Research indicates that a medication originally intended to slow down ALS progression is showing potential in hindering the self-renewing cancerous stem cells responsible for challenging existing treatments for aggressive grade 4 brain tumors.
Glioblastoma is the most prevalent and aggressive type of primary brain tumor found in adults. It is untreatable and incredibly aggressive, with patients facing a median survival time of only 18 months despite undergoing surgical removal and chemotherapy.
Recent innovative research headed by Dr. Arezu Jahani-Asl, a Canada Research Chair focused on Neurobiology of Disease at the University of Ottawa, presents robust evidence that the drug edaravone, which is used for ALS treatment, shows potential in inhibiting the cancer stem cells that currently pose a challenge to effective treatments for these deadly brain tumors.
The results from her team may significantly influence the fight against brain tumors, as brain tumor stem cells (BTSCs) are key players in the resistance to treatment and tumor recurrence seen in glioblastoma—a devastating form of cancer that affects approximately four individuals per 100,000 in Canada.
According to Dr. Jahani-Asl, finding effective methods to target these rare yet highly resistant cell populations could have a profound impact on global initiatives aimed at addressing brain cancer.
Here’s what the team at the University of Ottawa discovered: The drug edaravone (known by its brand name Radicava) can suppress the self-renewal and growth of brain tumor stem cells, indicating that repurposing this drug might become a powerful strategy against glioblastoma. Their findings were published in the journal Stem Cell Reports.
“We demonstrate that edaravone precisely targets cancer stem cells and works particularly well in conjunction with ionizing radiation,” she states. “The study implies that combining edaravone with radiation therapy can effectively eliminate cancer stem cells, potentially reducing the chances of therapy resistance and recurrence in glioblastoma patients.”
Ionizing radiation is utilized in cancer treatments to kill or hinder the growth of cancerous cells. The researchers plan to develop a protocol detailing the optimal dosage of edaravone when used alongside ionizing radiation and chemotherapy.
Repurposing existing drugs that have already been approved for human use is becoming a favored approach to tackle various cancer types. Drugs that have proven safe for use in humans could transition more quickly into clinical trials. Since edaravone is already an approved medication with a known safety profile, Dr. Jahani-Asl considers this repurposing for glioblastoma to be “very promising.”
In 2017, the U.S. Food and Drug Administration approved edaravone for ALS treatment, and in 2022, Health Canada sanctioned its oral formulation. It is also employed in stroke treatment.
Prior to the confirmatory study that illustrated how edaravone makes glioblastoma tumors more sensitive to radiation in animal models, the research team conducted a gene expression analysis on patient-derived brain tumor stem cells with and without the drug.
“Our analysis revealed significant changes in gene panels related to stemness and DNA repair mechanisms, which encouraged us to explore this new line of research,” explains Dr. Jahani-Asl.
Gaining deeper insights into the regulation of BTSCs is crucial in the fight against glioblastoma, with Dr. Jahani-Asl being a leading authority in this area. Her research initiatives at the Faculty of Medicine at uOttawa focus on creating innovative therapeutic approaches for severe brain disorders.
Any new treatment option for glioblastoma is anticipated to complement the current standard treatments, which include ionizing radiation and chemotherapy.
“Our objective now is to refine the dosage for a safe therapeutic range,” Dr. Jahani-Asl notes. “Once we determine a safe dose for use alongside other therapies, we will be well-prepared to advance this research into clinical applications.”
