Hey, I'm not happy about it either. But if you have a small placebo group and that group just happens to show a slow rate of progression (every ALS patient progresses differently), then it's tough to show great statistics in an overall study. There will be some patients who undoubtedly were great responders, but others simply weren't responders and they haven't nailed down the differentiating factor between responders and non responders. You really should go watch the video and you can decide for yourself. $CUR got killed for similar results....I haven't followed them lately so I don't know if they are still proceeding w/ their stem cell therapy in ALS or not.
Unfortunately, I don’t think the big catalyst event I was hoping from the US Trials is going to materialize. If you watch the Alliance for Regenerative Medicine presentation and Q&A, you can very easily pick up on the caution in Chaim’s message. Variability between ALS progression, NTF differentiation and response, placebo effect all make deriving statistically meaningful efficacy end points very difficult. The company acknowledges the need to try to identify responder sub-groups (sounds like very much like Neuralstem which fell off a cliff btw). They are funded through this year, but no doubt about it they will need to raise additional funds as they attempt a large cross-site multi-dose trial. Expect them to release other indications this year as well, but those will most likely be for the purpose of PR…any trials would again need additional funding. I don’t see enough upside here to wait it out for the next several years.