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Table 1 Examples of repositioned drugs, their targets and indications*

From: Drug repositioning for personalized medicine

Drug name Original target Original indication New target New indication References
Successful repositionings from approved drugs
Duloxetine Serotonin and norepinephrine reuptake Depression Serotonin and norepinephrine reuptake Stress urinary incontinence, fibromyalgia, chronic musculoskeletal pain [12]
Everolimus mTOR Immunosuppressant Unchanged Pancreatic neuroendocrine tumors [20]
Imatinib BCR-ABL CML KIT, PDGFRA GIST [14]
Minoxidil Unknown Hypertension Unknown Hair loss [24]
Nelfinavir HIV-1 protease AIDS Inhibits AKT pathway In clinical trials for multiple cancers [17]
Sildenafil PDE5 Angina Unchanged Erectile dysfunction, pulmonary arterial hypertension [130]
Sunitinib Multiple kinases GIST, renal cell carcinoma Unchanged Pancreatic neuroendocrine tumors [131]
Trastuzumab HER2 HER2-positive breast cancer Unchanged HER2-positive metastatic gastric cancer [129]
Successful repositionings from investigational drugs
Crizotinib MET kinase Clinical trials for anaplastic large-cell lymphoma EML4-ALK oncogene NSCLC [7]
Thalidomide Unknown Morning sickness (withdrawn) Inhibits tumor necrosis factor α production Leprosy [132]
Thalidomide Unknown Morning sickness (withdrawn) Inhibits angiogenesis Multiple myeloma [132]
Zidovudine Reverse transcriptase Failed clinical trials for cancer Reverse transcriptase AIDS [73]
Unsuccessful repositionings
Bevacizumab VEGF Multiple cancers Unchanged Failed clinical trial for gastric cancer [124]
Buproprion Unknown Depression Synergistic inhibition of appetite and energy expenditure Obesity (rejected by FDA owing to adverse effects) [126, 127]
Naltrexone Opioid receptors Opioid addiction Synergistic inhibition of appetite and energy expenditure Obesity (rejected by FDA owing to adverse effects) [126, 127]
Naltrexone Unknown Alcohol dependence Synergistic inhibition of appetite and energy expenditure Obesity (rejected by FDA owing to adverse effects) [126, 127]
Sunitinib Multiple kinases GIST, renal cell carcinoma Multiple kinases Failed clinical trials for multiple cancers [125]
  1. *Drugs are divided into successful and unsuccessful repositionings. Within successful cases, drugs are further divided according to whether they were approved at their time of repositioning. For each drug, the original target and indication is listed, along with the new target and indication. In many cases, it can be seen that the new indication is still based on the same target protein. CML, chronic myeloid leukemia; GIST, gastrointestinal stromal tumor; NSCLC, non-small-cell lung cancer.