ATTACHMENT C 
EXCEPTS FROM THE JULY 30, 1990, HUMAN GENE THERAPY 
SUBCOMMITTEE - ATTACHMENT C 
PROPOSED ADDITION TO APPENDIX D OF THE NIH GUIDELINES REGARDING 
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. Walters called on Dr. Parkman to present a review of the protocol. Dr. Parkman 
said provisional approval of this protocol had been granted at the last meeting with eight 
conditions to be met. Dr. Epstein asked that the conditions be restated so that the 
discussion could be focused. 
Dr. Wivel said the motion to approve this protocol was unanimously passed with the 
following eight provisos: 
1. That the consent form be revised, be reviewed and accepted by the RAC at 
its next meeting; 
2. That a stronger warning with regard to the potential for malignancy be 
inserted in the consent form; 
3. That a stop criterion of two therapy-related deaths be inserted; 
4. That intraperitoneal (i.p.) infusions not be utilized without further approval 
by this committee; 
5. That proceeding to Phase 2B of this protocol would require approval by 
the IBC and IRB; 
6. That full data from the Milan experiments and any related data be 
provided for review of a working group of this subcommittee prior to a 
meeting of the RAC and that a formal review of those data be brought to 
the RAC when this protocol comes up for approval; 
7. That a final version of the inclusion/exclusion criteria reflect the 
parameters that were addressed in the subcommittee discussion, including 
age, length of time on PEG- AD A; and, 
8. That there be a specific protocol for the follow-up evaluation of the 
immunological and clinical status. 
Recombinant DNA Research, Volume 14 
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