Gene Therapy Trial Reveals Possible Control Of HIV
A recent study presented at the annual American Society of Gene Therapy (ASGT) meeting found that hematopoietic (blood) stem cell therapy was promising in the management and treatment of HIV and AIDS.
Geoff Symonds, one of the authors of the study and now Chief Scientific Officer of Calimmune, Inc., said his research while at Johnson & Johnson and Calimmune — which involves the therapeutic use of hematopoietic stem cells with protective genes — has the potential to safely and effectively provide permanent control over HIV.
These hematopoietic stem cells, which help form and renew blood cells, are found in the bone marrow of adults and are valuable to HIV researchers because of their ability to renew themselves and differentiate into other specialized blood cells.
The double-blind Johnson & Johnson study tested stem cell-delivered gene therapy, or the introduction of engineered genes into cells or tissues, on 74 HIV-positive patients. “For half the patients, the introduced hematopoietic stem cells had anti-HIV genes that were to protect progeny cells (daughter cells) from HIV infection and replication,” Symonds said.
Because the introduced stem cells created healthy CD4 T-cells with protection from HIV, the measured “viral loads” in patients, or the amounts of the virus in a given blood sample, decreased and the CD4 T-cell number increased.
“This study is a major advance in the field and it paves the way for future treatment with this new therapeutic paradigm,” Symonds said at the ASGT meeting.
Additionally, there were no gene therapy adverse side-effects to the treatment. This approach may be groundbreaking when compared to Highly Active Anti-Retroviral Treatments (HAARTs), which use three to four drugs in combination, must be taken life-long, and are known to have a variety of negative side-effects in HIV/AIDS patients.
“If this is truly effective and the hematopoietic stem cells keep producing protected CD4 T-cells over the life of the patient, it may be a ‘one off’ or infrequent therapy that acts alone or in concert with a reduced requirement for HAART,” Symonds said.
The original study appeared in the March 2009 issue of Nature Medicine (abstract).
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