Importance of conducting clinical trials on the efficacy of DCVax-L for advanced and recurrent glioblastoma in Japan

Dr. Liau et al reported that in this study, adding DCVax-L to standard of care (SOC) (i.e., temozolomide) resulted in clinically meaningful and statistically significant extension of survival for patients with both newly diagnosed glioblastoma (nGBM) and recurrent glioblastoma (rGBM) compared with contemporaneous, matched external controls who received SOC alone.

In Japan, Avastin (anti-hVEGF monoclonal antibody) is recommended as the first-line drug for treatment against glioblastoma. Subsequently, temozolomide chemotherapy became widely used after randomized controlled trials demonstrated the efficacy of combination therapy with temozolomide and irradiation for glioblastoma, as well as maintenance therapy. However, temozolomide chemotherapy has limited survival benefit.

From December 2019 to April 2023, in cancer genomic medicine at a Japanese national university, new therapies for total of 2991 patients with malignances have been considered by cancer genome testing (Ncc oncopanel test: 679 cases, F1CDx test: 2312 cases). The new therapies for total of 43 advanced and recurrent glioblastomas, including Japanese pediatric patients, were considered by cancer genomic testing. Cancer genomic testing revealed an ATRX pathogenic variant in 32 advanced or recurrent glioblastoma cases. However, ATRX is one of the chromosomal remodeling factors, and antitumor drugs against ATRX pathogenic variants have not been developed.

In Japan, the treatments with DCVax-L for advanced and recurrent glioblastomas was not approved by the Ministry of Health, Labor and Welfare. The efficacy of DCVax-L therapy for advanced/recurrent glioblastoma reported by Dr. Liau et al. have had a major impact on the treatment of advanced and recurrent glioblastoma in Japan. In order to clarify the efficacy of DCVax-L treatment for advanced and recurrent glioblastoma in Japanese, a clinical study with a large cohort should be conducted in Japan in the future.

Glioblastoma, previously known as glioblastoma multiforme (GBM), is the most aggressive and most common type of cancer that originates in the brain, and has very poor prognosis for survival. Initial signs and symptoms of glioblastoma are nonspecific. They may include headaches, personality changes, nausea, and symptoms similar to those of a stroke. Symptoms often worsen rapidly and may progress to unconsciousness.

We do not have potential conflicts of interest.

Published in JAMA Oncology on June 06, 2023 by Kyoto@takumaH

Doctor specializing in cancer Medicine.
Doctor specializing in emerging infectious diseases.