topotecan / Generic mfg. - LARVOL DELTA

Dinutuximab Beta Added to Temozolomide-Based Chemotherapy for Children With Relapsed and Refractory Neuroblastoma: Results of the ITCC-SIOPEN BEACON Immuno Phase II Trial. (PubMed, J Clin Oncol) - "Within a randomized phase II setting, results observed with addition of dB to T-based chemotherapy in RR-HR-NB warrant further evaluation."

Journal • P2 data • Neuroblastoma • Oncology • Solid Tumor

Trilaciclib for the prevention of chemotherapy-induced myelosuppression: A systematic review and meta-analysis (ASH 2025) - "Chemotherapy regimens were heterogeneous and included etoposide-platinum (E/P) (either cisplatin or carboplatin) (n=5), gemcitabine/carboplatin (GCb) (n=2), topotecan (n=2), FOLFOXIRI (n=1), and Carboplatin/Paclitaxel/Tislelizumab (n=1)...It also showed a positive impact on progression-free and overall survival, without compromising chemotherapy effectiveness or increasing toxicity. These findings highlight Trilaciclib's potential as a valuable adjunct to chemotherapy in appropriately selected patients."

Retrospective data • Review • Breast Cancer • Colorectal Cancer • Febrile Neutropenia • Infectious Disease • Lung Cancer • Neutropenia • Non Small Cell Lung Cancer • Small Cell Lung Cancer • Solid Tumor • Thrombocytopenia • Triple Negative Breast Cancer

Atypical hemolytic uremic Syndrome Triggered by malignancy and drug exposure: A systematic review and meta-analysis (ASH 2025) - "Tacrolimus was the mostreported drug trigger (n=6)followed by gemcitabine (n=5), vincristine (n=5), bevacizumab (n=3), carfilzomib (n=3), 6-mercaptopurine(n=2), methotrexate (n=2), and mitomycin (n=2). Aflibercept, bactrim, bleomycin, capecitabine, cisplatin, cyclophosphamide, cytarabine,dasatinib, deferasirox, dinutuximab, estarylla, ketoprofen, L-asparaginase, modakafusp alfa, PEG-asparaginase, sunitinib, syntheticpsychoactive drugs, tamoxifen, and topotecan were reported as potential triggers for aHUS in one patient each...The pooled rate of treatment with eculizumab was 74% (95% CI, 0.629-0.842, p < 0.01, I2 = 72%),and the pooled rate of renal recovery was 65% (95% CI, 0.525-0.761, p < 0.01, I2 = 55%)...AKI and hematological abnormalities in these patients should prompt an emergent work-up and treatment. Current evidenceis primarily derived from case reports, so prospective trials are necessary to establish the incidence, associations, triggers, and outcomes..."

Retrospective data • Review • Acute Kidney Injury • Acute Lymphocytic Leukemia • Anemia • Atypical Hemolytic Uremic Syndrome • B Acute Lymphoblastic Leukemia • Biliary Cancer • Breast Cancer • Cholangiocarcinoma • Complement-mediated Rare Disorders • Genito-urinary Cancer • Hematological Malignancies • Hepatocellular Cancer • Hodgkin Lymphoma • Leukemia • Lung Cancer • Lymphoma • Multiple Myeloma • Nephrology • Neuroblastoma • Oncology • Ovarian Cancer • Pancreatic Cancer • Prostate Cancer • Renal Cell Carcinoma • Renal Disease • Solid Tumor • Thrombocytopenia • Urothelial Cancer

Tisotumab vedotin vs investigator's choice of chemotherapy as second- or third-line (2L/3L) treatment for Chinese patients (pts) with recurrent or metastatic cervical cancer (r/mCC) in innovaTV 301 (ESMO Asia 2025) - P3 | "This report presents the first analysis of efficacy and safety of TV in Chinese pts with r/mCC, based on data from both global and China extension portions within innovaTV 301. Eligible pts had r/mCC with disease progression following standard chemotherapy doublet ± bevacizumab ± anti-PD-(L)1 therapy, measurable disease per RECIST v1.1, and an ECOG performance status of 0-1. Pts were randomised 1:1 to receive TV monotherapy or investigator's choice of topotecan, vinorelbine, gemcitabine, irinotecan, or pemetrexed... TV as 2L/3L treatment demonstrated clinically meaningful improvements in OS, PFS, and ORR vs chemotherapy, with a manageable safety profile, for Chinese pts with r/mCC."

Clinical • Metastases • Cervical Cancer • Oncology • Solid Tumor

Idasanutlin in Combination with Chemotherapy or Venetoclax in Pediatric and Young Adult Patients with Relapsed/Refractory Solid Tumors (iMATRIX Idasa): Results of a Phase I/II, Multicenter, Multi-arm Study. (PubMed, Target Oncol) - P1/2 | "The safety profile and tolerable exposure of idasanutlin in pediatrics was similar to that reported in adults. Limited clinical activity was observed in patients with solid tumors. On the basis of the negative benefit-risk assessment, the study was terminated, and the overall pediatric development program for idasanutlin was discontinued."

Journal • P1/2 data • Febrile Neutropenia • Hematological Disorders • Neuroblastoma • Neutropenia • Oncology • Pediatrics • Solid Tumor • Thrombocytopenia

Poor response to systemic therapy upon progression on cyclin dependent kinase 4/6 inhibitors in HR+ Inflammatory Breast Cancer (SABCS 2025) - "RESULTS Among N = 33 patients evaluated, upon progression on standard of care CDKI and endocrine directed therapy (ET) combinations, patients received the following therapies with median ToT (months (min-max ToT)), respectively: capecitabine (N = 7; 3 (2-5)), everolimus/ET (N = 6, 3 (1-4)), Abraxane (N = 3, 3(2-10)), Eribulin (N = 2, 2.5(2-3)), capivasertib/fulvestrant (N = 1, 6(6)), taxol (N = 1, 5(5)), elacestrant (N = 1, 3(3)), fulvestrant (N = 1, 3(3)), tamoxifen (N = 1, 1(1)), intrathecal topotecan (N = 1, 1(1)), non standard/clinical trial (N = 4, 2.5(1-4)). CONCLUSION Patients with metastatic HR+ IBC demonstrate poor response to ET and cytotoxic chemotherapies upon progression on first line standard CDKI/ET, with a disproportionately high rate of death occurring in the first line setting due to advanced disease. Given previously reported low ToT on CDKI/ET in the first line and high incidence of brain relapse in this population, clinical trial design utilizing..."

Breast Cancer • HER2 Breast Cancer • HER2 Positive Breast Cancer • Hormone Receptor Breast Cancer • Hormone Receptor Positive Breast Cancer • Inflammatory Breast Cancer • Oncology • Solid Tumor • HER-2

Trastuzumab-deruxtecan for the management of leptomeningeal metastases from breast cancer (SNO 2025) - "IT chemotherapy included topotecan with trastuzumab, cytarabine with trastuzumab, and topotecan alone. Survival after LM diagnosis is increasing with novel therapy and our findings highlights the role of T-DXd even in the setting of low HER2. Our findings provide a single center experience in using trastuzumab-deruxtecan for LM in breast cancer patients."

Breast Cancer • HER2 Breast Cancer • HER2 Positive Breast Cancer • Hormone Receptor Breast Cancer • Oncology • Solid Tumor • ER • PGR

Monitoring therapy in leptomeningeal carcinomatosis in colorectal cancer using CSF liquid biopsy. (SNO 2025) - "In addition to systemic therapy, this patient was initially treated intrathecally with nivolumab, methotrexate, and topotecan, then weekly with intrathecal methotrexate. We show some discrepancy between CSF tumor load as measured by SNV and levels of chromosomal structural gain or loss. In summary, we show that longitudinal monitoring of LC using CSF LBx provides a reliable means for evaluating efficacy of therapy and perhaps can guide therapeutic plans."

Biopsy • Liquid biopsy • Brain Cancer • Colorectal Cancer • Oncology • Solid Tumor • BRAF

Results of a Pilot Trial of Infusion Rate Escalation During Convection Enhanced Delivery of Topotecan with a Multiport Catheter. (PubMed, World Neurosurg) - "Increasing the rate of infusion can allow for larger volume of distribution into enhancing tumor tissue."

Journal • Brain Cancer • Glioblastoma • Glioma • High Grade Glioma • Oncology • Solid Tumor

The Safety and Efficacy of Intravitreal Topotecan for the Treatment of Proliferative Vitreoretinopathy (clinicaltrials.gov) - P1 | N=50 | Not yet recruiting | Sponsor: Massachusetts Eye and Ear Infirmary | Trial completion date: Mar 2026 ➔ Mar 2027 | Trial primary completion date: Mar 2026 ➔ Dec 2026

Trial completion date • Trial primary completion date • Retinal Disorders

Elucidating the Pro-Tumor Myeloid Response to Locally-Delivered Topotecan in Recurrent Glioblastoma (SNO 2025) - "The transcription factor CEBPB is a key predicted master regulator of the MARCO-mesenchymal transcriptomic program. Our work therefore nominates CEBPB blockade as a therapeutic strategy for disrupting the pro-tumor macrophage population in the setting of CED of topotecan."

Brain Cancer • CNS Tumor • Glioblastoma • Oncology • Solid Tumor • CEBPB

Non-enhancing regions of Glioblastoma Harbor Quiescent Stem Like Cells that Contribute to Chemotherapy Resistance (SNO 2025) - "Cells were treated with temozolomide (TMZ, 1000 μM), paclitaxel (PTX, 50 μM), topotecan (TPT, 50 μM), vincristine (VCR, 50 μM), and doxorubicin (DOX, 20 μM) for 48 hours. NE infiltrating regions of glioblastoma maintain high proportions of cells that have characteristics of glioma stem cells transcriptionally. These regions have slow initial growth in in vitro and in vivo models, but are able to recapitulate solid tumor properties. Likely related to this quiescent property, NE regions are more resistant to chemotherapeutic agents."

Brain Cancer • Glioblastoma • Glioma • Solid Tumor • PROM1

Enhancing drug retention in H3 K27-altered diffuse midline glioma: Combining convection-enhanced delivery with systemic efflux inhibition (SNO 2025) - "However, systemic everolimus administration prolonged drug retention and potentiated the effects of efflux substrate drugs delivered via CED (e.g., topotecan, ponatinib). The observed synergistic effects highlight a promising strategy to overcome drug clearance limitations and improve treatment outcomes. Further clinical investigation is warranted to validate this approach in patients."

Brain Cancer • Diffuse Midline Glioma • Glioma • Solid Tumor

Application of surface-enhanced Raman scattering combined with artificial intelligence for multiplex detection of chemotherapeutic agents used in solid tumors. (PubMed, Anal Bioanal Chem) - "Carboplatin, epirubicin, gemcitabine, paclitaxel, topotecan, and docetaxel are widely used in combination regimens; however, there is still a lack of sensitive, rapid, and multiplex technologies for real-time monitoring of chemotherapeutic drug concentrations. Within the simulated therapeutic concentration range, quantitative regression yielded R2 ≥ 0.98. This AI-SERS platform offers advantages of multiplex detection, trace-level sensitivity, and strong anti-matrix interference, holding great promise for pharmacokinetic monitoring and individualized chemotherapy optimization across a broad range of solid tumors."

Journal • Breast Cancer • Endometrial Cancer • Gynecologic Cancers • Oncology • Ovarian Cancer • Solid Tumor

In Vitro Stability and Preclinical Safety Evaluation of High-Dose Intravitreal Topotecan in Rabbits: Impact of Dose and Concentration. (PubMed, Ophthalmol Sci) - "The results of this study may have clinical utility in the assessment of very high-dose topotecan as a salvage treatment of highly compromised eyes with recurrent or relapsed subretinal seeds and retinal tumors. Proprietary or commercial disclosure may be found in the Footnotes and Disclosures at the end of this article."

Journal • Preclinical • Alopecia • Eye Cancer • Hematological Disorders • Oncology • Ophthalmology • Retinal Disorders • Retinoblastoma • Solid Tumor

A selective RPL15 PROTAC degrader enhances anti-PD-1 immunotherapy in a murine melanoma tumor model. (PubMed, Oncogene) - "We previously showed that topotecan, a chemotherapeutic drug and topoisomerase I (TOP1) inhibitor, binds to ribosomal protein RPL15 and induces the secretion of DAMPs from cancer cells, which activate cGAS-STING signaling in dendritic cells...Here, we synthesized SN-38-conjugated pomalidomide (SN38-PROTAC) and showed that SN38-PROTAC induced ubiquitin-mediated degradation of RPL15, but not TOP1...The enhanced antitumor effects of SN38-PROTAC and anti-PD-1 antibody combination were abolished in STING-deficient mice. Our results indicate that SN38-PROTAC, which induces RPL15 degradation, has the potential to enhance ICI efficacy in PD-1-resistant cancer with low cytotoxicity."

Journal • Preclinical • Melanoma • Oncology • Solid Tumor • Targeted Protein Degradation • TOP1

COLLATERAL SENSITIVIES EMERGE DURING THE EVOLUTION OF CHEMORESISTANCE TO MAP CHEMOTHERAPY IN OSTEOSARCOMA (CTOS 2025) - "Objective: We aimed to model the evolution of chemoresistance to methotrexate, doxorubicin, and cisplatin (MAP) in vitro and to characterize collateral drug sensitivity and resistance patterns over time, potentially informing second-line treatment strategies in chemo resistant primary or recurrent disease. Five evolutionary replicates of MG63.3 osteosarcoma cells were treated with six complete cycles of pulsed, clinically relevant doses of cisplatin and doxorubicin, alternating with methotrexate...Collateral resistance emerged to etoposide, vincristine, and topotecan, while sensitivity increased to gemcitabine, cabozantinib, and palifosfamide... Our model reveals temporally dynamic, heterogeneous collateral responses during the development of chemoresistance to MAP in osteosarcoma. While cross-resistance to several agents emerged, collateral sensitivity to gemcitabine and cabozantinib supports a second line use for these agents. These data may inform rational sequencing..."

Oncology • Osteosarcoma • Sarcoma • Solid Tumor

Camptothecin in Cancer Therapy: Current Challenges and Emerging Strategies with Nanoemulsions. (PubMed, Pharmaceutics) - "Semi-synthetic analogs such as irinotecan (CPT-11) and topotecan (TPT) have been developed and approved for the treatment of various types of cancer; however, challenges related to drug resistance and side effects continue to arise. Therefore, nanomedicine and nanoparticle-based delivery systems, including nanoemulsions, liposomes, and antibody-drug conjugates (ADCs), emerge as promising strategies to improve the stability, bioavailability, and effectiveness of CPT, despite significant challenges such as scalability, pharmacokinetic variability, and regulatory requirements. This review discusses recent advances in CPT, its analogs, and these delivery platforms, highlighting its potential to optimize cancer therapy and reduce toxicity while outlining translational challenges such as scalability, pharmacokinetic variability, and regulatory requirements."

Journal • Review • Oncology • TOP1

A Study to Compare Sacituzumab Tirumotecan (MK-2870) Monotherapy Versus Treatment of Physician's Choice as Second-line Treatment for Participants With Recurrent or Metastatic Cervical Cancer (MK-2870-020/TroFuse-020/Gog-3101/ENGOT-cx20) (clinicaltrials.gov) - P3 | N=686 | Recruiting | Sponsor: Merck Sharp & Dohme LLC | Trial completion date: Oct 2028 ➔ Jun 2028 | Trial primary completion date: Oct 2028 ➔ Jun 2028

Monotherapy • Trial completion date • Trial primary completion date • Cervical Cancer • Oncology • Solid Tumor

Investigation of Immunogenic Cell Death Induced by Cytotoxic Anticancer Agents in Small Cell Lung Cancer (APSR 2025) - "Methods : We verified the ability of various cytotoxic anticancer agents (cisplatin, carboplatin, etoposide, SN-38 (an active metabolite of irinotecan), and topotecan) to induce ICD in multiple human SCLC cell lines (H841, SBC3, SBC5, etc.). Flow cytometry analyses demonstrated that CRT expression on cell surface was robustly induced by exposure with etoposide and topotecan, while extracellular HMGB1 release was notably increased by exposure with carboplatin and etoposide. Conclusion : These findings suggest that carboplatin, etoposide, and topotecan efficiently induce ICD in SCLC cells, potentially offering new therapeutic options when combined with ICIs."

Immunogenic cell death • IO biomarker • Lung Cancer • Oncology • Small Cell Lung Cancer • Solid Tumor • ANXA5 • CALR • HMGB1

Assessment of leachables in hospital pharmacy compounded topotecan conditioned in common off-label syringes for intravitreal use. (PubMed, Sci Rep) - "In contrast, BBraun Omnifix showed greater chemical stability, releasing only minimal silicone-related compounds and oleamide, suggesting a lower risk for intraocular use. Although these findings support BBraun Omnifix as a safer option, the findings underscore the need for toxicologically assessed, purpose-designed plastic PFSs made from low-extractable materials such as cyclic olefin polymer (COP) or copolymer (COC) for long-term drug storage."

Journal • Eye Cancer • Oncology • Retinal Disorders • Retinoblastoma • Solid Tumor

Trastuzumab-deruxtecan for the management of leptomeningeal metastases from breast cancer (WFNOS 2025) - "IT chemotherapy included topotecan with trastuzumab, cytarabine with trastuzumab, and topotecan alone. Survival after LM diagnosis is increasing with novel therapy and our findings highlights the role of T-DXd even in the setting of low HER2. Our findings provide a single center experience in using trastuzumab-deruxtecan for LM in breast cancer patients."

Breast Cancer • HER2 Breast Cancer • HER2 Positive Breast Cancer • Hormone Receptor Breast Cancer • Oncology • Solid Tumor • ER • PGR

Monitoring therapy in leptomeningeal carcinomatosis in colorectal cancer using CSF liquid biopsy. (WFNOS 2025) - "In addition to systemic therapy, this patient was initially treated intrathecally with nivolumab, methotrexate, and topotecan, then weekly with intrathecal methotrexate. We show some discrepancy between CSF tumor load as measured by SNV and levels of chromosomal structural gain or loss. In summary, we show that longitudinal monitoring of LC using CSF LBx provides a reliable means for evaluating efficacy of therapy and perhaps can guide therapeutic plans."

Biopsy • Liquid biopsy • Brain Cancer • Colorectal Cancer • Oncology • Solid Tumor • BRAF

Elucidating the Pro-Tumor Myeloid Response to Locally-Delivered Topotecan in Recurrent Glioblastoma (WFNOS 2025) - "The transcription factor CEBPB is a key predicted master regulator of the MARCO-mesenchymal transcriptomic program. Our work therefore nominates CEBPB blockade as a therapeutic strategy for disrupting the pro-tumor macrophage population in the setting of CED of topotecan."

Brain Cancer • CNS Tumor • Glioblastoma • Oncology • Solid Tumor • CEBPB

Enhancing drug retention in H3 K27-altered diffuse midline glioma: Combining convection-enhanced delivery with systemic efflux inhibition (WFNOS 2025) - "However, systemic everolimus administration prolonged drug retention and potentiated the effects of efflux substrate drugs delivered via CED (e.g., topotecan, ponatinib). The observed synergistic effects highlight a promising strategy to overcome drug clearance limitations and improve treatment outcomes. Further clinical investigation is warranted to validate this approach in patients."

Brain Cancer • Diffuse Midline Glioma • Solid Tumor