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Re: bladerunner1717 post# 169589

Thursday, 11/07/2013 11:24:40 PM

Thursday, November 07, 2013 11:24:40 PM

Post# of 257438
GERN - Just a closing summary of my position. Because in mix-it-up and dialogs with evolving positions like this one the point often seems to get lost or misconstrued or ... . And after this I'll leave it alone (until/unless there is new data). My three main points are:

1) GERN willingness to look squarely and openly at the data - In #msg-93839808 there is reference to CEO's stating that 'of course symptomotology will improve'. My simple response is similar to JQ's. CEO's response is literal truth since the abstract does show some (relatively small compared to Jakafi) - but my (not JQ's) larger point is that the CEO's statement is ignoring the more important truth that the data in hand indicates the improvement in clinically important QoL symptoms isn't much compared to other 'lesser' treatments like Jakafi. And that is a problem for GERN. Probably surmountable to some degree - but taking time and costing $. And ignoring or talking around the problem will make the solution take longer and cost more.

2) Will fixing "root cause" inherently beat Jakafi at symptoms? It might - but hardly obvious it must. There are plenty of cases in medicine and virtually every other field where broken things cause secondary breakage that doesn't inherently get fixed when the first is fixed. And right at this instant Imetelstat is looking like it might be such a case. And in some cases it is the secondary breakages that are more symptomatic and/or lethal. Too soon to say for sure that that is the case here with Imetelstat - but the data in hand says there is substantive risk.

3) Tafferi's abstract issues - my objection to the abstract is that it ignores the 'odd' mismatch between "solving the root cause" and little or no efficacy on the primary symptom. Which mismatch is probably the most interesting data from the trial. Doesn't really talk about the negatives. This is a little like the discussion earlier this week that even papers published in first class journals don't fairly present negatives like SAE. That said, as JQ noted on the earlier topic, it is hardly unique to any given biomedical author.

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