June 1, 1990, Human Gene Therapy Subcommittee Minutes 
therapeutic goals which says that, "If this gene correction protocol does not prove to be 
superior to the PEG- ADA alone, the protocol will be retired." He wanted to know 
whether this is singular therapeutic goal. Dr. Neiman said he thought this point had 
been clarified and that both scientific and laboratory evaluations, as well as clinical 
evaluations, could be reasonable goals. The question of gene therapy being a superior 
treatment to PEG-ADA alone is only one question to be answered. Clearly the 
investigators hope that gene therapy plus PEG-ADA will yield an incremental 
improvement. 
Dr. R. Murray asked if more specific goals need to be addressed specifically by the 
investigators. Dr. Neiman said this request had already been made by Dr. Parkman in 
his comments. 
Dr. Leventhal voiced concern over the current consent form and assumed it would have 
to be revised with the current revisions in the protocol. Dr. Childress said the 
subcommittee was not responsible for approving the consent form but should be more 
interested in the particular things to be disclosed to participants. 
Dr. Walters asked Dr. Childress to amplify previous comments he had made relative to 
the incentive of the protocol in absorbing costs of PEG-ADA treatment. Dr. Childress 
said his concern centered around absence of an incentive for medical benefit by 
withdrawal from PEG-ADA and was worried about the financial coverage itself being an 
incentive. Dr. Walters said he thought it was a question of families, whose insurance 
coverage did not include PEG-ADA, having the incentive of not experiencing the out-of- 
pocket costs for this treatment by placing a child on the protocol. Dr. Childress said that 
was a concern as well. 
Ms. Areen raised two issues that Dr. Parkman did not restate in his last summation, 
those of the necessity for intraperitoneal infusion and the amount of the blood drawn 
from the patient. Dr. Parkman said he considered the protocol an "intravenous 
protocol." If intraperitoneal infusion was to be used, it would require significant 
reassessment. Since this has been proven to be a less efficacious route of administration. 
Dr. Mahoney asked if the investigators would follow Dr. Parkman's suggestion of 
whether in vivo selection of ADA competent genes was as effective as making the 
selection ex vivo, that is giving cells without expanding instead of going ahead with Part 
two, and whether they would attempt to work with this hypothesis. 
Dr. Mahoney also wanted clarification of exclusion criteria for patients with pulmonary 
or liver disease, or other chronic disease. 
Ms. Meyers said she didn't understand what Dr. Mulligan was referring to in the second 
paragraph of his letter in mentioning commercial concerns in the protocol and the 
involvement of a company called Genetic Therapy, Inc. She asked that these questions 
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