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A new ruthenium cyclopentadienyl azole compound with activity on tumor cell lines and trypanosomatid parasites

This article was downloaded by: [New York University] On: 23 June 2015, At: 11:25 Publisher: Taylor & Francis Informa Ltd Registered in England and Wales Registered Number: 1072954 Registered office: Mortimer House, 37-41 Mortimer Street, London W1T 3JH, UK Journal of Coordination Chemistry Publication details, including instructions for authors and subscription information: http://www.tandfonline.com/loi/gcoo20 A new ruthenium cyclopentadienyl azole compound with activity on tumor cell lines and trypanosomatid parasites Esteban Rodríguez Arcea, Cynthia Sarnigueta, Tania S. Moraesb, Marisol Vieitesa, A. Isabel Tomazb, Andrea Medeirosc, Marcelo A. Cominic, Javier Varelad, Hugo Cerecettod, Mercedes Gonzálezd, Fernanda Marquese, M. Helena Garcíab, Lucía Oteroa & Dinorah Gambinoa a Departamento Estrella Campos, Cátedra de Química Inorgánica, Facultad de Química, Universidad de la República, Gral. Flores 2124, 11800 Montevideo, Uruguay b CCMM, Faculdade de Ciências da Universidade de Lisboa, Lisbon, Portugal c Institut Pasteur de Montevideo, Group Redox Biology of Trypanosomes, Montevideo, Uruguay d Grupo de Química Medicinal, Laboratorio de Química Orgánica, Facultad de Ciencias, Click for updates Universidad de la República, Uruguay e Centro de Ciências e Tecnologias Nucleares (C2TN), Instituto Superior Técnico, Universidade de Lisboa, Portugal Accepted author version posted online: 18 Jun 2015. To cite this article: Esteban Rodríguez Arce, Cynthia Sarniguet, Tania S. Moraes, Marisol Vieites, A. Isabel Tomaz, Andrea Medeiros, Marcelo A. Comini, Javier Varela, Hugo Cerecetto, Mercedes González, Fernanda Marques, M. Helena García, Lucía Otero & Dinorah Gambino (2015): A new ruthenium cyclopentadienyl azole compound with activity on tumor cell lines and trypanosomatid parasites, Journal of Coordination Chemistry, DOI: 10.1080/00958972.2015.1062480 To link to this article: http://dx.doi.org/10.1080/00958972.2015.1062480 Disclaimer: This is a version of an unedited manuscript that has been accepted for publication. As a service to authors and researchers we are providing this version of the accepted manuscript (AM). Copyediting, typesetting, and review of the resulting proof will be undertaken on this manuscript before final publication of the Version of Record (VoR). During production and pre-press, errors may be discovered which could affect the content, and all legal disclaimers that apply to the journal relate to this version also. 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This article may be used for research, teaching, and private study purposes. Any substantial or systematic reproduction, redistribution, reselling, loan, sub-licensing, systematic supply, or distribution in any form to anyone is expressly forbidden. Terms & Conditions of access and use can be found at http:// www.tandfonline.com/page/terms-and-conditions 5102 enuJ 32 52:11 ta ]ytisrevinU kroY weN[ yb dedaolnwoD Publisher: Taylor & Francis Journal: Journal of Coordination Chemistry DOI: http://dx.doi.org/10.1080/00958972.2015.1062480 A new ruthenium cyclopentadienyl azole compound with activity on tumor cell lines and trypanosomatid parasites ESTEBAN RODRÍGUEZ ARCEa, CYNTHIA SARNIGUETa, TANIA S. MORAESb, MARISOL VIEITESa, A. ISABEL TOMAZb, ANDREA MEDEIROSc, MARCELO A. COMINIc, JAVIER VARELAd, HUGO CERECETTOd, MERCEDES GONZÁLEZd, FERNANDA MARQUESe, M. HELENA GARCÍAb, LUCÍA OTEROa and DINORAH GAMBINO*a aCátedra de Química Inorgánica, Departamento Estrella Campos, Facultad de Química, Universidad de la República, Gral. Flores 2124, 11800 Montevideo, Uruguay bCCMM, Faculdade de Ciências da Universidade de Lisboa, Lisbon, Portugal cInstitut Pasteur de Montevideo, Group Redox Biology of Trypanosomes, Montevideo, Uruguay dGrupo de Química Medicinal, Laboratorio de Química Orgánica, Facultad de Ciencias, Universidad de la República, Uruguay eCentro de Ciências e Tecnologias Nucleares (C2TN), Instituto Superior Técnico, Universidade de Lisboa, Portugal As part of our efforts to develop organometallic ruthenium compounds bearing activity on both trypanosomatid parasites and tumor cells, a new Ru(II)-cyclopentadienyl clotrimazole complex, [RuCp(PPh ) (CTZ)](CF SO ) where Cp = cyclopentadienyl, CTZ = clotrimazole, was 3 2 3 3 synthesized and characterized. The compound was evaluated in vitro on T. cruzi (Y strain), the infective form of T. brucei brucei strain 427 (cell line 449), on three human tumor cell lines with different sensitivity to cisplatin (A2780, ovary; MCF7, breast; HeLa, cervix) and on J774 murine macrophages as mammalian cell model. The new compound is more cytotoxic on T. cruzi and on the tumor cell lines than the reference drugs (Nifurtimox and cisplatin, respectively). In addition, complexation of the bioactive CTZ to the {RuCp(PPh )} leads to significant increase of the 3 antiparasitic and antitumoral activity. To get insight into the potential “dual” mechanism of antiparasitic action emerging from the presence of Ru(II) and CTZ in a single molecule, the inhibitory effect of this new complex on the biosynthesis of T. cruzi sterols of membrane and the interaction with DNA were studied. Although the tested complex does not affect DNA, it affects the T. cruzi biosynthetic pathway of conversion of squalene to squalene oxide. According to the results here reported, [RuCp(PPh ) (CTZ)][CF SO ] could be considered a prospective 3 2 3 3 antiparasitic and/or antitumoral agent that deserves further evaluation. *Corresponding author. Email: dgambino@fq.edu.uy 1 5102 enuJ 32 52:11 ta ]ytisrevinU kroY weN[ yb dedaolnwoD Keywords: Organometallic ruthenium compound; Clotrimazole; Cancer; Trypanosomes 1. Introduction American Trypanosomiasis (Chagas disease) and Human African Trypanosomiasis (Sleeping sickness), diseases caused by the genetically related trypanosomatid parasites Trypanosoma cruzi and Trypanosoma brucei (Trypanosoma brucei gambiense and Trypanosoma brucei rhodesiense), respectively, constitute major health concerns in the developing world [1-6], although in the context of the current global society the impact of these diseases is not strictly geographically confined [7, 8]. The available chemotherapeutics are decades old and suffer from limited efficacy, undesirable collateral effects and development of resistance. Therefore, novel strategies for the development of more efficient and less toxic agents, that could also circumvent drug resistance, are urgently needed. Based on the recognition of common targets in both parasites, drugs that are active against both protozoa could offer an innovative approach for drug discovery [4]. In a not very different context, cancer is the second largest cause of death in developed countries, and the number of worldwide deaths from this disease is projected by WHO to rise to over 13.1 million people in 2030 [9]. Metabolic pathways of highly proliferative cells, like tumor cells and parasites, are expected to have resemblances that could lead to a correlation between antiparasitic and antitumor activities. In fact, several antitumoral drugs also show significant antiparasitic activity and vice versa [10]. The development of bioactive metal-based compounds through inorganic medicinal chemistry strategies has been recognized as a promising and attractive approach in the search for antitumoral and antiparasitic drugs [11-27]. In particular, ruthenium complexes have been the most widely studied non-platinum, metal-based anticancer candidates and hold potential as successful alternatives in cancer treatment [10, 13, 16, 18, 28, 29]. In this perspective, ruthenium-cyclopentadienyl complexes presenting N-heteroaromatic ligands have exhibited excellent antitumoral properties and are very promising agents for cancer therapy [30-34]. In addition, ruthenium classical coordination compounds and organometallic compounds have been recognized as prospective agents against trypanosomatid parasites [35-42]. The design of antiparasitic compounds which combine ligands 2 5102 enuJ 32 52:11 ta ]ytisrevinU kroY weN[ yb dedaolnwoD bearing antitrypanosomal activity and pharmacologically active metals in a single chemical entity could provide drugs capable of modulating multiple targets simultaneously. Our group has been working on the development of ruthenium complexes with different families of trypanosomicidal ligands [10, 21, 38-42]. In particular, Ru-p-cymene compounds with this type of ligands were developed and studied in depth [39-41]. Based on a similar approach, Sánchez-Delgado's group performed leading research in the field by developing ruthenium and other metal complexes with the bioactive azole derivative clotrimazole (CTZ, figure 1) as ligand [35, 37, 43-45]. CTZ is a well-known antifungal agent, acting, like other azole derivatives, as a sterol biosynthesis inhibitor in fungi. In particular, it inhibits the cytochrome P-450 dependent C(14) demethylation of lanosterol to ergosterol. The sterol biosynthetic pathways of trypanosomatids are similar to those of pathogenic fungi and therefore CTZ also displays moderate activity on T. cruzi [45, 46]. Five classical Ru-CTZ coordination compounds and four organometallic Ru-p-cymene CTZ compounds were developed by this group. Most of them displayed higher antiproliferative activity than free CTZ on T. cruzi suggesting a synergistic effect [35, 37, 44, 45]. The biological profile clearly changed when modifying the coordination sphere of the Ru(II), making further modification of the Ru(II)-CTZ environment interesting [10, 37]. As part of our efforts to rationally develop organometallic ruthenium compounds bearing activity on different trypanosomatid parasites but also on tumor cells, a new Ru(II)- cyclopentadienyl CTZ complex, [RuCp(PPh ) (CTZ)](CF SO ) (abbreviated as RuCpCTZ), was 3 2 3 3 synthesized and characterized in this work. The compound was evaluated in vitro on the epimastigote form of T. cruzi (Y strain), the infective form of T. brucei brucei strain 427 (cell line 449) and the tumor cell lines A2780 (ovary), MCF7 (breast) and HeLa (cervix). To test selectivity, the cytotoxicity on J774 murine macrophages as mammalian cell model was evaluated. In order to get insight into the potential “dual” mechanism of antiparasitic action, emerging from the presence of Ru(II) and CTZ in a single molecule, the inhibitory effect of the complex on the biosynthesis of T. cruzi membrane sterols and the interaction with DNA were studied. 2. Results and discussion 3 5102 enuJ 32 52:11 ta ]ytisrevinU kroY weN[ yb dedaolnwoD Clotrimazole (CTZ, figure 1) is a classic imidazole ligand with a single nitrogen coordination site (N1) capable of binding to a metal ion. It usually reacts readily under mild conditions [37, 44, 45, 47-50]. A Ru-cyclopentadienyl complex (figure 2) with this bioactive ligand was synthesized with high purity and good yield by the reaction of the precursor [RuCp(PPh ) Cl] 3 2 and CTZ (figure 1). Unfortunately, it was impossible to obtain adequate single crystals of the complex for performing structural characterization by X-ray analysis. Nevertheless, the compound was fully characterized in the solid state and in solution by using elemental analyses, conductometric measurements, and FTIR and 1H-, 13C- and 31P-NMR spectroscopies. Elemental analyses agree with the proposed formula. The molar conductivity value obtained for the complex in DMSO solution demonstrates that it is a 1:1 electrolyte 51. No conductivity changes were observed during at least 5 days at 25 °C, hence suggesting that the complex is stable in air in DMSO. Relevant solid FT-IR absorption bands were tentatively assigned to molecular vibration modes of the coordinated CTZ ligand 37, the triflate counterion 34 and the {RuCp} moiety 52. Three new medium to strong bands were identified in the low wavenumber region (600- 400 cm-1) at 572, 533 and 522 cm-1 that could correspond to metal-ligand vibrations. A detailed characterization of the new complex in solution was performed by NMR spectroscopy. The resonances were assigned on the basis of 1D 1H-NMR, and 2D homonuclear correlated spectroscopy (COSY) and heteronuclear correlation (HSQC) experiments as well as on the comparison with those for CTZ and other previously reported Ru(II)–CTZ complexes 37, 44, 45. Table 1 shows the 1H-NMR chemical shifts () of the complex. Figure 2 shows the corresponding numbering scheme. 1H-NMR integrations and signal multiplicities were in agreement with the proposed formula and structure. 1H-NMR resonance of the 5-cyclopentadienyl ring is in the characteristic range of cationic ruthenium(II) complexes [30]. The heteroaromatic ring protons of CTZ ligand are shielded upon coordination to Ru(II) centre, with exception of the protons adjacent to the coordinated N; these two protons, H2 and H4, are deshielded by 0.31 and 0.16 ppm, respectively. This effect, perhaps due to the influence of the organometallic moiety on the ring current of the heteroaromatic ligand, was also observed in other piano stool Ru(II) η5-cyclopentadienyl [34] and Ru(II) η6-arene compounds with pyridylpyrazole, pyridylimidazole [53], and phenoxazine ligands [54], coordinated by N of the heteroaromatic rings. 4 5102 enuJ 32 52:11 ta ]ytisrevinU kroY weN[ yb dedaolnwoD 13C-NMR spectra revealed the same general effect observed for the protons, although it was not possible to unequivocally identify all the carbons, showing high complexity in the 127- 138 ppm region with signals corresponding to both PPh and CTZ ligands. However, analysis of 3 the HSQC spectra allowed clear identification of C2 (138.2 ppm) and C6 (130.0 ppm) of the CTZ ligand. As previously reported, these are the most shifted CTZ carbon signals as a consequence of complexation [44]. The signal at 83.2 ppm corresponds to equivalent carbons of the Cp ligand [52]. Furthermore, a signal at 121.9 ppm corresponding to CF SO - counterion was 3 3 also observed [55]. 31P-NMR spectra of the complex show a single sharp signal for the triphenylphosphane coligand at ~41 ppm, and the expected deshielding upon coordination is in accord with the σ-donor character of this ligand. 2.1. Biological results 2.1.1. Antiparasitic activity The effect of RuCpCTZ on the epimastigote form of T. cruzi, Y strain, was evaluated and compared with that of CTZ. The results are presented in table 2. RuCpCTZ showed high cytotoxic activity on this parasite with an IC value in the submicromolar range. A six-fold 50 increase of activity was observed with respect to free CTZ. In addition, the complex showed an activity about 30 times higher than the reference drug Nifurtimox (IC = 8.0 µM) [56]. 50 For T. brucei brucei (strain 427), the new ruthenium compound showed growth inhibitory activity, inducing a dose-dependent antiproliferative effect on parasites treated for 24 h (table 2). CTZ displayed a lower cytotoxicity towards T. brucei (IC >25 μM) than against T. cruzi (IC 50 50 1.8 μM). This selectivity is in good agreement with the sterol content in each species and life cycle stage of the parasites; T. cruzi epimastigotes contain 40% ergosterol whereas bloodstream T. brucei contains predominantly cholesterol incorporated from the medium by a receptor- mediated endocytic mechanism [57]. As a consequence of complex formation, the potency of RuCpCTZ against the infective form of African trypanosomes is 40-fold higher (IC = 0.6 μM) 50 compared to the activity of the parental compound (IC >25 μM). This suggests that the RuCp 50 moiety of the CTZ complex is a major determinant for the biological activity of this compound against T. brucei. 5 5102 enuJ 32 52:11 ta ]ytisrevinU kroY weN[ yb dedaolnwoD In addition, RuCpCTZ showed a lower IC value on T. brucei brucei (strain 427) than 50 previously reported [Ru (p-cymene) (L) ]X complexes, where L = 5-nitrofuryl containing 2 2 2 2 thiosemicarbazones and X = Cl- or PF - [39]. 6 2.1.2. Cytotoxicity on J774 murine macrophages The specificity of the antitrypanosomal activity of RuCpCTZ and CTZ was evaluated by analyzing their cytotoxicity against a murine macrophage-like cell line (J774). The results are depicted in table 2. These results enable comparison of selectivity indexes (SI) of RuCpCTZ and CTZ calculated as SI = IC macrophages / IC parasites. 50 50 Although RuCpCTZ showed higher cytotoxicity than CTZ, RuCpCTZ showed fairly good selectivities towards both parasites (T. cruzi and T. brucei) with respect to mammalian cells (J774 murine macrophages). In the case of T. brucei, the comparison of the selectivity index values reveals that RuCpCTZ (SI = 3) is not only more potent but also more selective than CTZ (SI  2). 2.1.3. Cytotoxic activity in human tumor cells The cytotoxic activities of RuCpCTZ and CTZ were evaluated on three human cancer cells, ovarian A2780, breast MCF7 and cervical HeLa, representing common cancer diseases and also presenting different degrees of resistance to metallodrugs. The cells were treated with the compounds in the concentration range 100 nM - 100 µM during a 72 h incubation period. A colorimetric MTT assay was used to evaluate the cytotoxic activity measured as the half- inhibitory concentration (IC ). IC values found for RuCpCTZ ranged between 0.5 and 5 µM 50 50 while those obtained for CTZ spanned between 6 µM and 20 µM (table 3). The A2780 cells were particularly much more sensitive to both compounds, in particular to RuCpCTZ, than the MCF7 and HeLa cells. Complex formation seems to be responsible for the observed higher sensitivity of the cells to the Ru complex relative to the free ligand. Cisplatin was presented for comparison, since it is the metallodrug in clinical application. In the cell lines studied, the cytotoxic activity of RuCpCTZ is three to five times higher than that of the reference drug cisplatin. In addition, RuCpCTZ is 4- to 63-fold more active on A2780 tumor cells and 2- to 12-fold more active on MCF7 cells than a series of organometallic Ru compounds previously developed by our group 6 5102 enuJ 32 52:11 ta ]ytisrevinU kroY weN[ yb dedaolnwoD [41] and shows a significantly lower IC value on HeLa and MCF7 cells than recently reported 50 Ru(II) cytotoxic compounds [59, 60]. 2.2. Mechanism of antiparasitic action RuCpCTZ was designed to include a bioactive ligand and the pharmacologically relevant Ru-cyclopentadienyl center, searching for a dual mechanism of action by affecting different targets or biologically significant processes in the parasite. Accordingly, studies were performed in order to validate proposed targets and to get insight into the probable mechanism of antiparasitic action of RuCpCTZ. 2.2.1. Inhibition of sterol membrane biosynthesis CTZ mechanism of action involving -14 C demethylase inhibition from the sterol membrane biosynthesis pathway is well known [45, 46]. Therefore, the effect of RuCpCTZ on this biochemical pathway was studied [61]. The obtained results (figure 3) showed that, while ergosterol is present in all the studied treatments, its concentration seemed to be lower for those incubations treated with RuCpCTZ, CTZ or terbinafine than for the negative control. As expected, treatment with CTZ produced accumulation of lanosterol due to inhibition of this enzyme that catalyzes the conversion of lanosterol to ergosterol. On the other hand, the treatment with RuCpCTZ led to squalene accumulation, effect observed for terbinafine but not for free CTZ. The results indicate that RuCpCTZ affects sterol membrane biosynthesis at the same step as terbinafine, inhibiting squalene-2,3-epoxidase enzyme [62]. 2.2.2. Interaction with DNA Metal compounds and particularly ruthenium ones are usually able to interact with DNA. In addition, DNA has been proposed as a parasitic target for other Ru-CTZ complexes [45]. Therefore, this biomolecule was investigated as a potential target of RuCpCTZ. The well- established fluorescent DNA probe ethidium bromide (EB) was used to detect RuCpCTZ interaction with DNA. When EB is specifically intercalated into double stranded DNA, the {DNA-EB} adduct shows a strong fluorescence emission (λ = 510 nm) with a maximum at exc 594 nm in our experimental conditions [63, 64]. Results obtained for the titration of this {DNA-EB} adduct with RuCpCTZ are summarized in figure 4. No quenching in the emission of 7 5102 enuJ 32 52:11 ta ]ytisrevinU kroY weN[ yb dedaolnwoD {DNA-EB} was observed upon increasing Ru complex concentration. This result indicates no strong interaction (through intercalation or covalent binding) between the Ru complex and DNA, in agreement with what is observed in the direct quantification test for binding of RuCpCTZ to DNA described in section 4.5.2. The DNA binding level obtained by this method (nmol Ru / mg DNA ≈ 1) is negligible when compared to the values ranging from 40-300 nmol Ru / mg DNA reported for other Ru compounds that efficiently interact with DNA and for antitumor metal complexes having DNA as molecular target [39, 42, 65, 66]. 3. Conclusion A new organometallic ruthenium clotrimazole compound, [RuCp(PPh ) (CTZ)](CF SO ), was 3 2 3 3 synthesized and fully characterized. This compound showed high cytotoxic activity on trypanosomatid parasites (T. cruzi and T. brucei), validating our proposal of the development of broad spectrum metal-based drugs active against multiple trypanosomatid protozoa as a potentially innovative approach for antiparasitic drug discovery. In addition, the compound shows high cytotoxicity on three tumor cell lines with different sensitivity to that of cisplatin. The compound is more cytotoxic on the parasites and on the tumor cell lines than the reference antitrypanosomal and antitumoral drugs. In all cases [RuCp(PPh ) (CTZ)](CF SO ) is more 3 2 3 3 active than free CTZ. The experiments performed gave insight into the probable mechanism of antitrypanosomatid action of the ruthenium compound. It affects the T. cruzi biosynthetic pathway responsible for the conversion of squalene to squalene oxide. According to the results obtained, [RuCp(PPh ) (CTZ)](CF SO ) could be considered a prospective antiparasitic and/or 3 2 3 3 antitumor agent that deserves further development. 4. Materials and methods 4.1. Materials All common laboratory chemicals were purchased from commercial sources and used without purification. All syntheses were carried out under dinitrogen using Schlenk techniques and the solvents used were dried by standard methods [67]. The precursor [Ru(η5-C H )(PPh ) Cl] was 5 5 3 2 synthesized from RuCl , triphenylphosphane and cyclopentadienyl in ethanol according to a 3 previously reported procedure [68]. 8 5102 enuJ 32 52:11 ta ]ytisrevinU kroY weN[ yb dedaolnwoD 4.2. Synthesis of [RuCp(PPh ) (CTZ)](CF SO ) 3 2 3 3 [RuCp(PPh ) Cl] (0.5 mmol, 0.320 g) and AgCF SO (0.45 mmol, 0.116 g) were dissolved in 3 2 3 3 dried distilled dichloromethane. The reaction mixture was kept at room temperature for 1.5 h. CTZ (0.5 mmol, 0.172 g) was added and allowed to react at room temperature for about 2 h. The reaction was followed by TLC (stationary phase silica gel GF254; mobile phase petroleum ether/acetone 1:1v/v) until no reactants were detected. AgCl was filtered off on line under dinitrogen pressure and the solvent was evaporated in vacuo. The yellow complex was obtained by separation through silica column under N using petroleum ether/acetone (1:1v/v) as mobile 2 phase. [RuCp(PPh ) (CTZ)](CF SO ). Yield: 190 mg, 32%. Yellow solid. Anal (%) calc. for 3 2 3 3 C H ClF N O P RuS (%): C, 64.89; H, 4.42; N, 2.36; S, 2.71. Found: C, 64.75; H, 4.44; N, 64 52 3 2 3 2 2.38; S, 2.67. FT-IR (KBr, cm-1, m = medium, w = weak, s = strong): 3055 (mw), (C-H from arene), (C H ); 1479 (mw), 1446 (ms); 1430 (m), (C=N and/or C=C from arene); 696 (s), 5 5 520(s); 1273 (s), (CF SO ).  (DMSO) 380 nm ( = 2.8×103 M-1cm-1).  (DMSO) = 3 3 M 34.6 Scm2mol-1. 4.3. Physicochemical characterization C, H and N analyses were carried out with a Thermo Scientific Flash 2000 elemental analyzer. Conductimetric measurements were done over time (5 days) at 25 °C in 10−3 M DMSO solutions using a Conductivity Meter 4310 Jenway to determine the type of electrolyte and to assess the stability of the complex in such medium [51]. The FTIR absorption spectra (4000-300 cm–1) of the complex, the precursor and CTZ were measured as KBr pellets with a Shimadzu IRPrestige-21 instrument. UV-vis spectrum was measured in DMSO from 300-800 nm with a spectrophotometer Shimadzu UV 1603 instrument. 1H-NMR and 13C-NMR spectra were recorded in DMSO-d at 30 °C on a Bruker DPX-400 instrument (at 400 MHz and 100 MHz, 6 respectively). Homonuclear (COSY) and heteronuclear correlation experiments (2D-HETCOR), HSQC (heteronuclear single quantum correlation), were carried out with the same instrument. Tetramethylsilane was used as the internal standard. 31P{1H}-NMR spectrum was recorded in DMSO-d on a Bruker AVANCE III 400 instrument (at 161.98 MHz). Chemical shifts are 6 reported in ppm. 9 5102 enuJ 32 52:11 ta ]ytisrevinU kroY weN[ yb dedaolnwoD 4.4. Biological activity 4.4.1. Anti-T. cruzi activity Trypanosoma cruzi epimastigotes (Y strain) were grown at 28 °C in an axenic milieu (BHI-Tryptose) supplemented with 5% fetal bovine serum (FBS) as previously described [69, 70]. Cells from a 10-day-old culture (stationary phase) were inoculated into 50 mL of fresh culture milieu to give an initial concentration of 106 cells/mL. Cell growth was followed every day by measuring the absorbance of the culture at 600 nm. Before inoculation, the milieu was supplemented with the indicated amount of the studied compound from a freshly prepared stock solution in DMSO. Nifurtimox (Nfx) was used as the reference trypanosomicidal drug. The final concentration of DMSO in the culture milieu never exceeded 0.4%, and the control was run in the presence of 0.4% DMSO and in the absence of the studied compounds. No effect on epimastigote growth was observed due to the presence of up to 1% DMSO in the culture milieu. The percentage of inhibition (PGI) was calculated as follows: PGI (%) = {1 - [(Ap - A0p)/(Ac - A0c)]}  100, where Ap = A of the culture containing the studied compound at day 5; 600nm A0p = A of the culture containing the studied compound just after addition of the inocula 600nm (day 0); Ac = A of the culture in the absence of the studied compound (control) at day 5; 600nm A0c = A in the absence of the studied compound at day 0. To determine IC values (50% 600nm 50 inhibitory concentrations) parasite growth was followed in the absence (control) and presence of increasing concentrations of the corresponding compound. At day 5, the absorbance of the culture was measured and related to the control. The IC value was taken as the concentration of 50 compound under study necessary to reduce the absorbance ratio to 50%. 4.4.2. Activity on T. brucei brucei strain 427 The infective form of T. brucei brucei strain 427, cell line 449 (encoding one copy of the tet-represor protein: PleoR [71]), was aerobically cultivated in a humidified incubator at 37 °C with 5% CO in HMI-9 medium [72] supplemented with 10% (v/v) fetal calf serum (FCS), 2 10 U/mL penicillin, 10 µg/mL streptomycin and 0.2 g/mL phleomycin. The assay was performed as previously described [73]. Ten mM stock solutions of the compounds were prepared using DMSO as solvent and then diluted in culture medium to obtain seven experimental concentrations (from 25 to 0.3 µM). The maximum DMSO concentration used in the cytotoxicity assays did not exceed 1%, which is known to be nontoxic to T. brucei parasites. 10 5102 enuJ 32 52:11 ta ]ytisrevinU kroY weN[ yb dedaolnwoD Compounds, DMSO (up to 1%) or medium were immediately added at the concentrations described above and the culture plate was incubated at 37 °C with 5% CO . After 24 h, living 2 parasites were counted with a Neubauer chamber under the light microscope. Each condition was tested in triplicate. For each compound concentration, cytotoxicity was calculated according to the following equation: Cytotoxicity (%) = {(experimental value - DMSO control)/(growth control - DMSO control)} × 100. The data were plotted as percentage cytotoxicity versus drug concentration. IC 50 values were obtained from dose response curves fitted to a sigmoidal equation (Boltzmann model) or extrapolated from non-linear fitting plots. 4.4.3. Cellular viability assays in human tumor cells Three human tumor cell lines: A2780 ovarian, MCF7 breast and HeLa cervical (ATCC) were cultured in medium (Gibco, Invitrogen) RPMI 1640 (A2780) or DMEM containing GlutaMax I (MCF7, HeLa) supplemented with 10% fetal bovine serum and 1% antibiotics at 37 °C, 5% CO 2 in a humidified atmosphere (Heraeus, Germany). The cells were adherent in monolayers and upon confluency were harvested by digestion with trypsin–EDTA (Gibco). Cell viability was evaluated by using a colorimetric assay based on the tetrazolium salt MTT [74]. For this purpose, cells were seeded in 200 µL aliquots in complete media RPMI or DMEM into 96-well plates. Adequate cellular densities were chosen to ensure exponential growth of untreated control samples throughout the experiments (2-5 × 104 cells/well). For 24 h cells were allowed to adhere followed by the addition of dilution series of the test compounds in fresh medium (200 µL/well). Ligand and complex were first solubilized in DMSO and then in medium, and added to final concentrations in the range 100 nM - 100 µM. DMSO in cell culture medium did not exceed 1% (final concentration). DMSO does not show cytotoxic effect by itself at this concentration. After continuous treatment of the cells with the compounds for 72 h at 37 °C with 5% CO , the 2 medium was replaced by 200 µL of MTT solution in PBS (0.5 mg/mL). After 3-4 h incubation, MTT solutions were removed and the formazan product formed was dissolved in DMSO (200 µL/well). The cellular viability was evaluated by measuring the absorbance at 570 nm using a plate spectrophotometer (PowerWave Xs, Bio-tek Instruments, VT, USA). The cytotoxic effects of compounds were quantified by calculating the IC value based on nonlinear regression 50 11 5102 enuJ 32 52:11 ta ]ytisrevinU kroY weN[ yb dedaolnwoD analysis of dose-response curves (GraphPad Prism version 4.0). Evaluation was based on at least two independent experiments, each comprising six replicates per concentration. 4.4.4. Cytotoxicity on murine macrophages The J774 mouse macrophage cell line was cultivated in a humidified 5% CO /95 % air 2 atmosphere at 37 °C in DMEM medium supplemented with 10% (v/v) FCS, 10 U/mL penicillin and 10 µg/mL streptomycin. Stock solutions of the compounds to be tested were prepared as described for anti-T. brucei activity tests and diluted in culture medium to obtain seven experimental concentrations (from 100 to 0.5 µM). The maximum DMSO concentration used did not exceed 1%, which is known to be tolerated by murine macrophages from J774 cell line. Each condition was tested in triplicate. The cytotoxic effect of the compounds towards macrophages was evaluated by colorimetric assay of cell viability with a tetrazolium salt (WST-1 reagent). The absorbance of the formazan dye produced by metabolically active cells was measured at 450 nm (reference wavelength at 630 nm) with an EL 800 microplate reader (Biotek). The methodological procedure is essentially as described previously [39]. Cytotoxicity was calculated and IC values were obtained as described for cytotoxicity on T. brucei brucei. 50 4.5. Mechanism of action 4.5.1. Inhibition of the biosynthesis of membrane sterols Epimastigote form of T. cruzi (Tulahuen 2 strain) was maintained in an axenic medium (BHI-Tryptose). The experiments were carried out in cultures at 28 °C and with strong aeration. The studied compound was added to the culture (10×106 cells/mL) as a DMSO solution in a concentration equivalent to IC . Compound and parasites were incubated 72 h at 28 °C. Final 50 concentration of DMSO in the culture never exceeded 0.4%. A control including 0.4% DMSO was prepared. Positive controls with terbinafine were also incubated. After incubation the control and drug-treated parasites were centrifugated at 3000 rpm during 10 min and then the supernatants were discarded and the pellets were collected and suspended in 1 mL phosphate buffer (0.05 M, pH 7.4) and centrifugated at 3000 rpm during 10 min. The pellets were treated with 1 mL chloroform/methanol (2:1) during 12 h at 4 °C. After addition of saturated NaCl aqueous solution a new extraction with 600 μL chloroform and an extraction with 600 μL hexane were performed. The total volume of extraction was seeded on a silica TLC plate. Squalene was 12 5102 enuJ 32 52:11 ta ]ytisrevinU kroY weN[ yb dedaolnwoD identified by two chromatographic runs with hexane as mobile phase and ergosterol by one run with n-hexane:AcOEt (8:2). The TLC was developed by UV light and with iodine vapors. The compound was seeded together with a negative control, a positive control (terbinafine) and standards of squalene, lanosterol, cholesterol and ergosterol [75]. 4.5.2. Interaction with DNA Fluorescence studies. Experiments for competitive binding to calf-thymus DNA (ct-DNA, SIGMA, Type I, No. D-1501) with ethidium bromide (EB, SIGMA) were carried out in 10 mM Tris-HCl buffer at pH 7.4. Millipore® water was used for the preparation of all aqueous solutions. Fluorescence measurements were carried out on individually prepared samples to ensure the same pre-incubation time for all samples in each assay. Due to the low solubility of the complex in aqueous media, DMSO was used to prepare concentrated stock solutions followed by appropriate dilution to obtain the targeted concentration and the same content of DMSO (5% v/v) in the final samples. DNA stock solutions were prepared by hydrating ct-DNA in Tris-HCl buffer (1 mg/mL, ~2 mM.nuc-1) during 3-4 days at 4 °C, swirling the solution about 4-5 times a day until full dissolution was attained, and a clear solution was obtained. This solution was kept at 4 °C (in the refrigerator) between measurements and discarded after 4 days. The concentration of each stock solution was determined by UV spectrophotometry using the molar extinction coefficient ε = 6600 M−1cm−1 [76]. A 5 mM EB solution was prepared in (260nm) Tris-HCl buffer. ct-DNA was pre-incubated with EB at 4 °C for 24 h. Samples were prepared with a total concentration of DNA and of EB of 20 µM and 10 µM, respectively, varying the total complex concentration from 1-110 µM. They were incubated at 37 °C for 30 min. Samples with complex alone and samples with complex and EB but no DNA were used as blanks. Fluorescence spectra were recorded from 520 nm to 650 nm at an excitation wavelength of 510 nm on a Shimadzu RF-5301PC spectrofluorimeter. Fluorescence emission intensity was corrected for the absorption and emission inner filter effect at the maximum emission wavelength (594 nm) using the UV-Visible absorption data recorded for each sample according to the following equation [77]. 13 5102 enuJ 32 52:11 ta ]ytisrevinU kroY weN[ yb dedaolnwoD ct-DNA interaction experiments. The complex was tested for their global DNA interaction ability using native ct-DNA by a modification of a previously reported procedure [78, 79]. ct-DNA (50 mg) was dissolved in water (30 mL) overnight. Solutions of the complex in DMSO (spectroscopy grade) (1 mL, 10-3 M) were incubated at 37 °C with solution of ct-DNA (1 mL) during 96 h. DNA/complex mixtures were exhaustively washed to eliminate the un-reacted complex. Quantification of bound metal was done by atomic absorption spectroscopy on a Perkin Elmer 5000 spectrometer. Standards were prepared by diluting a metal standard solution for atomic absorption spectroscopy. 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Chem Soc., 114, 9253 (1992). 19 5102 enuJ 32 52:11 ta ]ytisrevinU kroY weN[ yb dedaolnwoD Table 1. 1H-NMR chemical shift values () in ppm for [RuCp(PPh ) (CTZ)](CF SO ) 3 2 3 3 and CTZ in DMSO-d at 30 °C. 6 1H-NMR/ (integration) (multiplicity) H Proton number [RuCp(PPh ) (CTZ)](CF SO ) CTZ 3 2 3 3 H2 7.75 (1, s) 7.44 (1, t) H4 7.07 (1, dd) 6.91 (1, t) H5 6.72 (1, dd) 6.73 (1, t) H8 6.79 (1, m) 6.80 (1, d) H11 7.38* (1, m) 7.40 (1, t) H9, H10, H14, H16, H18, H20, 6.79 (8, m) 7.32 (8, m) H22, H24 H15, H17, H21, H23 6.66 (4, m) 7.01 (4, m) Cp 4.50 (5, s) - PPh 7.38 (12, m), 7.27 (6, m), 7.21 (12, m) - 3 *overlapped with PPh 3 20 5102 enuJ 32 52:11 ta ]ytisrevinU kroY weN[ yb dedaolnwoD Table 2. In vitro activity (measured as the IC value, the half inhibitory concentration) against 50 T. cruzi, T. brucei brucei, cytotoxicity on murine macrophages and selectivity index (SI) values of RuCpCTZ and CTZ. T. cruzi T. brucei macrophages Compound SI (fold)a SI (fold)b IC / M IC / M IC / M 50 50 50 RuCpCTZ 0.25 ± 0.08 0.6 ± 0.1 1.9 ± 0.1 8 3 CTZ 1.8 ± 0.4  25 55.1 ± 0.5 31  2 a SI: IC macrophages / IC T. cruzi. b SI: IC macrophages / IC T. brucei brucei. Results are 50 50 50 50 the mean value of three different independent experiments. 21 5102 enuJ 32 52:11 ta ]ytisrevinU kroY weN[ yb dedaolnwoD Table 3. IC values found for RuCpCTZ and CTZ in three different human 50 cancer cells lines after 72 h incubation at 37 °C. Cisplatin is included for comparison. IC (µM) Compound 50 A2780 MCF7 HeLa CTZ 5.7 ± 0.9 20.5 ± 8.1 18.4 ± 7.9 RuCpCTZ 0.54 ± 0.1 5.4 ± 2.0 3.6 ± 1.1 Cisplatin 1.9 ± 0.1 a 28 ± 6.0 a 7.00 ± 2.70 b a From reference [33]. b From reference [58]. 22 5102 enuJ 32 52:11 ta ]ytisrevinU kroY weN[ yb dedaolnwoD Figure 1. Scheme for the synthesis of [RuCp(PPh ) (CTZ)](CF SO ). 3 2 3 3 23 5102 enuJ 32 52:11 ta ]ytisrevinU kroY weN[ yb dedaolnwoD CF SO 10 3 3 9 11 4 5 8 7 12 Cl 20 Ru N N 6 21 19 Ph P 3 2 14 13 22 Ph 3 P 18 24 23 15 17 16 Figure 2. Proposed chemical structure for [RuCp(PPh ) (CTZ)](CF SO ). 3 2 3 3 24 5102 enuJ 32 52:11 ta ]ytisrevinU kroY weN[ yb dedaolnwoD Figure 3. TLC of the lipidic extract of T. cruzi after treatment of the parasite with the compounds under study and terbinafine. The sterols used as standards are included. The TLC was revealed with vapors of iodine. Lane 1: RuCpCTZ, Lane 2: CTZ, Lane 3: terbinafine, Lane 4: negative control, Lane 5: ergosterol, Lane 6: lanosterol, Lane 7: cholesterol, Lane 8: squalene. Relevant pathways of the sterol biosynthesis are shown in the attached scheme (right). 25 5102 enuJ 32 52:11 ta ]ytisrevinU kroY weN[ yb dedaolnwoD 0 M 1 M 250 5 M 7.5 M 140 15 M m n 0 1 ) 200 2 3 3 5 0 5    M M M m n 120 5 = 150 4 4 0 5   M M 4 9 100 c 5 x 50 M @ ).u (  e 100 60 M F rro c 80 .a ( F 50 I % 60 I 0 40 540 560 580 600 620 640 0 10 20 30 40 50 60 /nm [RuCpCTZ]/M Figure 4. Left: Fluorescence data obtained for the competitive binding study of RuCpCTZ to {EB-DNA}. Right: Relative fluorescence intensity (%) at  = 594 nm with increasing em RuCpCTZ concentration. (C = 20 µM, C = 10 µM, samples prepared in 2% DMSO/Tris DNA EB HCl medium, 30 min incubation at 37 °C). 26 5102 enuJ 32 52:11 ta ]ytisrevinU kroY weN[ yb dedaolnwoD 27 5102 enuJ 32 52:11 ta ]ytisrevinU kroY weN[ yb dedaolnwoD