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Do gene silencing treatments look promising?

June 25, 2016

Huntington's Disease Youth Organization

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Do gene silencing treatments look promising?

Q. My friend (30) recently found out that he carries the mutated gene, so I have been trying to do some research about potential treatment possibilities and how the future looks.. Do gene silencing treatments look promising (as of 2016)?

And at what stage would these treatments have to start, after HD has been diagnosed, or before? My friend´s dad got the diagnosis later than most people, so I´m hoping my friend also will start to get symptoms late and that treatment options are much better at that point.. I know the onset may still come early though.

Another question I have is about the psychological changes that HD patients might get. Is this because of the general loss of neurons, so that dopamine neurons get affected like in Parkinson´s?

Thanks for this website, it´s nice to have somewhere to ask questions!

Preben, Young Adult, Denmark

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A. Hi Preben

Thanks for the questions.  I think that the gene silencing therapies do look promising.  We are just testing them for the first time in humans (the IONIS trial).  I think that the biological mechanism behind the treatment is fantastic.  The tricky problems to be solved right now are how to get that mechanism into the cells (without harming anything), to make sure it’s safe (that’s what the IONIS trial is for) and, in the longer term, work out how it is going to be paid for (providing it works for humans).

The stage at which this treatment should given is a really tricky question.  The best way I have of looking at this is to think that the treatment will not bring dead cells back to life, but should help struggling cells to work better and prevent them from dying.  What this means is that treatments would probably be most effective given earlier in the illness.   Finally, regarding the psychological changes.  These are really common in HD. Some symptoms, like apathy, seem to be strongly related to the biology of HD but others, like depression and irritability are probably caused by a mixture of internal and environmental factors. The symptoms with the “mixed” causes are usually very treatable (including depression, irritability and anxiety).

With best wishes