I’ve been taking a look both at your posts here and related papers.
Having read the paper in detail, I’m afraid that the results are not as positive as outlined by Dr Schneider in his press release. http://bit.ly/Z4CjHO
Let me explain. Essentially, the study had three phases. First phase
In the first, half of the participants were treated with GM1 and the remainder with placebo.
The symptoms of the GM1 treated group improved significantly (as assessed by a standard clinical scoring system) while the placebo group continued to deteriorate, as did a 'comparison' group who also did not receive treatment. Second phase – delayed start
After six months, the placebo group were changed over to GM1 – this was the 'delayed start' component of the trial or the second phase. While their condition improved, this was not to the same extent as those who had been on GM1 from the start.
This suggests either of two things:
• Firstly, the GM1 may have had a neuroprotective effect to slow down progression and because of this, those who were started on the drug later had a less profound effect.
• However, it could also be argued that GM1 has a purely symptomatic effect and that it is more effective when given at an earlier stage.Third phase- ‘wash-out’ phase
The key phase however is the third one when the GM1 treatment was stopped in both groups after 120 weeks. This is referred to as the 'wash out' phase.
If the GM1 was protective, although the patients patient’s symptoms would deteriorate as the condition progresses, those who received GM1 from the start should continue to progress at a slower rate than those who only started the GM1 after six months. This was not the case.
After a further year off treatment, the conditions of both groups were equal so the both groups had deteriorated to the same level. In addition, although the comparison (non-treated) group were not followed after 96 weeks, an extrapolation of the rate of deterioration suggests that they would have been at the same clinical stage as those who had stopped receiving GM1 treatment.
This provides strong evidence that the effect of the drug was primarily symptomatic rather than protective. Unfortunately, the researcher did not refer to the wash-out phase in his press release.
The authors published a paper in the same journal in 2010 where they reported the results of an earlier five year study (http://to.ly/hNFv
). Again the results were ambiguous and they could not draw any definite conclusions as to whether the effect of the drug is neuroprotective or symptomatic. In fact, they suggested that it was quite likely that GM1 was acting to make the remaining nerve cells more efficient.
You can be assured that we monitor all clinical research papers very closely and would be the first to report a study that shows a genuine neuroprotective effect. However, we do not wish to give false hope and unfortunately the results from this trial were not sufficiently robust to be considered as being neuroprotective.
I hope that this answers your questions, but please do not hesitate to contact me if you require further information.
Dr Kieran Breen
Director of Research and Innovation