as outlined in Appendixes B and D. A backup marrow or peripheral 
blood stem cell collection would be available which is 
genetically unmodified. 
5.3 Treatment Plan: All patients should be treated in the Protected 
Environment if available. 
a. The preparative ablative regimen will consist of the 
following systemic chemotherapy: 
Cyclophosphamide: 1.7 g/m 2 qd on days 1-3; mesna, 6 mg/kg will be 
given q6h during cyclophosphamide infusion. 
Thiotepa at a dose of 225 mg/m 2 by continuous infusion over 4 
days will be diluted in normal saline. 
b. Subsequently modified autologous bone marrow will be 
reinfused on day 9 after the last dose of chemotherapy 
following an interval of 6 days so as to protect the marrow 
from metabolites of the chemotherapy. Premedication with 
benadryl 25 mg and solucortef 100 mg ivb 30 minutes before 
reinfusion to prevent anaphylactic reactions. 
c. Treatment of patients in the Protected Environment on the 
12th floor of the Lutheran Pavilion is strongly urged. [ 
Patients will remain there until the attainment of 500 
granulocytes/mm 3 . 
d. Patients will receive bactrim DS po BID and ketoconazole 200 
mg po q8h as antibiotic prophylaxis or other antibiotics 
specified by the Infectious Disease group while hospitalized 
to protect patients against overwhelming infection. 
e. All blood products will be irradiated from the start of 
treatment and for three months following the transplant. 
f. GCSF (5 mcg/kg) will be given SC qd starting on day 1 after 
marrow harvesting and until the ANC is >10 , 000/ fid. 
5.4 Maintenance Therapy: Taxol will be given every three to four 
weeks following recovery of hematopoietic function after bone 
marrow transplantation. The starting dose will be 13 5 mg/m 2 
CI/24 hours, and will be given to 3 patients if hematologic, 
neurologic or mucocutaneous toxicity is < 2. Dose will be 
escalated to 200 mg/m 2 in the next cohort of 3 patients. If any 
of the patients develop grade 3 toxicity, 3 additional patients 
will be treated at the same dose level. If 2/6 patients develop 
grade 3 toxicity, it will be considered M.T.D. If there is no 
grade 3 toxicity, the dose will be further escalated by 
increments of 50 mg in cohorts of 3 patients until M.T.D. If the 
kinetics of hematopoietic recovery is such that increases in the 
doses of Taxol can be made, (a recovery of 2 000 neutrophils/cm mm 
100,000 platelets/cm mm at day 21 after the previous course of 
therapy) , hematologic recovery will be defined as an absolute 
neutrophil count (ANC) >2, 000/mm 3 and a platelet count 
>100, 000/m 2 . 
[670] 
Recombinant DNA Research, Volume 17 
