A gene strongly associated with Alzheimer’s disease has been neutralised in human brain cells for the first time.
Scientists have ‘turned off’ a protein associated with the apoE4 gene, which damages nerve cells, leading to dementia.
The researchers hope the finding could pave the way for a treatment that halts the disease but add therapies that are successful in the laboratory or on animals often fail in patients.
Having one copy of the apoE4 gene doubles a person’s risk of Alzheimer’s, while two copies increase an individual’s risk by 12 times. Around one in four people carry apoE4.
Alzheimer’s disease affects approximately 5.5 million people in the US.
Turning off gene could halt Alzheimer’s
The researchers, from the biomedical research organisation Gladstones Institutes in San Francisco, analysed nerve cells donated by Alzheimer’s patients with two copies of apoE4.
These cells were compared against those that did not produce the gene.
Results suggest the presence of apoE4 causes brain damage, which could be avoided by neutralising the gene.
The compound that turns off the protein behind apoE4 has only been tested on cells in the laboratory to date, with researchers working to improve the substance so it can be tested on patients.
Lead author Yadong Huang, said: ‘Drug development for Alzheimer’s disease has been largely a disappointment over the last 10 years.
‘Many drugs work, beautifully in a mouse model, but so far they’ve all failed in clinical trials.
‘One concern within the field has been how poorly these mouse models really mimic human disease.’
The findings were published in the journal Nature Medicine.
Alzheimer’s will be as manageable as HIV within 10 years
This comes after award-winning scientists predicted Alzheimer’s will be as manageable as HIV within 10 years.
Future dementia treatments will be taken before the condition develops to prevent symptoms rather than attempting to reverse them, according to Professor Michel Goedert, who was involved in discovering the importance of protein plaques in Alzheimer’s onset.
Professor Goedert, from the University of Cambridge, added: ‘Alzheimer’s will become something like HIV.
‘It’s still there but it has been contained or whittled down by drug treatments.
‘It will disappear as a major problem from society.’
Professor Goedert believes drugs under investigation for Alzheimer’s often fail due to them being taken too late into the disease’s progression.
His colleague Professor Bart De Strooper of University College London, with whom he shares the four-million euro Brain Prize money, added: ‘The mistakes we have made is the trials is that treatment has been given too late.
‘It’s like popping a statin to stop a heart attack.
‘But when we first started we knew almost nothing about Alzheimer’s and now we understand a huge amount.
‘In 10 years we will have a completely different picture.’
The scientists have been credited for changing the way doctors approach Alzheimer’s disease and other age-related brain disorders.
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