Natural product derivatives for inhibiting cellular necroptosis, ferroptosis and oxytosis
11819492 · 2023-11-21
Assignee
- Institut National De La Sante Et De La Recherche Medicale (Inserm) (Paris, FR)
- Centre National De La Recherche Scientifique (Cnrs) (Paris, FR)
- Sorbonne Universite (Paris, FR)
- Universite De Poitiers (Poitiers, FR)
- Centre Hospitalier Universitaire de Poitiers (Poitiers, FR)
Inventors
- Stéphane Bach (Sibiril, FR)
- Marie-Thérèse Dimanche-Boitrel (Melesse, FR)
- Claire Delehouze (La Roche Maurice, FR)
- Thierry Hauet (Mignaloux Beauvoir, FR)
Cpc classification
A61K31/352
HUMAN NECESSITIES
A61K45/06
HUMAN NECESSITIES
International classification
A61K31/352
HUMAN NECESSITIES
A61K45/06
HUMAN NECESSITIES
Abstract
The present invention relates to a compound of the following general formula (I): ##STR00001##
or a pharmaceutically acceptable salt and/or solvate thereof, for use as drug, particularly intended for inhibiting a programmed cell death route selected from the group consisting of ferroptosis, oxytosis and cellular necroptosis. The present invention also relates to a compound of general formula (I) for use as a drug for neuroprotection as well as for preventing and/or treating disorders associated with cellular necroptosis or ferroptosis. The present invention also relates to a pharmaceutical composition comprising a compound of general formula (I), or a pharmaceutically acceptable salt and/or solvate thereof. The present invention also encompasses the use of a compound of the general formula (I) for organs preservation.
Claims
1. A pharmaceutical composition comprising at least one compound of the following general formula (I): ##STR00006## or a pharmaceutically acceptable salt and/or solvate thereof, wherein: X.sub.1 represents a (C.sub.1-C.sub.6)alkyl an aryl, an aryl-(C.sub.1-C.sub.6)alkyl group or an OR.sub.X group, wherein R.sub.X is selected from a (C.sub.1-C.sub.6)alkyl, an aryl and an aryl-(C.sub.1-C.sub.6)alkyl group, X.sub.2 and X.sub.3 each represent, independently of each other, a hydrogen atom or a (C.sub.1-C.sub.6)alkyl group, Y.sub.1, Y.sub.2 and Y.sub.3 each represent, independently of each other, a hydrogen atom, a (C.sub.1-C.sub.6)alkyl, an aryl, an aryl-(C.sub.1-C.sub.6)alkyl group, an OH or an OR.sub.Y group, with at least one of Y.sub.1, Y.sub.2 and Y.sub.3 representing a (C.sub.1-C.sub.6)alkyl, an aryl, an aryl-(C.sub.1-C.sub.6)alkyl group or an OR.sub.Y group, wherein R.sub.Y is selected from a (C.sub.1-C.sub.6)alkyl, an aryl and an aryl-(C.sub.1-C.sub.6)alkyl group, and at least one pharmaceutically acceptable excipient.
2. The pharmaceutical composition according to claim 1, wherein the compound is of the following general formula (II): ##STR00007## or a pharmaceutically acceptable salt and/or solvate thereof, wherein R.sub.X represents a (C.sub.1-C.sub.6)alkyl group and R.sub.Y represents an aryl-(C.sub.1-C.sub.6)alkyl group.
3. The pharmaceutical composition according to claim 1, wherein the pharmaceutically acceptable excipient is selected from the group consisting of long-term stabilizers, drug absorption enhancers, viscosity reducers and solubility enhancers.
4. The pharmaceutical composition according to claim 3, wherein the pharmaceutically acceptable excipient is a solubility enhancer.
5. The pharmaceutical composition according to claim 1, wherein it further comprises at least one other active ingredient.
6. The pharmaceutical composition according to claim 5, wherein the other active ingredient is selected from the group consisting of another cellular necroptosis inhibitor, an apoptosis inhibitor, an autophagy inhibitor, a ferroptosis inhibitor, an inhibitor of mitochondrial permeability transition (MPT) pore-dependent necrosis, a cyclophilin inhibitor, a Cyclin-dependent kinase 5 (CDK5) inhibitor, a parthanatos inhibitor, a thrombin inhibitor, an antioxidant an inflammatory inhibitor and combinations thereof.
7. The pharmaceutical composition according to claim 6, wherein the other active ingredient is an antioxidant.
8. A combination product comprising: (i) at least one compound of the following general formula (I): or a pharmaceutically acceptable salt and/or solvate thereof, wherein: X.sub.1 represents a (C.sub.1-C.sub.6)alkyl an aryl, an aryl-(C.sub.1-C.sub.6)alkyl group or an OR.sub.X group, wherein R.sub.X is selected from a (C.sub.1-C.sub.6)alkyl, an aryl and an aryl-(C.sub.1-C.sub.6)alkyl group, X.sub.2 and X.sub.3 each represent, independently of each other, a hydrogen atom or a (C.sub.1-C.sub.6)alkyl group, Y.sub.1, Y.sub.2 and Y.sub.3 each represent, independently of each other, a hydrogen atom, a (C.sub.1-C.sub.6)alkyl, an aryl, an aryl-(C.sub.1-C.sub.6)alkyl group, an OH or an OR.sub.Y group, with at least one of Y.sub.1, Y.sub.2 and Y.sub.3 representing a (C.sub.1-C.sub.6)alkyl, an aryl, an aryl-(C.sub.1-C.sub.6)alkyl group or an OR.sub.Y group, wherein R.sub.Y is selected from a (C.sub.1-C.sub.6)alkyl, an aryl and an aryl-(C.sub.1-C.sub.6)alkyl group, and (ii) at least another active ingredient selected from the group consisting of another cellular necroptosis inhibitor, an apoptosis inhibitor, an autophagy inhibitor, a ferroptosis inhibitor, an inhibitor of mitochondrial permeability transition (MPT) pore-dependent necrosis, a cyclophilin inhibitor, a Cyclin-dependent kinase 5 (CDK5) inhibitor, a parthanatos inhibitor, a thrombin inhibitor, an antioxidant an inflammatory inhibitor and combinations thereof, separate from the at least one compound of formula (I), for simultaneous, separate or sequential administration.
9. The combination product according to claim 8, wherein the compound is of the following general formula (II): or a pharmaceutically acceptable salt and/or solvate thereof, wherein R.sub.X represents a (C.sub.1-C.sub.6)alkyl group and R.sub.Y represents an aryl-(C.sub.1-C.sub.6)alkyl group.
10. The combination product according to claim 8, wherein the other active ingredient is an antioxidant.
11. The pharmaceutical composition according to claim 3, wherein the pharmaceutically acceptable excipient is a drug absorption enhancer.
Description
BRIEF SUMMARY OF THE FIGURES
(1)
(2)
(3)
(4)
(5)
(6)
(7)
(8)
(9)
(10)
(11)
(12)
(13)
(14)
(15)
(16)
(17)
(18) In the above-mentioned figures, “6E11” refers to compound 1.
EXAMPLES
(19) The following abbreviations, commonly used in this field of art, have been used in the following examples: AGC: Protein kinase A, G, and C families (PKA, PKC, PKG) BSA: Bovine Serum Albumin CAMK: Ca.sup.2+/calmodulin-dependent protein kinases CMGC: CDKs, MAP kinases, GSK and CDK-like kinases CDK: Cyclin-dependent kinase CK1: Cell Kinases 1 (originally known as Casein Kinase 1) DMF: Dimethylformamide DMSO: Dimethylsulfoxide DTT: Dithiothreitol EC.sub.50: Half maximal effective concentration EDTA: Ethylenediaminetetraacetic acid EGTA: Ethylene glycol-bis(β-aminoethyl ether)-N,N,N,N-tetraacetic acid Et: Ethyl (CH.sub.2CH.sub.3) EtOAc: Ethyl acetate FACS: Fluorescence-activated cell sorting FADD: Fas-Associated Death Domain GSH: glutathione GSK: Glycogen synthase kinases GST: Glutathione S-transferase h: hour HAEC: Human Artery Endothelial Cells HB: hydrogen bond hPBLs: human Peripheral Blood Lymphocytes hRPE-1: human Retinal Pigment Epithelial cell line IC.sub.50: Half maximal inhibitory concentration Kd: dissociation constant kg: kilogram M: Molar MAP: Mitogen-Activated Protein kinases MD: Molecular dynamic Me: Methyl (CH.sub.3) mg: milligram MHz: MegaHertz min: minute(s) ml: milliliter mM: millimolar mmol: millimole MOPS: 3-(N-morpholino)propanesulfonic acid MTS: 3-[4,5-dimethylthiazol-2-yl]-5-[3-carboxymethoxy-phenyl]-2-[4-sulfophenyl]-2H-tetrazolium n: number of replicates in an experiment N: number of independent experiments NMR: Nuclear Magnetic Resonance PBS: Phosphate buffered saline PCR: Polymerase Chain Reaction RIPK1: Receptor-Interacting Protein Kinase 1 ROS: reactive oxygen species r.t: Room temperature SD: Standard Deviation STE: STE Kinases (Homologs of yeast STErile kinases) TK: Tyrosine Kinases TKL: Tyrosine Kinases-Like TNF-α: Tumor Necrosis Factor α μg: microgram μl: Microliter μM: Micromolar
(20) I. Synthesis of the Compounds According to the Invention
Example 1: Synthesis of the Compounds of General Formula (I)
(21) Compound 1 was prepared according to the method disclosed in Hauteville et al. Tetrahedron 1980, 37, p. 377-381.
(22) Said method can be generalized to obtain a compound of general formula (I), according the following reaction scheme:
(23) ##STR00005##
(24) The starting materials are commercially available, or can be easily prepared according to methods well-known of the one skilled in the art. Optionally, and if necessary, additional protection or deprotection steps well-known of the skilled person might be incorporated within the general procedure represented above.
(25) II. Biological Tests of the Compounds According to the Invention
Example 2: Cell-Based Screening of Chemical Libraries for Characterization of Necroptosis Inhibitors
(26) TNF-α can induce necroptosis in Jurkat cells (human T lymphocytes) when FADD is deleted. This model was used to screen various libraries of chemical compounds for characterization of new inhibitors of cellular necroptosis. Details on this cell-based assay can be found in Miao and Degterev (Methods Mol. Biol. 2009, 559, 79-93). The Jurkat FADD-deficient I 2.1 cell line used was purchased from ATCC and was maintained in RPMI 1640 medium (Gibco) containing Glutamax and 15% fetal calf serum (Life Technology). Necroptosis was induced by addition of 10 ng/ml of human recombinant TNF-α (Life Technology). Necrostatin-1 (Nec-1, Enzo Life Sciences) was used as model necroptosis inhibitor. Cells were maintained in 75 cm.sup.2 flask and passed every 2 or 3 days. Chemical collections analysed were formatted in 96-well plates with 80 molecules per plate at 10 mM in 100% DMSO. For each collection plate, two plates were prepared: one corresponding to necroptosis-induced with TNF-α, and the other without TNF-α to evaluate the intrinsic toxicity of the tested compound. Cells were seeded at 20000 cells/well, in 40 μl of medium, in a 96-well clear, flat bottom plate (CytoOne, Starlab) before treatment. Then, 40 μl of medium with or without TNF-α at 25 ng/ml were added to all wells in the corresponding plate. Immediately after TNF-α addition, 20 μl of diluted compound at 50 μM were added to the plates. Final concentration of each chemical compound was 10 μM at 0.1% DMSO. Eight positives (Nec-1 at 10 μM final) and eight negative (DMSO) controls were used in each plate to validate the assay. Cells were incubated at 37° C., 5% CO.sub.2 for 24 hours before performing MTS viability assay, described hereafter. Compounds were diluted before to treat cells. Liquid handling was performed using the Nimbus Microlab liquid handler (Hamilton Robotics) under microbiological safety workbench. The 10 mM compounds were diluted at 50 μM directly in cell medium.
(27) Compound 1 has emerged from this screening to be a very efficient necroptosis inhibitor, as discussed below.
Example 3: Anti-Necrotic Effect of Compound 1
(28) Effect on cell viability: Jurkat FADD-deficient I 2.1 cells were treated by TNF-α (10 ng/ml) and increasing concentrations of compound 1 (0.01-0.05-0.10-0.50-1.00-2.50-5.00-10.00-25.00-50.00 μM). Cells were incubated at 37° C., 5% CO.sub.2 for 24 hours before performing MTS viability assay. Cell viability was monitored using CellTiter 96® AQueous Non-Radioactive Cell Proliferation Assay (Promega, Fitchburg, WI, USA), based on the water-soluble tetrazolium compound MTS (3-[4,5-dimethylthiazol-2-yl]-5-[3-carboxymethoxy-phenyl]-2-[4-sulfophenyl]-2H-tetrazolium, inner salt) according to the manufacturers instructions. As it appears from
(29) Effect on two hallmarks of necroptosis: Jurkat FADD-deficient I 2.1 cells were treated by TNF-α (10 ng/ml) and increasing concentrations of compound 1 (0, 1, 5, 10, 20 and 50 μM). Cells untreated by TNF-α are used as control (light grey curves). Cells were incubated at 37° C., 5% CO.sub.2 for 24 hours before performing the measurements of both plasma membrane permeabilization and intracellular ATP levels. Dead cells were detected by FACS analysis of Propidium Iodide-stained nuclei (
Example 4: Compound 1 Cytotoxicity Assays
(30) On Human Peripheral Blood Lymphocytes (hPBLs): hPBLs were treated with increasing concentrations of compound 1 (0, 1, 5, 10, 20, 50 and 100 μM) for 24 hours. Viability was assessed by cell proliferation assay (MTS). % of cell viability was determined using the CellTiter 96® AQ.sub.ueous Non-Radioactive Cell Proliferation Assay (Promega). Data represent the quantitative analysis of six independent experiments with means±SD (n=6 individuals).
(31) As it appears from
(32) On the Human Retinal Pigment Epithelial Cell Line (hRPE-1): hRPE-1 cells were treated with increasing concentrations of compound 1 (0, 0.01, 0.05, 0.1, 0.5, 1, 2.5, 5, 10, 25 and 50 μM) for 24 hours. A colorimetric MTS assay was used to calculate the percentage of cell viability.
(33) As it appears from
Example 5: RIPK1 Autophosphorylation Assay and Binding Assays
(34) RIPK1 Autophosphorylation Assay: Human RIPK1 full length GST-tagged was baculovirally expressed in Sf9 cells according to manufacturer's instructions (Bac-to-Bac expression system, Invitrogen) and purified using gluthation-sepharose beads (GE Healthcare). The elution was made in 50 mM Tris-HCl, pH 8.0 buffer supplemented with 30 mM reduced gluthathione (Sigma). The protocol used to detect the enzymatic activity is adapted from Miao and Degterev (Methods Mol. Biol. 2009, 559, 79-93). Kinase reaction was initiated mixing 5 μl of eluted RIPK1, 5 μl of 3X kinase reaction buffer (5 mM MOPS pH 7.2, 2.5 mM β-glycerophosphate, 4 mM MgCl.sub.2, 2.5 mM MnCl.sub.2, 1 mM EGTA, 0.4 mM EDTA, 50 μg/ml BSA, 0.05 mM DTT), 2 μl H.sub.2O and 3 μl of the tested molecule. The mixture was kept on ice for 10 minutes. During the incubation, the ATP solution was prepared by mixing 5 μl of 3X kinase reaction buffer, 4 μl H.sub.2O, 6 μl cold ATP at 150 μM and 2 μCi of [γ-.sup.32P] ATP. The ATP solution and the tested inhibitor were added to the kinase and incubated for 30 minutes at 30° C. To stop the enzymatic reaction, 5 μl of loading buffer were added and solution was heated for 3 minutes at 95° C. 25 μl of each reaction were loaded per well in pre-cast NuPage 12% Bis-Tris gel (Life Technology). Necrostatin-1, a well-described inhibitor of RIPK1, was used as an internal control. Coomassie staining was performed in order to estimate the total amount of protein loaded on polyacrylamide gel. Autophosphorylated RIPK1 band was visualized on radiographic film after 6h exposition at −80° C.
(35) The results of this test obtained with compound 1 are indicated in
(36) Besides, as shown in
(37) Binding Assays:
(38) (i) Characterization of Kinase Targets of Compound 1 (“KINOMEscan Max”).
(39) This in vitro competition binding assay was used for the profiling of compound 1 against 456 kinases, including eight lipid kinases. This experimental approach quantitatively measures the ability of a compound to compete with an immobilized, active-site directed ligand. The assay is performed by combining three components: DNA-tagged kinase (e.g. RIPK1); immobilized ligand; and a test compound (here compound 1). The ability of compound 1 to compete with the immobilized ligand is measured via quantitative PCR of the DNA tag. The codes reported on the
(40) (ii) Determination of Dissociation Constant (Kd) of Compound 1 for RIPK1 Kinase.
(41) KdELECT is a service of DiscoveRx Corporation, Fremont, USA. This assay is based on a competition binding assay described hereabove. An 11-point 3-fold serial dilution of compound 1 was prepared in 100% DMSO in order to determine the dissociation constant (Kd) at two different temperatures, r.t and 4° C. Kd was then calculated with a standard dose-response curve (reported on
Example 6: In Vitro “Hypoxic Cold Storage” Viability Assays
(42) Human endothelial cells (HAEC) were grown to confluence, then synchronized using depleted media for 16h. For hypothermia/hypoxia, cells were washed twice with PBS then incubated in University of Wisconsin (UW) solution in 95% N.sub.2/5% CO.sub.2 atmosphere at 4° C. for 24 hours. Compound 1, Nec1 or Nec1s were added to the preservation solution at the indicated dilution (04) during hypoxia (
Example 7: In Silicon Analysis of the Theoretical RIPK1-6E11 Complex
(43) The predictive orientation for compound 1 was studied by in silico analysis of the theoretical RIPK1-6E11 complex. Stable contact residues defining a pharmacophore and determined on the most representative structural model included six key amino acids Lys30, Val47, Leu60, Leu78, Tyr88 and Leu90 (
Example 8: Effect of Compound 1 on Both Ferroptosis and Oxytosis Programmed Cell-Death Routes
(44) Murine hippocampal neuronal cell line HT22 was treated with 10 mM of (L)-glutamate (oxytosis initiator) or 1 μM erastin (ferroptosis initiator).
(45) In this assay, 5,000 cells were seeded per well and treated with increasing concentrations of compound 1 (2.50-5.00-10.00-25.00-50.00 μM) with or without 1 μM erastin or 10 mM L-glutamate. Cells were then incubated at 37° C., 5% CO.sub.2 for 24 hours before performing MTS viability assay. Cell viability was monitored using CellTiter 96® AQueous Non-Radioactive Cell Proliferation Assay (Promega, Fitchburg, WI, USA), based on the water-soluble tetrazolium compound MTS (3-[4,5-dimethylthiazol-2-yl]-5-[3-carboxymethoxy-phenyl]-2-[4-sulfophenyl]-2H-tetrazolium, inner salt) according to the manufacturers instructions. As it appears from
Example 9: Effect of Compound 1 on a Cellular Model of Age-Related Macular Degeneration (AMD)
(46) At it was previously shown (Hanus et al. Cell Death Discov. 2016, 2, 16054), NaIO.sub.3 is an oxidizing agent that induces necroptosis in retinal pigment epithelial cell line that can be inhibited by 200 μM of necrostatin-1. The experiment described in the cited literature was reproduced to detect the effect of compound 1. Human retinal pigment epithelial cell line, ARPE-19, was treated with 10 mM of NaIO.sub.3 as cellular model of age-related macular degeneration (AMD). In this assay, 10,000 cells were seeded per well and treated with 20 or 200 μM of compound 1 or Nec-1s (a specific RIPK-1-dependent necroptosis inhibitor) (
Example 10: Effect of Compound 1 on Cold-Induced Cell Death (Cold-Stress Preservation)
(47) At it was previously shown (Ahlenstiel et al. Transplantation 2016, 81(2), 231-239), cold storage induces a cell-death process. Porcine LLC-PK1 cells (kidney proximal tubule cell line) were stored at approximately 4° C. during 24 hours with or without the tested molecules. In this assay, 5,000 cells were seeded per well and treated with increasing concentrations of compound 1 or Nec-1s (0.01, 0.05, 0.10, 0.50, 1.00, 2.50, 5.00, 10.00, 25.00 and 50.00 μM) and stored for 24 hours at 4° C. Cells were then incubated at 37° C., 5% CO.sub.2 before performing MTS viability assay. Cell viability was monitored using CellTiter 96® AQueous Non-Radioactive Cell Proliferation Assay (Promega, Fitchburg, WI, USA), based on the water-soluble tetrazolium compound MTS (3-[4,5-dimethylthiazol-2-yl]-5-[3-carboxymethoxy-phenyl]-2-[4-sulfophenyl]-2H-tetrazolium, inner salt) according to the manufacturers instructions. As it appears from
Example 11: Effect of Compound 1 on H.SUB.2.O.SUB.2.-Induced Necrosis (Anti-Oxidant Property)
(48) The rat embryonic myoblastic H9C2 cells were cultured at 10,000 cells/well in 96-well-plates for 24 hours at 37° C./5% CO.sub.2. Then, cells were treated or not (DMSO) with 800 μM H.sub.2O.sub.2 for 24 hours after a 2 hours pretreatment or not (NT) with 10 μM compound 1 (comp 1), 10 μM Necrostatin-1s (Nec-1s), or 10 μM Ferrostatin-1 (Fer-1). LB corresponds to cells treated with a lysis buffer (100% of cell death). Cell cytotoxicity was determined by a colorimetric assay quantitatively measuring lactate dehydrogenase (LDH) released into the cytosol. As it appears from