Recombinant DNA Advisory Committee - 06/7-8/93 
breech of patient confidentiality in this case and was concerned that the current publicity 
will impact on patient enrollment in other gene therapy protocols. Dr. Walters explained 
that this request came on April 13, 1993, and that the expedited review procedures were 
not officially in effect at that time. Therefore, the revision was treated as a minor 
modification. 
Investigators' Responses-Drs. Blaese and Kohn 
Responding to Dr. Post's question, Dr. Blaese explained that the FDA has determined 
that the previous lot of LASN may be used only for patients on the existing protocol and 
will not be permitted for use in this amended protocol. Subsequently, a new production 
lot of LASN has been produced and will be available for use on the two newborns. 
Regarding Dr. Doi's question, Dr. Blaese said that fresh CD34( + ) cells will be used 
immediately without cryopreservation. In response to the confidentiality issue raised by 
Ms. Grossman, Dr. Blaese stated that patient confidentiality was maintained almost 3 
years for the initial ADA patients. Ultimately, the parents chose to publicize the 
encouraging results that are being observed in their children. In the case of these 
newborns, the parents voluntarily waived their confidentiality and encouraged press 
contact. Ms. Meyers expressed her concern on patients' families going public which 
could affect their ability to make proper informed consent. Drs. Miller and Leventhal 
agreed on the validity of Ms. Meyers' concern; however, this issue should not be the 
basis for disapproval of this minor modification. In regard to Dr. Walters' question 
about the time of diagnosis, Dr. Blaese said that these two families have previous 
children bom with ADA deficiency; therefore, the mothers were monitored for the 
defect. Following a positive diagnosis, the families expressed interest in pursuing stem 
cell gene therapy using cord blood and placenta. Regarding Ms. Meyers's question about 
Informed Consent documents, Dr. Blaese said that these documents have been prepared 
for each of the collaborating institutions. Ms. Meyers commented that all of these 
documents were not submitted for RAC review. Dr. Leventhal stated that if the 
additional documents are essentially the same as the one approved by the RAC, then 
they would be acceptable. 
Dr. Leventhal suggested that the withdrawal of PEG-ADA should be at the discretion of 
the Pis since they possess the expertise to make such a decision. She noted that bone 
marrow aspiration is a routine procedure for 6 month old children; therefore, the 
procedure is acceptable if properly described in the Informed Consent document. Dr. 
Kohn responded that the aspiration procedure is clearly stated in the Informed Consent 
document, and that the patients' parents have the right to refuse the procedure. Dr. 
Motulsky concurred with Dr. Leventhal's comments. 
Dr. Kohn stated that only fresh cord blood will be used for transduction and 
reconstitution of stem cells because the effects of cryopreservation are unknown. The 
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