A science conference in the UK has heard some good news which offers hope to cystic fibrosis (CF) patients worldwide.
At the BA Festival of Science in Liverpool this week, Dr. David Sheppard from the University of Bristol said a new drug therapy may represent a tremendous step forward in the treatment of cystic fibrosis.
According to Dr. Sheppard the early results with the drug VX-770 suggest that drug therapies which target defects at the root of the disease have the potential to greatly improve the quality of life of CF patients.
There is currently no cure for CF, which is a hereditary disease caused by a common single, defective gene disorder which affects about 70,000 people worldwide.
CF causes ducts and tubes in the body to become blocked by a thick, sticky mucus which affects the lungs, pancreas, the intestines, the liver and the reproductive organs.
Most individuals with cystic fibrosis die young, many in their 20s and 30s from lung failure but with the introduction of many new treatments, the life expectancy of a person with CF is increasing to ages as high as 40 or 50.
The treatment as a rule targets airway infection along with the encouragement of good nutrition and an active lifestyle.
The treatment for CF continues throughout a patient's life and is aimed at maximizing organ function, and therefore quality of life, but at best, current treatments serve only to delay the decline in organ function.
Treatment is tailored to the individual, because of the wide variation in disease symptoms and targets the lungs, gastrointestinal tract, the reproductive organs and psychological support; therapies such as transplantation and gene therapy aim to cure some of the effects of cystic fibrosis.
The most consistent aspect of therapy in cystic fibrosis is limiting and treating the lung damage caused by the thick mucus and infections, with the goal of maintaining quality of life.
Intravenous, inhaled, and oral antibiotics are used to treat chronic and acute infections and mechanical devices and inhalation medications are used to alter and clear the thickened mucus.
One of the most recognisable symptoms of CF is 'salty sweat', caused by the failure of the sweat ducts to reabsorb salt and existing treatments only alleviate symptoms, for example, physiotherapy to clear the air passages, antibiotics for lung infections and enzymes to aid the digestion of food.