{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,20]],"date-time":"2026-06-20T14:45:24Z","timestamp":1781966724666,"version":"3.54.5"},"reference-count":29,"publisher":"Oxford University Press (OUP)","issue":"8","license":[{"start":{"date-parts":[[2025,5,16]],"date-time":"2025-05-16T00:00:00Z","timestamp":1747353600000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/academic.oup.com\/pages\/standard-publication-reuse-rights"}],"funder":[{"DOI":"10.13039\/100009619","name":"Japan Agency for Medical Research and Development","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100009619","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2025,8,3]]},"abstract":"<jats:title>Abstract<\/jats:title>\n               <jats:sec>\n                  <jats:title>\u00a0<\/jats:title>\n                  <jats:p>This multi-institutional, double-blind, randomized, placebo-controlled phase III trial was designed to evaluate the efficacy and safety of Cellm-001, an autologous formalin-fixed brain tumor immunostimulant, for newly diagnosed glioblastoma with gross total resection to prolong overall survival (OS) and prevent recurrence after surgery. One hundred twelve patients are to be randomized 1:1 to either Cellm-001 with standard chemoradiotherapy (CRT) or saline solution with standard CRT. Randomization is based on the following stratified randomization criteria: age, Karnofsky Performance Status, and the presence or absence of photodynamic therapy (PDT). The primary endpoint is OS and secondary outcomes are progression-free survival (PFS), OS and PFS with and without radiographically residual lesions as subgroups, OS and PFS with and without PDT, p53-negative OS and PFS, high Cluster of Differentiation-8 score OS and PFS, OS associated with death in primary disease, and 24-month OS and PFS rates. All institutions received ethical committee approval and patient enrollment began in 2021.<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>Importance of the study<\/jats:title>\n                  <jats:p>Given the growing interest in immunotherapy (IMT), we developed an autologous formalin-fixed tumor vaccine (AFTV) manufactured from the patient\u2019s own glioblastoma multiforme (GBM) tissue in paraffin-embedded blocks made from the resected tumor and a double-blind, randomized phase IIB trial of AFTV with temozolomide in newly diagnosed GBM was conducted. The 3-year progression-free survival (PFS) rate for patients with gross total resection (GTR) on imaging tended toward improvement: 81% in the AFTV group versus 46% in the placebo group (P\u00a0=\u00a0.067). Based on these IIB results, the feasibility of conducting a phase III trial was confirmed for IIB-eligible patients with total resection. We here plan to conduct the world\u2019s first double-blind, randomized, placebo-controlled phase III trial using Cellm-001 to demonstrate autologous tumor immunostimulant efficacy. This IMT, in combination with sub-analyses (GTR, P53 status, CD8 score, and other factors) to be validated, is expected to be a breakthrough in effective standards of care for the treatment of GBM.<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>Trial registration<\/jats:title>\n                  <jats:p>Registry number: jRCT2031200153; Date of Registration: 20 \/October, \/2020; Date of First Patient Enrollment: 14 \/January\/, 2021.<\/jats:p>\n               <\/jats:sec>","DOI":"10.1093\/jjco\/hyaf078","type":"journal-article","created":{"date-parts":[[2025,5,16]],"date-time":"2025-05-16T08:25:10Z","timestamp":1747383910000},"page":"975-981","source":"Crossref","is-referenced-by-count":4,"title":["A randomized, placebo-controlled phase III trial of an autologous, formalin-fixed tumor vaccine for newly diagnosed glioblastoma: trial 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