2 
the public eye; otherwise, it can be viewed as simply another 
tool to help individuals overcome an illness."^ We would urge 
a comparable statement be included in the introduction to the 
"Points to Consider" document. 
In addition, PMA suggests that, in the preface, the potential 
benefits of this therapy also be noted. Somatic-cell diagnosis 
and potential therapy for human single gene defects such as 
Lesch-Nyhan disease, muscular dystrophy, cystic fibrosis, etc., 
represent initial targets of a major new approach for treating 
genetic diseases. Far broader applications are considered pos- 
sible if present day early tests demonstrate the workability 
of this new exciting technique. The science is advancing very 
rapidly, and there appears to be a general religious and ethical 
acceptance of this approach for possibly ameliorating human 
single gene defects. 
The "Points to Consider" document provides necessary guidance as 
to what information is required on the objectives and rationale 
of proposed research involving the use of recombinant DNA tech- 
nology in humans. Much of the information requested is compa- 
rable to that requested by the FDA in its "Points to Consider" 
document for products produced by recombinant DNA (rDNA) tech- 
nology. 
On the subject of preclinical studies, the animal species in 
which the studies are performed should be selected on a compound- 
by-compound basis, and NIH should not specify that all studies be 
done in primates. One should not assume that primates are the 
only animals that will predict toxicity in man. The preclinical 
studies should be done in any species in which the drug shows 
pharmacological activity on the theory that, if one can define a 
pharmacologically active dose, one can also define a toxic dose. 
Therefore, one should not eliminate other animal studies a 
priori , for, depending on the gene therapy, a different 
animal model may be preferable. 
Concerning the social issues cited in the "Points to Consider" 
document, we agree that there must be as full communication as 
possible among investigators and clinicians for products or 
procedures developed in the proposed study; however, disclosure 
should comport with relevant patent laws or regulations governing 
patent rights and proprietary information. Again, a clear dis- 
tinction should be made between somatic-cell therapy and germ 
line therapy. The questions posed in the Social Issues section 
of the document, standing alone, could create confusion in this 
1 Human Gene Therapy - A Background Paper (Washington, DC: 
U.S. Congress, Office of Technology Assessment, 
OTA-BP-BA-32 , December 1 984) 
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