Randomized, double‐blind, placebo‐controlled study of interferon‐γ 1b in Friedreich Ataxia

Lynch, D. R., Hauser, L. , McCormick, A. , Wells, M. , Dong, Y. N., McCormack, S. , Schadt, K. , Perlman, S. , Subramony, S. H., Mathews, K. D., Brocht, A. , Ball, J. , Perdok, R. , Grahn, A. , Vescio, T. , Sherman, J. W. and Farmer, J. M. (2019); Ann Clin Transl Neurol. doi:10.1002/acn3.731

Results
No difference was noted between the groups after 6 months of treatment in the mFARS or secondary outcome measures. No change was noted in buccal cell or whole blood frataxin levels. However, during an open‐label extension period, subjects had a more stable course than expected based on natural history data.

Conclusions
This study provides no direct evidence for a beneficial effect of IFN‐γ1b in FRDA. The modest stabilization compared to natural history data leaves open the possibility that longer studies may demonstrate benefit.



Randomized, double‐blind, placebo‐controlled study of interferon‐γ 1b in Friedreich Ataxia