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New Gene Therapy May Help Parkinson’s Patients

Posted in Drug Studies, Public Health

On Thursday, January 9th 2014, MedlinePlus reported that gene therapy may be helpful to patients suffering from advanced-stage Parkinson’s disease, as demonstrated by new research.

According to the researchers, this therapy, “called ProSavin, works by reprogramming brain cells to produce dopamine, the chemical essential for controlling movement”. (MedlinePlus)

This research comes from an English company, Oxford BioMedica, where lead researcher Kyriacos Mitrophanous is quoted, stating “We demonstrated that we are able to safely administer genes into the brain of patients and make dopamine, the missing agent in Parkinson’s patients”.

Patients with Parkinson’s, a disease characterized by insufficient dopamine in the brain, suffer deceased muscle control which may begin as a slight tremor, but “also commonly causes stiffness or slowing of movement.” (Mayo Clinic)

“‘The ProSavin study was a positive and important first step for a potential gene therapy for Parkinson’s disease,’ said Dr. Michael Okun, national medical director at the National Parkinson Foundation. ‘The results of this preliminary study revealed a promising safety profile, and it will be interesting to observe longer-term benefits and how ProSavin will compare to other therapies such as deep brain stimulation.’” (MedlinePlus)

While ProSavin has not yet proven itself more beneficial than levodopa, the mainstay in dopamine therapy for Parkinson’s disease, or deep brain stimulation, a technique for boosting dopamine production using electrical stimulation with wires and an external battery pack, gene therapy carries at least one theoretical advantage.

According to Mitrophanous, as the disease progresses over time, patients require more and more medication.  With gene therapy, the body is “tricked,” if you will, into creating the dopamine it needs itself.

“Patients injected with ProSavin had mild to moderate side effects. The most common while on medication were involuntary movements (dyskinesias) and switching between mobility and immobility, called on-off phenomena, which occurs as levodopa wears off.”  However, “All patients showed significant improvements in motor scores in the 12 hours after they stopped taking their other medications and at six months and a year after surgery, the researchers found.” (MedlinePlus)