Gene therapy is yet another approach to treating Parkinson's disease. A study of gene therapy in non-human primate models of PD is testing different genes and gene-delivery techniques in an effort to refine this kind of treatment. An early-phase clinical study is also testing whether using the adeno-associated virus type 2 (AAV2) to deliver the gene for a nerve growth factor called neurturin is safe for use in people with PD.

Another study is testing the safety of gene therapy using AAV to deliver a gene for human aromatic L-amino acid decarboxylase, an enzyme that helps convert levodopa to dopamine in the brain. Other investigators are testing whether gene therapy to increase the amount of glutamic acid decarboxylase, which helps produce an inhibitory neurotransmitter called GABA, might reduce the overactivity of neurons in the brain that results from lack of dopamine.

A Vaccine for Parkinson's?

Another potential approach to treating Parkinson's is to use a vaccine to modify the immune system in a way that can protect dopamine-producing neurons. One vaccine study in mice used a drug called copolymer-1 that increases the number of immune T cells that secrete anti-inflammatory cytokines and growth factors. The researchers injected copolymer-1-treated immune cells into a mouse model for PD.

The vaccine modified the behavior of supporting (glial) cells in the brain so that their responses were beneficial rather than harmful. It also reduced the amount of neurodegeneration in the mice, reduced inflammation, and increased production of nerve growth factors. Another study delivered a vaccine containing alpha-synuclein in a mouse model of PD and showed that the mice developed antibodies that reduced the accumulation of abnormal alpha-synuclein.


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While these studies are preliminary, investigators hope that similar approaches might one day be tested in humans.


The National Institute of Neurological Disorders and Stroke (NINDS) conducts and supports research on brain and nervous system disorders.