Oral mucositis is a side effect of radiation therapy to the head and neck. Symptoms of oral mucositis include dry mouth, thick saliva, mouth ulcers, bleeding, difficulty swallowing, talking, eating, and more.1 It can severely impact the quality of life of a patient undergoing cancer treatment. There is currently no official FDA-approved treatment for oral mucositis.
Check out this video to understand how to manage oral mucositis when it happens in a patient:
RRx-001 is a hypoxia (lack of oxygen) activated small molecule that leads to the displacement of nitric oxide, which ultimately inhibits inflammation. It also serves the purpose of stressing tumor cells, which is a double win-win for patients undergoing treatment for cancer with oral mucositis.2
This novel drug was a part of a Phase 2 randomized, multi-institutional trial where patients received varying frequencies of infusions of the drug along with a steroid or patients receiving standard of care for oral mucositis. They saw a similar adverse event profile across those being treated with RRx-001 and standard of care, with no severe adverse event profile being attributed to the drug itself.
In this small patient population (53 patients), RRx-001 showed a reduction of oral mucositis duration relative to the control. There is no evidence of a dose-related response on oral mucositis and RRx-001, meaning that administering more of the drug does not improve symptoms, which is surprising and unexpected. Some may argue that this can question if the drug is truly effective, especially considering the small patient cohort of this study. However, that is unlikely since there is a true biological mechanism by which RRx-001 works in reducing oral mucositis symptoms.
Overall, this study found that RRx-001 was a fairly safe drug to administer in cancer patients; further studies will be highly anticipated, especially in a larger patient population.
1 Bell A, Kasi A. Oral Mucositis. [Updated 2022 Jun 10]. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2022 Jan-. Available from: https://www.ncbi.nlm.nih.gov/books/NBK565848/
2 PREVLAR: Phase 2a randomized trial to assess the safety and efficacy of RRx-001 in the attenuation of oral mucositis in patients receiving head and neck chemoradiotherapy M. Bonomi, D. M. Blakaj, R. Kabarriti, K. Colvett, V. Takiar, M. Biagioli, et al. International Journal of Radiation Oncology, Biology, Physics DOI: 10.1016/j.ijrobp.2022.12.031 https://doi.org/10.1016/j.ijrobp.2022.12.031