CF Treatments

Question

Hi, I'm a CF, I am 15 years old. I am looking for new drugs. I'd like your response. Thank you.

Answer

Hello,
Your question is not very accurate. If I understand, you want information on new drugs used to treat cystic fibrosis. This is a very broad question that's difficult to answer completely. To be schematic, it should be distinguished:

- Treatments directed against the consequences of the disease: this may be the airway clearance techniques, antibiotic treatment against respiratory infections, treatment of diabetes mellitus, pancreatic enzymes to aid the digestion of food .. .

- And treatments designed to treat the disease mechanism. It may be gene therapy that involves inserting into a normal gene the cells: it is a path that is still in the research stage. It can be also drugs that aim to correct or improve the functioning of the sick gene: the research that has led to the development of drugs as ivacaftor gave very positive results in patients with quite rare mutations and, more recently, the combination of ivacaftor and lumacaftor that can be used in F508del homozygous patients. The results, however less clear than in the previous case, were considered enough to get the marketing right to be used in everyday life. It generates even better results hopes for other drugs that are being studied.

I suggest you talk to the doctor of your CF Center.

Hope this answer can help.
Best wishes
Gilles RAULT, MD, Roscoff CF Center

27.02.2016