New Hope from Fraunhofer Research – a Potential Therapy for KCNQ2

The Fraunhofer Institute for Translational Medicine and Pharmacology (ITMP) has recently announced in a press release the identification of a promising compound targeting KCNQ channels. These potassium channels are crucial for neuronal excitability and are directly implicated in the seizures seen in children with KCNQ2 encephalopathy.

Why is this discovery important?

• Compound Identified: JNJ-37822681, originally developed as an antipsychotic, turned out to be a strong opener of KCNQ2 channels—echoing the mechanism of the withdrawn anti-epileptic drug retigabine.
• Preclinical Evidence: In patient-derived neurons and animal models, JNJ-37822681 significantly reduced seizure-like activity and restored normal electrical signaling.
• Safety & Development Potential: Although shelved by Johnson & Johnson years ago, the compound has already been tested in hundreds of patients for safety in other indications—yet these valuable clinical data remain inaccessible.

Gratitude and Call to Action

As KCNQ2 e.V. Germany, we are deeply grateful to the dedicated researchers at Fraunhofer ITMP for their relentless pursuit of hope and their focus on real solutions for our children.

The next critical step lies with Johnson & Johnson: releasing the existing clinical data. Only then can Fraunhofer accelerate the transition into clinical studies—potentially bringing a treatment to our children much sooner. Every delay is a setback for families living with this devastating disease.

We urge J&J: Act responsibly and release the data—our families cannot wait.

Sources:

• German: https://www.itmp.fraunhofer.de/de/presse/Epilepsie_KCNQ2.html
• English: https://www.itmp.fraunhofer.de/en/press/Epilepsie_KCNQ2.html

Disclaimer:

KCNQ2 e.V. shares information about current scientific developments. This does not constitute medical advice. Families should always consult their physicians for treatment decisions.