Gene therapy in combination with blood stem cell transplants could hold the key to curing HIV

2011 Annual Report

Gene therapy in combination with blood stem cell transplants could hold the key to curing HIV

Dr. Hans-Peter Kiem

Dr. Hans-Peter Kiem

Hutchinson Center researchers broke new ground in the treatment of HIV this year by exploring the potential of blood stem cell transplantation to cure those infected with the AIDS-causing virus.

Drs. Hans-Peter Kiem and Keith Jerome were selected by the National Institutes of Health to lead a multifaceted team of scientists and institutions to determine whether a person’s own stem cells can be engineered to deny HIV entry into the body’s blood cells. They also will work to develop tools to eradicate existing reservoirs of infection in the body.

“Funding for research to find a cure for HIV-infected persons represents a paradigm shift,” said Jerome, an expert in viral infections at the Hutchinson Center and the University of Washington. “HIV has been an incurable, lifelong infection that at best sentences people to a lifetime of complex drug therapies. Now the research field is shifting to address the possibility of a cure. No one would have talked about this approach five years ago.”

One approach under investigation is stem cell transplantation, in which the infected patient’s own immune cells are genetically modified to be resistant to HIV by eliminating one of the receptors HIV needs to infect new cells. Led by Kiem, this method builds on the Hutchinson Center’s long-standing expertise in using transplantation to treat and cure blood cancers and some autoimmune diseases.

Stem cell transplantation to eliminate HIV infection has an intriguing precursor. In 2008, a group of German physicians published results of transplanting an American man who had leukemia and HIV. The so-called “Berlin Patient” received a new immune system from donor cells that also carried a rare genetic variation that made them resistant to the virus. Today, the patient is considered cured of HIV. However, few people have this genetic variation, so a way must be found to modify the patient’s own immune cells to resist HIV.