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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Morning sickness (withdrawn)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Morning sickness (withdrawn)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.