James “Brad” Elder, MD, Director of Neurosurgical Oncology at the Ohio State University Comprehensive Cancer Center, discusses a clinical trial evaluating the investigational gene therapy, AVB-101 in patients with frontotemporal dementia (FTD).
FTD is a neurodegenerative disorder associated with shrinking of the frontal and temporal anterior lobes of the brain. Symptoms include changes in social behavior and personality, and/or problems with language. People with behavior changes may have disinhibition, apathy and loss of empathy, hyperorality, agitation, compulsive behavior, and various other changes. Examples of problems with language include difficulty speaking or understanding speech. Some people with FTD also develop motor syndrome such as parkinsonism or motor neuron disease. There is a strong genetic component to FTDs. The three main genes responsible for familial FTD are MAPT, GRN, and C9orf72. However, the genetic cause of familial FTD cannot always be identified.
ASPIRE-FTD is a phase 1/2 open-label, multicenter, dose-escalation clinical trial currently enrolling patients. The goal is to evaluate the safety and efficacy of AVB-101, an investigational gene therapy being developed for patients with FTD with progranulin (GRN) gene mutations (FTD-GRN).
AVB-101 is an investigational one-time infusion containing non-mutated GRN. It is being developed to restore levels of progranulin in the brain, in hope of stopping or slowing progression of FTD.
For more information on the clinical trial and enrollment, click here.
To learn more about rare neurological diseases, visit https://checkrare.com/diseases/neurology-nervous-system-diseases/
