Methylprednisolone is a steroid used to reduce inflammation, and is believed to help improve the symptoms of Friedreich’s ataxia (FA).

How may methylprednisolone help FA patients?

The idea that inflammation may be involved in FA is based on both clinical observations and patient self-reports. Some individuals with FA have noticed that their neuromuscular function improved when they were taking anti-inflammatory medications for other conditions. A study published in January 2016 reported improvement of FA symptoms in a patient with that disease had received corticosteroid medications for nephrotic syndrome, a kidney condition. The investigators also saw improvement of neurologic symptoms in other FA patients treated with corticosteroids who did not have nephrotic syndrome.

The idea has also been supported by laboratory observations, looking at tissue from deceased individuals with FA and analysis of changes in mitochondria — the root of the damage to nerves and muscles caused by the genetic mutation causing FA.

Methylprednisolone in clinical trial for FA

Based on these observations, the Children’s Hospital of Philadelphia (CHOP), in collaboration with Friedreich’s Ataxia Research Alliance (FARA), has initiated a clinical trial (NCT02424435) to explore the possibility that methylprednisolone may help improve FA symptoms.

The trial aims to find out whether methylprednisolone improves mobility and overall function in FA patients, as measured by a timed 25-foot walking test and scores on a number of other tests of both upper and lower body function. Investigators also want to determine whether methylprednisolone has adverse effects and is well-tolerated.

The study began in June 2015, enrolling five children and five adults all diagnosed with FA based on genetic testing. Participants received a supply of 8 mg tablets of methylprednisolone, to be taken by mouth over a 28-day cycle. They visited CHOP several times over a six-month period for evaluation. Researchers measured performance scores at the start of the study and at 26 weeks.

The study is closed for enrollment, the data has been collected, and the analysis is underway. Results are expected by early 2018.

Note: Friedreich’s Ataxia News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.