Human Gene Therapy Subcommittee - 4/5/91 
forms at M.D. Anderson are taken very seriously. Prior to a session in which an 
informed consent document is discussed, the patient is shown a videotape that describes 
the therapy, the procedures, and the issues at stake for the patient. Also, the patient has 
met with a nurse who also has gone over the issues and has also met with the financial 
representatives of the institution. The patients at M.D. Anderson fall into two 
categories: (1) those who are Texas residents and are indigent; these patients have full 
access to the most advanced and expensive levels of care without bearing any financial 
responsibility; and (2) those patients who have insurance coverage. Before a patient 
proceeds with therapy, it is assured that all the financial issues have been resolved. Dr. 
Deisseroth stressed that the consent document presented to the subcommittee for review 
does not fully reflect the effort that goes into trying to educate the patient. He agreed 
with the committee's concerns about clarifying issues of each dimension of therapy, as 
well as the retroviral marking, and he will clarify the document. 
Dr. Deisseroth explained that the "sterile environment" mentioned in the consent form is 
a ward at M.D. Anderson equivalent to the transplant wards all over the country. It is a 
more contained environment in an effort to shield the patients from infection. It is 
always assured that a room in this ward is available for a patient who is undergoing 
either allogeneic bone marrow transplantation or an autologous program which has the 
potential for prolonged hematopoietic recovery in which the risks of infection for the 
patient are much greater. Whenever someone undertakes something new with 
hematopoietic reconstitution, such as adding a retrovirus marker, there is a potential for 
unexpected events. Therefore, those patients are put at the highest level of protection. 
Mr. Capron asked why the condition is included that the room will be supplied whenever 
available. Dr. Deisseroth explained there are instances in which the urgency of therapy 
would make the risk of waiting for a sterile environment greater than going ahead 
without it. Mr. Capron suggested they leave allusion to the sterile environment out of 
this consent form. Dr. Deisseroth felt it was a very good suggestion. 
Dr. Deisseroth described the choice of blast crisis as a model. He felt it was the clearest 
model, or disease setting, in which to clarify the issues of therapy in terms of autologous 
reconstitution, because the blast cells have a molecular marker which can be detected. 
This provides an opportunity not only to resolve the issue of whether relapse is arising 
from leukemia cells that contaminate the autologous marrow or from systemic disease, 
but also allows the exploration of other areas which pertain to normal hematopoiesis, to 
reconstitution, to isolation of the hematopoietic stem cell, all of which could be 
important in future gene therapy using hematopoietic reconstitution as a vehicle. 
Dr. Deisseroth explained that CML starts out as a very indolent disease which evolves 
into a life-threatening illness, a fulminant leukemic transformation. The proposed 
therapy involves converting the fulminant leukemic transformation to the indolent stage 
of the disease. The retroviral marker is used to identify the origin of the failure of that 
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Recombinant DNA Research, Volume 14 
