topotecan

The most promising salvage options for patients responding insufficiently to treatment are the chemotherapy combinations, topotecan/vincristine/doxorubicin (TVD), topotecan/cyclophosphamide/etoposide (TCE), ifosfamide/carboplatin/etoposide (ICE) or topotecan/cyclophosphamide (TopoCy), or [131I]-mIBG therapy. Early-phase clinical trials are also a possible option in this setting.

P1, N=11, Active, not recruiting, Children's Hospital Los Angeles | Recruiting --> Active, not recruiting

P1/2, N=266, Recruiting, Beijing Tide Pharmaceutical Co., Ltd

Platinum combined with etoposide, irinotecan, or topotecan was defined as SCLC-like CT, whereas platinum with taxanes or gemcitabine was considered NSCLC-like CT. When stratified by molecular subtype, patients with the SCLC subtype who received SCLC-like CT showed a longer mOS compared to those treated with NSCLC-like CT (15 [9.9-20.1] vs. 6 [3.9-8.1] months, respectively; p = 0.47), although this difference did not reach statistical significance. These findings suggest that molecular subclassification may help inform the choice of optimal systemic therapy in patients with LCNEC.

P3, N=531, Not yet recruiting, AbbVie

This study establishes ZPLD1 as a prognostic indicator and potential therapeutic target in BLCA, paving the way for precision oncology strategies integrating ZPLD1-guided patient selection and immune-drug combinations.

P3, N=466, Recruiting, Incyte Corporation | Not yet recruiting --> Recruiting

P3, N=642, Completed, Children's Oncology Group | Unknown status --> Completed

P1, N=99, Completed, Children's Oncology Group | Active, not recruiting --> Completed | Trial completion date: Sep 2025 --> Dec 2025

P3, N=1100, Recruiting, AstraZeneca | Not yet recruiting --> Recruiting

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.

Mirvetuximab soravtansine (MIRV) is an antibody-drug conjugate (ADC) composed of the DM4 payload conjugated to a folate receptor α (FRα)-targeting antibody via the cleavable sulfo-SPDB linker. The MIRV Phase 3 registrational trial (MIRASOL) showed superiority of MIRV vs. chemotherapy (paclitaxel, pegylated liposomal doxorubicin, or topotecan) in patients with high (≥ 75%) FRα-expression PROC, showing an objective response rate of 42% versus 16%, a median progression-free survival of 5.6 versus 4.0 months, and an overall survival of 16.5 versus 12.8 months. Here, we briefly review MIRV mechanism of action, pharmacokinetics, pharmacodynamics, and key clinical efficacy and safety data.