with CF need a transplant?
No. Transplantation is appropriate only for a patient who is severely ill and for whom all other forms of conventional treatment are no longer helpful. Not all patients at this stage of the disease are suitable for a transplant and some patients may choose not to go on the transplant list.

What is the survival rate for someone who has a transplant?
Although it is important not to place too much emphasis on averages when it comes to transplants, as some people sadly do not recover from a transplant and others are still thriving 10 years on, the statistics show that around 70% of those with CF survive for five years post transplant

Does the CF Trust support presumed consent?
We welcome the debate on the subject, and ultimately support any move that means more people with CF will get their life saving transplant, but we are more interested in the measures the Organ Donation Taskforce have introduced to improve the current situation as it may not be necessary to move to a system of presumed consent if we improve our rate of transplantation from existing donors who have chosen to sign the register.

Can someone with CF be an organ donor?
Yes they can, as not all the organs are affected by Cystic Fibrosis.

ABOUT THE CF TRUST

What does the CF Trust do?
The Cystic Fibrosis Trust is the UK's only national charity dealing with all aspects of Cystic Fibrosis (CF). It funds research to treat and cure CF and aims to ensure appropriate clinical care and support for people with Cystic Fibrosis.

What progress is being made in research?
When the CF Trust was formed in 1964. many children with CF didn't live past their 5th birthday. Now adults with CF are living into their 30s, 40s, 50s and beyond. This is due in part to the incredible progress that has been made in treating and understanding the condition. Our ground breaking gene therapy research is leading the world and making encouraging progress. We hope to be able to take the life-threatening aspect out of CF and clinical trials are underway. Read more on our research pages.

The CF Trust received a one-off grant of £1 million from the Department of Health for our gene therapy research programme in 2007/8.

Does the CF Trust work with other CF organisations around the world?
Yes the CF community is a close-knit one and we have links with all the major CF charities and organisations worldwide.

Does the Trust receive funding from pharmaceutical companies?
The CF Trust does from time to time receive grants from pharmaceutical companies who have an interest in CF, usually for specific things such as paying for the costs of a conference or for a particular publication. It will always be clearly indicated if we have received funding from a pharmaceutical company to help with one of our projects. The amount of funding received from pharmaceutical companies is small in relation to our overall income; last year it was roughly 0.5% of our total income.

GENE THERAPY AND OTHER RESEARCH

What is gene therapy?
Gene therapy is a way of treating or curing a disease by adding a copy of a healthy gene to do the job of a faulty one.

Is gene therapy a cure for CF?
Gene Therapy is not a cure for CF, but if effective, would prevent the lung damage that kills 90% of those with CF from developing further.

When will gene therapy be available?
As with all ground-breaking scientific research, it is difficult to predict a timescale. However we are conducting a pilot study in 2009-10 which will be followed by a major clinical trial in around 100 people with Cystic Fibrosis.

How much does it cost?
The CF Trust has spent well over £20 million on gene therapy to date and is committed on raising at least £4 million a year to fund this vital work.

Will gene therapy only benefit newborn babies or will older children and adults benefit too?
Gene therapy will benefit most people with CF. Although it cannot reverse lung damage, it will stop lungs from deteriorating further.

Is stem cell therapy an option for treating CF?
Stem cell therapy could, in the future, be of benefit to people with CF. However at present our main focus is our gene therapy research which is concerned with somatic cells.

Somatic cells are replenished from populations of stem cells, which have the unique ability to divide to produce both copies of themselves and other cell types. Stem cells also have the ability to replace damaged cells in the body that would otherwise not be replenished. This property has led scientists to investigate the possible use of stem cells in regenerative medicine and the treatment of diseases. If you use gene therapy on stem cells, it is hoped that you will also correct the genetic fault in all the cells it reproduces. However research into therapies using stem cells is still in its infancy.

A lot more basic information about stem cells and their behaviour is required before they can be used to develop treatments. Scientists currently do not know the location of the stem cells that produce the cells in the lungs or whether stem cell transplants to the lung will even be possible. It is not yet known how transplanted stem cells will behave inside the body. Stem cell therapy could unwittingly pass viruses and other disease causing agents to people who receive cell transplants. Some research has also raised the possibility that stem cells may turn cancerous. Consequently, the prospect of full clinical trials for any disease or condition is still some way off.

Does the CF Trust fund research involving animals?
The Cystic Fibrosis Trust is a member of the Association of Medical Research Charities (AMRC), which recognises that a minimal amount of strictly controlled work using animals is essential to make progress in the treatment of life-threatening diseases. New medicines are required by law to use animals during development and safety-testing and by law, animals cannot be used in research if there is an equally good non-animal alternative.

Does the CF Trust fund research involving human and hybrid human embryos?
The CF Trust supports the right of scientists to use human and hybrid embryos in research into life-threatening conditions. However at present we are not funding any research of this nature.

How can someone with CF get on clinical trials for gene therapy and other new treatments?
The gene therapy trials are taking place in Edinburgh and Oxford and 200 people with CF over 12 years old have already been recruited to take part in the run-in study. 100 of these will go on to take part in the multi-dose clinical trial. These people were chosen in close collaboration with their CF consultant to meet the requirements of the study.

Other research in CF takes place around the world in collaboration with CF centres. Each CF Centre may be involved in different areas of research at any point. This can be academic or clinical research and through the Centre themselves, Universities or pharmaceutical companies. Each CF Centre will be able to give information on current trials and further information on UK trials can be found on our webpage. Europe wide trials can be found on the European Trials Network.

SCREENING FOR CF

Can you tell if a baby has CF before it is born?
Yes in some cases. Women can have amniocentesis or CVS to test the embryo for CF whilst pregnant. Occasionally, if the baby has meconium ileus (a bowel blockage commonly associated with CF) this can be picked up on an ultrasound scan and CF may be suspected.

How will I know if I am a carrier of the gene that causes CF?
Most people do not know if they are a carrier. If you have CF in your extended family, you should ask your GP to be tested as you are more likely to be a carrier than the general population.

Are babies screened for CF at birth?
Yes since October 2007 all babies are screened for CF at birth as part of the heel-prick blood test.

What is PGD and PGH?
Pre-implantation Genetic Diagnosis (PGD) and Pre-implatantation Genetic Haplotyping is a way in which two known carriers of the CF gene can have a baby without CF. Both techniques involve screening embryos conceived by IVF.

However whereas PGD tests for a specific mutation in a gene, PGH is able to identify a CF-affected gene without needing to know the specific mutation.

Why isn't the CF Trust campaigning for everyone to be screened to see if they are a carrier?
The CF Trust recognises that screening everyone to see if they are a carrier of the CF gene would be very expensive and impractical. However we do recommend that anyone who is a blood relation of someone with CF is tested for carrier status. Read our factsheet on cascade screening.

OTHER

Can people with CF have children?
Fertility, or the ability to have children, is usually normal or only slightly reduced in women but most men with CF are infertile. This is due to an abnormality/absence of the vas deferens (the tube which carries sperm from the testis to the penis). The tubes are
effectively blocked or absent altogether. Although sperm is are produced and sexual function remains entirely normal, men with CF can seldom father children naturally. However, recent advances in in vitro fertilization and aspiration of sperm has allowed
some men with CF to father children with clinical assistance.

Some women with CF may be advised not to have children if their health is poor, as carrying a baby and giving birth could be detrimental to their health.

Can people with CF socialise with each other?
Those with CF are advised not to mix with each other as they have different bacteria or 'bugs' that grow in their lungs. These 'bugs' are rarely harmful to those who do not have CF but may be harmful to others who have CF but who do not have the same 'bugs'. The CF Trust has internet message boards for those with CF so they can interact safely.

Do people with CF have to pay prescription charges?
In England, people with certain medical conditions are exempt from paying prescription charges. At present Cystic Fibrosis is not one of these conditions. The Government are currently carrying out a review of prescription charges which was due to report in Autumn 2009. We hope that this review will abolish charges for those with CF.

Currently people in Wales do not pay prescription charges. Prescription charges are expected to be completely abolished in Northern Ireland by 2010 and Scotland by 2011.

>More info on prescription charges.

Are people with CF entitled to benefits?
Those with CF can be entitled to benefits, the most common is the Disability Living Allowance. It is given to people who need a lot of help with personal care or getting around, or both, because they are ill or disabled. For more information on benefits click here.

Does CF restrict employment opportunities?
As people with CF are living longer, healthier and more independent lives many more are taking the opportunity to work part-time or full-time. In the last report from the CF Registry, of those adults with CF who completed an employment questionnaire, around 70% were in work or education.

There can be issues for people with CF in employment regarding time off or type of working environment. Find out more by reading our employment factsheet.