mitoxantrone

Taken together, our results identify a distinct type of ribosome-enriched SGs that form through RNA-ribosome condensation rather than classical translational stress pathways. This mechanism provides a direct example of how a clinically used drug can reorganize cytoplasmic RNA-protein complexes, with possible consequences for mRNA regulation, cancer therapy, and neurodegenerative disease.

These findings demonstrate that PPA is unlikely to be a substrate of ABCG2 but functionally inhibits ABCG2-mediated efflux, contributing to the restoration of MTX sensitivity, although there may be additional mechanisms involved. PPA could be a promising MDR-reversal agent in ABCG2-driven chemotherapy resistance.

P=N/A, N=86, Not yet recruiting, Hunan Cancer Hospital; Hunan Cancer Hospital

Intratumoral mitoxantrone administration in a murine CD24-high glioma model extended survival, decreased tumor size, reduced Siglec-10+/TREM2+ cell populations, and increased anti-tumor CD8+ cells. These findings suggest that targeting the CD24/Siglec-10 axis with mitoxantrone may modulate the tumor microenvironment and enhance anti-tumor immunity. Keywords: CD24, Siglec-10, Mitoxantrone, Malignant brain tumor, Immunotherapy.

P2, N=206, Active, not recruiting, St. Jude Children's Research Hospital | Trial primary completion date: Jun 2025 --> Sep 2025

Mitoxantrone (MIT), an ICD inducer, and cisplatin (CDDP), a non-ICD inducer, were compared with PTT. Moreover, PTT showed a supra-additive effect in terms of therapeutic effect and anti-tumor activation when combined with an immune checkpoint inhibitor. In this study, we demonstrated that PTT induced ICD-related signaling and improved the response rate of ICI, which means PTT is a promising combination therapy with ICI.

P3, N=167, Terminated, Delta-Fly Pharma, Inc. | N=450 --> 167 | Trial completion date: Jun 2026 --> Jan 2026 | Recruiting --> Terminated; The data met the protocol-specified criteria for futility of the investigational treatment.

P4, N=114, Not yet recruiting, Daping Hospital and the Research Institute of Surgery of the Third Military Medical University

Cell lines with higher ABCG2 expression exhibited lower sensitivity to mitoxantrone, topotecan, and doxorubicin and diminished cytotoxic response. Mechanistically, p38 regulated the expression and membrane localization of oligomeric ABCG2, essential for its efflux activity. This study highlights p38 as a promising target to overcome ABCG2-mediated multidrug resistance and improve treatment outcomes for drug-resistant tumors.

P2/3, N=300, Recruiting, Shanghai Jiao Tong University School of Medicine | Phase classification: P1 --> P2/3 | N=50 --> 300

Here, we developed a novel whole-cell vaccine, termed MDLSD, by combining mitoxantrone (MTX)-induced ICD with the TLR9 agonist SD-101. Furthermore, mice cured by MDLSD developed long-term immunological memory, characterized by a recall response dominated by IFN-γ+ CD8+ and TNF-α+ T cells, enabling rejection of secondary tumor challenges. Collectively, our findings illustrate that the MDLSD vaccine synergizes the antigenic breadth of ICD with potent TLR9 stimulation to elicit robust DC activation, polyfunctional T-cell responses, and durable immunological memory, offering a compelling strategy for cancer immunotherapy.

P1, N=48, Active, not recruiting, National Cancer Institute (NCI) | Suspended --> Active, not recruiting