ALS drug shows promise in targeting glioblastoma stem cells

Glioblastoma is the most common – and the most malignant – primary brain tumour in adults. It's aggressive and incurable. Even with treatment including surgical removal and chemotherapy, the median survival for patients is just 18 months.

"We show that edaravone specifically targets cancer stem cells and is particularly effective in combination with ionizing radiation," she says. "The study suggests that edaravone 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."

Repurposing existing marketed drugs already approved for human use is an increasingly popular strategy of battling different types of cancer cells. And compounds that have already been proven safe could potentially advance swiftly to clinical trials. Since edaravone is an approved compound that has already been shown safe in humans, Dr. Jahani-Asl says repurposing the drug and its translation for glioblastoma is "very promising."

Edaravone was approved by the U.S. Food and Drug Administration in 2017 to treat ALS, and Health Canada approved the drug's oral formulation in 2022. It's also used to treat stroke.

"We found significantly deregulated gene panels related to stemness and DNA repair mechanisms which was the spark to begin a new major line of investigation," Dr. Jahani-Asl says.

Achieving new insights into BTSC regulation is at the forefront of efforts to battle glioblastoma, and Dr. Jahani-Asl is an international leader in this effort. Her research program at the uOttawa Faculty of Medicine is centered on developing novel therapeutic strategies for complex and devastating brain diseases.

Any new drug for glioblastoma is expected to be used in combination with the present standard treatments which includes ionizing radiation and chemotherapy.

"Our goal is now to try to optimize dosage for a safe therapeutic window," Dr. Jahani-Asl says. "Once we establish a dose that is safe for use in combination therapy, we will be well equipped with the knowledge to move this forward to clinic."

Source:

University of Ottawa