Figure 1
Translational roadmap for the use of human brain tissue as a step between animal research and clinical trials. Schematic illustration of how human-tissue resected from patients with epilepsy can be used to validate the effect of treatments found in animal models before proceeding to clinical trials for drug-resistant epilepsy, exemplified by NPY. (1) Basic research, with in vitro models, from several labs has shown that NPY has an inhibitory effect on epileptiform activity. (2) Chronic epilepsy models in vivo, such as the intrahippocampal kainate model, has provided key evidence demonstrating that NPY has an anti-epileptic effect in the chronic epileptic brain over a longer period of time. (3) The present study is a crucial validation step showing that NPY has an inhibitory effect on epileptiform activity in the target tissue, drug-resistant human hippocampal slices, minimising the risk for the next step of first-in-man clinical phase 1–2 study (step 4). The step 2 is interchangeable with step 3.
