Stem Cell Gene Therapy for HIV/AIDS
Prof. Hans Peter Kiem
Fred Hutchinson Research Center
April 10, 2014
Foege N130A, Wallace H. Coulter Seminar Room
The toxic effects of chemotherapy (chemotoxicity) on blood and bone marrow cells of cancer patients can be a significant barrier to treating tumors. I will discuss how the delivery of a gene that can protect bone marrow stem and progenitor cells from chemotoxicity could overcome this barrier. Patients with chemotherapy-resistant brain tumors with very poor chances of survival were given a transplant with their own bone marrow hematopoietic stem cells after the cells had been modified with a gene that protects these cells from chemotherapy. After the bone marrow transplant, patients were then given dose-intensified chemotherapy. We found that the patients were able to tolerate these chemotherapy doses better after transplant of the gene-modified bone marrow stem cells than did patients in previous studies who had received the same type of chemotherapy but without the gene-modified bone marrow stem cell transplant. We found that chemotherapy increased the number of gene-modified blood and bone marrow cells in these patients. These patients survived longer than predicted without any negative side effects from the transplanted cells or the treatment given. This strategy could be used to treat other types of cancer or even other diseases such as genetic disorders or HIV/AIDS where the level of corrected or HIV-protected cells need to be increased to higher levels to produce a therapeutic benefit. I will then discuss the potential applications of this approach for HIV / AIDS and some genetic disorders.
Hans-Peter Kiem, MD, FACP, is the José Carreras / E. Donnall Thomas Endowed Chair for Cancer Research; Associate Head of Transplantation Biology and a Full Member in the Clinical Research Division at Fred Hutchinson Cancer Research Center; he is a Professor of Medicine and Adjunct Professor of Pathology at the University of Washington School of Medicine. Dr. Kiem has extensive experience in pre-clinical and clinical studies involving stem cell biology including induced pluripotent stem cells, hematopoietic stem cell transplantation, and gene therapy. Dr. Kiem’s research focus has been the study of stem cell biology and cell engineering with applications involving genetic and infectious diseases and cancer. Dr. Kiem also has extensive experience in training students and postdoctoral fellows and has had more than 25 trainees in his lab over the past 15 years. Many of his trainees now hold tenured faculty positions in the US and in Germany. Given his strong clinical interest, Dr. Kiem is also the sponsor of two clinical stem cell gene therapy studies and the PI of grant to study stem cell gene therapy in patients with AIDS lymphoma.