A new pharmacological therapy against a severe kind of deficiency in Coenzyme Q10

Scientists from the University of Granada have developed a new pharmacological therapy using a molecule that presents similarities with one of the precursors of coenzyme Q10 (CoQ10). The therapy is effective against a severe type of deficiency in Coenzyme Q10, a rare mitochondrial disease that targets mostly children and for which there is currently no effective treatment.

A new pharmacological therapy against a severe kind of deficiency in Coenzyme Q10. Image credit: Ragesoss/Wikimedia Commons (CC BY-SA 3.0)

CoQ10 is a molecule synthesized in body cells with key functions in the cellular metabolism. Deficiency in CoQ10 is a syndrome featuring very diverse clinical manifestations.

Usually, cases that present neurological symptoms or those that manifest the multisystem variant do not respond to the conventional treatment with high doses of exogenous CoQ10, due to the low capacity of this molecule to cross biological obstacles and reach the nervous system.

In this regard, the focus of this research has been to assess a therapeutic option that is effective for severe cases of deficiencies in CoQ10. The working hypothesis of the researchers was based on recent studies in yeasts and cell cultures generated by some international groups.

The results indicate that the therapeutic molecule is capable of modulating the multiprotein complex for CoQ10 biosynthesis, so that endogenous CoQ10 synthesis is stimulated. “But we have also seen, and this is even more striking, that the therapeutic molecule is capable of reducing the levels of intermediary metabolites of CoQ10 that can be toxic to the power plant of the cells” explains Luis Carlos López García, a researcher of the UGR Biomedical Research Center.

Consequently, these metabolic changes induce an increase in the capability of cells to produce energy, together with a reduction in the molecular and histopathological markers characteristic of mitochondrial encephalomyopathies.

The final result is an improvement of the phenotypic characteristics of the animal model with a very significant increase in life expectancy: while the untreated mutant mice reach a maximum of 7 months of life, the treated mutant mice are able to reach up to 25 months of life, approaching the survival curve characteristic of healthy mice.

“There are some response mechanisms to the treatment that we still do not understand, so we are working on new experimental approaches that will help us to figure out these aspects, which should contribute to better understand the pathophysiology of mitochondrial diseases and to generate new therapeutic advances, for this and other mitochondrial disorders. At the same time, we are testing other molecules, with very interesting characteristics, that may be important not only for the treatment of severe diseases but also as nutraceuticals for the healthy population or for people without severe pathological conditions”, Prof. López observes. (University of Granada)

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