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A metabolite from a drug against diabetes, a possible treatment for neurodegenerative diseases

MIN-102, a metabolite from a drug against diabetes, could be useful for treating some neurodegenerative diseases, as scientists think. To confirm this hypothesis, a project has been launched with the involvement of the CSIC’s Instituto de Investigaciones Biomédicas de Barcelona, the Hospital Universitari 'Sant Joan de Déu' and the University of Lleida. The research project is led by the company Mynorix Therapeutics.

In the image, damage due to neuroinflammation can be seen. In A, in vitro culture of neurons and glia, in which the neurons are marked.  In B, culture of neurons and glia, where neuronal death has occurred by glial activation. Image: IIBB / CSIC.In the project, funded by the Spanish program RETOS, the scientific team will investigate the potential of MIN-102 for treating diseases such as the Rett syndrome, the optic atrophy type 1 (a disease that affects the optic nerve and is caused by a genetic change in the OPA1 gene), as well as mitochondrial diseases caused by mutations in the NDUFS1 gene, and neurodegenerative diseases like Huntington, Friedrich ataxia and Parkinson. All of them are related to neuroinflammation and to an impaired function of the mitochondria (the cell organelles that produce energy for the cell).  Some of them are rare diseases.  

The researchers will study the effects of the molecule on the mitochondrial function “in vitro” models. Also, the scientists will study ‘in vitro’ and ‘in vivo” the benefits for inflammation.

The team wants to find out whether the molecule has a therapeutic benefit for treating some of these diseases. If results are successful, the scientists will apply for designation of MIN-102 as an orphan drug in Europe (through the EMA, European Medicines Agency) and for the USA (Food and Drugs Administration, FDA).

The project will last two years and has a budget of more than a million Euros, which will be partially funded by European ERDF funds, through the programme RETOS of the Spanish Ministry of Science, Innovation and Universities.

Previous studies

As a matter of fact, Mynorix is now developing a new treatment for adrenoleukodystrophy, based on the MIN-102 molecule. To date, no pharmacological treatment has been found for this genetic disease linked to the X-chromosome, which causes progressive neurological impairment.

Previous studies found out that the MIN-102 molecule has protective effects on neurons, astrocytes and oligodendrocytes, and that preserves cells from oxidative stress as it increases mitochondrial biogenesis. Also, some tests have demonstrated that MIN-102 has positive effects against inflammation.

Carme Solá, a CSIC scientist and main investigator of the IIBB team, says: neuroinflammation is a common characteristic of all neurodegenerative diseases, therefore it is a possible target when it comes to minimizing neuronal damage in these pathologies.  In the research, we will study the anti-inflammatory and neuroprotective effects of MIN-102 for the Parkinson disease. We will test the molecule on cell cultures of glia cells and neurons, as well as on a mouse-model of the disease”.