Huntington’s disease trial test is ‘major advance’ –
Ajit
Patel Sanda Wellness
Scientists say they may have found
the world’s first blood
test that predicts when someone at risk is likely to get Huntington’s disease
and tracks how quickly damage to the brain occurs.
The study, in the Lancet Neurology,
suggests the prototype test could help in the hunt for new treatments.
Huntington’s
disease is an inherited and incurable brain
disorder that is currently fatal.
‘Disease
speedometer’
Around 10,00 people in the UK have
the condition and around 25,000 are at risk.
It is passed on through genes,
and children who inherit a faulty gene from parents have a 50% chance of
getting the disease
in later life.
People can develop a range of
problems including involuntary movements,
personality changes and altered behaviour and may be fully dependent
on carers towards the end of their lives.
In this study, an international
team – including researchers from University College London – looked at 200
people with genes for Huntington’s
disease – some of whom already had signs of the disease, and others at earlier
stages.
They compared them to some 100 people
who were not at risk
of getting the condition.
Volunteers
had several tests over three years, including brain scans and clinical check-ups
to see how Huntington’s
disease affected people’s thinking skills and movement as the condition became
more severe.
At the same time scientists looked
for clues in blood
samples – measuring a substance called neurofilament light chain (NFL) – released
from damaged brain
cells.
They found levels of the brain
protein were high in people with Huntington’s disease and
were even elevated in people who carried the gene for Huntington’s
disease but were many years away from showing any symptoms.
And researchers found NFL levels rose
as the condition worsened
and as people’s brains shrank over time.
Halt progress
Dr Edward Wild, at UCL, said:
“Neurofilament light chain has the potential
to serve as a speedometer in Huntington’s
disease, since a single blood test reflects how quickly the brain
is changing.
“We have been trying to identify blood biomarkers to help track the
progression of Huntington’s disease for well over a decade and this is the best
candidate we have seen so far.”
Researchers suggest it could be more rapid
and cheaper than current methods of measuring the progress of the disease, such
as invasive tests of spinal fluid and brain scans.
And they say the blood
test could be particularly helpful when checking if new treatments show any signs
of being able halt the progress
of the condition.
Commenting in the Lancet Neurology,
Prof Christopher Ross and Prof Jee Bang of John Hopkins University described
the study as “remarkable”.
They added: “The study represents a
major advance in the field of Huntington’s
disease and neurodegeneration in general…”
But they cautioned that it was important
to carry out further, larger trials to confirm the results.
Scientists working on the original
study agreed that further experiments
were needed to fully understand the pros and cons of the test, before it could
be of any help to patients.
They have launched a larger trial.
Cath Stanley, chief executive of Hungtington’s
Disease Association, said: “This is a ground breaking piece of research that
takes nearer to having a better understanding about Huntington’s
disease.”
No comments:
Post a Comment