School of Biomedical Sciences, The University of Queensland, St Lucia, Queensland, Australia.
J Neurochem. 2024 Dec;168(12):3911-3935. doi: 10.1111/jnc.15938. Epub 2023 Aug 18.
Nowadays, particularly in countries with high incomes, individual mutations in people affected by genetic epilepsies are identified, and genetic therapies are being developed. In addition, drugs are being screened to directly target specific mutations, and personalised medicine is possible. However, people with epilepsy do not yet benefit from these advances, and many types of epilepsies are medication-resistant, including Dravet syndrome. Thus, in the meantime, alternative and effective treatment options are needed. There is increasing evidence that metabolic deficits contribute to epileptic seizures and that such metabolic impairments may be amenable to treatment, with metabolic treatment options like the ketogenic diet being employed with some success. However, the brain metabolic alterations that occur in ion channel epilepsies are not well-understood, nor how these may differ from epilepsies that are of acquired and unknown origins. Here, we provide an overview of studies investigating metabolic alterations in epilepsies caused by mutations in the SCN1A and KCNA1 genes, which are currently the most studied ion channel epilepsies in animal models. The metabolic changes found in these models are likely to contribute to seizures. A metabolic basis of these ion channel epilepsies is supported by human and/or animal studies that show beneficial effects of the ketogenic diet, which may be mediated by the provision of auxiliary brain fuel in the form of ketone bodies. Other potentially more preferred dietary therapies including medium-chain triglycerides and triheptanoin have also been tested in a limited number of studies, but their efficacies remain to be clearly established. The extent to which brain metabolism is affected in people with Dravet syndrome, KCNA1 epilepsy and the models thereof still requires clarification. This requires more experiments that yield functional insight into metabolism.
如今,特别是在高收入国家,人们已经发现了受遗传癫痫影响的个体突变,并正在开发遗传疗法。此外,还在筛选针对特定突变的药物,并且可以进行个性化治疗。但是,癫痫患者尚未从中受益,许多类型的癫痫是药物难治性的,包括 Dravet 综合征。因此,在这期间,需要替代的有效治疗选择。越来越多的证据表明代谢缺陷会导致癫痫发作,并且这些代谢损伤可能是可以治疗的,例如生酮饮食等代谢治疗方法已经取得了一些成功。但是,离子通道性癫痫中发生的大脑代谢改变尚未得到很好的理解,也不知道它们与后天的、未知原因的癫痫有何不同。在这里,我们提供了对 SCN1A 和 KCNA1 基因突变引起的癫痫症中代谢改变的研究综述,这两种基因突变是目前在动物模型中研究最多的离子通道性癫痫症。这些模型中发现的代谢变化可能会导致癫痫发作。人类和/或动物研究表明生酮饮食具有有益作用,为酮体形式的大脑提供辅助燃料,从而支持这些离子通道性癫痫具有代谢基础。其他潜在的更优选的饮食疗法,包括中链甘油三酯和三庚酸,在有限数量的研究中也进行了测试,但它们的疗效仍有待明确。 Dravet 综合征、KCNA1 癫痫及其模型中的大脑代谢受影响的程度仍需阐明。这需要进行更多的实验,以深入了解代谢的功能。