Last week at a conference, I believe that I heard their CEO say that they were entering the final stage of their Phase I trial now and that they positioned the FDA to allow 2 operations a month instead of the one allowed now. I also believe that he said that they were seeking to set-up a second surgical team at a second site in anticipation of moving forward with the program.

Honestly Fafut_1 - they didn't speak about efficacy at the presentation I went to recently. The are only reporting safety of administration of the cells at this point. They want to increase the number of cells delivered per injection as well, as the current FDA protocol has a limit on it, so either they think they are having an effect and want to enhance it or they aren't seeing the effect they desire and think that dosing is going to make a difference....but I think that its all speculative right now as I don't think they gave an exact reason (nor did i have the thought in my head at the time to ask).

I would imagine that we will likely see results reported from at least the initial cohort of PALS by the end of this year. Right now, there is little ability to see where the cells went and what they did in a person after they get them implantation. Now, if we saw improvement, one might say "who cares where they went and what they did!", and i might be one of those people for awhile too....but then i think that we need to get some answers about what it is that they do when in the body for an extended period of time so that we can advance and science around it to have the greatest potential benefit. But right now, too soon to tell from the knowledge that i have on this matter.

Rob G, I see epw's point. It sounds like you are saying that if patients demonstrate positive results from this, or any other experimental treatment/drug, that you (and by inference the entire medical establishment)would want to know the WHy before they feel the desire to get this out to other patients.

Your statement, "...we need to get some answers about what it is that they do when in the body for an extended period of time so that we can advance and science around it to have the greatest potential benefit" is utterly unfathomable. Does it occur to any researcher that this is not an either/or proposition, that you continue to do the "WHy is this working" studies to your heart's content while simultaneously giving the treatment/drug to more patients?

Yes, science wants to know the Why, and I support that effort. But why must the lives of PALS be sacrificed on the altar of someone's odd sense of scientific purity? If a researcher's sensibility is offended by the knowledge that someone, somewhere is benefiting from a drug that the researcher has not yet figured out completely, well... I can live with that, and so might a bunch of PALS.

Rob, I undestand science need, but science has to understand patients needs as well. I somehow dont like the approach that someone who has als is already regarded dead by madical community.

Lets leave it for a while - I'll ask you just this - if the results are obvious and unmet so far (ie stopping or substantial slowing), do you still think that it has to go through whole process or it may be granted some special, really fast protocol? We all know this is last medical resort for todays pals....

ROb, thanks for the clarification. I hope that all researchers share your sentiments. I am not holding my breath on that.

I will continue to wonder why there is so much time involved in getting the data analyzed (statistics aren't done on an abacus any more) and how long it takes to analyze these results. For example, my wife (MS in statistics) was asked to check the statistical relationship between the incidence of leukemia and high voltage power poles (this was big in the late 90s). The document was lengthy and impressive. It took her about 30 seconds. She looked at a couple of the values and said, "It's garbage. There is no statistical relationship." But what about all these graphs and thick prose? "Don't matter. The results speak for themselves."

She wrote a three sentence report that was accepted by her superiors.

So I always wonder why it takes so damn long to gather the data, do the analysis, write the report, and have the report checked so we can proceed to the next step, along with full transparency of the data and the conclusions. The FDA and researchers pontificate on how careful they want to be, but in reality the efficacy of a treatment is apparent to statisticians in a matter of minutes. Not months or years. Minutes.

And why would the FDA take so damn long to decide what to do next? How many meetings, seminars, and discussions are actually required?

The writing of the paper might take a month or so, I will concede. I have 15 years experience as a technical writer for highly detailed documents, and know that every comma and period is double-checked. But you get someone to validate the data, another dealing with the stat package, and another putting it all together.

So in the clinical trial process, where is the delay point, and why is it there?