Safety Trials for C9orf72 Expansion
Safety trials for treatment of the faulty C9orf72 gene are due to start in the last quarter of this year both in the USA and UK. However, I learned at the John Radcliffe yesterday that these trials are likely to be delayed well into next year for bureaucratic reasons.
Treatment will be an intrathecal injection into the spine, so that the drug gets into the cerebro-spinal fluid that bathes the brain. The trial is likely to be double blind, so one could get a placebo injected. A single shot would last 6 months or more.
The people at the John Radcliffe have no control over who is selected to take part in the trial.
The point of the trial, as the title suggests, is to provide assurance that the treatment is indeed a safe one. They told me that they are pretty sure that it will be safe.
From my point of view, I place little hope that this treatment will arrive in time to help me. I might not be selected for the trial and if I was, I might be given the placebo. Even if all that were to work in my favour, it is likely to be some years before an approved treatment results. But I intend to accept an invitation to join it, if invited.
There was clear confidence at the John Radcliffe that a cure for this particular faulty gene is in the offing and that the whole scene could look very different in say 5 years time.
This is obviously good news for our family and our descendants. It is worth noting that the likelihood of our descendants having the faulty gene reduces by a factor of two for each generation.
I spoke at length with the doctor who is leading the C9orf72 Cohort Study and have agreed to join it.
Treatment will be an intrathecal injection into the spine, so that the drug gets into the cerebro-spinal fluid that bathes the brain. The trial is likely to be double blind, so one could get a placebo injected. A single shot would last 6 months or more.
The people at the John Radcliffe have no control over who is selected to take part in the trial.
The point of the trial, as the title suggests, is to provide assurance that the treatment is indeed a safe one. They told me that they are pretty sure that it will be safe.
From my point of view, I place little hope that this treatment will arrive in time to help me. I might not be selected for the trial and if I was, I might be given the placebo. Even if all that were to work in my favour, it is likely to be some years before an approved treatment results. But I intend to accept an invitation to join it, if invited.
There was clear confidence at the John Radcliffe that a cure for this particular faulty gene is in the offing and that the whole scene could look very different in say 5 years time.
This is obviously good news for our family and our descendants. It is worth noting that the likelihood of our descendants having the faulty gene reduces by a factor of two for each generation.
I spoke at length with the doctor who is leading the C9orf72 Cohort Study and have agreed to join it.
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