Cystic fibrosis affects around 30,000 children and adults in the United States and more than
twice that number worldwide. The genetic disease greatly limits the life expectancy of the people suffering from it and, while there have been great strides forward since the the late 1930s when it was first diagnosed as a specific disease, most people with cystic fibrosis can only hope to survive until their 30s or 40s.
The New England Journal of Medicine just published an article with very promising results for those suffering with cystic fibrosis:
The results of the trial, published in the New England Journal of Medicine, showed a combination of two drugs, lumacaftor and ivacaftor, improved the lung function of young patients and reduced their visits to hospital. It also cut the number of courses of hospital antibiotics they had to take after picking up infections, which is common. There was an improvement in their breathing, their weight and their quality of life.
The good news is that this treatment shows real promise. It also gives hope to researchers that treating the genetic defect can work. While it isn't a cure and there is no evidence yet that it can extend someone with CF's life expectancy, it does show that it can at the very least improve their standard of living. The drawback right now is cost:
However, the cost of the drugs is very high, which may place a question mark over their use. Ivacaftor alone costs $250,000 (£159,000) per patient per year in the USA. The price in the UK has not been revealed, but the Department of Health agreed to fund it for the small group for whom it was effective. The new combination will be scrutinised for cost-effectiveness by Nice, the National Institute for Care and Health Excellence.
Daily Kos community member TexMex has shared her family's
personal experience with the disease.