New advances in cystic fibrosis drug therapy

 

cystic fibrosis lungs

There are between 70,000 and 100,000 people living with this chronic life-threatening disease worldwide.

A new combination drug therapy could provide hope for thousands of cystic fibrosis sufferers and greatly improve their quality of life.

Cystic fibrosis is a hereditary disease that affects the respiratory and digestive systems. Due to DNA errors, unusually thick mucus is formed in the lungs of the sufferers that results in damage to the lungs. This can lead to infection, breathing issues and hinder digestion. Cystic fibrosis sufferers currently have to undergo rigorous daily treatment in order to eat and breath normally.

The life-threatening disease is estimated to affect 1 in every 2500 babies. The average life expectancy of a sufferer in the USA or UK is 35-40. Fortunately, as further research is conducted about this disease, the life expectancy rate has been steadily increasing.

Underdeveloped countries

There is a significant lack of information regarding cystic fibrosis in underdeveloped countries due to a lack of awareness. It was often thought that cystic fibrosis did not exist in these areas as it was being regularly misdiagnosed, with the symptoms being treated separately. The average life expectancy in these countries is 15 years or under which highlights the urgent need for increased awareness of the disease.

New treatments

The latest trials on cystic fibrosis use a combination of two experimental drugs, lumacaftor and ivacaftor, to treat patients. After 24 weeks, the researchers found that patients had improved lung and digestive function.

Since cystic fibrosis is caused by a number of DNA defects, the symptoms cannot be alleviated by one treatment alone. However, it is believed that this new combined drug regime will help around half of people with this condition, which would be a significant step towards eventually fighting this disease.

This therapy is currently being reviewed by regulators around the world. The ongoing research and campaigns to increase cystic fibrosis awareness give hope to cystic fibrosis sufferers across the globe.