Orphan Study

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Title	Article Date	Tags	Notes
UF researchers granted $1 million for epilepsy study	11/05/2015	Epidiolex, Orphan Study, Florida	The project is intended to treat 50 children from ages 2 to 16 living with epilepsy that have exhausted most of their treatment options, Carney said. Every step of the three-year study will be meticulously tracked. Patients will check-in at regular intervals and the researchers will examine how the children are responding, Carney said. All subjects will be clinically evaluated and seizure frequency will be recorded in a diary given to patients, according to the ClinicalTrials.gov website. “Through this research, we want to learn about what type of children respond, if they can tolerate the medication and if there are any side effects we need to worry about,” Carney said. The study will begin later this summer and participants are still being accepted, Carney said.The project is intended to treat 50 children from ages 2 to 16 living with epilepsy that have exhausted most of their treatment options, Carney said. Every step of the three-year study will be meticulously tracked. Patients will check-in at regular intervals and the researchers will examine how the children are responding, Carney said. All subjects will be clinically evaluated and seizure frequency will be recorded in a diary given to patients, according to the ClinicalTrials.gov website. “Through this research, we want to learn about what type of children respond, if they can tolerate the medication and if there are any side effects we need to worry about,” Carney said. The study will begin later this summer and participants are still being accepted, Carney said.
More Positive Results With Cannabidiol in Epilepsy	11/03/2015	Epidiolex, Studies, Orphan Study, gw pharm	More Positive Results With Cannabidiol in Epilepsy (You will need to google this title in order to get the article to avoid creating an account) They found a reduction of the total number of seizures by a median of 38% at 3 months and 31% at 6 months. These patients started on a cannabidiol (CBD) dose of 2 mg/kg per day, which was increased to a maximum tolerated dose or to 25 mg/kg per day. Of the 25 patients enrolled in the study, two discontinued treatment before 6 months. One boy stopped medication after a few months because of intolerable diarrhea, and another patient discontinued the drug because of persistent nausea/vomiting. One patient didn't report 6-month data at the time of the analysis. More than three quarters (77%) of the 22 remaining patients experienced fewer seizures after 6 months. However, he stressed that the study was uncontrolled, symptoms were self-reported by families, and there has been a lot of news coverage of medical marijuana for the treatment of epilepsy. "Many parents had been avidly desirous of their child going on this cannabidiol because of what they had read in the lay press, so they were primed to believe this cannabidiol might be beneficial." On the other hand, he said, it's important to keep in mind that these children have "horrible epilepsy" and most have been on more than 10 medications for seizure without success. "Any improvement to that degree in this population that is sustained is a pretty dramatic response."
Orphan drug designation spurs trials for cannabidiol in Dravet syndrome	03/21/2014	Charlotte's Web, Dravet syndrome, Orphan Study	In a survey and other anecdotal reports, parents of children with drug-refractory epilepsy syndromes have reported successful treatment with cannabidiol-enriched marijuana. But little high-quality evidence is available about the efficacy and safety of cannabidiol in patients with Dravet, Lennox-Gastaux, and other pediatric epilepsy syndromes, as shown by a recent Cochrane Collaboration review of four randomized trials totaling 48 patients with epilepsy that found no adverse effects with 200-300 mg daily over short periods but did not report on the efficacy of the drug.