Mitochondrial Treatments and Therapies

While mitochondrial disorders have no cure, there are some treatments available that can help reduce symptoms. Treatments vary by individual and depend on the type of mitochondrial disorder someone has been diagnosed with. Strides are being made in the field of gene therapy, gene editing and precision medicine that present great promise in improving the lives of those living with a mitochondrial disorder.

The ‘Mito Cocktail’ is the most common form of treatment, which involves a combination of vitamin supplements including but not limited to, acetyl-L-carnitine (ALCAR), coenzyme Q10 (CoQ10), alpha-lipoic acid (ALA), and creatine monohydrate (CM). This is an empirically based combination of vitamins, cofactors, and nutrients that are known to replace deficiencies that occur when the mitochondrial energy production system fails and to help its residual function act better. To learn more about the health benefits of CoQ10, click here to read this comprehensive review created by HealthyButSmart. 

Exercise has been scientifically proven as one of the most beneficial therapies (as long as the patient can tolerate it). When exercising, you produce more energy on a biochemical level, which improves mitochondrial function and increases the number of healthy mitochondria in the cells. Research indicates that exercise makes the body use oxygen more efficiently and increases exercise tolerance and metabolism. Patients should consult with their primary mitochondrial physician about incorporating an exercise plan as part of treatment. Here is a guide for exercise and mitochondrial myopathy. 

Mitochondrial Replacement Therapy (MRT): MRT aims to prevent the transmission of inheritable disorders caused by mutations in the mitochondrial genome. MRT involves transferring the nucleus from an oocyte containing pathogenic mitochondrial DNA to a donor oocyte that contains healthy mitochondrial genomes.

MRT is currently illegal in Canada and will require a change in the Assisted Human Reproduction Act (AHRA). To date, the UK is the only country that has legalized MRT. The Canadian mitochondrial community is investigating approaches to MRT: Mitochondrial Replacement Therapy: The Road to the Clinic in Canada and MitoCanada’s Position on MRT 

The Future of Therapy: Mitochondrial disorders are complex. The way a disorder manifests, genetics and symptom presentation, therapies like precision medicine are poised to be a game-changer as a new therapy. When the precise genetic cause of a mitochondrial disorder is identified, models can be created (either in the patient’s own cells or in animal models) to help scientists better understand the way in which biochemistry has caused a problem and how the functions of the organs are impaired. From there, therapies like mitochondrial supplements, nutrients, and exercise can be tested to precisely understand how helpful or harmful they are in any one type of mitochondrial disease. New compounds could be screened to quickly and effectively identify therapies that are precisely matched to each patient’s metabolism and disease.

Current Status of Mitochondrial Medicine – Dr. Marni Falk, a geneticist at the Children’s Hospital of Philadelphia (CHOP), is a Director of the the mitochondrial medicine frontier program. She is also a board of trustee member and a chair of the scientific and medical advisory board of the United Mitochondrial Disease Foundation. Click here to watch Dr. Falk’s video, it provides an excellent overview to the current status of mitochondrial medicine.