New gene therapy to tackle dementia
An experimental form of gene therapy has given hope of a significant advance in the treatment of dementia.
The therapy, in which a nerve growth factor delayed the loss of brain cells, led to increased metabolic activity in the brain of Alzheimer’s sufferers and a reduction in the decline of cognitive functions.
Though the study was small, the subjects seemed to show indications of a reduction in the advancement of their disease, according to Prof Mark Tuszynski of the University of California, San Diego, the study’s principal investigator.“If these effects are borne out in larger, controlled trials, this could be a significant advance over existing therapies for Alzheimer’s disease,” he said.
Prof Tuszynski presented his preliminary findings yesterday, which suggested that brain cells in eight volunteers responded to the insertion of growth factor-producing grafts.
Patients’ own skin cells were genetically modified in culture to produce Nerve Growth Factor, NGF, a naturally occurring protein that prevents cell death and stimulates cell function.
The NGF-producing cells were then surgically implanted into a deep brain region where cell degeneration occurs in Alzheimer’s disease, affecting the “cholinergic system”, which is important in memory and cognitive function.
Using a brain-scanning method, the patients showed increased metabolic activity in those areas of the brains after treatment with NGF, compared with non-treated Alzheimer’s disease patients. An autopsy of a patient who died showed active NGF production in the brain, and a growth response of brain cells to the NGF delivery.
Alzheimer’s affects one in 20 of those aged over 65 and nearly a quarter of those over 85, causing bouts of dementia, loss of memory, personality changes and, eventually, death.
There are half a million sufferers in Britain, although some predict that there will be as many as one million sufferers within two decades.