IRCCS Foundation "Carlo Besta" Neurological Institute, Milan, Italy.
IRCCS Istituto di Ricerche Farmacologiche "Mario Negri", Milan, Italy.
J Alzheimers Dis. 2018;62(3):1247-1259. doi: 10.3233/JAD-170770.
Translational neuroscience integrates the knowledge derived by basic neuroscience with the development of new diagnostic and therapeutic tools that may be applied to clinical practice in neurological diseases. This information can be used to improve clinical trial designs and outcomes that will accelerate drug development, and to discover novel biomarkers which can be efficiently employed to early recognize neurological disorders and provide information regarding the effects of drugs on the underlying disease biology. Alzheimer's disease (AD) and prion disease are two classes of neurodegenerative disorders characterized by incomplete knowledge of the molecular mechanisms underlying their occurrence and the lack of valid biomarkers and effective treatments. For these reasons, the design of therapies that prevent or delay the onset, slow the progression, or improve the symptoms associated to these disorders is urgently needed. During the last few decades, translational research provided a framework for advancing development of new diagnostic devices and promising disease-modifying therapies for patients with prion encephalopathies and AD. In this review, we provide present evidence of how supportive can be the translational approach to the study of dementias and show some results of our preclinical studies which have been translated to the clinical application following the 'bed-to-bench-and-back' research model.
转化神经科学将基础神经科学的知识与新的诊断和治疗工具的发展相结合，这些工具可能应用于神经疾病的临床实践。这些信息可以用于改进临床试验设计和结果，从而加速药物开发，并发现新的生物标志物，以便有效地早期识别神经障碍，并提供有关药物对潜在疾病生物学影响的信息。阿尔茨海默病（AD）和朊病毒病是两类神经退行性疾病，其发病的分子机制知之甚少，缺乏有效的生物标志物和治疗方法。因此，迫切需要设计预防或延迟发病、减缓进展或改善与这些疾病相关症状的疗法。在过去几十年中，转化研究为开发新的诊断设备和有希望的疾病修饰疗法提供了一个框架，用于治疗朊病毒脑病和 AD 患者。在这篇综述中，我们提供了转化方法对痴呆研究的支持证据，并展示了我们的一些临床前研究结果，这些研究结果遵循“从病床到治疗台再回到病床”的研究模式，已经转化为临床应用。
