A Drug for Cystic Fibrosis?

Cystic Fibrosis is a well known genetic lung disease. If you have Cystic Fibrosis you have mucus in your lungs that builds up and makes it difficult to breathe, also it opens you up to the possibility of recieving an infection. Cystic Fibrosis is the most common genetic disease in white people. 

In August, 1989 American and Candadian scientists discovered the gene responsible for Cystic Fibrosis. When this happened scientists believe that they would be able to quickly come up with a cure, or drug that would allow them to treat the disease. Now, almost 25 years later, a druge to treat the disease is possible. It took the scientists this long to test 600,000 possible chemical solutions that would help fix the incorrect protein that produced Cystic Fibrosis. The new drug is one called Kalyedco.

Kalyedco like all drug is not perfect. It does not cure the disease but it helps to control it. Also, it is very expensive, it can cost $300,000 per year. Another downside is it doesn't work for everyone with Cystic Fibrosis, only those with a certain type of mutation in that gene. The scientists that have helped to create Kalyedco are still on the search for a drug to help a greater amount of people with Cystic Fibrosis, but they have been able to accomplish much with Kalyedco. A woman that is on Kalyedco (she has the specific gene mutation necessary for it to work) is able to breathe and not feel as if her lungs are filled with mucous, instead they feel clear. For her this is enough, for the scientists not so much.

The only way currently to fix the protien and thus end Cystic Fibrosis comes from gene therapy. Apparently gene therapy is not easy though. There is a good chance that it will not work and takes a lot of time and effort. So as of now there is no definite  way to cure Cystic Fibrosis, but the fact that scienists were able to come up with a drug that benefits some is a good start.

Hopefully someday soon scientists will be able to help end Cystic Fibrosis. As they progress towards this ending goal it is good that they are at least capable of making drugs to help those currently struggling with it. Although the drug is defintely very expensive, like all other drugs over time it is bound to drop in price. It is important that we focus on developing drugs that help or even can cure diseases such as Cystic Fibrosis, its a long difficult task to find a solution to these issues and takes true dedication. If we could eliminate Cystic Fibrosis there is the possibility to eliminate other genetic diseases, this drug has gotten the ball rolling for more to come in this specific focus and hopefully some others as well. I hope that one day during my life time Cystic Fibrosis will no longer be a disease people have to worry about, and I hope it is not the only one like that.

http://www.npr.org/blogs/health/2013/01/02/168353348/drug-fulfills-promise-of-research-into-cystic-fibrosis-gene