Gene therapy treats Parkinson’s tremors
Researchers have used gene therapy to correct movement problems in macaque monkeys with Parkinson’s symptoms. The problem with current treatments for Parkinson’s disease is that the drugs lose effectiveness over time, and can cause serious side effects.
The team used a virus to introduce three genes into the brains of monkeys wih a form of Parkinson's. These genes are involved in the production of dopamine, a neurotransmitter responsible for controlling movement and the brain’s motor system. The three genes have been introduced into animals separately in the past, but never together.
Using specially designed probes to measure the dopamine levels in the brain, the team monitored the monkeys for up to three and a half years. Dopamine levels were restored to about half normal concentrations in the brain and the monkeys showed improved movement problems without any severe adverse side effects.
It is stressed that this therapy is only a treatment, not a cure, as it only targets one of the symptoms of Parkinson’s disease – the tremor and movement problems. But after this successful trial an early stage human clinical trial using this technique is underway. Once researchers find the optimal dose, they plan to move to larger phase II trials.