Groundbreaking Clinical Trials to Investigate CBD and THC for Treatment-Resistant Epilepsy

In a significant development for patients battling treatment-resistant epilepsy, the National Health Service (NHS) in the UK has announced two clinical trials set to explore the safety and efficacy of cannabidiol (CBD) and tetrahydrocannabinol (THC) in both adults and children. These pioneering studies are set to begin in 2025 and aim to bring new hope to those who have not responded to conventional epilepsy treatments.

Details of the Trials

Co-led by prominent researchers Prof Finbar O’Callaghan and Prof Helen Cross from University College London (UCL) and Great Ormond Street Hospital (GOSH), these trials will involve a total of 500 participants who suffer from medicine-resistant early-onset and genetic generalized epilepsies. The study’s design includes a randomized assignment of participants to receive either:

• CBD alone
• CBD combined with a small amount of THC
• A placebo

The trials will last for a period of 24 weeks and are open to recruitment from NHS facilities across the UK. The formulations under examination, developed by Ananda Developments, are identified as MRX2 (pure CBD) and MRX2T (CBD with THC).

Study Goals and Expectations

The overarching objective of these trials is to assess whether these cannabinoid-based medicines can effectively reduce the frequency and severity of seizures in patients. Beyond seizure control, the research will also examine the impact of treatment on various aspects of patients’ quality of life, including:

• Learning capabilities
• Sleep patterns
• Behavioral responses
• Stress and anxiety levels

Ananda Developments has highlighted the uniqueness of these trials, stating, “These will be the world’s first double-blind randomized controlled trials to investigate the use of CBD and THC together for epilepsy, and the first to evaluate the medicines in children and adults who have a broad range of difficult-to-treat epilepsies.”

Pathway to Approval

The potential success of these trials may lead to applications for regulatory approval from the Medicines and Healthcare Products Regulatory Agency (MHRA) and other relevant authorities for the use of these treatments in refractory epilepsy cases. At present, Epidyolex, a CBD medicine, is licensed for use within the NHS for patients suffering from Dravet syndrome and Lennox-Gastaut syndrome.

Current Recommendations and Need for Further Research

Despite the availability of CBD products since 2018, the National Institute for Health and Care Excellence (NICE) has not recommended Epidyolex for severe and treatment-resistant epilepsies, citing the need for more robust evidence on its safety and effectiveness.

Importance of the Upcoming Trials

Rebekah Smith, Deputy Chief Executive at Epilepsy Action, expressed optimism regarding the trials, stating, “It’s encouraging to see these long-anticipated trials for medicinal cannabis finally taking place. The need for more research on the effectiveness and safety of both the CBD and THC elements of cannabis is much needed, and announcing this research is a source of hope for many families.”

Smith’s comments reflect the sentiments of many who acknowledge the current limitations of existing treatments and the hope that these trials can challenge the stigma and misconceptions associated with medicinal cannabis.

Matt Hughes, Co-founder and Director of the Medcan Family Foundation, also welcomed the announcement, underscoring the importance of expanding access to CBD medicines for treating drug-resistant epilepsy. He remarked on the significance of including THC in the trials, noting, “We continue to urge government support in creating a pathway for the licensing of these more advanced medications.”

Looking Ahead

As the date for the trials approaches, there remains a consensus on the urgent need for comprehensive research in this area. The trials not only promise to shed light on the effectiveness of existing cannabis-based treatments but also offer a framework for potentially licensing more advanced cannabinoid medicines that families have found to be beneficial.

For more information regarding medical cannabis and epilepsy, interested individuals can visit the Epilepsy Action website.

Frequently Asked Questions