Stem cell therapy may be effective in HIV, early trial suggests.

Phase I clinical data suggest that autologous stem cell gene therapy may be effective in restoring immune function in HIV-infected adults.

Enzo Biochem presented the early results for its therapy, HGTV 43, during a session of the 8th Conference on Retroviruses and Opportunistic Infections held in Chicago, IL. HGTV 43 contains 3 Enzo-developed antisense genes designed to interfere with the functioning of 2 HIV-1 genes essential for virus growth in human cells.

In the 5 patient trial, the therapeutic genes were inserted into blood stem cells removed from the patients and then reinfused, in the hope that they would act as a reservoir of progenitor cells that would divide and develop into CD4+ cells, the cell type infected by HIV-1. The results showed that the 3 anti-HIV-1 genes could be successfully placed in stem cells, and that these stem cells survived, grew, and developed into CD4+ cells for a minimum of 6 months, the study's endpoint. Anti-HIV-1 antisense RNA was detected in all patients after 6 months and has been detected in 2 of the 5 subjects for periods up to 12 months.

An upcoming phase II trial involving 6-60 HIV patients with low T-cell counts will test for enhanced expression of CD4+ cells.