Diazepam nasal spray effective in Lennox-Gastaut syndrome
A new analysis of data from a phase 3 clinical trial suggests that an inhaled diazepam nasal spray (Valtoco, Neurelis) works about as well among patients with Lennox-Gastaut syndrome (LGS) as it does with other patients with pediatric encephalopathies.
LGS is a severe form of epilepsy that generally begins in early childhood and has a poor prognosis and seizures that are often treatment refractory. The findings of the analysis should be encouraging to physicians who may view patients with LGS as not benefiting from treatment, said Daniel C. Tarquinio, DO, who presented the results at the 2022 annual meeting of the Child Neurology Society.
Novel cannabis oil curbs tics in severe Tourette syndrome
An oral oil containing tetrahydrocannabinol (THC) and cannabidiol (CBD) led to a significant and meaningful reduction in motor and vocal tics in patients with severe Tourette syndrome, results of a double-blind, placebo-controlled, crossover study showed.
“In a methodologically robust manner (and independent of any drug company sponsorship), we provide evidence for the effectiveness of repeated dosing with THC:CBD versus placebo in tic
suppression, as well as reduction of comorbid anxiety and obsessive-compulsive disorder in severe Tourette syndrome,” neuropsychiatrist and lead investigator Philip Mosley, PhD, said in an interview.
The results offer support to people with Tourette syndrome who “want to approach their doctor to try medicinal cannabis when other drugs have not worked or are intolerable,” said
Dr. Mosley, of the Wesley Research Institute and QIMR Berghofer Medical Research Institute, Herston, Australia.
The study was published online in NEJM Evidence (2023 Jun 7. doi: 10.1056/EVIDoa2300012).
Twenty-two adults (mean age, 31 years) with severe Tourette syndrome received THC:CBD oil titrated upward over 6 weeks to a daily dose of 20 mg of THC and 20 mg of CBD, followed by a 6-week course of placebo (or vice versa). Six participants had not previously used cannabis.
The primary outcome was the total tic score on the Yale Global Tic Severity Scale (YGTSS; range, 0-50 with higher scores = greater tic severity).
The mean baseline YGTSS total tic score was 35.7. At 6 weeks, the reduction in total tic score was 8.9 with THC:CBD versus 2.5 with placebo.
A linear mixed-effects model (intention to treat) showed a significant interaction of treatment and visit number (P = .008), indicating a greater decrease (improvement) in tic score over time with THC:CBD, the study team reported.
On average, the magnitude of the tic reduction was “moderate” and comparable with the effect observed with existing treatments such as antipsychotic agents, the investigators noted.
The study was funded by the Wesley Medical Research Institute, Brisbane, and the Lambert Initiative for Cannabinoid Therapeutics, a philanthropically funded research organization at the University of Sydney, Australia.
Dr. Mosley reported no relevant financial relationships.