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相关概念视频

Preclinical Development: Overview01:28

Preclinical Development: Overview

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Preclinical development consists of a series of tests that ensure the safety and efficacy of a new therapeutic compound before it is tested in humans. There are four main phases to this process. First, safety pharmacology tests are conducted to ensure the drug does not produce any acutely harmful effects. These tests examine parameters such as bronchoconstriction, cardiac dysrhythmias, blood pressure changes, and ataxia. Next, preliminary toxicological testing is performed to determine the...
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Clinical Trials: Overview01:11

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Clinical development focuses on how the drug will interact with the human body and encompasses four key phases of clinical trials, each serving a specific purpose in assessing the safety and effectiveness of new drugs. These phases overlap and build upon one another. Phase I involves a small group of healthy volunteers (typically 20-80 individuals) or, in cases where significant toxicity is expected, patients with the targeted disease, such as cancer or AIDS. The volunteers are tested for...
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Drug Discovery: Overview01:26

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Drug discovery is a multifaceted process involving extensive screening, testing, and optimization of lead compounds to identify potential new drugs for therapeutic use. It combines several approaches, including screening large numbers of natural products, chemical modification of known active molecules, identification of new drug targets, and rational design based on biological mechanisms and drug-receptor structure. These approaches are carried out in both academic research laboratories and...
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Drug Administration and Therapy Phases: Overview01:26

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Drugs, the chemical agents used in diagnosing, treating, or preventing diseases, undergo a four-phase process of development: pharmaceutic, pharmacokinetics, pharmacodynamics, and therapeutic.
The pharmaceutical phase focuses on leveraging the physicochemical properties of the drug to design and manufacture an effective product. Variants include orally administered tablets or capsules, topical creams or ointments, and parenteral-delivery solutions or emulsions.
The pharmacokinetic phase...
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In Vitro Drug Release Testing: Overview, Development and Validation01:10

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In vitro dissolution and drug release tests assess how quickly and how much of a drug is released from its dosage form into an aqueous medium under standardized laboratory conditions. These tests are essential tools in pharmaceutical development and quality assurance, offering insight into the drug's performance before clinical use.During formulation development, dissolution testing identifies incomplete or inconsistent drug release issues. It also supports decisions on selecting the optimal...
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Drug Regulation01:25

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Drug regulation encompasses the management of drug usage by evaluating its safety and efficacy through assessments conducted by regulatory authorities. Regrettably, the history of drug regulation is marred by several catastrophic events. One such incident is the Elixir Sulfanilamide tragedy, in which the toxic compound diethyl glycol was included in a sweet-tasting medication, leading to numerous fatalities. This event prompted the enactment of the Food, Drug, and Cosmetic Act in 1938. Under...
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药物开发 药物开发

Sharon Cohen1, Simon Ducharme2, Jared R Brosch3

  • 1Toronto Memory Program, Toronto, ON, Canada.

Alzheimer's & dementia : the journal of the Alzheimer's Association
|December 25, 2025
PubMed
概括
此摘要是机器生成的。

米维尔西兰是一种RNA干扰疗法,在早期阿尔茨海默病患者中显示出良好的安全性和降低了粉样蛋白前体蛋白. 多次剂量进一步降低了sAPPβ,支持其在阿尔茨海默病和脑粉样血管病变治疗中的潜力.

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科学领域:

  • 神经科学是一个神经科学.
  • 药理学 药理学是指药理学的学科.
  • 遗传学 是一个遗传学.

背景情况:

  • 米维尔西兰是一种研究中的RNA干扰 (RNAi) 治疗药物,向中枢神经系统 (CNS).
  • 之前的研究表明,单次用米特西兰剂量有效降低了粉样β前体蛋白 (APP).
  • 这项研究报告了早期阿尔茨海默病 (EOAD) 患者的第一阶段试验中的临时安全性和药理动力学数据.

研究的目的:

  • 评估EOAD患者中单次上升剂量 (SAD) 和多次上升剂量 (MAD) 的安全性和耐受性.
  • 评估米特西兰对大脑脊髓液 (CSF) 溶性APPβ (sAPPβ) 水平的药动力学影响.
  • 为提供针对中枢神经系统的RNAi治疗阿尔茨海默病的初步临床数据.

主要方法:

  • 患有EOAD的患者随机分配到接受单次内耳注射剂量mietsiran (25-100mg) 或安慰剂.
  • 一小部分患者在洗后接受了开放式,多次上升剂量的 mivelsiran (50mg每6个月).
  • 通过不良事件 (AE) 的频率来评估安全性,通过CSFsAPPβ水平的变化来评估药理学.

主要成果:

  • 在SAD队列中,大多数AE是轻度/中度;在第1个月的CSFsAPPβ降低峰值为-84.5%,持续到第10个月 (-61.1%).
  • 在MAD队列中,没有报告严重或严重的副作用.
  • 在MAD的第一剂米特西兰后,CSFsAPPβ减少了-63.7%;第二剂导致进一步减少 (-83.8%).

结论:

  • 单剂和多剂量米特西兰在EOAD患者中通常耐受良好.
  • 米维尔西兰证明了强大的,持久的,与剂量相关的CSFsAPPβ的减少.
  • 结果支持进一步研究阿尔茨海默病和脑粉样血管病变的 mivelsiran.