AIDS/HIV disease is a viral infection that destroys the immune response. The disease can be treated with drugs, however these drugs do not eliminate the virus from the body. Hence currently the patient must remain on these drugs for the remainder of their life. These drugs are expensive and often cause toxicities, thus an improved treatment strategy for this disease is needed. Our project proposes pre-clinical studies to advance towards clinical trial a stem cell-based method of enhancing the patient's own immune response against the virus. This approach involves identifying anti-HIV receptors that will be introduced into the patient's own blood-forming stem cells. These cells will then be transplanted back into the patient, and the new receptors in those cells will direct those cells to turn into mature white blood cells that kill other cells infected by the AIDS virus (HIV). Since stem cells are stable following transplant, this approach has the potential to provide long-term immune control of virus replication, and may require only one or a few administrations. In this project we will perform all of the necessary pre-clinical development and characterization of this approach, and complete all of the necessary regulatory steps to achieve regulatory approval to begin testing of this strategy in humans.
Statement of Benefit to California:
The state of California and its economy are greatly impacted by AIDS/HIV disease. In 2007, the most recent year for which data is available, over 1100 Californians died from HIV disease. In 2008 nearly 68,000 Californians were living with an AIDS diagnosis. HIV infected individuals can be treated with anti-viral drugs, but these do not completely eradicate HIV from the body. Consequently HIV infected patients must remain on these drugs for the remainder of their life. These drugs agents are expensive and associated with toxicities. For these reasons a stem cell-based therapy that offered long-term control or elimination of HIV without the toxicities associated with current drug therapies would be advantageous. A stem cell-based treatment may have an additional advantage over current drug treatment in that it may need to be administered only once, or a limited number of times. Our proposed approach would provide a means to genetically engineer stem cells to enhance the patient's own immune response against the virus. It is possible that this approach could be used alone, or in conjunction with other therapies to provide improved control of HIV and prolong the life of the HIV infected patient.