Investigational Epilepsy Therapy for Children Shows Targeted Potential in Early Testing

Investigational Epilepsy Therapy for Children Shows Targeted Potential in Early Testing

Preclinical studies on The Cell Factory’s lead investigational biologic drug (CF-MEV-117) for the treatment of drug-resistant epilepsy in children has been shown to have dose-dependent anti-inflammatory and immunosuppressive activity. These results suggest a specific and targeted effect, an achievement in the drug development program.

The full study results will be presented at the International Society for Extracellular Vesicles (ISEV) meeting on May 18–21 in Toronto, Canada.

The Cell Factory, a subsidiary of Esperite Group, has been developing the extracellular vesicles (EVs) drug candidate CF-MEV-117 in collaboration with teams in pediatric regenerative medicine, neurology, gastroenterology, immunology and EVs science at Bambino Gesù Children’s Hospital in Rome, Mario Negri Institute for Pharmacological Research in Milan, and the Women’s and Children’s Health Department at University of Padua. The consortium expects to demonstrate that CF-MEV-117 can have a much broader therapeutic effect in brain regeneration and normalizing neuron function.

Several studies have showed that brain inflammation can be a key factor in the development of severe epileptic seizures, where pro-inflammatory molecules secreted by brain cells may be responsible for a status epilepticus (an epileptic seizure of five minutes or more, or more than one seizure within a five-minute period without the patient returning to normal between them). A treatment that could control immune responses and inflammatory processes in the brain could be the way to reduce or eliminate symptoms of epilepsy, or even prevent a relapse.

EVs are very small natural vesicles that are produced by cells and contain proteins, growth factors, messenger RNA and other molecules that can have therapeutic effects. Because they are very small and stable, they can pass through the blood-brain barrier and reach areas that are affected by epilepsy.

“The Cell Factory is focused on development, clinical translation and commercialization of the advanced extracellular vesicles (EVs) biologic drugs and autologous stem cell therapies. The Cell Factory goal is to master the development and production of extracellular vesicles drugs in treatment of different indications,” Frederic Amar, CEO of the Cell Factory, said in a press release.

In vitro studies showed that CF-MEV-117 can modulate human immune cell response. It was found to increase the numbers of a specific subset of T-cells, known as regulatory T-cells, essential for the suppression and control of immune responses, while reducing the numbers of B-cells and inhibiting B-cell differentiation. Importantly, the drug response was dependent on the dose used.

Results confirm the anti-inflammatory and immunomodulatory capacity of this new investigational drug with clinical potential. The next step is to confirm the CF-MEV-117 activity in animal models before initiating clinical studies.

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