Recombinant DNA Advisory Committee - 12/2-3/93 
RAC to date (See Attachment IV for complete data management report): 
Therapy 
Marking 
Total CT + M) 
RAC Approved 
35 
23 
58 
NIH Director Approved 
28 
22 
50 
Categories of RACApprovcd Protocols 
, ,, 
Cancer 
26 
4 
30 
Cystic Fibrosis 
4 
1 
5 
SOD/ADA 
1 
0 
1 
Acute Hepatic Failure 
0 
1 
1 
Familial Hypercholesterolemia 
1 
0 
1 
Gaucher Disease 
2 
0 
2 
Bone Marrow Marking/ Cancer 
0 
15 
15 
HIV(+) 
2 
2 
4 
The next review of human gene transfer data will be presented at the June 9-10, 1994, 
RAC meeting. Dr. Leventhal noted several issues that should be addressed by the 
working group prior to the June 1994 data reporting period. Pis should not be required 
to submit semi-annual data reports as a follow-up to protocols that have been closed for 
> 1 year. She stated concern about the issue of under- and over-accrual of patients into 
NIH-approved protocols. Another issue is the lack of reporting unexpected toxicity and 
adverse reactions. Any such observation should be reported to the RAC, particularly in 
light of recent severe adverse reactions encountered in the fialuridine, or FIAU, hepatitis 
drug trial at NIH. Dr. Janet Woodcock of the FDA stated that the FDA requires that 
any unexpected adverse reaction must be reported immediately. All events, expected or 
unexpected, are reported annually and at the close of each study. Dr. Leventhal 
suggested that Pis submit copies of FDA adverse reaction reports to the RAC. 
Discussion ensued regarding recent press reports and public expectations about human 
gene therapy and the current state of art of this new biotechnology. The majority of the 
human gene transfer trials that have been approved are Phase I/II studies; therefore, the 
trials are not designed to assess efficacy. These Phase I/II studies should be evaluated 
on the basis of whether proposed scientific objectives have been obtained. 
V. UPDATE ON THE OLDFIELD PROTOCOL ENTITLED: GENE THERAPY FOR THE 
TREATMENT OF BRAIN TUMORS USING INTRA-TUMORAL TRANSDUCTION 
WITH THE THYMIDINE KINASE GENE AND INTRAVENOUS GANCICLOVIR 
(#9206-0 19)/DR. RAM 
Dr. Walters called on Dr. Zvi Ram of the NIH, Bethesda, Maryland, to provide an 
update on this human gene transfer protocol. Dr. Ram stated that as previously reported 
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Recombinant DNA Research, Volume 18 
