Human Gene Therapy Subcommittee - 11/30/90 
Ms. Meyers noted that when parents sign a consent form for bone marrow 
transplantation, they expect direct benefit to their child; this protocol offers no 
benefits. Mr. Capron reminded the HGTS that in cases where a patient is seeking 
therapeutic relief, informed consent is given less freely than in cases where the 
patient is actually hoping to help solve disease problems for future patients. If the 
actual number of patients seeking redress is small, then a small addition to the 
insurance funds could solve the problem. However, if many people are seeking 
redress for minor injuries who would now claim larger injuries if there were funds to 
be recouped, then additional funds would not be the solution. The Department of 
Health and Human Services (DHHS) had been requested to fund such a study, but 
this social experiment has never been conducted. 
Dr. McCarthy said that the Ethics Advisory Board to the Secretary has studied the 
question and recommended that some form of Government compensation be 
provided. DHHS attempted to devise a system, but was unable to gain approval for 
instituting it from senior officials of the Department. This recommendation has 
been revived in a draft legislative proposal within DHHS. Given the current fiscal 
climate in the country, it is unlikely to pass if forwarded to the Congress. Renewed 
pressure is being placed on the Congress because of issues such as compensation as 
a result of research on acquired immune deficiency syndrome (AIDS) and human 
immunodeficiency virus (HIV) infection. 
However, Dr. McCarthy echoed Mr. Capron's comments as to the variety of 
institutional policies on this issue. Most injuries are short-term and minor 
institutions have not found this issue to be a major problem. In the case of human 
gene therapy, such injuries could be more long-term and expensive. This proposal 
addresses these issues in a manner consistent with non-human gene therapy research 
protocols. The document could be strengthened by explaining who to notify and 
where to get information on financial as well as medical matters if such occurrences 
take place. 
Dr. Parkman said that in terms of this protocol, the most untoward event that could 
occur would be insertional mutagenesis which would result in Jeukemia. It would be 
difficult to prove whether this recurrence was caused by the gene therapy or whether 
it was recurrence of the original disease. 
Dr. Mahoney said that he had limited knowledge of cancer biology but noted that it 
appeared that most cancer therapies were "tumorigenic." When chemotherapy or 
radiation therapy are used, the cells of the body are exposed to potentially 
carcinogenic effects. These multiple interactions greatly exceed insertional events 
which could occur when treating a small number of cells outside the body. 
Recombinant DNA Research, Volume 14 
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