I think a big part of the problem with many of these clinical trials is the timeframe. A few people progress rapidly with PD but , on average, PD patients get about 3 points per year worse on the UPDRS part 3 (motor symptoms) rating scale. If you halt progression this means you are 3 points per year better off, but over a 6 or 12 month period this is hard to differentiate from a placebo effect.
I think the way forward is to run trials over 3 or more years - at first sight a lot more expensive, but given the money we have wasted over the past decades, perhaps worthwhile for treatments like GDNF.
The other thing we need to do is look at combinations of therapies but that is a whole other topic…
The GDNF trial had a placebo effect that was quite large which perhaps indicated the presence of an inhibitor causing the Parkinson's and that the dilution of this inhibitor by the saline used in the placebo was responsible for the large placebo effect. This would reduce the differential between the GDNF group and the placebo group.
I agree that the measures of the PD symptoms are far too crude. I was on another drug trial that started in 2019. It was abandoned when the company failed to get a vaccine contract. They reevaluated all their research programmes and dropped my trial as unpromising. I realised much later that, although I wasn’t aware of any significant changes to my symptoms, I did have worsening dyskinesia. So, if this was real ( because it was abandoned I never found out which group I was in), I could have been able to reduce my meds.
The medics have been perfectly happy to tell patients that excruciating back pain is being caused by their minds remembering the trauma and pain of a now cured injury and not by anything physiological. Why not exploit this the other way around and train our brains to support our bodies?
Why are researchers ignoring the placebo effect? If a placebo was able to improve my Parkinson’s symptoms by just 10%, I would happily take that improvement. I went to a lecture in this at the Royal Institution. It was given by Jo Marchant, who wrote a book on the subject: “Cure”.
The GDNF procedure in my opinion is too invasive and high cost to ever be a success. I was quite surprised when PUK decided to try proceed with another trial. I did ask a question at the charities AGM on the value of continuing with this in light of a similar trial with CDNF. The Finish company 'Harantis' ran a phase 1 CDNF trial to prove the safety of their product. The procedure use was the same as in the GDNF trials. It was proved successful and showed promise. However the ultimate aim of the company was to produce a product that could be administered subcutaneously. They have since identified a molecule, HER-096, from the CDNF which looks to be ideal in crossing the BBB. Phase1B trials are to be started the 2nd half of 2024. This is explained far more clearly than I can manage in the company presentation- https://herantis.videosync.fi/fy-2023-report
We can only hope that a rich investor may come forward to help involve uk patients but for some it will be too late. Could you cover the subject of brain donation to help research I’m interested in doing it when the time comes but hopefully not for a while 😉
I think a big part of the problem with many of these clinical trials is the timeframe. A few people progress rapidly with PD but , on average, PD patients get about 3 points per year worse on the UPDRS part 3 (motor symptoms) rating scale. If you halt progression this means you are 3 points per year better off, but over a 6 or 12 month period this is hard to differentiate from a placebo effect.
I think the way forward is to run trials over 3 or more years - at first sight a lot more expensive, but given the money we have wasted over the past decades, perhaps worthwhile for treatments like GDNF.
The other thing we need to do is look at combinations of therapies but that is a whole other topic…
The GDNF trial had a placebo effect that was quite large which perhaps indicated the presence of an inhibitor causing the Parkinson's and that the dilution of this inhibitor by the saline used in the placebo was responsible for the large placebo effect. This would reduce the differential between the GDNF group and the placebo group.
I agree that the measures of the PD symptoms are far too crude. I was on another drug trial that started in 2019. It was abandoned when the company failed to get a vaccine contract. They reevaluated all their research programmes and dropped my trial as unpromising. I realised much later that, although I wasn’t aware of any significant changes to my symptoms, I did have worsening dyskinesia. So, if this was real ( because it was abandoned I never found out which group I was in), I could have been able to reduce my meds.
The medics have been perfectly happy to tell patients that excruciating back pain is being caused by their minds remembering the trauma and pain of a now cured injury and not by anything physiological. Why not exploit this the other way around and train our brains to support our bodies?
Why are researchers ignoring the placebo effect? If a placebo was able to improve my Parkinson’s symptoms by just 10%, I would happily take that improvement. I went to a lecture in this at the Royal Institution. It was given by Jo Marchant, who wrote a book on the subject: “Cure”.
The GDNF procedure in my opinion is too invasive and high cost to ever be a success. I was quite surprised when PUK decided to try proceed with another trial. I did ask a question at the charities AGM on the value of continuing with this in light of a similar trial with CDNF. The Finish company 'Harantis' ran a phase 1 CDNF trial to prove the safety of their product. The procedure use was the same as in the GDNF trials. It was proved successful and showed promise. However the ultimate aim of the company was to produce a product that could be administered subcutaneously. They have since identified a molecule, HER-096, from the CDNF which looks to be ideal in crossing the BBB. Phase1B trials are to be started the 2nd half of 2024. This is explained far more clearly than I can manage in the company presentation- https://herantis.videosync.fi/fy-2023-report
We can only hope that a rich investor may come forward to help involve uk patients but for some it will be too late. Could you cover the subject of brain donation to help research I’m interested in doing it when the time comes but hopefully not for a while 😉