By CRG Staff

Story Landis, Ph.D., is head of the Stem Cell Task Force at the U.S. National Institutes of Health (NIH) and director of the National Institute of Neurological Disorders and Stroke (NINDS).

The stated purpose of the stem cell task force is to (advance stem cell research). Am I right to assume, based on their affiliations, that the members of the task force all have an interest in advancing the same aim?

Yes, we do. The task force was set up after President Bush, in his address in 2001, announced that NIH could conduct and fund research using embryonic stem cells, and he asked NIH to put together a task force that would accomplish those aims. So the task force has been in place since 2001; and interestingly enough, given the Obama executive order, which has completely changed the landscape for NIH funding and stem cell research. Given the advances in stem cell research and the new opportunities provided by the Executive Order, we rethink what the task force is doing and make sure that the membership reflects the new goals.

How has the task force's membership changes since 2001?

These are basically the members who were in place eight years ago. There may have been some turnover, but the same institutes are represented - those that support human embryonic stem cell research. One change that we should make would be include program staff with expertise in induced pluripotent stem cell biology.

With the last administration there were some fairly clear ethical boundaries on the use of human embryonic stem cells, and of course there's also an ethical issue in terms of the benefits that could be derived from research using stem cells. Do you find yourself or the Stem Cell Task Force dealing much with that push and pull?

The Stem Cell Task Force has focused its attention on identifying and supporting the best science. We have also been engaged in providing resources for human embryonic stem cell research including funding a stem cell bank to provide stem cells lines and training courses to teach scientists how to work with stem cells. Between 2001 and 2009, the National Academy of Sciences/Institute of Medicine and the International Society for Stem Cell Research played a key role in developing guidelines for the derivation of human embryonic stem cells and their uses. The discussions during the Bush administration about concerns about responsible derivation and responsible use either happened in the President's Council on Bioethics or, to a very large extent, were happening in the National Academy of Sciences or in the ISSER or in states like California or Connecticut or Maryland. NIH had marching orders from the President regarding what kind of stem cell research we would fund and how we would fund it.

So what are your marching orders now?

The recent executive order asked NIH to come up with guidelines that would enable us to support responsible and scientifically worthy research. Now the focus is on actually implementing those guidelines as expeditiously as we can.

 Once Obama issued the executive order, a small working group of policy experts from the NIH Office of the Director and scientists with stem cell expertise from the institutes developed draft guidelines. NIH then sought public comments on the draft guidelines. We received more that 49,000 comments and based on that input modified  the guidelines to address all the very useful input that we got. The preamble to the final guidelines which were published in August summarizes the comments and the changes that were made in response to them. One of the important changes was to create a mechanism for central NIH review of eligibility of stem cell lines and a registry that would list them. We have now created a website so that individuals or institutions can submit information so that lines can be considered for eligibility. Within a day or two of opening up the  website, complete information on 12 lines had been submitted and partial information on another 50. Each week additional requests for consideration are added. The website allows scientists and the public to check progress.

And the guidelines include limits on how embryos can be obtained and what kind of research can be funded?

The guidelines propose certain criteria - for instance, that the embryos donated for derivation of lines should be from in vitro fertilization for reproductive purposes and no longer needed for that purpose - and that the donation should be made with informed consent. While detailing the requirements for donation, the guidelines provide for the different review processes for donations before the final guidelines were released as compared to after. I think those guidelines establish clear limits on what NIH will  fund and the conditions under which embryos should be donated. And there are two very specific pieces at the end of the NIH guidelines that restrict uses of embryonic stem cells in NIH funded research.

What sense do you have of the direction of stem cell research?

I think most people have focused on using human embryonic stem cells to generate tissues for transplantation. As you probably know, the FDA approved the first clinical trial using embryonic stem cells in humans, the Geron trial for spinal injury, in January of 2009. While the public believes that cell replacement will be the first and probably most important use, many in the scientific community, including at NIH, believe that as important, and maybe even more important, is the use of human embryonic stem cells to understand human pathogenesis and also to test potential therapeutic strategies.

Human embryonic stem cell lines have been generated from embryos identified through preimplantation genetic diagnosis to carry serious disease mutations, such as Huntington's disease or cystic fibrosis. Cells from those lines can be turned into different classes of cells - for example, secretory cells for cystic fibrosis or neurons for Huntington's disease, that will allow us to understand disease pathogenesis and to develop assays to screening for treatments. So while there's a public focus on transplantation, many of us think that elucidating pathogenesis and drug screening are even more important.

For instance, we don't really know why the mutant Huntington's gene kills cells. Studies have been done in mouse models but to date the mice don't get Huntington's disease in a way that neurologists and neuropathologists would recognize. In contrast,  research on the human embryonic stem cell lines that carry the mutation can give us important  clues about what kills cells in Huntington's. And you can imagine there are other diseases where preimplantation genetic diagnosis can identify embryos carrying the disease such as lysosomal storage diseases.

Diseases where the genetic component is clear.

Yes, where the mutations in the gene are absolutely defined as causing devastating diseases, and people who choose to do in vitro fertilization so that they can be confident that the blastocysts that are implanted don't carry the disease can donate the ones that  carry the disease for research. I got a call once from a woman who knew that both she and her husband carried mutations in the cystic fibrosis gene. She wanted to know how to donate blastocysts that would be homozygous for the disease mutation. I referred her to one of the societies that deals with in vitro fertilization.

There is a recent controversy in Britain regarding the creation of animal-human "hybrid" embryos, which were first allowed and now have been abandoned. Am I right in thinking that this type of study would not be approved for NIH funding?

NIH guidelines say NIH funding can't be used for that - and NAS and ISSCR guidelines say "don't do that" - but we don't have laws that prohibit it.

I should note that the stem cell task force is primarily charged with implementing NIH guidelines, not with creating guidelines or dealing with these kinds of questions.

Being in the position of implementing these policies, do you get a sense of when new policy is headed your way - for instance, when Bush was elected?

When Clinton left office and Bush took office, I was a scientific director, which meant that I was not involved in or responsible for trans-NIH issues. So that knowledge was way above my pay grade at that time!

Did you and others at the NIH have an idea of what policy shifts were coming ahead of the 2008 election?

Remember, both McCain and Obama had as part of their campaign commitments that they would enhance NIH funding for stem cell research. So I think most of us expected that whoever won the election, there would be changes to the Bush policy.

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