Recombinant DNA Advisory Committee - 12/1-2/94 
response to RAC’s stipulations. The time required for investigators to adequately respond to these stipulations 
varies. The RAC is advisory to the NIH Director. The NIH Director retains the final authority to 
approve/disapprove any human gene transfer protocol. 
II-A CHAIR REPORT ON NIH-APPROVED ACCELERATED REVIEW PROTOCOLS/DR. WALTERS 
Dr. Walters explained that the following 3 Accelerated Review protocols were approved since the September 
12-13, 1994, RAC meeting: (1) Adenovirus Mediated Gene Transfer for Cystic Fibrosis: Safety of Single 
Administration in the Lung (lobar instillation) (Protocol #9409-091). This study will be conducted by Dr. 
Henry Dorkin, New England Medical Center, Tufts University, Boston, Massachusetts; and Dr. Allen Lapey, 
Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts. Drs. Straus and Ginsburg, 
and Mr. Capron reviewed and approved the lobar instillation arm of the protocol through the accelerated 
review process. However, the reviewers decided that the aerosol administration arm of the study required full 
RAC review. The reviewers’ recommendation was based on the fact that aerosol administration is an 
unprecedented route of vector delivery that may raise significant safety issues. (2) High Dose Chemotherapy 
and Autologous Bone Marrow plus Peripheral Blood Stem Cell Transplantation for Patients with Lymphoma or 
Metastatic Breast Cancer Use of Marker Genes to Investigate the Biology of Hematopoietic Reconstitution in 
Adults (Protocol #9411-092). This study will be conducted by Dr. Dan Douer, Kenneth Norris Jr. 
Comprehensive Cancer Center and Hospital, University of Southern California, Los Angeles, California. Drs. 
Haselkorn, Saha, and Secundy approved this protocol. Dr. Haselkorn commented that the investigators are 
serious and capable, and he noted that such an approval may not have been granted to less qualified 
investigators. (3) A Phase I Study of Vaccination with Autologous, Irradiated Melanoma Cells Engineered to 
Secrete Human Granulocyte-Macrophage Colony Stimulating Factor (Protocol #9411-093) will be conducted by 
Dr. Glen Dranoff, Dana Farber Cancer Institute, Harvard Medical School, Boston, Massachusetts. Drs. 
Secundy, Brinckerhoff, and Smith approved this protocol which closely parallels Dr. Jonathan Simons’ RAC- 
approved (Protocol #9303-040). Dr. Walters remarked that transition to Accelerated Review has been smooth 
and has led to a significant reduction in the number of protocols reviewed by the full RAC. This allows the 
RAC to focus on broader issues. 
II-B. CHAIR REPORT ON MINOR MODIFICATIONS OF NIH-APPROVED HUMAN GENE TRANSFER 
PROTOCOLS/DR. WALTERS 
Dr. Walters stated that 10 minor modifications were approved to RAC-approved human gene transfer 
protocols since the September 12-13, 1994, RAC meeting (see Attachment III for a complete listing of minor 
modifications). 
III. SEPTEMBER 12-13, 1994, RAC MINUTES 
The RAC approved a motion made by Dr. DeLeon and seconded by Dr. Chase to accept the September 12- 
13, 1994, RAC minutes (with the incorporation of minor editorial changes) by a vote of 18 in favor, 0 opposed, 
and no abstentions. 
IV. UPDATE ON NIH/FDA CONSOLIDATED REVIEW OF HUMAN GENE TRANSFER PROTOCOLS/DRS. 
WIVEL AND NOGUCHI 
Dr. Walters stated that on November 22, 1994, Dr. Alan Goldhammer, Director of Technical Affairs of the 
Biotechnology Industry Organization, Washington, D.C., submitted comments on the proposed NIH/FDA 
consolidated review process. Dr. Walters called on Drs. Wivel and Noguchi to present an update on this 
process. 
Dr. Wivel noted that the RAC recommended approval of proposed amendments to the NIH Guidelines at its 
September 12- 13, 1994, meeting. These amendments accommodated the NIH/FDA consolidated review 
process through the inclusion of minor editorial changes. Although the NIH Office of General Counsel 
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