A phase 1, first-in-child, multicenter study to evaluate the safety and efficacy of the oncolytic herpes virus talimogene laherparepvec in pediatric patients with advanced solid tumors

The survival rates for pediatric patients with relapsed and refractory tumors are poor. Successful treatment strategies are currently lacking and there remains an unmet need for novel therapies for these patients. We report here the results of a phase 1 study of talimogene laherparepvec (T-VEC) and explore the safety of this oncolytic immunotherapy for the treatment of pediatric patients with advanced non-central nervous system tumors. T-VEC was delivered by intralesional injection at 10 plaque-forming units (PFU)/ml on the first day, followed by 10 PFU/ml on the first day of week 4 and every 2 weeks thereafter. The primary objective was to evaluate the safety and tolerability as assessed by the incidence of dose-limiting toxicities (DLTs). Secondary objectives included efficacy indicated by response and survival per modified immune-related response criteria simulating the Response Evaluation Criteria in Solid Tumors (irRC-RECIST). Fifteen patients were enrolled into two cohorts based on age: cohort A1 ( = 13) 12 to ≤21 years old (soft-tissue sarcoma, = 7; bone sarcoma, = 3; neuroblastoma, = 1; nasopharyngeal carcinoma, = 1; and melanoma, = 1) and cohort B1 ( = 2) 2 to