Yes I am a doctor and I know several of the people involved with the company and the trial. Although medicine classifies diseases like cancer or neuro-degenerative diseases according to the organ they occur in or their behaviour, in fact the genetic basis may be quite diverse meaning they respond to different treatments. While it would have been amazingly good for all bowel cancer to respond well to a certain treatment, we have come to realise that some treatments for what appears to be the same disease work better in sub-classes of patients. So I never expected all of the patients to respond well to NTcell.There is no way to be sure they are suffering from exactly the same disease.
The problem with the trial results is that the placebo patients have had an unexpectedly strong response, and the 120 unit implants didn't show much efficacy at all. Also the diversity in responses within the groups was so great that the standard deviation in the small numbers was so large that even the relatively good response in the 80 unit cohort couldn't reach statistical significance.
So in order to reach a conclusion in subsequent follow up we require that the placebo response reduce, the actively treated groups to show a durable and improving response, and if possible, the response of all 4 actively treated patients to become a bit more consistent to decrease the size of the error measurement. And then the problem also becomes one of what is different about the patients that might make one patient respond when others did not, so patients who can benefit can be selected. That is what the regulatory body will want to know.
The company will look at the 52 week data for the 40 and 80 unit cohorts to see if this appears to be happening. If its not then they will make a decision about how much of the available funding to allocate to NTcell Parkinsons Disease.
They have two other alternatives that they are considering evaluating instead of PD
1. Retinal Degeneration
2. Neurogenic Hearing loss.
These should be simpler, cheaper and faster to pursue than PD.
The final strategic element is the Pericyte Protection collaboration with the Centre for Brain Research, where they hope to identify the compounds being secreted by NTcell which seem to make Neural Pericytes from PD patients and Alzheimers patients resistant to toxins. If a particular compound can be identified then it could be an attractive agent to synthesise as a drug.
In addition there is the Diabecell news which I didn't expect, but for Otsuka to have sunk tens of millions into its development and now to consider to worthy of trial is significant.
In all honesty if the 52 week results show don't suggest that NTcell has made some durable improvement in the actively treated patients, then LCT is effectively back to square one with its NTcell program, looking to find a better alternative than PD.
No-one knows if the PD trial results could show a benefit in 3 or 5 years time, but LCT can't wait that long.
However the Diabcell news is lurking in the back ground and just could be a dark horse. Otsuka is "big pharma", and if Diabecell turns out to be commercially viable then it could play a large part in LCT's future.
Bob Elliot was forthright in admitting that they set the bar too high on the trial protocol, they simply believed that NTcell would not only halt PD but reverse it sufficiently that the size of the effect would mean a small number of patients would be sufficient to show a statistically significant the result at 6 months. It was an error of judgement.
However the first treated patient will be coming up to the 4 year anniversary, when one would expect any placebo effect to have well and truly dissipated. If the improvement in the PD is still sustained, it would be further encouragement that NTcell does live up to its potential in some patients.
LCT is not going out of business, especially if the Diabecell card is turned over and turns out to be a trump card.
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