Some Days You Just Don't Want To Enter That Maze Ever Again

Yesterday was one.

It's easy to be lured into that maze entrance with announcements and press releases and "promising" approaches.  Been there. Done that.  We always seem to get lost inside.

The Big Biomarker Trial Announcement

Boy, did that ever sound good.  Three million dollars.  Two trials. "Promising" biomarkers.  Walk right into that maze and see what a wonderful destination we find.  Whoa.  Wait a minute.  I really don't want to enter that maze if it ends up anything like our last Phase 2 trial of NP001. 

My understanding is that the $3 million in grants announced yesterday were specifically for professional CRO services from NEALS for Phase 2 trials.  That's certainly not chopped liver, but it's not flexible like cash.  Do those kinds of grants mean that investigators and drug developers and organizations will not be tempted to challenge the old Phase 2 model? 

And now we come to NP001 itself.  We already learned from Dr. McGrath's comments at the big ALS symposium in December that high LPS PALS were the responders in the old Phase 2 trial in 2011.  And we also know from experiences of PALS in that trial that some PALS experienced improvements and then had terrible crashes during the washout period where the LPS biomarker was tracked.  And no NP001 was made available to any of those trial volunteers after that. 

Will this trial be designed more humanely? 

And what will we have at the end of another long Phase 2 trial?  More certainty on biomarkers but still nothing accessible for PALS?  Will Neuraltus still be passing the tin cup to investors to come up with the $30-$35 million dollars they say they need for a Phase 3 trial? 

Urgency, anyone? 

Or are we just expected to enter the maze again and see where it takes us? 

And if anyone is worried about the dangers of GM604 if granted FDA Accelerated Approval, we can let them talk to the widows and orphans of the brave NP001 trial volunteers about safety and risk and the old approval process. 

The ALS Registry Annual Meeting

And we had an opportunity to step into yet another maze yesterday afternoon.  Per @CDCEnvironment the ALS Registry Annual Meeting will not be webcast this year because of budget limitations.  That's certainly a huge step backwards in transparency in a project that people care about.  It's also not fair to those who advocate for tens of millions of dollars for that project.  We step into another maze when we have to wait for a written report on the meeting that is published far after the fact.  We get lost when we don't see the interaction and questions and answers and project leadership dynamically. 

And we also learned yesterday from @CDCEnvironment that the annual report of Registry results that we was promised every year after the initial report last summer will be delayed.  "The 2nd & 3rd report (i.e. 2012-2013) will be combined into 1 report. Therefore, it will be released in early 2016."  Oh, it's like we just took a bad turn in the maze again. 

The shortest distance between two points isn't in a maze.   Transparency and challenges to the status quo are healthy, and you just get lost when you walk right into that adjective-laden door.