Recombinant DMA Advisory Committee - 
which he would take up with ORDA staff. He asked for other comments on the 
minutes. 
There being none he asked Dr. Post for a motion. Dr. Post moved that the minutes of 
the October 16, 1990, meeting of the RAC be accepted with staff making a review on the 
language in the paragraph describing the motion about Appendix K. Dr. Krogstad 
seconded the motion. There being no further discussion on the motion, the Chair put it 
to a vote and it passed unanimously by a vote of 15 in favor, 0 opposed, and no 
abstentions. 
Dr. McGarrity noted the presence of the Chair of the Human Gene Therapy 
Subcommittee (HGTS), Dr. LeRoy Walters. Dr. McGarrity thanked Dr. Walters for his 
past efforts and welcomed him to the committee meeting. 
Dr. McGarrity said that one of the contingencies of approval of human gene therapy 
protocols to this point is that investigators return to the RAC and give timely reports on 
their work to date, including any minor changes, modifications, or problems that the 
investigators may have witnessed. If major changes were envisioned, the investigators 
must return to the HGTS and the RAC for approval, but that minor modifications could 
be approved by Chairs of the HGTS and RAC in consultation with committee or 
subcommittee members without a formal vote of the RAC. Dr. McGarrity then called 
on Dr. W. French Anderson to present an interim report on the ongoing human gene 
therapy protocol on treatment of severe combined immunodeficiency syndrome (SCID) 
with the human adenosine deaminase (ADA) gene. 
INTERIM REPORT ON THE HUMAN GENE THERAPY 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. Anderson began by noting his close working relationship with Drs. Rosenberg, 
Blaese, and Culver in the one human gene transfer protocol and the two human gene 
therapy protocols that have been approved to date, the N2/TIL protocol, the ADA 
protocol, and the TNFjjl protocol. Any future protocols would continue to be a group 
effort. Therefore, it is appropriate that the Principal Investigator (PI) on each of the 
protocols present an update for the committee. 
Dr. R. Michael Blaese noted that there were some new committee members present and 
he reviewed a bit of the rationale for the ADA protocol. The protocol had two major 
components: (1) an immune enhancement by giving children infusions of autologous T 
cells; and (2) genetic correction with the retroviral-mediated gene transfer of the ADA 
gene into the autologous T cells that are reinfused into the children. 
Recombinant DNA Research, Volume 14 
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