Introduction: Parkinson's disease (PD) is a common and chronic movement disorder with no therapy yet proven to alter the underlying advancing pathology. Gene delivery of trophic factors, which have shown disease modifying potential in preclinical PD models, are now being evaluated in early clinical trials.
Areas covered: This review discusses early experiences with glial-derived neurotrophic factor in PD, the initial studies using AAV2-neurturin in PD patients, the lessons learned from these studies and the future directions of this therapy.
Expert opinion: Gene therapy has emerged as a potential breakthrough in the treatment of PD and early clinical trials using AAV2-neurturin, a trophic factor that has shown the ability to protect dopaminergic degeneration in preclinical PD models, are underway. While trophic protection of dopamine neurons would be a significant breakthrough, PD remains a widespread disorder that involves neurodegeneration across multiple cellular types. We believe that these initial studies with AAV2-neurturin are significant steps toward the realization of gene delivery of trophic factors as a viable therapy, though the ultimate goal must be that of comprehensive neurorestoration.