Researchers from Calgary’s Hotchkiss Brain Institute and the Arthur J.E. Child Comprehensive Cancer Centre say adding high doses of niacin (Vitamin B3) to the standard treatment for glioblastoma may significantly delay disease progression. Their findings were published in the peer-reviewed Journal of Neuro-Oncology following positive results in an early-phase clinical trial. The Phase I–II study found that 82.3 per cent of patients who received niacin alongside surgery, radiation and chemotherapy were alive without their cancer progressing six months after treatment. According to the study, historical data shows about 54 per cent of patients typically reach that point without progression. Edward Waldner is one of the patients in the trial. He was diagnosed with glioblastoma in 2024 after seeking medical care following months of worsening fatigue and changes to his walking. “I could barely walk anymore, like I was stumbling, dragging my heels – and just tired,” Waldner said. “I did a CAT scan in Lethbridge, and the doctor came back and he entered the room, and I knew it was bad. He told me what’s going on, and he said you’ve got to go to Calgary [for further treatment], you have a big mass on your brain.” Doctors at the Arthur J.E. Child Comprehensive Cancer Centre in Calgary diagnosed Waldner with glioblastoma, an aggressive and typically fatal cancer. Waldner agreed to participate in the niacin trial after undergoing surgery, at a point when standard treatment options were limited. “We instantly jumped on and said, ‘Why not. Let’s help out with the study,’” he said. “If it helps me, it can help somebody else – and I think it’s helping me.” Dr. Gloria Roldan Urgoiti is a Calgary oncologist and co-authour of the study. “Glioblastoma is the most aggressive form of brain cancer in adults and is considered incurable,” she said. “The first treatment is always surgery, but in the brain, the surgery is limited by the function of the brain. So, the surgeon does the most extensive surgery that it can be done, depending on the location of the tumor, and after that, we do oral chemotherapy.” Roldan Urgoiti says the cancer eventually progresses. A diagnosis is considered fatal. “We hope that adding niacin in this dose … Prolongs the time to progression and maybe also prolongs survival.” Waldner’s wife, Leslie Masuk, says taking part in the research has helped their family cope with a diagnosis that often comes with bleak expectations. “This drug study has been a game changer for our whole family,” she said. “It offers hope and positivity. We feel like we’re being we’re doing something for the glioblastoma community.” Masuk said it has given them hope for Waldner – and for future patients. “It gives you hope when there’s hopelessness,” she said. “Not too many people get to say that, that you’re helping science.” The study involved patients aged 18 to 75 with newly diagnosed glioblastoma. Participants received controlled-release niacin in addition to standard treatment. Researchers gradually increased the niacin dose to determine the highest amount patients could safely tolerate. “We have to emphasize here that we are using niacin here, not as a vitamin, but as a medication,” said Hotchkiss Brain Institute researcher Wee Yong. “So, in that regard, we escalated the dose of niacin so that it can now be used as a therapeutic. So for all drugs, there can be potential side effects if used in an uncontrolled manner.” According to the study, the maximum tolerated dose was 2,000 milligrams per day. Higher doses caused serious side effects including liver toxicity and dangerously low platelet counts, leading researchers to stop further dose escalation. Beyond delaying progression at six months, the interim analysis showed patients had a median progression-free survival of 10 months, meaning half of the participants went at least that long before their cancer worsened. The study showed median overall survival was 17 months, compared to about 15 months typically seen with standard treatment alone. Researchers also examined outcomes based on a genetic marker known to influence prognosis. MGMT is a gene involved in DNA repair that can affect how well tumours respond to chemotherapy. According to the study, patients whose tumours lacked MGMT promoter methylation, a group that usually responds more poorly to treatment, still showed progression-free survival rates of nearly 78 per cent at six months. The study builds on earlier laboratory research showing that niacin, also known as vitamin B3, can stimulate immune cells in the brain. According to the researchers, glioblastoma suppresses the immune system, limiting the body’s ability to attack tumour cells. “Our aim then was to discover medications that could rejuvenate the compromised immune system, so that the immune system can now defeat the tumor,” Yong said. “After screening over 1,000 potential medications, we discovered that niacin was effective in rejuvenating immune cells.” Researchers emphasized the findings are preliminary and say they should not be interpreted as proof that niacin improves long-term survival. The study was designed with a planned second phase to determine whether the early improvement seen at six months holds up in a larger group of patients, and to better assess safety over time. To do that, the research team is continuing to recruit patients with newly diagnosed glioblastoma through Alberta cancer centres, including sites affiliated with the University of Calgary and University of Alberta. The trial aims to enroll a total of 48 patients, a number set in advance to allow meaningful comparison with historical treatment outcomes. Final enrolment is expected by late 2026 or early 2027, after which a full analysis will be conducted. Waldner said he feels good now and remains active. “I feel really good,” he said. “I feel 100% physically. I go to the gym. I workout a lot. I try to not to think too negative.” The research is supported by the Canadian Institutes of Health Research and the Alberta Cancer Foundation.
