Gerhard Bauer, an American researcher and expert on ‘mother’ stem cells, during the 50th congress of the American Society of Hematology (ASH) in San Francisco, announced that human testing for an AIDS treatment based on genetically modified stem cells could begin within 5 years. Bauer has been working for the past 10 years on modified stem cells that could repair strongly compromised immune systems in HIV patients. “For this reason the apparent success obtained by German researchers on an American patient with AIDS and leukemia reinforces the idea that we are on the right path.”
Last month the German researchers announced that their patient underwent a bone marrow transplant, introducing stem cells taken from a donor with a natural immunity to HIV, and after the operation he lived for 20 months with no sign of the AIDS virus. “The result does not surprise me,” added Bauer, the next director of the UC Davis Institute for Regenerative Cures in Sacramento while presenting preliminary results obtained from mice at the American Society of Hematology announcing that ‘the first tests on human beings could begin within five years.’
The animal model developed by Bauer has allowed him to experiment with a new gene therapy. “The German case is nothing other than gene therapy. We are working on something similar. We want to genetically modify stem cells to replicate, or imitate natural immunity to the AIDS virus. In this sense, what happened in Germany suggests to us that genetic engineering of stem cells could be the right path for curing diseases.”
Scientists coordinated by Bauer removed human skin cells from patients and made them regress developmentally giving them characteristics similar to stem cells. Cells obtained in this manner, called induced pluripotent stem cells (iPS) are able to differentiate into numerous other types of cells, including hematopoietic stem cells. Hematopoietic stem cells are bone marrow stem cells that develop into the cells of the immune system.
Bauer said, “If we are able to replace normal immune system cells with immune cells resistant to HIV, we will be able to cure AIDS.” His research project has allowed for the development of various anti-HIV genes which will be inserted into the induced pluripotent stem cells through gene therapy and viral vectors.
“The final goal is in the near future to use skin cells from HIV affected patients to obtain iPS cells engineered to also to avoid problems with rejection that can occur in transplants.”