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Bringing balance to the force–regulatable gene therapy for epilepsy

Gene Therapy volume 26, pages 347–349 (2019)Cite this article

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Epilepsy is a chronic disorder of the brain affecting 1% of the population, making it one of the most common neurological conditions [1]. It is characterised by recurrent seizures that may be partial or generalised to the entire brain and sometimes accompanied by loss of consciousness forming a severe handicap to a healthy lifestyle. It has no cure and an estimated 30% of patients do not respond to antiepileptic drugs, the benefit of which is often mitigated by multiple side effects and reoccurring seizures despite treatment [2, 3]. In pharmaco-resistant epilepsies, surgical resection is sometimes the only effective treatment, and although it has a high chance of success, the problem is that it might also affect eloquent cortex surrounding the epileptic focus [4].

On the path to finding new cures, gene therapy using viral vectors to deliver therapeutic genetic material to the epileptic focus has been of interest [5, 6]. Gene delivery to the brain is challenging given the physical barriers in the form of the skull and the selective blood–brain barrier. This is further confounded by the intricate anatomy and size of arguably the most complex organ of the body. However, over the past decade steady progress has been made through improvements in vector design, discovery of vectors that cross the blood–brain barrier and access to advanced surgical techniques. Given the vast number of neurological conditions potentially amenable to gene therapy, these are welcome advancements. Life-saving, life-changing or quality of life enhancing gene therapy clinical trials for such conditions [7,8,9,10] have further fuelled the appetite to develop this treatment modality.

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Funding

AJR receives funding from the UCL Therapeutic Acceleration Support Fund. AAR receives funding from the UK Medical Research Council (MR/R025134/1, MR/R015325/1, MR/M00676X/1 and MR/N026101/1), Action Medical Research (GN2485), the European Union’s Horizon 2020 research and innovation programme (Grant No 666918), Asociación Niemann Pick de Fuenlabrada and Apollo Therapeutics.

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  1. UCL School of Pharmacy, University College London, 29-39 Brunswick Square, London, WC1N 1AX, UK

    Arnaud J. Ruiz & Ahad A. Rahim

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  1. Arnaud J. Ruiz
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  2. Ahad A. Rahim
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Correspondence to Ahad A. Rahim.

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Ruiz, A.J., Rahim, A.A. Bringing balance to the force–regulatable gene therapy for epilepsy. Gene Ther 26, 347–349 (2019). https://doi.org/10.1038/s41434-019-0083-6

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