Safety Study of AAV hTert and Klotho Gene Transfer Therapy for Dementia

Abstract

A sponsored, interventional, non-randomized study without a control group using a novel and proprietary central nervous system gene transfer method to deliver AAV hTert and Klotho genes to five patients with mild or moderate dementia was performed to primarily evaluate safety. Clinical response data was gathered as a secondary interest. The therapy demonstrated a very high safety profile with no serious adverse effects identified. Clinical evaluation of the patients over the course of the one year follow up yielded significant findings with all five patients demonstrating evident reversal of Dementia symptoms such as sustained cognitive improvement as measured by the Folstein exam. Telomere analysis was performed before and after the therapy. A measurable elongation of the participants telomeres was identified, and biological age was reduced as chronological age increased.