Dear Abdulaziz,

Although stem cell therapies are starting to show some clinical promise for some diseases, I doubt that effective stem cell therapy for CF will happen in the next decade. Too difficult to get the stem cells to where they are needed and make them do something useful. CF patients have lots of sticky mucus, that makes any apically-applied therapy tricky - gene therapy has been struggling with this for years.

However the Lumacaftor-Ivacaftor approach looks promising for a broader CF population (http://www.ncbi.nlm.nih.gov/pubmed/25981758).

BW, Simon

Dear Simon

many thanks for you reply to my question

Hello

Ivacaftor clearly is beneficial in a specific subgroup of CF patients, but I am not as impressed with Lumacaftor-Ivacaftor in the homozygote deltaF508 patients, so time will tell whether this therapy will be that impactful. We are beginning some work looking at tissue specific cells in CF. This work is very early as our group is just starting.

Thanks!

Hi Don,

Just out of interest, what cells are you looking at and how would you deliver them? I'm surrounded by stem cell people and have a side-interest in CF so it would be great to get your perspective.

Thanks, Simon