Recent uncertainty about whether federal funds can be used for for human embryonic stem cell research has “cast a cold chill” through the field, says Collins.

Question: How is the NIH dealing with a recent court injunction against using federal funds for embryonic stem cell research?

Francis Collins:  The injunction that insisted that federal funds could not be used for human embryonic stem cell research came as a great surprise since it was based on a new interpretation of legislative language that’s been in place for 14 years.  It absolutely cast a cold chill through the field of human embryonic stem cell research with investigators who had banked a lot of their career in working in this area suddenly questioning whether they had a career at all.  This resulted here in our intramural program, scientists who work here on this campus in Bethesda, having to close down experiments immediately.  Individuals working in universities and medical centers who had grants from NIH were able to continue with the funds they’d already received, but were not going to be able to get renewed the next time they came back for an annual renewal under the conditions of this injunction.  

And we had other grants that had already been through the first level of peer review and were poised to get funded that had to be shelved because of the terms of the injunction.  I’m pleased and reassured and encouraged to say that that has over the course of two-and-a-half very tumultuous weeks now resulted in a stay of that injunction from the Court of Appeals.  But it’s a temporary stay and we don’t know in the longer term where this might go.  

That means that the field continues to be feeling very whipsawed and uncertain about its future.  I’ve spoken to many scientists involved in human embryonic stem cell research who are quite troubled about this and who, in many instances are questioning whether this is an area that they can continue to work without feeling as if their whole program could be pulled out from under them by another event of this sort.  

The promise of human embryonic stem cell research remains somewhat uncertain.  This is a very new field, but it is potentially one of the most exciting developments in many years and to have this after many years of being slowed down by this kind of intense scrutiny and oversight, and now maybe even stopped, is troubling indeed.  And it will certainly lead to a higher proportion of this work going on in other countries in which ought to be a concern to anybody who is worried about the American leadership in science and the economic consequences that flow from that.  It’s a very uncomfortable and unstable situation right now.

Question: What are some of the main bioethical issues with this kind of stem cell research?

Francis Collins: Well first of all, I think the bioethical considerations about deriving stem cell lines have been worked out.  These are derived from excess embryos that are obtained during in vitro fertilization and that are otherwise doomed to be discarded, and if they’re derived with full consent of the donor couple and with no financial inducements, then the general consensus is that is a legitimate way to have these lines generated and that once generated they can be used for federal funding.  That’s what the Obama Executive Order said back in March of 2009.  So that part of the process I think has been well traveled.  

Perhaps you are also asking though about the use of such stem cell lines when it comes to therapeutics.  And that really falls into the same kind of discussion you have whenever you are contemplating a new therapy that hasn’t really been done on humans before, what is the likelihood of effectiveness and what is the risk of danger of a bad outcome?  Those things, of course, have been getting debated now for several years with protocols that are proposed in the private sector and of course, the first one of those, the Geron trial for using human embryonic stem cells for spinal cord injury patients has been approved by the FDA and is about to enroll its first patients.  

So there are uncertainties there, there was a concern and there probably will be for quite a long time until we have more actual experience that human embryonic stem cells could in fact grow into tumors.  They are pluripotent which means they can make all different cell types and one of the ways you test to see if a cell line is pluripotent is that it’s capable of making tumors in an immune deficient mouse.  So that potential is there and great care has to be taken to be sure you have minimized that risk.  

But I see this very much in the long line of experiences of radically new therapies that reached the point that is it arguable appropriate to begin to try this and very carefully to find human situations with close oversight and a very rigorous informed consent so that those that are involved in the research know exactly what they are getting into and have a chance to say no if they don’t think it’s a good balance of benefits and risks.

Recorded September 13, 2010
Interviewed by David Hirschman