I often feel a bit mixed after CF is in the news or on TV. It's either portrayed in drama as a 'terminal illness' where sufferers are pale, poor skinny souls without a hope. Or in the news as the NEXT BIG BREAK THROUGH disease. A cure is imminent! We can all throw out the nebulisers and look back on these days and laugh at the hours of physio endured!
The truth is, it is mostly neither.
Gene therapy is ground-breaking science, it really is. And like others in the CF community, I am hopeful that one day this may be a therapeutic treatment for cystic fibrosis. But I also believe we are many years away from this, and after the science, the funding will also delay this further. I'm also not hugely excited about a 3% increase in lung function. Isaac's can vary by 15% from one clinic visit to next. 3% and lung function stable? That would be, promising, but not exactly epic (as Ise would say), is it? I believe this is only the beginning of the long story of gene therapy. It is progress, and I am hopeful, but I'm not yet excited. And people saying to me how fabulous this is doesn't help much - I must look a bit deflated when I am less enthusiastic than they are. Sorry guys. But we've been here before.
Gene therapy was the buzz phrase when Isaac was a baby. We asked our consultant about it then, and she looked kind of flat in the same way. She surmised it might only work well in healthy lungs, not already destroyed by infection, inflammation and scarring. We asked at what age Isaac might have signs of this, and she replied age 5, maybe. When he way a baby, 2013 was talked about as a time when we might hope gene therapy drugs to be in the clinical setting. 2015 and we're still many years away. And yes, he probably does have all these signs already, but the trial showed positive results for those less well, so it is far from hopeless even though the wait continues.
A consultant once told us that they did not believe in a cure for CF. Which sounds like a crushing blow, but I think I agree. Rather, there will be many new therapies (small molecule drugs, new antibiotics….) that put together will hold off decline.
So great, the gene therapy results have raised the profile of CF (which is always good, helps raise funds, as well as awareness) and people are excited for us. Thank you. I'm just not sure that Press publicity accurately reflects what we should actually be excited about. Other trials results are a lot less sexy, but no less important.
In other Moly news;
One puppy, one less chicken. Oops.
Fibrogel is the evil cousin of Movicol - can anyone really drink that stuff? Back to the Movicol battles.
Summer holidays are fast approaching, and the Moly's will be going camping soon. Obie's first trip. We may be home sooner than we think.
My baby is 3! And starting pre-school soon - where did all the time go?
Isaac won most improved player of the year for his football team, and Anouk lost her first tooth and has her first horse show soon. Not sure which she is most excited about.
Have a great day.
