Drug Delivery to the Brain: Challenges and Progress

摘要 请求为题为“向大脑输送药物：挑战和挑战”的Keystone专题讨论会提供支助。 进展，由罗伯特·索恩博士和雷纳·本达扬博士组织。会议将于 布雷肯里奇，科罗拉多，2023年1月26日至39日。 本次Keystone研讨会旨在解决对创新、新技术的迫切科学需求。 中枢神经系统（CNS）药物输送的方法。事实上，目前缺乏疾病修饰 阿尔茨海默氏症、帕金森氏症和其他中枢神经系统疾病的治疗是最重要的未满足的 我们时代的健康需求。我们有能力有效地挖掘蛋白质、寡核苷酸和 基因疗法用于治疗中枢神经系统疾病受到典型的脑功能不足的严重限制， 全身或中枢给药后产生的暴露。甚至许多口服小剂量 分子受到大脑暴露限制。这些限制的主要原因是 存在于关键CNS界面的物理和生化屏障，包括水平的血脑屏障 脑血管系统，血液和脑脊液之间的其他专门屏障，以及进一步 阻碍脑脊液和CNS组织之间药物交换的障碍。中枢神经系统给药研究 处于生理学、药理学、制药学等许多不同领域的交叉点， 科学、神经科学、神经外科、工程学、遗传学和血管生物学等。人惊讶 迄今为止，只有少数几次会议专门讨论了与中枢神经系统有关的多学科挑战 药物输送本次研讨会的目标是（一）汇集国际专家和初级 来自多个研究领域的研究人员，旨在交流新思想和集思广益 解决现有CNS药物递送挑战的方法，以及（ii）突出新方法和观点， 改变CNS药物递送研究的方式，并最终改变 领域会议将包括一些简短的演讲，旨在整合最新的发展和新的 研究方向从外地。

ABSTRACT Support is requested for a Keystone Symposia conference entitled Drug Delivery to the Brain: Challenges and Progress, organized by Drs. Robert Thorne and Reina Bendayan. The conference will be held in Breckenridge, Colorado from January 26-39, 2023. This Keystone Symposia conference is designed to address the urgent scientific need for innovative, new approaches to central nervous system (CNS) drug delivery. Indeed, the current shortage of disease-modifying treatments for Alzheimer’s, Parkinson’s and other CNS diseases represent among the most significant unmet health needs of our time. Our ability to effectively tap into the vast potential that protein, oligonucleotide, and gene therapies have for treating CNS disorders has been sharply limited by the typically insufficient brain exposure that results after their systemic or central administration. Even many orally administered small molecules suffer from brain exposure limitations. Among the primary reasons for these limitations are the physical and biochemical barriers that exist at key CNS interfaces, including the blood-brain barrier at the level of the cerebrovasculature, other specialized barriers between the blood and cerebrospinal fluid, and further obstacles hindering drug exchange between cerebrospinal fluid and CNS tissue. CNS drug delivery research lies at the crossroads between many different fields, including physiology, pharmacology, pharmaceutical science, neuroscience, neurosurgery, engineering, genetics, and vascular biology, among others. Surprisingly few conferences to date have focused exclusively on the multidisciplinary challenges associated with CNS drug delivery. The goals of this symposium are (i) to bring together international experts and junior investigators from multiple research fields for the purpose of exchanging new ideas and brainstorming novel solutions to existing CNS drug delivery challenges and (ii) to highlight new methods and perspectives with the potential to change how CNS drug delivery research studies are performed and, ultimately, to transform the field. Sessions are to include a number of short talks chosen to integrate late-breaking developments and new research directions from the field.