Results of a Phase 1 Open-Label, Dose-Escalation Study of Gene Therapy With AAV2-hAQP1 for Grade 2 and 3 Radiation-Induced Late Xerostomia and Parotid Gland Hypofunction.
Int J Radiat Oncol Biol PhysMay 29, 2026 (epub)
Michael T Brennan, Michael J Passineau, Deborah Saunders et al.
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In 30 seconds
This Phase 1 open-label, multicenter study evaluated the safety and efficacy of AAV2-hAQP1 gene therapy in 24 patients with grade 2 and 3 radiation-induced late xerostomia. The treatment was well-tolerated, with no serious adverse events reported. At 12 months, participants showed significant improvements, including a mean percent change from baseline in unstimulated whole saliva flow rates of 112.5%.
Key findings
- At month 12, the mean percent change from baseline in XQ Total Score was -39.5%.
- 67% of participants reported an improvement of ≥8 points in XQ Total Score.
- The mean GRCQ-Symptom Change Score at month 12 was 3.8.
- The mean percent change from baseline in UWSFR at month 12 was 112.5%.
Why it matters
The study highlights a potential new treatment for chronic xerostomia, a common and debilitating side effect of head and neck cancer radiation therapy, which can significantly impact patients' quality of life.