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Riboswitch-controlled IL-12 gene therapy reduces hepatocellular cancer in mice.

Matthias J Düchs¹, Ramona F Kratzer², Pablo Vieyra-Garcia³

¹Research Beyond Borders, Boehringer Ingelheim Pharma GmbH & Co. KG, Biberach an der Riss, Germany.

Frontiers in Immunology

|April 1, 2024

Summary

Hepatocellular carcinoma and liver metastases pose significant challenges. Researchers developed a novel adeno-associated virus (AAV) gene therapy to safely deliver Interleukin-12 (IL-12) for effective cancer treatment.

Keywords:

AAV Tet-ON aptazyme riboswitch cancer il-12 immunotherapy inducible gene expression regulatable gene therapy

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Electroporation-Mediated Delivery of Cas9 Ribonucleoproteins and mRNA into Freshly Isolated Primary Mouse Hepatocytes

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Published on: June 2, 2022

Area of Science:

Oncology
Gene Therapy
Immunology

Background:

Hepatocellular carcinoma (HCC) and liver metastases represent significant unmet medical needs.
Interleukin-12 (IL-12) exhibits potent anti-tumor properties but is limited by severe toxicity.
Adeno-associated virus (AAV) vectors offer potential for targeted gene delivery.

Purpose of the Study:

To evaluate the efficacy and safety of AAV-mediated IL-12 gene delivery for treating liver tumors.
To establish controlled cytokine production using a tetracycline-inducible riboswitch system.
To assess the therapeutic potential of AAV-huIL-12 as a clinical candidate.

Main Methods:

Systemic delivery of AAV vectors carrying murine or human IL-12 genes to orthotopic liver tumors in mice.
Utilized a tetracycline-inducible K19 riboswitch for controlled IL-12 expression.
Analyzed STAT4 phosphorylation, interferon-γ (IFNγ) production, T cell infiltration, tumor regression, and vector tolerability.

Main Results:

AAV-mediated IL-12 expression successfully induced anti-tumor immune responses, including STAT4 phosphorylation and IFNγ production.
Significant tumor regression was observed in treated mice.
A safe and efficacious vector dose was determined through detailed efficacy and tolerability analyses.
Bioactive human IL-12 expression was achieved in a dose-dependent and tetracycline-inducible manner.

Conclusions:

Tissue-specific AAV vectors with riboswitch-controlled expression provide a safe and effective strategy for delivering potent proinflammatory cytokines.
This approach holds promise for advancing vector-based cancer immunotherapy for liver malignancies.
AAV-mediated IL-12 gene therapy represents an attractive platform for treating difficult-to-treat liver cancers.