HIV/AIDS: 'Functional Cure' On The Way?

By Matthew Klickstein , Mar 11, 2013 03:17 PM EDT

California-based Sangamo BioSciences, Inc. believes that it has taken HIV/AIDS research to a new height and developed what the firm is calling a "functional cure."

At the 20th Conference on Retroviruses and Opportunistic Infections (CROI) in Atlanta, Ga. March 3 through 6, Sangamo unveiled exciting new data on its HIV/AIDS functional cure research.

In Sangamo's first presentation at the Atlanta conference, the firm showed that its SB-728-T treatment of HIV-infected subjects has led to "durable reconstitution of the immune system" thanks to an increase in CD4+ central memory T-cells (TCM) and CCR5-protected TCM. This is an important development due to TCM being "long-lived, self-renewing cells that have the ability to remember and react against foreign antigens including HIV."

According to Sangamo's March 6 press release, the firm can now utilize certain cell surface marker and gene expression profiles to anticipate which patients will best respond to the SB-728-T treatment.

"These important data extend our understanding of why SB-728-T treatment improves the immune system as well as the conditions required for optimal engraftment of ZFN-modified T-cells," Sangamo's Vice President of Therapeutic Development and Chief Medical Officer Dale Ando, MD, says in the release. "[The data] confirm that SB-728-T meets the key immunologic requirements for immune reconstitution in HIV-infected individuals."

Ando added that he believes the data further shows that Sangamo can better distinguish how to "optimize" its future clinical trials regarding the specialized treatment.

Sangamo's research is particularly groundbreaking in light of the fact that the durable reconstitution of the immune system in HIV-infected subjects has never before been observed after a single treatment stemming from any other therapeutic research, according to Rafick-Pierre Sekaly, Ph.D., co-director and chief scientific officer, The Vaccine & Gene Therapy Institute of Florida (VGTI Florida).

Sekaly's laboratory carried out this analysis.

"Improvement in the overall health of the immune system of HIV-infected individuals, as demonstrated by treatment with SB-728-T, is a key step along the path to developing an immunologic approach to controlling and potentially eliminating the virus," Sekaly says.

An infected person's immune system is affected by HIV through the disease's killing off of CD4+ T cells. Currently, the regimen for HIV/AIDS victims includes a daily treatment with antiretroviral therapy (ART), which suppresses viral load in the blood of most subjects. This treatment, however, does not rid the body of HIV-infected cells and a lot of patients do not see their CD4+ T-cell rising back to normal levels.

"SB-728-T treatment, by eliminating the co-receptor, CCR5, which is necessary for HIV entry to CD4+ cells, is designed to provide a CCR5-negative population of CD4+ T-cells that cannot be infected by HIV but are able to fight opportunistic infections and enable the immune system to control and eliminate the virus," Sangamo's release says. "The data presented today demonstrate that SB-728-T treatment leads to unprecedented durable increases in total CD4+ T cells that are correlated with increases in TCM and ZFN-mediated CCR5-modified TCM. "

Sangamo's President and CEO Edward Lanphier says the company will engage in Phase 2 trials of the SB-728-T treatment, which will build on the earlier research that turned up the announced data. The information garnered from these new trials will be released later this year.

"These exciting data support our development program for SB-728-T as a potential functional cure for HIV/AIDS," Edwards says.

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