Patient with recurrent grade 4 astrocytoma responding favorably to intranasal delivery of NEO100, highly pure perillyl alcohol: illustrative case.

backgroundBetter treatments are needed for patients with grade 4 gliomas, especially in the setting of recurrence. The authors are developing NEO100, a clinical-grade version of the natural monoterpene perillyl alcohol, as a cancer therapeutic to be administered via intranasal (IN) delivery to patients with recurrent glioma. OBSERVATIONS: A 40-year-old woman was diagnosed with an isocitrate dehydrogenase 1-mutant, CNS WHO grade 4 astrocytoma harboring an unmethylated MGMT promoter. She underwent surgery and standard chemoradiation treatment with temozolomide (TMZ), but after 6 cycles of adjuvant TMZ, the tumor recurred. The patient was started on daily IN NEO100 at 288 mg 4 times a day, administered using a nebulizer and nasal mask. Routine MRI revealed steady tumor regression over the course of 13 months of daily IN NEO100, to the point where the tumor became inconspicuous. There were no serious adverse events, and her quality of life improved and remained high. LESSONS: The authors present a case in which IN cancer therapy with NEO100 was well tolerated and was associated with striking tumor regression, providing further evidence that this novel conceptual approach to cancer therapy might become useful for the improved treatment of recurrent glioma. https://thejns.org/doi/10.3171/CASE24683.