Human Gene Therapy Subcommittee - 11/30/90 
Dr. Zallen asked how the success of the transplant would be affected if a parent 
withdrew a child from the protocol before infusion of the marked cells, so that the 
child had the option to be reinfused with only 70% of the cells which were removed. 
Dr. Mirro said that enough cells would be harvested to allow for reconstitution with 
only 2/3 of the cells originally removed. If the parents declined the protocol, the 
child could still have an autologous transplant without transduction or an allogeneic 
transplant. There were a number of safety measures built into the protocol to 
ensure there would be no harm to the children in terms of their clinical outcomes. 
Dr. Parkman said that despite the fact that the data says there will be 60-70% long- 
term survival, there is nothing in the informed consent document to tell the patient 
that he/she will be expected to take part in long-term follow-up. Dr. Brenner 
replied that St. Jude has a post-chemotherapy clinic which follows all patients for 
life. 
Dr. Epstein amended his motion to allow for provisional approval based on 
presentation of these facts to the RAC. Dr. Erickson concurred. Dr. Zallen asked if 
an assent document was necessary since the protocol dealt with children. Dr. 
Parkman explained that the requirement for an assent document differs from state to 
state. 
Dr. Epstein asked for clarification about the current procedures at St. Jude's relative 
to assent forms. Dr. Mirro said the standard procedure does not include forms. 
However, the Bone Marrow Transplant Program has begun to use assent forms for 
children over seven years of age. There is no requirement in the State of Tennessee 
for an assent form; but in the case of this protocol, one would be developed. 
Ms. Meyers said more information on publicity and privacy should be included in the 
consent forms. Dr. Brenner said that information is in the summary statement, and 
he would include it in the informed consent document. 
Dr. Walters asked Dr. Mclvor to clarify all the stipulations being placed on the 
motion for provisional approval before taking a vote on the motion. 
Dr. Mclvor said that the motion to provisionally approve the protocol will have the 
following stipulations: 
1. That there be more information in the protocol and the consent form as 
to long-term follow-up; 
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Recombinant DNA Research, Volume 14 
