CFF Gathering at Nemours

[Guest guest]

The meeting we attended today was very informal and focused primarily

on fundraising. There were less than 20 people there but we enjoyed

meeting some new people including a lovely CF grandmother and the

husband of a CF'r. We toured the CF care center and I saw (and tried

out) my first vest. I look forward to Abby's being able to wear one.

The head Dr. (can't remember her name) of the care center spoke to us

and answered questions. Of course, like you all I always want to

know the answer to the billion dollar question. " How do you see a

cure or control being developed and how long do you anticipate it to

take? " I realize she hasn't a clue but I can't help asking. She

gave a fairly generic answer in regard to gene therapy - that they

were attempting to get around the barriers which inhibit the delivery

of the healthy gene - and that was one way she saw great potential.

Another was one I wasn't familiar with which involves using drug

therapy to target the specific malfunction in the CFTR - depending on

the mutation. If it is DeltaF508, which prevents the CFTR from

folding properly getting it hung up where it is destroyed by built in

defense mechanisms, the drug might make the body produce more of the

protein thereby increasing the chance that some would make it to the

cell wall and serve as a chloride channel. Other mutations with

different malfunctions in CFTR would be given different forms of the

drug which would counteract their specific problem. I think she

called it " phenyl buterates or buterol " or something that sounds like

that. I plan to check into it more. She didn't say how long before

it might be available

This is all my little interpretation of what I HEARD her say so don't

take a lot of stock in it. It sounded encouraging. If anyone knows

about this and I have erred in explaining it please correct me.

Joe (Dad of Kelsi, 7; Chloe, 2 1/2; and Abby, 15mos.w/cf)