Abby's Bronch/Gene Therapy Trial

[Guest guest]

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

[Guest guest]

Deb,

We elected not to stay in during the bronch. We had to help hold her

down for the IV (twice) and when they finally got the drug in her

we'd seen enough. We stayed until she started smiling at them all

doped up and they put her in the " straight jacket " . So I don't know

if she coughed or not. I do know she cried one time and they had to

give her more anesthetic. This caused everything to slow down more

because of the delayed recovery.

Joe

> In a message dated 7/15/2003 7:53:05 PM Central Daylight Time,

> reamsfarm@m... writes:

>

>

> > Joe

>

> I am so glad to hear everything went well for Abby with her

bronch!!

> 's bronch went well also the only thing I think that was

the worst thing for

> her was putting in the IV. She really really hates that!!. They

told us she had

> some mucus more in her lung area then her airway they also said it

looked red

> in some areas (I hope that doesn't mean anything bad) but like you

we are

> waiting for Friday to get hear the results. Our doctor was talking

to us about a

> new powdered Tobi which would be like an inhaler which would be a

lot easier

> for us he said we might be a good candidate for it if they do a

study here. I

> hope so!!

> Were you able to stay in the room when they did Abby's bronch? We

were and it

> really scared me how much she was coughing but they said when they

put the

> scope down her throat it is in a really sensitive area that make

them cough. It

> was really neat to watch it on the TV screen!! It is amazing how

they do

> thing!!! doesn't remember a whole lot about the actual

scope going down

> her throat but she hated the nebulizer stuff they used to numb her

throat I

> though she was going to get sick. Thank God she didn't have any

food in her tummy

> or I think she would have. OK now that I have grossed you out I am

going to go

> but I just wanted to let you know all went well in Iowa too. Cya

Deb A

>

>

>

[Guest guest]

In a message dated 7/16/2003 12:07:21 PM Central Daylight Time,

reamsfarm@... writes:

> We elected not to stay in during the bronch. We had to help hold her

> down for the IV (twice) and when they finally got the drug in her

> we'd seen enough. We stayed until she started smiling at them all

> doped up and they put her in the " straight jacket " . So I don't know

> if she coughed or not. I do know she cried one time and they had to

> give her more anesthetic. This caused everything to slow down more

> because of the delayed recovery.

>

> Joe

Joe,

I am sorry to hear she had a rough time which I know it is hard on everyone

that is involved. Our daughter really wanted someone to stay in so that is why

I really wanted to even though she didn't know if we were there or not. She

did very well she was awake and alert as soon as they wheeled her out of the

room. Well, I hope you hear a good results and I hope we do too. Deb A