The safety, tolerability, and effectiveness of PTL-101, an oral cannabidiol formulation, in pediatric intractable epilepsy: A phase II, open-label, single-center study

Mitelpunkt, Alexis; Kramer, Uri; Kedem, Moran Hausman; Fink, Efrat Zilbershot; Orbach, Rotem; Chernuha, Veronika; Fattal‐Valevski, Aviva; Deutsch, Lisa; Heffetz, Daphna; Sacks, Hagit

doi:10.1016/j.yebeh.2019.07.007

Cited by 36 publications

(29 citation statements)

References 25 publications

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“…However, inter-individual variations were still high. This formulation has been proven safe and effective in a paediatric population with treatment-resistant epilepsy [ 1 ]. Another group recently reported a 4.4-fold increase in C max , enhanced bioavailability, and shorter T max with a SEDDS-CBD formulation based on VESIsorb ® technology compared to a control CBD oil [ 51 ].…”

Section: Novel Cbd Medical Products In Developmentmentioning

confidence: 99%

“…Cannabidiol (CBD) is a phytocannabinoid used globally for a variety of indications, but with few approved medicinal applications. Purified CBD is only licensed in treatment-resistant, rare paediatric forms of epilepsy [ 1 , 2 , 3 , 4 , 5 ]. Ongoing clinical trials are being conducted in potential indications such as anxiety, schizophrenia, addiction, post-traumatic stress disorder, graft-versus-host disease, cancer and inflammatory bowel disease.…”

Section: Introductionmentioning

confidence: 99%

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Towards Better Delivery of Cannabidiol (CBD)

Millar¹,

Maguire

Yates³

et al. 2020

Pharmaceuticals

153

118

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Cannabidiol (CBD) has substantial therapeutic potential, but its development as an effective drug by the pharmaceutical industry is hindered by intrinsic characteristics such as low bioavailability, low water solubility, and variable pharmacokinetic profiles. Importantly, lack of patentability of the drug substance also limits the likelihood of an expensive, full development programme in anything other than orphan indications. Potential avenues to overcome these issues with CBD include self-emulsifying drug delivery systems, improved crystal formulations and other solid-state delivery formulations, which are mostly in the pre-clinical or early clinical stages of development. This review identifies issues compromising current delivery of solid-state CBD, and how advanced pharmaceutical development strategies can enable CBD to realise the full potential as a successful therapeutic agent.

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Section: Novel Cbd Medical Products In Developmentmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Towards Better Delivery of Cannabidiol (CBD)

Millar¹,

Maguire

Yates³

et al. 2020

Pharmaceuticals

153

118

View full text Add to dashboard Cite

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“…Over the last six years, medical publications regarding epilepsy treatment with cannabis oil extracts and pure CBD can be divided into several groups: retrospective surveys and chart reviews of patients independently treated with artisanal cannabis by their caretakers which was reported to physicians; retro-spective chart reviews of CBD-enriched cannabis oil use, as directed by physicians; and open-label followed by placebo-controlled prospective studies of US Food and Drug Administration (FDA)-approved pure CBD oil (Epidiolex®); in addition to anecdotal reports of other "pure CBD" compounds used. 33 Porter et al surveyed parents who belong to a Facebook group that used CBD extracts to treat their children's seizures. Nineteen out of 150 participants in the group responded: 84% reported a reduction in seizure frequency; of these, 11% (2/16) experienced a positive effect and became seizure-free.…”

Section: Current Clinical Experiencementioning

confidence: 99%

Medical Cannabis for Intractable Epilepsy in Childhood: A Review

Ben‐Zeev

2020

Rambam Maimonides Med J

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In recent years, cannabis has been gaining increasing interest in both the medical research and clinical fields, with regard to its therapeutic effects in various disorders. One of the major fields of interest is its role as an anticonvulsant for refractory epilepsy, especially in the pediatric population. This paper presents and discusses the current accumulated knowledge regarding artisanal cannabis and Epidiolex®, a United States Food and Drug Administration (FDA)-approved pure cannabidiol (CBD), in epilepsy management in pediatrics, by reviewing the literature and raising debate regarding further research directions.

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“…11,19,20 Several open-label expanded access programs have been initiated to study this pharmaceutical formulation of CBD for the treatment of TRE in patients with other seizure etiologies, but to date, there are limited data on other epilepsy syndromes, including many of the severe genetic epilepsies. 9,[21][22][23][24][25][26] We evaluated the effectiveness of add-on treatment with a 24% CBD-based oil in patients with TRE.…”

Section: Introductionmentioning

confidence: 99%

An Open Retrospective Study of a Standardized Cannabidiol Based-Oil in Treatment-Resistant Epilepsy

Marchese

Vari

Balagura

et al. 2022

Cannabis and Cannabinoid Research

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Introduction: Cannabidiol (CBD) has antiseizure properties but no psychoactive effects. Randomized controlled trials of an oral, pharmaceutical formulation of highly purified CBD are promising; however, data regarding other formulations are sparse and anecdotal. We evaluated the effectiveness of add-on therapy with a standardized CBD-based oil in treatment-resistant epilepsy (TRE) patients. Materials and Methods: An open retrospective study was carried out on patients with refractory epilepsy of different etiology. We reviewed clinical data from medical charts and caregiver's information. Participants received add-on with 24% CBD-based oil, sublingually administered, at the starting dose of 5-10 mg/[kg$day] up to the maximum dose of 50 mg/[kg$day], based on clinical efficacy. Efficacy was evaluated based on patients being seizure free or experiencing at ‡ 50% improvement on seizure frequency. Tolerability and suspected adverse drug reaction data were also analyzed. Results: We included 37 patients (46% female) with a median age of 16.1 (range: 2-54) years. Twenty-two (60%) patients suffered from epileptic encephalopathy, 9 (24%) from focal epilepsy, and 6 (16%) from generalized epilepsy. Mean follow-up duration was 68 (range: 24-72) weeks. The average age at seizure onset was 3.8-2.1 years (range: 7 days-21 years). The median achieved CBD-based oil dose was 4.2-11.4 (range: 0.6-50) mg/[kg$day]. At 40-month follow-up, 7 (19%) patients were seizure free, 27 (73%) reported > 50% improvement, 2 (5%) patients reported < 50% improvement, and 1 patient discontinued therapy due to lack of efficacy. Weaning from concomitant antiepileptic drugs was obtained after 24 weeks from CBD introduction in 10 subjects. Mild and transitory adverse events, including somnolence or loss of appetite, occurred in nine (25%) patients. Discussion and Conclusion: We showed the efficacy of a CBD-based oil formulation with few significant side effects in patients with TRE of various etiologies.

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The safety, tolerability, and effectiveness of PTL-101, an oral cannabidiol formulation, in pediatric intractable epilepsy: A phase II, open-label, single-center study

Cited by 36 publications

References 25 publications

Towards Better Delivery of Cannabidiol (CBD)

Towards Better Delivery of Cannabidiol (CBD)

Medical Cannabis for Intractable Epilepsy in Childhood: A Review

An Open Retrospective Study of a Standardized Cannabidiol Based-Oil in Treatment-Resistant Epilepsy

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