Recombinant DNA Advisory Committee - 01/14/93 
Director. Dr. Royston said that the Points to Consider responses are in his office. 
Dr. Zallen noted that Dr. Royston has stated that if he had known it was possible to 
request RAC review of a single patient protocol, he would have made that request. Dr. 
Zallen asked Dr. Sobol if he believed that was the case. Dr. Sobol said that he did not 
feel that Dr. Royston's statement was true, given the time frame involved. Dr. Zallen 
reminded Dr. Sobol that he had obtained the patient's tissue in August of 1992. Dr. 
Sobol agreed that the patient's tissue was available, and it was their intention to request 
RAC review at that time. However, the patient's clinical condition deteriorated, and 
they were unaware that the RAC would review a single patient protocol. Dr. Sobol said 
that it is his understanding that the patient's clinical deterioration precluded submission 
of the protocol in time for consideration. 
Dr. Royston stated that a significant amount of time elapsed between the time that the 
patient's cells were transduced and gene expression was demonstrated. Approval would 
not have been possible from the RAC in November 1992 because pertinent data was not 
available. Adequate data was not available until December 1992, at which time the 
December 4, 1992, meeting had already passed. Dr. Sobol acknowledged that the earlier 
conclusion, namely, that the investigators could have pulled the relevant information 
together in time for the December 4, 1992, meeting, was not a correct statement. 
XIV. STATEMENT-DR. WILLIAMS 
Dr. Walters called on Dr. R. Michael Williams to present his statement to the RAC. Dr. 
R. Michael Williams introduced himself as the Chairman of the Cancer Consulting 
Group, Professor of Medicine at Northwestern University, and Chief Medical Officer of 
the Cancer Treatment Centers of America. Dr. Williams explained that he is a 
practicing oncologist with a Ph.D. in immunology. 
Dr. Williams stated that he is in support of Dr. Healy's decision to grant a 
compassionate permit for the use of gene therapy at the San Diego Regional Cancer 
Center. Dr. Williams said that he sees the devastation and the hopelessness that cancer 
brings to families every day. Dr. Williams stated that the RAC needs to be open to new 
ideas and respond to new therapies quickly. Every avenue and every possibility to offer 
hope and to save lives should be pursued. 
Previous speculations, which raised fears about biological disasters emanating from gene 
research, have simply not come to pass. Discussions can and should continue, but people 
die every day from cancers that might be treatable by gene therapy-based techniques. 
Cancer patients have the right to be empowered as part of the treatment team that 
decided which therapy is right for them. Compassionate use of a therapy is just that, 
compassion. 
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Recombinant DNA Research, Volume 17 
