12 
He thought it possible the working group will receive a proposal dealing 
with "erihancive scmatic-cell gene therapy, not just gene therapy that 
relates to a disease." 
Mr. Rifkin contended that human gene therapy is "not just an extension of 
normal medical technology. In many ways, it has similarities; but also as 
you knew, it's qualitatively different because we're new looking at changing 
genetic instructions in the code for human beings." 
Dr. Walters said different issues arise in considering unintended side 
effects of scmatic-cell human gene therapy than are raised by deliberate 
germ line interventions. The working group thinks it unlikely inadvertent 
germ line effects will occur in scmatic cell gene therapy since somatic-cell 
gene therapy is analogous to organ transplantation. However, the Working 
Group on Human Gene Therapy is asking investigators to look very carefully 
for unintended germ line effects in pre-clinical studies. Dr. Walters 
said even if unintended effects on the patients' germ line occurred, such 
effects would simply be one factor in the overall risk/benefit calculation 
for a therapy which would most likely be administered to children who would 
otherwise die in the near future. 
Dr. Walters said the first proposals would involve treatment of disease and 
not erihancive gene therapy. The points to consider document specifically 
states in Section I-A-l "why is the disease selected for treatment . . . . " 
Mr. Rifkin suggested the composition of the working group be reexamined and 
the expertise of this working group broadened to include theologians, anthro- 
pologists , and psychologists and a "wide range of people . " He also said 
the membership of the RAC should be expanded. He argued that human gene 
therapy is a social and cultural issue as well as a medical issue, and the 
precedents set in the next five years will influence developments in this 
area for the next 25 years. He said "it's not good enough just to have 
consultants . . . . " 
Dr. Walters said the Working Group on Human Gene Therapy is caught between 
opposing canps of critics on this issue; seme critics state the working 
group needs more technical members while other critics state the working 
group needs greater representation in ethics, laws, and the social sciences. 
The working group has tried to strike a balance; it consists of approx- 
imately four laboratory scientists, three clinicians, three ethicists, 
three lawyers , and two public policy experts . The current members of the 
working group are individuals who have published for the past fifteen years 
on the technical, ethical , legal, and social issues surrounding human gene 
therapy. Dr. Walters said as experience is accumulated, the working group 
may discover it requires expertise in other areas and could be augmented 
with additional members should the need become apparent . However, hew 
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