Saturday 13 October 2012

George Jenkins OBE, Chair of CF Trust speaks about his experiences at NACFC

I have now spent two full days meeting and talking with the world' s leaders in the science, research and treatments of cystic fibrosis.  

I have heard of the rapid and very real advances in recent understanding of the science behind CF, the root cause of the disease and now really do understand the defect that drives the disease, the reason that CF cells do not "breathe" in the way they should and how that leads to the thick sticky mucus and infection we all know so well.  

I can also understand that this recent advance has been won on the back of many years of very hard and determined research by dedicated individuals.  One guy I was speaking to had worked for twenty years on this subject, twenty years of hard work and some blind alleys that have now led to the very start of a "Road Map", a route that has led to the discovery of ivacaftor and upon which further work will, I hope and pray, bring the potential benefits of this drug the many, many more people with cystic fibrosis.

I have been so impressed by this real acceleration in drug research, clearly so have other drug manufacturers who for some time have been watching developments and are now committing to the endeavour.  I know that some of that is generated by the quest for corporate profits, but such profits will only flow if they deliver real drugs for real people.

My son Adam did not and sadly will not get the benefit of these advances and I know that more years of research, development, advances and set backs will be needed until the goal we seek is achieved.  But my investigations,  questioning  and challenge so far this weekend have proved to me that real progress is being made for the benefit of all.


  1. As I have read this...I am grateful for all the advances that is been made through a lot of hard work to find a cure for Cystic a mother of a 27 year old.. everyday that goes by is a HOPE that a cure will soon be found so I can keep my son with me ....Thank you to all those who are working hard to make this happen soon.God Bless you all!!!!

  2. Kalydeco (Ivafactor) would help about 3-4% of the CF population in the UK. It has been shown to improve lung function (FEV1) by 5-10% and slightly reduce exacerbations which is great. Related "small molecule" therapies may help people with the most common mutation dF508. So far they have only demonstrated safety and increased sweat chloride levels. These therapies are exciting and important but they will only bring incremental benefits. Around $100m has been committed, by the CFF and Vertex, to this particular programme in the US. Now Genzyme are also investing in this "small molecule" area. Why no mention of Gene Therapy at the NACFC? What Eric Alton and team are doing in the UK is far more profound and had the potential to cure rather than just tweak. 10% of the money being spent on small molecules would supercharge the Gene Therapy programme here. Is the real problem that the big drug companies don't want to see a cure since it is not going to be as lucrative as a gradually released series of incrementally better drugs (akin to the ever-lasting lightbulb problem)?

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