I have just caught this snippet of information on the radio. Apparently, a team of scientists at Oxford University have had good results with an injection of something called "ProSavin" directly into the brain. Obviously early days. I will try to find out more about it and let you all know. (my mind was otherwise occupied with attempting to answer the 'phone!)
Hi AB
this is, I think the same as Jims' gene therapy in Research. It is very interesting though a symptomatic treatment rather than a cure.
i may be mistook about both points!
cheers
this is, I think the same as Jims' gene therapy in Research. It is very interesting though a symptomatic treatment rather than a cure.
i may be mistook about both points!
cheers
hope you dont mind AB if I add my amateur interpretation of the news?
the genes are in a modified virus.
they are put into the striatum (the area that is most affected by the lack of dopamine from the Subs. Nigra. and which controls motor movements)
these genes produce three enzyzmes (aromatic amino acid dopa decarboxylase, tyrosine hydroxylase and GTP-cyclohydrolase 1) which turn (i think) tyrosine into dopamine.
so the infected cells start to produce dopamine just where its needed most.
what it doesnt do is (I presume)
- replace the original cells
- send dopamine to other areas of the brain sourced by the SN
- stop the destructive process
questions
- will the desctruction of cells then start in the striatum??? or does it only happen in the SN?
- can a similar technique be used for dementia? no point being able to walk if you dont know where you are going!
still a very heartening development, particularly for those near the end of the levadopa phase. hopefully there are no nasty side effects.
CORRECTIONS VERY WELCOME!!!
the genes are in a modified virus.
they are put into the striatum (the area that is most affected by the lack of dopamine from the Subs. Nigra. and which controls motor movements)
these genes produce three enzyzmes (aromatic amino acid dopa decarboxylase, tyrosine hydroxylase and GTP-cyclohydrolase 1) which turn (i think) tyrosine into dopamine.
so the infected cells start to produce dopamine just where its needed most.
what it doesnt do is (I presume)
- replace the original cells
- send dopamine to other areas of the brain sourced by the SN
- stop the destructive process
questions
- will the desctruction of cells then start in the striatum??? or does it only happen in the SN?
- can a similar technique be used for dementia? no point being able to walk if you dont know where you are going!
still a very heartening development, particularly for those near the end of the levadopa phase. hopefully there are no nasty side effects.
CORRECTIONS VERY WELCOME!!!
I saw this on Sky news last night (Ezinda had drawn our attention to it). Just 15 people have had the treatment in UK and France. The middle aged lady interviewed who had obviously had PD for a number of year said it had helped her.
Am I being over optimistic in hoping that the news broken by Sky yesterday is a major step forward. Sure there are lots of hurdles to overcome before the treatment is widely adopted not least convincing the government and the NHS to release the funds for it's use but I find it strange that it has not received a great deal of comment in this forum.
As I say maybe I'm being optimistic rather than realistic but to be it seems like a breakthrough. Your views welcomed everyone
As I say maybe I'm being optimistic rather than realistic but to be it seems like a breakthrough. Your views welcomed everyone
the following is not to be believed, quoted, relied on etc
In my slightly humble opinion, the word 'breakthrough' is rarely appropriate.
This is another treatment - possibly a very good treatment - probably similar in timing and effect to DBS but without the hardware.
Its too early to say that it doesnt have side-effects for example. If it gives people another 10 years of reasonable health then its great. I can't see people having it until after they have been through the drug option. Perhaps the most interesting thing is if the vector works so it can be used with other enzymes on other sites.
But as , i believe, the first gene treatment this is definitely a significant milestone.
Dave, what I believe this might mean for your wife is that in 10 to 12 years time when levadopa has stopped being as useful, an operation is available to give her another 10 to 12 years. And in the next 20 to 24 years time who knows what will be developed. I would think of it as a safety net to stop the worst happening.
end of unreliable b*******
In my slightly humble opinion, the word 'breakthrough' is rarely appropriate.
This is another treatment - possibly a very good treatment - probably similar in timing and effect to DBS but without the hardware.
Its too early to say that it doesnt have side-effects for example. If it gives people another 10 years of reasonable health then its great. I can't see people having it until after they have been through the drug option. Perhaps the most interesting thing is if the vector works so it can be used with other enzymes on other sites.
But as , i believe, the first gene treatment this is definitely a significant milestone.
Dave, what I believe this might mean for your wife is that in 10 to 12 years time when levadopa has stopped being as useful, an operation is available to give her another 10 to 12 years. And in the next 20 to 24 years time who knows what will be developed. I would think of it as a safety net to stop the worst happening.
end of unreliable b*******
hi everyone,i too am surprised at the lack of comments here about this, for me , well callme emotional buy i praactically burst into tears when i heard this ....it is a breakthrouggh,itacts to kkick start our brains to procuce more dopermine were its needed, what else is ther that does this,this is amazing, and im not jumping for joy at the injecting your brain bit but man, i soo need this, im36 and im at the end with leverdoper, as iv had it near on 10 years already , i cant continue like this !!!!!!!!!!!!!!!!!! so i do hope this treatment becomes availbe quickly
nicky
nicky
lets hope its available as soon as possible. are you in line for dbs?
Hi,
Just thought that those of you that like "powerpoint-logic" might like to see the following presentation on ProSavin:
http://www.oxfordbiomedica.co.uk/userfiles/file/presentations/ProSavin_Interim_Data_Analysis_December_2011.pdf
I personally quite like slides 9 and 10 (factual data
). My understanding of it is that :
1. ProSavin seems to produce a very important beneficial impact in the first 3 months of treatment. The impact is positive for all stages of PD they tested, but it seems to be particularly strong for those in advanced stage of PD.
2. The impact from month 3 to 6 is still beneficial but much smaller. By the end of month 6, the PD symptoms are around 33% better than at the start (as measured by the UPDRS scale).
3. After month 6, PD seems to start progressing again (symptoms get worse), but they seem to progress at the "normal" rate from the 33% lower point one got to in month 6. In other words, ProSavin seems to take us back a few years in our PD development path. It's impossible to tell from the data shown how many years one "goes back", but if you asked me to guess from the very little data shown, I'd venture somewhere between 2 to 5 years back (but, of course, this is very, very rough guess and might be totally wrong).
I was also encouraged by the mention in the last slide that "Enhanced construct identified; could provide more than 10-fold increase in DA production" - which suggests they might be looking at test an even better version!!
So, while none of these results seem to be fully confirmed yet (they need to run more tests), it all sounds very encouraging to me. My applause to the team working on this - I personally find it very encouraging !!!
Warm regards to all,
lfs
Just thought that those of you that like "powerpoint-logic" might like to see the following presentation on ProSavin:
http://www.oxfordbiomedica.co.uk/userfiles/file/presentations/ProSavin_Interim_Data_Analysis_December_2011.pdf
I personally quite like slides 9 and 10 (factual data
). My understanding of it is that : 1. ProSavin seems to produce a very important beneficial impact in the first 3 months of treatment. The impact is positive for all stages of PD they tested, but it seems to be particularly strong for those in advanced stage of PD.
2. The impact from month 3 to 6 is still beneficial but much smaller. By the end of month 6, the PD symptoms are around 33% better than at the start (as measured by the UPDRS scale).
3. After month 6, PD seems to start progressing again (symptoms get worse), but they seem to progress at the "normal" rate from the 33% lower point one got to in month 6. In other words, ProSavin seems to take us back a few years in our PD development path. It's impossible to tell from the data shown how many years one "goes back", but if you asked me to guess from the very little data shown, I'd venture somewhere between 2 to 5 years back (but, of course, this is very, very rough guess and might be totally wrong).
I was also encouraged by the mention in the last slide that "Enhanced construct identified; could provide more than 10-fold increase in DA production" - which suggests they might be looking at test an even better version!!
So, while none of these results seem to be fully confirmed yet (they need to run more tests), it all sounds very encouraging to me. My applause to the team working on this - I personally find it very encouraging !!!
Warm regards to all,
lfs
very interesting slides
from the info the obvious (though probably NAIVE AND WRONG) conclusions to me are
- the bigger the dose , the more neurons make dopamine
- these neurons make a fixed amount X
- the amount from the SN (Y) decreases so the total amount available is Y + X with Y decreasing at about 5% per year. you will be back to where you started from in n years where Y-Yn=X
questions - how many neurons can be activated (increasing X)
- once the mechanism for the destruction of SN neurons is understood can a similar procedures introduce an enzyme or something that can stop it or slow it down
(increasing n)
- what about about the areas of the brain outside the striatum that also receive dopamine (and produce non-motor symptoms?)
when n is greater than life expectancy, hey presto - a cure of sorts, at lest for motor functions.
from the info the obvious (though probably NAIVE AND WRONG) conclusions to me are
- the bigger the dose , the more neurons make dopamine
- these neurons make a fixed amount X
- the amount from the SN (Y) decreases so the total amount available is Y + X with Y decreasing at about 5% per year. you will be back to where you started from in n years where Y-Yn=X
questions - how many neurons can be activated (increasing X)
- once the mechanism for the destruction of SN neurons is understood can a similar procedures introduce an enzyme or something that can stop it or slow it down
(increasing n)
- what about about the areas of the brain outside the striatum that also receive dopamine (and produce non-motor symptoms?)
when n is greater than life expectancy, hey presto - a cure of sorts, at lest for motor functions.
perhaps the most interesting question is will the newly dopamine producing cells be subject to attack too - is it the location of the cells in the SN or the fact that they produce dopamine or something else that makes them vulnerable? or is it just a matter of being close enough to a diseased cell to be contaminated? it might take years for that to become clear.