A study led by two researchers, both hailing from the Hotchkiss Brain Institute and Arnie Charbonneau Cancer Institute, has revealed promise for the common and inexpensive vitamin supplement, niacin (B3), in treatments for the cancer glioblastoma — a condition whose treatment has hardly budged since the 1970s.
Glioblastoma is an aggressive form of brain cancer that has garnered the reputation of being notoriously difficult to treat through standard practices such as surgery, radiation, and chemotherapy. While as much of the tumor as possible is attempted to be removed, the ability for cells to spread into surrounding brain tissue makes complete removal extremely difficult. Even after difficult treatment, it is common for the cancer to return.
At the University of Calgary, Dr. Voon Wee Yong, PhD, a neuroscientist part of the recent study, became aware of the potential treatment avenue during an earlier series of studies on how the immune system affects the brain. Screening more than a thousand compounds in search of the one that could “wake up” the immune cells, he noticed how niacin stood out — mice with brain tumors lived longer with the treatment than the ones who didn’t receive it. This success was promising enough to encourage Yong to move on to a study involving human subjects in notable collaboration with Gloria Roldan Urgoiti, MD, an oncologist specializing in brain cancers.
The goal of this new study was to determine whether the vitamin could be used to help restore the dampened immune cells in order to potentially fight the tumor cells by controlling niacin release alongside the recommended chemotherapy and radiotherapy. One of the first things the team needed to find was the highest dose that could be safely administered before side effects became a problem — finding 2,000 milligrams a day to be the ceiling. While the most common side effect complaint was flushing (warm, reddened skin the vitamin is known for and usually mild), at higher doses the team ran into more serious blood-related side effects. With this ceiling, the study shifted to looking for evidence that niacin may improve the lifetime of patients without worsening the cancer (progression-free survival). The researchers pledged to stop the trial if the progression-free survival failed to improve by at least 20% compared to older studies.
From the 24 patients, the 20% threshold was surpassed when, after six months, 82% of participants had not experienced a progression of their cancer — representing a 28% improvement compared to previous studies, according to the researchers. The team sees these early findings as promising for niacin’s effectiveness against the incurable cancer, though results are still preliminary and involve a relatively small group of patients. Looking toward the future, they hope to expand the study with 48 participants and conduct their final analysis by the end of 2026 or in early 2027.
Roldan Urgoiti, a clinical associate professor at the Cumming School of Medicine at the University of Calgary, commented on these early results: “Glioblastoma is the most aggressive brain cancer in adults. Survival of patients with this condition hasn’t changed significantly for 20 years.” She continued, “Anything that may help should be explored, but it requires strict protocols and safety monitoring.”
