Dementia drug potential hailed
Key points
- A drug aimed at helping people with a rare illness could hold promise for patients with Alzheimer's disease, says a UK scientist.
- A team from the Royal Free Hospital in North London, led by Professor Mark Pepys, think that the reason the body cannot break down the plaques is because another body protein, called SAP, coats the amyloid and disguises it from the immune system.
- In addition, the drug showed no signs of being toxic to mice, and doctors were able to move straight to tests on a small number of human patients.
- Professor Pepys told BBC News Online that these preliminary results were "encouraging" - but would have to be repeated on far more patients.
A drug aimed at helping people with a rare illness could hold promise for patients with Alzheimer's disease, says a UK scientist.
An international team including scientists from London and Southampton believes the discovery has a chance of providing an effective treatment for this incurable brain disease.
A number of diseases - including Alzheimer's - are characterised by the formation of "plaques", fibrous deposits of protein called amyloid around cells in the affected organ.
Even though these amyloid plaques are unnatural, the body seems unable to dispose of them, and they progressively affect more and more tissue.
It is strongly suspected that in Alzheimer's, the plaques may kill brain cells, playing a key role in the progression of dementia.
Breakthrough
A team from the Royal Free Hospital in North London, led by Professor Mark Pepys, think that the reason the body cannot break down the plaques is because another body protein, called SAP, coats the amyloid and disguises it from the immune system.
If this SAP could be removed, researchers suggested, perhaps the body could start to remove the amyloid, perhaps halting or even reversing the disease.
Now they have found a drug which removes SAP from the body.
It is designed to latch onto more than one SAP molecule, and then be recognised and extracted from the blood by the liver.
In mouse experiments, not only does it radically reduce the amount of SAP circulating in the bloodstream, but also appears to lead to a reduction in the overall amount of amyloid.
Safe in humans
In addition, the drug showed no signs of being toxic to mice, and doctors were able to move straight to tests on a small number of human patients.
"We don't have to get the drug into the brain, which is a great advantage"
Professor Mark Pepys, Royal Free Hospital, London
These all had an illness called systemic amyloidosis - in which plaques form and gradually disrupt various organs in the body, including, in some cases, the heart and kidneys.
These eventually cause the organ to fail, with devastating consequences to the patient.
Again, no significant side effects appeared, and blood levels of SAP fell.
More importantly, the condition of most of the patients did not worsen while they were on the drug.
Brain access
Professor Pepys told BBC News Online that these preliminary results were "encouraging" - but would have to be repeated on far more patients.
Written and revised by
Andrey Nailor, M.D.
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