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Parkinson's disease gene therapy shows promise

6 September 2004
By BioNews
Appeared in BioNews 274

The first patient to receive experimental gene therapy treatment for Parkinson's disease is healthy, and has seen an improvement in his symptoms. Fifty-five year-old Nathan Klein, one of 12 people enrolled on a trial taking place at New-York Presbyterian Hospital, received the treatment a year ago. Doctors injected a virus carrying a therapeutic gene into Klein's brain in a effort to halt the progression of the disease. A further six patients have since undergone the treatment, and the gene therapy does not appear to have triggered side effects in any of them.

Klein claims to have experienced an improvement of 40-60 per cent in overall symptoms when taking low doses of medication, and a 10-20 per cent when he is not. To carry out the treatment, the scientists cut through Klein's skull to gain access to the region of the brain that controls movement. The symptoms of Parkinson's disease - uncontrollable shaking, rigidity of the limbs, slowness of movement and impaired balance and co-ordination - are caused by a gradual loss of dopamine-producing brain cells in this area of the brain.

Existing treatments for Parkinson's disease involve replacing the dopamine, a chemical that controls brain activity. But in the new trial, the doctors did not want to target dopamine production directly, as they thought many patients might already be resistant to its effects. Instead, the team injected a gene called GAD (glutamic acid decarboxylase) into Klein's brain, in order to trigger production of a protein called GABA (gamma-aminobutyric acid). It was hoped that this brain chemical would then 'quiet' the over-activity of the brain that causes the unwanted body movements. The results of phase I of the trial suggest the treatment is safe and effective, say the team, who published their findings in the journal Chemistry and Industry.

UK Parkinson's disease expert Roger Barker, who was not involved in the study, said that the lack of adverse effects is good news, but since Klein is still on low doses of medication, it is not yet possible to say whether the gene therapy was better than existing treatments for the condition. Phase II of the trial is set to begin at the start of 2005.

First Parkinson's Gene Therapy Patient Passes One Year
Newswise |  2 September 2004
Gene therapy 'cure' for Parkinson's sufferers
Daily Express |  4 September 2004
25 June 2007 - by Ailsa Stevens 
A US study, published in the journal The Lancet last week, reported that all twelve Parkinson's patients who took part in the world's first gene therapy trial for brain disease improved markedly without experiencing side-effects. Under the care of Dr Michael Kaplitt and colleagues of the New...
19 April 2007 - by Dr Jess Buxton 
US researchers have reported encouraging initial results from a trial of a new type of gene therapy treatment for Parkinson's disease (PD). The Phase 1 clinical trial, designed to test the safety and effectiveness of the approach, involved 12 patients with advanced PD. After a year...
30 January 2006 - by BioNews 
Gene therapy could help rescue a promising treatment for Parkinson's disease (PD) shelved over safety fears, New Scientist magazine reports. The drug, glial-derived neurotrophic factor (GDNF), was withdrawn in September 2004 - just five months after it had been approved for use in patients. The biotech company that made the treatment...
26 August 2003 - by BioNews 
A US man affected by Parkinson's disease became the first patient to receive experimental gene therapy treatment for his illness last week. Fifty-five year-old Nathan Klein is one of 12 people enrolled on a new trial taking place at New-York Presbyterian Hospital, in which doctors will inject a therapeutic gene...
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