High-Dose Chemo Patients to Get Stem Cells With the MDR-1 Gene

Article

GAITHERSBURG, Md--Genetic Therapy, Inc. has begun phase I testing of a gene therapy protocol designed to protect patients' blood from the destructive effects of high-dose chemotherapy. The trial, headed by Kenneth H. Cowan, MD, PhD, of the NCI, will include approximately 18 metastatic breast cancer patients being treated at the NIH.

GAITHERSBURG, Md--Genetic Therapy, Inc. has begun phase I testingof a gene therapy protocol designed to protect patients' bloodfrom the destructive effects of high-dose chemotherapy. The trial,headed by Kenneth H. Cowan, MD, PhD, of the NCI, will includeapproximately 18 metastatic breast cancer patients being treatedat the NIH.

Patients who respond to chemotherapy and show no bone marrow involvementwill undergo both peripheral blood stem cell and bone marrow stemcell harvesting, 70% of which will be cryopreserved.

CD34+ cells, which include stem cells, will be separated fromthe remaining 30% and incubated with a retroviral vector containingthe human multiple drug resistance gene (MDR-1) in order to transferthe resistance trait to the cells. These cells along with thecryopreserved unmodified cells will be reintroduced followinghigh-dose chemotherapy.

It is hoped that the MDR-1 stem cells will contribute to the regenerationof the blood system and provide blood cells that have greaterresistance to subsequent chemotherapy.

Recent Videos
Nirav Shah, MD, MSHP, associate professor of medicine, at the Medical College of Wisconsin
Bhagirathbhai R. Dholaria, MD, an associate professor of medicine in malignant hematology & stem cell transplantation at Vanderbilt University Medical Center
Reena Sharma, MD, an adult metabolic consultant at Salford Royal Hospital
Mark Hamilton, MD, PhD, a hematology-oncology and bone marrow transplant (BMT) cell therapy fellow at Stanford University
Barry J Byrne, MD, PhD, the chief medical advisor of MDA and a physician-scientist at the University of Florida
Barry J Byrne, MD, PhD, the chief medical advisor of MDA and a physician-scientist at the University of Florida
Sarah Larson, MD, the medical director of the Immune Effector Cell Therapy Program in the Division of Hematology/Oncology at David Geffen School of Medicine at University of California, Los Angeles (UCLA)
David Porter, MD, the director of cell therapy and transplant at Penn Medicine
David Porter, MD, the director of cell therapy and transplant at Penn Medicine
Georg Schett, MD, vice president research and chair of internal medicine at the University of Erlangen – Nuremberg
Related Content
© 2025 MJH Life Sciences

All rights reserved.