Message Boards for the CF Trust

Replies: 11 Pages: 1

colster

Posts: 39
From: Southampton, UK
Registered: 06-Nov-2006

new CF drug
Posted: 15-Dec-2006 16:17

Reply

Looks very interesting.

Question though, why is the uk cf trust not involved?

http://www.israel21c.org/link.jsp?enDispWho=Articles^l1480&enZone=Health

Darling

Posts: 731
From: Here
Registered: 06-Feb-2004

Re: new CF drug
Posted: 15-Dec-2006 16:29

Reply

Why would the CF Trust be involved? (being Devil's advocate

)

Already funding lots of research over here into treatments and very well focussed. It's not like new treatments elsewhere will be kept secret if successful.

Good spot and interesting reading

ladybird

Posts: 226
From: Northants
Registered: 05-Nov-2006

Re: new CF drug
Posted: 15-Dec-2006 17:02

Reply

Some of us on the parents board have been following this for a while now and one has actually met some of the team involved. We will be going over to Israel in February all being well and will at least talk to them on the phone (hubby has spoken to the CF nurse there already). I'm also hoping to meet up with a physiotherapist whilst there as I believe the physio is very different there (more pilates/yoga based), but i could be wrong.

This drug (PTC124) will only be useful for those with a "stop" gene (or "nonsense" mutation) as I understand it. The most common genetic mutation (W1282X)in Israel is one of those! Ocean has this gene so it's of great hope to us. So far things are looking positive.

If anyone has any questions about the trials, then feel free to pass them to me and IF I get the chance I will ask them!

Shadow x

cupcake

Posts: 141
From: england
Registered: 02-Mar-2004

Re: new CF drug
Posted: 15-Dec-2006 17:03

Reply

I think that its possible that only CF patients with a certain gene would benefit from this treatment, and not many pwcf in the uk have the gene that this treatment can help. I could be wrong but I'm sure I read this on the parents site a while ago.

Message was edited by: cupcake who was too slow and was beaten by someone with much more knowlegde on this sudbect

sunseasand

Posts: 74
Registered: 10-Nov-2006

Re: new CF drug
Posted: 15-Dec-2006 17:20

Reply

Even if this new drug is not of any benefit to most pwcf in uk, I feel any new drug which benefits cf will help in the long run all pwcf. It gives better understanding of how cf works and also who knows they may then be able to make drugs like these that will benefit all. Everything lately seems to be fitting in like a puzzle and one day lets hope that puzzle will be finished and there will be a cure for all pwcf. I did not know the benefits of the Dead Sea for people with lung probs, that also sounds cool. from sunseasand.

ladybird

Posts: 226
From: Northants
Registered: 05-Nov-2006

Re: new CF drug
Posted: 15-Dec-2006 18:49

Reply

I don't think we'll make it to the Dead Sea when we're there unfortunately. I don't really relish the idea of keeping three young children out of the water (or at least the water out of their eyes - believe me that HURTS) there either. I've been there a few times and can't think of much else to do there (I'm sure hubby will put me right on that one though).

I've spoken to a geneticist about this drug and she explained the theory behind it. I'm not sure I've got it right, but I believe it will work by blanketing the relevant gene (the "stop" gene) effectively making the pwcf a carrier. They won't actually BE a carrier, they will still have CF and any previous damage won't be reversed, but it could halt the progression of CF.

I could, however, have that completely wrong!

softie

Posts: 1,070
From: Croydon, Surrey
Registered: 07-Feb-2004

Re: new CF drug
Posted: 15-Dec-2006 19:42

Reply

While this new drug is being taken, people with the right mutation will produce enough working CFTR (the protein coded for by the CF gene) to have a reduction in symptoms. As soon as they stop taking it then they will start to deteriorate at the normal rate. Also, while it may reduce symptoms enough that the lungs can reverse some damage, much of the damage will be irreversible.

I assume the Trust isn't in on this because doctors in this country would be able to find too few patients with this mutation to make it worth the effort of setting up trials. The American company will be able to make money out of their large Jewish population and the worldwide population of peple with that particular mutation, and it is worth it for doctors in Israel as they have such a very large population with that mutation.

As far as helping the rest of us... Well, probably not. Each mutation is different and needs treating differently, so what is learnt about that mutation will be of little relevance to what works for other genes. This doesn't apply to gene therapy, which aims to add a completely working copy of the gene, and should work whatever pre-existing copies of the gene there are.

Richard.

Tim Davies-Moss

Posts: 145
From: Shropshire
Registered: 06-Nov-2006

Re: new CF drug
Posted: 16-Dec-2006 16:53

Reply

please excuse my ignorance but what is the stop gene ?

maybe i have never heard of it because it does not effect me.. but am very interested

ladybird

Posts: 226
From: Northants
Registered: 05-Nov-2006

Re: new CF drug
Posted: 16-Dec-2006 17:07

Reply

I could be wrong, but I think they generally end in X.

Something along the lines of them finishing a thread of DNA rather than causing a blip in a line.

I'll shut up now as genetics isn't my strong point and that's about all I gleaned from a meeting with a geneticist. I'll ask hubby when he gets home if no-one else comes up with a better explanation!

Lucy

Posts: 87
From: North England
Registered: 09-Feb-2004

Re: new CF drug
Posted: 16-Dec-2006 18:59

Reply

No good for those of us who are homozygous for df508 then?

softie

Posts: 1,070
From: Croydon, Surrey
Registered: 07-Feb-2004

Re: new CF drug
Posted: 16-Dec-2006 19:54

Reply

> No good for those of us who are homozygous for df508
> then?

Nope, 'fraid not. However, there are a number of teams who are looking into drugs that encourage DF508 to be expressed and to operate correctly when expressed, so there is hope for the homozygous DF508ers amongst us!

Richard.

sally 1

Posts: 128
From: oldham
Registered: 09-Nov-2006

for ladybird
Posted: 16-Dec-2006 21:53

Reply

crickey how did that happen?

Confused of Oldham

Message was edited by: sally 1