Unfortunately, there are limited treatments or therapies and no proven cure(s) for mitochondrial disease.
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.
Gene therapies are showing promise as an effective way to treat some forms of mitochondrial disease. The most prominent gene therapy is the Mitochondrial Replacement Therapy (MRT), aims to prevent the transmission of heritable disorders caused by mutations in the mitochondrial genome. MRT is a pre-conception intervention that involves transferring the nucleus from an oocyte that contains pathogenic mitochondrial DNA to an enucleated donor oocyte that contains only normal mitochondrial genomes. MRT is currently illegal in Canada and will require a change in the Assisted Human Reproduction Act (AHRA). The UK is the only country to date that has legislated the legalization of MRT. Canadian scientists and clinicians are investigating the approach to MRT in Canada and have published a research paper“Mitochondrial Replacement Therapy: The Road to the Clinic in Canada”.
The Future of Therapy will be taking a precision medicine approach. Due to the complexity of disease manifestation, from at the genetic level and symptom presentation from person-to-person, a precision medicine approach for mitochondrial disease is the path to future therapies and clinical application. When the precise genetic cause is identified, models can be created (either in the patient’s own cells or in invertebrate and vertebrate animal models) that will help to better understand the way in which the biochemistry has caused a problem and how the functions of the organs and are impaired. From there, therapies such as mitochondrial medicine supplement regimens, nutrients, and exercise can be tested to precisely understand whether they are helpful or harmful in any one type of mitochondrial disease. Additionally, new compounds could be screened to more quickly and effectively identify therapies that are precisely matched to each patient’s metabolism and their 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
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