2024 April | Samuel W Terman, MD MS, Laura Kirkpatrick, MD, Lisa F Akiyama, MD, Wadih Baajour, MD, Deniz Atilgan, MD, Maria Kristina C Dorotan, MD, Hyoung Won Choi, MD, Jacqueline A French, MD
The article reviews the current state of the epilepsy drug and device pipeline, highlighting major innovations in diagnostic and therapeutic products presented at the 17th Epilepsy Therapies and Diagnostics Development conference. It discusses various therapeutic approaches targeting genetics, ion channels, neurotransmitters, and other potential first-in-class interventions such as stem cells, glycogen metabolism, cholesterol, the gut microbiome, and novel electrical neuromodulation methods. Therapeutics under development include drugs targeting refractory seizures and promoting precision medicine through disease-modifying approaches. Devices for seizure treatment and monitoring are also reviewed.
Key drug developments include XEN1101, a next-generation potassium channel opener showing promising efficacy in clinical trials, and BHV-7000, a compound with potential for treating refractory seizures. Other drugs include OV329, a GABA aminotransferase inhibitor for rare epilepsies, and ganaxolone, a positive allosteric modulator of GABA_A receptors approved for CDKL5 deficiency disorder. Newer drugs such as brivaracetam, radiprodil, and basimglurant are also under investigation for various epilepsy types.
Genetic therapies include STK-001, an ASO for Dravet syndrome, and AMT-260, an AAV-based gene therapy for refractory temporal lobe epilepsy. CAP-002, an AAV vector-based product for STXBP1 mutations, is also under development. Other innovations include NMT.001, an RNA-based therapy targeting miR-134, and VAL-1221, an antibody-enzyme fusion for glycogen metabolism disorders.
Neurostimulation devices such as Vagus Nerve Stimulation (VNS), Responsive Neurostimulation (RNS), and Deep Brain Stimulation (DBS) are discussed as palliative options for drug-resistant epilepsy. New devices such as Starstim and EASEE are also under development.
Seizure detection devices and electronic seizure diaries are reviewed for their role in improving seizure monitoring and clinical management. The National Institute of Neurological Disorders and Stroke (NINDS) is highlighted for its role in promoting device development through funding mechanisms.
The article concludes that despite the increase in available antiseizure medications, there remains a need for further innovation in developing more effective, better-tolerated treatments for epilepsy.The article reviews the current state of the epilepsy drug and device pipeline, highlighting major innovations in diagnostic and therapeutic products presented at the 17th Epilepsy Therapies and Diagnostics Development conference. It discusses various therapeutic approaches targeting genetics, ion channels, neurotransmitters, and other potential first-in-class interventions such as stem cells, glycogen metabolism, cholesterol, the gut microbiome, and novel electrical neuromodulation methods. Therapeutics under development include drugs targeting refractory seizures and promoting precision medicine through disease-modifying approaches. Devices for seizure treatment and monitoring are also reviewed.
Key drug developments include XEN1101, a next-generation potassium channel opener showing promising efficacy in clinical trials, and BHV-7000, a compound with potential for treating refractory seizures. Other drugs include OV329, a GABA aminotransferase inhibitor for rare epilepsies, and ganaxolone, a positive allosteric modulator of GABA_A receptors approved for CDKL5 deficiency disorder. Newer drugs such as brivaracetam, radiprodil, and basimglurant are also under investigation for various epilepsy types.
Genetic therapies include STK-001, an ASO for Dravet syndrome, and AMT-260, an AAV-based gene therapy for refractory temporal lobe epilepsy. CAP-002, an AAV vector-based product for STXBP1 mutations, is also under development. Other innovations include NMT.001, an RNA-based therapy targeting miR-134, and VAL-1221, an antibody-enzyme fusion for glycogen metabolism disorders.
Neurostimulation devices such as Vagus Nerve Stimulation (VNS), Responsive Neurostimulation (RNS), and Deep Brain Stimulation (DBS) are discussed as palliative options for drug-resistant epilepsy. New devices such as Starstim and EASEE are also under development.
Seizure detection devices and electronic seizure diaries are reviewed for their role in improving seizure monitoring and clinical management. The National Institute of Neurological Disorders and Stroke (NINDS) is highlighted for its role in promoting device development through funding mechanisms.
The article concludes that despite the increase in available antiseizure medications, there remains a need for further innovation in developing more effective, better-tolerated treatments for epilepsy.