IFN-γ for Friedreich ataxia: present evidence.

Abstract

IFN-γ-1b is currently US FDA approved as an orphan drug for the treatment of chronic granulomatous disease and severe malignant osteopetrosis. It is administered via subcutaneous injection and is a potential therapy for Friedreich ataxia (FRDA), a rare degenerative neurological condition. Ongoing Phase II and III trials in both adults and children with FRDA were preceded by a small Phase I, open-label trial in children that showed that IFN-γ-1b was reasonably well-tolerated and improved overall neurological function as measured by the Friedreich Ataxia Rating Scale after 12 weeks of treatment, though the primary outcome measure of frataxin level showed no improvement. Although there is an established dose of IFN-γ-1b prescribed for the current indications, the efficacy and tolerability of these dose levels in the FRDA population remains the subject of ongoing investigation.

• Authors: McKenzie Wells, Lauren Seyer, Kimberly Schadt, David R Lynch
• Journal: Neurodegenerative disease management (Neurodegener Dis Manag) Vol. 5 Issue 6 Pg. 497-504 (Dec 2015) ISSN: 1758-2032 [Electronic] England
• PMID: 26634868 (Publication Type: Journal Article)

Chemical References

• Neuroprotective Agents
• Recombinant Proteins
• interferon gamma-1b
• Interferon-gamma

Topics

• Animals
• Clinical Trials as Topic
• Friedreich Ataxia (drug therapy)
• Humans
• Interferon-gamma (adverse effects, pharmacokinetics, pharmacology, therapeutic use)
• Neuroprotective Agents (adverse effects, pharmacokinetics, pharmacology, therapeutic use)
• Recombinant Proteins (adverse effects, pharmacokinetics, pharmacology, therapeutic use)