Repurpose terbutaline sulfate for amyotrophic lateral sclerosis using electronic medical records

Hyojung Paik, Ah Young Chung, Hae Chul Park, Rae Woong Park, Kyoungho Suk, Jihyun Kim, Hyosil Kim, Ki Young Lee, Atul J. Butte

Research output: Contribution to journalArticlepeer-review

33 Citations (Scopus)


Prediction of new disease indications for approved drugs by computational methods has been based largely on the genomics signatures of drugs and diseases. We propose a method for drug repositioning that uses the clinical signatures extracted from over 13 years of electronic medical records from a tertiary hospital, including >9.4 M laboratory tests from >530,000 patients, in addition to diverse genomics signatures. Cross-validation using over 17,000 known drug-disease associations shows this approach outperforms various predictive models based on genomics signatures and a well-known "guilt-by-association" method. Interestingly, the prediction suggests that terbutaline sulfate, which is widely used for asthma, is a promising candidate for amyotrophic lateral sclerosis for which there are few therapeutic options. In vivo tests using zebrafish models found that terbutaline sulfate prevents defects in axons and neuromuscular junction degeneration in a dose-dependent manner. A therapeutic potential of terbutaline sulfate was also observed when axonal and neuromuscular junction degeneration have already occurred in zebrafish model. Cotreatment with a β2-adrenergic receptor antagonist, butoxamine, suggests that the effect of terbutaline is mediated by activation of β2-adrenergic receptors.

Original languageEnglish
Article number8580
JournalScientific reports
Publication statusPublished - 2015

ASJC Scopus subject areas

  • General


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