In a first successful Phase II clinical trial, funded by Neurologix, Inc, researchers used a gene therapy called NLX-P101 to reverse symptoms of Parkinson’s disease. The success of the gene therapy is the result of 20 years of research and the first to be used for Parkinson’s disease or any neurological disorder. Gene therapy normalizes chemical signals in Parkinson’s disease
The gene therapy works by normalizing chemical signals in the brain, eliminating symptoms of tremor, stiffness and difficulty moving. Typically, patients with the disease walk with a shuffling gait, finding it difficult to pick up their feet from rigidity of the limbs.
The treatment uses a gene known as glutamic acid decarboxylase (GAD). An inert virus is used to get the gene into the brain. GAD then signals brain cells to produce GABA that binds to the brain’s neurons, normalizing defects found in patients with Parkinson’s disease.
As a control, patients were given a sham therapy while they wereawake so they could respond to commands to move their limbs from the researchers, making them believe they were receiving the gene therapy. The control group was also given a saline injection instead of the gene, using the same delivery system as the therapy group.
The findings are published in the Lancet Neurology that could be a major advance for treating Parkinson’s disease, but longer trials would be needed to assess safety of the treatment.
In the study, the gene therapy dramatically improved symptoms of Parkinson’s disease in over half of the patients, compared to 14 percent of the control group at six months, with improvement seen at one month. Dr. During says the success of the Phase II trial means the NLX-P101 therapy is closer to being used in clinical practice for treatment of the debilitating neurological disorder. Neurologix, Inc., co-founded by Dr. Kaplitt, is seeking FDA approval for a Phase III clinical trial.
To learn more about How to Fight Parkinson's disease, we invite you to attend our educational conference on Monday, March 28th, 2011 at the Tampa Westshore Marriott in Tampa, Florida.
The conference is a unique educational opportunity for Parkinson’s disease (PD) patients, families, and caregivers to learn about the latest advances in PD research, medication and treatment innovations that greatly improve quality of life.
To learn more about the conference or the Parkinson Research Foundation, please visit us at http://parkinsonresearchfoundation.org/ As always please consult your physician before beginning a new health regimen. Thank you for your continued support.
References: http://www.emaxhealth.com/1020/gene-therapy-treats-parkinson%E2%80%99s-disease-second-clinical-trial
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