7 September, 2016

Stephen Nyangoma and Vian Rajabdazeh attended the first day (7 September 2016) of the annual UK Cystic Fibrosis Conference, a two-day event with over 400 delegates, held in Nottingham.

The day was designed for a multi-disciplinary clinical, research and academic audience – which provided the latest of CF research and innovation, and focused upon the opportunities raised by the development of precision and personalised medicine.

There has been a recent paradigm shift in the way in which an individual is treated for CF, with treatment targeting the Cystic Fibrosis Transmembrance conductance Regulator CFTR molecule, rather than solely treating the secondary symptoms that are precipitated by the mutated CFTR protein.

In terms of directly targeting the CFTR and depending on which mutation an individual possesses, there are several approaches, to improve the conductance and potentiation of the CFTR channel (to improve the conductance of chloride ions through), and to amplify the number of CFTR proteins and increase the efficiency of the protein.  Treatments can act on improving the conductance of chloride ions through the channel or increase protein expression to the cell surface. Orkambi is an example of combined treatment (Ivacaftor and Lumcaftor), both exhibiting different mechanisms of action.

A highlight of the conference was a talk by Professor Robert Ford, Chair in Structural Biology, University of Manchester, presenting the structure of CFTR molecule and demonstrating how lumcaftor binds to this. Lumacaftor assists CFTR protein during the folding process, and also increases the number that are trafficked to the cell surface.

The day was enlightening, and it was interesting to study, at the molecular level, the 'mechanisms of action' of the drugs we analyse in pharmacovigilance.  

Further details on the CF Trust can be seen here: https://www.cysticfibrosis.org.uk/.