Defect causing Huntington disease may have been corrected in a patient for the first time according to BBC.
An experimental drug injected into the spinal fluid tend to lower the toxic protein in the brain.
A research team, at university college London says there is now hope that the deadly disease can be stopped.
Experts say this could be the biggest breakthrough in neurodegenerative disease treatment in over 50 years.
It is one of the most devastating diseases.
What is Huntington?
It's an unstoppable death of brain cells, leaving patients permanently decline in movement,memory, behaviour and ability to think clearly.
Huntington generally affect people in their prime of 30s to 40s.
Patients die around 10 and 20 years of its symptom.
It is caused by error in the section of the DNA known as huntingin gene.
Normally,this section contain instructions for the production of protein called "huntingin" which is vital for brain development.
But a genetic error the protein to turn it to deadly killer of brain cell.
The treatment is designed to stop the gene.
On the trial,about 46 patients had the drug injected into the fluid to bathe the brain and spinal cord.
This was done at the "Leonard Wolfson Experimental Neurology center" at the National neurology and neurosurgery in London.
Although,there was fear that the injection could have effect of fatal meningitis.
But the first in-human test shows the drug was safe,well tolerated in patients and to large extent reduce level of huntington in the brain.
Prof John handy who was awarded the breakthrough prize for works on Alzheimer's,told BBC:"this is potentially the biggest breakthrough in neurodegenerative diseases in the past 50 years.
This sounds like a hyperbole,in a year I might be embarrassed by saying that,but that's how I feel at the moment."
The UCL scientist who was not involved in the research says same approach might be use to mitigate neurodegenerative disorders that features build-up of toxic proteins in the brain.
Having been tested and confirmed effective in stopping Huntington,it would certainly be a great step in curbing or at least reducing other neurodegenerative disorders.
An experimental drug injected into the spinal fluid tend to lower the toxic protein in the brain.
A research team, at university college London says there is now hope that the deadly disease can be stopped.
Experts say this could be the biggest breakthrough in neurodegenerative disease treatment in over 50 years.
It is one of the most devastating diseases.
What is Huntington?
It's an unstoppable death of brain cells, leaving patients permanently decline in movement,memory, behaviour and ability to think clearly.
Huntington generally affect people in their prime of 30s to 40s.
Patients die around 10 and 20 years of its symptom.
It is caused by error in the section of the DNA known as huntingin gene.
Normally,this section contain instructions for the production of protein called "huntingin" which is vital for brain development.
But a genetic error the protein to turn it to deadly killer of brain cell.
The treatment is designed to stop the gene.
On the trial,about 46 patients had the drug injected into the fluid to bathe the brain and spinal cord.
This was done at the "Leonard Wolfson Experimental Neurology center" at the National neurology and neurosurgery in London.
Although,there was fear that the injection could have effect of fatal meningitis.
But the first in-human test shows the drug was safe,well tolerated in patients and to large extent reduce level of huntington in the brain.
Prof John handy who was awarded the breakthrough prize for works on Alzheimer's,told BBC:"this is potentially the biggest breakthrough in neurodegenerative diseases in the past 50 years.
This sounds like a hyperbole,in a year I might be embarrassed by saying that,but that's how I feel at the moment."
The UCL scientist who was not involved in the research says same approach might be use to mitigate neurodegenerative disorders that features build-up of toxic proteins in the brain.
Having been tested and confirmed effective in stopping Huntington,it would certainly be a great step in curbing or at least reducing other neurodegenerative disorders.
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