Discovery of a selective, state-independent inhibitor of Na


Journal

Scientific reports
ISSN: 2045-2322
Titre abrégé: Sci Rep
Pays: England
ID NLM: 101563288

Informations de publication

Date de publication:
09 09 2020
Historique:
received: 16 04 2020
accepted: 06 08 2020
entrez: 10 9 2020
pubmed: 11 9 2020
medline: 18 5 2021
Statut: epublish

Résumé

The voltage-gated sodium channel isoform Na

Identifiants

pubmed: 32908170
doi: 10.1038/s41598-020-71135-2
pii: 10.1038/s41598-020-71135-2
pmc: PMC7481244
doi:

Substances chimiques

NAV1.1 Voltage-Gated Sodium Channel 0
NAV1.2 Voltage-Gated Sodium Channel 0
NAV1.3 Voltage-Gated Sodium Channel 0
NAV1.4 Voltage-Gated Sodium Channel 0
NAV1.5 Voltage-Gated Sodium Channel 0
NAV1.6 Voltage-Gated Sodium Channel 0
NAV1.7 Voltage-Gated Sodium Channel 0
NAV1.8 Voltage-Gated Sodium Channel 0
Sodium Channel Blockers 0
Guanidine JU58VJ6Y3B

Types de publication

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

Langues

eng

Sous-ensembles de citation

IM

Pagination

14791

Subventions

Organisme : NINDS NIH HHS
ID : R44 NS081887
Pays : United States
Organisme : NIH HHS
ID : R01 GM117263-01A1
Pays : United States

Références

Dib-Hajj, S. D., Yang, Y., Black, J. A. & Waxman, S. G. The Na
pubmed: 23232607
Cox, J. J. et al. An SCN9A channelopathy causes congenital inability to experience pain. Nature 444, 894–898 (2006).
pubmed: 17167479 pmcid: 7212082
Goldberg, Y. et al. Loss-of-function mutations in the Nav1.7 gene underlie congenital indifference to pain in multiple human populations. Clin. Genet. 71, 311–319 (2007).
pubmed: 17470132
Yang, Y. et al. Mutations in SCN9A, encoding a sodium channel alpha subunit, in patients with primary erythermalgia. J. Med. Genet. 41, 171–174 (2004).
pubmed: 14985375 pmcid: 1735695
Faber, C. G. et al. Gain of function Na
pubmed: 21698661
Payandeh, J. & Hackos, D. H. Selective ligands and drug discovery targeting the voltage-gated sodium channel Nav1.7. In Voltage-gated Sodium Channels: Structure, Function and Channelopathies, Vol. 246 (ed. Chahine, M.) 271–306 (Springer International Publishing, Berlin, 2018).
McKerrall, S. J. & Sutherlin, D. P. Nav1.7 inhibitors for the treatment of chronic pain. Bioorg. Med. Chem. Lett. 28, 3141–3149 (2018).
pubmed: 30139550
Mulcahy, J. V. et al. Challenges and opportunities for therapeutics targeting the voltage-gated sodium channel isoform Na
doi: 10.1021/acs.jmedchem.8b01906 pubmed: 31012583
Ahern, C. A., Payandeh, J., Bosmans, F. & Chanda, B. The hitchhiker’s guide to the voltage-gated sodium channel galaxy. J. Gen. Physiol. 147, 1–24 (2016).
pubmed: 26712848 pmcid: 4692491
Stevens, M., Peigneur, S. & Tytgat, J. Neurotoxins and their binding areas on voltage-gated sodium channels. Front. Pharmacol. 2, 71 (2011).
pubmed: 22084632 pmcid: 3210964
Israel, M. R., Tay, B., Deuis, J. R. & Vetter, I. Sodium channels and venom peptide pharmacology. In Advances in Pharmacology, vol. 79, 67–116 (Elsevier, Amsterdam, 2017).
Bosmans, F. & Swartz, K. J. Targeting voltage sensors in sodium channels with spider toxins. Trends Pharmacol. Sci. 31, 175–182 (2010).
pubmed: 20097434 pmcid: 2847040
Wang, X. et al. Inhibitors of Ion Channels, Patent PCT/US2006/042882, 2006.
Fulp, A., Marron, B., Suto, M., J. & Wang, X. Inhibitors of Voltage-Gated Sodium Channels, Patent PCT/US2006/031390, 2006.
Kawatkar, A., S. et al. Bicyclic Deriatives as Modulators of Ion Channels, Patent PCT/US2006/017699, 2006.
McCormack, K. et al. Voltage sensor interaction site for selective small molecule inhibitors of voltage-gated sodium channels. Proc. Natl. Acad. Sci. 110, E2724–E2732 (2013).
pubmed: 23818614
Zhang, M.-M. et al. Cooccupancy of the outer vestibule of voltage-gated sodium channels by micro-conotoxin KIIIA and saxitoxin or tetrodotoxin. J. Neurophysiol. 104, 88–97 (2010).
pubmed: 20410356 pmcid: 2904204
Shen, H. et al. Structural basis for the modulation of voltage-gated sodium channels by animal toxins. Science 362, eaau2596 (2018).
pubmed: 30049784
Ahuja, S. et al. Structural basis of Nav1.7 inhibition by an isoform-selective small-molecule antagonist. Science 350, aac5464 (2015).
pubmed: 26680203
Alexandrou, A. J. et al. Subtype-selective small molecule inhibitors reveal a fundamental role for Nav1.7 in nociceptor electrogenesis, axonal conduction and presynaptic release. PLoS ONE 11, e0152405 (2016).
pubmed: 27050761 pmcid: 4822888
Varney, M. Roche: At the Forefront of R&D Innovation and Breakthrough Treatments. https://www.roche.com/dam/jcr:5c999124-c278-4549-8e94-4475cc741de1/en/2016_roche_cowen_presentation.pdf (2016)
Schmalhofer, W. A. et al. ProTx-II, a selective inhibitor of Na
pubmed: 18728100
Xiao, Y. et al. Tarantula huwentoxin-IV inhibits neuronal sodium channels by binding to receptor site 4 and trapping the domain II voltage sensor in the closed configuration. J. Biol. Chem. 283, 27300–27313 (2008).
pubmed: 18628201 pmcid: 2556013
Deuis, J. R. et al. Pharmacological characterisation of the highly Na
pubmed: 28106092 pmcid: 5247677
Moyer, B. D. et al. Pharmacological characterization of potent and selective Na
pubmed: 29723257 pmcid: 5933747
Flinspach, M. et al. Insensitivity to pain induced by a potent selective closed-state Nav1.7 inhibitor. Sci. Rep. 7, 39662 (2017).
pubmed: 28045073 pmcid: 5206724
McKerrall, S. J. et al. Structure- and ligand-based discovery of chromane arylsulfonamide Nav1.7 inhibitors for the treatment of chronic pain. J. Med. Chem. 62, 4091–4109 (2019).
pubmed: 30943032
Graceffa, R. F. et al. Sulfonamides as selective Na
pubmed: 28324649
McDonnell, A. et al. Efficacy of the Nav1.7 blocker PF-05089771 in a randomised, placebo-controlled, double-blind clinical study in subjects with painful diabetic peripheral neuropathy. Pain 159, 1465–1476 (2018).
pubmed: 29578944
Rothenberg, M. E. et al. Safety, tolerability, and pharmacokinetics of GDC-0276, a novel Na
doi: 10.1007/s40261-019-00807-3 pubmed: 31338799
Walker, J. R. et al. Marked difference in saxitoxin and tetrodotoxin affinity for the human nociceptive voltage-gated sodium channel (Na
pubmed: 23077250
Thomas-Tran, R. & Du Bois, J. Mutant cycle analysis with modified saxitoxins reveals specific interactions critical to attaining high-affinity inhibition of hNa
pubmed: 27162340
Tsukamoto, T. et al. Differential binding of tetrodotoxin and its derivatives to voltage-sensitive sodium channel subtypes (Nav1.1 to Nav1.7). Br. J. Pharmacol. 174, 3881–3892 (2017).
pubmed: 28832970 pmcid: 5647187
Fleming, J. J., McReynolds, M. D. & Du Bois, J. (+)-saxitoxin: A first and second generation stereoselective synthesis. J. Am. Chem. Soc. 129, 9964–9975 (2007).
pubmed: 17658800
Mulcahy, J. V., Walker, J. R., Merit, J. E., Whitehead, A. & Du Bois, J. Synthesis of the paralytic shellfish poisons (+)-gonyautoxin 2, (+)-Gonyautoxin 3, and (+)-11,11-dihydroxysaxitoxin. J. Am. Chem. Soc. 138, 5994–6001 (2016).
pubmed: 27138488
Andresen, B. M. & Du Bois, J. De novo synthesis of modified saxitoxins for sodium ion channel study. J. Am. Chem. Soc. 131, 12524–12525 (2009).
pubmed: 19678702 pmcid: 2770901
Walker, J. R., Merit, J. E., Thomas-Tran, R., Tang, D. T. Y. & Du Bois, J. Divergent synthesis of natural derivatives of (+)-saxitoxin including 11-saxitoxinethanoic acid. Angew. Chem. Int. Ed. Engl. 58, 1689–1693 (2019).
pubmed: 30488599 pmcid: 6426452
Lipkind, G. M. & Fozzard, H. A. A structural model of the tetrodotoxin and saxitoxin binding site of the Na
pubmed: 8130328 pmcid: 1275657
Penzotti, J. L., Fozzard, H. A., Lipkind, G. M. & Dudley, S. C. Differences in saxitoxin and tetrodotoxin binding revealed by mutagenesis of the Na
pubmed: 9826589 pmcid: 1299940
Tikhonov, D. B. & Zhorov, B. S. Modeling P-loops domain of sodium channel: Homology with potassium channels and interaction with ligands. Biophys. J. 88, 184–197 (2005).
pubmed: 15475578
Shen, H., Liu, D., Wu, K., Lei, J. & Yan, N. Structures of human Na
pubmed: 30765606
Goral, R. O., Leipold, E., Nematian-Ardestani, E. & Heinemann, S. H. Heterologous expression of Na
pubmed: 25916202
Satin, J. et al. A mutant of TTX-resistant cardiac sodium channels with TTX-sensitive properties. Science 256, 1202–1205 (1992).
pubmed: 1375397
Gingras, J. et al. Global Nav1.7 knockout mice recapitulate the phenotype of human congenital indifference to pain. PLoS ONE 9, e105895 (2014).
pubmed: 25188265 pmcid: 4154897
Shields, S. D. et al. Insensitivity to pain upon adult-onset deletion of Nav1.7 or its blockade with selective inhibitors. J. Neurosci. 38, 10180–10201 (2018).
pubmed: 30301756 pmcid: 6596201
Minett, M. S. et al. Distinct Nav1.7-dependent pain sensations require different sets of sensory and sympathetic neurons. Nat. Commun. 3, 791 (2012).
pubmed: 22531176 pmcid: 3337979
Yeomans, D. C. et al. Recombinant herpes vector-mediated analgesia in a primate model of hyperalgesia. Mol. Ther. 13, 589–597 (2006).
pubmed: 16288901
Cooper, B. Y., Vierck, C. J. & Yeomans, D. C. Selective reduction of second pain sensations by systemic morphine in humans. Pain 24, 93–116 (1986).
pubmed: 3951883
Tzabazis, A. Z. et al. Selective nociceptor activation in volunteers by infrared diode laser. Mol. Pain 7, 18 (2011).
pubmed: 21426575 pmcid: 3070669
Yeomans, D. C., Pirec, V. & Proudfit, H. K. Nociceptive responses to high and low rates of noxious cutaneous heating are mediated by different nociceptors in the rat: behavioral evidence. Pain 68, 133–140 (1996).
pubmed: 9252008
Loggia, M. L., Juneau, M. & Bushnell, C. M. Autonomic responses to heat pain: Heart rate, skin conductance, and their relation to verbal ratings and stimulus intensity. Pain 152, 592–598 (2011).
pubmed: 21215519
Theile, J. W., Fuller, M. D. & Chapman, M. L. The selective Nav1.7 inhibitor, PF-05089771, interacts equivalently with fast and slow inactivated Nav1..7 channels. Mol. Pharmacol. 90, 540–548 (2016).
pubmed: 27587537
Alles, S. R. A. et al. Sensory neuron-derived Na
pubmed: 32128393 pmcid: 7030926

Auteurs

H Pajouhesh (H)

SiteOne Therapeutics, South San Francisco, CA, 94080, USA.

J T Beckley (JT)

SiteOne Therapeutics, Bozeman, MT, 59715, USA.

A Delwig (A)

SiteOne Therapeutics, South San Francisco, CA, 94080, USA.

H S Hajare (HS)

Department of Chemistry, Stanford University, Stanford, CA, 94305, USA.

G Luu (G)

SiteOne Therapeutics, South San Francisco, CA, 94080, USA.

D Monteleone (D)

SiteOne Therapeutics, South San Francisco, CA, 94080, USA.

X Zhou (X)

SiteOne Therapeutics, South San Francisco, CA, 94080, USA.

J Ligutti (J)

Neuroscience Department, Amgen Research, Thousand Oaks, CA, 91320, USA.

S Amagasu (S)

Neuroscience Department, Amgen Research, Thousand Oaks, CA, 91320, USA.

B D Moyer (BD)

Neuroscience Department, Amgen Research, Thousand Oaks, CA, 91320, USA.

D C Yeomans (DC)

Department of Anesthesiology, Perioperative, and Pain Medicine, Stanford University, Stanford, CA, 94305, USA.

J Du Bois (J)

Department of Chemistry, Stanford University, Stanford, CA, 94305, USA.

J V Mulcahy (JV)

SiteOne Therapeutics, South San Francisco, CA, 94080, USA. john.mulcahy@site1therapeutics.com.

Articles similaires

[Redispensing of expensive oral anticancer medicines: a practical application].

Lisanne N van Merendonk, Kübra Akgöl, Bastiaan Nuijen
1.00
Humans Antineoplastic Agents Administration, Oral Drug Costs Counterfeit Drugs

Smoking Cessation and Incident Cardiovascular Disease.

Jun Hwan Cho, Seung Yong Shin, Hoseob Kim et al.
1.00
Humans Male Smoking Cessation Cardiovascular Diseases Female
Humans United States Aged Cross-Sectional Studies Medicare Part C
1.00
Humans Yoga Low Back Pain Female Male

Classifications MeSH