Escuela de Tecnología Médica, Facultad de Salud, Universidad Santo Tomás, Santiago 8370003, Chile.
Molecular & Clinical Pharmacology, ICBM, Faculty of Medicine, University of Chile, Santiago 8330111, Chile.
Biomolecules. 2024 Jun 8;14(6):673. doi: 10.3390/biom14060673.
One of the biggest problems in the treatment of idiopathic Parkinson's disease is the lack of new drugs that slow its progression. L-Dopa remains the star drug in the treatment of this disease, although it induces severe side effects. The failure of clinical studies with new drugs depends on the use of preclinical models based on neurotoxins that do not represent what happens in the disease since they induce rapid and expansive neurodegeneration. We have recently proposed a single-neuron degeneration model for idiopathic Parkinson's disease that requires years to accumulate enough lost neurons for the onset of motor symptoms. This single-neuron degeneration model is based on the excessive formation of aminochrome during neuromelanin synthesis that surpass the neuroprotective action of the enzymes DT-diaphorase and glutathione transferase M2-2, which prevent the neurotoxic effects of aminochrome. Although the neurotoxic effects of aminochrome do not have an expansive effect, a stereotaxic injection of this endogenous neurotoxin cannot be used to generate a preclinical model in an animal. Therefore, the aim of this review is to evaluate the strategies for pharmacologically increasing the expression of DT diaphorase and GSTM2-2 and molecules that induce the expression of vesicular monoamine transporter 2, such as pramipexole.
特发性帕金森病治疗中的最大问题之一是缺乏能够减缓其进展的新药。左旋多巴仍然是治疗这种疾病的明星药物,尽管它会引起严重的副作用。新药临床试验的失败取决于基于神经毒素的临床前模型的使用,而这些模型不能代表疾病中的情况,因为它们会诱导快速和广泛的神经退行性变。我们最近提出了一种特发性帕金森病的单神经元退化模型,该模型需要多年时间才能积累足够数量的神经元丢失,从而引发运动症状。这种单神经元退化模型基于神经黑色素合成过程中氨基酸过度形成,超过了 DT-黄递酶和谷胱甘肽 S-转移酶 M2-2 的神经保护作用,这些酶可以防止氨基酸的神经毒性作用。尽管氨基酸的神经毒性作用没有扩张作用,但这种内源性神经毒素的立体定向注射不能用于在动物中产生临床前模型。因此,本综述的目的是评估增加 DT 黄递酶和 GSTM2-2 表达的药理学策略,以及诱导囊泡单胺转运体 2 表达的分子,如普拉克索。