Activity-based protein profiling reveals off-target proteins of the FAAH inhibitor BIA 10-2474

A recent phase 1 trial of the fatty acid amide hydrolase (FAAH) inhibitor BIA 10-2474 led to the death of one volunteer and produced mild-to-severe neurological symptoms in four others. Although the cause of the clinical neurotoxicity is unknown, it has been postulated, given the clinical safety pro...

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Veröffentlicht in:Science (American Association for the Advancement of Science) Jg. 356; H. 6342; S. 1084
Hauptverfasser: van Esbroeck, Annelot C M, Janssen, Antonius P A, Cognetta, 3rd, Armand B, Ogasawara, Daisuke, Shpak, Guy, van der Kroeg, Mark, Kantae, Vasudev, Baggelaar, Marc P, de Vrij, Femke M S, Deng, Hui, Allarà, Marco, Fezza, Filomena, Lin, Zhanmin, van der Wel, Tom, Soethoudt, Marjolein, Mock, Elliot D, den Dulk, Hans, Baak, Ilse L, Florea, Bogdan I, Hendriks, Giel, De Petrocellis, Luciano, Overkleeft, Herman S, Hankemeier, Thomas, De Zeeuw, Chris I, Di Marzo, Vincenzo, Maccarrone, Mauro, Cravatt, Benjamin F, Kushner, Steven A, van der Stelt, Mario
Format: Journal Article
Sprache:Englisch
Veröffentlicht: United States 09.06.2017
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ISSN:1095-9203, 1095-9203
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Abstract A recent phase 1 trial of the fatty acid amide hydrolase (FAAH) inhibitor BIA 10-2474 led to the death of one volunteer and produced mild-to-severe neurological symptoms in four others. Although the cause of the clinical neurotoxicity is unknown, it has been postulated, given the clinical safety profile of other tested FAAH inhibitors, that off-target activities of BIA 10-2474 may have played a role. Here we use activity-based proteomic methods to determine the protein interaction landscape of BIA 10-2474 in human cells and tissues. This analysis revealed that the drug inhibits several lipases that are not targeted by PF04457845, a highly selective and clinically tested FAAH inhibitor. BIA 10-2474, but not PF04457845, produced substantial alterations in lipid networks in human cortical neurons, suggesting that promiscuous lipase inhibitors have the potential to cause metabolic dysregulation in the nervous system.
AbstractList A recent phase 1 trial of the fatty acid amide hydrolase (FAAH) inhibitor BIA 10-2474 led to the death of one volunteer and produced mild-to-severe neurological symptoms in four others. Although the cause of the clinical neurotoxicity is unknown, it has been postulated, given the clinical safety profile of other tested FAAH inhibitors, that off-target activities of BIA 10-2474 may have played a role. Here we use activity-based proteomic methods to determine the protein interaction landscape of BIA 10-2474 in human cells and tissues. This analysis revealed that the drug inhibits several lipases that are not targeted by PF04457845, a highly selective and clinically tested FAAH inhibitor. BIA 10-2474, but not PF04457845, produced substantial alterations in lipid networks in human cortical neurons, suggesting that promiscuous lipase inhibitors have the potential to cause metabolic dysregulation in the nervous system.A recent phase 1 trial of the fatty acid amide hydrolase (FAAH) inhibitor BIA 10-2474 led to the death of one volunteer and produced mild-to-severe neurological symptoms in four others. Although the cause of the clinical neurotoxicity is unknown, it has been postulated, given the clinical safety profile of other tested FAAH inhibitors, that off-target activities of BIA 10-2474 may have played a role. Here we use activity-based proteomic methods to determine the protein interaction landscape of BIA 10-2474 in human cells and tissues. This analysis revealed that the drug inhibits several lipases that are not targeted by PF04457845, a highly selective and clinically tested FAAH inhibitor. BIA 10-2474, but not PF04457845, produced substantial alterations in lipid networks in human cortical neurons, suggesting that promiscuous lipase inhibitors have the potential to cause metabolic dysregulation in the nervous system.
A recent phase 1 trial of the fatty acid amide hydrolase (FAAH) inhibitor BIA 10-2474 led to the death of one volunteer and produced mild-to-severe neurological symptoms in four others. Although the cause of the clinical neurotoxicity is unknown, it has been postulated, given the clinical safety profile of other tested FAAH inhibitors, that off-target activities of BIA 10-2474 may have played a role. Here we use activity-based proteomic methods to determine the protein interaction landscape of BIA 10-2474 in human cells and tissues. This analysis revealed that the drug inhibits several lipases that are not targeted by PF04457845, a highly selective and clinically tested FAAH inhibitor. BIA 10-2474, but not PF04457845, produced substantial alterations in lipid networks in human cortical neurons, suggesting that promiscuous lipase inhibitors have the potential to cause metabolic dysregulation in the nervous system.
Author van Esbroeck, Annelot C M
van der Stelt, Mario
Lin, Zhanmin
Hendriks, Giel
den Dulk, Hans
Florea, Bogdan I
Fezza, Filomena
Hankemeier, Thomas
Soethoudt, Marjolein
Kushner, Steven A
De Petrocellis, Luciano
Allarà, Marco
van der Kroeg, Mark
van der Wel, Tom
Janssen, Antonius P A
Di Marzo, Vincenzo
Cravatt, Benjamin F
Kantae, Vasudev
Baak, Ilse L
Baggelaar, Marc P
de Vrij, Femke M S
Cognetta, 3rd, Armand B
Overkleeft, Herman S
Maccarrone, Mauro
De Zeeuw, Chris I
Ogasawara, Daisuke
Shpak, Guy
Deng, Hui
Mock, Elliot D
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  surname: Deng
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  surname: Allarà
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  organization: Department of Experimental Medicine and Surgery, Tor Vergata University of Rome, Via Montpellier 1, 00133 Rome, Italy
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  orcidid: 0000-0002-8188-6733
  surname: Lin
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  organization: Department of Neuroscience, Erasmus Medical Centre, 3000 CA, Rotterdam, Netherlands
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BackLink https://www.ncbi.nlm.nih.gov/pubmed/28596366$$D View this record in MEDLINE/PubMed
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References 28853735 - Nat Chem Biol. 2017 Jul 18;13(8):817
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Snippet A recent phase 1 trial of the fatty acid amide hydrolase (FAAH) inhibitor BIA 10-2474 led to the death of one volunteer and produced mild-to-severe...
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SubjectTerms Amidohydrolases - antagonists & inhibitors
Analgesics - adverse effects
Analgesics - chemistry
Analgesics - metabolism
Analgesics - pharmacology
Anti-Anxiety Agents - adverse effects
Anti-Anxiety Agents - chemistry
Anti-Anxiety Agents - metabolism
Anti-Anxiety Agents - pharmacology
Cell Line, Tumor
Clinical Trials, Phase I as Topic
Cross Reactions
Cyclic N-Oxides - adverse effects
Cyclic N-Oxides - chemistry
Cyclic N-Oxides - metabolism
Cyclic N-Oxides - pharmacology
Humans
Neurons - drug effects
Neurons - metabolism
Protein Interaction Maps
Pyridazines - pharmacology
Pyridazines - therapeutic use
Pyridines - adverse effects
Pyridines - chemistry
Pyridines - metabolism
Pyridines - pharmacology
Urea - analogs & derivatives
Urea - pharmacology
Urea - therapeutic use
Title Activity-based protein profiling reveals off-target proteins of the FAAH inhibitor BIA 10-2474
URI https://www.ncbi.nlm.nih.gov/pubmed/28596366
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