At the moment, there are limited treatment options for glioblastoma, a rare form of brain cancer. While surgery, radiation, chemotherapy, and electric-field therapy are all utilized in the fight against glioblastoma, these tumors are difficult to treat—and many of these therapies cannot be used in pediatric patients. Identifying novel treatments is crucial to saving lives.

A potential therapy currently being explored for pediatric glioblastoma is gallium maltolate. Gallixa explains that gallium maltolate shows anti-tumor properties; the therapy has the potential to stop cancerous cells from reproducing. It can also cross the blood-brain barrier, allowing targeted treatment for brain cancers like glioblastoma. 

According to OncLive, the U.S. Food and Drug Administration granted Orphan Drug designation to gallium maltolate for the potential treatment of pediatric glioblastoma. The therapy also received Orphan Drug designation earlier this year for the treatment of glioblastoma in adults. This designation, which comes with incentives such as fee waivers and seven years of market exclusivity upon drug approval, is granted to therapies intended to treat, diagnose, or prevent rare conditions. The designation comes following preclinical results which suggest that gallium maltolate more than doubles survival rates in pediatric patients compared to those not treated. 

Researchers are also exploring the safety, efficacy, and tolerability of gallium maltolate for relapsed or refractory glioblastoma in a Phase 1 clinical study (although this is in adults). Participants will receive either 500mg daily or every other day, or 1000 or 1500mg daily, of gallium maltolate. Researchers seek to understand the maximum tolerated dose, as well as how the treatment improves survival rates. 

About Glioblastoma

Classified as a stage IV astrocytoma, glioblastoma is a highly aggressive brain tumor that is capable of making its own blood supply. An estimated 20% of all brain tumors are glioblastoma, but this percentage is much smaller in children. These tumors are more common in men than women. Additional risk factors include prior radiation therapy or certain genetic disorders. 

About 500 children in the United States are diagnosed with glioblastoma each year. Symptoms can include:

• Nausea and vomiting
• Double or blurred vision
• Speech and memory difficulties
• Headache
• Drowsiness
• Mood changes
• Weakness on one side of the body
• Seizures