Originally posted by humy
I don't know if it was double blind. But somehow I wouldn't think the number of them dying or showing a slower or faster cancer spread would be greatly effected by the placebo effect!
Having said that, I am still rather concerned that they didn't give specific statistics on that (I wonder "why didn't they?" ) and also their slightly vague statement of "...sho ...[text shortened]... ses..." without elaborating on exactly what they mean by that. These, to me, are not good signs.
The article says it was a phase I trial, which is typically a very small trial on healthy
volunteers to ensure the drug doesn't cause harms. They have conducted a trial on 600 cancer patients and the article implies there is no standard treatment arm, which would be normal in a phase I trial as they are looking for harms rather than efficacy. This trial is strange for a phase I trial as it is huge, normally they have around 10 volunteers, and it is on cancer patients rather than healthy volunteers. Since they do not talk about a comparison arm they appear to be comparing with baseline. Until they have done a large trial where the comparison is with a standard treatment group and at least five years follow up then there is no proof that this drug is any good.
Another point is that they are looking at surrogate measures such as tumour size. This is a technical outcome, not a clinical one. Clinical outcomes are things the patient cares about such as mortality, pain, well-being. The reason for this distinction is that a procedure's technical success is not the same as clinical success - summed up by the old quote: "The operation was a complete success - but the patient died."
What you were reading was marketing, not science. The theoretical description of the procedure is promising, but what are the adverse effects? Is the immune system permanently off the leash? Will people survive for a couple of years only to be destroyed by their own immune systems? Is it really better than standard treatment? If I'm right and there is a single arm with comparison with baseline then we don't actually know if the drug is any good. All they've shown so far is that the drug doesn't kill cancer patients quickly.