Repurposing the Selective Oestrogen Receptor Modulator Tamoxifen for the Treatment of Duchenne Muscular Dystrophy

Authors

  • Elinam Gayi School of Pharmaceutical Sciences, University Medical Center University of Geneva, CMU 5-6 Rue Michel-Servet 1, Geneva, Switzerland
  • Laurence A. Neff School of Pharmaceutical Sciences, University Medical Center University of Geneva, CMU 5-6 Rue Michel-Servet 1, Geneva, Switzerland
  • Hesham M. Ismail School of Pharmaceutical Sciences, University Medical Center University of Geneva, CMU 5-6 Rue Michel-Servet 1, Geneva, Switzerland
  • Urs T. Ruegg School of Pharmaceutical Sciences, University Medical Center University of Geneva, CMU 5-6 Rue Michel-Servet 1, Geneva, Switzerland
  • Leonardo Scapozza School of Pharmaceutical Sciences, University Medical Center University of Geneva, CMU 5-6 Rue Michel-Servet 1, Geneva, Switzerland
  • Olivier M. Dorchies School of Pharmaceutical Sciences, University Medical Center University of Geneva, CMU 5-6 Rue Michel-Servet 1, Geneva, Switzerland. olivier.dorchies@unige.ch

DOI:

https://doi.org/10.2533/chimia.2018.238

Keywords:

Duchenne muscular dystrophy, Oestrogens, Repurposing, Serm, Tamoxifen

Abstract

Drug discovery is a long, expensive and risky process. Evaluating drugs that have already been proved safe for use in humans and testing them for a new indication greatly reduces the time and monetary costs involved in finding treatments for life-threatening conditions. Here tamoxifen, a drug that is used for the treatment of breast cancer, is investigated in a mouse model of Duchenne muscular dystrophy. Tamoxifen was efficacious in countering the symptoms of the disease without affecting the underlying genetic cause. Based on these results, tamoxifen has been tested in other forms of muscle disease with success. Drug repurposing may not only be a cost-effective manner for treating a variety of diseases, it may also help us uncover common mechanisms between conditions that were previously thought to be unrelated.

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Published

2018-04-25

How to Cite

[1]
E. Gayi, L. A. Neff, H. M. Ismail, U. T. Ruegg, L. Scapozza, O. M. Dorchies, Chimia 2018, 72, 238, DOI: 10.2533/chimia.2018.238.