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Perspectives on Neuroscience
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Wilson disease-treatment perspectives.

Tomasz Litwin1, Karolina Dzieżyc1, Anna Członkowska1

  • 1Second Department of Neurology, Institute Psychiatry and Neurology, Warsaw, Poland.

Annals of Translational Medicine
|June 11, 2019
PubMed
Summary

New Wilson disease (WD) treatments are being researched due to limitations of current copper-lowering drugs. Future therapies focus on novel agents, non-chelating drugs, and cell/gene therapy for improved safety and efficacy.

Keywords:
ChelatorsWilson disease (WD)coppergene therapyzinc salts

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Area of Science:

  • Genetics
  • Neurology
  • Pharmacology

Background:

  • Wilson disease (WD) is a genetic disorder causing toxic copper accumulation in organs like the liver and brain.
  • Current treatments, chelators and zinc salts, are decades old and have limitations, including neurological deterioration and adverse events.
  • Many patients experience persistent deficits or non-adherence due to side effects and complex regimens.

Purpose of the Study:

  • To review current research into novel therapeutic strategies for Wilson disease.
  • To explore alternative treatments addressing the limitations of existing copper-lowering therapies.

Main Methods:

  • Review of ongoing research into new Wilson disease treatments.
  • Focus on agents with improved safety profiles and efficacy in copper removal, particularly from the brain.
  • Exploration of non-chelating drugs and cell/gene therapy approaches.

Main Results:

  • Research is exploring tetrathiomolybdate salts and CNS-penetrable trientine for enhanced copper removal.
  • Methanobactin represents a non-chelating drug candidate in preclinical studies.
  • Cell and gene therapy are also under investigation as future treatment modalities.

Conclusions:

  • Existing Wilson disease treatments have significant limitations, necessitating the development of new therapeutic options.
  • Future research is focused on safer, more effective copper chelation, non-chelating agents, and advanced cell/gene therapies.
  • These emerging treatments aim to improve outcomes for patients with Wilson disease, especially those with neurological involvement.