EPIXCHANGE

Innovative gene therapies for epilepsy treatment

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 Obiettivo del progetto (Objective)

'A significant part of the costs of neurological diseases on society is associated with epilepsy. About 30-40% of the patients are refractory to pharmacological treatments, which are mostly symptomatic and often have side effects. In few cases surgical intervention is considered and no treatments interfering with or preventing the development of epilepsy are currently available. In this context, EPIXCHANGE aims at exploring, providing the basis for clinical application and implementing in the industrial arena new and unconventional strategies for the therapy of partial epilepsy advancing the state-of-the-art in the field. To achieve this, a strategic partnership will be created, including two internationally recognized academic institutions (UniFE, ULund) and one SME (NsGene) developing encapsulated cell biodelivery (ECB) based therapeutic products. ECBs will be transferred to the academic partners, while knowledge and technology on animal models, viral vectors and BDNF-producing cells will be transferred to the SME. This partnership will implement a joint research programme, which will enable to exploit the complementary competencies and technologies available at each participant site and will increase the knowledge-sharing and technology transfer, as well as the mutual understanding and penetration of the different cultural settings and skills required for both academic and industrial sectors, thus improving partners’ RTD capability and competitiveness. This will be combined with transfer of knowledge on complementary skills. Experienced researchers will be also recruited to bring top level experience in techniques that are not present in the consortium. Annual workshops will be organized to benefit the transfer of knowledge programme and the dissemination of results both within the consortium and towards the scientific community.'

Introduzione (Teaser)

Gene and cell therapy are emerging as novel alternatives to existing treatments. A European consortium is evaluating the implementation of such innovative approaches for the treatment of epilepsy.

Descrizione progetto (Article)

Epilepsy is a chronic neurological condition characterised by seizures. Depending on the affected area of the brain, epilepsy can be subcategorised into generalised or partial. Unfortunately, in almost 40% of the cases current medications fail to treat seizures, indicating a need for novel therapies.

The EU-funded 'Innovative gene therapies for epilepsy treatment' (http://www.epixchange.eu/ (EPIXCHANGE)) project proposes to address this issue by developing unconventional therapies for the treatment of epilepsy. These strategies entail either the administration of encapsulated cells or viral vectors for the delivery of therapeutic factors.

So far, scientists have generated cell lines that produce and release neurotransmitter molecules capable of stopping epileptic seizures. These cells have been encapsulated for delivery to the epileptic brain, and their efficacy is currently being investigated in experimental models. Viral vectors capable of modulating the local expression of various factors are also being evaluated. The outcome of these pre-clinical studies will form the basis for future clinical trials in humans.

During the first part of the project, two workshops were organised one on 'Animal model of epilepsy' and the other on 'EC project management'. Additional workshops took place in the second year of the project with focus on the safety of gene and cell therapy products and on gene transfer in the CNS. Distinguished speakers attended to present the latest advancements in gene and cell therapy for neurological diseases.

Dissemination of the project activities through the website, social media and local media coverage raised public awareness regarding the hot topic of gene therapy in CNS. Special oral- and lab-based presentations to local high-school students was a further step to communicate the importance of research to society.