Innovative gene therapy trial for Parkinson’s disease

A trial for a new gene therapy, known as AXO-Lenti-PD, aimed at improving the supply of dopamine in the brains of people with Parkinson's disease has been launched by researchers at UCL and University College London Hospitals (UCLH). In people affected by Parkinson's disease (PD), parts of the brain become progressively damaged over many years resulting in motor symptoms including tremor, rigidity and bradykinesia (slowness of movement). This deterioration is caused by a loss of dopamine-producing neurons (nerve cells) in an area of the brain called the substantia nigra. In turn, this leads to a reduction in the availability of dopamine, which acts as a neurotransmitter (a chemical released by neurons) to send signals to other neurons, and has a role in controlling movement and balance. Gene therapy works by introducing genes via an inactivated viral vector (virus) to specific cells providing them with the genetic instructions needed to change their fate. AXO-Lenti-PD, also known as OXB-102, is a next generation gene therapy and is being tested for the first time by UCL and UCLH researchers at the National Institute for Health Research (NIHR) UCLH Clinical Research Facility, Leonard Wolfson Experimental Neurology Centre, Queen Square. The therapy has been developed by Oxford BioMedica and Axovant Sciences Ltd for the treatment of PD, and contains three genes that are responsible for producing dopamine, delivered in a lentiviral vector.