- Brindisi, Margherita;
- Maramai, Samuele;
- Gemma, Sandra;
- Brogi, Simone;
- Grillo, Alessandro;
- Di Cesare Mannelli, Lorenzo;
- Gabellieri, Emanuele;
- Lamponi, Stefania;
- Saponara, Simona;
- Gorelli, Beatrice;
- Tedesco, Daniele;
- Bonfiglio, Tommaso;
- Landry, Christophe;
- Jung, Kwang-Mook;
- Armirotti, Andrea;
- Luongo, Livio;
- Ligresti, Alessia;
- Piscitelli, Fabiana;
- Bertucci, Carlo;
- Dehouck, Marie-Pierre;
- Campiani, Giuseppe;
- Maione, Sabatino;
- Ghelardini, Carla;
- Pittaluga, Anna;
- Piomelli, Daniele;
- Di Marzo, Vincenzo;
- Butini, Stefania
We report the discovery of compound 4a, a potent β-lactam-based monoacylglycerol lipase (MGL) inhibitor characterized by an irreversible and stereoselective mechanism of action, high membrane permeability, high brain penetration evaluated using a human in vitro blood-brain barrier model, high selectivity in binding and affinity-based proteomic profiling assays, and low in vitro toxicity. Mode-of-action studies demonstrate that 4a, by blocking MGL, increases 2-arachidonoylglycerol and behaves as a cannabinoid (CB1/CB2) receptor indirect agonist. Administration of 4a in mice suffering from experimental autoimmune encephalitis ameliorates the severity of the clinical symptoms in a CB1/CB2-dependent manner. Moreover, 4a produced analgesic effects in a rodent model of acute inflammatory pain, which was antagonized by CB1 and CB2 receptor antagonists/inverse agonists. 4a also relieves the neuropathic hypersensitivity induced by oxaliplatin. Given these evidence, 4a, as MGL selective inhibitor, could represent a valuable lead for the future development of therapeutic options for multiple sclerosis and chronic pain.