Optimization of a nicotine degrading enzyme for potential use in treatment of nicotine addiction.


Journal

BMC biotechnology
ISSN: 1472-6750
Titre abrégé: BMC Biotechnol
Pays: England
ID NLM: 101088663

Informations de publication

Date de publication:
02 08 2019
Historique:
received: 09 05 2019
accepted: 23 07 2019
entrez: 4 8 2019
pubmed: 4 8 2019
medline: 14 1 2020
Statut: epublish

Résumé

Smoking and tobacco use continue to be the largest preventable causes of death globally. A novel therapeutic approach has recently been proposed: administration of an enzyme that degrades nicotine, the main addictive component of tobacco, minimizing brain exposure and reducing its reinforcing effects. Pre-clinical proof of concept has been previously established through dosing the amine oxidase NicA2 from Pseudomonas putida in rat nicotine self-administration models of addiction. This paper describes efforts towards optimizing NicA2 for potential therapeutic use: enhancing potency, improving its pharmacokinetic profile, and attenuating immunogenicity. Libraries randomizing residues located in all 22 active site positions of NicA2 were screened. 58 single mutations with 2- to 19-fold enhanced catalytic activity compared to wt at 10 μM nicotine were identified. A novel nicotine biosensor assay allowed efficient screening of the many primary hits for activity at nicotine concentrations typically found in smokers. 10 mutants with improved activity in rat serum at or below 250 nM were identified. These catalytic improvements translated to increased potency in vivo in the form of further lowering of nicotine blood levels and nicotine accumulation in the brains of Sprague-Dawley rats. Examination of the X-ray crystal structure suggests that these mutants may accelerate the rate limiting re-oxidation of the flavin adenine dinucleotide cofactor by enhancing molecular oxygen's access. PEGylation of NicA2 led to prolonged serum half-life and lowered immunogenicity observed in a human HLA DR4 transgenic mouse model, without impacting nicotine degrading activity. Systematic mutational analysis of the active site of the nicotine-degrading enzyme NicA2 has yielded 10 variants that increase the catalytic activity and its effects on nicotine distribution in vivo at nicotine plasma concentrations found in smokers. In addition, PEGylation substantially increases circulating half-life and reduces the enzyme's immunogenic potential. Taken together, these results provide a viable path towards generation of a drug candidate suitable for human therapeutic use in treating nicotine addiction.

Sections du résumé

BACKGROUND
Smoking and tobacco use continue to be the largest preventable causes of death globally. A novel therapeutic approach has recently been proposed: administration of an enzyme that degrades nicotine, the main addictive component of tobacco, minimizing brain exposure and reducing its reinforcing effects. Pre-clinical proof of concept has been previously established through dosing the amine oxidase NicA2 from Pseudomonas putida in rat nicotine self-administration models of addiction.
RESULTS
This paper describes efforts towards optimizing NicA2 for potential therapeutic use: enhancing potency, improving its pharmacokinetic profile, and attenuating immunogenicity. Libraries randomizing residues located in all 22 active site positions of NicA2 were screened. 58 single mutations with 2- to 19-fold enhanced catalytic activity compared to wt at 10 μM nicotine were identified. A novel nicotine biosensor assay allowed efficient screening of the many primary hits for activity at nicotine concentrations typically found in smokers. 10 mutants with improved activity in rat serum at or below 250 nM were identified. These catalytic improvements translated to increased potency in vivo in the form of further lowering of nicotine blood levels and nicotine accumulation in the brains of Sprague-Dawley rats. Examination of the X-ray crystal structure suggests that these mutants may accelerate the rate limiting re-oxidation of the flavin adenine dinucleotide cofactor by enhancing molecular oxygen's access. PEGylation of NicA2 led to prolonged serum half-life and lowered immunogenicity observed in a human HLA DR4 transgenic mouse model, without impacting nicotine degrading activity.
CONCLUSIONS
Systematic mutational analysis of the active site of the nicotine-degrading enzyme NicA2 has yielded 10 variants that increase the catalytic activity and its effects on nicotine distribution in vivo at nicotine plasma concentrations found in smokers. In addition, PEGylation substantially increases circulating half-life and reduces the enzyme's immunogenic potential. Taken together, these results provide a viable path towards generation of a drug candidate suitable for human therapeutic use in treating nicotine addiction.

Identifiants

pubmed: 31375100
doi: 10.1186/s12896-019-0551-5
pii: 10.1186/s12896-019-0551-5
pmc: PMC6679477
doi:

Substances chimiques

Bacterial Proteins 0
Nicotine 6M3C89ZY6R
Monoamine Oxidase EC 1.4.3.4

Types de publication

Journal Article Research Support, N.I.H., Extramural Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

56

Subventions

Organisme : National Institute on Drug Abuse
ID : R43 DA044064
Pays : United States

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Auteurs

Thomas Thisted (T)

Antidote Therapeutics, Inc, 708 Quince Orchard Road, Suite 250-C, Gaithersburg, MD, 20878, USA.

Zuzana Biesova (Z)

Antidote Therapeutics, Inc, 708 Quince Orchard Road, Suite 250-C, Gaithersburg, MD, 20878, USA.

Celine Walmacq (C)

Antidote Therapeutics, Inc, 708 Quince Orchard Road, Suite 250-C, Gaithersburg, MD, 20878, USA.

Everett Stone (E)

Department of Molecular Biosciences, The University of Texas at Austin, Austin, TX, USA.

Max Rodnick-Smith (M)

Department of Molecular Biosciences, The University of Texas at Austin, Austin, TX, USA.

Shaheda S Ahmed (SS)

Alcyomics Ltd, Bulman House, Regent Centre, Gosforth, Newcastle upon Tyne, NE3 3LS, UK.

Stephen K Horrigan (SK)

Noble Life Sciences, PO Box 242, Woodbine, MD, 21797, USA.

Bo Van Engelen (B)

Antidote Therapeutics, Inc, 708 Quince Orchard Road, Suite 250-C, Gaithersburg, MD, 20878, USA.
Maastricht University, P. Debyeplein 1, 6229 HA, Maastricht, NL, USA.

Charles Reed (C)

Antidote Therapeutics, Inc, 708 Quince Orchard Road, Suite 250-C, Gaithersburg, MD, 20878, USA.

Matthew W Kalnik (MW)

Antidote Therapeutics, Inc, 708 Quince Orchard Road, Suite 250-C, Gaithersburg, MD, 20878, USA. mkalnik@atibio.com.

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Classifications MeSH