Intranasal Delivery of Perillyl Alcohol (NEO100) as a New Treatment Strategy for Glioma

Background: Perillyl alcohol (POH) is a naturally occurring monoterpene that is being developed as an intranasally delivered agent for the treatment of brain-localized malignancies. Clinical trials with glioma patients in Brazil have yielded preliminary evidence that this approach might be able to achieve therapeutic activity and result in prolonged survival of patients. Methods: NEO100, a highly pure, current good manufacturing practice-produced version of POH, is being evaluated in a Phase I/IIa clinical trial with recurrent glioblastoma patients in the United States. Patients self-administer POH/NEO100 as a mist with a nasal mask over the course of 15 minutes, four times a day, every day. Results: The treatment regimen is well tolerated, even if maintained over several years. It correlated with improved survival when compared to historical controls. Conclusion: There is human data demonstrating that this novel approach could become useful for the treatment of malignant glioma.