Safety of retrovirus gene therapy, Temin, 
In addition, retrovirus therapy should only be used for human disease after it has 
been established to be safe and effective in animal model systems. With the problems of 
extrapolating from cell culture models to determine the efficiency of expression of 
retrovirus vectors, primates should be the final test subjects. 
The National Institutes of Health Recombinant DNA Working Group on Human 
Gene Therapy has prepared "Points to Consider in the Design and Submission of Human 
Somatic-Gene Therapy Protocols" which should ensure safety and effectiveness of 
somatic gene therapy of human disease with retrovirus vectors before human trials begin. 
B. Cost And Effectiveness 
These questions are no different than for any other new medical therapy. This 
kind of therapy will be very expensive because of the sophisticated laboratory monitor- 
ing. However, it probably will be cheaper than heart transplantations. My position as to 
cost is that a country spending close to $300 billion a year on the military can afford to 
introduce any therapy that has the potential to correct extremely serious, life-threatening 
conditions. 
Although primate and other animal studies will give some idea about the pos- 
sible effectiveness of this therapy, there is no way to determine the effectiveness of this 
therapy in correcting these genetic diseases without human experimentation. Given that 
the vector system is safe, as indicated above, and that the gene to be introduced is ex- 
pressed in primate models, human experimentation is justified to determine effectiveness. 
C. General Social Effects 
As with any powerful new technology, some people raise concerns that somatic 
gene therapy of human disease with retrovirus vectors will increase the possibility of the 
development of other new technologies that have undesirable consequences (Talbot, 
1986). Since these are hypothetical consequences, they can be treated on their own 
roogi Recombinant DNA Research, Volume 12 
