Curing HIV is the goal. New stem cell clinical trial could get us there

March 5, 2015 | by Nicole White

Curing HIV, not simply controlling its replication, is the ultimate goal of HIV researchers. A new clinical trial at City of Hope could put that goal within reach. The trial will test an innovative new therapy that modifies the stem cells of patients with HIV to make them resistant to infection with the virus. Here's how it works:
HIV A novel treatment for HIV will be offered via clinical trial at City of Hope. The therapy edits a patient's stem cells to prevent production of a protein the virus needs to infect healthy cells.
The AIDS virus relies on a protein called CCR5 to penetrate and infect cells. For the trial, researchers will use a zinc finger nuclease, or ZFN, to modify blood stem cells from patients infected with HIV. The ZFN acts as a pair of molecular scissors that cuts the CCR5 gene out of the cells. Without the gene, there is no CCR protein – and researchers hope that when these cells are infused back into the patient, their immune system will be resistant to HIV. “It’s erasing the gene, and once it’s erased, it never can reappear,” said John Zaia, M.D., in an interview with BuzzFeed News. Zaia is the Aaron D. Miller and Edith Miller Chair in Gene Therapy at City of Hope, chair of the Department of Virology and principal investigator of the trial.He and other City of Hope researchers will work with the University of Southern California and Sangamo Biosciences, the biopharmaceutical company that developed the ZFN. The study is funded by the California Institute for Regenerative Medicine (CIRM) and was recently approved by the Food and Drug Administration. “While we have a number of drugs that are effective in holding HIV at bay, we have nothing that cures it,” Zaia said. “In addition, for many patients, these medications come with significant long-term problems so there is a real need for a therapy that can help eradicate the virus from a patient completely. That is where our work is focused.” CIRM awarded $5.6 million to the trial as part of its Strategic Partnership Award to fund the trial. The amount will be matched by Sangamo. “There are more than 1 million Americans living with HIV/AIDS today,” said Jonathan Thomas, Ph.D., J.D., chair of the CIRM governing board. “This kind of work may be a first step towards finding a cure for them. And while the research is being done here in California, the potential impact is clearly a global one. That’s why we have been supporting this project for the past five years, through early testing to this point where we can now see if it works in people.” The clinical trial will include people with HIV/AIDS who have had a poor response to standard therapies. The first phase will determine whether the approach is safe for these patients, identify any potential side effects and assess how the gene-modified stem cells perform when returned to the body. This program extends work by Sangamo, which has safely treated more than 70 HIV patients using the same technology to cut out CCR5 in mature T cells. The trial will be part of City of Hope’s new Alpha Clinic for Cell Therapy and Innovation. The CIRM-funded clinic is devoted to bringing stem cell clinical trials to patients battling currently incurable diseases. Learn more about HIV research using the ZFN in this "elevator speech" from principal investigator Zaia:** Learn more about City of Hope’s new Alpha Clinic for Cell Therapy and Innovation.


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