Recent results of a Turkish trial have demonstrated the need for further research into IFN-gamma as a treatment for Friedreich’s ataxia (FA). This study showed that the therapy resulted in significant improvements in stance and walking skills, which contradicted a prior Phase 3 study that illustrated no ability to slow or stop disease progression. Medical professionals must investigate IFN-gamma further in order to truly understand its effects.
About the Turkish Study
Titled “Efficacy and Tolerability of Interferon Gamma in Treatment of Friedreich’s Ataxia: Retrospective Study” and published in Archives of Neuropsychiatry, this clinical trial was conducted at Faculty of Medicine of Erciyes University in Turkey and focused on the safety and efficacy of IFN-gamma.
14 patients participated in the trial. Together, they had a mean age of 29.6 and had been living with FA for an average of 9.4 years. The majority (11) were being treated with an experimental therapy called idebenone prior to the study. The mean length of treatment with IFN-gamma was 7.2 months, and the effects of the treatment were assessed on the Scale for the Assessment and Rating of Ataxia (SARA).
Results demonstrated:
- After three months, researchers noted sustained improvement in walking skills
- After six months, patients’ stance was greatly improved
- No changes in other motor functions measured by SARA
- 71.4% of participants experienced a common adverse effect of treatment, typically flu-like symptoms
- Most events were reported to be mild
- Two patients discontinued treatment
These results led researchers to define IFN-gamma as safe and partially effective.
Previous Research
Horizon Pharma investigated IFN-gamma in the past as a treatment for Friedreich’s ataxia. They market the product under the name Actimmune, and it has already received FDA approval for two other rare conditions. Preclinical data that demonstrated the drug’s ability to increase frataxin pushed Horizon to indicate INF-gamma as a treatment for FA.
In a Phase 2 trial, researchers discovered that subcutaneous injections of Actimmune given every three weeks were able to significantly lower disease severity while being well-tolerated by patients. These results did not translate to the subsequent Phase 3 trial. After six months, participants did not see a change in disease severity when compared to those receiving a placebo.
While these results were discouraging, Horizon continued to study the drug in an open-label extension study. Disease progression was noticed to be slightly slower, but there was little significant evidence. After this trial, the pharmaceutical company discontinued Actimmune’s development for FA.
About Friedreich’s Ataxia
Friedreich ataxia is a rare, hereditary disease that is characterized by movement and neurological symptoms. Affected individuals typically notice the effects around age 15, where they will experience difficulty moving and slurred speech. Symptoms will continue and may expand to problems with vision and hearing, cardiac arrhythmia, chest pain, fatigue, shortness of breath, foot abnormalities, scoliosis, and diabetes mellitus. A mutated FXN gene is responsible for this condition. This gene normally produces frataxin, which is necessary for proper mitochondrial function. When it is mutated, this process is interrupted and the mitochondria can no longer perform their functions. It is inherited in an autosomal recessive pattern. Treatment is symptomatic.
Find the source article here.
