posted 08-24-2000 08:13 AM         

Human pluripotent stem cells are a unique scientific and medical resource. They can develop into most of the specialized cells and tissues of the body, such as muscle cells, nerve cells, liver cells, and blood cells and they can divide for indefinite periods in the laboratory, making them readily available for research, and potentially, treatment purposes. Scientists derived these unique cells from human embryos and from non-living fetuses.

The establishment of human pluripotent stem cell lines represents a major step forward in the understanding of human biology. These unique cells have captured the interest of scientists and the public, particularly patients and their advocates. Although such research promises new treatments and, possibly even cures for many debilitating diseases and injuries, including Parkinson=s disease, diabetes, heart disease, multiple sclerosis, burns and spinal cord injuries, the NIH acknowledges that the ethical issues related to this research need due consideration.

Federal law currently restricts the use of Department of Health and Human Services (DHHS) funds for human embryo research. DHHS funds cannot be used for the derivation of stem cells from human embryos. The Congressional restriction, however, does not prohibit funding for research utilizing human pluripotent stem cells because such cells are not embryos.

The purpose of the NIH Guidelines is to prescribe procedures to help ensure that NIH-funded research in this area is conducted in an ethical and legal manner. By issuing these Guidelines, the NIH aims to enhance both the scientific and ethical oversight of this important arena of research and the pace at which scientists can explore its many promises. These Guidelines will encourage openness, help make certain that researchers can make use of these critical research tools, and help assure public access to the practical medical benefits of research using these cells.

In an effort to help ensure that any research utilizing human pluripotent stem cells is appropriately and carefully conducted, the NIH sought the advice of scientists, patients and patient advocates, ethicists, clinicians, lawyers, the National Bioethics Advisory Commission (NBAC), members of Congress, among others in drafting these Guidelines. The draft Guidelines were published for public comment in the Federal Register and after reviewing and considering all comments, the NIH will publish the final NIH Guidelines in the Federal Register on August 25, 2000.

The Guidelines prescribe the documentation and assurances that must accompany requests for NIH funding for research using human pluripotent stem cells derived from human embryos or fetal tissue.

• For studies using cells derived from human embryos, NIH funds may be used only if the cells were derived from frozen embryos that were created for the purposes of fertility treatment and were in excess of clinical need.

• The Guidelines prohibit the use of inducements, monetary or otherwise, for the donation of the embryo. There must also have been a clear separation between the fertility treatment and the decision to donate embryos for this research.

• Investigators who propose to use human pluripotent stem cells from fetal tissue will be expected to follow both the Guidelines and all laws and regulations governing human fetal tissue and human fetal tissue transplantation research.

• The Guidelines require that the informed consent specify whether or not information that could identify the donor(s) will be retained.

• They require that the donation of human embryos or fetal tissue be made without any restriction regarding the individual(s) who may be the recipient of the cells derived from the human pluripotent stem cells for transplantation.

• They also require review and approval of the derivation protocol by an Institutional Review Board.

• The informed consent should include statements that the embryos or fetal tissue will be used to derive human pluripotent stem cells for research, that may include human transplantation research; that derived cells may be kept for many years; that the research is not intended to provide direct medical benefit to the donor; and, for cells derived from embryos, that embryos donated will not be transferred to a woman=s uterus and will not survive the stem cell derivation process.

• The informed consent must also state the possibility that the results of the research may have commercial potential, and that the donor will not receive any benefits from any such future commercial development.

As required by law, NIH funds cannot be used for the derivation of pluripotent stem cells from human embryos. The Guidelines also set forth several other areas of research that are ineligible for NIH funding, including: 1) research in which human pluripotent stem cells are utilized to create or contribute to a human embryo; 2) research utilizing pluripotent stem cells that were derived from human embryos created for research purposes; 3) research in which human pluripotent stem cells are derived using somatic cell nuclear transfer; 4) research utilizing human pluripotent stem cells that were derived using somatic cell nuclear transfer; 5) research in which human pluripotent stem cells are combined with an animal embryo; and 6) research in which human pluripotent stem cells are derived using somatic cell nuclear transfer for the purposes of reproductive cloning of a human.

A request for NIH funds for research using these cells must include a signed assurance that the cells were derived from human embryos in accordance with the Guidelines and that the institution will maintain documentation in support of the assurance.

This assurance must also affirm that:

• The human pluripotent stem cells to be used in the research were, or will be, obtained through a donation or through a payment that does not exceed the reasonable costs associated with the quality control, processing, transportation, preservation, and storage of the stem cells.

• The proposed research is not a class of research that is ineligible for NIH funding.

Investigators must also submit:

• A sample informed consent document, with patient identifier information removed, and a description of the informed consent process along with documentation of IRB approval of the derivation protocol.

• An abstract of the scientific protocol used to derive human pluripotent stem cells along with a title of the research proposal that proposes the use of human pluripotent stem cells.

Investigators requesting NIH funds for research using pluripotent stem cells will need to provide documentation that they are in compliance with the Guidelines prior to receiving NIH funds for this class of research. Submitted documentation will be reviewed by a newly-created NIH working group called the Human Pluripotent Stem Cell Review Group (HPSCRG). 

Members of the working group will:

• Review documentation of compliance with the Guidelines for funding requests that propose the use of human pluripotent stem cells

• Advise the NIH Center for Scientific Review Advisory Committee (CSRAC) of the outcome of their review, which, if appropriate, will be approved by the CSRAC. This decision will be forwarded to the funding Institute or Center.

• Hold public meetings when a request proposes the use of a line of human pluripotent stem cells that has not been previously reviewed by the HPSCRG. 

In no event will NIH fund research or allow existing funds to be used for research using human pluripotent stem cells derived from human embryos or human fetal tissue until the derivation protocol has received HPSCRG review and CSRAC approval. Continued compliance with the Guidelines is a term and condition of the NIH award.

Additional information about stem cells can be found on the NIH Web site at http://www.nih.gov/news/stemcell/index.htm. (The final guidelines will be available on this site as soon as they are released.)