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Alzheimer's gene therapy treatment enters phase II trial

28 September 2009

By Dr Jay Stone

Appeared in BioNews 527

Dr R. Scott Turner and his team of the Memory Disorders Program at Georgetown University, US, have begun recruiting patients to take part in a gene therapy trial, which hopes to test whether gene therapy using the nerve growth factor (NGF) gene could be used to slow the progression of Alzheimer's disease.

Alzheimer's disease is the most common cause of dementia, affecting around 417,000 people in the UK alone. Proteins in the brains of affected people begin to stick together and form what are termed 'plaques', which in turn interfere with cell function and eventually lead to cell death. At the beginning stages of the disease, patients can often feel they are having lapses of memory or simply having trouble finding the right words, but as the disease progresses the symptoms worsen and it is eventually fatal.

The new gene therapy treatment, called CERE-110, consists of the genetic information for a protein called 'nerve growth factor' (NGF), which is a survival factor for the neuronal cells in the brain. Once injected into the affected areas of the patient's brain, it is hoped that it will help protect the cells so they can continue to function properly, thus slowing the progression of the disease.

Fifty patients classified as having a mild form of Alzheimer's disease - and so competent to consent for themselves - will be eligible for the trial. All participants will undergo the neurosurgery but only half of the patients will actually receive the CERE-110 treatment, with the other half getting a non-effective placebo. The study is sponsored by the Alzheimer's Disease Cooperative Study (ADCS) through a grant from the National Institute on Aging (a part of the NIH, National Institutes of Health) in association with Ceregene, Inc.

SOURCES & REFERENCES | 22 September 2009
ScienceDaily | 23 September 2009


07 September 2015 - by Chris Hardy 
An experimental gene therapy used in patients with Alzheimer's disease appears to slow down neural degeneration....
15 October 2012 - by Dr Nicola Davis 
Up to three experimental drugs will be trialled to determine if they can be used to prevent the development of Alzheimer's disease...
11 April 2011 - by Alison Cranage 
International scientists including researchers at Cardiff University, UK and the University of Pennsylvania, USA have discovered five genetic variations associated with Alzheimer's disease. The findings are published in two papers in the journal Nature Genetics...
27 September 2010 - by Alison Cranage 
US scientists have identified a gene which they suggest is associated with Alzheimer's and could help uncover the causes of the disease....
05 July 2010 - by Chris Chatterton 
Stories about a genetic test to see if you would live to 100 abounded in the UK press last week. Was this hype or something more? The stories arose following the publication of a paper in Science where researchers claimed to have identified regions of the genome linked to exceptional longevity...

25 April 2005 - by BioNews 
Six patients taking part in a gene therapy trial for Alzheimer's disease are showing an improvement in their condition, a US team reports. Scientists at the University of California, San Diego (UCSD) have shown that injecting genetically modified skin cells into the brain appears to slow the effects of the...
24 September 2004 - by BioNews 
The first patient in a gene therapy trial for Alzheimer's disease, 63-year-old Ron Shellady, was treated recently at Rush University Medical Centre in Chicago, US. The two-year study will assess the safety of the technique, on 6-12 participants with a mild to moderate form of the disease. The...
07 July 2004 - by BioNews 
Performing gene therapy on mice can prevent the symptoms of a hereditary brain disease called spinocerebellar ataxia (SCA). This discovery, made by a research team from the University of Iowa, US, has prompted hopes of similar treatments for related brain disorders such as Huntington's and Alzheimer's. Unlike other gene therapy...

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