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.