Recombinant DNA Advisory Committee- 9/12-13/94 
I. CALL TO ORDER 
Dr. Walters (Chair) called the meeting to order and stated that notice of the meeting and 
proposed actions were published in the Federal Register on August 23, 1994 (59 FR 43426) 
as required by the NIH Guidelines for Research Involving Recombinant DNA Molecules 
(NIH Guidelines). He noted that a quorum was present and outlined the order in which 
speakers would be recognized. The primary and secondary reviewers will present their 
comments regarding the proposal, followed by responses from the principal investigators 
(Pis). The Chair will then recognize other committee members, ad hoc consultants, other 
NIH and Federal employees, the public who have submitted written statements prior to 
the meeting, followed by the public at large. 
Dr. Walters welcomed two new scientific members to the RAC: (1) Robert P. Erickson, 
M.D., Professor, Department of Pediatrics, Molecular and Cellular Biology, University of 
Arizona, Tucson, Arizona; and (2) R. Jude Samulski, Ph.D., Director, Gene Therapy 
Center, University of North Carolina, Chapel Hill, North Carolina. 
Overview 
Dr. Walters mentioned that the Office of Recombinant DNA Activities (ORDA) has 
moved to a new office space located at 6006 Executive Boulevard, Suite 323, MSC 7052, 
Bethesda, Maryland 20892-7052. The telephone and FAX numbers are unchanged, Phone 
(301) 496-9838/FAX (301) 496-9839. Dr. Harold Varmus, Director of NIH, is to address 
the RAC regarding RAC review criteria. It was noted that 8 protocols will be reviewed at 
this meeting, 2 on cystic fibrosis, 4 on cancer, 1 on mild Hunter syndrome, and another on 
peripheral artery disease. One protocol by Dr. Jack L. Gluckman has been withdrawn. 
There will be a discussion on the proposed actions to the NIH Guidelines regarding NIH 
and Food and Drug Administration (FDA) consolidated review of human gene transfer 
protocols. 
Dr. Walters stated that the RAC has approved 57 gene therapy protocols (47 approved by 
the NIH Director) and 24 gene marking protocols (22 approved by the NIH Director). 
Dr. Walters noted an article published by Dr. James Wilson and his colleagues on their 
study of familial hypercholesterolemia (Protocol #9110-012) in Nature Genetics (Vol. 6, 
pp. 335-341, 1994), with an accompanying commentary by David Weatherall in the same 
issue of the journal (pp. 325-326). There is correspondence in the same journal (Vol. 7, 
pp. 349-350) by Michael S. Brown, Joseph L. Goldstein, Richard J. Havel and David 
Steinberg questioning the interpretation of the results from the first patient study. These 
authors also proposed a set of criteria for evaluating success of gene therapy data on 
familial hypercholesteremia. 
Dr. Walters announced that the FDA will hold a public meeting to discuss gene vector 
production issues immediately following the first day of the RAC meeting. Notice of this 
meeting was published in the Federal Register on August 30, 1994 (59 FR 44739). 
Recombinant DNA Research, Volume 20 
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