title: 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.affiliation: Departments of Pediatrics & Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, USA.|Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
authors.forename: McKenzie
authors.initials: M
authors.lastname: Wells
authors.affiliation: Departments of Pediatrics & Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, USA.|Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
authors.forename: Lauren
authors.initials: L
authors.lastname: Seyer
authors.affiliation: Departments of Pediatrics & Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, USA.|Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
authors.forename: Kimberly
authors.initials: K
authors.lastname: Schadt
authors.affiliation: Departments of Pediatrics & Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, USA.|Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
authors.forename: David R
authors.initials: DR
authors.lastname: Lynch
mesh_terms: D000818:Animals; D002986:Clinical Trials as Topic; D005621:Friedreich Ataxia / Q000188:drug therapy*; D006801:Humans; D007371:Interferon-gamma / Q000009:adverse effects / Q000493:pharmacokinetics / Q000494:pharmacology / Q000627:therapeutic use*; D018696:Neuroprotective Agents / Q000009:adverse effects / Q000493:pharmacokinetics / Q000494:pharmacology / Q000627:therapeutic use*; D011994:Recombinant Proteins / Q000009:adverse effects / Q000493:pharmacokinetics / Q000494:pharmacology / Q000627:therapeutic use
chemical_list: D018696:Neuroprotective Agents; D011994:Recombinant Proteins; C554125:interferon gamma-1b; D007371:Interferon-gamma