Re: Abby's Bronch/Thank you Joe!!

[Guest guest]

Joe,

Thank you for taking the time to give us this update. Like most

parents of small children hope is one thing that I have in abundance!!

Eventhough, I have only one child with CF the other two maybe

carriers, so I am very hopeful of their future and their offsprings

not having to deal with CF.

I hope Abby feels good very soon.

Be well,

mom of a 9 wcf, Venanzio 7 nocf, Pepe 3 nocf

> Our 11 month old Abby had her bronchoscopy today and all went

well.

> Dr. Terry Spencer performed it and said that he saw very little

> mucous on one side and what he suctioned out looked clear. He said

> he wouldn't be surprised if she did not have Pa in her lungs. Said

> it could just be in her upper airways and was unsure how or if they

> would go about eradicating it if that was the case. Personally,

I'll

> be surprised if none grows from down there since it grew from the

> first bronch - even though they treated it aggressively. After all

> if it's shown up in the upper airways after all that Tobi etc. then

> what's to say it isn't still in her lungs. I hope and pray I get

> surprised. We'll find out Friday what the culture is doing.

>

> What I really wanted to share was what Dr. Spencer told us about

the

> gene therapy clinical trial. Abby yanked out her IV before they

got

> her drugged and they had to call the " IV people " back up there so

> they could re-do it. This gave us about 45 minutes with Spencer

and

> we asked a lot of questions. The trial was his favorite subject.

As

> you know, Phse. IIb of the aerosolized gene therapy trial using the

> AAV vector is underway nationwide and Dr. Spencer along with Dr.

> Flott are heading this up at Shands in Gainesville with 100

> patients. He said he was extremely encouraged by what he saw in

the

> first group (30 patients) and believes this vector holds real

> promise. It doesn't seem to illicit any serious immune response

like

> the adenovirus and doesn't need any " helper viruses " to transfect

the

> cell.

>

> He said that when the gene was discovered in '89 that everyone

> expected that there would be gene therapy available by the mid-

> nineties and people have been discouraged by the slow pace. He

feels

> as though things are moving again and even went as far as to say 5

> years was a real possibility. As you know the barriers have

largely

> had to do with the vectors since many have caused bad immune

> responses or had other side effects which compounded problems. He

> said the AAV seemed to be working and according to him there were

no

> problems with it so far. I realize that many of you may have grown

> tired of getting excited about a cure just to see it fail because

of

> some crazy reason but I was very encouraged today by Dr. Spencer's

> comments.

>

> Joe