Gene therapy to fight epilepsy

GO-Bio round 8 – Prof. Regine Heilbronn – Charité – Berlin University Hospital, Institute of Virology, Benjamin Franklin Campus


Beneficiary: Charité – Berlin University Hospital 

Project description

Epilepsy is one of the most common chronic disorders of the central nervous system. Epileptic seizures are a result of excessive electrical discharges in brain neurons. Neurologists speak of thunderstorms raging in the brain. Focal epilepsy is difficult to treat despite medical advances. Surgical removal of the affected brain area can help, but it does not guarantee a seizure-free life. A great many patients with epilepsy develop memory, learning and emotional disorders – which negatively impacts their quality of life and ability to work.

The GO-Bio team led by Berlin-based virologist Regine Heilbronn and neurobiologist Christoph Schwarzer has developed a highly innovative molecular approach to focal epilepsy treatment: it is based on a type of gene therapy whereby the genetic information for a special neuropeptide called dynorphin is introduced into the affected brain area. There is uptake of the genetic material through the neurons, which start to produce and store neuropeptides. The procedure is particularly unique in that the agents inhibiting a seizure are released into the system only if the nerve tissue is over-excited – such as when a seizure starts. The team led by Heilbronn calls this approach "drug on demand" gene therapy.

As transporter genes, the Berlin-based start-up team use adeno-associated viruses (AAV for short), which are reported to be extremely safe. The GO-Bio team now aims to develop gene therapy to treat patients with drug-resistant temporal lobe epilepsy (TLE) as part of a project entitled "EpiBlock". Researchers have already demonstrated in tests on mice that the substances were released in a manner that was, both spatially and temporarily, extremely limited. For a period of several months epileptic seizures in the animals were suppressed completely.

During the first phase of GO-Bio, the team from Charité in Berlin hopes to drive the pre-clinical development of its gene therapy, thereby achieving proof of concept. Furthermore, the transporter gene will be produced as a drug for use in clinical studies. After the company has been established, the second phase of GO-Bio will see clinical trials.