Glioblastoma multiforme is one of the most common and difficult-to-treat adult brain tumors. With an estimated 10,000 annual new diagnoses in the United States and a near 100% recurrence rate, safe and effective brain-penetrant therapies are essential to combat this disease. However, developing drugs that effectively treat GBM has proved exceedingly challenging to date.
The current standard of care for GBM, surgical resection followed by radiation therapy and temozolomide, is largely ineffective, with 95% of patients suffering a relapse. Furthermore, temozolomide has been the only first-line approved anti-GBM drug in the last 20 years, with limited efficacy and a high rate of resistant patients. Additionally, between 1983 and 2020, only 4 out of 161 orphan drugs were approved for GBM treatment, as well as one implant.