HIV/AIDS Fact Sheet

CIRM funds many projects seeking to better understand HIV/AIDS and to translate those discoveries into new therapies.

Description

HIV, or the human immunodeficiency virus, is a virus that infects cells of the immune system, undermining the body’s ability to fight disease. Eventually infection can lead to symptoms of AIDS (Acquired Immune Deficiency Syndrome), which includes susceptibility to infections, cancers and other diseases, and eventually causes death. The CDC estimated that in 2010, more than 1.148 million people in the U.S. were infected with HIV.

Stem cell approaches to treating people with HIV primarily involve replacing the person’s immune system with one that the virus can’t infect. Hope that this approach could work were boosted in late 2010 when scientists reported that Timothy Ray Brown, also known as the "Berlin Patient”, had effectively had his HIV “cured”. As part of a treatment for leukemia, Brown had received a bone marrow transplant 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. Without CCR5, HIV wasn’t able to infect these replacement immune cells and Brown has been able to go off his medications.

The problem is that there aren’t enough people with naturally occurring CCR5 mutations to serve as bone marrow donors for all HIV patients. Instead, scientists are hoping to create CCR5 mutations. They first plan to remove the blood-forming stem cells in a person’s bone marrow and mutate the CCR5 gene. The idea is that those genetically altered cells would then repopulate the person’s blood system with one that lacks CCR5 and that HIV won’t be able to infect.

Clinical Stage Programs

City of Hope

The City of Hope team plans to mutate the CCR5 gene using a technology called a zinc finger nuclease, which is essentially a pair of molecular scissors developed by Sangamo Biosciences that snips an exact spot on the CCR5 gene. Early evidence in animals suggests that when those cells are reintroduced, they create an immune system that HIV can’t infect. The team has begun a clinical trial with the procedure.

Calimmune

The Calimmune team is using a method called RNA interference to block the CCR5 gene from generating a protein. A blood system generated from these cells will lack CCR5 and block HIV infection. The team has begun a clinical trial with the procedure.

University of California, Davis

The team at is taking a patient’s blood forming stem cells and inserting 3 anti-HIV genes into them and then returning them to the individual. The anti-HIV genes are then passed on to all new immune system cells and make them resistant to HIV. Because AIDS-related lymphoma is linked to the constant immune cell stimulation caused by HIV infection, getting rid of the virus should prevent return of the cancer.

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.

CIRM Grants Targeting HIV/AIDS

Researcher name Institution Grant Title Grant Type Approved funds
Mehrdad Abedi University of California, Davis Stem Cell Gene Therapy for HIV in AIDS Lymphoma Patients Disease Team Therapy Planning I $66,880
Zack Jerome University of California, Los Angeles Stem Cell Programming With Chimeric Antigen Receptors to Eradicate HIV Infection Early Translational IV $4,925,166
Irvin Chen University of California, Los Angeles Development of a humanized mouse model for testing anti-HIV HSPC gene therapy strategies in HIV-1 infected mice. Early Translational from Disease Team Conversion $1,505,000
Mark Anderson University of California, San Francisco Generation of a functional thymus to induce immune tolerance to stem cell derivatives Basic Biology V $1,191,000
John Zaia City of Hope A Phase I, Open-Label Study To Assess The Safety, Feasibility and Engraftment of Zinc Finger Nucleases (ZFN) CCR5 Modified Autologous CD34+ Hematopoietic Stem/Progenitor Cells (SB-728MR-HSPC) with Escalating Doses of Busulfan In HIV-1 (R5) Infected Subjec Strategic Partnership III Track A $5,583,438
John Zaia City of Hope The Innovation-Alpha Clinic for Cellular Therapies (I-ACT) – A Program for the Development and Delivery of Innovative Cell-based Treatments and Cures for Life-threatening Diseases. Alpha Stem Cell Clinics $8,000,000
Paula Cannon University of Southern California Site-specific gene editing in hematopoietic stem cells as an anti-HIV therapy Tools and Technologies III $1,499,400
Mehrdad Abedi University of California, Davis Stem Cell Gene Therapy for HIV Mediated by Lentivector Transduced, Pre-selected CD34+ Cells in AIDS lymphoma patients Clinical Trial Stage Projects $8,414,265
David Baltimore California Institute of Technology Immunotherapy for HIV infection using engineered hematopoietic stem/progenitor cells Quest - Discovery Stage Research Projects $1,586,934
David DiGiusto City of Hope Development of RNA-based approaches to stem cell gene therapy for HIV Early Translational II $3,097,160
Irvin Chen University of California, Los Angeles Stem Cells: A New Avenue of HIV Research and New Approaches to HIV Treatment Conference $24,456
John Zaia City of Hope ZINC FINGER NUCLEASE-BASED STEM CELL THERAPY FOR AIDS Disease Team Research I $14,536,969
Irvin Chen University of California, Los Angeles HPSC based therapy for HIV disease using RNAi to CCR5. Disease Team Research I $9,905,604
Irvin Chen University of California, Los Angeles Genetic modification of the human genome to resist HIV-1 infection and/or disease progression SEED Grant $616,800
Zack Jerome University of California, Los Angeles Human Embryonic Stem Cell Therapeutic Strategies to Target HIV Disease Comprehensive Grant $2,401,903
Geoff Symonds Calimmune, Inc. GENE-MODIFIED HEMATOPOIETIC STEM/PROGENITOR CELL BASED THERAPY FOR HIV DISEASE Disease Team Research I $8,278,722
Mark Anderson University of California, San Francisco Stem cell differentiation to thymic epithelium for inducing tolerance to stem cells Transplantation Immunology $1,314,089
Total:
$72,947,786.00

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