12/07/87 
Dr. Gottesman said she was uncertain whether the subcommittee needed to ask people again; 
the subcommittee was considering the worst case situation. 
Dr. O’Reilly said that the bone marrow transplants were first tried on diseases fatal in the first 
year. Over the last twenty years, bone marrow transplants had progressively been prescribed 
for different diseases. 
Dr. Walters proposed that a statement should be drafted. Dr. Anderson stated that the Preclinical 
Data Document (PDD) does not have to be repeated. Ms. Areen said that the ground rules need 
not be changed. 
Dr. Parkman turned to the revision of the Points To Consider . Ms. Witherby stated that the Points 
to Consider will be reviewed on a yearly basis. Mr. Capron suggested that nothing formal should 
be done. 
Dr. Walters suggested that closure be sought on the safety, efficacy, and alternative therapies 
questions. Dr. Anderson argued that closure is not possible, and that he will submit a limited 
clinical protocol. Dr. Epstein stated that the minutes of the meeting can reflect this in the 
summary. 
Dr. Parkman commented that the Points to Consider can be answered differently. Dr. Erickson 
cited the Hirschfield letter, middle paragraph, p.3. The subcommittee then adjourned for lunch. 
Following lunch, Mr. Capron spoke of the danger of exploitation associated with "last hope 
cases." First, premature submission could occur, that is, when the likelihood of benefit is very 
small. Second, the potential for harm remains great, even if the benefits to others should appear. 
Dr. Anderson asked what the view of the Food and Drug Administration (FDA) regarding a small 
test would be, should the NIH subcommittee approve one. Dr. Miller answered that the FDA 
makes no distinction between large clinical trials and small tests. He would later provide FDA 
comments to the subcommittee; FDA had no fundamental objections. 
Information Document 
Ms. Witherby said she and Dr. Walters would continue to edit the information document. She 
asked members of the subcommittee for comments, particularly regarding the scientific portions. 
Dr. Walters requested that the comments be received by December 21, 1987. Dr. Parkman 
commented that the document is quite good. 
Ms. Witherby requested suggestions for where to distribute copies of the completed document. 
Issues for Future Meetings 
Dr. Cook-Deegan stated that Dr. Mulligan will report on "Gene Transfer and Gene Therapy" at the 
UCLA meeting. A short presentation on vaccine development and approval would be useful. 
Dr. Parkman requested discussion of tissue specificity and receptor sites. 
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