Attachment VI - Page 3 
5. In section l.B.L.b.(l), we suggest tlie addition of 
questions about: (1) the methods for assaying potenc y of 
the product used to treat cells or administered to a 
patient; and (2) the consistency of the product lot-by-lot, 
If different lots will be useef on the same patient or on 
different patients; and (3) the stability of the product 
under the projected conditions of storage. 
6. In section I.B.2.C., we suggest the addition of a section 
that would request a description of previously-reported 
similar human studies (Including foreign studies) and their 
results . 
7. Section I . B . 2 . c . ( 1) ( c ) Is relevant to gene therapy whether 
or not a retroviral system Is used, and should therefore be 
relocated just prior to I.B.2.c.(l). 
8. In section I . B . 2 . c . ( 1 ) ( d ) , we believe that the phrase 
"specifically germllnc cells" should be deleted because 
apprehension about an effect on gametes should be not 
greater than for other critical tissues including, for 
example, blood or bone cell precursors. A clearer, more 
appropriate way of posing the question would be, "What Is 
the specificity of transformation or other effects for the 
target cells?" 
9. In section I . B . 2 . c . ( 1) ( e) , the first sentence would be more 
clear If revised to read, "Describe animal experiments 
completed or in progress employing protocols similar to 
that proposed . " 
10. We believe that section I.B.3. Is the appropriate place for 
inclusion of "selection of subjects," rather than as a 
separate section I.C. 
11. In section I.B.3.e., we suggest that the first sentence of 
the section Is sufficient. The remainder requires of the 
Investigator extremely complex speculation and should be 
de le t ed . 
12. In section I.D., the Initial paragraph under "Informed 
consent" would provide sufficient guidance, without points 
1-5, If the following were added to the paragraph: 
"The consent form must adhere to the requirements of 
45 CFR 46 and 21 CFK 50. Special attention must be 
paid to the applicability of the additional elements 
of Informed consent listed In these regulation. 
(Include a copy of the patient consent form as part of 
the documentation requested In 1’art III, below.)" 
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