CBD & THC: A Promising Epilepsy Treatment Backed by Science

Understanding CBD and THC in Epilepsy

Cannabis contains over 100 cannabinoids, but two are most prominent: tetrahydrocannabinol (THC) and cannabidiol (CBD). THC is the psychoactive compound that produces a “high,” while CBD is non-intoxicating and increasingly recognized for its therapeutic benefits.

In epilepsy treatment, CBD is the star. It interacts with brain receptors and modulates neural excitability without inducing the mind-altering effects of THC. However, low amounts of THC may enhance CBD’s effectiveness through what’s known as the “entourage effect,” where cannabinoids work synergistically to amplify benefits.

The Science: Clinical Trials and Breakthroughs

CBD’s reputation as an antiepileptic emerged from anecdotal reports and early observational studies. The turning point came with randomized controlled trials (RCTs)—the gold standard in medical research.

Epidiolex: A Landmark Approval

In 2018, the U.S. FDA approved Epidiolex, a purified CBD extract developed by GW Pharmaceuticals (now part of Jazz Pharmaceuticals), for treating two rare, severe forms of epilepsy: Dravet syndrome and Lennox-Gastaut syndrome (LGS). Later, it was approved for tuberous sclerosis complex (TSC) as well.

Key trial results showed:

• Up to 50% reduction in seizure frequency in some patients

• A favorable safety profile compared to traditional antiepileptics

• Significant improvement in quality of life, especially in pediatric patients

Additional Studies

Since then, multiple peer-reviewed studies have reinforced the efficacy of high-CBD, low-THC therapies:

• A 2020 review in Epilepsia concluded that CBD reduces convulsive seizures with statistically and clinically significant effects.

• Trials conducted in Europe, Israel, and Australia have shown consistent positive outcomes, even in drug-resistant cases.

• Low-THC components are increasingly studied for their synergistic effects, helping stabilize CBD’s action.

Mechanism of Action: Why CBD Works

CBD does not act on traditional seizure-modulating targets like sodium channels or GABA receptors. Instead, it influences:

• Endocannabinoid system (ECS): Balances neurotransmitter release

• TRPV1 receptors: Modulate temperature and pain, implicated in seizure thresholds

• Adenosine signaling: Helps regulate inflammation and neuronal excitability

These pathways make CBD particularly useful in complex, treatment-resistant epilepsies where other drugs fail.

Safety and Tolerability

One of CBD’s biggest advantages is its safety profile. Unlike many antiepileptic drugs (AEDs), it does not cause cognitive dulling, mood changes, or addiction. Most side effects are mild to moderate, including:

• Fatigue

• Diarrhea

• Appetite changes

• Elevated liver enzymes (when used with valproate)

These are manageable with dose adjustment or monitoring, making it suitable even for children and vulnerable populations.

Regulatory Developments: Global Momentum

While the U.S. FDA was the first major regulator to approve a CBD-based epilepsy drug, other regions are following suit:

Europe

• EMA (European Medicines Agency) approved Epidyolex (the EU version of Epidiolex) for Dravet and LGS.

• Countries like Germany, UK, France, and Ireland allow prescription CBD for epilepsy, with insurance reimbursement in some cases.

Canada

• Medical cannabis has been legal since 2001.

• Health Canada allows CBD prescriptions for epilepsy under the ACMPR (Access to Cannabis for Medical Purposes Regulations).

Australia and Israel

• Both countries lead in cannabinoid research and allow high-CBD epilepsy treatments, even for pediatric patients, under special access schemes.

Regulatory Hurdles

Despite progress, some countries maintain strict controls. Standardization, quality control, and physician education remain challenges that need coordinated action.

Investment Perspective: Market and Opportunity

The epilepsy market is large and underserved. Globally, over 50 million people live with epilepsy, and up to 30% are drug-resistant. This creates massive demand for innovative treatments.

Market Highlights

• The global cannabis pharmaceuticals market is expected to reach $5.3 billion by 2028, with epilepsy as a key segment.

• Epidiolex alone generated over $730 million in revenue for Jazz Pharmaceuticals in 2023.

• Investors are increasingly interested in biosynthetic cannabinoids, which promise cost-effective and consistent production at scale.

Innovation Beyond Epidiolex

Next-gen therapies go beyond purified CBD:

• Full-spectrum extracts with a tailored cannabinoid mix

• CBDV (Cannabidivarin), a lesser-known cannabinoid, shows promise in preclinical epilepsy models

• Nanoemulsion technology for improved bioavailability

• AI-driven formulations optimizing dosage and ratios

Startups and biotechs are racing to bring novel cannabis-derived neurology treatments to market. IP protection, clinical validation, and global reach will determine who wins.

Patient Impact: Stories That Matter

Anecdotes abound of children with Dravet syndrome going from 300 seizures a week to a handful a month after starting CBD therapy. For many families, these treatments are life-changing—not just for seizure control but for enabling development, speech, and mobility.

Pediatric neurologists increasingly include cannabinoid therapy in their toolkit, especially after exhausting conventional AEDs.