Drug combination shows positive effect in models of FA - Ataxia UK

Drug combination shows positive effect in models of FA

Post Published: October 20, 2022

A study led by researchers at the London Ataxia Centre, in collaboration with UC Davis California and Brunel University London, has shown that a combination of resveratrol and a multiple sclerosis treatment called dimethyl fumarate had positive effects in cell and animal models of Friedreich’s ataxia.

Frataxin is a protein that is found in the mitochondria (the cell’s energy producers). In Friedreich’s ataxia (FA) there is a reduced amount of the frataxin protein. This is caused by a mutation in the frataxin gene, in other words, a change in the DNA sequence.

To make a protein, DNA is copied into instructions called messenger RNA (mRNA). These instructions are then read by the cell’s protein-making factories called ribosomes, which make the frataxin protein. In FA, less protein is made because there is a change in the DNA sequence, which disrupts this process.

In this study led by the London Ataxia Centre, the researchers tested eight different drugs in skin cells of people affected by FA to see whether they increased frataxin mRNA, the making of new mitochondria, and improved antioxidant response (all of which are reduced in FA). In these experiments, they found that dimethyl fumarate (DMF) and resveratrol worked best compared to the others. DMF is a drug that has been approved in the UK to treat relapsing multiple sclerosis. Resveratrol is naturally occurring and is found in the skin of red grapes. It is thought to act like an antioxidant.

The researchers next decided to test whether treatment with both DMF and resveratrol together could be an effective approach.  They found that DMF and the combination of DMF and resveratrol improved mitochondrial function in cerebellar cells of a mouse model of FA, while resveratrol by itself did not make a significant difference. They also found that treatment with both drugs improved mitochondrial function in cerebellar cells of another FA mouse model.

Next, the researchers tested whether the drug combination was effective in a longer-term animal study using a mouse model of FA. They found after four months of treatment with both DMF and resveratrol, mice had improved performance in a test measuring balance and coordination compared with mice that were not treated with the drugs.

Overall, this study shows that combining DMF and resveratrol could be a valuable approach to treating FA and warrants further investigation.

Dr Rosella Abeti, the leading researcher on the project, said: “I am very pleased that we paved a new way to target FA. Our drug combination approach could lead to better treatments for patients with this incurable disease. We are also very grateful to FARA for awarding a grant to our collaboration and making this project possible”.

You can access the full research paper here.

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