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Embryonic stem cells can mature into all cells of the immune system. These, or adult blood-forming stem cells, could be used to create a replacement immune system that cannot be infected by HIV. Learn more about this image by clicking on it or see more of CIRM's stem cell images on our Flickr Photostream.

CIRM funds several research projects investigating ways of modifying adult blood-forming stem cells or embryonic stem cells to create a replacement immune system that is resistant to HIV infection.

If you want to learn more about CIRM funding decisions or make a comment directly to our board, join us at a public meeting. You can find agendas for upcoming public meetings on our meetings page.

Learn more about stem cell research:
Stem Cell Basics Primer | Stem Cell Videos | What We Fund

Find clinical trials:
CIRM does not track stem cell clinical trials. If you or a family member is interested in participating in a clinical trial, please see the national trial database to find a trial near you: clinicaltrials.gov

Description

Hopes for a stem cell cure or therapy for HIV infection were boosted in late 2010 when scientists reported that Timothy Ray Brown, also known as the "Berlin Patient”, had effectively had his HIV “cured”. Brown had received a bone marrow transplant as part of a treatment for leukemia that came from a donor whose cells were resistant to HIV infection.

The person who donated the bone marrow had a genetic mutation in a gene called CCR5, which makes a protein that is required for HIV to enter cells. The problem is that there aren’t enough people with naturally occurring CCR5 mutations to serve as bone marrow donors for all HIV patients.

Rather than relying on naturally resistant donors, CIRM is funding two disease teams worth almost $35 million that have the goal of engineering a person’s own bone marrow to make the cells resistant to HIV.

A $14 million CIRM grant to researchers at the City of Hope in Duarte led by Dr. John Zaia will induce a CCR5 mutation with the help of molecular scissors—an engineered molecule called a zinc-finger nuclease, a technology developed by Richmond, Calif., company Sangamo BioSciences Inc (here is a summary of that work).

CIRM is also providing $20 million to a UCLA team, headed by Dr Irvin Chen (here is a summary of that work). Working with California biotech, CalImmune, the researchers are studying a technique called RNA interference as a method to block expression of the CCR5 gene and in turn blocking HIV infection.

In either case, the goal is to induce a CCR5 mutation in the patient’s own blood-forming stem cells then reintroduce those cells into the patient. The hope is that these cells then repopulate the immune system. Restoring a competent immune system with cells that are no longer a target for HIV is why eradication or “cure “ becomes a possibility.

CIRM is also providing funds to scientists at UCLA to study other therapeutic strategies using stem cells to target HIV and genetic modification of the human genome to help the immune system resist HIV infection or disease progression. CIRM is providing further funds to City of Hope to develop other RNA based approaches to stem cell gene therapy for HIV. 

Jeff Sheehy, HIV/AIDS patient advocate member of the CIRM Governing Board, and John Zaia, leader of the City of Hope CIRM HIV Disease Team, discuss stem cell transplant strategies for the treatment of HIV/AIDS.

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