Breakthrough with Gene Therapy

About two weeks ago Tom and I went into London for a special meeting for CF. The CF Trust had sent out the invitation beginning of December stating they wanted to update us on gene therapy. I knew that we would hear one of two things: it worked, or it didn't work. I was not looking forward to going if the latter was true. Thank goodness Tom decided to blow off busy season work and come with.

I'll give a bit of background on gene therapy. What it is meant to do is get a normal functioning gene into the core of the mutated gene, which is buried in the small airways of the lung under cilia and a whole lot of mucus. If this can happen (it takes no less than a miracle!), the normal gene overrides the mutated gene and the CF goes away...or Jeb becomes a carrier like me, Tom and Aiden (and btw, 1 out of 25 people who are CF carrier...sorry, had to get that message out!). The hard part of gene therapy is tricking the body into thinking this new gene should be there and not trying to get rid of what the body thinks is a virus.

Ok. So, the update. First we listened to the head of the Advisory Committee, and Australian who had nothing but great things to say about the research. Then we listened to a French member of the advisory team tell us basically how dna is created and processed. Not only could we not understand the content, which was so far over our heads, but we couldn't really understand what he was saying due to the thick French accent!

Then the man of the hour came. The Dr. who is leading the team, which is working tirelessly in Edinburgh, Oxford and London. He gave the background of how this started - 10 years ago they tried it and it improved lung function 25%. Not good enough. They started over. This time they gave the gene in a lipid (slippery stuff designed to get it through everything and into the small airway) with a new nebuliser. The result after 2 weeks - 50% improvement, 4 weeks - 100%, 6 weeks 100% and 9 weeks 100%. It then started slipping back at 12 weeks, when a new dose would be given. As he was showing us this on the chart you could feel everyone holding their breath and as those 100% marks came up, the auditorium was filled with sighs of relief from 1000 people who have been waiting for this. Some a whole lot longer than us.

So, the next steps. A multidose trial, first with sheep, who have lungs very similar to humans. That will last 18 months. Then with 150 UK CF patients, that will also last 18 months. If it works, it will go worldwide for 6 months and then out to market in the UK. I was a bit worried that it would take awhile to get to the states. No, it will go out to the world within weeks, if not days.

5 years. It's not a cure, but it means a nebuliser every month for 30 minutes. No physical therapy. No worry of nasty bugs that cause lung disease. It's just one step closer to a normal life...whatever that may mean.

I did speak with a friend in the states, who has Elyse, a little girl born 2 days after Jeb and also has CF. The states are working on drugs to correct the gene. And it looks like it's getting close - so, if that comes out in the next 3 years, it will only make Jeb stronger for when gene therapy comes. It's what we've been praying for. And there is a wave 2 product called Lentivirus, which was used once - kept the lung healthy for 2 years. It's in it's infancy, but there is so much hope!

We walked away feeling very encouraged, but still praying. Hope you will continue your prayers for Jeb also.