A first-in-human peptide vaccine targeting H3K27M; encouraging early findings in 8 adults with diffuse midline glioma
Year: 2023
Type: article
Abstract: A case series reported promising findings for a 27mer H3K27M targeted peptide vaccine in 8 patients with diffuse midline glioma (DMG).1 This was a report from a compassionate access program delivering a first-in-human therapy to patients with recurrent disease. DMG, typically presenting in children and young adults, is a highly aggressive primary tumor within the central nervous system; however, unfortunately there are no proven effective systemic therapies for DMG, and it remains an incurable cancer.2 This deadly tumor typically occurs in the midline structures of the brain and is characterized by a mutation in K27M genes,3 which produces a tumor antigen that can be targeted for vaccination strategies. All 8 patients in this series had received prior treatment with standard radiation and temozolomide, and 1 patient also received lomustine (CCNU). The 8 patients were 4 females and 4 males, and their ages ranged from 18 to 35 years with a mean of 28 years. Patients represented a variety of different human leukocyte antigen (HLA) types. The median progression-free survival (PFS) of the 8 patients after vaccination was 6.2 months and the median overall survival (OS) was 12.8 months. The PFS and OS figures in patients with recurrent DMG were encouraging with the caveat that there was an inherent selection bias in this series, as it was not a prospective clinical trial. There were also inconsistent variables across the patient population including the co-administration of immune checkpoint inhibitors on a variable schedule in most, but not all, of the 8 patients. The differing characteristics and treatments prior to being enrolled in this cohort limit some interpretations that can be made based on this study.
Source: Neuro-Oncology
Institutions: Duke University, Duke Medical Center
Language: English
Subfield: Genetics
Field: Medicine
Domain: Health Sciences
Open Access status: bronze
Funder: National Cancer Institute
OpenAlex ID: W4388410937