Methylprednisolone
Based on clinical observations and growing evidence of inflammation in FA, Dr. David Lynch at the Children’s Hospital of Philadelphia conducted a small exploratory study. This was an open-label study to explore safety, tolerability, and efficacy of methylprednisolone treatment in 11 individuals with FA. In addition, the study explored if methylprednisolone had any effects on biomarkers associated with FA.
INACTIVE: Stages of Development for Methylprednisolone
The drug development process can be thought of as a series of stages, and drugs must successfully pass through each stage to become available to patients. This treatment has been evaluated, and the program has been discontinued. Thus, it is not in the pipeline.
A secondary inflammatory response has been proposed to be present in FA, as revealed in autopsy studies and in the alteration of immune pathways in microarray analysis. Inflammation in FA raises the possibility of a therapeutic benefit from anti-inflammatory steroid treatment, as inflammation may directly underlie multiple complications of FA including cardiomyopathy. In support of this theory are clinical observations and patient self-reports of improvement of ataxia symptoms following the prescription of steroids for indications other than the primary FA diagnosis.
2015: Dr. David Lynch at the Children’s Hospital of Philadelphia conducted a small exploratory open-label study of safety, tolerability, and efficacy of methylprednisolone treatment in 11 individuals with FA. In addition, the study explored if methylprednisolone had any effects on biomarkers associated with FA.
Methylprednisolone was found to be well-tolerated. The efficacy outcomes were mostly unchanged except for a modest improvement in the pediatric cohort for 1-minute walk (P<.05).
No changes in frataxin levels were detected in buccal cells.