A promising small study, which took place over six months, is raising big hopes for scientists who are devoted to helping Parkinsons Disease patients. It involves an experimental gene therapy that is injected into the brain. Researchers say it less characteristic tremor, stiffness as well as other movement symptoms.
According to Michael Kaplitt, MD, PhD, who developed the gene therapy, this is the first successful clinical trial that compares a gene-based treatment to other treatments in Parkinsons or any neurological disorder. Dr. Kaplitt is vice president for research in the department of neurological surgery at Weill Cornell Medical College in New York City and a co-founder of the company Neurologix, which is developing the treatment and funded the study.
In gene therapy, the genes are transferred into targeted cells via harmless viruses. Other gene-based treatments have shown promise for the treatment of Parkinsons, but those studies were conducted using primates. Previous trials that went onto to treat humans didnt show any benefits.
Forty-five patients who had moderate to advanced Parkinsons symptoms that were not adequately controlled with conventional treatments took part in the study. They were treated at seven different medical centers throughout the United States. Half got the gene therapy, which involved the infusion of genetic material directly into a key brain region through tiny holes drilled in the brain. The other half were treated with a placebo.
