High-Dose Niacin Shows Promise in Glioblastoma: Early Trial Reports 28% Improvement in Disease Progression
Key Insights
A Phase I/II clinical trial found that high-dose vitamin B3 (niacin) combined with standard therapy improved six-month progression-free survival to 82% in glioblastoma (search) patients, compared to a typical 54%.
The study enrolled 24 patients and demonstrated that niacin can restore function in weakened immune cells, enhancing their ability to target and destroy tumor cells.
Researchers at the University of Calgary caution that the small sample size, short follow-up, and lack of a randomized control group limit the findings, and larger controlled trials are needed.
High-dose vitamin B3 (niacin) may enhance immune activity and improve short-term disease control in glioblastoma (search) when added to standard therapy, according to early clinical findings published in the Journal of Neuro-Oncology. The study, led by researchers at the University of Calgary's Hotchkiss Brain Institute and Arnie Charbonneau Cancer Institute, reported that 82% of patients showed no disease progression at six months, compared to the typical 54% observed in previous studies — a 28% improvement.
Glioblastoma (search) is the most aggressive brain cancer in adults, with a median survival of roughly 12 to 18 months. Standard treatment typically includes surgery to remove as much of the tumor as possible, followed by radiation therapy and chemotherapy. Even with these interventions, the cancer frequently returns, and survival rates have not changed significantly in 20 years.
"Glioblastoma (search) is the most aggressive brain cancer in adults. Survival of patients with this condition hasn't changed significantly for 20 years," said lead study author Dr. Gloria Roldan Urgoiti, a clinical associate professor at the Cumming School of Medicine (CSM). "Anything that may help should be explored, but it requires strict protocols and safety monitoring."
Reawakening the Immune System
The research program, developed jointly by Roldan Urgoiti and neuroscientist Dr. Wee Yong, began in Yong's laboratory using mice, where early experiments showed that niacin extended survival. Those preclinical results prompted the team to launch a Phase I and II clinical trial in humans.
"Normally, the immune system will try to counter and prevent tumor growth; however, this brain cancer suppresses the immune system," said Yong, a professor at the CSM. "Niacin treatment rejuvenates immune cells so they can do what they are supposed to do: attack and kill the cancer cells. I see it as an ongoing 'battle for the brain.'"
The trial was designed to identify the highest safe dose and evaluate the potential benefits of controlled-release niacin when combined with standard chemotherapy and radiotherapy. Researchers established a benchmark before the study began: if progression-free survival at six months did not improve by at least 20% compared with previous studies, the trial would be stopped. The early findings from 24 patients exceeded that target.
Cautions and Limitations
Despite the promising early results, the researchers urged caution. The study's small sample size, short follow-up, and lack of a randomized control group posed significant limitations. Larger, controlled trials are needed to confirm the findings.
The researchers also emphasized that high doses of vitamins, including niacin, can be toxic and may cause harm if not carefully monitored by medical professionals. Dr. Marc Siegel, Fox News senior medical analyst who was not involved in the study, noted that niacin does have some side effects, including skin flushing. "There probably will end up being a small impact here, so this is useful information, though clearly not a cure," he added.
Next Steps
The study is continuing, and the team plans to enroll an additional 24 patients by the end of 2026 or early 2027 for the next phase of the trial, which will assess niacin's safety and immune system activation. The research is supported by the Canadian Institutes of Health Research and the Alberta Cancer Foundation.