Human Gene Therapy Subcommittee - 11/30/90 
I. CALL TO ORDER 
Dr. LeRoy Walters (Chair) called the meeting to order at 9:00 a.m., on November 
30, 1990. He noted that some additional materials had been provided in table 
folders. He called on Dr. Wivel to update the subcommittee on actions taken 
regarding the TNE^il protocol and the ADA protocol which were approved 
previously. 
II. INTERIM REPORTS ON THE HUMAN GENE THERAPY PROTOCOLS 
ENTITLED TREATMENT OF SEVERE COMBINED IMMUNODEFICIENCY 
SYNDROME (SCID) DUE TO ADENOSINE DEAMINASE (ADA) DEFICIENCY 
WITH AUTOLOGOUS LYMPHOCYTES TRANSDUCED WITH A HUMAN ADA 
GENE AND GENE THERAPY OF PATIENTS WITH ADVANCED CANCER 
USING TUMOR INFILTRATING LYMPHOCYTES TRANSDUCED WITH THE 
GENE CODING FOR TUMOR NECROSIS FACTOR 
Dr. Wivel asked Dr. Anderson, one of the investigators on the ADA protocols, to 
give the first update on the protocol entitled Treatment of Severe Combined 
Immunodeficiency Syndrome (SCID) Due to Adenosine Deaminase (ADA) Deficiency 
with Autologous Lymphocytes Transduced with a Human ADA Gene. 
Dr. Anderson reported that a four year old girl was the only patient treated to date 
in this protocol, and that she had undergone three courses of treatment. There were 
indications that she was doing well but cautioned that more data were necessary 
before making any final reports. A formal report with laboratory data is expected to 
be presented at the next meeting of the Human Gene Therapy Subcommittee 
(HGTS). 
Dr. Anderson noted that the Food and Drug Administration (FDA) had given final 
approval for the protocol. Thp first treatment had taken place within five hours of 
the approval. The dose had b|een escalated with each treatment, and the third 
treatment was 1.2 X 10^® cells. The only side effects noted at this level were slight 
flushing of the cheeks and a 1/2 degree temperature rise. Both symptoms 
disappeared within a half an hour following treatment. The fourth escalation would 
be to 1.5 X 10^° cells. The expected maximum dose of 3 X 10^° cells would be 
reached on the sixth escalation. The investigators will continue to monitor side 
effects. 
Dr. Wivel reported that the FDA had requested specific changes to the TNF^jl 
protocol entitled Gene Therapy of Patients with Advanced Cancer using Tumor 
Infiltrating Lymphocytes (TIL) Transduced with the Gene Coding for Tumor Necrosis 
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Recombinant DNA Research, Volume 14 
