Recombinant DMA Advisory Committee- 9/12-13/94 
motion endorses the process proposed by the FDA. Dr. Wivel said there is no need for 
another Federal Register announcement in order to have a vote on this proposal. The 
results of the RAC action, when approved by the NIH Director, will be published in the 
Federal Register. 
Committee Vote 
The RAC approved a motion made by Dr. Miller and seconded by Dr. Zallen to accept 
the following: (1) the FDA proposal submitted by Dr. Noguchi; (2) adopt the Categories 
for Accelerated Review that were approved by the RAC at its March 3-4, 1994, meeting, as 
guidelines for proposals that will not require RAC review (until such criteria have been 
established by an ad hoc review committee proposed by Dr. Varmus); (3) FDA and the 
RAC will establish a subcommittee to examine the consolidated review process for human 
gene transfer protocols; and (4) accept the proposed amendments to the NIH Guidelines 
to reflect this revised consolidated review process (including acceptance of Appendix M 
and incorporation of necessary editorial changes). 
The motion was approved by a vote of 15 in favor, 0 opposed, and 1 abstention. 
Acceptance of the proposed amendments to the NIH Guidelines is contingent on review 
and approval of these amendments by NIH and FDA legal counsel, the NIH Director, and 
the FDA Commissioner. 
Ms. Meyers thanked Dr. Noguchi for his efforts in crafting the FDA proposal. Dr. 
Merchant (Viagene, Inc., San Diego, California) commented from his vantage point as a 
former NIH and FDA employee that the RAC vote is very pertinent for the rapidly 
evolving field of human gene therapy. By not allowing the RAC to evolve into a Study 
Section that involves itself constantly with "nuts and bolts," the RAC will be able to really 
concentrate on the novel applications of gene therapy. It was Dr. Merchant’s opinion that 
the more time that the RAC spends in a truly deliberative and advisory capacity and the 
less time with simple review issues, the more effective the RAC can be in helping the 
American public. 
XIV. ADDITION TO APPENDIX D OF THE NIH GUIDELINES REGARDING A HUMAN 
GENE TRANSFER PROTOCOL ENTITLED: RETROVIRAL-MEDIA TED TRANSFER 
OF THE IDURONATE-2-SULFATASE GENE INTO LYMPHOCYTES FOR 
TREATMENT OF MILD HUNTER SYNDROME ( MUCOPOLYSACCHARIDOSIS TYPE 
II)/ DR WHITLEY 
Review-Dr. Erickson 
Dr. Walters called on Dr. Erickson to present his primary review of the protocol 
submitted by Dr. Chester B. Whitley, University of Minnesota, Minneapolis, Minnesota. 
The overall purpose of this study is to evaluate the possibility of treating Hunter syndrome 
(mucopolysaccharidosis type II) , a severe heritable disease, by a form of gene therapy 
using a LXSN-class vector, L2SN. This vector is a retrovirus genetically-modified to carry 
the normal gene for human iduronate-2-sulfatase (IDS), which is lacking in patients with 
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