11 
Dr. Anderson said the proposed subsection on beta- lactam antibodies is not 
relevant to human gene therapy procedures since the first clinical protocols 
will probably be based on procedures using modified bone marrow cells. These 
cells will be removed fran the patient, modified, vvashed, and returned to the 
patient. Antibiotics which could cause an allergic reaction will not be present 
in the materials administered to the patient. 
Dr. Gottesman said Dr. Miller's absolute statement prohibiting the use of 
beta- lactam antibiotics does not make sense since it does not specify vhen 
these antibiotics may not be used. This prohibition could be interpreted as 
applying to steps in the preparation of the retroviral vector in which an 
antibiotic might be necessary. 
Dr. Walters said Dr. Miller's fifth suggestion was to add to Section I-B-l-b-(l ) 
questions on the methods for assaying the potency of the product, the consistency 
of the product lot-by-lot, and the stability of the product under conditions of 
storage . 
Dr. Murray said this proposed language had also been considered at the April 
1985 meeting; at this time, it makes no sense to assume the initial clinical 
trials will involve commercial production. The initial gene therapy protocols 
will be administered on a patient-by-patient basis. 
Dr. Anderson said Dr. Miller's proposed language applies in FDA reviews of 
drugs and biologies; however, the Working Group on Human Gene Therapy is not 
attempting to perform an FDA review. 
Dr. Gartland asked whether the working group would review a proposal differently 
if FDA does not review the proposal . FDA statutes do not apply in all cases; 
for example FDA statutes do not apply to protocols vhich do not involve inter- 
state commerce. 
Dr. Gottesman said she viewed Dr. Miller's intimation that FDA might not review 
protocols as a threat the working group might be required to perform an FDA 
review. Dr. Gottesman said an FDA style review is not the working group's 
mandate or interest. 
Drs. Anderson and Gottesman then explained that the words "potency," "consistency," 
and "stability" are appropriate language for drugs and biologicals but are not 
the correct terms to apply to human gene therapy. Genes do not have "potency," 
Rather the analogous term for a gene is "level of expression." The analogous 
information request for "stability" in gene therapy protocols is a request for 
information on the cell line vhich will be producing the retroviral vector. 
Cell lines producing retroviral vectors are stored in liquid nitrogen and are 
stable indefinitely under these conditions. "Expression in time" is the 
appropriate analogous term for "stability" of a gene. The language pertinent 
to genes is found in detail in the working group document. 
Dr. Anderson said patients' bene marrow cells, vhich are critical to the proce- 
dure, will not be stored. He compared bone marrow transplants in human gene 
therapy to other organ transplantation. He said it makes no more sense to 
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