A cure may be on the horizon for Parkinson’s as scientists have found an experimental drug can slow down its progression and stop symptoms.
NLY01 has been called ‘amazingly protective’ as preliminary trials showed it blocked brain cells from dying.
Parkinson’s strikes 120,000 patients in the UK and one million in the US. It gradually destroys cells in the part of the brain that controls movement.
It can cause muscle stiffness, slow movement, tremors, sleep disturbance, chronic fatigue, an impaired quality of life and severe disability.
Johns Hopkins University researchers, who led the trial of NLY01, expect the drug to proceed to clinical trials on humans later this year.
NLY01 has been called ‘amazingly protective’ as preliminary trials by Johns Hopkins University researchers showed it blocked brain cells from dying (stock)
If successful, it will become one of the first drugs to directly target the progression of Parkinson’s, rather than just blocking its symptoms.
Professor Ted Dawson, who led the three-part study on mice and human brain tissue, said: ‘It [NLY01] is amazingly protective of target nerve cells.’
Celebrity sufferers Michael J Fox and Billy Connolly have increased the awareness of Parkinson’s, which claimed the life of boxer Muhammad Ali.
But there is currently no cure and no way of stopping the progression. The new study, published in Nature Medicine, offers some hope.
NLY01 binds to glucagon-like peptide-1 receptors on the surface of certain cells. Animal studies have showed these drugs have a protective effect on brain cells
Similar drugs, such as liraglutide, are already used in treating type 2 diabetes to increase insulin levels in the blood.
Professor Dawson and colleagues tested NLY01 on three major cell types in the human brain called astrocytes, microglia and neurons.
WHAT IS PARKINSON’S?
Currently, about 127,000 people in the UK have Parkinson’s, while figures suggest there are around one million sufferers in the US.
It causes muscle stiffness, slowness of movement, tremors, sleep disturbance, chronic fatigue, an impaired quality of life and can lead to severe disability.
It is a progressive neurological condition that destroys cells in the part of the brain that controls movement.
Sufferers are known to have diminished supplies of dopamine because nerve cells that make it have died.
There is currently no cure and no way of stopping the progression of the disease, but the new findings offer hope.
They speculated it may stop astrocytes – star-shaped cells that help neurons communicate – from being turned aggressive and killing neurons.
Astrocytes can be ‘activated’ when microglia secrete chemical signals.
But in trials on lab-grown human brain cells, researchers treated microglia with NLY01 and found they were able to turn the activating signals off.
In another trial, mice were injected with alpha-synuclein, the protein known to drive Parkinson’s disease, and treated them with NLY01.
Another set of mice given the injections but not the drug showed motor impairment over the course of six months in behavioural tests.
However, mice treated with NLY01 maintained normal physical function and had no loss of dopamine neurons – suggesting it protected them.
A third experiment found the drug can also extend the lives of mice with Parkinson’s by around 120 days – a 31 per cent boost.
Dr Dawson cautioned that NLY01 is merely experimental and must still be tested for its safety and effectiveness in humans.
However, because of the safety profile of other similar drugs, he does not anticipate any major roadblocks in terms of side effects.
Dr Beckie Port, research manager at Parkinson’s UK, said: ‘This is promising news, particularly as existing research supports the potential of diabetes drugs that work in the same way as NLY01.
‘Up to now, most research has focused on existing diabetes drugs, like exenatide, to treat Parkinson’s, but NLY01 may have some advantages over these: it may be able to penetrate the brain and reach the brain cells affected by Parkinson’s more easily, which could make it more effective.
‘We eagerly anticipate the next stage for this drug, clinical trials, to show whether it is truly able to slow the progression of Parkinson’s – something no current treatment can do.’