Drug used to slow ALS progression shows promise in treating deadly brain cancer

An old drug could bring new hope for people suffering from the most common, and deadly, primary brain tumour in adults.

uOttawa researcher Dr. Arezu Jahani-Asl, who holds a Canada Research Chair in Neurobiology of Disease, leads a team that has found a drug currently used to slow progression of the neurological disorder ALS (amyotrophic lateral sclerosis) shows promise in treating glioblastoma.

Jahani-Asl’s team found evidence that the drug — called edaravone — shows promise in suppressing the self-renewing cancerous stem cells that challenge treatment of glioblastoma. Those brain tumour stem cells are at the core of treatment resistance and tumour recurrence in patients with glioblastoma.

Finding ways to target those cells could have major implications for global efforts to combat brain cancer, said Jahani-Asl. Her team reported on the findings in the journal Stem Cell Reports.

Patients diagnosed with the aggressive form of cancer have an average survival rate of about two years, something that has not improved significantly in recent years despite progress in understanding the tumour.

Tragically Hip front man Gord Downie, who died in 2017, and former Ottawa Centre NDP MP Paul Dewar, who died in 2019, both put a face to the disease that affects about four per 100,000 people in Canada, according to the Brain Tumour Foundation of Canada. It affects more men than women and has a peak incidence of between the ages of 45 and 75.

Jahani-Asl said her research shows that edaravone specifically targets cancer stem cells “and is particularly effective in combination with ionizing radiation. The study suggests that edaravone, given in combination with ionizing radiation can be effective in eradicating cancer stem cells, and thus is expected to decrease the chance of resistance to therapy and recurrence in glioblastoma patients.”

That could extend patients’ survival rates, although Jahani-Asl said it is too soon to know the real-world impact of the drug.

Standard treatment for glioblastoma involves surgery followed by ionizing radiation and chemotherapy. Because of the nature of the tumours — which are diffuse and have finger-like tentacles that typically infiltrate the brain — surgery cannot remove the entire tumour. The goal of the ionizing radiation and chemotherapy is to kill the remaining tumour cells, she said.

The promising findings have been seen in animal models. The next step, said Jahani-Asl, is finding the best ways to optimize drug dosage in combination with ionizing radiation and chemotherapy.

Testing drugs for human use is typically slow and extremely costly. Repurposing drugs, such as the one being tested in Jahani-Asl’s lab can significantly speed up that advancement from research to clinical trials because the drugs have already been approved for human use. It is one reason there has been a growing interest in trying to find new uses for existing drugs, especially when it comes to battling cancer.

“We think it is very promising,” she said. “All I can say is it would take less time and effort than a newly developed drug, but I can’t give you a time line.”

Jahani-Asl’s research program at the uOttawa Faculty of Medicine is focused on developing novel therapeutic strategies for complex and devastating brain diseases.

Jahani-Asl said she has been moved by the glioblastoma patients and family members she has met.

“This is such a deadly disease. It is almost a death sentence. It is a heartbreaking situation for any family.”

She said her research has received donations from patients’ families who want to see the outlook for patients improve. “It has been very touching.”