13 new stem cell lines get NIH approval

Washington -- The federal government officially began funding new human embryonic stem cell research on Dec. 2, when the National Institutes of Health declared that 13 human stem cell lines met NIH ethics guidelines released in July.

The 13 lines -- derived by two institutions -- are the first of hundreds under NIH review or expected to be submitted for approval, said NIH Director Francis S. Collins, MD, PhD. The exact number of privately developed stem cell lines is not known, but the agency sent notifications to several hundred investigators and institutions about the availability of NIH funding.

But Dr. Collins added that NIH is carefully reviewing the applications to ensure they meet ethics guidelines. Stem cell lines must come from excess embryos at in vitro fertilization clinics to be eligible for use in federal research. The embryos must be provided by donors who were informed of their options and who provided clear written consent, among other requirements.

"I want to re-emphasize just how careful NIH is being and how seriously we're taking the responsibility to be sure that these lines are being developed in a fashion that is ethically above reproach," Dr. Collins said.

For instance, the NIH Advisory Committee to the Director on Dec. 4 withheld an approval recommendation for one stem cell line of 28 submitted by Harvard University because the donor's consent had lapsed, said NIH spokeswoman Jenny Haliski. The panel approved the other 27 lines. The advisory committee and NIH staff are reviewing stem cell applications, and Dr. Collins provides the final go-ahead.

NIH approval of the 13 lines marks the reopening of a source of funding denied to investigators using human embryonic stem cells since President Bush's August 2001 executive order, which banned the use of federal money for research on lines derived after the order. President Obama signed an executive order in March lifting that ban.

NIH had been holding back $21 million worth of institute and stimulus act money for 31 research grants. Dr. Collins said he expects to award at least another $10 million and possibly much more in early 2010 as NIH reviews additional research grant applications.

Eleven of the 13 NIH-approved lines were derived by Children's Hospital Boston and its George Q. Daley Laboratory and will be used to study rare congenital diseases -- including marrow failure -- and to develop sources of new cells for transplants, said M. William Lensch, PhD, a senior researcher in the Daley Lab. About one-half to two-thirds of patients who would benefit from blood stem cell transplants do not have a matching donor, he said.

The other two stem cell lines were derived at Rockefeller University in New York.

Human embryonic stem cells are interesting to researchers for their pluripotency, or ability to form new cell types. For example, one line of research is focusing on the potential to develop replacement neural cells for patients with damaged spines. The Food and Drug Administration is considering applications for such research in humans.

Opponents of human embryonic stem cell research -- which necessitates the destruction of human embryos -- point to emerging work on induced pluripotent stem cells, or mature cells reversed into pluripotency, as an alternative.

Although Lensch and his colleagues work with all types of cells -- including induced pluripotent cells -- research on human embryonic stem cells also is needed, he said. "It really all has to be on the table, because you don't know where the best information -- or hopefully, the breakthroughs -- are going to come from. Having as many avenues of research to you as possible seems to be the best way to go forward."

Research on embryonic stem cells also will broaden understanding of human development, said Mark O. Lively, PhD, president of the Federation of American Societies for Experimental Biology.

Lively said the new federal support for embryonic stem cell work is especially important because private funding for biomedical research has shrunk during the recession.

But even the federal support is limited. Stimulus act funding will be exhausted at the end of 2010, which will pose difficulties in maintaining the pace of biomedical research started with the stimulus act, Dr. Collins acknowledged. "That's a broad problem that many of us are concerned about."