A phase 1 study to assess the safety, tolerability and effectiveness of PV-10 (Rose Bengal Sodium) in neuroendocrine tumours metastatic to the liver

作者：Timothy Price, Laura Depauw, Gabrielle Cehic, Eric A. Wachter, Ruben Sebben, Jessica Reid, Susan J. Neuhaus, Anas Alawawdeh, Ian Kirkwood, Rahul Solanki, Mark D. McGregor, Lisa Leopardi, Dominic Rodrigues, Guy J. Maddern · 发表于：British Journal of Cancer · 年份：2025 · DOI：10.1038/s41416-025-02976-9 · 被引用次数：3 · 研究领域：Neuroendocrine Tumor Research Advances、Radiopharmaceutical Chemistry and Applications、Lung Cancer Research Studies

BACKGROUND: Metastatic neuroendocrine neoplasms (mNEN) require new treatment options. Intralesional (IL) PV-10 is an autolytic chemotherapy that may elicit an adaptive immune response. METHODS: This phase 1 study evaluated IL PV-10 administered percutaneously to hepatic lesions in patients with progressive mNEN. IL PV-10 was injected in a single lesion per treatment cycle. A treatment cycle could be repeated after ≥ 6 weeks if multiple targetable lesions were present. The primary endpoint was safety. RESULTS: Twelve patients were enrolled with a median age of 66 years (range 47-79). All patients had progressive disease at enrolment and received prior somatostatin analogues; 10 patients had peptide receptor radionuclide therapy (PRRT) treatment. One lesion was injected per cycle for all 12 patients. Reported grade 3 side effects were photosensitivity (1 patient), face oedema (1 patient), elevated transaminases (1 patient), hypertension (2 patients). Response rate was 42% of injected lesions with patient-level disease control of 84%, PFS 9.4 months and median OS 24.0 months. CONCLUSIONS: IL PV-10 elicited no safety concerns. Encouraging evidence of local and systemic disease control was seen in a heavily pre-treated, progressing mNEN population. CLINICAL TRIAL REGISTRATION NUMBER: NCT02693067.