Session I | Session II
Report: Status of Implementation of the Federal Stem Cell Research Policy
- Hearing Before the Senate Appropriations Subcommittee on Labor, Health and
Human Services, and Education - September 25, 2002
Arlen Specter (R-PA), Ranking Minority Member; Patty Murray (D-WA); and Kay Bailey Hutchison (R-TX).
Elias Zerhouni, M.D., Director of NIH
Senator Deborah Ortiz, California State Senate;
Dr. Curt Civin, Departments of Oncology and Pediatrics, Johns Hopkins University;
Dr. George Daley, Whitehead Institute, Massachusetts Institute of Technology; Dr. Roger Pedersen, Department of Surgery, Cambridge University;
Dr. Gerald Schatten, Department of Cell Biology-Physiology, Pittsburgh Development Center, Magee-Women's Research Institute.
Purpose of Hearing: The purpose of the hearing was to examine NIH's implementation of President Bush's August 2001 stem cell policy. Specifically, the Subcommittee was interested in testimony from scientists working in the field regarding their efforts to obtain the existing stem cell lines listed on the NIH registry.
Senator Specter chaired the hearing in the Chairman's absence, and discussed the Subcommittee's extensive efforts, including 13 prior hearings, on this issue. He said, "the President last year established a policy limiting stem cells to 63 or 67 lines, in that range. And a big issue arises as to whether that is adequate to carry on the research."
Statements of Witnesses
Panel One: Dr. Zerhouni testified that while the field of embryonic stem cell research is
extremely exciting, expectations must be tempered by the enormous scientific challenges that must be
addressed before therapies are developed. He used several charts to illustrate the "puzzle" of scientific
issues that must be explored, and the challenging and time consuming technical steps that are required
between derivation of stem cells from an early blastocyst and full characterization of a stem cell line.
In addition to explaining the steps NIH has already taken to implement the President's decision, Dr. Zerhouni
noted that he has established a Stem Cell Task Force to be led by Dr. James Battey, Director of the National
Institute on Deafness and Other Communication Disorders. One of the first issues to be addressed by the
task force is the dearth of experienced investigators in the field.
Panel Two: Deborah Ortiz, a state legislator from California, described a recently enacted State law on stem cell research that she sponsored. She said the bill makes stem cell research legal in California with the appropriate ethical review and includes prohibitions on the sale of embryos for profit. She said, "we ask that Congress respect California's will to protect Californians and assure that that right will be protected and not preempted by any subsequent law."
Dr. Pedersen, a stem cell researcher previously from the University of California at San Francisco (UCSF), who moved to England in order to work without the constraints of the President's policy, described his frustrations with that policy. Although praising NIH for its efforts, he suggested that the delay in Federal funding has "delayed the benefit of research to patients with the degenerative disease." He argued against a central U.S. repository, indicating that such an effort could lead to additional delays in the accessibility of the lines.
Dr. Schatten testified that although the NIH registry lists 71 stem cell lines, he has been able to identify "just a handful," and to obtain only two. He said that while investigators in other countries are interested in collaborating with him, he believes that American science should be "conducted on American soil." He argued for increased funding for supplements to existing NIH grants. In a dramatic criticism of the President's policy, he said, "Would Galileo have been satisfied if he could have looked at 65 or 71 stars? Maybe. But he wouldn't have discovered our place in the solar system unless Jupiter traveled through that narrow field."
Dr. Civin, an adult stem cell researcher, discussed his interest in embryonic stem cells (ES) as a way of understanding the molecular pathways by which such cells can proliferate without differentiating. One of his scientific goals is to use this knowledge to push the same "molecular buttons" in adult stem cells. He expressed frustration with the fact that only a small number of lines are available, and that access to those is hampered by the time, difficulty, and expense of negotiating material transfer agreements. Dr. Civin stated that the cost of $5,000 per cell line is 50 to 100 times greater than what he and his colleagues are accustomed to paying in other settings. He also reported that after months of negotiating with scientists in India, he was told that the Indian government has put an indefinite hold on sending ES lines out of their country. Dr. Civin argued strongly in favor of an NIH-funded repository where NIH would characterize the lines and serve as a distribution center.
Dr. Daley also testified about his difficulties in obtaining the available stem cell lines. He argued in favor of a broader policy that would permit both nuclear transfer and the derivation of new lines. He described some of his own research that involved both the use of stem cells derived by nuclear transfer (cloning) and gene therapy to treat a mouse with so-called "bubble boy" disease. Finally, he argued strongly in favor of a federally-funded stem cell repository which would maintain "consistent, standard operating procedures for the culture and maintenance of the cell lines."
Senator Specter's questions were aimed at eliciting more concrete information from the witnesses regarding whether the existing stem cell lines are sufficient for research. Dr. Zerhouni explained that as a scientific matter, this is "not knowable" at the present time. Senator Hutchinson, while generally supportive of the President's policy, expressed concern that talented researchers in her state would move to other states, such as California, in order to take advantage of more liberal policies regarding stem cell research. Senator Murray expressed concern that the existence of the California law might prohibit California researchers from receiving NIH funds. In response, Dr. Zerhouni explained that the state law does not bar state researchers from receiving NIH funds.
The specific questions follow:
- You heard the testimony about the sharp limitations on the availability of stem cell research lines. According to the information which our subcommittee has been able to glean, only five of these lines are available to stem cell researchers. The question is: Is there a sufficient number of stem cell lines available for the required research?
- You're working with the sources of the 78 lines, which were approved by the president as of August 9. Is that adequate?
- Dr. Zerhouni, when will we know whether the existing stem cell lines available are adequate? Can you give me a timeline as to when we'll know? And Dr. Zerhouni, if you can supplement what you have said with a timeline as to when you think you might know, we will be interested to know that.
- Dr. Pedersen, to what extent do you think your example will be followed by others in leaving the United States to go to places like Great Britain where you can get funding on stem cell research?
- Dr. Pedersen, you're saying the number of stem cell lines for clinical delivery transplantation is zero?
- I would like you, Dr. Schatten, Dr. Civin, Dr. Daley, Senator Ortiz and Dr. Pedersen, to provide in writing what you would like to have available. Dr. Daley, you have talked about nuclear transfer studies. I'd like to get the specifics as to what you have in mind. Give me your insights as to what legislation you would like to see enacted.
- I think the President's policy was meant to allow use of the lines that are available to do the necessary testing. But then I hear your stories about trying to get lines, and it doesn't seem to be working. I have researchers at UT Southwestern who are very concerned about availability. I now have another concern: now perhaps some of our good scientists will be lured to California. Dr. Zerhouni, now that California is looking at providing state support for this research-and so many of our institutions are public institutions that do get private help, but also Government support-what can we do to keep everything balanced, so that scientists don't migrate to the West Coast?
- The amount of support that is available from private and state funds is not comparable to Federal funding. Does it become complicated if an investigator is being supported with Federal funds and then wants to take the next step into clinical studies. Do you have to stop doing the federally funded research?
- Can you use embryonic stem cells, under the present law or the President's policy, to do that research?
- I would like to ask other members of the panel if they feel that the current differentiation is enough to allow people to go forward.
I think many of us were very concerned about the President's decision to limit stem cell lines a year ago. Now that California has moved ahead, we are concerned about a drain of researchers from our states. Dr. Zerhouni, if state funds are used for embryonic stem cell research, will researchers in California or any other states that enact laws like this receive NIH funds in the future or will they be prohibited from receiving those funds?
Prepared by Gemma Flamberg, OD/OLPA, October 9, 2002