[Frankfurt, Germany] – A recent study has investigated a novel approach to treating ataxia telangiectasia (AT), a rare neurodegenerative disorder. The findings, published in Lancet Neurology, offer hope for patients with this condition.
AT is a progressive disorder that can lead to severe physical and cognitive disabilities. While corticosteroids can improve neurological function in some patients, their use is limited due to side effects and potential for symptom recurrence.
The ATTeST study (Safety and efficacy of intra-erythrocyte dexamethasone sodium phosphate in children with ataxia telangiectasia) evaluated the safety and efficacy of intra-erythrocyte delivery of dexamethasone sodium phosphate (Dexa-RBC) compared to placebo in children with AT. Dexa-RBC is a new method of delivering corticosteroids that can potentially reduce side effects.
Although the study did not achieve its primary goal of improving neurological function, it did demonstrate that Dexa-RBC is generally well tolerated. There were no serious adverse events reported, and common steroid side effects were not observed.
Based on the findings, researchers plan to continue studying Dexa-RBC in a specific subgroup of AT patients aged 6-9 years old. The hope is that this approach may offer a more effective and safer treatment option for these individuals.
Zielen et al. Lancet September 2024.