admin Imagine treating cancer with just one pill a day. Right now, a research team at the University of Alberta is working to turn that vision into reality.
Professor Luc Berthiaume has spent decades searching for a way to fight cancer with minimal side effects.
“I came here to find a new way to kill cancer cells while reducing harm to the body,” he explained.
Years ago, Berthiaume proposed an unconventional approach to cancer treatment—one that was met with scepticism during his graduate studies at the Université de Sherbrooke in Quebec. But today, clinical trials are proving that his theory works by targeting mitochondria within cancer cells.
“It inhibits the signalling within cells that causes abnormal, uncontrolled growth,” he said. “Not only does our drug stop tumour growth, but it also suffocates cancer cells, starving them of energy—an effect that is far more pronounced in cancerous cells than in healthy ones.”
His groundbreaking treatment disrupts the cell’s metabolism, preventing it from converting oxygen into energy—potentially saving lives.
“It’s the spread, or metastasis, of cancer that is the primary cause of cancer-related deaths,” explained PhD candidate Rony Pain, who is assisting in Berthiaume’s lab.
A Pill That’s Changing Lives
The theory was first tested on cells, then in mice, and eventually in cancer patients with only months to live.
“Our pill, Zelenirstat, can be taken at home—no hospital visits, no injections, just one easy-to-take pill,” Berthiaume said. “The compound is water-soluble, enters the bloodstream quickly, and directly targets tumours.”
The first patient to take the pill was treated at the Cross Cancer Institute in Edmonton. Since then, the trial has expanded to other leading Canadian cancer centres, including the BC Cancer Care Centre, Princess Margaret Hospital in Toronto, and the Centre Hospitalier de l’Université de Montréal.
The Phase 1 clinical trials, designed to assess the safety of the drug, have already yielded remarkable results.
“We had a lymphoma patient whose tumour shrank by 30% in just two to three weeks of treatment. That was a huge breakthrough—it confirmed we were on the right track,” Berthiaume said.
One patient, initially given only months to live, survived for 18 months while taking the pill.
“The Phase 1 trial not only demonstrated that the drug was safe but also showed clear signs of effectiveness,” he added.
Fast-Tracking a Life-Saving Treatment
Zelenirstat’s success has attracted international attention, including from the US Food and Drug Administration (FDA).
“We’ve been granted fast-track designation as an orphan disease treatment by the FDA,” Berthiaume said. “This means that once we complete Phase 2, we could begin commercialising and distributing the drug without needing to go through Phases 3 and 4.”
The drug has shown the most promise in treating blood cancers, particularly lymphoma and acute myeloid leukaemia. However, it may also be effective against aggressive solid tumours in the brain, lungs, and ovaries.
Pain’s PhD research is exploring how Zelenirstat could benefit patients with triple-negative breast cancer—a particularly aggressive form that lacks the usual receptors targeted by traditional treatments.
Pain has been working on the project for four years and is excited by the drug’s potential.
A Gentler Alternative to Traditional Treatments
Unlike chemotherapy and radiation, which often come with debilitating side effects, Zelenirstat is designed to be easier on the body.
“One of the most exciting things about our drug is that it’s just one pill a day with minimal side effects,” Berthiaume said.
However, before Zelenirstat can reach more patients, it must undergo Phase 2 clinical trials—a process that requires significant funding.
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