SPARK project receives millions in funding for a new epilepsy therapy

Charité clinician Regine Heilbronn and her research team convince the Federal Ministry of Education and Research (BMBF) at the GO-Bio Competition, daring to take the leap from the laboratory to medical application. The scientists were chosen for their advanced epilepsy therapy on April 18, 2018 at the “German Biotechnology Days 2018.”

 

Professor Regine Heilbronn is one of the winners of this year’s selection round of the BMBF GO-Bio funding initiative, which supports start-up researchers with cutting-edge ideas from the life sciences during the early project phases. The team led by Regine Heilbronn, Director of the Institute of Virology at Charité – Universitätsmedizin Berlin, has developed an advanced therapy for focal epilepsy together with Prof. Christoph Schwarzer of the Medical University of Innsbruck and is receiving more than three million euro for this. The therapy is a “Drug on Demand” gene therapy based on adeno-associated viral (AAV) vectors that are injected once into the affected brain area so that they induce the production and storage of seizure-inhibiting neuropeptides in the focus. When the transduced neurons are exposed to high-frequency excitation, as is the case before the onset of seizures, neuropeptides are released so that the development of epileptic seizures is completely suppressed for a period of many months. Unlike conventional drugs, the release is strictly focal and limited over time. With this new therapy concept, seizures can be prevented over the long term, side effects can be minimized, and losses in learning and memory can be prevented. Prof. Axel Radlach Pries, Dean of Charité, is thrilled about receiving the funding: “The gene vector-based therapy project planned by Professor Heilbronn has the potential to open a new chapter in the treatment of epilepsy because of the long-term, stimulation-controlled drug release that can be achieved.”

Ready for the start-up and vector production

In 2017 SPARK Berlin accompanied the research of Regine Heilbronn and her team. SPARK Berlin is part of Berlin Health Innovations, the joint technology transfer unit of BIH and Charité, which Stiftung Charité and BIH jointly fund. Here, participants receive mentoring, coaching and financial support to accelerate the translation of their discoveries into clinically relevant drugs, diagnostics and therapies.

“I am delighted by the fruits of the efforts of BIH to further develop the culture of innovation in all areas, from digital to pharmaceutical. The additional GO-Bio funding will now enable the team led by Professor Heilbronn to quickly bring their innovation to the patient,” says Dr. Rolf Zettl, CFO of BIH and the Board Member responsible for Berlin Health Innovations. At the end of the three-year GO-Bio funding, the finished product should already be available for clinical testing. The innovative “Drug on Demand” concept also opens up new therapeutic perspectives for other chronic diseases that have been difficult to treat up to now.

Focal epilepsies that are difficult to treat

The potential for the new therapy is high. “Epilepsy is one of the most common chronic diseases of the central nervous system, affecting more than five million people in the EU alone,” Professor Regine Heilbronn says. The most common form of focal epilepsy is temporal lobe epilepsy, from which approximately 600,000 patients suffer chronically in Europe alone. This form of focal epilepsy is very hard, or even impossible to treat with drugs alone. Patients often develop memory, learning and emotional control disorders that significantly affect their quality of life and societal participation. Depending on the accessibility of the epileptic focus, a neurosurgical removal of the affected tissue is possible, but many patients shun from open-brain surgery, even more since lasting seizure-freedom cannot be garanteed.

“We have developed truly innovative data and a unique therapeutic concept, but we have also benefited greatly from the support of Stiftung Charité and BIH, as well as the participation in the SPARK program, from the mentoring, pitch training and intensive coaching in business matters,” Regine Heilbronn says. “Since its founding, Stiftung Charité has been committed to a culture of innovation in which research and translation to the clinical go hand in hand. The impulses in technology transfer that we have provided in recent years are now having more and more of an impact. This is also impressively demonstrated by the accolades that Professor Heilbronn has achieved,” says Professor E. Jürgen Zöllner, Chairman of Stiftung Charité.

 

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