Gene Therapy question

[Guest guest]

Joe,

Did you ask Dr. Spencer when we might see our little ones get a chance to be

in the trial? I remember saying to him last year that it made sense to me that

the youngest would do the best if you assume they have the least amount of lung

damage. He agreed, but said at that point the youngest would be 12 years in

the clinical trial (then) coming up.

Keeley

Mom to Leila, 1yr. wcf and ZZ alpha, and Hadley 3 yrs. nocf

Re: Gene Therapy question

Dr. Spencer, who is, along with Dr. Flott heading up this Phse.2b at

Shands said they had 30 patients in Phse2 and upped it to 100

patients in 2b.

[Guest guest]

Keeley,

He told us the same thing. I guess the consensus was that they

didn't want to use the real little ones. Maybe it would've been

harder to tell if it was working. I'm only speculating. He may have

told me they had some 6-year-olds, I'm not sure. He told us so much

I've forgotten a lot of it. The thing that stuck with me was his

overall optimism about it. He mentioned that we needed to keep Abby

healthy and minimize lung damage so that if it is available in " say

five years " then she can take full advantage. Now of course I

realize he doesn't know that for sure but just knowing that someone

with his level of knowlege of it is optimistic enough to use the five

years as an example makes me feel good. He was the doctor that gave

us Abby's diagnosis and he didn't whitewash anything. Although he

was compassionate he didn't sugar coat the news. He even said that

the present median life expectancy was about 30 years. It wasn't

until later that I had a doctor tell me that a baby now may likely do

much better if the treatment curve continues as it is going now. In

other words, Dr. Spencer is a pretty conservative guy. I only pray

that the good results so far in the trial continue. They've been

slapped back several times because of problems with the vector

illiciting counter-productive immune responses but according to him

so far it seems to be working.

Joe

Dad of Kelsi,6 Chloe,2 and Abby(CF), 11 1/2 mos.(8/7)

> Joe,

>

> Did you ask Dr. Spencer when we might see our little ones get a

chance to be in the trial? I remember saying to him last year that

it made sense to me that the youngest would do the best if you assume

they have the least amount of lung damage. He agreed, but said at

that point the youngest would be 12 years in the clinical trial

(then) coming up.

>

> Keeley

> Mom to Leila, 1yr. wcf and ZZ alpha, and Hadley 3 yrs. nocf

> Re: Gene Therapy question

>

>

> Dr. Spencer, who is, along with Dr. Flott heading up this Phse.2b

at

> Shands said they had 30 patients in Phse2 and upped it to 100

> patients in 2b.

>

>