Recombinant DMA Advisory Committee - 5/30-31/91 
Dr. Walters asked whether it were possible for a patient to undergo the interleukin and TIL 
therapy but not take part in the gene marking study. Dr. Lotze said there was no requirement 
that all patients undergo all parts of the study in order to receive the therapy. He agreed to 
add a sentence to the informed consent form to make this clear. 
Dr. Gellert moved that the RAC grant conditional approval to this protocol pending receipt 
of data on the quantitative aspects of the PCR. Dr. Post seconded the motion. Dr. McGarrity 
called for further discussion on the protocol. 
Dr. Hirano noted that the protocol had been divided into four arms with 5 patients in each 
arm. She asked how many of these patients would actually receive the treatment. Dr. Lotze 
clarified that the plan is to have all patients treated. He said that the earlier version of the 
protocol specified four groups, but that this had been amended and the different approaches 
concatenated into a single protocol. 
There being no further discussion on the proposal, Dr. McGarrity called for a vote on Dr. 
Gellert's motion to conditionally approve the protocol pending receipt of the results of the 
PCR analysis. The motion passed unanimously by a vote of 18 in favor, none opposed and 
no abstentions. 
Dr. McGarrity then noted that Dr. Gellert had been out of the room when he presented 
certificates of service to other retiring members of the committee and he presented Dr. 
Gellert with his certificate and thanked him for his service. 
Noting that the meeting was running ahead of the contemplated schedule. Dr. McGarrity 
called on Dr. Anderson to present an update on the ADA gene therapy studies that had been 
approved by the RAC previously and which were now ongoing at NIH. 
VIII. UPDATE ON THE ONGOING ADA HUMAN GENE THERAPY TRIAL: 
Dr. Anderson said the first patient had started therapy on September 14, 1990 and had now 
had 6 infusions. A second patient had been started on January 31, 1991, and this patient has 
had 3 infusions to date. 
He said the first patient has continued to improve in every aspect of her existence and that 
approximately three weeks previous she had been challenged with a tetanus toxoid vaccination 
which took and was a very strong positive and she continues to do well in terms of not being 
ill. He said the investigators were concerned that the parents had begun to treat the child as 
if she were normal and that there still should be concerned since she still is at risk of lethal 
infections due to an incomplete immune system. 
Dr. McGarrity asked about her levels of isohemagglutinin. Dr. Anderson said that these are 
now normal. Dr. Mclvor asked what level of ADA was present in the blood stream of the 
patients and what fraction of cells are ADA-positive or positive for transduction with the virus. 
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Recombinant DNA Research, Volume 14 
