BACKGROUND:  The host immune system is crucial to final resolution of viral infection. Unfortunately, many viruses, such as HIV-1 and hepatitis C virus (HCV), can evade the immune system. There is no pharmaceutical treatment that can eradicate these viruses from infected individuals. The fate of these infected individuals hinges on the findings of new therapeutic approaches, which has been a long-standing goal of HIV/AIDS research.

INNOVATION:  Researchers at UCLA have developed a therapeutic technology that combines the advances in gene therapy, stem cell technology and immune therapy. This technology enables the generation of anti-viral T cells from the stem cells of a patient who fails to generate effective anti-viral T cells. This approach involves harnessing stem cells from a patient and delivering into these cells a cloned human T cell receptor gene; the T-cell receptor is the receptor on T cells that recognizes antigens on pathogens and cancers (in this case, the antigen is the HIV gag protein). The modified stem cells will be re-introduced into the patient where the cells would develop into mature, anti-viral T cells that can functionally respond to antigen and produce responses specific to the patient's infectious profile.

POTENTIAL APPLICATIONS 

• New therapeutics against infectious diseases, such as HIV/AIDS, HCV infection and influenza infection
• New therapeutics against cancers
• Effective in vivo generation of mature, functional viral-specific T cells

ADVANTAGES

• An autologous cell therapy, no drug toxicity and few safety concerns
• Currently, no known system exists that allow the genetic manipulation of human stem cells to produce specific anti-viral T-cells
• Effective in vivo generation of mature, functional viral-specific T cells

DEVELOPMENT-TO-DATE:  The technology has been tested in vivo against HIV-1 infection in a humanized mouse model.

Reference: UCLA Case No. 2009-252