New Therapy Developed for Severe Form of Mitochondrial Disease

According to a story from Medical Xpress, a team of scientists affiliated with the University of Granada have developed a new potential treatment for a form of mitochondrial disease that is linked to a deficiency of CoQ10, a coenzyme. This disease mostly affects kids and there are no currently available therapies.

About Mitochondrial Disease

Mitochondrial diseases are a group of genetic disorders that causes the mitochondria not to function properly. The mitochondria are an essential organelle that is found in most types of cells in the body, with red blood cells being the only exception. They are responsible for generating energy for the cell. Mitochondrial diseases are usually caused by mutations of the mitochondrial DNA or the nuclear DNA. Symptoms tend to be the worst when the issue affects cells that use a lot of energy, such as the muscles or parts of the brain. These symptoms affect many aspects of bodily function and include poor growth, poor muscle coordination, dementia, neurological issues, muscle weakness, breathing disorders, vision problems, digestive disorders, hearing problems, disease of the kidney, liver, and heart, and learning disabilities. Treatment options are limited in number and in their effectiveness. To learn more about mitochondrial diseases, click here.

Exciting Potential

This treatment is still highly experimental has yet to be tested on humans; however, results with animal models have been very encouraging so far. The researchers found that the therapy was able to restore mitochondrial function, allowing cells to produce energy again. The drug was also able to dramatically increase lifespan for mice affected with a model of the disease. An untreated mouse only survived for about seven months, but with treatment lifespan increased to 25 months, which is much closer to the lifespan of a healthy mouse. 

While an obvious next step is to begin testing the drug in clinical trials with human patients, the researchers also want to conduct more tests with the drug in order understand certain response mechanisms caused by the treatment. These experiments could help reveal new information about the mechanism behind mitochondrial diseases.

Check out the original study here.


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