Gene-Edited Stem Cell Therapy for HIV/AIDS
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial explores a new gene-edited stem cell treatment for individuals with HIV/AIDS. The main goal is to determine if this treatment, called SB-728mR-HSPC, is safe and can be successfully administered following a specific pre-treatment with the drug busulfan. The trial includes two groups, each receiving a different dose of busulfan. It suits HIV-positive individuals on stable medication with controlled virus levels and without serious heart issues. Participants should not have conditions like active hepatitis or recent drug or alcohol abuse. As a Phase 1 trial, this research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this innovative therapy.
Will I have to stop taking my current medications?
The trial does not specify if you need to stop taking your current medications, but you cannot use AZT or maraviroc in your cART regimen. You also cannot be on any investigational agents, biological, chemotherapy, or radiation therapy.
Is there any evidence suggesting that this treatment is likely to be safe for humans?
Research has shown that gene-edited stem cell therapy, such as SB-728mR-HSPC, is generally safe. Animal studies found that this treatment is well-tolerated, with animals surviving after receiving it. In another study, patients received a similar gene-edited stem cell treatment and demonstrated promising safety results. These findings suggest the treatment could be safe, but further research is needed to confirm this in humans.12345
Why are researchers excited about this trial's treatment?
Unlike the standard treatments for HIV/AIDS, which typically involve antiretroviral therapy to control the virus, SB-728mR-HSPC offers a groundbreaking approach by using gene-edited stem cells. This treatment targets a key protein called CCR5, which the virus uses to enter human cells. By editing the genes of stem cells to disrupt the CCR5 protein, the therapy aims to create HIV-resistant immune cells. Researchers are excited because this approach could potentially reduce the need for lifelong medication and transform how HIV/AIDS is managed.
What evidence suggests that this treatment might be an effective treatment for HIV/AIDS?
Research has shown that SB-728mR-HSPC is promising in fighting HIV/AIDS. This treatment involves altering certain genes in stem cells to block the CCR5 gene, which HIV uses to enter cells. Studies have demonstrated that this method can reduce HIV levels by 80-95%. Additionally, the gene-editing process has successfully modified up to 72.9% of the targeted cells. Participants in this trial will receive SB-728mR-HSPC in different cohorts, with Cohort 1 targeting busulfan AUC levels of 8,000 µM*min (+/- 1,000) and Cohort 2 targeting 12,000 µM*min (+/- 1,000). These findings suggest that SB-728mR-HSPC could help control HIV more effectively by preventing it from entering cells.12678
Who Is on the Research Team?
Amrita Y. Krishnan, MD
Principal Investigator
City of Hope Medical Center
Are You a Good Fit for This Trial?
This trial is for HIV-1 infected patients under 75 years old, with controlled virus levels due to consistent antiretroviral therapy (cART) for at least a year, and specific CD4+ T-cell counts. They must not have significant heart issues or psychosocial conditions affecting study participation. Excluded are those with active hepatitis, pregnant or nursing women, recent investigational drug use, substance abuse history, certain hematologic diseases, recent opportunistic infections or malignancies.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Conditioning
Participants receive a two- or three-day course of busulfan to prepare for infusion of genetically modified cells
Treatment
Participants are infused with SB-728mR-HSPC, genetically modified hematopoietic stem/progenitor cells
Follow-up
Participants are monitored for safety and effectiveness after treatment, with assessments at 9-12 months post-infusion
What Are the Treatments Tested in This Trial?
Interventions
- SB-728mR-HSPC
Trial Overview
The trial tests the safety of SB-728mR-HSPC infusion in HIV/AIDS patients after busulfan conditioning. SB-728mR-HSPC is a genetically modified stem/progenitor cell product aimed at altering the immune system to resist HIV more effectively.
How Is the Trial Designed?
2
Treatment groups
Experimental Treatment
busulfan AUC levels: 12,000 µM\*min (+/- 1,000)
target busulfan AUC levels: 8,000 µM\*min (+/- 1,000)
Find a Clinic Near You
Who Is Running the Clinical Trial?
City of Hope Medical Center
Lead Sponsor
Sangamo Therapeutics
Industry Sponsor
California Institute for Regenerative Medicine (CIRM)
Collaborator
Citations
Gene-Edited Stem Cell Therapy for HIV/AIDS
SB-728mR-HSPC is unique because it involves gene-editing of hematopoietic stem cells to disrupt the CCR5 gene, which is crucial for HIV to enter cells. This ...
Promising Stem Cell therapy in the Management of HIV and ...
The outcome was an 80-to-95 percentage reduction in HIV levels, suggesting that stem-cell-based genetic engineering with a CAR might be a ...
NCT02500849 | Safety Study of Zinc Finger Nuclease ...
The objective of the study is to evaluate the safety and feasibility of giving autologous SB-728mR-HSPC to HIV-1 (R5) infected patients who are being treated ...
Preclinical development and qualification of ZFN-mediated ...
Our results demonstrate effective biallelic CCR5 disruption in up to 72.9% of modified colony forming units from adult mobilized HSPC with maintenance of ...
In vivo selection of anti-HIV-1 gene-modified human ...
Hematopoietic stem/progenitor cell (HSPC)–based anti-HIV-1 gene therapy holds great promise to eradicate HIV-1 or to provide long-term remission through a ...
A Phase I, Open-Label Study To Assess The Safety, Feasibility ...
Success of this therapy would establish the safety of a possible future cure for HIV-1/AIDS.
Preclinical development and qualification of ZFN-mediated ...
Our results demonstrate effective biallelic CCR5 disruption in up to 72.9% of modified colony forming units from adult mobilized HSPC with ...
CRISPR-Edited Stem Cells in a Patient with HIV and Acute ...
We transplanted CRISPR-edited CCR5-ablated hematopoietic stem and progenitor cells (HSPCs) into a patient with HIV-1 infection and acute lymphoblastic leukemia.
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