MEDICAL RECORD 
CONTINUATION SHEET for either: 
NIH 2514-1, Consent to Participate In A Clinical Research Study 
NIH 2514-2, Minor Patient’s Assent to Participate In A Clinical Research Study 
! STUDY NUMBER: 
CONTINUATION: page _JL of JLL pages. 
CFTR gene into the lung cells using a modified cold- like virus as a 
transporter. The safety, effectiveness, and practicality of this approach 
will be evaluated during the course of the protocol. 
The modified virus to be used is called AdCFTR. It is a modified adenovirus, 
that is, a laboratory-altered virus that can infect cells in the lung like a 
"cold" virus, but unlike a "cold" virus, AdCFTR cannot reproduce itself in 
human lung cells. AdCFTR has had inserted into it the normal human CFTR gene, 
which directs the production of the CFTR protein essential for normal function 
of the lung. The AdCFTR virus is capable of carrying the normal human CFTR 
gene into the lung cells of animals where it produces the normal human CFTR 
protein. Also, in the laboratory, AdCFTR can transfer the normal CF gene to 
lung cells recovered from individuals with CF, and these cells then produce 
the normal human CFTR protein. If AdCFTR can achieve this in the lung cells 
of live animals, and in cells recovered from the lungs of individuals with CF, 
it is reasonable to expect that this may also be achieved in the lung cells of 
individuals with CF when these cells are still in the lung. The hope is, that 
this may then compensate for the lack of a normal gene in the lung cells of 
these individuals. There is no guarantee that this will occur or that if it 
does it will make a difference in the lung function, general clinical status, 
or prognosis of individuals participating in the protocol. 
Overview of Cystic Fibrosis 
Cystic fibrosis is a common disorder affecting 1 in every 2000 births in 
Caucasians. It is acquired by receiving abnormal, instead of normal, CFTR 
genes from both parents. The median survival age of individuals with this 
disorder is 28 years. This means that 50% of people with cystic fibrosis will 
die before the age of 28. Lung disease is the major site of illness 
throughout the lives of most cystic fibrosis patients, and ninety percent of 
the deaths in cystic fibrosis result from lung disease. Current therapies to 
treat the respiratory manifestations of CF include frequent chest percussion 
and physiotherapy to mobilize (cough up) the thick mucus and antibiotic 
therapy to reduce infections. Applications of these therapies and rigorous 
attention to nutritional status (a good diet) have helped individuals with CF 
live longer. You are now receiving the best presently available therapy for 
your lung disease. 
New therapies for the respiratory manifestations of CF are being evaluated at 
the National Institutes of Health and elsewhere. These therapies include 
recombinant DNase, a human protein that is produced in the laboratory and can 
cut DNA, one of the major components of the infected mucus in CF. Preliminary 
studies have demonstrated that when administered as an aerosol (mist) to the 
lung DNAse improves the ability to cough out the infected mucus and hence 
PATIENT IDENTIFICATION 
CONTINUATION SHEET for either: 
Recombinant DNA Research, Volume 16 
[773] 
25-0099 
