This month research scientists from NeuroCycle Therapeutics, Inc. (NCT) will begin research on a potential next generation treatment of Dravet Syndrome in labs located within the Milwaukee Institute for Drug Discovery, which is directed by Dr. Stafford. Dravet Syndrome (DS) is one of the most pharmacoresistant epilepsy syndromes with an estimated incidence of 1 per 15,700 live births in the US and causes frequent, severe seizures. Patients with DS typically have initial seizure onset at 6–9 months of age, often associated with fever. Comorbidities include sleep disturbance; autistic features; and cognitive, memory, and motor impairments. In >80% of patients, DS results from a spontaneous loss-of-function mutation in the Scn1a gene encoding brain voltage-gated sodium channel type-I (NaV1.1).
In the work at UWM, Dr. Aric Madayag and Dr. Eva Fekete will test subtype-selective GABAA receptor modulators, including one compound discovered right here in the labs of Prof. Cook, in models of DS using juvenile SCN1A heterozygous knockout mice. Prof. Arnold (UWM) and Prof. Kearney (Northwestern University) serve as consultants for these studies. These mice recapitulate the key symptoms of Dravet syndrome and provide data considered to be highly translatable to human disease.
About Neurocycle Therapeutics
NeuroCycle Therapeutics, Inc. is a privately held pharmaceutical company unlocking the potential of small-molecule CNS therapeutics to transform patient’s lives. The company is currently focused on building a strong pipeline of compounds that selectively target specific GABAA receptor subtypes to treat diseases related to central sensitization including chronic itch and chronic pain, and rare epilepsy syndromes such as Dravet Syndrome. NCT’s lead program, NCT10004, is currently in IND-enabling studies and is expected to begin Phase 1 trials in healthy volunteers in early 2020.
For more information, please visit www.neurocycletherapeutics.com.