Charité spin-off develops gene therapy against epilepsy | City of Health Berlin

Thursday, June 23, 2022 – Author: ham

Focal epilepsy is often a difficult drug to treat. The new gene therapy developed by the Charité spin-off gives hope. This approach has already been successful in animal experiments. Clinical trials will now follow.

Repeated seizures are extremely stressful. Patients must take medication all the time to prevent relapse. However, in focal epilepsy, where the origin of the seizure is concentrated in a certain area of ​​the brain, drugs often fail and have serious side effects. Some patients may also undergo surgery. But even epilepsy surgery does not guarantee permanent relief from seizures.

“Unfortunately, we can’t really help many people with disabilities,” says Professor Regine Heilbronn of Charité Clinic for Neurology and co-founder of EpiBlok GmbH, a spin-off of Charité and the Medical University of Innsbruck. “That’s why we’ve developed a conceptually new therapeutic approach.”

The missing neuropeptide is replaced

The new approach is based on the finding that the concentration of the neuropeptide dynorphin is often too low in focal epilepsy. A small protein ensures that groups of nerve cells cannot discharge at the same time, as in an epileptic seizure. In gene therapy, researchers introduce the dynorphin gene into affected nerve cells using a gene vector. Dynorphin gene vehicles are adeno-associated viral vectors (AAVs) that have already been clinically approved for gene therapy in some diseases.

In mice, the researchers were able to demonstrate that the animals produce and retain dynorphin peptide after treatment. After a single application, seizures were reliably suppressed for several months, the researchers said.

“Drug on demand” – Therapy

“In this case, it is a ‘drug on demand’ therapy: Nerve cells release the stored peptide only when it is needed,” explains prof. Christoph Schwarzer, neuropharmacologist at the University of Innsbruck and co-founder of EpiBlok. “It’s a case where nerve cells are constantly aroused, like at the beginning of an epileptic seizure. Dynorphin then suppresses the excitement and the storm recedes. “

A clinical study is being prepared

A team led by Regine Heilbronn and Christoph Schwarzer is now planning the first clinical trial. “With EpiBlok Therapeutics GmbH, we want to produce the AAV vector in larger quantities and in the required high quality so that we can start an initial clinical study,” says Heilbronn.

When EpiBlok GmbH was founded, the Regine Heilbronn team was supported by the SPARK-BIH program through funding, coaching and mentoring. Regine Heilbronn has already received € 3.3 million from the GO Bio program for preclinical gene therapy studies against focal epilepsy based on AAV. According to Charité, EpiBlok is their first by-product to seek access to gene therapy treatment.

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