Mukherjee Abhiyanta, Biswas Soumojit, Roy Ipsita
Department of Biotechnology, National Institute of Pharmaceutical Education and Research, S.A.S. Nagar, Punjab, India.
Acta Pharmacol Sin. 2025 Jun;46(6):1511-1538. doi: 10.1038/s41401-024-01455-w. Epub 2025 Jan 31.
Immunotherapy has emerged as a promising therapeutic approach for the treatment of neurodegenerative disorders, which are characterized by the progressive loss of neurons and impaired cognitive functions. In this review, active and passive immunotherapeutic strategies that help address the underlying pathophysiology of Huntington's disease (HD) and prion diseases by modulating the immune system are discussed. The current landscape of immunotherapeutic strategies, including monoclonal antibodies and vaccine-based approaches, to treat these diseases is highlighted, along with their potential benefits and mechanisms of action. Immunotherapy generally works by targeting disease-specific proteins, which serve as the pathological hallmarks of these diseases. Additionally, the review addresses the challenges and limitations associated with immunotherapy. For HD, immunotherapeutic approaches focus on neutralizing the toxic effects of mutant huntingtin and tau proteins, thereby reducing neurotoxicity. Immunotherapeutic approaches targeting flanking sequences, rather than the polyglutamine tract in the mutant huntingtin protein, have yielded promising outcomes for patients with HD. In prion diseases, therapies attempt to prevent or eliminate misfolded proteins that cause neurodegeneration. The major challenge in prion diseases is immune tolerance. Approaches to overcome the highly tolerogenic nature of the prion protein have been discussed. A common hurdle in delivering antibodies is the blood‒brain barrier, and strategies that can breach this barrier are being investigated. As protein aggregation and neurotoxicity are related, immunotherapeutic strategies being developed for other neurodegenerative diseases could be repurposed to target protein aggregation in HD and prion diseases. While significant advances in this field have been achieved, continued research and development are necessary to overcome the existing limitations, which will help in shaping the future of immunotherapy as a strategy for managing neurological disorders.
免疫疗法已成为治疗神经退行性疾病的一种有前景的治疗方法,这些疾病的特征是神经元逐渐丧失和认知功能受损。在这篇综述中,讨论了通过调节免疫系统来解决亨廷顿舞蹈病(HD)和朊病毒疾病潜在病理生理学的主动和被动免疫治疗策略。重点介绍了目前用于治疗这些疾病的免疫治疗策略,包括单克隆抗体和基于疫苗的方法,以及它们的潜在益处和作用机制。免疫疗法通常通过靶向疾病特异性蛋白质起作用,这些蛋白质是这些疾病的病理标志。此外,综述还讨论了与免疫疗法相关的挑战和局限性。对于HD,免疫治疗方法侧重于中和突变型亨廷顿蛋白和tau蛋白的毒性作用,从而降低神经毒性。针对突变型亨廷顿蛋白侧翼序列而非聚谷氨酰胺序列的免疫治疗方法已为HD患者带来了有希望的结果。在朊病毒疾病中,治疗方法试图预防或消除导致神经退行性变的错误折叠蛋白。朊病毒疾病的主要挑战是免疫耐受。已经讨论了克服朊病毒蛋白高度致耐受性的方法。递送抗体的一个常见障碍是血脑屏障,目前正在研究能够突破这一屏障的策略。由于蛋白质聚集与神经毒性相关,正在为其他神经退行性疾病开发的免疫治疗策略可重新用于靶向HD和朊病毒疾病中的蛋白质聚集。虽然该领域已取得重大进展,但仍需要持续的研究和开发来克服现有局限性,这将有助于塑造免疫疗法作为管理神经系统疾病策略的未来。