suppress your cough. You will be asked to gargle a local anesthetic (lidocaine) which will 
numb your mouth and throat. Next, lidocaine will be sprayed to further numb the back of 
your throat and your voice box. While lying down, a medicine (midazolam) will be given 
through the needle in your vein which will make you very sleepy. The bronchoscope, 
which is a flexible tube smaller in diameter than your little finger, will be placed through 
your nose or mouth and down your windpipe. Your windpipe is much larger than the 
bronchoscope so you will be able to breathe around it without difficulty. Lidocaine will be 
sprayed into your windpipe and airways through the bronchoscope to numb your airways 
and reduce your coughing. If during the bronchoscopy you become more awake and 
experience discomfort, more sedative (midazolam) will be given. To administer the virus 
containing the normal cystic fibrosis gene, the bronchoscope will be directed toward one 
segment of the lung. The airway will be cleared of secretions by suctioning through the 
bronchoscope. A small tube will be inserted through a channel in the bronchoscope and 
positioned in the desired airway. The virus will be instilled and the airway to that 
segment will be temporarily plugged by the bronchoscope for up to 30 minutes. 
Afterward, the bronchoscope will be removed. You will be asked to lie quietly for 30 
minutes. After another two hours you will be able to eat and drink normally. During the 
bronchoscopy, your heart rate and oxygen level in your blood will be measured 
continuously. Your blood pressure will be measured every several minutes. A chest xray 
will be taken afterward to check for any problems. 
3. Post-gene therapy monitoring. After the bronchoscopy, you will remain in the 
hospital for 10-14 days. During this time you will be monitored for any complications of 
the tests. This will be done by asking you repeatedly about any change in symptoms, by 
physical exams, by blood tests, by pulmonary function tests, by chest x-rays and by high 
resolution computed tomography of the chest. You will be tested to see if any virus can be 
found in your nose, sputum, blood, urine, or stools. After discharge from the hospital, you 
will be seen weekly in the outpatient clinic for 3 months. 
4. Follow-up bronchoscopies to test for the safety and effectiveness of the gene therapy. 
A repeat bronchoscopy will be done during the first week after gene therapy, and again 
after 6 weeks and 3 months. For these tests, you will be prepared for the bronchoscopy 
the same as above. The bronchoscope will be introduced into the airway as before. A small 
amount of fluid will be injected and sucked out of the airway where the virus was instilled. 
The same will be done in a similar airway in the other lung. A small brush on a wire will 
be placed through the bronchoscope and the wall of the airway will be gently brushed to 
remove a small number of the lining cells. Finally, a small pinchers on a wire will be 
placed into the bronchoscope and 6 small pieces of lung tissue (each the size of a grain of 
rice) will be removed from the lung. Because there are no pain nerves in the lung tissue, 
taking the pieces of lung will not hurt. 
5. Need for long term follow-up. Because gene therapy is a very new form of 
treatment, the long term effects are not known. To gain the most information and to 
provide the greatest safety, periodic evaluations will be needed. Your agreement to 
participate in the study means that you recognize the need for a prolonged relationship 
with the study and appreciate the need for continued evaluations. This will entail annual 
evaluations that include history, physical examination, chest xray, and routine blood tests. 
6. Consent for autopsy. To fully evaluate the effects and safety of gene therapy, it will 
be necessary to obtain as much information as possible. In the eventual occurrence of 
your death, evaluation of your lungs and other organs will be a very valuable method to see 
the full effects of gene therapy. Therefore your participation in this study means that you 
agree to an autopsy. This agreement must also be known by your next of kin so that person 
can follow with your wishes. 
Recombinant DNA Research, Volume 16 
[843] 
