Your donation will make a difference:
Cystic Fibrosis Trust

Frequently Asked Questions about cystic fibrosis

The basics

What is cystic fibrosis?
How common is cystic fibrosis?
What causes cystic fibrosis?
Can you catch cystic fibrosis?
Is cystic fibrosis known by any other name?
How are people diagnosed with cystic fibrosis?
What are the symptoms of cystic fibrosis?
Are there different types of cystic fibrosis?
Are men as likely to have cystic fibrosis as women?
Does cystic fibrosis affect Black, Asian and minority ethnic groups?
What is the life expectancy of someone with cystic fibrosis?






 







 
 

Day to day

How does cystic fibrosis affect daily life?
Is there anything that children with cystic fibrosis can't do?
Do people with cystic fibrosis require a special diet?
Does CF affect fertility?
Can people with cystic fibrosis socialise with each other?
Do people with cystic fibrosis have to pay prescription charges?
Are people with cystic fibrosis entitled to benefits?
Does cystic fibrosis restrict employment opportunities?

 

Treatment and transplants

Does treatment start as soon as a baby is diagnosed?

Where do people with cystic fibrosis get their care?

Will everyone with cystic fibrosis need a transplant?
What is the survival rate for someone who has a transplant?
Does the Cystic Fibrosis Trust support 'soft opt-out' organ donation?
Can someone with cystic fibrosis be an organ donor?




 
   

Screening for cystic fibrosis

Is there antenatal screening for cystic fibrosis?
How will I know if I am a carrier of the gene that causes cystic fibrosis?
Are babies screened for cystic fibrosis at birth?
What is PGD and PGH?
Why isn't the Cystic Fibrosis Trust campaigning for everyone to be screened to see if they are a carrier?

 

Gene therapy and other research

Is gene therapy a cure for cystic fibrosis?
How much does it cost?
Will gene therapy only benefit newborn babies or will older children and adults benefit too?


 
 



 

Stem Cell therapy

What is stem cell therapy?
Is stem cell therapy approved in the UK for the treatment of cystic fibrosis?
Is stem cell therapy dangerous?
Is the Cystic Fibrosis Trust funding research into stem cell therapy?
What research is the Cystic Fibrosis Trust funding?
Should patients pay to go abroad for stem cell therapy?
There are media reports on stem cell treatment being carried out in the Dominican Republic; is this a safe option? 
Could stem cell therapy one day provide a way of beating cystic fibrosis? 
How can I keep up to date with the latest news and research on stem cell treatments?

 

About our research

What progress is being made in research? 
Does the Cystic Fibrosis Trust fund research involving animals?
Does the Cystic Fibrosis Trust fund research involving human and hybrid human embryos?
How can someone with cystic fibrosis get on clinical trials for gene therapy and other new treatments?
Does the Cystic Fibrosis Trust receive any money from the UK Government, Scottish Parliament or Welsh Assembly?
Does the Cystic Fibrosis Trust work with other cystic fibrosis organisations around the world?
Does the Trust receive funding from pharmaceutical companies?



 



The basics...

What is cystic fibrosis?

Cystic fibrosis 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 cystic fibrosis?

Around 10,500 people in the UK have cystic fibrosis, that's 1 in every 2,500 babies born. Cystic fibrosis affects around 100,000 people around the world.

What causes cystic fibrosis?

Cystic Fibrosis is a genetic condition. One person in 25 carries the faulty cystic fibrosis gene usually without knowing, over two million people in the UK. If two carriers have a baby, the child has a one in four chance of having cystic fibrosis.

Can you catch cystic fibrosis?

No, cystic fibrosis can't be caught or developed; it can only be caused by inheriting two copies of the faulty gene. You could be a gene carrier without having cystic fibrosis.

Is cystic fibrosis known by any other name?

Cystic fibrosis is also known as CF, mucovoidosis, or mucoviscidosis.

How are people diagnosed with cystic fibrosis?

All newborn babies in the UK are now screened for cystic fibrosis shortly after birth using the heel prick blood test. This tests for the most common mutations of the gene that causes cystic fibrosis.

Around one in 10 children with cystic fibrosis are diagnosed before, at or shortly after birth due to a condition called meconium ileus that causes the gut to become 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 cystic fibrosis gene, may be diagnosed later in life, after they have become unwell and developed symptoms.

What are the symptoms of cystic fibrosis?

In people with cystic fibrosis the lungs make thicker sputum (mucus) than normal, which can trap bacteria in the small airways and lead to infection. Symptoms that typically develop include persistent cough, wheezing, shortness of breath and breathing difficulties and repeated chest infections.

Thickened mucus secretions block the normal flow of digestive juices from the pancreas, which means food can not be 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, constipation and prolonged diarrhoea.

Other symptoms can include sinus infections, and nasal polyps. Some adults with cystic fibrosis may also get cystic fibrosis related diabetes, arthritis, osteoporosis and liver problems

Symptoms usually first develop within the first year of life, but in a minority of cases may not appear until later in childhood or beyond. The severity of symptoms can vary and not all people with cystic fibrosis will have every symptom.

Are there different types of cystic fibrosis?

There are over 1,500 identified mutations of the cystic fibrosis gene. Cystic fibrosis is a very complex condition that affects people in different ways. Some suffer more with their digestive system than the lungs. Others have no problem with their pancreas.

Are men as likely to have cystic fibrosis as women?

Yes, cystic fibrosis is as common in both sexes.

Does cystic fibrosis affect Black, Asian and minority ethnic groups?

Yes, cystic fibrosis affects people of many ethnic backgrounds, although it is more common in the Caucasian population.

What is the life expectancy of someone with cystic fibrosis?

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 cystic fibrosis currently stands at 41 years old. This means that according to the statistics on our clinical CF Registry, currently half of those with cystic fibrosis will live to over 41 years old, although a baby born today can be expected to live longer.

Day to day...

How does cystic fibrosis affect daily life?

Cystic fibrosis affects everyone differently, but for many it involves a rigorous daily treatment regime including physiotherapy, oral, nebulised and occasionally intravenous antibiotics, and taking enzyme tablets with food. Some people with cystic fibrosis will have a feeding tube overnight.

For those who are very ill with cystic fibrosis and with very poor 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 cystic fibrosis can't do?

Children with cystic fibrosis should be encouraged to do things that their peers do, although there are a few things that should be avoided because they may harbour particular bugs which can cause problems for people with cystic fibrosis.

Do people with cystic fibrosis require a special diet?

In most people with cystic fibrosis 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 cystic fibrosis do not absorb and digest their food easily. This is called ‘pancreatic insufficiency’.

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

Does cystic fibrosis affect fertility?

Fertility, or the ability to have children, is usually normal or only slightly reduced in women but most men with cystic fibrosis 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 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 (extracting sperm directly from the testes) has allowed some men with cystic fibrosis to father children with clinical assistance. Some women with cystic fibrosis 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 cystic fibrosis socialise with each other?

People with cystic fibrosis should not meet 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 cystic fibrosis but may be harmful to others who have cystic fibrosis but who do not have the same 'bugs'. We offer internet message boards for people with cystic fibrosis so they can interact safely.

Do people with cystic fibrosis have to pay prescription charges?

In England cystic fibrosis is currently not one of the medical conditions that makes people exempt from paying prescription charges. Currently people in Wales, Scotland and Northern Ireland do not pay prescription charges. More information on prescription charges.

Are people with cystic fibrosis entitled to benefits?

Those with cystic fibrosis can be entitled to benefits.

Does cystic fibrosis restrict employment opportunities?

As people with cystic fibrosis 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, around 70% of those adults with cystic fibrosis who completed an employment questionnaire were in work or education. 

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

Treatment and transplants

People with cystic fibrosis often 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. People with cystic fibrosis may also need a transplant. Lung transplants are the most common type for people with cystic fibrosis, as usually these are the organs most affected by cystic fibrosis. However, some people will have problems with their liver, kidneys or pancreas, and may need these organs transplanted.

Does treatment start as soon as a baby is diagnosed?

Yes. Most babies with cystic fibrosis 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 cystic fibrosis get their care?

The majority of adults with cystic fibrosis 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. Find a list of specialist CF centres.

It is vitally important that those with cystic fibrosis receive appropriate healthcare to ensure a better quality and length of life. Therefore, we strongly recommend that people with cystic fibrosis 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.

Will everyone with cystic fibrosis need a transplant?

No. Transplantation is only appropriate 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?

It is important not to place too much emphasis on statistics when it comes to transplants, as some people sadly do not recover from a transplant and others are still thriving 10 years on, but the statistics show that around 70% of those with cystic fibrosis survive for five years post transplant.

Does the Cystic Fibrosis Trust support 'soft opt-out' organ donation?

We support any move that means more people with cystic fibrosis will have a chance of receiving a successful transplant.

Can someone with cystic fibrosis be an organ donor?

Yes they can, as not all the organs are affected by cystic fibrosis.

Screening for cystic fibrosis

Is there any antenatal screening for cystic fibrosis?

Yes in some cases. Women can have amniocentesis or CVS to test the embryo for cystic fibrosis whilst pregnant. Occasionally, if the baby has meconium ileus (a bowel blockage commonly associated with cystic fibrosis) 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 cystic fibrosis?

Most people do not know if they are a carrier. If you have cystic fibrosis 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 cystic fibrosis at birth?

Yes, since October 2007 all babies are offered screening for cystic fibrosis shortly after birth as part of the heel-prick blood test.

What is PGD and PGH?

Pre-implantation Genetic Diagnosis (PGD) and Pre-implatantation Genetic Haplotyping (PGH) are ways in which two known carriers of the cystic fibrosis gene can have a baby without cystic fibrosis. 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 cystic fibrosis affected gene without needing to know the specific mutation.

Why isn't the Cystic Fibrosis Trust campaigning for everyone to be screened to see if they are a carrier?

We recognise that screening everyone to see if they are a carrier of the cystic fibrosis gene would be very expensive and impractical. However, we do recommend that anyone who is a blood relation of someone with cystic fibrosis is tested for carrier status.

Gene therapy and other research

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 cystic fibrosis?

Gene therapy is not a cure for cystic fibrosis, but effective gene therapy could halt or prevent the lung damage that causes 90% of deaths of people with cystic fibrosis.

How much does it cost?

The Cystic Fibrosis Trust has invested £30 million in gene therapy to date. The Wave 1 product is now in Phase 2B trials. If the Phase 2 trial is successful (ie the gene therapy product improves lung function), it is likely that a pharmaceutical partner will be sought to take the gene therapy product to a Phase 3 clinical trial. Phase 3 trials can cost in excess of £100 million and the Cystic Fibrosis Trust and its supporters would not be able to fund this stage of research themselves.

Will gene therapy only benefit newborn babies or will older children and adults benefit too?

Gene therapy would benefit most people with cystic fibrosis and works regardless of age as it replaces the faulty gene mutation with a correct version. Although it cannot reverse lung damage, it would stop lungs from deteriorating further.

Stem cell therapy

What is stem cell therapy?

Somatic cells (biological cells that form the body of an organism) 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 early.

There are two types of stem cells. The first are embryonic stem cells, which as their name suggests, are derived from embryos. The other is induced Pluripotent Stem Cells (iPS also called iPSC). These are created by taking a patient’s own mature cells (such as blood cells) and converting them back to become stem cells.

Is stem cell therapy approved in the UK for the treatment of cystic fibrosis?

Stem cell therapy in the treatment of cystic fibrosis has not been approved for use in either Europe or America and the treatment has not successfully undergone clinical trials.

Is stem cell therapy dangerous?

A lot more basic information about stem cells and their behaviour is required before they can be used to develop treatments for CF. 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.

Is the Cystic Fibrosis Trust funding research into stem cell therapy?

It is in the research strategy published in April 2013. At the moment we have not been funding research in this area. We are beginning to fund research in the area of iPS.

What research is the Cystic Fibrosis Trust funding?

We have provided a small grant to University of Cambridge for some preliminary laboratory based research, which involves genetic editing in iPS cells from people with CF.

Should patients pay to go abroad for stem cell therapy?

Cystic fibrosis is a devastating condition so it is understandable that patients will seek new treatments to reduce the burden of the disease and prolong their life. However patients are advised that stem cell therapy recently reported in the news is unproven and has not undergone rigorous clinical trials so the risks of the treatment are currently unknown.

I’ve seen some media reports about successful stem cell treatment being carried out in the Dominican Republic. Can you tell me whether this is a safe option for me?

The treatment being carried out in the Dominican Republic has not been has not been approved by the Regulatory Authorities in USA or Europe (FDA and EMA). It is unregulated and unproven and there is no approved scientific evidence that the procedure being undertaken in the Dominican Republic is safe or effective. The Trust would not encourage people living with cystic fibrosis to currently undertake any type of stem cell treatment in the UK or abroad.

Could stem cell therapy one day provide a way of beating cystic fibrosis?

Yes, but it must be combined with genetic editing. A lot more research is needed to help us understand how this would work to ensure its safety.

How can I keep up to date with the latest news and research on stem cell treatments?

The Cystic Fibrosis Trust website carries up to date news of its latest research ventures so check it frequently. In addition, there is a stem cell catapult centre based at Guys Hospital . The Cell Therapy Catapult was established in 2012 as a centre of excellence in innovation, with the core purpose of building a world-leading cell therapy industry in the UK.

About our research

We are the UK's only national charity dealing with all aspects of cystic fibrosis. We fund research to improve cystic fibrosis care and treatment, and aim to ensure appropriate clinical care and support for people with cystic fibrosis.

What progress is being made in research?

Ever since we began in 1964 we have funded research into every aspect of cystic fibrosis. Together with our national and international partners we continue to increase our understanding of cystic fibrosis and improve understanding, treatments and management of cystic fibrosis.

We are also involved with social research, exploring the non-medical impact of cystic fibrosis, and its implications for quality and length of life.

Read more on our research pages.

Does the Cystic Fibrosis Trust fund research involving animals?

Yes we do. We are 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 legally required to use animals during development and safety-testing.

Does the Cystic Fibrosis Trust fund research involving human and hybrid human embryos?

The Cystic Fibrosis 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 cystic fibrosis get on clinical trials for gene therapy and other new treatments?

The Phase 2 clinical trial is currently taking place at the Gene Therapy Consortium's two clinical sites in London and Edinburgh. For information visit the consortium's website.

Other research in cystic fibrosis takes place around the world in collaboration with specialist 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 can be found on our UK clinical trials page. Europe-wide trials can be found on the European Trials Network.

Does the Cystic Fibrosis Trust receive any money from the UK Government, Scottish Parliament or Welsh Assembly?

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

Does the Cystic Fibrosis Trust work with other cystic fibrosis organisations around the world?

Yes, the cystic fibrosis community is a close-knit one and we have links with all the major cystic fibrosis charities and organisations worldwide.

Does the Trust receive funding from pharmaceutical companies?

From time to time we receive grants from pharmaceutical companies who have an interest in cystic fibrosis, 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.

Research

The Trust is committed to funding and supporting cutting-edge research to find new and better treatments for cystic fibrosis. Take a look at some of that work and the progress that is being made.

Discover more

What is cystic fibrosis?

Find out more about cystic fibrosis, its diagnosis and how it is treated, as well as useful links to our publications and other organisations who can help.

Read on

More information

If you would like to find out more about any of our campaigns, publications or anything else, get in touch and we'll be happy to help.

Get in touch
Related Posts Plugin for WordPress, Blogger...