Investigational drugs for the treatment of cancer cachexia: a focus on phase I and phase II clinical trials

Expert Opin Investig Drugs. 2019 Aug;28(8):733-740. doi: 10.1080/13543784.2019.1646727. Epub 2019 Jul 26.


Introduction: Cachexia is frequent in chronic diseases and especially during cancer development. Multiple definitions of cachexia have been proposed; it may be considered a multifactorial complex syndrome that presents with progressive unintentional weight loss and wasting of muscle mass and adipose tissue. Area covered: This article covers phase-I and phase-II clinical trials of investigational drugs for cancer cachexia. We performed a search on PubMed with keywords as cancer cachexia, phase-I/phase-II trial, drug, identifying articles relevant to this review. Studies were conducted using compounds, including anabolic agents such as ghrelin analogs, selective androgen receptor modulators, as well as anti-inflammatory drugs such as thalidomide, OHR, anti-interleukin antibody, cannabinoids, and omega-3 supplements. We also describe the mechanisms of action of these molecules and their phase-I and phase-II study design. The major outcomes were appetite stimulation, weight gain, improvement of muscle mass and function, modulation of inflammation, and quality of life. Expert opinion: The molecules discussed act on molecular pathways involved in cancer cachexia; they modulate appetite, anabolic effects, inflammation and direct interaction with muscle. Considering the multifactorial aspects of the cachexia syndrome, the combination of these drugs with metabolic and nutritional interventions may represent the most promising therapeutic approach to cancer cachexia.

Keywords: Cachexia; cachexia treatment; cancer; investigational drug; phase I clinical trial; phase II clinical trial.

Publication types

  • Review

MeSH terms

  • Appetite / drug effects
  • Cachexia / drug therapy*
  • Cachexia / etiology
  • Cachexia / physiopathology
  • Clinical Trials, Phase I as Topic
  • Clinical Trials, Phase II as Topic
  • Drugs, Investigational / pharmacology
  • Drugs, Investigational / therapeutic use*
  • Humans
  • Inflammation / drug therapy
  • Inflammation / pathology
  • Neoplasms / complications*
  • Nutritional Support / methods
  • Quality of Life
  • Research Design


  • Drugs, Investigational