4 ms·
I see no reason why it isn't real, unfortunately a trial of 1 does not a cure make, however it does seem very hopeful, even if there is a long way to go with mo
by SeanDav 12y ago
I see no reason why it isn't real, unfortunately a trial of 1 does not a cure make, however it does seem very hopeful, even if there is a long way to go with more extensive trials.
- ekianjo 12y agoIndeed. We see these kind of n=1 reports every now and then and media jump on the hype gun without understanding drug development at all.
- deleted 12y ago[deleted]
- dekhn 12y agoStatistically and probabilistically speaking, we can make an estimate (with error bars) of the value of the treatment, even with a trial of only a few people.
- gfodor 12y agoit was a trial of two, and the success ratio was 50%. so there is some actual statistical data there.
- adinb 12y agoTake a look at http://www.mayoclinicproceedings.org/article/S0025-6196(14)00332-2/fulltext http://www.mayoclinicproceedings.org/article/S0025-6196(14)0... and the included video. The first patient did have a recurrence of a single tumor that was treated locally, with her marrow remaining completely clear. The second patient had his marrow cleared and all but a couple tumors in his legs resolved. And the virus penetrated those tumors and allowed full 3D mapping of them due to tags attached to the virus itself (it sounded like a thyroid produced chemical was attached to the virus as a tag). The implication was that because of the detailed mapping that it would be possible to resolve/remove the remaining tumors in the leg muscles of the second patient. So, the answer is quite a bit much more complex than 50%. Both patients cleared the myeloma from their marrow. Almost all (numerous!) tumors were removed, from a single treatment in each patient. Edit: Here's a link to the editorial article that accompanies the paper: http://www.mayoclinicproceedings.org/article/S0025-6196(14)00368-1/fulltext http://www.mayoclinicproceedings.org/article/S0025-6196(14)0...
- gfodor 12y agoyeah so in that case 'a trial of 1 proves nothing' is especially misleading. this seems like very strong evidence.
- SeanDav 12y agoWhat happens if it turns out that the treatment is fatal in 30% of cases? A trial of 2 would not necessarily show this type of thing. That is why one cannot extrapolate this without much more information i.e. more trials.
- gfodor 12y agonobody is saying that more trials are unnecessary. my point is to emphasize the limited data so far is compelling, and that it goes beyond a 'trial of 1'.