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 _L of Pipages. 
improving lung function. Aerosolized antiproteases (proteins which can 
protect the lung from being "chewed up" by enzymes released by inflammatory 
cells in the CF lung) such as alpha 1-antitrypsin and recombinant secretory 
leukoprotease inhibitor are also being evaluated. Initial studies show that 
they decrease inflammation in the lung. Amiloride, a medicine which alters 
the flow of salt and water in and out of cells, has also been administered 
directly to the lung. While the results of these therapies are encouraging, 
the results are all preliminary. These medicines are not approved for general 
use, and they do not correct the basic defect which causes CF (the gene 
abnormality) . 
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A more radical approach to CF therapy is lung transplantation. Through 1991, 
lung transplantation procedures have been carried out in 312 individuals with 
CF worldwide. Overall, there has been a 3 year survival rate of approximately 
52%, that is, approximately one-half of the individuals undergoing these 
procedure will be alive 3 years later. While this procedure effectively cures 
the lung abnormalities of CF, the patient is left with a number of significant 
problems. The transplanted or original lung may become reinfected with 
bacteria from the nasal sinuses. Also, the patient will have to receive 
immunosuppressive therapy (medicine to prevent the body reacting against the 
new lung) for the rest of their life because of a significant risk of 
transplant rejection. Perhaps more significant, is the fact that there is a 
very small pool of lungs available for transplantation. At any given time the 
usual waiting period for donors is 9 to 12 months. 
Overview of Protocol 
'1 
Because CF is a hereditary disorder with an identified genetic defect and 
because most of its clinical manifestations in the tracheobronchial tree (the 
branching structure that carries air from the voice box to the air sacs), it 
is reasonable to try to treat CF by delivering the normal gene to the cells 
lining the tracheobronchial tree. How can the normal gene be delivered to 
these cells? The delivery system in this protocol involves placing the normal 
CFTR gene into AdCFTR, a virus that has been modified in the laboratory, that 
can infect cells in the lung like a "cold" virus but cannot reproduce itself 
in human lung airway cells. In this fashion, AdCFTR will be used like a 
shuttle to "ferry" the normal gene to the lung cells, thus compensating for 
the lack of normal CFTR genes. AdCFTR will be administered to the respiratory 
tract of individuals with CF at two sites, the nose and the airways. 
Administration to the nose will be under direct vision through a nasal 
speculum (a small cone-like device that can easily be inserted into the nose). 
Administration to the airways will be through a bronchoscope (a flexible 
fiberoptic tube that can, with appropriate sedation and anesthesia, be placed 
via the nose or mouth into the lung; see below for a more detailed 
explanation) . AdCFTR will be placed in a small amount of fluid known as a 
vehicle. This vehicle is composed of a dilute mixture of salts and buffered 
PATIENT IDENTIFICATION 
CONTINUATION SHEET for either: 
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Recombinant DNA Research, Volume 16 
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