Conclusion
In summary, many of the most promising preclinical data on novel approaches to improve antiepileptic drug delivery are aimed at either disrupting or bypassing the BBB, including Trojan horse technology that would allow targeting of large (and water insoluble) molecules through the BBB to epileptic brain regions responsible for seizure generation. The most important aspect is that such novel delivery approaches will allow clinical testing for antiepileptic activity of large molecules that were previously barred from development of epilepsy drugs. There may be a whole new world of effective epilepsy drugs that were excluded from experimental, and clinical evaluation simply because they could not cross the BBB. Researchers and practitioners in the field eagerly await the results—with cautious optimism and with high hopes.
Ultimately, our best chance for effectively treating and preventing epilepsy will involve therapies that, on the one hand, safely and specifically regulate seizure generation and, on the other hand, enhance the safety and tolerability by avoiding systemic toxicity. Once clinical trials of new delivery techniques have been conducted, the hope is that we will soon have novel epilepsy therapeutics that successfully and safely stop seizures in more patients than is possible today.
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Schmidt, D., Holmes, G.L. Commentary: Novel delivery approaches for antiepileptic drugs: Hope and hurdles. Neurotherapeutics 6, 381–382 (2009). https://doi.org/10.1016/j.nurt.2009.01.008
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DOI: https://doi.org/10.1016/j.nurt.2009.01.008