Entries in Gene Therapy (1)


Gene Therapy Is Successful as Parkinson's Treatment, Study Says

Digital Vision/Thinkstock(COLUMBUS, Ohio) -- There is no magic bullet to cure diseases like Parkinson's. Certain therapies like deep brain stimulation and dopamine drugs are not often successful. But a new study published in Lancet Neurology provides results from a successful phase II gene therapy trial in patients suffering from Parkinson’s disease.

A hallmark of Parkinson’s disease is uncontrolled tremors and movement impairment due to abnormal brain circuitry. In the study, the authors use a sophisticated approach to inject genetic material into the brain cells responsible for motor functions in an attempt to correct the abnormal functioning.

Forty-five patients aged 30-75 years with moderate to advanced Parkinson’s disease were enrolled in the trial, with half of them receiving the gene therapy and the other half getting a “sham surgery." The patients were then tested after six months for their motor score which was based on speech, tremor at rest and facial expressions.

In the results of the trial, researchers found that patients receiving gene therapy had a 23.1-percent improvement in their motor score as compared to 12.7-percent improvement in the control group. Based on these results, the authors conclude that gene therapy can be further developed as a potential treatment for Parkinson’s disease. This is a first-ever successful phase II clinical trial of a gene therapy for Parkinson’s or any other neurological disorder. The phase I trial was published in 2007.

Unlike ceep brain stimulation which involves the implantation of a medical device called a brain pacemaker, gene therapy does not involve any electrical variables. However, it still remains to be seen if the improvements with this therapy are long-lasting.

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