Mitochondrial disease (MD) is a heterogeneous group of pathologies caused by deficits in the ability of cells to generate sufficient energy to function. MD are neuromuscular diseases that mostly affect children, leading to severe multi-organ failure and death. Current treatments are limited to palliative care and vitamins or other supplements without clear indication of their effectiveness. MitoCBD has identified Cannabidiol (CBD), a compound found in cannabis without psychoactive activity, as a potential novel treatment for MD.
- To develop an innovative treatment for MD based on repurposing cannabidiol (CBD), a drug with known anti-inflammatory, neuroprotective, and anti-epileptic effects.
Problem to Solve
1 in every 5000 children is born with mutations that render their mitochondria, the cellular energy generators, unable to function. These pathologies, known as mitochondrial disease, affect high energy-requiring tissues, like brain and muscles, leading to severe disability and death. To date, there is no effective treatment or cure for mitochondrial disease. Therefore, there is an urgent need to identify innovative and effective treatments for these orphan diseases.
MitoCBD provides an innovative therapeutic approach to treating mitochondrial disease by proposing the use of cannabidiol (CBD). CBD is a well-tolerated, non-psychoactive, neuroprotective and anti-inflammatory drug that has already been approved by the FDA, which could help to fast-track its commercialization.
CBD has shown extremely positive results in one of the best in vivo models of mitochondrial disease, ameliorating the motor and social deficits, as well as extending the lifespan of the animals, in stark comparison to other drugs currently undergoing clinical trials for MD. These results highlight the potential of CBD as a treatment for MD.