Anonymous stem cell scientist frankly answers questions about the field

Of course I have to ask you about the recent U.S. court ruling essentially declaring all federal funding of human ES cell work illegal. What’s your take on this?

ANSWER: The ruling has so many flaws in it. It is purely political and the judge was crass. The legal wangling is nauseating.

And the impact?

ANSWER: For the moment, it is disastrous. Many are not getting the NIH funding that they were counting on to continue their studies. The research is screeching to a halt. Hundreds of people will lose their jobs. Enormous effort will be wasted. It’s painful. I am sure that some people will simply quit doing hES cell research completely and others who were considering it, will not start. The freeze is chilling the field, which maybe was the judge’s intention. The question is whether this is a transient icy period or an ice age. When will this be resolved? I’m an optimist by nature, but I’m discouraged at this point. Beyond us researchers, more important than the impact on us, this decision is a slap in the face to millions of patients and their families. The judge dashed their hopes.

What’s going to continue even during this “freeze”?

ANSWER: You have to remember that there is a trap of being too ethno-centric in science and viewing everything as revolving around the U.S….so there is a tremendous amount of outstanding hES cell research going on internationally that will continue unabated. Focusing on the U.S. alone…. Privately-funded research will go on as will CIRM-funded research such as the work in your lab…..CIRM will keep the ball rolling and I hate to think where we’d be without CIRM right now, but we need more overall. If there is no quick resolution, at least 100-200 hES cell projects around the country will completely end. That’s a catastrophe. More generally, I think it will discourage private companies from doing hES cell work too.

In that regard, what do you think of Geron’s news recently about their Phase I clinical trial for hESC?

ANSWER: Honestly, it’s both exciting and terrifying to me after all this time to see the first trial move forward. If there are negative outcomes, we will all be in the soup.

You mean teratoma?

ANSWER: Correct, or some other serious, unexpected problem. Phase I trials are unpredictable as you go from rodent to human. And there are so many enemies of hES cells out there waiting and hoping for bad news…and they’ve won a significant, if temporary victory. They may be fired up and I expect more lawsuits.

As more hES cell clinical trials start over the next couple years, the risk goes up because all it takes is one piece of bad news from one of them to ignite a firestorm that maybe torches them all. It is a paradox though because more clinical trials are essential. ACT and then others will get theirs going. These trials are crucial, but we are all kind of holding our breath. Especially with the ban of NIH-funded research, we need some good news from industry.

Beyond hESC and as we navigate the current hESC ban, what do you think about the prospects of iPS cells for getting into the clinic? We just recently saw a paper on apparently effective iPS cell derived neurons for treating a Parkinson’s like disease in rats. I was encouraged by it.

ANSWER: I’m excited, but we do not want to get ahead of ourselves with iPS cells. We have to be extraordinarily careful with the current technology. I personally would not conduct a clinical trial with the current generation of iPS cells and I think the FDA would be difficult to convince at the pre-clinical stage. At the moment at least, the power of iPS cells is matched by potential problems such as teratoma and more malignant tumors.  So caution should rule…cautious excitement if that makes sense.

Can you see any solutions to these hurdles?

ANSWER: Absolutely…potential ones at least. But they are not easy fixes as you well know.  It is very possible that we need a fundamentally different methodology that makes iPS cells, one that creates iPS cells that are at least no more tumorigenic than hES cells are.

Where do you see the iPS field going and where will it be say in a year?

ANSWER: Methodology and Disease Modeling will dominate over the next twelve months with a surge of interesting pre-clinical studies.  I do not personally believe we will see a good non-genetic method by the end of 2010 that has clinical relevance. I hope I’m mistaken on that, but I doubt it very much. I think it will be next year or even further down the road before we see a legit totally non-genetic method, but I have a bad feeling we’ll get some methods published that claim they are bona fide non-genetic, but turn out not to be….Let’s see, otherwise the flood of disease modeling papers will continue.  Overall I do not see iPS as a field being much further at the end of this year than we are today. But, I hope 2011 will be the big blowout year when their clinical potential becomes more realized. I’m excited, but after all these years I’m patient and I worry… a lot.

What’s your perception of the iPS cell field?

ANSWER: The iPS cell field is moving so fast and furiously. In some ways it’s too much a race between different groups. Some races in science have produced good things over the years and move fields forward more quickly, but there is a big risk with speed, especially when you are talking about things from a pre-clinical and clinical perspective.

Some see it as an international battle between U.S. and non-U.S. researchers, but I’d prefer to think of us as more united than that. In any case, there are big bumps ahead and those we have to somehow traverse.

What about the role of industry in iPS cell research?

ANSWER: I think companies like iPieirian are interesting and there are others.  Industry was betting more on hES cells, but we shall see what with the ban…..Unfortunately there’s likely to be some patent tug of wars on iPS cell technology. ACT recently has been putting out aggressive press releases about their IP position in the iPS cells field, which is very surprising to me. What do they have? Since they didn’t publish anything in this area, we will have to wait and see. Many iPS cell researchers have filed patent applications, but I think only Yamanaka’s has been granted…that’s a patent in Japan. You’ll see this snowballing in terms of the number of patent applications. I firmly believe that Yamanaka deserves the patents.

How about big pharma and stem cells more generally?

ANSWER: They are jumping in in some cases, cautiously watching in others. You’ll probably see some buyouts of the small companies. For example, if Geron or ACT has a successful trial, they could very well be swallowed up.

What do you think of the stem cell field’s prospects more generally?

ANSWER: Despite my angst at the moment, I’m very optimistic overall. You can just feel the momentum and energy at meetings. I think iPS cells have helped reinvigorate people and generated a lot of excitement….even if I’m skeptical of their clinical use in the near future. We will get through this temporary NIH problem and hES cell research will get back in gear. And of course adult stem cell research is going well too. My perspective is that we will need the 3 big cannons….adult stem cell, ES cell, and iPS cell research…to help all the people out there with various conditions. If you eliminate one of them such as ES cells, you eliminate your best hope for many diseases.