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The Role of Nitisinone in Tyrosine Pathway Disorders

  • Orphan Diseases (B Manger, Section Editor)
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Abstract

Nitisinone 2-(2-nitro-4-trifluoromethylbenzoyl)cyclohexane-1,3-dione (NTBC), an effective herbicide, is the licensed treatment for the human condition, hereditary tyrosinaemia type 1 (HT-1). Its mode of action interrupts tyrosine metabolism through inhibition of 4-hydroxyphenylpyruvate dioxygenase (HPPD). Nitisinone is a remarkable safe drug to use with few side effects reported. Therefore, we propose that it should be investigated as a potential treatment for other disorders of tyrosine metabolism. These include alkaptonuria (AKU), a rare disease resulting is severe, early-onset osteoarthritis. We present a case study from the disease, and attempts to use the drug both off-label and in clinical research through the DevelopAKUre consortium.

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Compliance with Ethics Guidelines

Conflict of Interest

Edward Lock reports that he holds a patent WO Patent 93/00080, for use of NTBC in tyrosinaemia type 1, licensed to Swedish Orphan Biovitrum AB .

Lakshminarayan R. Ranganath and Oliver Timmis report no conflict of interest.

Human and Animal Rights and Informed Consent

This article does not contain any studies with human or animal subjects performed by any of the authors.

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Authors and Affiliations

  1. School of Pharmacy and Biomolecular Sciences, Liverpool John Moores University, Byrom Street, Liverpool, L3 3AF, UK

    Edward Lock

  2. Department of Clinical Biochemistry, Royal Liverpool University Hospital, Prescot Street, Liverpool, L7 8XP, UK

    Lakshminarayan R. Ranganath

  3. AKU Society, 66 Devonshire Road, Cambridge, CB1 2BL, UK

    Oliver Timmis

Authors
  1. Edward Lock
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  2. Lakshminarayan R. Ranganath
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  3. Oliver Timmis
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Corresponding author

Correspondence to Oliver Timmis.

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Lock, E., Ranganath, L.R. & Timmis, O. The Role of Nitisinone in Tyrosine Pathway Disorders. Curr Rheumatol Rep 16, 457 (2014). https://doi.org/10.1007/s11926-014-0457-0

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  • DOI: https://doi.org/10.1007/s11926-014-0457-0

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