Recombinant DMA Advisory Committee - 6/9-10/94 
the IRB of any changes in the protocol required by the RAC. Dr. Post commented that 
this protocol would have qualified for the Accelerated Review process. 
Committee Motion 
The RAC approved a motion made by Dr. Miller and seconded by Ms. Buc to accept the 
protocol submitted by Drs. Helen Heslop, Malcolm Brenner, and Robert Krance of St. 
Jude Childrens Research Hospital, Memphis, Tennessee, by a vote of 14 in favor, 0 
opposed, and no abstentions. 
VIII. WORKING GROUP ON DATA MANAGEMENT-SEMI-ANNUAL DATA 
MANAGEMENT REPORT/DR. SMITH 
Dr. Smith summarized the semi-annual data reports submitted by the Pis of NIH- 
approved human gene transfer protocols. A total of 219 patients have been entered on 
MH-approved human gene transfer studies (68 subjects since the last reporting period). 
Of the 72 protocols recommended for approval by the RAC, 63 have been approved by 
the NIH Director, 39 are considered active, 10 are considered closed, 21 are pending 
FDA approval, and 9 are pending NIH Director approval (due to failure of the 
investigators to meet the RACs stipulation requirements for approval). 
A new column has been added to the Semi-Aimual Data Management Report entitled, 
"Biological Efficacy." This column is applicable only to gene therapy protocols. The 
term "published" is entered in this column for protocols in which demonstration of 
biological efficacy has been published in a peer-reviewed journal. 
The "In Vivo Evidence of Gene Transfer" column has been modified to include 
additional information regarding gene expression. For the gene marking studies, 
information will be given to indicate detection of marked cells in the recipients. For 
gene therapy protocols, information will be collected regarding detection of the 
transgene in recipients' cells and expression of the gene product. Such information is 
irrelevant for vaccine protocols. 
Dr. Smith made the following recommendations regarding future semi-annual data 
reports: (1) demonstration of the virus or gene product in a site other than the target 
should be reported as an "adverse effect", (2) investigators should indicate the number of 
subjects who have undergone autopsy and provide relevant data, and (3) a letter should 
be sent to investigators who have not enrolled any subject onto the protocol for several 
years inquiring whether the study should be considered "closed." 
Dr. Chase asked about the compliance rate for data reporting. Ms. Debra Wilson of the 
NIH Office of Recombinant DNA Activities (ORDA) said that the response rate is over 
90%. Dr. Ross asked if information regarding the number of eligible patients identified 
versus the number of subjects entered. Dr. Smith said that with one exception (Dr. 
Deisseroth's study) such information is not requested or available. 
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