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Postmortem findings hint at Alzheimer's gene therapy effect

07 September 2015

By Chris Hardy

Appeared in BioNews 818

An experimental gene therapy used in patients with Alzheimer's disease appears to slow down neural degeneration.

The preliminary findings are based on the post-mortem brains of patients with Alzheimer's disease who participated in a phase I clinical trial to test the safety of the treatment, running between 2001 and 2012 at the UC San Diego. The study was designed to assess whether nerve growth factor (NGF) – a protein that occurs naturally in our bodies and is essential for cell growth, maintenance and survival – might safely slow or reduce the spread of brain-cell death in Alzheimer's disease.

The results confirm earlier findings that none of the patients suffered adverse effects, but also showed that all the patients had responded to treatment, suggesting NGF had been taken up by the brain cells. Degenerating neurons showed signs of axonal sprouting (an indication of neuronal repair and recovery) in all ten of the Alzheimer's disease brains studied. This was true even in brains that had been given the gene therapy ten years previously.

'All of the Alzheimer's disease brains showed anatomical evidence of a growth response to the growth factor,' said the principal investigator, Professor Mark Tuszynski, of the UC San Diego Translational Neuroscience Institute and the VA Medical Center, San Diego. 'This means that growth factors as a class consistently result in activation of dying cells in human neurodegenerative disorders.'

Eight of the patients in the study had ex-vivo gene therapy, where a skin biopsy was taken from each patient and genetically modified so that the cells expressed NGF, before being injected directly into the brains of the patients.

The remaining two patients had in-vivo therapy, where a modified virus carrying NGF was injected into the brain, where it altered the cells so that they too expressed NGF. Both strategies involved injections straight into the brain because NGF is too big to cross the blood-brain barrier; an injection elsewhere in the body would not have affected the brain.

Phase II trials testing the efficacy of the treatment are currently ongoing, and results from these trials will be crucial. It remains to be seen whether the effects seen in the brain have any effect on disease symptoms such as memory loss and mental function.

Results of the study were published in JAMA Neurology.


17 October 2016 - by Annabel Slater 
A new gene therapy has slowed the progress of early Alzheimer's disease in mice...
11 July 2016 - by Sarah Gregory 
Researchers looking at multiple genes have developed risk scores that could identify those most likely to develop Alzheimer's disease in later life...
11 April 2016 - by Rebecca Carr 
A gene therapy for children with a rare but life-threatening genetic disorder that severely weakens the immune system has been recommended for approval by the European Medicines Agency...
11 April 2016 - by Dr Barbara Kramarz 
A gene called FOXF2 has been linked to small vessel disease in the brain, a condition associated with a higher risk of stroke and dementia...
07 December 2015 - by Chris Hardy 
Scientists have identified a link between the product of the BRCA1 gene, variants which can cause breast and ovarian cancer, and Alzheimer's disease...

26 August 2014 - by Claire Downes 
Researchers have identified a connection between DNA methylation and Alzheimer's disease, gaining a further understanding into the underlying causes of this neurodegenerative condition....
18 August 2014 - by Chris Hardy 
A man without a working copy of a gene thought to be necessary for healthy brain function - and linked with Alzheimer's disease - has a 'grossly normal cognitive status'. Targeting this gene in people at-risk for the disease could help to protect them against it...
28 September 2009 - by Dr Jay Stone 
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...
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...
17 April 2001 - by BioNews 
Doctors at the University of California, San Diego, have surgically implanted genetically modified cells into the brain of a patient suffering from Alzheimer's disease. A 60-year old woman was the first person to have gene therapy to combat the effects of the disease, in a procedure that involved drilling...

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