Innovative therapies for Batten disease and childhood epilepsy

Can the use of cannabis-derivatives reduce the distressing symptoms associated with Batten disease and childhood epilepsies?

Dr Stephanie Hughes
University of Otago, Dunedin

What is the problem and who does it affect?

Both Batten disease and epilepsy are neurological conditions. Epilepsy is a highly prevalent disease in New Zealand, affecting both adults and children. About 450 New Zealand children develop the condition each year. One third of those affected have seizures with major social, psychological, physical and cognitive consequences. Severe epilepsies starting in childhood have a mortality rate of 25% by 20 years of age.

Batten disease is a rare condition with new cases affecting around four New Zealand children and their families each year. It is a childhood neurodegenerative disorder that is characterised by seizures, blindness, dementia, and premature death. Sadly, Batten disease is always fatal with the life expectancy of children with late infantile Batten disease being eight to 12 years.

 

What is the project hoping to achieve?

Dr Hughes’ research focuses on drug and gene therapy for Batten disease. In this study, Dr Hughes and her team will test the effectiveness of cannabis derivatives in reducing symptoms in Batten disease and epilepsy.

Several children with severe epilepsy in New Zealand have recently been prescribed the treatment Sativex. The aim of this study is to test the effectiveness of two active ingredients of medicinal cannabis in reducing and/or preventing the development of Batten disease. Knowledge gained from this work will establish the likelihood of patient benefits from cannabis derivatives and, if successful, provide evidence towards funding a clinical trial in patients.

In parts of the US, children can have their epilepsy treated with cannabis oil, however, the research is still lacking. Dr Hughes believes a successful trial would be used to support the development of a human clinical trial and government funding of a much needed drug therapy for Battens and other childhood epilepsies.