Re: Cleveland gene therapy report

[Guest guest]

It looks really promising, but I just take a " wait and see " approach

now. When my son was first diagnosed, I watched the research and

read every scrap of information about cf. I got really excited about

trials, only to be heartbroken when they didn't pan out. And

by " heartbroken " I really mean it just felt like my heart was crushed

each time I read the results of another unsuccessful treatment.

The things that I see with this particular study that are not so good

are that:

the site of administration was the nasal passages - noses aren't

lungs, so there's a chance that the good results won't translate to a

good treatment.

they used a " wash " and are going to try to develop an aerosol - to

me, this says they don't know if the aerosol will actually deliver to

to the lungs in a way that the gene transfer will " take "

and finally, the study group was PAINFULLY SMALL, with only twelve

participants. (what WERE they thinking???)

On the positive end, I noticed that:

ALL of the participants finished the study (this seems unusual and

good, but again, only twelve were involved.)

The treatment actually had a positive effect at a cellular level at

the site of administration (often during a study, things just don't

work like they did when they tried it in the test tube.)

And, most notably, this thing is going to Phase IIb, and STILL LOOKS

PROMISING. If you go back and look at other cf studies, you'll see

that this doesn't happen very often. Usually, Phase II is where they

notice that the treatment just doesn't do anything and it doesn't

make it to Phase III. Just the idea that it has made it this far

without being shot down is noteworthy.

I know I may sound a little jaded, but after getting so excited about

aav gene therapy, gsh, dha, and so on and so forth, only to realize

that this wasn't the silver bullet, well, I guess I just try to be a

little guarded with my optimism now. Also, being from Ohio myself,

it is a little shocking that something better than Drew Carey might

emerge from Cleveland... (just kidding, of course we all know

Cleveland Rocks.)

-sorry to all those not in the U.S., this humor will probably be

lost on you.

~

> I keep scrolling the posts & haven't seen anything yet

> on the new postings about the progress of the gene

> therapy trial in Cleveland. It made our local news &

> several people mentioned it to us. Having only been

> involved in the CF " loop " for a few months, I'm not

> sure what to make of this. Is it promising? I'm

> hopeful that it is, but thinking that it may not be

> since nobody here has mentioned it.

>

> W.