Abstract More than two million people worldwide are affected by life-threatening, invasive fungal infections annually. Candida species are the most common cause of nosocomial, invasive fungal infections and are associated with mortality rates above 40%. Despite the increasing incidence of drug-resistance, the development of novel antifungal formulations has been limited. Here we investigate the antifungal mode of action and therapeutic potential of positively charged, synthetic peptide mimics to combat Candida albicans infections. Our data indicates that these synthetic polymers cause endoplasmic reticulum stress and affect protein glycosylation, a mode of action distinct from currently approved antifungal drugs. The most promising polymer composition damaged the mannan layer of the cell wall, with additional membrane-disrupting activity. The synergistic combination of the polymer with caspofungin prevented infection of human epithelial cells in vitro, improved fungal clearance by human macrophages, and significantly increased host survival in a Galleria mellonella model of systemic candidiasis. Additionally, prolonged exposure of C. albicans to the synergistic combination of polymer and caspofungin did not lead to the evolution of tolerant strains in vitro. Together, this work highlights the enormous potential of these synthetic peptide mimics to be used as novel antifungal formulations as well as adjunctive antifungal therapy. Subject terms: Antifungal agents, Target identification, Target validation __________________________________________________________________ Fungal infections are severely underestimated as a cause of mortality, and alternative drugs are urgently needed. Here, Schaefer et al. show that a synthetic polymer mimicking defensins shows different, but synergistic activity with known antifungals. Introduction Modern medicine often relies on invasive medical interventions or drugs which can compromise the patient’s immune system. An unfortunate consequence of these undeniably successful treatments for life-threatening diseases like cancer is severe infections caused by opportunistic pathogens^[60]1,[61]2. Among these opportunists are fungal pathogens, including Candida, Aspergillus, Cryptococcus, and Pneumocystis species^[62]1–[63]3. More recently, increasing numbers of opportunistic fungal infections caused by Aspergillus, Mucorales, and Candida species have been observed in COVID-19 patients with severe respiratory syndromes in intensive care units^[64]4. These and other factors result in over 2 million invasive fungal infections annually worldwide, with alarmingly high mortality rates and more than 1.5 million deaths^[65]3. Candida spp. is the fourth most common cause of hospital-acquired infections, and mortality rates from systemic Candida infections exceed 40%, even with antifungal intervention^[66]3,[67]5. Among all Candida species, Candida albicans accounts for around 50% of Candida bloodstream infections^[68]3,[69]6. Novel pathogenic species, such as the multi-drug-resistant Candida auris have emerged, potentially through adaptations to higher ambient temperature due to global climate change^[70]7,[71]8. Indeed, C. auris and C. albicans were listed as two of the four critical group pathogens in the World Health Organization’s first-ever fungal priority pathogens list, emphasising the need for new treatment options^[72]9. There are currently only four classes of antifungal drugs approved for the treatment of invasive Candida infections – azoles (e.g., fluconazole), polyenes (e.g., amphotericin B), echinocandins (e.g., caspofungin), and flucytosine^[73]10. Their application is limited by undesired drug-drug interactions (azoles), detrimental off-target side-effects (polyenes), and the increasing occurrence of drug resistance (azoles, flucytosine, and echinocandins)^[74]10,[75]11. Resistance of Candida spp. occurs mainly due to target over-expression, modification of the drug target, or upregulation of drug-efflux pumps^[76]11. The urgency to find new treatment options against Candida spp. was highlighted by the Centers for Disease Control and Prevention’s (CDC) 2019 classification of Candida spp. as a serious threat to human health with equal standing to multi-drug-resistant bacteria such as Pseudomonas aeruginosa^[77]12. However, discovering novel targets for antifungal drugs is complicated by the evolutionary similarity of eukaryotic human and fungal cells, and the antifungal development pipeline is dominated by compounds from established classes, which are likely to result in similar complications^[78]10,[79]13. Exceptions are fosmanogepix and ibrexafungerp which are first-in-class and undergoing clinical trials^[80]14,[81]15, but ultimately the emergence of resistance and clinical success for these new classes remain to be seen^[82]14. Combination therapy can decrease the development of resistance or re-sensitise resistant strains by acting on multiple targets^[83]16–[84]18. It can also reduce toxicity to the host by decreasing required drug concentrations^[85]16. In nature, antifungal peptides (AFPs) prevent and combat fungal infections in all domains of life^[86]19. Most interact with the fungal cell membrane, damaging the cell wall or membrane or causing intracellular stress^[87]19. Employing those potent natural effectors as a drug is hampered by several issues; the membrane-active AFPs are often toxic to the expression host in biotechnological synthesis, chemical synthesis of peptides is expensive and complicated by their sequence specificity, and susceptibility to host proteases generally limits the applicability of AFPs^[88]19. These issues can be circumvented by mimicking cationic amphiphilic properties of AFPs synthetically^[89]20,[90]21. Various polymeric synthetic structures show promising antifungal properties, including β-peptides^[91]22,[92]23, poly(2-oxazoline)s^[93]24,[94]25, polycarbonates^[95]26, polyacrylamides^[96]27,[97]28, and peptidopolysaccharides^[98]29. Owing to advances in polymerisation techniques, particularly reversible-deactivation radical polymerisation (RDRP), synthetic macromolecules can be produced in a facile manner with precise control over molecular weight and composition^[99]30. However, one of the remaining challenges for RDRP is the synthesis of strictly homogeneous sequence-defined molecules, which would resemble AFPs with precisely defined sequences^[100]31. Due to this limitation, synthetic copolymers are usually synthesised with statistically distributed monomers or as a block copolymer, which to a certain degree provide control over sequence and dispersity^[101]31. Our group previously synthesised and screened a library of synthetic polyacrylamides inspired by AFP structures for activity against C. albicans and biocompatibility^[102]27. We identified polymers that outperformed amphotericin B in terms of their therapeutic index against C. albicans in vitro^[103]27. In the current work, we determined the mode of action of our most promising polymer compositions and investigated the in vitro and in vivo therapeutic potential of synergistic combinations of our polymers with existing antifungals with a view to enhancing efficacy, minimising toxicity and preventing the emergence of antifungal drug resistance. Results and discussion Synthesis and characterisation of amphiphilic polyacrylamides that mimic AFPs Inspired by the physicochemical properties of antimicrobial peptides, we previously synthesised random acrylamide copolymers using photo-induced reversible-deactivation radical polymerisation^[104]27,[105]30. The polymer characteristics that conferred the highest activity against C. albicans and best biocompatibility with mammalian host cells (measured by in vitro therapeutic indexes) were short polymers with a degree of polymerisation (X[n]) of 20 and an optimal balance of hydrophilic to hydrophobic groups^[106]27. Here, we synthesised four ternary polyacrylamides with these characteristics (Fig. [107]1 and Table [108]1), which previously demonstrated the most promising properties: LP (linear, pentyl), LH (linear, heptyl), CB (cyclic, benzyl), and CX (cyclic, hexyl), named by their distinct hydrophobic features. The successful synthesis and purification were confirmed by ^1H nuclear magnetic resonance (NMR) spectroscopy and refractive index-based size-exclusion chromatography (SEC). We achieved nearly complete monomer conversion of >98% and observed narrow, unimodal molecular weight distributions with a dispersity (Ð) between 1.09 and 1.12 (Supplementary Figs. [109]S1-[110]S15). Fig. 1. Chemical structures of polyacrylamides synthesised for this study. Fig. 1 [111]Open in a new tab R represents the different side chains and x indicates the targeted number of hydrophobic residues within the molecule. Table 1. Composition, targeted degree of polymerisation (X[n]), calculated molecular weight (M), experimental number-average molecular weight (M[n]) and dispersity (Ð) of polymers employed in this study Polymer Targeted polymer composition (% positively charged/hydrophilic/hydrophobic functionality) Experimental polymer composition (% positively charged/hydrophilic/hydrophobic functionality)^a Targeted X[n] Calculated M (g/mol) ^b M[n] (g/mol) ^c Ð ^c LP 50/20/30 50/21/29 20 3700 6300 1.12 LH 50/25/25 50/25/25 20 3800 6400 1.09 CB 50/15/35 50/15/35 20 3900 6600 1.10 CX 50/25/25 49/26/25 20 3800 6100 1.09 [112]Open in a new tab ^adetermined by ^1H NMR spectroscopy before polymerisation. ^bcalculated by ChemDraw (version 19.0) for Boc-protected polymers based on targeted composition and X[n], rounded to the nearest 100. ^cdetermined by SEC using poly(methyl methacrylate) standards. Polyacrylamides are active against drug-resistant, clinical C. albicans isolates Our previous work showed that our polymers were active against C. albicans and other ascomycetes including Candida glabrata (Nakaseomyces glabratus), Candida krusei (Pichia kudriavzevii), and Saccharomyces cerevisiae, as well as the basidiomycete Cryptococcus neoformans^[113]27. Despite a high tolerance of C. neoformans towards AmpB and fluconazole, and intrinsic resistance of S. cerevisiae and C. glabrata towards fluconazole, all were susceptible to the four candidate polymers^[114]27. This suggested a mode of action that is different to AmpB and fluconazole. To explore this further, the minimum inhibitory concentration (MIC, growth inhibition of >90% at 24 h) of the polymers against antifungal drug-resistant strains of C. albicans (Table [115]2) was assessed using slightly modified Clinical and Laboratory Standards Institute (CLSI) guidelines^[116]32,[117]33. Table 2. Antifungal activity of polymers and selected antifungal drugs against C. albicans clinical isolates C. albicans strain Phenotype Minimum inhibitory concentration[24h, 90%] in µg/mL (µM) Polymers Antifungal drugs LP LH CB CX AmpB Flu Cas SC5314 Wild type 64–128 (24–47) 16–32 (6–11) 64 (22) 64 (23) 0.5–1 (0.5–1.1) 0.25–0.5 (0.8–1.6) 0.25–0.5 (0.2–0.5) 110.12 Gsc1-mutation ↑ 256 (95) 16 (6) ↑ 128 (44) ↑ 128 (46) 1–2 (1.1–2.2) ↑↑ >8 (>26) ↑↑ 4–8 (3.6–7.3) EU0108 Erg11-/Erg5-mutation, enhanced Cdr-efflux 64 (24) 8–16 (3–6) ↓ 16–32 (6–11) 32–64 (11–23) ↑ 2 (2.2) ↑↑ >8 (>26) 0.5–1 (0.5–0.9) EU0012 Erg3-mutation 128–256 (47–95) 16–32 (6–11) 64–128 (22–44) 64–128 (23–46) ↑↑ 2–4 (2.2–4.3) ↑↑ >8 (>26) 0.5–1 (0.5–0.9) EU1008 Erg3-/Erg11-mutation 32–64 (12–24) 8–16 (3v6) ↓ 32 (11) ↓ 16–32 (6–11) ↑ 2 (2.2) ↑↑ >8 (>26) 0.5–1 (0.5–0.9) EU0136 Erg6-deficient ↑ 256 (95) 16–32 (6–11) ↑ 128 (44) 64–128 (23–46) ↑↑ 4 (4.3) ↑↑ >8 (>26) 0.5 (0.5) EU0992 Enhanced Cdr-efflux 64–128 (24–47) 16 (6) 64–128 (22–44) 64–128 (23–46) 1–2 (1.1–2.2) ↑↑ >8 (>26) 0.5–1 (0.5–0.9) EU0989 Enhanced Cdr-efflux 128 (47) 8–16 (3–6) 64 (22) 64–128 (23–46) 1–2 (1.1–2.2) ↑↑ >8 (>26) 0.5–1 (0.5–0.9) EU0981 Enhanced Mdr-efflux 64 (24) 8–16 (3–6) 64 (22) 32–64 (11–23) 1–2 (1.1–2.2) ↑↑ >8 (>26) 0.25–0.5 (0.2–0.5) EU0999 Enhanced Mdr-efflux 64–128 (24–47) 8–16 (3–6) 64–128 (22–44) 32–64 (11–23) 1–2 (1.1–2.2) ↑↑ >8 (>26) 0.25–0.5 (0.2–0.5) [118]Open in a new tab Minimum inhibitory concentrations were determined by the concentration at which the respective compound inhibited >90% of fungal growth compared to an untreated control after 24 h at 35 °C. AmpB Amphotericin B, Flu Fluconazole, Cas Caspofungin. ↑↑ indicates resistance, ↑ indicates increased tolerance (up to two-fold increase in MIC), and ↓ indicates a decreased MIC compared to the wild type. C. albicans strain 110.12 is resistant to azoles and caspofungin, the latter due to a mutation in the echinocandin target Gsc1, an essential β-1,3-glucan synthase subunit^[119]34. The MICs for the polymers LP, CB and CX were only slightly increased compared to wild type, and the clinical isolate was at least as susceptible as the wild type to polymer LH. AmpB-tolerant (EU0108, EU1008) or -resistant (EU0012, EU0136) strains have various mutations in enzymes involved in the biosynthesis of ergosterol (Erg3, Erg5, Erg6, Erg11), the target of AmpB^[120]35,[121]36. The strains are also azole-resistant, which has been attributed to increased drug efflux at least in strain EU0108^[122]35,[123]36. However, all polymers were active against those clinical isolates with even decreased MICs against the AmpB-tolerant strains. LH was fully active against the AmpB-resistant strains. A slight increase in MIC was observed for LP, CB, and CX. The fluconazole-resistant C. albicans strains EU0992 and EU0989 have an increased Cdr-mediated drug efflux, and EU0981 and EU0999 show more Mdr-dependent export activity, while all these strains are normal in sterol biosynthesis (O. Bader, personal communications)^[124]37. The antifungal activity of the polymers was not affected by those mutations, suggesting that they are not transported out by Cdr- or Mdr-related efflux pumps. Comparing the MICs of the most active antifungal polymer LH against C. albicans (8–32 µg/mL, 3–11 µM) to reported MICs of antimicrobial peptides reveals them to be active at similar concentrations. For example, the membrane-lytic peptides LL-37 (human) and melittin (bee venom) inhibit C. albicans growth at concentrations of 64 µg/mL (14 µM) and 11–22 µg/mL (4–8 µM), respectively^[125]38,[126]39, while the intracellularly acting histatin 5 (human) exhibited slightly lower MICs against C. albicans (4–8 µg/mL, 3–5 µM)^[127]40,[128]41. Some synthetic cationic peptides composed of 9–11 amino acids have shown MICs against C. albicans in an equivalent range of 8–32 µg/mL (6–23 µM) and also interfere with fungal membranes^[129]42,[130]43. Overall, each antifungal drug-resistant C. albicans strain tested was as susceptible to polymer LH, if not more, as the wild type. This indicates a distinct mode of action of LH. The different activity pattern of the other polymers against drug-resistant strains may indicate slight differences in modes of action between the polymers. We therefore investigated the potentially novel modes of action of our polymers. Transcript profiling to investigate the mode of action of the antifungal polymers We compared the transcriptome of C. albicans cells grown for 1 h in the presence of sub-inhibitory concentrations of the antifungal polymers to untreated control. A non-active polymer, poly(hydroxyethyl acrylamide) (poly-HEA), was also included for comparison (chemical characterisation for poly-HEA in Supplementary Figs. [131]S14 and [132]15 and Table [133]S1) as well as the membrane-active antimicrobial peptide LL-37^[134]38,[135]44. An overview of the global transcriptomic differences of C. albicans in response to the polymers and LL-37 was gained through hierarchical clustering and principal component analyses of normalised gene expression data. These analyses showed that the transcriptome of cells exposed to the antifungal polymers LP, CB, and CX clustered together (Supplementary Fig. [136]S16). Surprisingly, the hierarchical clustering revealed that the transcriptomic patterns for these three polymers were more similar to the non-toxic poly-HEA (Supplementary Fig. [137]S16A), while our most active candidate LH clustered separately after applying either of the two statistical methods (Supplementary Fig. [138]S16A, B). The antimicrobial peptide LL-37 clustered separately from all polymers. Next, we looked for biological functions and pathways where there was an overrepresentation of up- or downregulated genes associated with the function or pathway in the datasets by performing Gene Ontology (GO) term^[139]45 and Kyoto Encyclopedia of Genes and Genomes (KEGG) pathway enrichment^[140]46 analyses (Fig. [141]2). Similar to our clustering analyses, we noted that the non-toxic poly-HEA differed from the four antifungal polymers and that among the polymers, LH showed the most distinct pattern with the highest number of differentially expressed genes. Therefore, we focussed our studies on the effect of polymer LH on C. albicans since it demonstrated the best activity against clinical C. albicans isolates in comparison to the other three polymers (Table [142]2) and triggered a distinct transcriptomic response in C. albicans (Fig. [143]2 and Supplementary Fig. [144]S16) in the present study. Additionally, a previous study highlighted the potential of polymer LH due to its superior antifungal activity while showing comparatively low damage to red blood cells and murine fibroblasts^[145]27. Fig. 2. Antifungal polymers cause transcriptomic responses associated with impaired protein glycosylation, membrane stress, and cell wall damage in C. albicans. [146]Fig. 2 [147]Open in a new tab A Gene Ontology (GO) term enrichment analysis, based on molecular function^[148]45, and B KEGG (Kyoto Encyclopedia of Genes and Genomes) pathway enrichment analysis^[149]46 of RNA microarray data after treating C. albicans SC5314 for 1 h at 30 °C with sub-inhibitory concentrations of polymers and LL-37. Statistically significantly up- (red) or downregulated (green) gene groups associated with GO terms and KEGG pathways compared to the untreated control are shown. Diameter of circles reflects the percentage of genes differentially regulated in the associated pathway or term and the shading represents the adjusted p-value (calculated by hypergeometric distribution and adjusted by Benjamini–Hochberg correction for multiple testing). GO terms are additionally ordered by their assigned parental processes. Functional GO term enrichment with the GO term finder tool at Candida Genome Database^[150]45 (Fig. [151]2A) and KEGG pathway enrichment analyses^[152]46,[153]47 (Fig. [154]2B) were performed on the sets of differentially expressed genes. Both analyses indicated that the amphiphilic antifungal polymers caused damage to the cell membrane and cell wall, leading to the metabolic arrest of C. albicans. GO terms associated with transporter activity were enriched in the downregulated gene set, indicating general stress and metabolic arrest of the cells (Fig. [155]2A). More specifically, oligopeptide transmembrane transporter activity was enriched in the set of genes downregulated following treatment with LH. Together with the overrepresentation of genes involved in lipid and protein binding, this supports the hypothesis of C. albicans sensing a toxic, peptide-like structure with amphiphilic properties. The KEGG pathway mitogen-activated protein kinase (MAPK) stress response was overrepresented in upregulated genes upon treatment with the antifungal polymers (Fig. [156]2B and Supplementary Fig. [157]S17 after treatment with polymer LH). An upregulation of genes in the MAPK signalling pathway is typical for cell wall and osmotic stress or starvation and leads to cell cycle arrest and cell wall remodelling^[158]48,[159]49. The metabolic arrest of the fungal cells was further indicated by the enrichment of the KEGG pathways ribosome, biosynthesis of amino acids, and oxidative phosphorylation in the downregulated gene set upon treatment with the antifungal polymers. Additionally, GO terms associated with glycosylation processes were enriched in the upregulated gene set, specifically hydrolase activity against O-glycosyl compounds and hexosyltransferase activity – a parental gene set of mannosyltransferase activity (Fig. [160]2A). Similarly, one significantly overrepresented KEGG pathway enriched in upregulated genes was protein processing in the endoplasmic reticulum (ER) (Fig. [161]2B). A more detailed look (Supplementary Fig. [162]S18) revealed that C. albicans cells treated with LH strongly upregulated genes associated with glycosylation and ER-associated degradation of misfolded protein (ERAD), together suggesting a disruption in the correct glycosylation of proteins. In support of this hypothesis, we also observed upregulated genes in the N-glycan biosynthesis pathway (Supplementary Fig. [163]S19). GO terms relating to membrane stress were enriched in the upregulated gene set (Fig. [164]2A). This included calcium ion transmembrane transport, suggesting increased Ca^2+ influx and membrane damage. KEGG pathway enrichment analysis also revealed a transcriptional signature expected from damage to the cell membrane (Fig. [165]2B), particularly with polymer LH. This includes an overrepresentation of glycosylphosphatidylinositol (GPI)-anchor biosynthesis and glycerolipid biosynthesis in the upregulated gene set, and steroid biosynthesis in the downregulated gene set. In contrast, treatment with the non-toxic poly-HEA or the antimicrobial peptide LL-37 did not elicit a significant enrichment of these glycosylation-related GO terms and KEGG pathways. This shows that it is not a general response to the presence of polymers or to bioactive peptide(-like) substances. Instead, we found an enrichment of upregulated genes with GO terms associated with DNA, protein, and small molecule binding. This may indicate that C. albicans reacts to small, peptide(-like) molecules, however, they do not seem to influence glycosylated proteins. We found nearly no KEGG pathways to be enriched for differentially regulated genes in the presence of poly-HEA. In the presence of LL-37, genes involved in MAPK signalling were significantly upregulated, indicating a fungal stress response to a toxic compound. Additionally, the KEGG pathways ribosome, biosynthesis of amino acids, and oxidative phosphorylation were significantly enriched in the downregulated gene set upon treatment with LL-37, indicating an expected metabolic arrest of the fungal cells. Those KEGG pathways were also overrepresented in the presence of the antifungal polymers, but not the non-toxic poly-HEA. Notably, genes in the KEGG pathway “protein processing in the ER” were specifically upregulated upon treatment with the antifungal polymers, but not poly-HEA or LL-37 (Fig. [166]2B and Supplementary Fig. [167]S18, showing polymer LH vs. LL-37), again highlighting that ER protein processing and MAPK signalling responses are specific for polymers with antifungal activity. In addition, we found that the gene expression profiles of C. albicans exposed to our polymers are very distinct from published profiles under exposure to polyenes, azoles or echinocandins^[168]50. This again suggests a unique mode of action of our synthetic polymers. Currently approved antifungals have specific cellular targets, and mutations in the affected enzymes are one of the main reasons for the development of resistance^[169]10. In contrast, antimicrobial peptides have multiple modes of action, decreasing the likelihood of the development of resistance^[170]51. For example, a Musca domestica (housefly) AFP triggers responses in C. albicans that include reaction to oxidative stress, cell wall and membrane maintenance, protein synthesis, and energy metabolism^[171]52. A bacterium-derived, membranolytic antifungal lipopeptide (jagaricin)^[172]53 similarly induces a broad transcriptional response, comprising the upregulation of cell wall organisation/biogenesis and calcium ion transmembrane transport genes, and downregulation of transmembrane transport for substances such as oligopeptides^[173]37. Our antifungal polymers induced a similar transcriptional response in C. albicans. Altogether, these data led us to hypothesise that the antifungal polymers likely target multiple processes in C. albicans – they damage the cell wall and permeabilise the cell membrane, and they target protein glycosylation and thereby induce ER stress. O-mannosylation, calcineurin, MAPK signalling, and a phosphoinositide regulator are required for C. albicans to survive polymer exposure To gain further insights into the target of the polymers, we screened selected deletion mutants of C. albicans for their growth at sub-inhibitory polymer concentrations (LP, LH, CB, CX). Mutants were chosen to represent suspected target and resistance pathways, including cell wall organisation and stress response, membrane composition and inositol signalling, protein glycosylation (O- and N-linked mannosylation), calcineurin pathway, osmotic and oxidative stress response (MAPK signalling), drug efflux, and polyamine uptake (Supplementary Data [174]S1). Growth curves were compared based on the time to reach half-maximal absorption (Supplementary Data [175]S1). This growth speed index is positive for beneficial mutations (green shading in Supplementary Data [176]S1) and negative for detrimental ones (red shading in Supplementary Data [177]S1). The deletion of genes important for cell wall organisation and stress response had little effect on growth in the presence of polymers (Supplementary Data [178]S1), with the exception of ire1, which showed no growth in the presence of LH. IRE1 encodes a protein kinase of the unfolded protein response and cell wall organisation^[179]54. Interestingly, IRE1 was also upregulated in the presence of LH (Supplementary Fig. [180]S18). Among the mutants for genes relevant to membrane composition and inositol signalling (Supplementary Data [181]S1), inp51 – lacking a phosphatase involved in the maintenance of phosphoinositide levels and thus cell wall and membrane integrity^55 – exhibited no growth in the presence of all polymers. INP51 deletion has been shown to increase susceptibility to cell wall-active compounds^[182]55. The deletion of ERG5, involved in ergosterol biosynthesis, did not change the susceptibility to the polymers. This agrees well with the susceptibility of clinical AmpB-tolerant isolates towards the polymers (Table [183]2) and indicates that ergosterol biosynthesis likely does not affect the polymers’ mode of action. Deletion of genes contributing to O-linked protein mannosylation (PMT1, PMT3, PMT4, PMT5)^[184]56 consistently increased susceptibility of C. albicans (Supplementary Data [185]S1), suggesting that these activities are involved in the response to all antifungal polymers. In contrast, gene deletions affecting N-linked mannosylation (MNN13, MNN14, MNN15, MNN22, MNN4, MNN9, MNS1, MNT4, OCH1)^[186]56 resulted in no change in susceptibilities. Calcineurin and MAPK signalling are crucial in fungal development and response to environmental stress^[187]57. Two calcineurin pathway deletion mutants (crz1, mid1) showed no growth with normally sub-inhibitory polymer concentrations (Supplementary Data [188]S1), and their genes’ expression was also upregulated in C. albicans exposed to the antifungal polymers, but not poly-HEA. Two MAPK signalling deletion mutants (hog1, pbs2) also showed no growth in the presence of polymers. HOG1 similarly has shown upregulation after exposure to the antifungal polymers, most prominently for LH and CX (Supplementary Fig. [189]S17 for LH-treatment). Hence, the antifungal polymers seem to cause stress in C. albicans, and both calcineurin and MAPK pathways appear to be essential for fungal survival. In agreement with the clinical drug-efflux mutants (Table [190]2), neither gain-of-function (in MRR1 and TAC1) nor deletion (of MRR1, SNQ2, TAC1) of genes involved in drug efflux had a consistent impact on susceptibility. The AFP histatin 5 is actively transported across the fungal membrane by polyamine transporters like Dur31 in C. albicans to exert its intracellular antifungal effect^[191]58. Our synthetic polymers share characteristics like cationic charge with both histatin 5 and polyamines, and their presence led to the upregulation of DUR31. However, deletion of DUR31 or the related DUR35^[192]59 resulted in a slightly increased (dur31) or unchanged susceptibility (dur35) (Supplementary Data [193]S1), suggesting that the polymers are not primarily taken up by those transporters. We also investigated selected C. glabrata (Nakaseomyces glabratus) mutants (marked blue in Supplementary Data [194]S1). While none of the tested C. albicans mutations were beneficial, the deletion of C. glabrata ERG5 and INO2 increased tolerance to all antifungal polymers. Both genes are associated with membrane composition and inositol signalling. C. glabrata cdr1, lacking a drug-efflux pump-encoding gene, also showed increased growth with polymers, except LH. In contrast, deletion of MNN2, coding for an N-linked mannosyltransferase, had the highest benefit for LH-treated C. glabrata cells. Despite these slight differences, our data still suggests a very similar mode of action against C. glabrata. Like in C. albicans, deletion of genes encoding proteins involved in membrane composition and inositol signalling (INP51, INP53, ISC1), O-linked protein mannosylation (PMT1, PMT2) and MAPK signalling (PBS2) were more susceptible to the polymers, while the deletion of genes coding for proteins in the calcineurin pathway (CNA1, CRZ1, MID1) resulted in no growth in the presence of antifungal polymers. In sum, the mutant screening agreed with our analyses of the transcriptome and suggested a mode of action connected to protein glycosylation and general stress of the polymers against C. albicans. Some similarities (involvement of MAPK and calcineurin signalling) were detected in a previous C. albicans mutant screen with the antifungal lipopeptide jagaricin, for which disruption of membrane integrity was suggested as the primary mode of action^[195]37,[196]53. This suggests a similar effect of the synthetic polymers, especially as our gene expression analyses also showed possible interference with the plasma membrane (Fig. [197]2). Polymer LH lyses C. albicans cell membranes The polymers are inspired by antimicrobial peptides, which lyse bacterial and fungal membranes^[198]60. Membrane damage by the synthetic polymers was also suggested by gene expression analyses (Fig. [199]2) and mutant screening (Supplementary Data [200]S1). We therefore investigated the membranolytic potential of polymer LH with a C. albicans strain constitutively expressing GFP in the cytosol (Fig. [201]3A–G). Two antifungal compounds were included – AmpB, which has membranolytic activity^[202]61,[203]62, and tunicamycin, which inhibits N-glycosylation of proteins, leading to cell death without membrane lysis^[204]63. Additionally, tethered membranes isolated from C. albicans and erythrocytes were incubated with polymer LH and non-toxic poly-HEA to investigate their potential to lyse synthetic membranes (Fig. [205]3I, J). Fig. 3. Polymer LH causes membrane lysis in C. albicans. [206]Fig. 3 [207]Open in a new tab Detection of fluorescence of C. albicans expressing cytoplasmic GFP (ADH1-GFP) after 6 h at MIC assay conditions for (A) untreated C. albicans cells, B cells treated with 1× MIC Amphotericin B (AmpB), C 1× MIC tunicamycin, D 0.25× MIC polymer LH, E 0.5× MIC polymer LH, and F 1× MIC polymer LH to investigate lytic activity of the compounds. Scale bars in (A–F) represent 10 µm. G The GFP signal was quantified from at least 50 C. albicans-GFP cells per biological replicate (n = 3), and then averaged and normalised to the respective untreated control. H Colony forming units (CFU) were determined by backplating and normalised to the inoculum (n = 3 biological replicates). Statistical significance in (G) and (H) was determined by Dunnett’s ordinary one-way ANOVA multiple comparisons analysis (compared to untreated (100%) in (G) or inoculum (100%) in (H), *p < 0.05 [G: p = 0.0148, H: p = 0.0296], **p <  0.01 [G: p = 0.0051], ***p < 0.0005 [H: p = 0.0002], ****p < 0.0001) with ns indicating non-significant [H: p = 0.4987]. Average conductance (G[m]) of tethered membranes isolated from C. albicans yeast (black) or hyphae (grey), or from erythrocytes (red) after the addition of increasing concentrations (c) of (I) antifungal polymer LH and (J) non-toxic poly-HEA in RPMI medium at 37 °C (n = 3 biological replicates). Error bars in (G–J) represent the standard deviation (SD) around the mean. Source data are provided as a Source Data file. Untreated C. albicans-GFP cells showed a prominent intracellular GFP signal (Fig. [208]3A), which was absent in AmpB-treated cells (Fig.[209]3B, G) where only 0.3% were viable as determined via backplating (Fig. [210]3H). The loss of GFP signal was due to membrane lysis, and not cell death per se, as tunicamycin-treated cells showed a less prominent loss of intracellular GFP (Fig. [211]3C), even though only 3% of cells were viable. The polymer LH (Fig. [212]3D–F) led to membranolytic activity, as seen by GFP signal loss, in a concentration-dependent manner with a proportional decrease in cell viability (36% at 0.5× MIC, 3% at 1× MIC at the assay conditions). Interestingly, at concentrations below MIC, we observed cell aggregates, which suggest a change in the surface properties of C. albicans cells. To confirm the permeabilisation of membranes by polymer LH as part of its mode of action against C. albicans, tethered membranes composed of lipid mixtures isolated from C. albicans yeast or hyphae, or from erythrocytes, were incubated with polymer LH (Fig. [213]3I) or non-toxic poly-HEA (Fig. [214]3J). Polymer LH led to a concentration-dependent increase in membrane conductance, indicating membrane permeabilisation by pore formation (Fig. [215]3I and Supplementary Fig. [216]S20A). The increase in membrane conductance was highest in the case of lipids from C. albicans yeast (Fig. [217]3I, black) and weaker for those from C. albicans hyphae (grey). Membranes composed of erythrocyte lipids were more stable and showed lower conductance before the addition of polymer LH and also at low concentrations of polymer LH, compared to membranes composed of C. albicans membranes. In contrast to polymer LH, poly-HEA did not lead to an order of magnitude differences in membrane conductance when increasing its concentration in any of the three lipid mixtures (Fig. [218]3J), indicating no lytic effect. As a control, the same experiment was performed with antimicrobial peptide LL-37 (Supplementary Fig. [219]S20B), for which membrane permeabilisation has been reported as the primary mode of action against C. albicans^[220]38,[221]44. Like polymer LH, LL-37 permeabilised C. albicans membranes at a concentration of 8 µg/mL. However, it also strongly affected tethered membranes isolated from erythrocytes, indicating a lower selectivity of LL-37 compared to polymer LH. LH damages mannans attached to cell wall proteins, enhances phagocytosis, and affects yeast-to-hypha transition Mutant screening and gene expression analyses suggested that glycosylated proteins might be primarily affected by the polymers. A major group of glycosylated proteins in C. albicans are the cell wall proteins. These are both O- and N-mannosylated as they pass through the ER and Golgi on their way to the wall. Once attached to the wall through GPI anchors or Pir linkages, the short O-mannan chains remain buried in the inner cell wall layer, whereas the long N-mannan chains protrude out from the cell surface forming an outer fibrillar layer of the cell wall (Fig. [222]4A)^[223]64. To investigate the effects of LH on the cell wall structure of C. albicans, fungal cells were incubated at sub-inhibitory concentrations for 6 h and analysed by transmission electron microscopy (TEM, Fig. [224]4A–D). Compared to the no-treatment control (Fig. [225]4A), treatment with the non-toxic poly-HEA resulted in no major ultrastructural changes to the cell wall (Fig. [226]4B). A reduction of the outer cell wall N-mannan layer was observed after treatment with tunicamycin, an inhibitor of N-glycosylation (Fig. [227]4C)^[228]63. Treatment with a sub-inhibitory concentration of LH (Fig. [229]4D) disrupted the arrangement of the N-mannan fibrils, supporting our transcriptome and mutant screen data. At these sub-inhibitory concentrations, LH did not cause any obvious disruption of the membrane. Fig. 4. Cell wall changes in C. albicans and immune cell response to LH-treated C. albicans. [230]Fig. 4 [231]Open in a new tab Transmission electron microscopy (TEM) micrographs of the C. albicans cell wall after 6 h incubation in SD medium at 30 °C with (A) no additives, B with poly-HEA, C with tunicamycin, or D antifungal polymer LH, at sub-inhibitory concentrations of the antifungal compounds. The scale bars in (A–D) represent 100 nm. E The amount of glucan and mannan in cell walls isolated from C. albicans wildtype (SC5314) and och1 mutant cells after 6 h of incubation in SD medium (untreated) and with sub-inhibitory concentrations of polymer LH, poly-HEA, tunicamycin, and caspofungin was determined by HPLC. Glucose (from glucan) and mannose (from mannan) represented 55% and 45% of the dry weight of the cell wall in wild-type untreated cells. The graph shows the proportions of glucan (grey bars) and mannan (striped bars) relative to those observed in untreated C. albicans wild-type cells. F Proportion of C. albicans cells not taken up over 15–30 min by human monocyte-derived macrophages. C. albicans cells were pre-treated with LH for one hour before putting them into contact with the macrophages (n = 6 biological replicates). Statistical significance in (E) was determined by Dunnett’s repeated measures ANOVA multiple comparisons analysis (*p <  0.05 [0 vs 8 µg/ml: p = 0.0173; 0 vs 16 µg/ml; p = 0.0192]). Error bars in (F) represent the standard deviation (SD) around the mean. Source data are provided as a Source Data file. To confirm the transcriptome and TEM observations quantitatively, cell walls from C. albicans were isolated after 6 h incubation, and after acid hydrolysis, mannose (from mannans) and glucose (from glucans) were separated and detected by high-performance liquid chromatography (HPLC) (Fig. [232]4E). Exemplary HPLC spectra are shown in Supplementary Fig. [233]S21. In accordance with the TEM observations (Fig. [234]4D), treatment with sub-inhibitory concentrations of polymer LH decreased the proportion of mannan relative to untreated cells. In contrast, poly-HEA treatment had no effect on the relative proportion of glucan and mannan. Consistent with our observations in the TEM images (Fig. [235]4C), treatment with the N-glycosylation inhibitor tunicamycin reduced the mannan content relative to untreated cells. The proportion of mannan in C. albicans och1 mutant cells was reduced relative to wild-type cells, as expected for an N-mannosylation mutant and consistent with previous observations^[236]62. Finally, the β-glucan synthase-inhibiting antifungal drug caspofungin had the expected effect of decreasing the glucan and increasing mannan, relative to the untreated control^[237]63. Overall, our measurements were therefore in good agreement with the expected outcome four our controls and supported our notion of a defective protein glycosylation in C. albicans after treatment with polymer LH. Components of the C. albicans cell wall are also recognised by innate immune cells^[238]65. We hypothesised that the LH-induced changes to the cell wall structure (Fig. [239]4D) could also impact immune recognition and clearance by human macrophages. To test this, we challenged C. albicans cells that were preincubated with or without polymer LH at sub-inhibitory concentrations with primary human monocyte-derived macrophages (hMDMs). Primary immune cells such as hMDMs show donor-dependent differences in their uptake efficiency. We therefore chose the time point (15 or 30 min) for each donor when 30–50% of untreated C. albicans cells were not yet phagocytosed by the specific macrophages. We found that LH pre-treatment significantly increased clearance of C. albicans by primary hMDMs, even at sub-inhibitory concentrations between 4–16 µg/mL (Fig. [240]4F). Concentrations above 16 µg/mL of polymer LH, however, resulted in decreased clearance (Supplementary Fig. [241]S22A). These are most likely due to toxic effects on the hMDMs, which became apparent at 128 µg/mL after 30 min (Supplementary Fig. [242]S22B, measured by LDH release). It has been previously reported that C. albicans mutants with N- or O-linked glycosylation defects are more efficiently phagocytosed than the wild type^[243]66,[244]67. With our TEM, HPLC, and transcriptome data, this suggests that LH disrupts the mannan layer and induces cell wall remodelling which increases phagocytosis by macrophages. Thus, in vivo, the application of polymer LH could potentiate fungal clearance by innate immune cells. The cytokine release by primary human peripheral blood mononuclear cells (PBMCs) to pre-treated C. albicans was characterised by the pro-inflammatory cytokines IL-1β, IL-6, and TNF (Supplementary Fig. [245]S23). The increased clearance by hMDMs (Fig. [246]4F), was reflected by significantly decreased IL-1β, IL-6, and TNF responses of PBMCs at an LH concentration of 4 µg/mL (Supplementary Figs. [247]S23). Higher LH concentrations resulted in no significant changes in the release of IL-1β and TNF and a significant increase of IL-6. The decrease in TNF agrees with previous observations on C. albicans N-mannosylation defective mutants with impaired recognition by immune cells^[248]68,[249]69. As mannoproteins in C. albicans cause a pro-inflammatory immune response^[250]56,[251]65, these data support our notion that polymer LH can act on protein glycosylation. The cell wall of C. albicans is adaptive and constantly remodels, including when C. albicans’ transitions from yeast to hyphae, a process that is commonly associated with virulence^[252]65. Hence, we tested C. albicans under hypha-inducing conditions (37 °C and 5% CO[2]) in the presence of antifungal polymers at sub-inhibitory concentrations over 4, 6 (to measure hypha length), and 24 h (to measure microcolony diameter) (Supplementary Fig. [253]S24). Treatment with each polymer, especially LH, reduced the hyphal length and the diameter of microcolonies. Besides lytic activity against synthetic membranes isolated from C. albicans hyphae (see Fig. [254]3I, grey line), a reduced speed of hyphae formation could be beneficial for clearance and protection of epithelial cells, as the formation of hyphae drives cell invasion and infection of human epithelial cells by C. albicans^[255]70, however, further studies are necessary to confirm this correlation for polymer LH. Polymer LH prevents in vitro infection of human epithelial cells by C. albicans synergistically with caspofungin or fluconazole During systemic infection, C. albicans hyphae invade the human epithelial barrier and allow it to spread via the bloodstream to distal organs^[256]71. The yeast-to-hypha transition is also important for common superficial infections, such as those of the vaginal mucosa, which affect approximately 75% of women worldwide at least once in their lifetime^[257]72,[258]73. To investigate the therapeutic potential of polymer LH, we used an in vitro human epithelial cell model (HECM). Monolayers of vaginal epithelial cells (A-431) were infected with C. albicans, after the addition of different drug dilutions with minimum preincubation time. After 24 h, epithelial damage to A-431 cells was assayed by lactate dehydrogenase (LDH) release. Despite being a rather simple model, it has been routinely used to simulate vaginal candidiasis in vitro^[259]74,[260]75. First, we examined the biocompatibility of polymer LH in the HECM in the absence of C. albicans (Supplementary Fig. [261]S25, light-grey bars). Only at concentrations above 128 µg/mL (4–8× MIC against C. albicans) we observed more than 50% damage. Previously, polymer LH has similarly been reported to cause more than 50% damage against murine fibroblasts at 128 µg/mL or higher^[262]27. Based on the therapeutic index – the ratio of cytotoxic concentration to MIC – polymer LH, with its therapeutic index of 4–8, outperformed AmpB (therapeutic index 2–4)^[263]27. For the HECM in the present study, this would theoretically present a therapeutic window for polymer LH of up to 128 µg/mL. However, in the HECM at concentrations between 16 and 128 µg/mL, polymer LH did not prevent damage by C. albicans to the vaginal epithelial cells (Supplementary Fig. [264]S25, dark-grey bars). One possibility for the unexpected failure of LH to prevent damage in the HECM could be the bioavailability. To test this hypothesis, we pre-treated C. albicans cells with the polymer for 1 h at concentrations between 16 and 512 µg/mL before infection. Damage by C. albicans was strongly reduced with this altered protocol in a concentration-dependent manner up to 256 µg/mL (34% of untreated infection control at 256 µg/mL, Supplementary Fig. [265]S26A). This supports the hypothesis that poor bioavailability in the HECM reduces the antifungal properties of the polymer. We next took supernatants from uninfected vaginal epithelial cells, treated with LH, and added them to C. albicans in a conventional MIC assay. These were unable to inhibit C. albicans growth in vitro, even when the initial concentration of polymer LH exceeded the MIC (Supplementary Fig. [266]S26B). This suggests that the presence of human cells reduces the concentration of polymer in the supernatant. In contrast, the common antifungal drugs AmpB, caspofungin, and fluconazole (among others) successfully reduced damage by C. albicans to the epithelial cells in the HECM (Supplementary Table [267]S2). Since the polymer LH alone unexpectedly did not inhibit damage by C. albicans in the HECM, we studied combinations of LH with established antifungal compounds, first at MIC assay conditions and without human cells (Supplementary Figs. [268]S27 and [269]28, and Table [270]S3). Synergy was defined as a minimum two-fold decrease in MIC in the presence of the other drug (fractional inhibitory concentration (FIC) index ≤0.5). Antagonistic drug combinations result in an FIC index of at least 4, i.e., a MIC increase of at least two-fold. Indifferent (FIC index = 1), and additive (FIC index between 0.5 and 1) effects were also considered. The compounds selected for our synergy studies differ in their targets: cell wall (Calcofluor White, Congo Red, caspofungin, nikkomycin Z); cell membrane (AmpB, cetyltrimethylammonium bromide (CTAB), dodecyltrimethylammonium bromide (DTAB), sodium dodecyl sulfate (SDS)); or intracellular processes (fluconazole, cycloheximide, tunicamycin, FK506, geldanamycin), which in turn could again affect cell membrane or wall composition. Seven out of these thirteen antifungal compounds showed a synergistic or strong additive effect (FIC index below 0.6) with LH in vitro and were therefore tested in the HECM. In addition to the LDH release assay, propidium iodide was used to visualise dead epithelial cells after 24 h of infection (Fig. [271]5, Supplementary Fig. [272]S29). Fig. 5. Synergistic effects of LH in combination with selected antifungal drugs against C. albicans SC5314 during vaginal epithelial cell infections. [273]Fig. 5 [274]Open in a new tab Damage to vaginal epithelial (A-431) cells (infected by C. albicans and uninfected) after treatment with polymer LH and A caspofungin (Cas) or B fluconazole (Flu) and their respective combinations was measured by LDH release (n = 4 biological replicates, SD around the mean). Damage to A-431 cells was normalised to untreated infection control (for infected samples) or a Triton-X-treated 100% lysis control (uninfected samples; each indicated by a dotted line). Source data are provided as a Source Data file. (C–J) show fluorescence microscopy images of the scenarios represented in (A) and (B) to visualise morphological changes and the viability of vaginal epithelial cells by staining with 1 µg/mL propidium iodide. Scale bars represent 100 µm. Although LH alone did not prevent damage by C. albicans (Supplementary Fig. [275]S26), it did so in combination with the antifungal drugs caspofungin or fluconazole, even at normally sub-inhibitory concentrations (Fig. [276]5). Strikingly, only 0.03 µg/mL of caspofungin (i.e., 8× less than its MIC of 0.25 µg/mL in the HECM) combined with 4 µg/mL LH was required to reduce host cell damage to 2% with no visible cytotoxicity (Fig. [277]5A). Similarly, a low dose of 0.03 µg/mL fluconazole (again 8× less than its MIC in the HECM) combined with 16 µg/mL LH reduced vaginal epithelial cell damage to 13%, while remaining biocompatible (Fig. [278]5B). Without LH, these low antifungal drug concentrations did not prevent infection with over 50% epithelial cell damage. This finding was supported by fluorescence microscopy (Fig. [279]5C–J), where the same combinations resulted in healthy vaginal epithelial cells and low doses of antifungal drugs did not significantly protect the epithelial cells from damage at up to 0.25 µg/mL. Combination with LH therefore reduced the MIC for established antifungal drugs up to eight-fold, showing its potential as a synergistic agent for antifungal applications. The other antifungal compounds with strong additive or synergistic behaviour with LH in the in vitro pre-screen (nikkomycin Z, cycloheximide, tunicamycin, FK506, geldanamycin), did not reduce damage to epithelial cells to less than 45% of the no-drug control, and in some cases showed no significant synergism (Supplementary Fig. [280]S29). Some of those compounds are cytotoxic at elevated concentrations, and indeed, the combination of LH with cycloheximide or geldanamycin caused damage to uninfected human cells. Combination of polymer LH and caspofungin prolongs survival in an invertebrate model of C. albicans infection Next, we assessed whether the synergism of polymer LH with caspofungin protected against fungal infection in vivo. For this, we used the well-established Galleria mellonella (greater wax moth) model of systemic candidiasis by injecting larvae with an infectious dose of C. albicans followed by treatment with LH and caspofungin^[281]76–[282]79. Even though G. mellonella is an invertebrate organism, the model has several advantages for testing the virulence of Candida spp. and antifungal activity of candidate antifungals, such as ease-of-use, growth at 37 °C, and its many similarities to the mammalian innate immune system^[283]76–[284]79. We first determined the acute toxicity of polymer LH, caspofungin, and AmpB in larvae. Lethal effects of LH on larvae were observed at 500 mg/kg or higher (Supplementary Fig. [285]S30A). No toxicity was observed for caspofungin at doses up to 100 mg/kg (Supplementary Fig. [286]S30B, blue line), AmpB showed toxicity at 100 mg/kg after 4 d (Supplementary Fig. [287]S30B, purple line). Therefore, both LH and caspofungin outperformed AmpB in terms of toxicity, and we determined that doses of LH up to 250 mg/kg and caspofungin up to 100 mg/kg could be used to treat larvae. The synergistic combination of LH with fluconazole was not tested, because it was not active against C. albicans in the presence of 50% (v/v) or more of foetal bovine serum in MIC assays, in contrast to the combination of LH and caspofungin which remained active in the presence of serum (Supplementary Table [288]S4). To simulate systemic candidiasis, we infected each larva with 1 × 10^5 C. albicans cells in one proleg, treated them in another proleg, and monitored survival over 14 days (Fig. [289]6). All uninfected, water-treated G. mellonella larvae survived for 14 d. In contrast, untreated and water-mock treated C. albicans-infected larvae died after 2 d (difference not significant at p = 0.674). Fig. 6. Treatment with polymer LH in combination with caspofungin (Cas) prolongs survival of C. albicans-infected Galleria mellonella larvae. [290]Fig. 6 [291]Open in a new tab G. mellonella larvae were infected with 1 × 10^5 C. albicans cells (except uninfected control, grey) and treated after 2 h with caspofungin (Cas; 5 mg/kg: blue; 100 mg/kg: blue dotted), polymer LH (250 mg/kg, green dashed), and the combination of 5 mg/kg Cas and 250 mg/kg LH (green and blue dashed). Untreated, infection controls are shown in black (C. albicans only) or black dashed (C. albicans, injected with water). Survival of G. mellonella was monitored over 14 d. Nineteen larvae per condition were tested. Statistical significance was determined for infected larvae treated with the combination (5 mg/kg Cas + 250 mg/kg LH) compared to infected larvae treated with the respective dose of a single drug by Log Rank (Mantel–Cox) pairwise comparison (**p < 0.01 [here: p = 0.003], ***p < 0.001). At the concentrations used here, the single compounds exhibited no toxicity against G. mellonella, as shown in Supplementary Fig. [292]S30. Source data are provided as a Source Data file. Treatment with 100 mg/kg caspofungin alone protected 94.7% of the larvae from death, whereas only 10.5% of larvae survived to 14 d at 5 mg/kg (Fig. [293]6). All infected larvae treated with polymer LH alone at 250 mg/kg succumbed by day 3, however, the same dose in combination with low-dose caspofungin (5 mg/kg) significantly increased the survival of C. albicans-infected larvae (5 mg/kg caspofungin vs. combination: p = 0.003; 250 mg/kg LH vs. combination: p < 0.001; water vs. combination: p < 0.001). Compared to low-dose caspofungin treatment, the survival after 14 d increased four-fold to 42.1%. Notably, 100% of infected larvae treated with the synergistic combination survived until day 6 post-infection. This therefore demonstrates in vivo synergy between polymer LH and caspofungin and emphasises the in vivo potential of the synthetic polymer LH in combination therapy. Such combination therapy approaches of compounds which are individually inactive or only marginally active in vivo have received more attention recently. Examples include an AFP mimic (brilacidin) potentiating caspofungin and a small organic molecule (imidazopyrazoindole) synergising with azoles^[294]17,[295]18. Notably, the possibility of re-sensitising drug-resistant fungi by the combination of active compounds is promising^[296]17,[297]18,[298]20. The key advantages of synthetic polymers are stability and cost-effective production at scale^[299]20,[300]30, which could solve many issues, especially in low-income countries^[301]9. In vitro evolution leads to tolerance of C. albicans to LH, but not to the combinations of LH with caspofungin or fluconazole A major obstacle in the development of antifungal drugs is the emergence of resistance. In this study, we follow the definition of resistance and tolerance suggested by Berman and Krysan^[302]80; where resistance is the clinically observed ineffectiveness of a drug against a fungal pathogen and tolerance is the slow growth of a less susceptible fungal strain at normally inhibitory concentrations in vitro^[303]80. An in vitro evolution assay (Fig. [304]7A) was used to determine whether prolonged exposure to polymer LH, caspofungin, fluconazole or their synergistic combinations results in the emergence of tolerant C. albicans variants. Growth at 1× MIC over 14 d (Fig. [305]7B) showed an increasing tolerance for the single drug treatments after 9–10 d incubation. A less pronounced tolerance developed for the combination of fluconazole and LH, while essentially no change in growth was seen for the combination of caspofungin and LH. Fig. 7. In vitro evolution experiment of C. albicans challenged with 1× MIC of caspofungin (Cas), fluconazole (Flu), polymer LH, and the synergistic combinations. [306]Fig. 7 [307]Open in a new tab A Experimental setup of the in vitro evolution experiment. B Growth of C. albicans was monitored by absorbance over 14 d and normalised to the untreated controls. Filled circles highlight strains that were selected for whole genome sequencing and empty circles highlight strains additionally analysed for their MIC against antifungal compounds in Supplementary Table [308]S5. Source data are provided as a Source Data file. C Genomes of the isolated strains with the highest tolerance to antifungal drugs were sequenced and analysed for their relative copy number for Flu- and LH-evolved strains. Each point in (C) represents the mean normalised read depth compared to wild-type C. albicans strain SC5314 at t = 0 for a gene (Y-axis) on its chromosome position (X-axis), colour-coded by allele. Positions of the centromeres are indicated by red circles. The MICs for Flu and LH are indicated on the right, where MIC in SC5314 is depicted in green and higher values scale to red. After 14 d, an aliquot of the cells was incubated at 2× MIC for 24–48 h. If growth was observed, the sample was plated, single colonies were isolated and their MIC to the antifungal polymers (LP, LH, CB, and CX) and antifungal drugs (AmpB, fluconazole, caspofungin, tunicamycin) was determined (Supplementary Table [309]S5). Isolates with an increased MIC were selected for further analysis (Fig. [310]7B, filled circles – increased tolerance to LH, empty circles – no increase in LH tolerance). In agreement with their growth pattern over 14 d, we did not find any stably tolerant isolates after treatment with combinations of LH and caspofungin or fluconazole (MICs in Supplementary Table [311]S5). To identify the genetic basis for LH tolerance, we sequenced the genomes of three independent LH-evolved strains with high MIC (evo-LH1, evo-LH2, and evo-LH3, filled black circles in Fig. [312]7B), and a fluconazole-evolved strain (evo-Flu1) as control. Evo-LH1 showed an increase in MIC against LH to 64–128 µg/mL and was also more tolerant to the polymers LP, CB, and CX. The MICs towards established antifungal drugs (AmpB, caspofungin, fluconazole, tunicamycin) remained unchanged. Interestingly, evo-Flu1 not only showed increased tolerance to fluconazole (MIC 2 µg/mL) but also to caspofungin and the polymers. The caspofungin-evolved strain (evo-Cas1) developed an increased MIC for caspofungin (2 µg/mL), but we did not observe any cross-tolerance. To investigate the genomic mechanisms of C. albicans adaption to LH or fluconazole, we examined the gene counts for the evolved strains compared to wild-type C. albicans SC5314 (Fig. [313]7C, Supplementary Fig. [314]S31). In all strains, including the parental wild-type SC5314, loss of heterozygosity was detected in the left arm of chromosome 2 (Supplementary Fig. [315]S31). Analysis of the evolved strains revealed major ploidy changes in some cases: In evo-Flu1, we observed aneuploidy of chromosome R and trisomy of chromosome 5. Both are associated with fluconazole resistance^[316]81,[317]82, and the chromosome 5 trisomy has been shown to be driven by amplification of the ERG11 and TAC1 genes^[318]83. For evo-LH1 and evo-LH2, we found aneuploidy in chromosome R, and trisomy in chromosome 2, respectively. Notably, chromosome 2 trisomy and tetrasomy have been linked to adaption to the ER stressor tunicamycin^[319]84. For the strain evo-LH3, which showed only a minor change in MIC, we found no large-scale ploidy changes. We did not find mutations or copy number variations on the gene level that could explain the drug tolerance phenotype, although some differences were observed between our isolated C. albicans strains on the nucleotide level (Supplementary Data [320]S2). We conclude that major chromosomal aberrations probably drive tolerance to polymer LH in C. albicans. Together with our transcriptomics and chemical-genetic screening evidence, we therefore postulate that polymer LH has multiple targets, which differ from those of known antifungal drugs. Importantly, in our in vitro evolution experiment we found no development of genetically stable tolerance by combinatorial treatment of LH with caspofungin or fluconazole, in further support of our hypothesis of distinct targets. The strong synergistic action with caspofungin against C. albicans on human epithelial cells in vitro and G. mellonella larvae in vivo, together with the reduced tolerance development suggests that the amphiphilic polymer LH is a promising antifungal lead for combination therapy with well-established drugs. In summary, we investigated four synthetic polymers, which were inspired by amphiphilic antimicrobial peptides and which kill drug-resistant clinical C. albicans isolates. Our findings reveal that the most promising polymer, LH, exerts its activity on C. albicans by a putatively novel mode of action. We found evidence that it targets protein glycosylation, and also interferes with the fungal membrane, which together leads to fungal cell death. The combination of LH with caspofungin is particularly promising in its therapeutic potential since it inhibits infection of human epithelial cells by C. albicans at otherwise sub-inhibitory concentrations and additionally increases fungal uptake by human macrophages. Moreover, the synergistic combination of polymer LH and caspofungin prolonged survival in an in vivo model of systemic candidiasis. In addition to these promising synergistic effects, which prevent C. albicans infection in vitro and protect in vivo, the combination of polymer LH and caspofungin did not lead to any tolerant C. albicans strains after prolonged exposure in vitro, highlighting the therapeutic potential of polymer LH as an antifungal lead, particularly for combination therapy. Future experiments may allow to further optimise polymer LH, e.g., by investigating the effect of monomer sequence or block order within the polymer on its activity. This way, it may be possible to retain the promising antifungal activity even as a stand-alone formulation, which would then also be envisioned to be tested in vertebrate in vivo infection models. Methods Ethics statement The research presented here complies with all relevant ethical regulations. The blood donation procedure was approved by the Jena institutional ethics committee (Ethik-Kommission des Universitätsklinikums Jena, Permission No 2207–01/08). All donors gave written informed consent and did not receive any compensation. Age, sex or gender of the donors was not taken into account. Materials for polymer synthesis Ethylenediamine (Sigma-Aldrich, ≥99%), N-amylamine (Sigma-Aldrich, 99%), N-benzylamine (Sigma-Aldrich, 99%), N-heptylamine (Sigma-Aldrich, 99%), N-cyclohexanemethylamine (Sigma-Aldrich, 98%), di-tert-butyl dicarbonate (Sigma-Aldrich, 99%), N-hydroxyethyl acrylamide (Sigma-Aldrich, 97%), triethylamine (TEA) (Scharlau, 99%), trifluoroacetic acid (TFA) (Sigma-Aldrich, 99%), chloroform (Merck), dichloromethane (DCM) (Merck), tetrahydrofuran (THF) (Merck), diethyl ether (Merck), hexane (Merck), dimethyl sulfoxide (DMSO) (Merck), dimethylacetamide (DMAc) (Sigma-Aldrich), thionyl chloride (Sigma-Aldrich, 99%), acrylic acid (Sigma-Aldrich), deuterated DMSO (Cambridge Isotope Laboratories, Inc.), 2-(butylthiocarbonothioylthio)propanoic acid (BTPA, Boron Molecular) and 5,10,15,20-tetraphenyl-21H,23H-porphine zinc (ZnTPP) (Sigma-Aldrich) were used as received. Acryloyl chloride synthesis Acryloyl chloride was synthesised according to the previously reported procedure^[321]85, with slight changes^[322]27. Briefly, acrylic acid (41.2 mL, 1.2 equiv) was added dropwise to 36.3 mL of thionyl chloride at 0 °C over 45 min under nitrogen. The mixture was stirred for 12 h at 40 °C. The product was collected by in situ distillation under atmospheric pressure. Synthesis of monomers Cationic monomer - tert-butyl (2-acrylamidoethyl)carbamate tert-Butyl (2-acrylamidoethyl)carbamate was prepared according to the previously reported procedure^[323]27,[324]86,[325]87 Ethylenediamine (0.33 mol) was dissolved in chloroform (400 mL). Di-tert-butyl dicarbonate (0.03 mol) was dissolved in 100 mL of chloroform and was added dropwise to the ethylenediamine solution over 4 h at 0 °C while stirring and continued overnight at room temperature. After filtering the white precipitate, the organic phase was washed with 200 mL of Milli-Q water six times and then dried using MgSO[4]. Solids were separated by filtration, and chloroform was evaporated, resulting in a pale yellow oil. THF (100 mL) was added to dissolve the obtained oil. TEA (1.2 equiv) and acryloyl chloride (1.1 equiv) were added dropwise to the solution at 0 °C with N[2] bubbling. The reaction mixture was stirred at room temperature for 2 h. Afterwards, THF was removed by rotary evaporation. The crude product was dissolved in chloroform (150 mL) and washed with 0.1 M HCl solution (1 × 75 mL), saturated NaHCO[3] (1 × 75 mL), brine (1 × 75 mL), and water (1 × 75 mL). The organic phase was dried using MgSO[4] and filtered, and the remaining solvent was removed by rotary evaporation. The product was further purified by repeated precipitation steps in hexane to yield the Boc-protected monomer as a fine white powder, which was dried in vacuo. Synthesis of hydrophobic monomers A standard procedure, as previously reported^[326]27,[327]87 was employed for the synthesis of four hydrophobic monomers ((N-pentylacrylamide, N-heptylacrylamide, N-cyclohexanemethyl)acrylamid e, and N-benzylacrylamide) from their corresponding amines (N-amylamine, N-heptylamine, N-cyclohexanemethylamine, or N-benzylamine) using acryloyl chloride: The specified amount of amine was dissolved in THF with a ratio of 6 mL of THF per 1 mmol amine. TEA (1.2 equiv) and acryloyl chloride (1.2 equiv) were added in a dropwise manner at 0 °C with N[2] bubbling and further stirred overnight at room temperature. The by-products were filtered, and the solvent was removed by rotary evaporation. The crude product was dissolved in chloroform (1.5× THF volume), washed sequentially with 0.1 M HCl, saturated NaHCO[3], brine, and water using half of the chloroform volume for each wash. The organic phase was dried with MgSO4 and basic Al2O3 and filtered to remove solids. Finally, the solvent was removed by rotary evaporation and dried in vacuo to yield the acrylamide monomer. Random copolymerisation by photo-induced electron/energy transfer-reversible addition-fragmentation chain transfer (PET-RAFT) polymerisation The linear, random copolymers were synthesised using a slight modification of the general one-pot protocol reported previously^[328]88 Briefly, stock solutions of the monomers were prepared with a concentration of 33% (w/w) in DMSO. ZnTPP was dissolved in DMSO at a concentration of 1 mg/mL. The RAFT agent BTPA was added to a 4 mL glass vial in an amount corresponding to the targeted X[n] of 20 and dissolved in DMSO. Monomer stock solutions were added into the vial to a final monomer concentration of 25% (w/w) in DMSO, corresponding to the targeted ratios. The photocatalyst (ZnTPP) was added at 100 ppm relative to the monomers. The vial was sealed with a rubber septum, and the headspace was degassed with N[2] for 10 min. The vial was then placed under a green light-emitting diode light (λ = 530 nm) for 20 h to produce the Boc-protected copolymers. The copolymers were analysed by size-exclusion chromatography (SEC) and ^1H nuclear magnetic resonance (NMR) to examine the monomer conversion, polymer composition, and molecular weight distribution. Then, the polymer was purified by precipitating in a diethyl ether/hexane mixture (3:7), followed by centrifugation (5000 × g for 5 min, 0 °C). The precipitate was dissolved in acetone or methanol and reprecipitated twice more. The polymer was then dried in vacuo prior to Boc-group removal. Polymer deprotection TFA was used to remove Boc-protecting groups based on our group’s previously reported protocol^[329]88 Briefly, the polymer was dissolved in DCM (∼7% (w/w) polymer), followed by the addition of TFA (20 mol equivalent with respect to Boc groups). The mixture was stirred at room temperature for 3 h and precipitated into diethyl ether. The precipitate was isolated by centrifugation, dissolved in acetone, and reprecipitated twice more. The polymer was then dried in vacuo, and ^1H NMR analysis was used to determine the removal of Boc-protective groups and to examine the targeted X[n]. Polymer characterisation ^1H NMR spectra were obtained using a Bruker AVANCE III spectrometer (300 MHz, 5 mm BBFO probe) or a Bruker AVANCE III 400 spectrometer (400 MHz, 5 mm BBFO probe). Deuterated DMSO was used as a solvent to determine the polymer composition and conversion at concentrations of ∼10–20 mg/mL. All experiments were run with a gas flow across the probes at 535 L/h with sample spinning and at a temperature of 25 °C. All chemical shifts were stated in parts per million (ppm) relative to tetramethylsilane. SEC analysis was performed using a Shimadzu liquid chromatography system equipped with a Shimadzu refractive index detector and three MIX C columns operating at 50 °C. DMAc (containing 0.03% (w/v) LiBr and 0.05% (w/v) 2,6-dibutyl-4-methylphenol) was used as the eluent at a flow rate of 1 mL/min. The system was calibrated using narrow poly(methyl methacrylate) (PMMA) standards with molecular weights from 200 to 10^6 g/mol. Media and buffers for biological experiments Phosphate-buffered saline (pH 7.4) A 10× phosphate-buffered saline (PBS) stock (1.37 mol/L sodium chloride, 0.027 mol/L potassium chloride, 0.08 mol/L disodium hydrogen phosphate, 0.02 mol/L potassium dihydrogen phosphate) was prepared by dissolving 80.06 g sodium chloride, 2.01 g potassium chloride, 11.36 g disodium hydrogen phosphate, and 2.72 g potassium dihydrogen phosphate in 900 mL of double-distilled water and the pH was adjusted to 7.4, before adding double-distilled water up to a final volume of 1 L. The solution was autoclaved for sterilisation. A 1× PBS solution was obtained by dissolving 100 mL of 10× PBS in 900 mL of sterile double-distilled water. Yeast extract peptone dextrose medium The yeast extract peptone dextrose (YEPD; 1% (w/v) yeast extract, 2% (w/v) mycological peptone, and 2% (w/v) d-glucose) broth was prepared by dissolving 4 g yeast extract and 8 g mycological peptone in double-distilled water up to a total volume of 360 mL. After autoclaving, 40 mL of filter-sterilised 20% (w/v) d-glucose was added. For YEPD plates, 8 g agar was added to the solution before autoclaving. Synthetic defined medium Synthetic defined (SD) medium was prepared by dissolving 6.7 g yeast nitrogen base (YNB) without amino acids, and 0.395 g complete supplement mixture were dissolved in 900 mL double-distilled water, adjusted pH to 6.0 with HCl and NaOH, and autoclaved. Afterwards, 100 mL of filter-sterilised 20% (w/v) d-glucose was added. When required, 5 mL of a filter-sterilised 5 mg/mL uridine solution was added, too. Modified Roswell Park Memorial Institute (RPMI)-1640 medium for C. albicans studies To 1 L of RPMI-1640 medium (with l-glutamine, without bicarbonate), 18 g d-glucose and 34.53 g 3-(N-morpholino)propane-1-sulfonic acid (MOPS) were added. Afterwards, the pH was adjusted with HCl and NaOH to 4.0 and filter-sterilised. Polymer and antifungal stock solutions Polymer stock solutions were prepared at a concentration of 10 mg/mL (3.4–3.7 mM, depending on the respective polymer composition) in sterile distilled water and stored at 4 °C. Before use, the stock solutions were sonicated for approx. 3 min. Antifungal drug stocks were prepared at different stock solutions in sterile distilled water or DMSO, as summarised in Table [330]3. Table 3. Concentrations, medium and storage temperature of antifungal stocks Antifungal compound Stock concentration Medium Storage temperature mg/mL mM Amphotericin B (AmpB) 1–2 1.1–2.2 DMSO 4 °C Calcofluor white 50 5.5 DMSO −20 °C Caspofungin 1 0.9 Distilled water −20 °C Cetyltrimethylammonium bromide (CTAB) 10 27.4 Distilled water Room temperature Congo red 6 8.6 Distilled water 4 °C Cycloheximide 5 17.8 Distilled water 4 °C Dodecyltrimethylammonium bromide (DTAB) 10 32.4 Distilled water Room temperature FK506 5 6.2 DMSO −20 °C Fluconazole 1–2 3.3–6.5 DMSO 4 °C Geldanamycin 5 8.9 DMSO −20 °C LL-37 1 0.2 Distilled water −20 °C Nikkomycin Z 5 10.1 Distilled water 4 °C Sodium dodecyl sulfate (SDS) 10 34.7 Distilled water Room temperature Tunicamycin 5 5.9 DMSO 4 °C [331]Open in a new tab Culture conditions of fungal strains The yeasts were routinely streaked on YEPD agar and incubated for 1–2 days at 30 °C (37 °C for clinical isolates). Cultures on agar plates were stored for up to two weeks at 4 °C. Long-term stocks were stored at −80 °C in 50% (v/v) sterile glycerol from an overnight culture. Overnight cultures were prepared by inoculating colonies from a YEPD plate in YEPD broth and shaking overnight at 30 °C at 180 rpm (37 °C for clinical isolates). Fungal strains A complete list of used fungal strains is attached in Supplementary Data [332]S3. For most assays, the C. albicans reference strain SC5314 was used, unless otherwise indicated. Minimum inhibitory concentration (MIC) assay and evaluation of drug interactions The MICs of polymers against different strains of C. albicans were determined via the broth microdilution method according to Clinical and Laboratory Standards Institute (CLSI) guidelines for fungal susceptibility testing, with slight modifications^[333]32,[334]33. Briefly, the C. albicans strains were grown on YEPD plates for 48 h at 30 °C (37 °C for clinical isolates). One colony was emulsified in 1 mL of sterile Milli-Q water. Cells were counted using a haemocytometer and adjusted to 2–5 × 10^6 cells/mL. The cell suspension was diluted 1:1000 in the modified RPMI-1640 medium (supplemented with d-glucose and MOPS, pH 4.0) to obtain the 2× concentrated stock suspension. A two-fold dilution series of the 100 μL polymer solution was added into 96-well microplates (final concentration between 4 and 512 μg/mL), followed by the addition of 100 μL of fungal cell suspension. The 96-well plates were incubated for 24 h at 35 °C in a humidified chamber, wells were resuspended, and the absorbance was measured at 405 nm with a microtiter plate reader. Additionally, AmpB, fluconazole, and caspofungin were tested at final concentrations between 0.125 and 16 (AmpB) and 0.06–8 μg/mL (fluconazole and caspofungin). DMSO controls at the respectively used final concentrations, no-polymer and no-cell controls were included in all experiments. The MIC value was defined as the lowest concentration of the respective polymer that showed growth inhibition of >90% compared to the untreated control. Three independent biological replicates were carried out (unless otherwise indicated). To evaluate interactions of the polymer LH with selected antifungal compounds (see Table [335]3), C. albicans cells were treated and prepared as described above. The drug-dilution plates were prepared separately for each drug before combining them. For that, a four-fold dilution series of the respective antifungal drug was added into a 96-well microplate along the rows. The same was performed with polymer LH, diluting it in a 96-well microplate along the columns. Then, 50 µL of antifungal were combined with 50 µL of polymer LH, resulting in a two-fold dilution series of the respective compounds. Thereby, one row and one column acted as controls only containing one compound. Drug interactions were classified according to their fractional inhibitory concentration (FIC) indices. The FIC index was calculated as described in the following Eq. ([336]1), where c[A/B] are the concentrations of compounds A or B, respectively, in combination resulting in growth inhibition >90%, and MIC[A] and MIC[B] are the MICs of compound A or B, respectively, alone: [MATH: FIC index=cAMICA+cBMICB :MATH] 1 A combination was called synergistic if the FIC index was below 0.5, antagonistic for an FIC index above 4 and values in between as additive (between 0.5 and 1.0) or indifferent (between 1 and 4). Three independent biological replicates were carried out. The MICs of polymer LH against C. albicans SC5314 in all used media are displayed in Supplementary Table [337]S6. RNA isolation, microarray and KEGG pathway/GO term enrichment analysis For RNA isolation, C. albicans SC5314 was grown in YEPD broth overnight, diluted 1:50 in YEPD broth and subcultured for 4 h (30 °C, 180 rpm). The cells were washed three times in PBS (2500 × g, 1 min) and adjusted to approx. 5 × 10^7 cells/mL in SD broth. The cell suspension was added to a final concentration of approx. 5 × 10^6 cells/mL into sterile glass flasks containing 6 mL SD medium supplemented with no polymer (for the untreated control) or 32 (LH, LL-37), 64 (CB and CX), or 128 µg/mL (LP and poly-HEA) polymer, corresponding to their MICs (Table [338]2) and being sub-inhibitory at the conditions of this assay (Supplementary Table [339]S6). The flasks were incubated for 1 h at 30 °C and 180 rpm. Three biological replicates were performed for each condition. Cell viability was ascertained by backplating on YEPD agar. Fungal cells were harvested for subsequent RNA isolation (2500 × g, 2 min, 4 °C) and handled on ice from that step onwards. RNA was then isolated using an RNeasy mini kit (QIAGEN) by mechanical disruption with acid-washed glass beads following instructions in the manual. The concentration and quality of RNA were checked by Nanodrop ND-1000 (ThermoScientific) and Bioanalyzer 2100 (Agilent). The Quick Amp Gene Expression Labeling Kit (Agilent) was used to synthesise Cy5-labelled cRNA. A common reference (RNA from a mid-log-phase-grown C. albicans SC5314^[340]89) was labelled with Cy3 following the same procedure. The dye incorporation was assured by spectrophotometric measurement using a NanoDrop ND-1000. Samples and common references were cohybridised on Agilent arrays (AMADID 026869)