A latest study has shown that gene therapy may be successful in treating the dreaded brain degenerative disease – Parkinson’s disease. Neurologists are hopeful that it could be useful in other neurologic disorders too.
This was a phase 2 trial where the therapy gene was given to PD patients. This gene helped improve motor scores in patients with advanced stages of Parkinson’s disease. PD is characterized by tremors and muscle rigidity. Lead author Andrew Feigin, associate professor of neurology and molecular medicine at The Feinstein Institute for Medical Research, Manhasset, New York and his team conclude, “The use of somatic-cell gene transfer to alter gene expression in well-characterized brain neurochemical systems offers a novel alternative to conventional pharmacological or surgical treatment.” Their findings are published online March 17 in Lancet Neurology. The study was funded by Neurologix.
The study population was divided into therapy group (22 patients) and placebo or dummy surgery group (23 patients). Motor scores fell from baseline in both groups at the 1-month assessment, but the decrease in the treatment group was greater. After six months scores in the gene therapy group improved by 8.1 points on the UPDRS motor score while the sham-operated group improved by 4.7 points. Investigators had defined a clinically meaningful change in UPDRS motor score as an improvement of 9.0 points. Eight of the 16 patients in the treatment group, but only 3 of the 21 sham-operated patients, showed this level of improvement 6 months after treatment.
Study researcher Dr. Michael Kaplitt, vice chairman for research in the Department of Neurological Surgery at Weill Cornell Medical College in New York said this study, “brings us much closer to having a gene therapy that might be ready for general use.” He added, “I think we are now helping to facilitate and to accelerate the development of a whole host of gene therapies … for diseases such as Alzheimer's disease, epilepsy [and] depression.”
Dr. William Weiner, a professor of neurology at the University of Maryland Medical Center, who studies Parkinson's disease and was not involved in the study said, “It's important that there was a change, but it was a very small change,” said. The idea that inserting a single gene is the answer for neurodegenerative diseases is probably simplistic, Weiner added. He also notes that gene therapy does not cure the disease, it only treats the symptoms. The findings still need to be confirmed in a larger group of patients, since this study involved just 22 individuals, and it remains to be seen how long the benefits last.
Parkinson's UK welcomed the study, but said further research was needed.
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