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Parkinson's disease gene therapy trial begins

26 August 2003
By BioNews
Appeared in BioNews 222

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 into the patient's brain in a effort to halt the progression of the disease. The US Food and Drug Administration granted permission for the trial to go ahead last year, after the US and New Zealand team published results that showed the technique worked in rats.

The team, lead by neurosurgeon Michael Kaplitt, first 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 coordination - 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 into Klein's brain, which should in turn trigger production of a protein called GABA (Gamma-Aminobutyric acid). It is hoped that this brain chemical will then 'quiet' the overactivity of the brain that causes the unwanted body movements. 'Dopamine is like the key to the car. But if you hotwire the car, you don't need the key' said Kaplitt.

Kaplitt stresses that the trial is primarily to assess the safety of the approach: 'My goal is not to try to cure Parkinson's disease' he said. 'It's to provide a better treatment that we can build on to make the next advance'.

First Parkinson's gene therapy patient keeps faith
Reuters |  20 August 2003
Genes put into brain to hinder Parkinson's
The Chicago Tribune |  19 August 2003
Gene therapy used to treat patients with Parkinson's
The New York Times |  19 August 2003
Parkinson's gene therapy study begins
Yahoo Daily News |  19 August 2003
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...
6 September 2004 - by BioNews 
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...
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