World Stem Cell Summit
The Summit was very well attended, and is unique among Stem Cell meetings, in that it was much more “patient centric” than any of the other meetings I have attended. Most of the meetings are either scientists presenting for scientists, or scientists presenting for investors. Bernie Seigel, whose Genetic Policy Institute sponsored and ran the meeting, is more interested in educating the public, and in particular the various patient advocacy groups whose lives are affected by the diseases. So that education is really the theme of his meeting. Since the meeting was in Wisconsin, the home of Embryonic (and now iPS cell) technology, the meeting was also meant to be a show case for Dr. James Thompson and the entire Wisconsin Stem Cell initiative, and included a very visible and enthusiastic Wisconsin Governor Doyle.
(For those not familiar, iPS induced pluripotent stemcells, are basically skin cells which are turned “back” in time to something like embryonic stem cells by the introduction of a series of genes).
I was more than a little surprised then, when the “tenor” of my panel became somewhat adversarial. The theme from the research community seemed to be “we don’t know enough about what causes these (CNS) diseases, so we think that all resources should go to basic research to try to fill these “gaps” (I am putting it nicely). I heard one esteemed gentlemen on the panel say that anyone doing otherwise was putting the cart before the horse; and another (based on research he did in 1994) state that we don’t know enough about the cells we are contemplating using, and we should wait for much more research on that front before proceeding.
You may imagine some surprise then, when I presented and announced that Neuralstem would be doing a human trial for ALS, and that we had completed all of the pre clinical work; that we were polishing up the IND application, and would be filing our IND with the FDA in the next several weeks. There was even further surprise when I announced that Eva Feldman of Michigan and the Taubman Institute, one of the World’s leading ALS clinicians and researchers, would be the Principle Investigator for the trial, and that the surgeries would be done at Emory University, under the guidance of Dr. John Glass, the director of the ALS Clinic at Emory who will act as our site principle investigator. John is also a world renown ALS clinician and researcher. Finally, when I also recognized the contribution of (equally renown and respected) ALS clinicans from Columbia, Hershey Medical Center, The Mayo Clinic; and an equally well respected team of surgeons who helped pull together our surgical protocol, there was, as the writers like to say, an awkward silence. While it is possible that none of the researchers on the panel had ever heard of Neuralstem, and were quite ready to write us off as not a “serious” science effort, the truth was of course, quite the opposite, and you could see it dawn on them. And I can report to you now quite confidently that not only are we doing world class science at Neuralstem, but clearly world leading as well. None of the other stem cell technologies are even remotely in the same “place” that we are with respect to moving the technology into humans for a disease of the spinal cord. This is not however, a good thing for society at large. In my mind the lack of progress in these other technologies is a function of some very real problems which need to be overcome.
In brief, the embryonic stem cell technology, which clearly can create motor neurons, is still simply too unstable for human use. And while Dr. Thompson (generally credited with discovering Embryonic stem cells) spoke eloquently on the potential of his new iPS cell technology, he also acknowledged that it would be at least three to five years before they really knew if these cells could do all the same things as embryonic stem cells (and if they have the same flaws), and then at least another five to ten years after that before potential therapeutics could be designed. In the meantime he proffered, they will make excellent research tools in the lab.
When the presentation period was over, and the panel “discussion” period began, Mark Noble (a charming man, brilliant researcher and sort of elderly statesman from Rochester University) posited that a trial like ours, in view of the gap in knowledge about what causes the disease, could serve no purpose. “We know” he said..”what will happen in such a trial, some patients will have a good result, some will not, and we will have learned nothing, because we don’t know yet what causes the disease”. I responded by saying that if our trial results in “some patients” having a good result, that would be a remarkably positive thing, as there currently is nothing that ALS patients can avail themselves of, which gives any positive results; and that I was convinced that every patient and patient’s family member in the room (a very large number) would agree with me.
There were other things said, (I suspect I shall not be invited back) of the same tenor. Perhaps no statement more clearly distinguishes the difference between industry and academia than his. Each needs the other I believe, and I believe that it is healthy to have each pushing the other. Each has its proper role; academia making sure that industry doesn’t cut corners or do shoddy research just to “get” to a product sooner; and industry pushing and prodding and constantly reminding academia that there are patients out there on the other end of all of this who don’t have the luxury of time.
Finally, there was also a report on the many fraudulent “clinics” around the world that are taking advantage of the desperation of families searching for something, anything, for their loved ones. One man, who had traveled the world with his son after his son had been diagnosed with ALS, had documented in great detail these evil places; and appeared to be on a one man crusade to try to bring their practices to light for the vast patient populations upon whom they prey. It was impossible not to be moved. I have written on this subject before, and hope to be of some help to him in his efforts.
For those of you who are interested, I will post his web site information after getting his approval to do so.