Scientists Explore Common Vitamin as Potential Brain Cancer Treatment - Video

High-dose vitamin B3 may help strengthen the immune response against glioblastoma when added to standard treatment, according to early results from a small clinical trial, published in the Journal of Neuro-Oncology.

Glioblastoma is an aggressive brain cancer that is difficult to treat because tumour cells can spread into surrounding brain tissue. Standard treatment typically involves surgery followed by radiation and chemotherapy, but the cancer frequently returns.

Researchers at the University of Calgary are investigating whether high-dose niacin, a form of vitamin B3, can help overcome one of glioblastoma's major challenges: suppression of the immune system.

The approach was first supported by experiments in mice, where niacin treatment extended survival. Researchers subsequently launched a combined Phase I and Phase II clinical trial to assess safety, determine an appropriate dose and look for early evidence of treatment benefit.

The trial is testing controlled-release niacin alongside standard chemotherapy and radiotherapy. Researchers are particularly examining progression-free survival, which measures how long patients live without their cancer worsening.

The study had a predefined threshold for continuing treatment. Researchers planned to stop the trial if fewer than 20% more patients remained progression-free at six months compared with results from previous studies.

However, preliminary findings from the first 24 participants showed that 82% had no cancer progression at six months. Researchers report this was 28% higher than the benchmark from earlier studies.

The findings are considered encouraging but remain preliminary. The trial is small and is ultimately expected to include 48 participants, with final analysis expected by late 2026 or early 2027.

Researchers caution that niacin should not be considered a proven glioblastoma treatment based on these early findings. High doses can cause adverse effects and may be harmful without medical supervision.

Larger trials will be needed to confirm whether the early signal translates into longer survival or better disease control.

REFERENCE: Gloria Roldan Urgoiti, et al.; A phase I-II study of niacin in patients with newly diagnosed glioblastoma: safety and interim phase II analysis. Journal of Neuro-Oncology, 2025; 176 (1) DOI: 10.1007/s11060-025-05351-z