Six months ago, I published a Top 10 list of questions and predictions for the stem cell field. How did I do? A mixed bag. Below are the questions and predictions from October 2010 (in blue), along with an update as to how my predictions fared.
1) How will Geron’s Phase I Clinical Trial go? Will GRNOPC1 be safe? When will we know? Barring any major catastrophic events, it may be a year before we find out how the trial is going. Time intervals between patients and the relatively long latency of teratoma make this a slower process than all of the impatient watchers would like, but that’s just the way it goes. The pre-clinical data on 2000 rodents support the notion that this is a safe therapy so while we cannot be sure how things will go in a human context, we can be cautiously hopeful. I think the odds are better than 50-50 it will be safe, but we’ll see.
- Where do things stand now in Spring 2011 and how did I do with my prediction? Everything is still up in the air at this point with Geron’s trial. The Washington Post semi-“outed” the first patient in the trial, perhaps with his consent. The patient seems upbeat, but no details were revealed about how he is doing. We need to respect patient privacy.
2) What will happen with other potential Phase I ESC-based Trials? When will ACT’s trial get the green light? I think we will see at least one more trial begin in early-mid 2011, most likely ACT’s.
- Where do things stand now in Spring 2011 and how did I do with my prediction? I had heard through the grapevine that ACT’s trial would not start until early 2011. The good news is that I was wrong and ACT got the green light from the FDA in late 2010. As for GERN, we will have to be patient to see how the actual trial goes, but it is very exciting.
3) Where do we stand with IPS cells? I think the IPS cell field continues to surprise in a good way. We are closer (relatively speaking) to the clinic than I ever would have imagined at this point, but still years off.
- Where do things stand now in Spring 2011 and how did I do with my prediction? I think I was right that we are years away from iPS cells making to the clinic. If anything there are more concerns now than there were 6 months ago about the potential use of iPS cells in the clinic. I still hold out some hope for that however.
4) Is the new RNA-based IPS method going to be consistently as good as it seems on first glance? My guess would be that the new RNA-based approach will not be as efficient in most researchers’ hands as was reported in the recent paper simply because those guys who published it are the experts at it and have been working on it for a long time. However, I think the RNA approach may very well ultimately win the day as the best.
- Where do things stand now in Spring 2011 and how did I do with my prediction? I was right about the RNA iPS cell technique. Most researchers seem to not be able to get it to work well or at all. I’m still hopeful with some tweaks that it can in the end be the best, safest method. We’ll see….
5) What are the hurdles still ahead for IPS cells? I’d like to see more studies of IPS cell safety and tumorigenicity. Are they more prone, even when made with RNAs, to form malignant tumors than ESC? Do they form teratoma more readily than ESC? We just do not know this stuff, but it’s crucial. I think even non-genetic methods of making IPS cells may yield cells that are more tumorigenic than ESC.
- Where do things stand now in Spring 2011 and how did I do with my prediction? I was mostly right on this one. Recent studies suggest that regardless of the method used to make iPS cells that they have genetic and epigenetic alterations that might make them more tumorigenic. The tumorigenicity of iPS cells, in a clinically relevant setting, remains unaddressed.
6) What will be the next big advance in adult stem cell research/therapeutics? It’s hard to predict, but there will be some exciting developments in the next year for sure. But on the down side, it may be that it will become clearer in the coming year that unlike ESC, adult stem cells just do not possess the ability to grow actual organs and tissues.
- Where do things stand now in Spring 2011 and how did I do with my prediction? I think it is still an open question as to whether adult stem cells can make organs or tissues. Meanwhile, studies with ES cells continue to advance with organ building and seem very exciting.
7) What will happen with the pending court cases before Judge Lamberth and the DC Appeals Court? My prediction is that the ultimate ruling on this case will be given by the Appeals Court. Lamberth, if he rules, will rule against ES cell research, but he may stay his own ruling, and if he doesn’t the DC Appeals Court will do so and take over. How will the Appeals Court rule? I don’t know, but I’ll be an optimist and say in favor of ES cell research.
- Where do things stand now in Spring 2011 and how did I do with my prediction? I’m still hopeful, but I have to say I’m very surprised that we have had no ruling in the case and not even any news in the case for so long. I don’t see that as a positive or negative development, and the court still could rule negatively. Be assured the case has not disappeared and there will be a ruling…in fact it could come any time.
8. Will Congress pass a bill dealing with the D-W amendment and codify the legality of federal funding for ES cell research? No.
- Where do things stand now in Spring 2011 and how did I do with my prediction? I was right, but that was kind of a no-brainer.
9) What will be the outcome, from a stem cell research perspective, of the 2010 midterm elections? As I blogged before, I think it will be really bad. Republicans will control the House, but I’m hopeful not the Senate. We’ll have more Republican Guvs who will stir up trouble in 2011. NIH and the FDA may find themselves under a harsh magnifying glass (like one in held out in the Sun) of Extremist Freshman Congresspeople.
- Where do things stand now in Spring 2011 and how did I do with my prediction? I was right about this one too, but it didn’t take a psychic to guess that Republican control of the House was coming and would cause trouble for science.
10) Biggest surprise in the coming year for stem cells? I think someone will publish a method of reprogramming that is fundamentally different than what we’ve seen so far.
- Where do things stand now in Spring 2011 and how did I do with my prediction? So far, I am wrong about this one and I may stay wrong. I’m still hoping that RNA iPS methodology may evolve, but I’m also still awaiting some iPS method that is really outside the box.