Very interesting - thanks for sharing that. This is not the first targeted therapy that has been a bust but, IMO, targeted therapy will be the 4th treatment choice, in the future, once they finally unlock the secrets to it.

The advantage of the type of whitepaper data, published by the researcher, from a regulatory perspective, would enable them to get a "fast track" with the FDA for an NDA (New Drug Approval). This is the fastest way to conduct the different tracks to validate safety and efficacy. It is typically only granted for treatment of life threatening or incurable diseases. Double blind studies would be part of the data required by the FDA in the first phase of testing. The FDA actually has to ok the clinical trial protocols, is proactive in them and frequently changes them based on preliminary test results, data, etc. This is also the reason that some drugs are removed from the market after their approval by the FDA. Typically it takes about 10 years for a new drug to get FDA approval, if not on the fast track, and even longer for OTC drugs.

It may appear to look insidious that the researcher moved to Germany but in reality this happens quite often as the EU is less stringent on testing on human subjects. Quite a few clinical trials are done in Europe. Our litigious society stymies human trials very often in the US. Besides, nobody wants the placebo!

Also, most drugs (and their indications for use) are found by "accident" anyway. It is very bold to target a specific condition.

I don't really pay a lot of attention to preliminary data based on animal studies, which most of the raw data is based on. It does make for sensational headlines on slow news days (and is a welcome departure from politics ;-)