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First Parkinson's Gene Therapy Patient Passes One Year
September 02, 2004The first ever patient to have undergone gene therapy for Parkinson's appears to have come through phase I without a hitch, suggesting that the therapy is safe and effective, reports Marina Murphy in Chemistry & Industry.
'We have yet to analyse efficacy data, with the seventh of the planned twelve subjects only just having undergone gene transfer. . . But there have been no adverse effects related to the gene therapy so far,' said lead researcher Matthew During, of the University of Auckland. He continues, 'Our therapy is extremely safe and we hope there will be some symptomatic improvement.'
It is just over a year since the first patient, Nathan Klein, had a virus carrying a gene injected in a part of his brain. He claims to have experienced an improvement of 40-60% in overall symptoms when he is on his medication, and a 10-20% improvement when he is not. Prior to the surgery, he habitually suffered from a tremor on his right side.
Roger Barker, a Parkinson's expert at the University of Cambridge, who was critical of the decision to go ahead with the trial, said the fact that there has been no adverse effects is good news, but because the patient is on low doses of his medication, it is not possible to determine whether the gene therapy is any better than a more aggressive drug regime, or subthalamic nucleus (STN) deep brain stimulation.
The treatment uses a harmless virus to introduce a gene into a part of the brain that is overactive in Parkinson's patients, causing jerky movements. Introducing this gene leads to the production natural chemicals that inhibit the overactive brain cells. During's group is the only one conducting human gene therapy trials in Parkinson's and experts think that gene therapy is 20 years ahead of stem-cell therapy for Parkinson's disease.
Phase II should start at the beginning of 2005.
Society of Chemical Industry
Roger Barker, a Parkinson's expert at the University of Cambridge, who was critical of the decision to go ahead with the trial, said the fact that there has been no adverse effects is good news, but because the patient is on low doses of his medication, it is not possible to determine whether the gene therapy is any better than a more aggressive drug regime, or subthalamic nucleus (STN) deep brain stimulation.
The treatment uses a harmless virus to introduce a gene into a part of the brain that is overactive in Parkinson's patients, causing jerky movements. Introducing this gene leads to the production natural chemicals that inhibit the overactive brain cells. During's group is the only one conducting human gene therapy trials in Parkinson's and experts think that gene therapy is 20 years ahead of stem-cell therapy for Parkinson's disease.
Phase II should start at the beginning of 2005.
Society of Chemical Industry
Gene Therapy Current Events and Gene Therapy News RSSResearch reveals lipids' unexpected role in triggering death of brain cells
The lipid that accumulates in brain cells of individuals with an inherited enzyme disorder also drives the cell death that is a hallmark of the disease, according to new research led by St. Jude Children's Research Hospital investigators.
No-entry zones for AIDS virus
The AIDS virus inserts its genetic material into the genome of the infected cell. Scientists of the German Cancer Research Center have now shown for the first time that the virus almost entirely spares particular sites in the human genetic material in this process. This finding may be useful for developing new, specific AIDS drugs.
Cornell researchers identify a weak link in cancer cell armor
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Treatment to improve degenerating muscle gains strength
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Iowa State University researcher discovers key to vital DNA, protein interaction
A researcher at Iowa State University has discovered how a group of proteins from plant pathogenic bacteria interact with DNA in the plant cell, opening up the possibility for what the scientist calls a "cascade of advances."
Scientists successfully reprogram blood cells
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Immune therapy can protect against or treat later lymphoma
Specially developed immune system cells that target the common Epstein-Barr virus can protect immune-suppressed bone marrow transplant recipients against lymph system disease and cancers that arise from the viral infection.
Caltech researchers show efficacy of gene therapy in mouse models of Huntington's disease
Researchers at the California Institute of Technology (Caltech) have shown that a highly specific intrabody (an antibody fragment that works against a target inside a cell) is capable of stalling the development of Huntington's disease in a variety of mouse models.
Immunotherapy demonstrates long-term success in treating lymphoma
Targeted immunotherapy has been an attractive new therapeutic area for a number of cancers because it has the potential to destroy tumor cells without damaging surrounding normal tissue. New study results demonstrate high success rates using specialized white blood cells to prevent or treat lymphoma associated with the Epstein-Barr virus (EBV-lymphoma) in patients who have received a hematopoietic stem cell transplant (HSCT).
Toward bold new anti-cancer medicines
Bold new strategies in the battle against cancer may turn forms of the disease that presently are incurable into manageable conditions that can be controlled for long periods of time.
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