What is cystic fibrosis?
Cystic fibrosis is one of the UK’s most common life-threatening genetic conditions, and affects over 10,500 in the UK alone. It’s caused by inheriting two copies of a defective gene that one in 25 people in the UK carry, usually without knowing it. If two carriers have a baby, the child has a one-in-four chance of having cystic fibrosis.
Cystic fibrosis affects the movement of salt between cells, causing the liquid in the lungs and digestive system to be thicker than usual. This can make it difficult to breathe and cause repeated lung infections, as well as issues with digesting food and absorbing nutrients. Half of those who died from CF last year were aged under 32, though babies born with the condition today could be expected to live much longer.
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Can cystic fibrosis be cured?
Cystic fibrosis is a genetic condition, and as such cannot be cured. However, there are treatments being developed all the time that are helping to improve the lives of people with the condition, including new ‘precision medicines,’ which the Cystic Fibrosis Trust has been campaigning for alongside the CF community for several years.
Why can’t people with cystic fibrosis meet each other?
In the past, people with CF were allowed to spend time with each other and some will even remember going to ‘CF camps’, where they were able to meet and spend time with others with the condition.
However, in the early 1990s it was discovered that the bug B. cepacia could pass between people with cystic fibrosis. This is called cross-infection.
When the spread of B. cepacia was discovered, it was recommended that people with the bug be segregated. This eventually came to be the policy for all people with cystic fibrosis when it was discovered that people with CF could pass a number of dangerous bugs between each other. While in the US, a rule exists called the ‘six foot rule’ which says that people with CF must be kept a minimum of six feet apart. today in the UK it is recommended that people with CF do not meet or spend time with each other.
Where this isn’t possible – for example, in families where two children have the condition – stringent cross-infection procedures are taken to ensure that people with CF are kept safe and do not contract dangerous bugs.
Why do people with CF contract bugs?
A number of bugs can be harmless to people who don’t have CF, but because of the thick, sticky mucus that sits in the bodies of people with CF, these usually harmless bugs can settle in the lungs (known as colonising) and cause devastating damage.
People with CF can carry a number of different bugs, including Pseudomonas aeruginosa, Burkholderia cepacian, Staphylococcus aureus, H. influenza, Aspergillus, MRSA and Nontuberculous Mycobacterium abscessus, some of which can make people with CF ineligible for lung transplant.
About CF bugs
Why do some people with CF need lung transplants?
Some people with CF will need a lung transplant because of the damage that is done to their lungs by repeated bacterial infections. This is not a procedure that is taken lightly, and deciding when to list someone with CF for transplant is a very complicated decision involving a number of factors, like how unwell the person is and what bugs they are infected with.
It is still important that people with CF do not meet, even after they have had a lung transplant, as they can still carry and spread dangerous germs to other people with cystic fibrosis.
Do you still have CF once you have had a lung transplant?
A lung transplant, or other organ transplant – for example transplant of the pancreas or liver, which can also be damaged by cystic fibrosis – will replace a damaged organ with a healthy one. However, as cystic fibrosis is a genetic condition, the rest of the person’s body will continue to have cystic fibrosis, and some CF treatments may still be needed after a transplant.
What is haemoptysis?
Haemoptysis is the medical term for coughing up blood. Not all people with CF will experience haemoptysis, and most will only experience it to a small degree. Haemoptysis is often a sign of increased infection, as infection irritates small blood vessels and can cause them to bleed. While coughing up blood can be very scary, it is treated just like any lung infection; with antibiotics or airway clearance techniques. If haemoptysis is very severe, it might require hospitalization.
What is a clinical trial and why do they matter?
Clinical trials are studies designed to investigate the effects of new treatments on human health. These treatments might be new medicines, medical devices or behavioural and lifestyle changes. People with CF may take part to trial new and emerging CF treatments, and some of the treatments that were trialled through clinical trials in the past are now used commonly to treat aspects of the condition. Hear from Caitlin about her experiences taking part in a CF clinical trial.
The Cystic Fibrosis Trust started the UK’s first CF clinical trials database, the Trials Tracker, which allows people with CF to find trials they are eligible for and where they are running.
About clinical trials
Is it possible to ‘fake your way’ onto a clinical trial?
Due to the digitized nature of hospital records, it is almost impossible to falsify them, and to end up on a clinical trial that you are not eligible to take part in. Most people with CF have a strong relationship with their CF team, who would have full record of their health. Even if, like in Casualty, an A&E doctor had knowledge of a suitable CF trial for a patient they were treating, they would need to liaise with the patient’s CF team to refer them.
Every trial has an ‘eligibility criteria’. Before you are entered in to a clinical trial, you will need to attend a ‘screening visit’. This will be an appointment with a member of the local trial team, which will determine whether it is okay for you to take part in the trial. This will involve checking your health and medical history against the inclusion and exclusion criteria of a trial. This screening process will be explained to you before hand, and will only take place if you have given your consent to take part in the trial.
Being told you’re not eligible to take part in a particular clinical trial can understandably be very disappointing, however you may find you are eligible to participate in a different trial. It’s really important that clinical trials have strict eligibility criteria in place to make sure the trial is conducted as safely and accurately as possible.
What happens if you experience side effects during a trial?
Clinical trials are carefully controlled, regulated and organised to minimise risk to participants. Serious side effects are very rare and clinical trials are now safer than ever.
New medications will have been checked using human and animal cells before ever being administered to humans, and early trials for new medications may involve heathy volunteers rather than people with the condition the drugs are being tested to treat. However, as with any new medical treatment, there is always a small possibility of unpleasant or potentially serious side effects. It’s worth remembering that not all trials are for new medications – some trials involve commonly prescribed medications to see if treatments effective for other conditions are also effective in cystic fibrosis.
Every trial has an independent Data Safety Monitoring Board (DSMB), and if anything happens during a trial, such as a bad reaction, it is immediately reported to the DSMB. The board will stop a trial if there is any doubt about the safety of the treatment.