Phase II investigational oral drugs for the treatment of radio/chemotherapy induced oral mucositis

ABSTRACT

Introduction: Oral mucositis is a significant unmet clinical need for many cancer patients. The biological complexity of mucositis’ pathogenesis provides a number of mechanistic targets suitable as pharmacologic targets. The diversity of targets has stimulated drug development in search of an effective intervention. In this paper, we review a range of agents that are currently being evaluated.

Areas covered: Drugs for management of oral mucositis vary in formulation, route of administration and biological target. Most propose to interrupt the initiation of injury by suppressing activation of the innate immune response or countering oxidative stress, or minimizing downstream inflammatory responses. Overwhelmingly, the population most studied is patients being treated with concomitant chemoradiation for cancers of the head and neck as this is the cohort that most consistently suffers severe mucositis for long periods of time. The Phase 2 pipeline is robust. Preliminary data reported for a number of agents is optimistic. Genomics may be important in interpreting and comparing responses to agents across widely demographically diverse populations.

Expert opinion: Oral mucositis remains a significant toxicity for patients undergoing cancer treatment. Incremental reports of successes have been noted for a number of targeted agents.

KEYWORDS:

• Oral mucositis
• biological targets
• phase 2 studies

Article highlights

• Oral mucositis remains a severe complication of cancer treatment with high morbidity for the patients

• Palifermin is approved for the prevention/treatment of oral mucositis associated with conditioning regimens in preparation for HSCTs

• Several palliative agents including rinses and gels may provide palliative benefit but are generally ineffective in mechanistically impacting the course of mucositis

• Several Phase II clinical trials on new agents for mucositis seem promising for the management of this condition

This box summarizes key points contained in the article.

Declaration of interest

S. Sonis is an employee at Biomodels LLC and a partner at Primary Endpoint Solutions. 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.

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

Additional information

Funding

This paper was not funded.