Gene therapy to treat Huntingtons slows progression by 75%

THIS is the way to use GMO technology, not for messing about with our food supply!



From this article - Huntington's disease successfully treated for first time

One of the cruellest and most devastating diseases – Huntington's – has been successfully treated for the first time, say doctors.
The disease runs through families, relentlessly kills brain cells and resembles a combination of dementia, Parkinson's and motor neurone disease.
An emotional research team became tearful as they described how data shows the disease was slowed by 75% in patients.

Someone mentioned this to me yesterday.

from UCL

“Trial results come through in numbers and graphs, but behind each datapoint is an incredible patient who volunteered to undergo major neurosurgery to be treated with the first gene therapy we’ve ever tested in Huntington’s disease. That is an extraordinary act of bravery for the benefit of humanity.

“My patients in the trial are stable over time in a way I’m not used to seeing in Huntington’s disease – and one of them is my only medically-retired Huntington’s disease patient who has been able to go back to work.”
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AMT-130 is a gene therapy that permanently introduces new functional DNA into a person’s cells. It consists of particles of a harmless, empty virus, plus a set of instructions encoded in custom-made DNA.
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The AMT-130 DNA becomes a permanent addition to the neuron. It contains a set of instructions for making a molecule of RNA which has been designed to bind to the RNA that is produced when a cell is making the huntingtin protein. When AMT-130 RNA binds to the cell’s own huntingtin RNA, it summons an enzyme to destroy it. As a result, the huntingtin message is deleted and less of the protein is made – permanently.

Sounds a much better use of the science to me too. Huntingtons is a horrible disease.

Hopefully the early results will prove to continue to protect these people indefinitely!

The comment about the cost of managing the disease would be huge is valid. Expensive as the gene therapy is, the cost to families without the treatment is daunting.