subject: Invisible Scalpel Vs Parkinson's Disease - Scalpel, Parkinson, Medicine - Pharmaceutical [print this page] Nervous system has been considered incurable Parkinson's disease, recently there has been a new breakthrough treatment. Weil Medical College of Cornell University, United States MichaelKaplitt professor and his colleagues first tried to gene therapy for Parkinson's disease for 11 Male And a Female Was The "Trojan horse-type" therapy pilot study, and had made significant progress. Acceptance testing of the Parkinson's patients have improved their ability to control the body has been significantly improved. Kaplitt professor discussion group the first time published the results of this study, the paper published in the prestigious medical journal "Lancet" (Lancet), on (Lancet2007; 369:2097-2105).
Encouraging CASE NathanKlein 59-year-old was suffering Parkinson disease is one of many patients. New York PortWashington Klein, a former television producer, and, like other patients, Klein had to endure frequent tremors, muscle rigidity and Sports Can not, imbalance and other symptoms and, ultimately, to the extent of life can not take care of themselves. He once complained that his life had been tortured Parkinson's disease, "chaotic." Disease Damage to his voice, so that he could not walk properly and activities, and even his hands could not even hold a cup steady.
Fortunately, in 2003, Professor Klein has become Kaplitt the first experimental group received gene therapy volunteers. Kaplitt and his colleagues, Professor target area in the brain implant a specific gene to suppress the brain's nerve impulses sent over-excited. After surgery, his symptoms were apparent ease. Now, in addition to start walking outside when the slightly trembling, Klein and other typical symptoms of Parkinson's disease have miraculously disappeared. Klein said that the effect of this incredibly excited, and now, he just took a number of routine maintenance treatments can be.
Genes are inserted, blocking the progress of Parkinson's disease
The pilot phase, Kaplitt professors were chosen 11 men and one female Parkinson's patients. Current surgical treatment of Parkinson's disease mainly through the "deep brain stimulation" operation, the completion of the one electrode placed in the brain of a specific location, so that the signal contact with the electrode to produce the corresponding stimulus program, the formation of a normal circuit loop . The experimental design professor Kaplitt targets to be achieved and surgery, as patients in order to block and inhibit the activities of signal over-active cycle. The reason is that patients with over-excited loss of the normal brain neurotransmitters?? -aminobutyric acid (GABA). Gene therapy is aimed at this mechanism to promote increased production in patients with brain GABA. Therefore, gene therapy has focused on encoding glutamic acid carboxylase (GAD) of the gene. GAD is to promote central neurotransmitter (GABA) generated enzymes, and GABA help to control the chemical signals between nerve cells. Professor
Kaplitt said that they implement the principles of gene therapy are: to have lost pathogenicity of viruses as vectors, the GAD gene into which, by the viral vectors will be delivered to patients with GAD gene target regions within the brain?? Hypothalamus the end of the nuclear (STN), and to promote the formation of GABA in the region, play a reduced excitability of nerve signals, while increasing inhibitory signals to balance the role of brain activity.
Implementation of gene therapy, using a 3-month follow-up observation for a time, until one year after surgery. After 3 months, patients generally reflected significant improvements in physical condition of the side, shaking, cramps and other symptoms ease, alleviate symptoms of implantation sites focus on the gene under the control hemi-brain side of body. According to grade the severity of Parkinson's disease, with an average improvement of 25% to 30%, some patients even recovered 65%. Preoperative medication in the treatment of patients, this gene therapy to improve the rate of 24% of the total; while patients taking the drug, gene therapy to improve the results slightly, about 27%. Patients with symptoms are more concentrated in one side of the body, because the researchers give more consideration to the safety of the test itself, will limit the scope of this study controlled side of the body, the control circuit of brain areas.
Year observation period, all patients without any serious side effects, and tremor, limb rigidity, muscle spasms, or difficulty in walking all the symptoms have been effectively improved. Kaplitt professor mentioned in the text, these 12 patients have tried all kinds of medication, but symptoms of Parkinson's disease has not been a corresponding significant improvement. He explained that the current treatment of Parkinson's disease drugs can only control the disease to some extent, but not completely alleviate the disease, and drugs can cause side effects such as transaction psychosis. This shows that gene therapy to alleviate symptoms of Parkinson disease profound, may become the focus of future research in neuroscience.
Baton of Use of gene therapy for Parkinson's disease had already set a precedent. Back in April, in a large conference had made a similar study reported that the pilot program, intends to form a gene specific input by the brain of patients, also confirmed the safety and efficacy of the foreseeable benefits. Prior to this study also shows that the brain in Parkinson's disease subthalamic nucleus activity over parts of the phenomenon appears to block signals back to the nervous system, thus impeding the body's motion control. Therefore, this gene therapy trial design is also based on previous research in this area based peers. This, Kaplitt Professor's comments: "We are only just starting, there are many large-scale testing needs to continue to complete. However, the preliminary results are very encouraging."
In the same period, "The Lancet" magazine's commentary, Vancouver, Pacific Parkinson's Disease Research Center JonStoessel professor, said the gene therapy walk in the forefront of neurodegenerative diseases. He also sent a question: Does gene therapy is certainly an original deep brain stimulation surgery is more effective and more beneficial to patients?
Kaplitt professors hope the latest end of this year on the efficacy of gene therapy as a standard clinical classification. He also pointed out that the gene therapy of the most important inherent advantages: gene therapy without surgical procedures as the original as installed in the patient's body line source, the battery of a class of foreign body infection caused by a device, implanted in the brain to avoid the risk of new power , do not worry there will be inconsistencies in electrical rhythm. Based on conclusions drawn from animal experiments, the efficacy of gene therapy can be maintained for several years. Therefore, Kaplitt Professor, if patients have the opportunity to opt-in deep
