Small molecule drugs with immunomodulatory effects in cancer

Research output: Contribution to journalArticle


Immunotherapy shows promise for positively changing the landscape ofthe management of many advanced solid tumors, including gastrointestinal (GI) malignancies. Many of these developments have been focused on vaccinebased, monoclonal antibody therapies and more recently, checkpoint inhibitors, although many small molecule inhibitors can function as immunomodulators. Small molecule compounds have several advantages over conventional immunotherapeutic agents including: ease of production and the potential for oral administration. There is a potential niche for small molecule immunomodulators to enhance the efficacy of existing immunotherapeutic and cytotoxic agents. This article focuses on two categories of small molecule compounds with immunomodulatory effects: IDO and MEK inhibitors. Indoleamine-2, 3-dioxygenase (IDO) is known for its effects in tumor immunity. IDO inhibitors are generally well-tolerated and have the potential to enhance anti-tumor responses when combined with checkpoint inhibitors. MEK inhibitors affect signal transduction of the RAS-RAF-MEK pathway and numerous MEK inhibitors are currently being investigated in solid tumors. Small molecule immunomodulators are currently being investigated for their potential role in augmenting the effects of conventional immunotherapeutic agents although further research is required to identify those patients most likely to respond to combination therapy.

Original languageEnglish (US)
Pages (from-to)2463-2468
Number of pages6
JournalHuman Vaccines and Immunotherapeutics
Issue number10
StatePublished - Jan 1 2015
Externally publishedYes


  • Gastrointestinal malignancies
  • IDO inhibitors
  • Immunotherapy
  • MEK inhibitors
  • Small molecule

ASJC Scopus subject areas

  • Immunology and Allergy
  • Immunology
  • Pharmacology

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