23rd February 2012 6 – 8:30 University College London
Roger Barker of the Cambridge Centre for Brain Repair set the context for the meeting by playfully challenging the speakers to come up with a better treatment regime than the present armoury of drugs and Deep Brain Stimulation. The question of whether they did was never resolved but what followed was a stimulating and tantalising glimpse of the leading edges of cell and growth factor research.
Sonja Kriks of the Memorial Sloan-Kettering Cancer Center New York took us through the process of creating Dopaminergic Neurons from human embryo stem cells. I can not summarise the complex process but
a) using embryonic cells gets around the objections to foetal cell treatments.
b) Embryo-derived cells were relatively easy to culture in large numbers
c) The survival rates for these cells when introduced into the brain were very high. They established themselves quickly and survived in sufficient numbers to be effective.
d) So far there had been no rejection or side effects in mice, rats and a monkey
It looks like they will be applying to start clinical trials soon
Alan Whone from Bristol then took us through the state of play on GDNF (growth factor) treatments where a nourishing and restorative fluid is piped in the areas of the brain affected by PD. After promising in itial results in the USA and in Bristol some years ago, clinical trials were halted after a group of monkeys paralleling the trials began to develop problems caused by the delivery method of a pump in the abdomen delivering a steady stream of GDNF via a thin tube into the striatum. The group in Bristol are planning a new trial where the GDNF is delivered via a slow infusion through several fine tubes connected to a port set, like some hearing aids into the bone behind the ear and in size and appearance resembling an earphone socket on an iPod. This avoids the need for an abdominal pump. The GDNF is administered every few week via the port. The team were also looking at using bone marrow (which is itself a source of GDNF) as a delivery medium via intravenous injection.
It was pointed out that GDNF, though very effective also encouraged the growth of Serotonin-ptoducers and that this could cause Dyskinesias to worsen.
Professor Anders Bjorklund from Lund in Sweden gave 2 talks: the first was on Cell Replacement by transplanting foetal brain cells into patients. This had been tried and discontinued in the 1990s and had not been judged a success at the time, as the cells did not appear to affect symptoms. However a later follow up on the patients showed that some their UPDRS scores began to improve after 3-5 years possibly because the foetal cells needed to mature and needed time to take on a role in the brain. So transplanting had not been a failure, it just needed more time to be allowed before passing judgement.
His second talk was on NURR1 a protein that plays a major role as a transcription factor, regulating the way in which Dopaminergic and other cells develop. This seems an exciting and productive field, since NURR1 has a key role in producing new dopaminergic cells, it also regulates GDNF and functions as a neuroprotective agent.
We never did test out Roger Barker’s challenge from the start of the evening – none of the speakers had the magic bullet or could give a date for their discoveries to produce widespread improvements in treatments but I left feeling very optimistic. There was a real sense of discovery and progress in the meeting. I was also impressed by the way the speakers reacted to each other, seeing immediately what uses they could make of the work being described. Could it be that the answer is out there, but it needs several people to come together with their pieces of the jigsaw puzzle?
very interesting thanks for making the effort.
Many thanks Dewey. This is all very positive and optimistic.
I would only be really happy when all the wonderful research step out of the Lab
and the science is applied to individuals. At the moment, what real chance have we got to really slow down or push back for good this monster of a condition
ever changing from day to day if not hour to hour sometimes? a very slim one!
Sorry everyone, I do not feel optimistic but awful.
Roger Barker I believe will be the pioneer in a major break through within 18 months. Yes I am a bit biased as he's been my consultant since my diagnosis 4 years ago. But he's honest and sincere. You may say its blind faith but if cells have been regenerated to restore sight, there is no reason why we shouldn't all be a little confident in our futures. I am on many studies, and even in the past few years it seems that people are secretly excited, again it might be me who's reading hope when there's not. But we have to hope, cell therapy is the future not only for us but also for the medical profession as a whole, fingers (toes, arms, legs, teeth!!) crossed.
The tremendous commitment I have seen among the Research Projects funded by Parkinson's UK is one of my reasons for optimism.
Speaking of optimism, I should have mentioned that this particular LRMN meeting was sponsored by the Cure Parkinson's Trust, run by Tom Isaacs - one of the most optimistic people I have come across.