According to a report in today’s New York Times, the European Medicines Agency has recommended that gene therapy be approved for use in a rare disease called lipoprotein lipase deficiency. Should the European Commission follow this recommendation, as it usually does, it would be the first time gene therapy would be approved for use “in the Western World.”
The field of gene therapy has been historically hampered by disappointing results from hundreds of clinical studies, as well as by issues with safety. But bringing a gene therapy such as this one to market could open the door for investment into future gene therapy approaches, including those for Parkinson’s disease (PD).
An approval ‘is really potentially going to change the way the field is looked at,’ said Jeffrey Ostrove, chief executive of Ceregene, a gene therapy company in San Diego [and a Michael J. Fox Foundation (MJFF) awardee working to apply the technique to PD]. Some pharmaceutical companies have been reluctant to invest in the field, he said, because ‘there are no approved products in the major markets they sell in.’
Having such an approved product could help to alleviate concerns within the industry regarding safety and efficacy. It could also help to publicly de-mystify gene therapy approaches that have tended to be shrouded in mystery, and help to garner support for such approaches across a wider circle of researchers, says Jamie Eberling, PhD, associate director of research programs at MJFF.
Gene therapy works by attempting to change the expression of a person’s genes. In PD, the approach primarily is focused on restoring the function of dopaminergic neurons in the brain that have died during the course of the disease.
Several PD gene therapy trials are currently in the clinic, including one from Ceregene studying their therapy, CERE-120. CERE-120 aims to limit this loss of dopamine neurons by introducing the gene that makes the protein neurturin into targeted areas of the brain. Neurturin is a type of protein called a trophic factor that promotes the survival, growth and function of neurons. You can think of trophic factors as “fertilizer for the brain.”
The therapy recommended for approval in lipoprotein lipase deficiency is called Glybera, from Dutch pharmaceutical company uniQure.