working days. Grade 4 (life-threatening) reactions and patient deaths while on treatment will 
be reported to the NCI by phone within 24 hours. A written report will follow within 10 
working days. 
Written reports will be sent to: 
Investigational Drug Branch 
Cancer Therapy Evaluation Program 
P.O.Box 30012 
Bethesda, MD 20824 
The end-point for part 1 will occur when the patient has received approximately 6 infusions of 
transduced, non-selected cells. A thorough immunologic evaluation will be performed at this 
time. All patients will progress to part 2.A. of the protocol. Part 1 will require approximately 
6 patient admissions to the Clinical Center and will last from 6-9 months. Part 1 may result 
in significant benefit to the patient from a measurable improvement in immune function or 
clinical status. We will also be able to evaluate the survival in the patient’s blood of the 
infused ADA gene-modified T cells and to determine whether autologous T cell infusions may 
have a selective growth advantage in vivo. 
7.2 Part 2; Infusion of Transduced Cells Selected for Expression of ADA. 
Part 2.A. of this study will involve approximately six monthly infusions of transduced 
lymphocytes selected for expression of the introduced genes. The procedure and possible 
complications of this part are identical to those in part 1. Again, cells will be infused as 
quickly as selection provides a transduced lymphocyte population with a normal blood level of 
ADA. Serial sampling of the patient’s blood to insert the ADA gene should permit genetic 
correction of a broad selection of the patient’s T cell repertoire since presumably different 
immune specificities will be present in different proportions at the different time points. 
In part 2.B., the dose of infused gene-transduced selected lymphocytes will be escalated at 
approximately monthly intervals in half log increments from 10® /kg to reach a target level of 
1-3 xlO^/kg. The patients will then receive approximately 6 monthly infusions of selected 
cells at this cell concentration. 
Recombinant DNA Research, Volume 14 
[231] 
