Incorporating upper motor neuron health in ALS drug discovery.

Incorporating upper motor neuron health in ALS drug discovery.
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DOI:
10.1016/j.drudis.2018.01.027
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发表时间:
2018-03
影响因子:
7.4
通讯作者:
Ozdinler PH
Ozdinler PH
中科院分区:
医学2区
文献类型:
--
作者:
Dervishi I;Ozdinler PH

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肌萎缩侧索硬化症 (ALS) 是一种复杂的疾病,影响运动神经元回路。在过去20年的临床试验连续失败后,依达拉奉最近被批准为第二个治疗ALS的药物。这引起了该领域的兴奋,并揭示了改进临床前检测以持续取得成功的必要性。在这里,我们重点关注上运动神经元 (UMN) 病理学在 ALS 中的重要性和相关性,并讨论将 UMN 存活纳入临床前检测将如何改善临床试验的纳入标准,并加快 ALS 和相关运动神经元疾病的药物发现工作。
Amyotrophic lateral sclerosis (ALS) is a complex disease, affecting the motor neuron circuitry. After consecutive failures in clinical trials for the past 20 years, edaravone was recently approved as the second drug for ALS. This generated excitement in the field and revealed the need to improve preclinical assays for continued success. Here, we focus on the importance and relevance of upper motor neuron (UMN) pathology in ALS, and discuss how incorporation of UMN survival in preclinical assays will improve inclusion criteria for clinical trials and expedite the drug discovery effort in ALS and related motor neuron diseases.
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