IMPORTANT INFORMATION

  • CFTR.info is an online educational resource on the genetics of cystic fibrosis (CF) and the role of CF transmembrane conductance regulator (CFTR) in normal human physiology as well as in CF.
  • The information contained herein is intended for healthcare professionals working within the European Union (EU).
  • The content of this website has been developed independently by a group of CF healthcare professionals and is for general information purposes only.
  • All of the information about CFTR and CFTR modulators has been obtained from referenced sources and is in the public domain.
  • This website is not intended to promote or advocate a particular course of treatment or any specific drug, whether licensed or unlicensed, nor is it intended to imply or suggest that any drug be used for any purpose other than that which for which it is licensed in the EU.

ABBREVIATED TERMS & CONDITIONS

  • While we endeavour to keep the information up to date and correct, we make no representations or warranties of any kind, express or implied, about the completeness, accuracy, reliability, suitability or availability with respect to the website or the information and related graphics, or products and treatments, contained on the website for any purpose. Any reliance you place on such information is therefore strictly at your own risk.
  • In no event will we be liable for any loss or damage including without limitation, indirect or consequential loss or damage, or any loss or damage whatsoever arising from, or in connection with, the use of this website.
  • Through this website you are able to link to other websites which are not under our control. We have no control over the nature, content and availability of those sites. The inclusion of any links does not necessarily imply a recommendation or endorse the views expressed within them.
  • Every effort is made to keep the website up and running smoothly. However, we take no responsibility for, and will not be liable for, the website being temporarily unavailable due to technical issues beyond our control.

Please see the full Terms & Conditions before using this website.

 

I have read and understood the above

Clinical management of CF » CFTR modulators » Summary

Summary

3 of 3

Click on the tabs to move through the sequence.

To summarise:

Clinical investigations are underway to evaluate the safety and efficacy of combining potentiators and correctors together. It is important to note, however, that CFTR modulators have been developed to ‘repair’ specific mutation defects and not entire classes of mutations.

 

Potentiators and correctors

 

Potentiators increase the flow of Cl through CFTR channels at the cell surface.

 

 

 

Correctors increase the cellular processing and delivery of CFTR proteins to the cell surface.

 

Combination therapies


There is increasing interest in the use of combination therapies. Combining a corrector and potentiator may serve to augment CFTR function and is currently under investigation in clinical trials.5

Comparison chart


This table shows an overview of CFTR modulators that are either approved or are in development for the treatment of CF.6

 

3 of 3
References
  1. Sloane PA. Curr Opin Pulm Med 2010;16:591–7
  2. O’Sullivan BP & Freedman SD. Lancet 2009;373:1891–1904
  3. Rogan MP. Chest 2011;139;1480–90
  4. Welsh MJ et al. Cystic fibrosis. In Valle D et al (Eds). OMMBID. The McGraw-Hill Companies Inc 2004. Part 21, chapter 201
  5. Clinicaltrials.gov. http://clinicaltrials.gov/ct2/show/NCT01225211
  6. Adapted from Rogan MP et al. Chest 2011;139;1480–90