Table 2.
Effects of exogenous leptin replacement or leptin antagonism in animal models and in human diseases
Leptin agonism
BackgroundFeaturesClinical outcomeReference
Mouse 
ob/ob Obesity, excessive food intake, infertile Decrease of body weight, food intake, serum insulin and glucose level; normalization of body fat percentage; increase of total activity and lean mass Pelleymounter et al., 1995; Halaas et al., 1995  
db/db Obesity, excessive food intake, infertile, hyperglycemia Not responding Halaas et al., 1995; Farooqi et al., 1999  
Human 
Congenital leptin deficiency Early-onset obesity, hyperphagia, alteration of the immune function Weight loss, decreased basal metabolic rate, increased physical activity level, increased basal and stimulated serum gonadotropin concentration Farooqi et al., 1999  
Acquired leptin deficiency (lipodystrophy) Generalized lack of body fat, insulin resistance, hypertriglyceridemia, polycystic ovary syndrome Decreased average triglyceride level, glycosylated hemoglobin, and plasma glucose level; improved metabolic control Oral et al., 2002; Santos and Cortés, 2020  
Mutation in the LEPR gene Early-onset obesity, hyperphagia, impaired pubertal development, reduced secretion of growth hormone and thyrotropin Not responding (other possible therapies: bariatric surgery or setmelanotide treatment) Clément et al., 1998  
General obesity Dysmetabolic syndrome, leptin resistance Not responding  
Leptin antagonism (rodent studies only) 
Pathology Antagonizing molecule Clinical outcome Reference 
Inflammation and Autoimmunity 
Chronic liver fibrosis Mutated leptin Reduced IFN-γ levels, attenuated liver fibrosis, improved survival Elinav et al., 2009  
Chronic experimental colitis Mutated leptin Reduced systemic and mucosal pro-inflammatory cytokines and clinical severity; increased T reg cell number Singh et al., 2013  
EAE Neutralizing leptin antibody or soluble LepR chimera Slowed disease progression, reduced relapses, FOXP3+CD4+ T cell induction and pro-inflammatory T cell proliferation blockade De Rosa et al., 2006  
SLE Neutralizing leptin antibody Hampered pro-inflammatory Th17 cell response Yu et al., 2013  
Cancer 
Aggressive breast cancer LepR antagonist Increased average survival time Otvos et al., 2011a  
Acute myelocytic leukemia Neutralizing LepR monoclonal antibody Decreased leukemic cell number and angiogenesis within the bone marrow Iversen et al., 2002  
Leptin agonism
BackgroundFeaturesClinical outcomeReference
Mouse 
ob/ob Obesity, excessive food intake, infertile Decrease of body weight, food intake, serum insulin and glucose level; normalization of body fat percentage; increase of total activity and lean mass Pelleymounter et al., 1995; Halaas et al., 1995  
db/db Obesity, excessive food intake, infertile, hyperglycemia Not responding Halaas et al., 1995; Farooqi et al., 1999  
Human 
Congenital leptin deficiency Early-onset obesity, hyperphagia, alteration of the immune function Weight loss, decreased basal metabolic rate, increased physical activity level, increased basal and stimulated serum gonadotropin concentration Farooqi et al., 1999  
Acquired leptin deficiency (lipodystrophy) Generalized lack of body fat, insulin resistance, hypertriglyceridemia, polycystic ovary syndrome Decreased average triglyceride level, glycosylated hemoglobin, and plasma glucose level; improved metabolic control Oral et al., 2002; Santos and Cortés, 2020  
Mutation in the LEPR gene Early-onset obesity, hyperphagia, impaired pubertal development, reduced secretion of growth hormone and thyrotropin Not responding (other possible therapies: bariatric surgery or setmelanotide treatment) Clément et al., 1998  
General obesity Dysmetabolic syndrome, leptin resistance Not responding  
Leptin antagonism (rodent studies only) 
Pathology Antagonizing molecule Clinical outcome Reference 
Inflammation and Autoimmunity 
Chronic liver fibrosis Mutated leptin Reduced IFN-γ levels, attenuated liver fibrosis, improved survival Elinav et al., 2009  
Chronic experimental colitis Mutated leptin Reduced systemic and mucosal pro-inflammatory cytokines and clinical severity; increased T reg cell number Singh et al., 2013  
EAE Neutralizing leptin antibody or soluble LepR chimera Slowed disease progression, reduced relapses, FOXP3+CD4+ T cell induction and pro-inflammatory T cell proliferation blockade De Rosa et al., 2006  
SLE Neutralizing leptin antibody Hampered pro-inflammatory Th17 cell response Yu et al., 2013  
Cancer 
Aggressive breast cancer LepR antagonist Increased average survival time Otvos et al., 2011a  
Acute myelocytic leukemia Neutralizing LepR monoclonal antibody Decreased leukemic cell number and angiogenesis within the bone marrow Iversen et al., 2002  

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