Gaucher's Disease Clinical Trial
OBJECTIVES: I. Transfer the human glucocerebrosidase (GC) gene into peripheral blood stem
cells (PBSC) obtained from patients with type I Gaucher disease using a retroviral vector.
II. Transplant the autologous transduced PBSC in these patients. III. Measure the carriage
and expression of the transferred gene and its duration in peripheral blood leukocytes.
IV. Assess the clinical effects of transplanting genetically corrected PBSC.
PROTOCOL OUTLINE: Patients undergo autologous transplantation using peripheral blood stems
cells (PBSC) stimulated with filgrastim (G-CSF) and transduced with a retroviral vector
containing the human glucocerebrosidase gene (R-GC). Patients may receive up to 4
transplants if a deficient glucocerebrosidase level is found in peripheral leukocytes 1
month following transplantation.
The G-CSF-stimulated PBSC are enriched for CD34-positive stem cells and transduced with the
R-GC vector. Stem cells with normal gene activity are selected for transplantation.
Patients are followed every month for 6 months, every 6 months for 18 months, and then
annually thereafter.
;
Primary Purpose: Treatment
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