What is cystic fibrosis cure?

Answer 1

In truth, there is no cure for cystic fibrosis; the disease is most commonly caused by a genetic mutation. The mutation is caused by an incorrect protein made by the "Cystic Fibrosis Transmembrane Conductance Regulator" which is responsible for managing most of the body's sodium-chloride channel. The problem is the protein is not made correctly into a 3-dimensional biological structure (or it's not coded in the right way) and so the body's detection system throws it out. Without this gene, the body is unable to process water and salt correctly. This in turn causes multiple systematic infections and even failures. Due to inability to process water, a CF patient's body cannot make thin and easily removable mucus. This mucus is used by the body to lubricate organs and pick up pathogens (germs and stuff that are capable of attacking the body.) Normally a person's body is able to dispose or throw out the mucus quite easily (i.e. when you get sick, your body amps up fluid and mucus production in order to rid the body of infection, hence the reason for a runny nose or wet cough) but in Cystic Fibrosis the mucus is not hydrated with enough water and salt, so it becomes uncontrollably thick. At first, a person with the most common mutation of CF (Delta-f508) will display malnourishment due to their inability to process enzymes. For those who need simpler explanations, the mucus in the organ "the pancreas" is so thick that most of the body's tools to break down and eat food are trapped. This means the body cannot absorb or use food. This leads to weight loss and death if untreated. Further on in childhood, a CFer (as we call ourselves) starts to cough and succumb to infections in the lungs and elsewhere. The lungs are filled with mucus in order to catch germs in the air we breathe in order to prevent people from getting sick (sometimes you still get sick though) and for other reasons as well. The mucus in CF patients, however, is too thick for the body to get rid of and so the bacteria sit there and grow until they become too much for the body's immune (defense) system to fight. This causes scarring and destruction of the lung tissue, which in turn decreases the body's ability to absorb oxygen and eventually, its ability to breathe. This will likely be the reason for the patient's death when they can no longer tolerate the loss of lung function. Other ailments for CF patients-due to the genetic mutation-are: liver failure from mucus buildup which stops the liver from being able to detoxify food and other things; liver bile can also become too thick and acidic; inadvertently it can cause kidney failure from the antibiotics which are used to treat infections; infertility among males is also at 99%-women on the other hand can have a tough time getting pregnant and at having birth due to thick mucus preventing sperm from entering the female reproduction system and due to the fact the patient will have a tough time supporting not only themselves but also the child within them (another problem to consider is the affects of the medication the mother needs to take to treat her CF on the baby.) Diabetes (CFRD-Cystic Fibrosis Related Diabetes) can also be caused by CF due to possible steroid treatment and the fact that the pancreas can get blocked up with mucus even more and be unable to release insulin to decrease sugar levels. Another problem which is inadvertently caused by CF is increased cholesterol levels in part because doctors told CF patients to go out and eat whatever food they want as much as they can-which included fast food-expecting that CF patients wouldn't live long enough to suffer from high cholesterol but as new treatments have come out which prolong the life of CFers, people are now suffering from this. Constipation is also prevalent due to the body's lack of support for the sodium chloride channel. The sinuses are also stuffed with mucus and can form polyps with numerous nasal infections. When lung function is bad enough, a lung transplant occurs (if the patient is able to receive a donor that is) and then the problem of the body rejecting the organ can come up. Medicines are needed to stop the body from attacking the new lungs, but then this weakens the immune system and increases the likelihood of getting a new infection that can weaken or kill the patient. Towards the end, CF patients will have to deal with a lot of bad pain. This can be treated with pain medicine (that is if the doctor actually cares whether or not the patient is in pain and will help treat them) but sometimes it won't help much. Throughout their life, a CF patient will feel much pain, from pulled muscles and scratched throats from the coughing, to stomach pain and side effects from powerful antibiotics. Fortunately, the CF Foundation (CFF) has worked vigorously to increase the lifespan and livelihood of us CFers and now the average life expectancy went from 5 years of age to around 30 years of age in the last 60 years.

All in all, the reason there is no cure for CF is because medical science is currently unable to correct or infuse a working CFTR protein. Only when this is accomplished can most of the mutations from Cystic Fibrosis be truly "cured." For those of us who were born with CF and have been alive more than a decade, a cure will not cure us, but will stop the progression and destruction of the disease. The problem is that since so much damage has already been done to people who have lived a while with CF, a working protein will not fix the damage just stop it.

My Sources: I have CF and have dealt with most of this. For more information, go to www.cff.org.