New Experimental System to Screen Drugs for Friedreich’s Ataxia

New Experimental System to Screen Drugs for Friedreich’s Ataxia

A recent study from researchers based in France described a new experimental system that can be used to identify drugs to treat Friedreich’s ataxia (FA). The research report, titled “A Yeast/Drosophila Screen to Identify New Compounds Overcoming Frataxin Deficiency,” appeared in the journal Oxidative Medicine and Cellular Longevity.

FA is a rare condition of the nervous system that involves neuronal death, and according to the Friedrich’s Ataxia Research Alliance (FARA), approximately one in every 50,000 people in the United States have FA. This disease is associated with co-existing medical conditions including scoliosis, heart disease, and diabetes. Unfortunately, the disease has no cure or effective treatments that can halt its progression, and because it is a genetic, inherited disorder, people with Friedreich’s ataxia could potentially be identified and treated early, preventing its progression.

Led by Alexandra Seguin of the Institut Jacques Monod in Paris, France, the team used genetically modified yeast and a fruit fly model of FA to create FA-like cells on which to test several different drugs. Researchers accessed the French National Chemical Library, which contains 5,500 compounds, and the Prestwick collection, which contains 880 compounds. According to the authors: “Half of the compounds selected in yeast appeared to be active in flies (…) and one of the two compounds with highest activities in this assay partially rescued the heart dilatation phenotype resulting from heart specific depletion of frataxin. The unique complementarity of these two frataxin-deficient models, unicellular and multicellular, appears to be very efficient to select new compounds with improved selectivity, bringing significant perspectives towards improvements in FA therapy.”

The authors conclude, “Altogether, the present results open new and promising ways to decipher the molecular basis of frataxin deficiency and to develop original compounds with some efficiency to treat FA.”

The two different models, yeast and fruit fly, proved to be efficient to screen chemicals that could be used as future medications for FA. Other researchers can now use this system to help identify molecules that may be developed into FA medications. The finding gives promise to those suffering from FA, as currently there are no effective treatments.

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