New pharmacological therapy for a rare mitochondrial disease

New pharmacological therapy for a rare mitochondrial disease https://noticiasdelaciencia.com/upload/images/11_2018/9208_45685.jpg?21

New pharmacological therapy for a rare mitochondrial disease





Scientists from the University of Granada (UGR) (Spain) have developed a new pharmacological therapy, which shows similarities with one of the precursors of coenzyme Q10 (CoQ10), which is effective against a severe mode of deficiency in Coenzyme Q10 (CoQ10) , a rare mitochondrial disease that affects mostly children and for which there is currently no effective treatment.

 

His work, which has just been published in EMBO Molecular Medicine, has demonstrated the effectiveness of this experimental treatment in an animal model (mice), generated at the UGR, with severe deficiency in CoQ, encephalomyopathy and premature death, through the administration chronic of an analogue of one of the precursors that eukaryotic cells use to synthesize CoQ.

 

Coenzyme Q10 is a molecule synthesized in the cells of the body and that has essential functions in the cellular metabolism. Deficiency in CoQ10 is a syndrome with very heterogeneous clinical manifestations. Usually, cases that present neurological symptoms or those that manifest the multisystem variety do not respond to conventional treatment with high doses of exogenous CoQ10, due to their low capacity to cross biological barriers and reach nerve tissue.

 

In this context, the objective of this work has been to evaluate a therapeutic option that is effective for the most severe cases of deficiencies in CoQ10. The working hypothesis of the researchers was based on previous studies in yeast and cell cultures generated in recent years by various international groups, including the UGR.

 



 

Researchers who have participated in the study. (Photo: UGR)

 

The results show that the therapeutic molecule is capable of modulating the CoQ synthesis complex, so that endogenous CoQ synthesis is stimulated. "But we have also seen, and this is even more important, that the therapeutic molecule is capable of reducing the levels of intermediary metabolites of CoQ that can be toxic to the power plant of the cells," explains the Center's researcher. of Biomedical Research of the UGR Luis Carlos López García, one of the authors of the work.

 

Consequently, these metabolic changes induce an increase in the ability 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.

 

"The therapeutic results we have obtained are very important, so one of the medium-term objectives is to make the translation to the clinic," says López. On the other hand, from an intellectual point of view "there are some response mechanisms to treatment that we still do not understand, so we are working on new experimental designs that help us understand these aspects, which should contribute to better understand the physiopathology of mitochondrial diseases and to generate new therapeutic advances, for this and other mitochondrial pathologies. 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 without severe disease states ".

 

Most of the experimental work of this research has been developed by Agustín Hidalgo and Eliana Barriocanal, hired by FPU and the Junta de Andalucía, respectively, in the CTS-101 group of the Biomedical Research Center of the University of Granada.

 

The work has also had important collaborations, among which are those of Professor Mohammed Bakkali, of the Department of Genetics of the UGR, and that of Professors Juan Duarte and Miguel Romero, as members of the Unit of Excellence "UNETE". (Source: UGR / DICYT)
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