5.3 Treatment Procedures 
GTI 0101 (04/08/93) 
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(Refer to section 6.0 for dose modification and toxicity management) 
5.31 Harvest of Hematopoietic Stem Cells : 
5.311 Peripheral Blood Stem Cell Harvest : 
Peripheral blood stem cells will be harvested after mobilization with the growth 
factor G-CSF. 
Study Days P1-P7 : G-CSF will be given at a dose of 10 /xg/kg/day 
subcutaneously for a period of 7 consecutive days. The drug will be given as a 
single daily injection and the patient may receive acetaminophen or other non- 
aspirin analgesics for any possible associated fever or bone pain. 
Study Davs P5-P7 : On days 5, 6, and 7 the patient will undergo apheresis 
procedures for the collection of peripheral blood mononuclear cell fractions with 
an expected yield of 1 X 10 8 cells per kilogram body weight. 
5.312 Bone Marrow Stem Cell Harvest : 
Study Day Ml : Bone marrow harvest from the iliac crest region will be 
performed according to standard bone marrow transplant procedures. Appropriate 
anesthesia will be used. A marrow harvest will consist of approximately one liter 
in volume and > 10 8 nucleated cells / Kg. Harvested marrow will be filtered 
through the collection kit filters to remove large particulate matter including bony 
fragments. Further processing of the marrow will be done to enrich for nucleated 
cells. 
5.32 Separation of CD34+ Population of Hematopoietic Cells : 
All the cells resulting from the marrow harvest or the peripheral blood stem cell 
harvest will be processed to remove contaminating red cells. The enrichment for 
CD34+ cells will be performed using the CellPro Stem Cell Separator. This 
procedure involves the incubation of the cells with a biotinylated murine anti- 
human CD34 antibody 12.8, washing the cells, and passing them over a avidin 
Biogel column. The cells adhering to the column will be collected after 
mechanical dislodgement. After enrichment a sample of CD34+ cells will be 
saved for further analysis and to use as pre-gene therapy controls for colony- 
forming and other assays. 
Recombinant DNA Research, Volume 17 
[809] 
