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Enkephalin

Enkephalins are endogenous pentapeptides that function as neurotransmitters and neuromodulators in the central and peripheral nervous systems, primarily known for their role in regulating and relief by activating specific receptors. Discovered in 1975 by John Hughes and Hans Kosterlitz through isolation from porcine brain tissue, these peptides were identified as natural ligands for opiate receptors, marking a pivotal advancement in understanding endogenous control mechanisms. The two primary forms are (Tyr-Gly-Gly-Phe-Met) and (Tyr-Gly-Gly-Phe-Leu), which share an identical N-terminal sequence but differ at the , enabling their selective interactions with biological targets. Biosynthesized from the precursor protein proenkephalin (PENK) through proteolytic cleavage, enkephalins are produced in various tissues, including the , , adrenal glands, and , yielding multiple copies per precursor molecule in humans—specifically six sequences and one . Their activity is tightly regulated by rapid enzymatic degradation via peptidases such as enkephalinases, which limits their duration of action and contributes to their role in fine-tuned physiological responses. As part of the broader endogenous opioid system—which includes , dynorphins, and nociceptin—enkephalins belong to one of four major families derived from distinct precursors, highlighting their evolutionary conservation across vertebrates for modulating stress and survival-related behaviors. Enkephalins exert their effects mainly through binding to delta-opioid receptors (DOR) with high affinity, and to a lesser extent mu-opioid receptors (MOR), both G-protein-coupled receptors distributed throughout the (e.g., periaqueductal gray matter and substantia gelatinosa of the ), peripheral nerves, and immune cells. In pain modulation, they inhibit the release of excitatory neurotransmitters like from nociceptive afferents, producing analgesia particularly at sites of inflammation where they can be released by immune cells. Beyond analgesia, enkephalins influence reward processing, stress responses, gastrointestinal motility (e.g., inhibiting ), and immune function, with met-enkephalin also acting as an opioid growth factor to regulate . Dysregulation of enkephalinergic systems has been implicated in conditions such as substance use disorders, underscoring their therapeutic potential in and .

Discovery and Nomenclature

Historical Identification

The discovery of enkephalins emerged from the burgeoning field of opioid research in the early , following the identification of specific receptors in the . In 1973, researchers demonstrated the existence of these receptors through radioligand binding studies on , prompting a search for endogenous ligands that could explain morphine's effects without external drugs. This context was further intensified by the isolation of beta-endorphin from pituitary extracts in 1975, highlighting the presence of natural -like peptides. In 1975, John Hughes and Hans Kosterlitz at the initiated a targeted effort to identify activity in tissue distinct from known peptides like beta-endorphin. They fractionated extracts from pig using techniques such as gel filtration and ion-exchange chromatography to isolate low-molecular-weight components (800–1200 Da) exhibiting properties. Initial evidence came from bioassays monitoring the inhibition of electrically stimulated contractions in isolated tissues: the mouse vas deferens for delta- selectivity and the guinea pig ileum for mu- activity, revealing opioid-like depression of release separate from other peptides. This work was first reported in a seminal paper detailing the purification and properties of the ligand, termed "enkephalin" provisionally. Confirmation of enkephalins as pentapeptides followed rapidly through advanced analytical methods. In late 1975, Hughes and colleagues sequenced the peptides using the dansyl-Edman degradation procedure and verified their structures via , synthesizing analogs to match the natural compounds' potency in bioassays. These findings were published in , establishing enkephalins as the first identified endogenous from brain tissue and integrating them into the broader endogenous opioid system alongside .

Naming Conventions

The term "enkephalin" was coined in 1975 by John Hughes and colleagues to describe the endogenous opioid pentapeptides they isolated from porcine tissue, deriving the name from the Greek words en (ἐν, meaning "in") and kephalē (κεφαλή, meaning "brain" or "head") to emphasize their cerebral origin. This nomenclature distinguished the brain-specific peptides from broader classes of endogenous s, such as —initially a generic term for morphine-like substances proposed around the same time but later refined to refer primarily to peptides derived from pro-opiomelanocortin, like β-endorphin—and endomorphins, opioids discovered in 1997 with high selectivity for μ-opioid receptors. In , enkephalins follow International Union of Pure and Applied Chemistry (IUPAC) conventions for peptides, with denoted as L-tyrosylglycylglycyl-L-phenylalanyl-L-methionine (commonly abbreviated as H-Tyr-Gly-Gly-Phe-Met-OH) and as L-tyrosylglycylglycyl-L-phenylalanyl-L-leucine (H-Tyr-Gly-Gly-Phe-Leu-OH). Over time, the term evolved to encompass not only these core pentapeptides but also longer variants derived from the proenkephalin precursor, such as Met-enkephalin-Arg⁶-Phe⁷, reflecting the processing of the proenkephalin gene product identified in the early . The 1970s saw significant debates in the scientific literature over opioid peptide nomenclature, driven by the rapid discovery of multiple ligands and the initial broad application of "endorphin" to all endogenous opioids, which led to confusion in distinguishing structural and functional families. These discussions, involving key researchers like Hughes, Kosterlitz, and Goldstein, culminated in standardized terminology by the 1980s, classifying peptides by their biosynthetic precursors—proenkephalin for enkephalins, pro-opiomelanocortin for endorphins, and prodynorphin for dynorphins—to provide clarity for ongoing research.

Chemical Structure and Types

Amino Acid Sequences

Enkephalins are pentapeptides characterized by their conserved N-terminal sequence Tyr-Gly-Gly-Phe, which is critical for their , followed by a variable C-terminal residue that distinguishes the primary variants. , the methionine-containing form, has the sequence Tyr-Gly-Gly-Phe-Met. Its molecular formula is C<sub>27</sub>H<sub>35</sub>N<sub>5</sub>O<sub>7</sub>S, with a molecular weight of 573.7 . Leu-enkephalin, the leucine-containing form, has the sequence Tyr-Gly-Gly-Phe-. Its molecular formula is C<sub>28</sub>H<sub>37</sub>N<sub>5</sub>O<sub>7</sub>, with a molecular weight of 555.6 Da. A key structural feature common to both is the N-terminal residue, which is essential for and activity due to its phenolic hydroxyl group interacting with receptor pockets. The C-terminal methionine in Met-enkephalin versus in Leu-enkephalin influences receptor subtype affinity, with the hydrophobic side chain variations modulating selectivity, particularly for δ-opioid receptors where both exhibit nanomolar potency but subtle differences in μ-receptor . These sequences are derived from larger precursor proteins through post-translational processing.

Precursor Proteins

Enkephalins are primarily synthesized from the proenkephalin (PENK) precursor protein, encoded by the located on the long arm of human at position 8q12.1. This gene produces a preproenkephalin polypeptide consisting of 267 , which includes a 24-amino-acid at the followed by the proenkephalin propeptide. The precursor serves as a polyprotein template, embedding multiple sequences that are later cleaved to yield mature enkephalins. The structure of proenkephalin features six copies of the pentapeptide (Tyr-Gly-Gly-Phe-Met) and one copy of the pentapeptide (Tyr-Gly-Gly-Phe-Leu), interspersed throughout the propeptide chain. These sequences are positioned such that five of the seven enkephalins are flanked by pairs of basic residues, facilitating proteolytic processing, while the remaining two are adjacent without such dibasic sites. This arrangement allows for the generation of multiple identical or similar peptides from a single precursor molecule, optimizing efficient production in neurons and other expressing cells. The PENK gene and its encoded precursor exhibit high evolutionary conservation across mammals. This conservation underscores the fundamental role of proenkephalin in signaling, maintained through selective pressure on the embedded enkephalin motifs. While alternative precursors exist for other peptides—such as pro-opiomelanocortin (POMC), which yields —enkephalins are predominantly derived from PENK, distinguishing their biosynthetic pathway.

Biosynthesis and Metabolism

Gene Expression and Processing

The preproenkephalin (PENK) gene, which encodes the precursor protein for enkephalins, is primarily transcribed in neurons of the (CNS) and the . Transcriptional regulation of the PENK gene involves response elements () located in its promoter and enhancer regions, which bind CRE-binding protein (CREB) to mediate -dependent activation. In CNS neurons, such as those in the , depolarization and signaling synergistically enhance PENK expression through these , contributing to adaptive responses in neural circuits. Similarly, in adrenal chromaffin cells, elevation—often triggered by stimulation—upregulates PENK transcription via CRE-mediated pathways, facilitating enkephalin production under physiological . Following transcription and , the preproenkephalin precursor undergoes extensive post-translational to generate active enkephalin peptides. This involves endoproteolytic at paired basic sites (Lys-Arg or Lys-Lys) by prohormone convertases PC1/3 and PC2, which are expressed in neuroendocrine cells and neurons. PC1/3 preferentially produces intermediate fragments, such as peptide B and larger enkephalin-containing peptides, while PC2 facilitates more complete to yield smaller, active forms like and . Subsequent trimming of C-terminal basic residues by carboxypeptidase E (CPE) is essential to produce the mature, bioactive pentapeptides, ensuring proper amidation and functionality. PENK gene expression exhibits tissue-specific patterns, with particularly high levels in the and , regions critical for modulation and stress responses. This expression is dynamically regulated by , including corticotropin-releasing factor (), which upregulates PENK mRNA in hypothalamic nuclei during acute stressors like isolation or immobilization. Enkephalins are then released from synaptic terminals through activity-dependent vesicular , where triggers calcium influx and fusion of large dense-core vesicles containing the peptides, allowing modulation of nearby neural activity.

Enzymatic Degradation

Enkephalins are rapidly inactivated through hydrolysis by membrane-bound ectopeptidases, which limits their duration of action and contributes to their role as short-acting neurotransmitters. The primary enzymes responsible for this degradation are neutral endopeptidase (NEP, 3.4.24.11, also known as CD10 or ) and aminopeptidase N (APN, 3.4.11.2, CD13). NEP preferentially cleaves the Gly³-Phe⁴ amide bond within the core sequence of both (Tyr-Gly-Gly-Phe-Met) and (Tyr-Gly-Gly-Phe-Leu), generating inactive fragments that further facilitate clearance. APN, a zinc-dependent exopeptidase, initiates degradation by excising the N-terminal residue, producing a (Gly-Gly-Phe) that is subsequently processed by other peptidases. This enzymatic cascade results in an extremely short for enkephalins, typically ranging from 2 to 5 minutes in circulating blood and neural tissues due to the high efficiency of these hydrolases. (ACE, EC 3.4.15.1) plays a secondary role, particularly in degrading extended enkephalin variants like -Arg⁶-Phe⁷, but contributes minimally to the breakdown of the canonical pentapeptides. The rapid turnover ensures precise spatiotemporal control of enkephalin signaling, preventing prolonged activation of receptors. NEP exhibits prominent distribution, with high activity in the brush-border membranes of renal proximal tubules and in regions rich in peptides, such as the and , thereby modulating local enkephalin concentrations in these organs. Similarly, APN is ubiquitously expressed on cell surfaces, including in the and peripheral tissues, complementing NEP's action. Pharmacologically, inhibition of NEP has been explored to extend enkephalin . , a metabolized to the active NEP inhibitor thiorphan, elevates endogenous enkephalin levels in the , reducing fluid secretion without central effects, as demonstrated in clinical use for acute . This approach underscores the therapeutic potential of targeting enkephalin degradation pathways while highlighting the enzyme's role in peripheral regulation.

Receptors and Mechanism of Action

Primary Receptor Interactions

Enkephalins primarily bind to the delta-opioid receptor (DOR), with demonstrating high affinity characterized by Ki values in the range of 2-7 across , rat, and mouse models. also exhibits strong selectivity for DOR, with a Ki of approximately 2 in receptors, showing a slight preference over other subtypes. This binding profile underscores the role of enkephalins as endogenous agonists predominantly targeting DOR to mediate their physiological effects. In addition to DOR, enkephalins display moderate affinity for the mu-opioid receptor (MOR), with showing Ki values around 19-28 nM and around 8 nM, indicating lower potency compared to DOR. Affinity for the kappa-opioid receptor (KOR) is notably lower, with Ki values exceeding 1 μM for both , resulting in minimal interaction at this subtype. DOR, the primary target of enkephalins, is abundantly distributed in the , particularly in the and , where it modulates neuronal activity. In the periphery, DOR is highly expressed in the , contributing to regulatory functions in enteric . The affinity of enkephalins for DOR is subject to allosteric modulation by sodium ions, which reduce potency by stabilizing an inactive receptor conformation. This sodium-dependent effect, observed in biochemical assays, highlights a key regulatory mechanism influencing enkephalin efficacy at DOR.

Signaling Pathways

Upon binding to their G protein-coupled receptors, primarily the delta (δ) and mu (μ) opioid receptors, enkephalins activate pertussis toxin-sensitive Gi/o proteins, leading to the dissociation of the Gαi/o subunit from the Gβγ complex. The Gαi/o subunit directly inhibits adenylate cyclase activity, thereby reducing intracellular cyclic AMP (cAMP) levels and subsequent protein kinase A (PKA) activation, which modulates downstream effectors such as ion channels and gene transcription. This cAMP suppression is a core mechanism for rapid cellular responses, including decreased neuronal excitability. The Gβγ subunits freed upon Gi/o activation exert direct effects on s: they open G protein-gated inwardly rectifying (GIRK) channels, promoting K⁺ efflux and membrane hyperpolarization, which inhibits firing. Concurrently, Gβγ inhibits voltage-gated calcium (Ca²⁺) channels, particularly N-type channels, reducing Ca²⁺ influx and thereby suppressing presynaptic release, such as glutamate or in nociceptive pathways. These modulations contribute to the acute inhibitory tone elicited by enkephalins. For sustained signaling, enkephalin-bound receptors recruit β-arrestins following by kinases (GRKs), initiating receptor desensitization, internalization via clathrin-coated pits, and trafficking to endosomes, which limits further activation. β-Arrestin recruitment also scaffolds (MAPK) pathways, particularly extracellular signal-regulated kinase (ERK1/2), enabling long-term adaptations like or cellular proliferation through cascades involving and Raf. This ERK activation occurs independently of Gi/o in some contexts, highlighting biased signaling potential. Tissue-specific variations in these pathways underscore enkephalin's versatility. In neurons, Gi/o-mediated presynaptic inhibition predominates, where reduced Ca²⁺ entry curtails excitatory transmitter release at synapses, as observed in spinal dorsal horn circuits. In immune cells, such as macrophages and T lymphocytes, enkephalin signaling via μ and δ receptors modulates release; for instance, it suppresses pro-inflammatory cytokines like TNF-α and IL-6 through inhibition, while enhancing IL-10, thereby regulating inflammatory responses.

Physiological Roles

Pain Modulation

Enkephalins, primarily acting through delta-opioid receptors (DOR), play a crucial role in endogenous analgesia by inhibiting nociceptive transmission at the spinal level. In the , enkephalin-containing in the substantia gelatinosa (laminae I and II of the dorsal horn) release met- and to presynaptically inhibit the release of excitatory neurotransmitters such as and glutamate from primary afferent nociceptors. These mechanisms include presynaptic inhibition of the release of excitatory neurotransmitters and postsynaptic hyperpolarization of nociceptive neurons, reducing the propagation of pain signals to higher centers. Seminal studies have demonstrated that enkephalin immunoreactivity is densely localized in these superficial laminae, confirming their direct involvement in gating pain transmission. Supraspinally, enkephalins contribute to pain modulation by enhancing descending inhibitory pathways, particularly in the (PAG) matter. Here, enkephalins bind DOR on , disinhibiting projection neurons that activate serotonergic and noradrenergic systems in the and , respectively. These descending projections then synapse onto spinal enkephalinergic , amplifying local inhibition of . This mechanism integrates emotional and sensory aspects of relief, with DOR activation in the PAG shown to synergize with mu-opioid pathways for robust antinociception in preclinical models. Clinical evidence supports enkephalins' role in pain states, with of enkephalin analogs like D-Ala²-D-Leu⁵-enkephalin (DADLE) reducing behaviors in animal models of and by mimicking endogenous release. In humans, (CSF) levels of are elevated in , suggesting compensatory activation of the enkephalinergic system during ongoing . Unlike mu-opioid agonists such as , which primarily target mu-receptors and induce significant respiratory depression, enkephalins via DOR exhibit minimal impact on respiration and show greater selectivity for , potentially due to upregulated DOR expression in damaged nerves.

Stress Response

Enkephalins are released in key brain regions such as the and during acute , contributing to the modulation of the body's response. This release helps regulate the hypothalamic-pituitary-adrenal () axis by acting through delta opioid receptors (DOR) to inhibit (CRH) neurons in the paraventricular nucleus of the (PVH). Specifically, activation of DOR on these CRH neurons suppresses CRH secretion, thereby attenuating the downstream activation of the axis and limiting excessive release. In the context of reward and aversion processing, enkephalins play a protective role against stress-induced , the reduced ability to experience pleasure. Downregulation of enkephalin signaling in the , a key reward center, has been shown to underlie the development of anhedonia following exposure. Additionally, enkephalin levels are elevated in certain models of post-traumatic stress, potentially as a compensatory mechanism to mitigate and aversion-related behaviors. Enkephalins also interact with the catecholaminergic system to dampen outflow. In the , enkephalins co-localize with catecholamines in chromaffin cells and exert an inhibitory effect on their release through DOR activation, reducing the sympathetic surge during . This autocrine or paracrine feedback helps prevent overactivation of the . Experimental studies in demonstrate dynamic changes in enkephalin expression under conditions. For instance, chronic variable significantly increases proenkephalin mRNA levels in the , a striatal subregion, reflecting an adaptive upregulation to cope with prolonged . Similar elevations in enkephalin-related transcripts have been observed in response to acute stressors, highlighting the system's responsiveness.

Clinical and Research Implications

Therapeutic Applications

Enkephalin analogs, such as D-Ala²-D-Leu⁵-enkephalin (DADL), have been explored for intrathecal delivery to manage spinal pain, particularly in cases of opioid tolerance. In clinical trials during the 1980s, intrathecal administration of DADL produced analgesia in patients with chronic pain, including those unresponsive to mu-opioid agonists like morphine, due to its selectivity for delta-opioid receptors (DOR) and lack of cross-tolerance. Further studies in the mid-1980s confirmed its efficacy in cancer patients with intractable pain, demonstrating dose-dependent pain relief without significant respiratory depression. These trials highlighted DADL's potential for targeted spinal analgesia, though development was limited by challenges in stability and delivery. Neutral endopeptidase (NEP) inhibitors, including derivatives of thiorphan, represent another approach by elevating endogenous enkephalin levels through blockade of enzymatic degradation, thereby enhancing their antinociceptive and pro-absorptive effects in the . This strategy has shown promise for treating (IBS), particularly the diarrhea-predominant subtype, where enkephalins regulate motility and secretion via DOR activation. Preclinical and early clinical evidence indicates that dual enkephalinase inhibitors (targeting NEP and N) reduce visceral and improve bowel function without the constipating side effects of mu-opioids. Emerging therapies include gene therapy approaches involving overexpression of the preproenkephalin (PENK) gene to sustain enkephalin production in chronic pain models, with potential applications in neurodegenerative pain conditions like neuropathic pain associated with disease progression. A phase I clinical trial using a herpes simplex virus vector expressing human PENK demonstrated safety and dose-dependent pain reduction in cancer patients, suggesting feasibility for broader chronic pain management. As of 2025, intranasal enkephalin formulations like Envelta (NES100) are undergoing IND-enabling studies for neuropathic pain, aiming to improve delivery via nanotechnology. Selective DOR agonists such as ADL5859 and ADL5747, which are enkephalin-derived analogs, advanced to phase II clinical trials in the early 2010s for the treatment of chronic and neuropathic pain. While they showed no seizure risks at therapeutic doses, the trials did not meet primary efficacy endpoints. A key advantage of enkephalin-based therapies, particularly those targeting DOR selectivity, is their lower abuse potential compared to traditional mu-opioid agonists, as they elicit reduced release in reward pathways and minimal respiratory depression or dependency. This profile positions DOR-selective enkephalin analogs as safer alternatives for long-term , addressing the opioid crisis while maintaining potent analgesia.

Pathological Associations

Postmortem brain studies have revealed reduced expression of the proenkephalin (PENK) gene, which encodes enkephalins, in individuals with , in brain regions such as the compared to controls. This downregulation is thought to contribute to dysregulated endogenous signaling, potentially exacerbating mood dysregulation, as evidenced by decreased enkephalin levels in and cortical tissue from depressed patients and victims. Evidence for PENK reductions in is inconsistent. In , particularly , enkephalins exhibit upregulation in the following activity, as demonstrated in animal models where a single dose of —a -inducing agent—led to long-lasting increases in PENK mRNA and enkephalin protein levels. This upregulation is associated with effects mediated primarily through delta opioid receptors (DOR) in the , where DOR activation inhibits neuronal excitability and reduces susceptibility by modulating transmission and during hypoxic episodes. Such findings suggest that enhanced enkephalin signaling may serve as an endogenous protective mechanism against recurrent . Chronic opioid use, such as prolonged administration, downregulates enkephalin systems, including decreased proenkephalin mRNA expression in key brain regions like the , which contributes to the development of analgesic tolerance. This adaptive response involves reduced endogenous synthesis and receptor desensitization, leading to diminished pain relief efficacy over time and heightened vulnerability to dependence, as the body's natural enkephalin-mediated analgesia is suppressed. In cancer research, particularly models, enkephalin analogs like [D-Ala², D-Leu⁵]enkephalin (DADLE)—a selective DOR agonist—have been shown to inhibit tumor growth by inducing in cells, with studies from the late 2010s and early 2020s demonstrating reduced and increased activation . These effects highlight the potential antitumor role of enkephalinergic signaling, where DOR activation promotes pathways, offering insights into novel therapeutic strategies for hormone-refractory malignancies.