Phase II drugs that are currently in development for the treatment of cachexia

Anne-Marie C. Dingemans*, Judith de Vos-Geelen, Ramon Langen, Annemie M. W. Schols

*Corresponding author for this work

Research output: Contribution to journalArticleAcademicpeer-review


Cachexia is a syndrome presenting with progressive unintentional weight loss and wasting and weakness of skeletal muscle. Cachexia is prevalent in cancer and in chronic diseases including chronic obstructive pulmonary disease (COPD). Areas covered: The authors searched trial registers for current Phase II clinical trials on cachexia. Twelve studies were found with 11 compounds, including the anti-inflammatory drugs thalidomide, OHR/AVR118, celecoxib, VT-122, omega-3 supplements, and anabolic agents such as ghrelin analogues, MT-102, BYM338 and ruxolotinib. The authors note that one of the studies related to COPD while the others were related to different cancers. Herein, the authors describe the mechanisms of action and their Phase II study design. Expert opinion: The compounds under study affect several pathways involved in cachexia by modulating inflammatory activity, anabolic potential, digestion and direct interaction with the muscle. Due to the multifactorial aspects of cachexia syndrome, combinations of these new drugs with nutritional intervention is probably the most promising approach. Furthermore, future studies should include interventions in pre-cachetic patients, as this stage might be more responsive to treatment. Future studies will benefit from well-defined end points and improved measures of cachexia, providing new insight into the disease. This insight, in combination with the elucidation of cachexia's underlying mechanism, will yield new treatment strategies in the near future.
Original languageEnglish
Pages (from-to)1655-1669
JournalExpert Opinion on Investigational Drugs
Issue number12
Publication statusPublished - Dec 2014


  • appetite stimulants
  • body composition
  • cachexia
  • cancer
  • chronic obstructive pulmonary disease
  • cytokine antagonists
  • ghrelin
  • myostatin

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