What is afoot?

Question

vx-770, vx 809-miglustat, ptc124, DNAase, all these are related to CF, but how are things coming along? What are all these? How has the financial crisis these last two years affected the research? Is gene therapy still being researched, or has it been abandoned? Would it be possible to make custom lungs and pancreas? I do not know if I am overly anxious, but I do not know the pace of the research and I do not see any substantial advancement for the next 5-6 years.

Answer

Dear friend,
VX770 will soon be commercially available for CF patients who carry the mutation G551D.
The administration of VX809 in conjunction with the administration of VX770, is being investigated and we expect the results.
The PTC124 has been found to be effective for stopping mutations in the group of mutations, whose name ends in X (e.g. W1282X & G542X). The first results are encouraging and come from USA and Israel and the research is under progress.
DNAse is commercially available for a long time now.
Greece may suffer greatly from the financial crisis, something that is not the case for e.g. Canada or Germany, therefore the scientific research is not affected.
The research about the cure of CF is intensive and promising. One part of it has to do with gene therapy and is under continuous development.
It is normal for you to be anxious, but it takes many years of intensive and systematic research to produce a new drug that is safe and effective to administer to patients.
It may take more than 5-6 years for a real breakthrough to take place.

Friendly
Dr. Stavros Doudounakis

23.01.2012