Gene Therapy

There are always new treatments for Parkinson's being researched. One of the most intriguing approaches to me is gene therapy. The idea of gene therapy is to use a carrier molecule, called a vector, to insert a gene into a patient's targeted cells. This newly inserted gene then helps to create the functions that are needed in the patient's cells. For a good overview of gene therapy please visit the following website. http://www.ornl.gov/sci/techresources/Human_Genome/medicine/genetherapy.shtml#whatis

One biotechnology company, Neurologix Inc, has just been granted permission to begin a Phase II study of a gene therapy protocol to treat advanced Parkinson's Disease. Their approach is a non-dopamingeric approach to reestablishing motor function. Non-dopaminergic means that the company is not replacing dopamine in the patient's brain or working directly to increase the amount of dopamine available to the patient.

Neurologix's approach is to increase the amount of GABA (another neurotransmitter) in the patient's brain. GABA is generally known as an inhibitory neurotransmitter, meaning that it's affect in the brain is to reduce the amount that neurons fire. Dopamine loss in Parkinson's patients results in a reduced amount of GABA being released as it is dopamine in the affected area of the brain that triggers the release of GABA. The reduction in GABA in turn causes neurons to fire more frequently than normal as the inhibitory GABA is not as plentiful. The increase in neuronal firing causes motor coordination issues in Parkinson's patients. By increasing the amount of GABA in the brain directly the hope is to decrease the neuronal firing thereby decreasing the amount of motor issues. If this approach is successful it will mean that there will be another potential therapy for people with advanced Parkinson's who are no longer responding to dopamine replacement therapy.

Of course another positive to this study being successful will be gene therapy progressing to a point where it is effective in helping to treat such a complicated disease as Parkinson's.

To read more about this study please visit this website.
http://www.earthtimes.org/articles/show/neurologix-initiates-recruitment-for-phase-2-parkinsons-disease-trial,509688.shtml