Research Project: A Feasibility Study of Bezafibrate in Mitochondrial Myopathy.
Principal Investigators: Prof Rita Horvath
As a clinical neurologist with an interest in neuro-genetic disorders, including mitochondrial diseases, my work has a strong translational focus.
I am currently working on a phase II, open label, proof of concept study that aims to identify whether use of bezafibrate can improve mitochondrial function in people with the m.3243A>G mutation. Bezafibrate is an already licensed drug that can be prescribed for high blood triglyceride levels. Based on pre-clinical studies it appears to have broader metabolic effects than simply reducing triglyceride levels, and in some models its use has improved mitochondrial function.
In my study, participants take bezafibrate for 12 weeks, and undergo a number of assessments pre and post treatment to determine the treatment effect. These assessments include: muscle biopsy, muscle and cardiac MRI, exercise testing and standardised questionnaire completion.
Results are expected later in 2017.
Sponsor/funder: Newcastle University, Confidence in Concept Fund