Memory loss has been reversed in mice with Alzheimer's disease following gene therapy.
A study led by brothers Professor Lars Ittner and Dr Arne Ittner of the Macquarie University Dementia Research Centre in Sydney, Australia, has shown that the gene therapy not only halts the progression of memory loss, but it can also reverse the effects when applied to mice with advanced Alzheimer's disease.
Discussing the findings, Professor Ittner said: 'We were completely surprised. They actually recovered their memory function and their ability to learn returned. So, two months after we treated the mice at very old ages, these mice suddenly behaved like their normal siblings.'
By introducing genetic material into the cells of affected mice, the researchers were able to activate an enzyme known as p38gamma. Previous research by the team revealed that this enzyme, when activated, is protective against the development of Alzheimer's disease. Their latest research builds on this, using gene therapy to enhance the activity of p38gamma in mice with established memory loss.
Results from the study suggest that this gene therapy may be useful in treating other forms of dementia, such as frontotemporal dementia, which typically affects a younger population. As there were no adverse events reported in the mice, even those treated with high doses over a longer period, the team are planning to trial the therapy in humans.
'There is no comparable therapy out there and no other gene therapy either,' said Professor Ittner. 'This provides hope, as there is a lot of therapy out there focussed on prevention, but not much for those already affected by the disease.'
'It will be exciting to see how over ten years of basic research to understand the mechanisms of Alzheimer's disease will finally transition into clinical development to eventually benefit those most in need, people living with dementia' he added.
The study was published in the journal Acta Neuropathologica.