Bartus Raymond T, Herzog Christopher D, Bishop Kathie, Ostrove Jeffrey M, Tuszynski Mark, Kordower Jeffrey H, Gasmi Mehdi
Ceregene, Inc., San Diego, CA 92121, USA.
Parkinsonism Relat Disord. 2007;13 Suppl 3:S469-77. doi: 10.1016/S1353-8020(08)70052-X.
To develop CERE-120 (AAV-NTN) as a novel therapy for Parkinson's disease (PD) that might restore function of degenerating dopamine neurons and prevent further degeneration.
A nonclinical program demonstrated that NTN expression can be predictably controlled following CERE-120 administration, provides clear evidence of efficacy in numerous animal models and is safe at dose multiples that far exceed those required for efficacy. Preliminary, open label evidence in PD subjects offers corroborative support for these observations.
CERE-120 may represent an important, novel therapy for PD, though the clinical data require confirmation with additional clinical tests, including an ongoing multi-center, double-blinded controlled trial.
开发CERE-120（腺相关病毒-神经营养素-4，AAV-NTN）作为帕金森病（PD）的一种新型疗法，该疗法可能恢复退化的多巴胺能神经元的功能并防止进一步退化。
一项非临床研究项目表明，给予CERE-120后，神经营养素-4（NTN）的表达可得到可预测的控制，在众多动物模型中提供了明确的疗效证据，并且在远远超过疗效所需剂量倍数的情况下是安全的。帕金森病受试者的初步开放标签证据为这些观察结果提供了佐证支持。
CERE-120可能代表一种重要的帕金森病新型疗法，尽管临床数据需要通过额外的临床试验来确认，包括正在进行的多中心双盲对照试验。
