Recombinant DNA Advisory Committee - 12/3-4/92 
manuscripts describing the adenosine deaminase (ADA) deficiency protocol was rejected 
from a prominent journal because the results had been reported to the public. 
Dr. Leventhal said that the goals of the protocol can be separated from clinical 
observations in terms of gene transfer. Investigators should be required to submit only 
data regarding the endpoints that relate to gene transfer; therapeutic benefits do not 
have to be reported in this format. 
Dr. Post emphasized that investigators should submit copies of published articles and 
abstracts that have been presented to the RAC. One of the reasons for this data 
reporting exercise is to assist the RAC with future protocol approvals, but the other 
reason is to identify categories of protocols that might qualify for a different level of 
review based on experience. He asked Dr. Leventhal if she has developed any 
suggestions for future directions. 
Dr. Leventhal said that a large number of patients have not been entered into the 
protocols; approximately 40 patients to date. Data indicates that patients who have 
received either the neo R gene and the LNL6 vector have not experienced any problems. 
Only 12 of these 40 patients have actually been shown to possess gene-marked cells. Dr. 
Post inquired if these 12 patients represented sufficient information with regard to safety 
and efficacy of gene transfer. Specifically, could the RAC recommend that neo R and 
LNL6 marking experiments no longer require RAC review? Would the Food and Drug 
Administration (FDA) and IRB approval be sufficient? Dr. Leventhal said that although 
she could not represent the entire RAC, she believed that these particular marker 
protocols could be approved solely by the FDA and the IRB if the vector was prepared 
by the same manufacturer in a standard way. If the RAC can be confident that the FDA 
will proceed carefully with the approval of new vector preparations, then the committee 
is certainly approaching the point where these markers will be considered safe. 
Drs. Leventhal and Post recommended that publications that were submitted by the 
investigators in response to the data reporting letter should be distributed to all of the 
members of the RAC. 
Dr. Murray suggested that the reporting format should include vector information. Dr. 
Secundy supported the development of a standard data reporting format. Dr. 
Dronamraju stated that investigators should submit positive data as well as negative data 
and include information regarding the socio-economic status of these patients. Dr. 
Leventhal explained that the RAC would have to wait for a longer period of time to 
receive positive results in the form of publications and abstracts. This delay would 
preclude obvious concerns regarding confidentiality. 
Recombinant DNA Research, Volume 16 
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