Is there a cure for mitochondrial diseases?

Mitochondrial diseases have many, many causes and therefore will have many, many cures. Many drug trials take years -- and treatment advances happen one small step at a time. Which means in the case of mitochondrial diseases that the one cure, one timeline, simply does not exist but with research we will move forward faster.

Drug development for mitochondrial disease has only just begun. The road to successful new drugs and drug therapies is riddled with incredible successes and forgotten failures. Recent studies estimate an average timeline to bring a drug to market is 13 years and $1 billion; 75% of the costs of drug development are associated with compounds failing in early stage development. Additionally, 90% of new drugs fail in the second and third phases.

The Foundation for Mitochondrial Medicine identifies one barometer of success: supporting and accelerating the development of the most promising mitochondrial research and treatments of the many, many forms of mitochondrial disease.

At this point there are only a few drugs for mitochondrial disease that have reached early phases of the FDA drug development approval process. The more resources that are available, the more researchers can augment the path to the cure including an increase in clinical drug trials. The Foundation’s accelerator fuels connections, encourages scientific collaboration, and will bring forth people, ideas and partnerships to set the wheels in motion to develop treatments, sooner and quicker.

While there is no cure for mitochondrial diseases, there are treatments and therapies that have proven to lessen or manage the symptoms as well as slow the advancement of the disease. Genetic testing can help determine whether or not you will pass the disease to your children. Sometimes, avoiding specific harmful substances that have been linked to mitochondrial diseases can help prevent the disease; your doctor can provide more information about those kinds of substances.

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Important: This content reflects information from various individuals and organizations and may offer alternative or opposing points of view. It should not be used for medical advice, diagnosis or treatment. As always, you should consult with your healthcare provider about your specific health needs.