An update on pharmacotherapies in active development for the management of cancer regimen-associated oral mucositis

ABSTRACT

Introduction

Oral mucositis (OM) is a common toxicity of cytotoxic cancer regimens and remains one of the most painful, injurious, and treatment-disrupting side effects of radiation and ablative therapy. Despite its frequency and impact, approved definitive preventive or therapeutic options remain limited.

Areas covered

This review focuses on mechanistically active small molecules and biologicals that are under clinical development for the prevention of oral mucositis. The authors have excluded those compounds and devices for which their indication is limited to symptoms management.

Expert opinion

OM remains a significant unmet clinical need. The commercial opportunities for an effective treatment have been characterized as a global market of $1 billion (US). The formulations of drugs under development vary considerably but share some several commonalities in their development scheme. All new agents are given prophylactically and are being evaluated in patients treated with concomitant chemoradiation therapy for head and neck cancer. The primary efficacy outcome for most trials is dependent on the assessment of mucositis using the scoring scale established years ago by the World Health Organization. The development activity for OM is robust with a diverse range of agents. New drug applications for effective therapeutic options for OM should be ready for review within the next few years.

KEYWORDS:

• Oral mucositis
• new agents
• chemotherapy
• radiation
• toxicity

Article Highlights

1. Oral mucositis is a common painful and costly complication of many cytotoxic cancer regimens.

2. Despite its frequency and impact, there are few approved definitive preventive or treatment options.

3. Several agents or biologicals are under active clinical development. These drugs are administered prophylactically, and all are being evaluated in patients being treated with concomitant chemoradiation therapy for oral cavity or oropharyngeal squamous cell carcinomas.

4. A variety of studies identify genomic features that define risk for oral mucositis.

5. As more variables (metabolomics, proteomics, microbiomics, etc.) are added to this analytic mix, the accuracy and consistency of oral mucositis risk prediction are likely to improve.

Declaration of interest

S Sonis and A Villa are employees of Primary Endpoint Solutions, LLC. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

Reviewer Disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Additional information

Funding

This manuscript has not been funded.