Ayoub Nahla
Department of Pharmacology and Toxicology, Faculty of Medicine, Umm Al-Qura University, Makkah 24375, Saudi Arabia.
J Clin Med. 2025 Mar 16;14(6):2021. doi: 10.3390/jcm14062021.
Botulinum toxin (BoNT), produced by Clostridium botulinum, has transitioned from being a lethal neurotoxin to a versatile therapeutic agent. Its ability to inhibit neurotransmitter release by targeting Soluble N-ethylmaleimide-sensitive factor Attachment Protein Receptor (SNARE) proteins underpins its applications in treating conditions such as spasticity, dystonia, chronic pain, and overactive bladder. The clinical and pharmacological properties of BoNT have been extensively studied, with significant advancements in its therapeutic use, safety profile, and understanding of associated adverse effects. This comprehensive review aims to consolidate historical developments, molecular mechanisms, clinical applications, and challenges associated with BoNT, with a focus on expanding its therapeutic scope while ensuring safety and efficacy. A narrative approach was used to analyze and synthesize insights from 155 references spanning experimental studies, clinical trials, and reviews. Key topics included BoNT's historical milestones, mechanisms of action, therapeutic applications, and adverse events. BoNT demonstrates remarkable efficacy in a wide range of medical and cosmetic applications. In movement disorders such as dystonia and spasticity, it reduces muscle overactivity and improves functional outcomes. In chronic pain management, including migraines and neuropathic pain, BoNT significantly alleviates symptoms by modulating neurotransmitter activity. Cosmetic use for conditions like glabellar lines and hyperhidrosis highlights its precision and safety when administered appropriately. For conditions like strabismus and blepharospasm, BoNT effectively restores muscle control, reducing involuntary contractions. In urological applications, BoNT has proven to be an effective therapy for overactive bladder, offering significant symptom relief in refractory cases. However, concerns about long-distance effects, where the toxin may spread beyond the injection site to affect distant muscles or systems, have been reported in certain high-dose or sensitive populations. These findings emphasize the importance of dose optimization and patient-specific approaches. Adverse effects such as localized pain, hematoma, dysphagia, and systemic effects, particularly in high-risk groups, underscore the need for careful monitoring. The development of immunogenicity, leading to neutralizing antibodies, remains a challenge that impacts long-term therapeutic efficacy. Emerging research on novel serotypes, including BoNT/X, and innovations in delivery mechanisms, offer promising avenues to address current limitations. Advances in optimizing dosing regimens and refining injection techniques have also contributed to minimizing complications and improving outcomes across diverse patient populations. BoNT remains a cornerstone in neurology and cosmetic medicine, with its therapeutic potential still expanding. The balance between efficacy and safety, driven by innovations in formulation and application, underscores the importance of continued research. Future directions should focus on minimizing adverse effects, reducing immunogenicity, and exploring novel indications to further enhance its clinical utility.
肉毒杆菌毒素(BoNT)由肉毒梭菌产生,已从一种致命的神经毒素转变为一种多功能治疗剂。它通过靶向可溶性N - 乙基马来酰亚胺敏感因子附着蛋白受体(SNARE)蛋白来抑制神经递质释放的能力,是其用于治疗痉挛、肌张力障碍、慢性疼痛和膀胱过度活动症等病症的基础。BoNT的临床和药理特性已得到广泛研究,在其治疗用途、安全性概况以及对相关不良反应的认识方面都取得了重大进展。本综述旨在整合与BoNT相关的历史发展、分子机制、临床应用和挑战,重点是在确保安全性和有效性的同时扩大其治疗范围。采用叙述性方法分析和综合了来自155篇参考文献的见解,这些文献涵盖实验研究、临床试验和综述。关键主题包括BoNT的历史里程碑、作用机制、治疗应用和不良事件。BoNT在广泛的医学和美容应用中显示出显著疗效。在肌张力障碍和痉挛等运动障碍中,它可减少肌肉过度活动并改善功能结果。在慢性疼痛管理中,包括偏头痛和神经性疼痛,BoNT通过调节神经递质活性显著缓解症状。在眉间纹和多汗症等美容用途中,适当给药时突出了其精确性和安全性。对于斜视和眼睑痉挛等病症,BoNT可有效恢复肌肉控制,减少不自主收缩。在泌尿外科应用中,BoNT已被证明是治疗膀胱过度活动症的有效疗法,在难治性病例中可显著缓解症状。然而,在某些高剂量或敏感人群中,已报告存在对远距离效应的担忧,即毒素可能扩散到注射部位以外影响远处肌肉或系统。这些发现强调了剂量优化和个体化治疗方法的重要性。局部疼痛、血肿、吞咽困难等不良反应以及全身效应,特别是在高危人群中,凸显了仔细监测的必要性。免疫原性的产生导致中和抗体,仍然是影响长期治疗效果的一个挑战。对新型血清型(包括BoNT/X)的新兴研究以及给药机制的创新,为解决当前局限性提供了有希望的途径。优化给药方案和改进注射技术的进展也有助于减少并发症并改善不同患者群体的治疗结果。BoNT仍然是神经学和美容医学的基石,其治疗潜力仍在不断扩大。由制剂和应用创新驱动的疗效与安全性之间的平衡,强调了持续研究的重要性。未来的方向应集中在尽量减少不良反应、降低免疫原性以及探索新的适应症,以进一步提高其临床效用。