Therapeutic targeting of BCL-2 during CART cell production augments potency through non-apoptotic adaptive changes
Targeting the BCL-2 family of proteins, key regulators of cellular apoptosis, with BH3-mimetics has been a major therapeutic goal to overcome cancer cell death resistance. However, beyond their canonical roles in apoptosis, BCL-2 proteins also play vital roles in cellular metabolism, signaling, and, increasingly, immune cell regulation. T cells in particular depend heavily on BCL-2 family proteins during ontogeny and maintenance, yet the broader consequences of pharmacologically inhibiting anti-apoptotic BCL-2 proteins in T cells remain underexplored. Our group has previously demonstrated that BCL-2 inhibition with the BH3-mimetic venetoclax induces gene expression and plasticity changes in murine T cells. Building upon this, we here investigate whether venetoclax-driven T cell reprogramming can be leveraged to enhance adoptive cell therapies, specifically using chimeric antigen receptor (CAR) T cells targeting CD19 as a model system. Our findings reveal that venetoclax treatment during ex vivo expansion of CART cells, prepared from T cells from healthy donors or chemotherapeutically pretreated patients, potently augments antitumor efficacy in a BCL-2-dependent manner. Transcriptomic and functional analyses demonstrate that venetoclax reprograms CART cells by regulating signaling pathways (e.g., IL-2/STAT5 and PI3K/AKT) and metabolic programs (e.g., OXPHOS and glycolysis), yielding CART cells with superior fitness and effector functionalities. These results highlight a novel therapeutic approach using anti-apoptotic drugging to enhance the performance of adoptive T cell therapies and support further examination of venetoclax and other BH3-mimetics as immune modulators.
Authors
- Peter A. Riedell (ORCID: https://orcid.org/0000-0003-2719-0580)
- Erting Tang (ORCID: https://orcid.org/0000-0001-5348-799X)
- Lishi Xie (ORCID: https://orcid.org/0000-0003-2376-6685)
- James L. LaBelle (ORCID: https://orcid.org/0000-0001-6776-4695)
- Jun Huang (ORCID: https://orcid.org/0000-0003-0271-4384)
- Nada S. Aboelella (ORCID: https://orcid.org/0000-0002-9009-9897)
- Nicholas Asby (ORCID: https://orcid.org/0000-0002-1675-7601)
- Ryan S. Park (ORCID: https://orcid.org/0000-0001-9896-4585)
- Justin P. Kline
- Tony Pan
- Sidney Wang
- Joshua D. Ho
Institutions
- University of Chicago (US)
Publication Details
- Journal
- Signal Transduction and Targeted Therapy
- Published
- 2026-04-27
- DOI
- https://doi.org/10.1038/s41392-026-02655-y
- Primary Topic
- CAR-T cell therapy research
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- AbbVie
- National Cancer Institute