Recombinant DNA Advisory Committee - 09/14-15/92 
Mr. Capron moved that the revised motion for approval should be restricted to the two 
protocols which involve a fully autologous system. Dr. Parkman seconded the motion. 
Dr. Murray stated that the newly amended motion would be to approve the two 
autologous bone marrow transplantation protocols allowing the FDA to established the 
required vector testing standards. 
Dr. Parkman suggested a friendly amendment to this motion. The language should be 
changed to read "utilizing an FDA approved vector." Although the FDA must ultimately 
approve these vectors, the RAC should not defer its responsibility. Ms. Buc agreed and 
said that the RACs purpose is to set its own standards for what is appropriate and 
should not defer to FDA. Dr. Wivel reminded the committee members that the RAC 
review process is independent and parallel to the FDA review process. Mr. Barton 
accepted this change as a friendly amendment. Dr. Parkman stated that based on this 
clarification, he withdrew the friendly amendment to Mr. Barton's motion. 
Dr. Leventhal called the question. Dr. Murray stated that the motion now on the table 
is to approve the two autologous protocols as written with the expectation that the FDA 
will convey to the RAC its standards for approval. The motion to approve the two 
autologous protocols passed by a vote of 14 in favor, 2 opposed, and 2 abstentions 3 . 
Dr. Parkman asked if any patients have been enrolled in the standard protocol utilizing 
peripheral blood stem cells from identical twins. Dr. Schuening stated that the first 
patient is currently being treated. Dr. Leventhal asked why the autologous patients 
receive G-CSF for seven days and the normal donors in this twin protocol receive G-CSF 
for five days when peripheral blood collection begins on day four. Dr. Schuening said 
that he is not the PI on these clinical protocols and referred the RAC to the background 
section of the protocol for the rationale of the various procedures. The optimal time to 
harvest peripheral blood progenitor cells is between four and seven days. It is unclear at 
this time what the most optimal day is for harvest. 
Dr. Leventhal asked if there is any evidence that the gene marking process may slow 
down the repopulation process. Dr. Schuening explained that the only difference 
between this marking protocol and the ongoing clinical protocol is that the donors will 
undergo a fourth leukapheresis in order to obtain enough cells for the marking 
procedure. 
Dr. Walters asked how may patients worldwide have had their peripheral blood stem 
cells transferred to another patient. Dr. Parkman said that one recent report cited 25 
lymphoma patients. Dr. Leventhal added that there is extensive data regarding 
autologous stem cell transplant following chemotherapy. There is currently no data 
3 Dr. D. Miller abstained. 
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Recombinant DNA Research, Volume 16 
