By Debbie Strickland

Associate

Nearly two years after human pluripotent stem cells (PSCs) were first isolated, the National Institutes of Health has finalized guidelines to deal with the political, legal and ethical questions sparked by the achievement. The new rules allow such research to receive NIH funding and are expected to draw legions of academic researchers to a field dominated up to now by private efforts.

"By pulling in some of the top minds in the academic community, we will start seeing more progress in research into the differentiation of such cells into various cell types important for treating [human disease]," said Robert Lanza, vice president of medical and scientific development at Worcester, Mass.-based Advanced Cell Technology Inc., a privately held company that is among the handful of leaders in stem cell research.

"Private industry has led the way so far," said Michael Werner, director of federal government relations and bioethics counsel at the Biotechnology Industry Organization in Washington. "But now that there is federal money available, the work in this area will dramatically increase."

Even the two landmark 1998 projects that first isolated human PSCs - one conducted at Johns Hopkins University in Baltimore, the other at the University of Wisconsin in Madison - were privately funded.

Human PSCs are capable of developing into many types of specialized cells, offering the possibility of a renewable source of replacement cells and tissue to treat a myriad of diseases, conditions, and disabilities, including Parkinson's and Alzheimer's diseases, spinal cord injury, stroke, burns, heart disease, diabetes, osteoarthritis and rheumatoid arthritis. According to the NIH, "There is almost no realm of medicine that might not be touched by this innovation."

Final Guidelines Look A Lot Like Earlier Draft

"The guidelines are pretty much what we expected," said Werner. "They are not dramatically different from the proposed guidelines." A draft was released last December for public comment. (See BioWorld Today, Dec. 6, 1999, p. 1.)

Lanza called the NIH's position "laudable, and considering the political climate, this will advance the cause." In the U.S., he noted, embryo research is highly controversial, but Lanza expects much of the political animosity to blow over as people begin to understand the technology. "We're talking about a completely undifferentiated ball of cells, something that's pre-implantation [as the source for human PSCs]," he said. "Does a completely undifferentiated ball of cells warrant the same rights as a child or adult who may die [without a stem-cell-based therapy]?"

Perhaps the thorniest matter in developing the guidelines was squaring the desire of NIH officials - notably former Director Harold Varmus - to fund research on PSCs with a federal law banning research on embryos, from which the cells are derived.

The NIH guidelines solve the problem neatly and in accordance with the opinion of the agency's legal counsel: Funding for the derivation of human pluripotent stem cells is prohibited, but funding is allowed for research that uses the cells after they have been collected.

"If you're a researcher, you can use federal money to do research on embryonic stem cells," said Werner, "but you have to obtain the cells from somebody who is not using federal dollars to get them."

That's not to say the NIH isn't interested in the cells' origins. A request for NIH funds for research using these cells must include a signed assurance that the cells were derived from leftover frozen embryos created for the purposes of fertility treatment. The rules also mandate an extensive informed consent process and permit no payments beyond those necessary to cover reasonable costs associated with the cells' collection and processing.

"The guidelines are an effort of the NIH to try to achieve a balance," said Werner, "to say we're not being cavalier about this, that [the research] is going to be tightly regulated and tightly controlled, but that the potential is so great we need to go forward."

A new NIH working group, the Human Pluripotent Stem Cell Review Group, will review grant applicants' documentation for compliance and will hold public meetings when a request proposes the use of a line of human pluripotent stem cells that has not previously been reviewed.

In addition to maintaining the ban on funding for derivation of PSCs, the guidelines set forth several other areas of research that are ineligible for federal dollars:

¿Research in which human PSCs are used to create or contribute to a human embryo.

¿Research using PSCs that were derived from human embryos created for research purposes.

¿Research in which human PSCs are derived using somatic cell nuclear transfer.

¿Research in which human PSCs are combined with an animal embryo.

¿Research in which human PSCs are derived using somatic cell nuclear transfer for the purposes of reproductive cloning of a human.

For Advanced Cell Technology, the new federal dollars open the possibility of new collaborations with academic researchers, though Lanza added that the guidelines are not as "forward thinking" as those recently promulgated by the British Science and Ethics Commission, which recommended allowing human therapeutic cloning.

Publicly traded firms working in this field include Geron Corp., of Menlo Park, Calif., and StemCells Inc. (formerly CytoTherapeutics Inc.), of Sunnyvale, Calif. Shares of Geron (NASDAQ:GERN) rose 4.6 percent to $25.50 Wednesday, but those of StemCells (NASDAQ:STEM) soared 37 percent to close at $7.375.

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