Novel CBD gelatin bead formulation reduced seizures by 82% in children with treatment-resistant epilepsy | Research

The takeaway

PTL-101, an oral CBD formulation designed for better absorption, reduced median seizure count by 82% in 16 children with treatment-resistant epilepsy, with 56% achieving 50%+ reduction.

Pediatric neurologists, epilepsy families, and pharmaceutical formulators.

82% seizure reduction

What the researchers found

Median seizure count reduced by 81.9% from baseline. Monthly seizure frequency decreased by 73.4% (p<0.05). 56% of patients were responders (50%+ reduction), and 2 patients became seizure-free. 73% of caregivers reported improved condition; 82% reported reduced seizure severity. Treatment adherence was 96.3%.

Why it matters

CBD bioavailability is a major challenge. This gelatin bead formulation (PTL-101) addresses that issue, and the 82% seizure reduction and high adherence (96%) suggest the formulation matters for clinical outcomes.

The numbers in context

16 enrolled, 11 completed; mean age 9.1 years; average dose 13.6 mg/kg/day; 81.9% median seizure reduction; 56% responders; 2 seizure-free; adherence 96.3%; AEs: insomnia 25%, somnolence 18.8%.

How the study worked

Phase II, open-label, single-center study. 16 pediatric patients with TRE (history of 4+ failed AEDs). 4-week baseline, 2-week titration (up to 25 mg/kg or 450 mg), 10-week maintenance. Average maintenance dose 13.6 mg/kg/day.

What this study cannot tell us

Open-label, no control group, single center, small sample (16). 5 patients dropped out. The 82% reduction may partly reflect regression to the mean or placebo effects without a control group.

How to read the evidence

Preliminary: small open-label phase II study without controls.

When this study was published

Published in 2019.

The bigger picture

Multiple CBD formulations are now being developed to improve on the original Epidiolex formulation. PTL-101's gelatin bead technology represents one approach, and its high response rate warrants larger controlled trials.

Questions still open

How does PTL-101 compare head-to-head with Epidiolex? Does the improved bioavailability allow lower effective doses?

Common questions

What makes PTL-101 different from other CBD products?

PTL-101 uses gelatin matrix beadlets designed to improve CBD absorption and maintain consistent transit through the digestive system, potentially improving bioavailability.

How effective was PTL-101 for seizures?

Median seizure count dropped 82%, 56% of children had at least 50% fewer seizures, and 2 patients became seizure-free during the 12-week study.

Read the original research

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

Epilepsy & behavior : E&B, 98(Pt A), 233-237

View on PubMed ↗Publisher’s paper ↗

Citation

Mitelpunkt, Alexis; Kramer, Uri; Hausman Kedem, Moran; Zilbershot Fink, Efrat; Orbach, Rotem; Chernuha, Veronika; Fattal-Valevski, Aviva; Deutsch, Lisa; Heffetz, Daphna; Sacks, Hagit. (2019). The safety, tolerability, and effectiveness of PTL-101, an oral cannabidiol formulation, in pediatric intractable epilepsy: A phase II, open-label, single-center study.. Epilepsy & behavior : E&B, 98(Pt A), 233-237. https://doi.org/10.1016/j.yebeh.2019.07.007