Human Gene Therapy Subcommittee - 4/S/91 
I. CALL TO ORDER: 
Dr. LeRoy Walters (Chair) called the meeting to order at 9:00 a.m., on April 5, 1991. 
He outlined the agenda for the meeting, noting that for the first time over 1,000 pages of 
material were circulated to members in advance of the meeting. He noted that he 
anticipated an additional agenda item in the form of a discussion of Mr. Capron's letter 
of November 26, 1990, if time permitted. Dr. Walters stated it would be his policy as 
Chair of the subcommittee to recognize speakers in the following order: primary and 
secondary reviewers, other members of the subcommittee, investigators on the proposed 
protocol, and then the public. He added that he felt it important to maintain the timing 
allotted on the agenda for each item in order to be fair to all investigators who had 
submitted protocols for review. 
Dr. Walters then called on Dr. Michael Blaese to present the first agenda item. 
II. PROGRESS REPORT ON ADA PROTOCOL ENTITLED: TREATMENT OF SEVERE 
COMBINED IMMUNODEFICIENCY DISEASE (SCID) DUE TO ADENOSINE 
DEAMINASE (ADA) DEFICIENCY WITH AUTOLOGOUS LYMPHOCYTES 
TRANSDUCED WITH A HUMAN ADA GENE: 
Dr. Blaese reported that the first patient started undergoing treatment on September 14, 
1990, and received her sixth infusion of gene-modified cells on April 4, 1991. A second 
patient began treatment on January 30, 1991, and has received two infusions of gene- 
modified cells to date. 
Dr. Blaese recounted the protocol in detail and discussed some of the goals of the 
protocol in light of the ongoing experiments. Due to the investigators' desire to ensure 
maximum safety to the patient, the studies had not progressed as rapidly as desirable. 
However, he was delighted with progress to date. This protocol demonstrated that it was 
possible to do routine apheresis from peripheral veins in children as young as 4 years of 
age with no difficulty, with no untoward effects in the children and with harvest of a 
suitable number of lymphocytes. 
It was possible to consistently derive T cell cultures from the immunodeficient children 
and introduce the ADA gene into those cultured lymphocytes. Further, the investigators 
were able to routinely infose the cultured lymphocytes into children, and the only side 
effect had been a brief episode of fever which abated after 4-5 hours with Tylenol 
therapy. One of the goals of the protocol had been to test the feasibility of using 
cryopreserved cells; however, this had not been attempted but would be done as the 
protocol evolved. 
Dr. Blaese presented data showing that it was not necessary to administer interleukin-2 
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Recombinant DNA Research, Volume 14 
