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🚀 !Incredible Clinical Trial for Frontotemporal Dementia Begins in the U.S.! 🚀 AviadoBio's ASPIRE-FTD Phase 1/2 Clinical Trial is now open at The Ohio State University Wexner Medical Center! ASPIRE-FTD recruits participants to evaluate AVB-101, a gene therapy designed to stop FTD progression caused by GRN mutations. The study is also ongoing in Europe, and aims to restore progranulin levels! The therapy is delivered to the thalamus via a minimally invasive procedure. Why This Matters: 🧠 FTD impacts those under 65, causing personality changes, behavioral issues, and loss of language and mobility. With no current treatments, this trial offers a new course of therapy. 🧠"People with FTD-GRN have no disease-modifying treatments, and the impact is profound for patients and families," said Dr. James Elder, neurosurgeon at The Ohio State University Wexner Medical Center #GeneTherapy #FTD #NeurodegenerativeDisorders #ClinicalTrials #MedicalResearch #Neurology #Neurosurgery #OhioState #AviadoBio #InnovationInMedicine #GRNMutations #AVB101 #EarlyOnsetDementia #MedicalBreakthroughs Image: Fig. 1a | Intrathalamic gene therapy delivery in sheep. MRI image of sheep skull demonstrating simulated bilateral intrathalamic infusion (blue) into the brain. https://lnkd.in/ebgB5Vz3
Experienced Senior Project Leader, Clinical Trials; Biotech and Pharma. Delivering timely solutions in clinical trials for enhanced drug development
2wCongrats to AvadioBio starting the battle for Frontotemporal dementia patients (FTD) is a cruel disease, and gene therapy offers a glimmer of hope. FTD damages brain cells, leading to personality changes, language difficulties, and movement problems. In some cases, mutations in the progranulin (GRN) gene cause FTD. People with these mutations have lower levels of progranulin, a protein important for brain health. FTD gene therapy aims to replace the faulty GRN gene with a healthy copy. This is done by delivering a virus (modified to be safe) carrying the healthy gene directly to brain cells. The hope is that brain cells will start producing normal levels of progranulin, slowing, or stopping disease progression. A Targeted Approach: This approach targets a specific area in the brain called the thalamus, which has connections to regions affected by FTD. This delivery method aims to: Increase effectiveness by getting the therapy where it is needed most. Reduce potential side effects by delivering a lower dose.FTD gene therapy is still in initial stages, Phase 1/2 trials. However, it holds promise as a potential one-time treatment for FTD caused by GRN mutations.