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Rhabdoid tumors as a novel target for PSMA-directed CAR T cell therapy
Aroshi Mitra1, Tianyi Zhou1, Jacky Wu1
1Center for Nuclear Receptors and Cell Signaling, Department of Biology and Biochemistry, University of Houston, Houston, TX 77204, USA.
Molecular Therapy. Oncology
|February 2, 2026
Summary
Prostate-specific membrane antigen (PSMA) is a promising target for ultra-rare pediatric rhabdoid tumors. PSMA-directed CAR T cells show potent anti-tumor activity in preclinical models, offering new therapeutic hope.
Area of Science:
- Oncology
- Immunotherapy
- Pediatric Malignancies
Background:
- Rhabdoid tumors are aggressive pediatric cancers with limited treatment options.
- Identifying novel therapeutic targets is crucial for improving patient outcomes.
Purpose of the Study:
- To investigate prostate-specific membrane antigen (PSMA) as a potential immunotherapeutic target in rhabdoid tumors.
- To evaluate the efficacy of PSMA-directed chimeric antigen receptor (CAR) T cells against rhabdoid tumors.
Main Methods:
- Transcriptomic data analysis identified high PSMA expression in rhabdoid tumor cell lines.
- PSMA expression was validated in cell lines and primary tumor specimens.
- PSMA-directed CAR T cells were tested for cytotoxicity against rhabdoid tumor cells in vitro and in vivo in xenograft models.
Main Results:
- Rhabdoid tumor cell lines and primary specimens showed significant PSMA expression.
- PSMA CAR T cells exhibited potent, antigen-specific killing of rhabdoid tumor cells in vitro.
- In vivo studies demonstrated significant tumor regression and CAR T cell infiltration in xenograft models.
Conclusions:
- Prostate-specific membrane antigen (PSMA) is a viable therapeutic target for rhabdoid tumors beyond prostate cancer.
- Preclinical data support the development of PSMA-directed immunotherapies for this pediatric malignancy.
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