Rosuvastatin, marketed by AstraZeneca under the brand name Crestor, belongs to a class of medicines called statins that have been used for years to lower cholesterol levels in people at risk for coronary artery disease. It currently is being investigated as a potential treatment for Friedreich’s ataxia (FA).

FA is a lifelong disease not only without a cure, but also without a treatment that can prevent or slow its progression.  Investigators are hopeful that exploring the action of rosuvastatin in patients with FA will uncover potential targets for therapy.

How rosuvastatin works

It lowers levels of low-density lipoprotein cholesterol (LDL-C), or “bad cholesterol,” while raising levels of high-density lipoprotein cholesterol (HDL-C), known as “good cholesterol.” Apolipoprotein A-1 (ApoA-1) is one of the main components of HDL-C, and statin drugs raise HDL-C mainly by increasing the production of ApoA-1.

Scientists at the University of Pennsylvania discovered that people with FA have lower levels of ApoA-1 than people without the disease. It is thought that rosuvastatin can raise levels of ApoA-1 in people with FA.  Understanding the effects of rosuvastatin on ApoA-1 levels may shed light on new potential targets for treating FA.

Clinical trials in FA

The potential application of rosuvastatin in people with FA is being explored in a Phase 1 clinical trial (NCT02705547) sponsored by the Children’s Hospital of Philadelphia, in collaboration with Friedreich’s Ataxia Research Alliance (FARA).

The trial is in its early stages and is still recruiting participants. It is considered exploratory and is open-label, meaning that all participants will know they are receiving the drug. Participants, who must be adults with genetically confirmed FA, will receive 10 mg of rosuvastatin for 12 weeks. At the end of this period, blood levels of ApoA-1 will be analyzed to see if rosuvastatin significantly raises levels from baseline.  Participants also will be monitored for evidence of adverse effects.

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