Experiences with Cannabidiol in Patients with NCL Disease

Introduction: Neuronal ceroidlipofuscinoses (NCLs) are lysosomal storage diseases with progressive neuronal degeneration. Symptom onset and combination of symptoms can vary with respect to different NCL forms. Mostly, first symptoms develop in early childhood or at school age. All forms have in common progressive decline in motor, language, cognitive and visual skills and the occurence of seizures. Usually patients die in childhood or early adulthood. Cannabidiol (CBD) is currently used for different indications like therapy-resistant epilepsy. The question is often raised whether the CBD shows also a positive effect in patients with neurodegenerative brain diseases.

Material/Methods: We analysed a cohort of 8 patients with different forms of NCL. All of them received CBD as off-label, palliative therapy due to different indications.

Results: We analysed 8 patients with NCL disease (CLN1, CLN2, CLN 3, CLN7) who received treatment with CBD. The patients started with therapy at different stages of the disease. CBD was well tolerated in 7/8 patients. Dosages ranges from 4,3 to 20 mg/kg bodyweight. The indications for CBD were therapy-resistant epilepsy, movement disorders (dystonia, myoclonus), restlessness, psychosis. One of the eight patients showed a deterioration of his psychosis under treatment with CBD so that the CBD was stopped at a low dosage. Another patient developed a secretion and coughing problem under high dosage of CBD which was refractory after dosage was reduced. 7/8 families reported an alleviation of symptoms under treatment with CBD. In 3/8 patients we were able to stop one of their medication after starting with CBD.

Discussion: The cohort was very variable regarding indication, NCL-type and stage of the disease. All in all the CBD was well tolerated in the patients at dosages between 4,3- 16,8 mg/kg bodyweight, despite of one patient with psychosis. In this patient we started CBD in a bad period of psychosis (as ultima ratio), so that it is unclear if the deterioration was a side effect or the dosage was too low with an inappropriate efficacy.

Conclusion: CBD was well tolerated in the majority of our cohort of NCL patients and the parents reported a positive efficacy. Nevertheless, it has to be considered that we only report retrospectively about individual cases in this cohort and we did not perform a study with clealy defined endpoints. But a study with indications and endpoints would help to answer the question of efficacy of CBD in patients with NCL.