Rockefeller stem cell lines among first to be NIH-approved

by JOSEPH BONNER

Two human embryonic stem cell lines derived at Rockefeller are among the first to be approved for use in federally funded research since the National Institutes of Health adopted new guidelines in July 2009.

The approval means that cell lines derived at the university can be made available to any scientist, including those supported by NIH grants, for further research. The two lines, called RUES1 and RUES2, are among the initial 13 to be approved and represent the first new lines of human embryonic stem cells available for NIH-funded research since the restrictions on using federal money for work on stem cells were loosened under President Barack Obama.

RUES1 and RUES2 were derived in the Laboratory of Molecular Vertebrate Embryology, headed by Ali H. Brivanlou, in 2005 using private funds. The embryos used for the derivation of the lines were originally generated for reproductive purposes and subsequently donated for research purposes following consent of the donors. The stem cell lines were shared with nine different researchers at eight different institutions before an application was filed with the NIH.

“I am very happy that the Rockefeller-derived stem cell lines will now be available to any NIH-supported researcher,” says Dr. Brivanlou, who is Robert and Harriet Heilbrunn Professor at Rockefeller. “It is humbling to think that future discoveries by NIH-funded researchers will be based on this primary work done at Rockefeller.”

President George W. Bush announced on August 9, 2001 that only those hESC lines derived prior to that date would be eligible for research funded by federal money. In the end, 21 human embryonic stem cell lines were included on the original NIH Registry, but scientists criticized these lines as flawed — some had damaged chromosomes and all were kept viable using mouse feeder cells that introduced animal viruses into the human cells — and unfit for use in human patients.

“There was an extraordinary outpouring of support from Rockefeller’s community of benefactors to keep the university’s stem cell studies moving forward,” says Marnie Imhoff, vice president for development. “More than $8 million was contributed by nearly 40 trustees, Rockefeller University Council members and other friends of the university. Then in June 2005, The Starr Foundation made a $50 million commitment to fund collaborative stem cell studies at Rockefeller, Memorial Sloan-Kettering and Weill Cornell.”

In March 2009, President Obama issued Executive Order 13505: Removing Barriers to Responsible Scientific Research Involving Human Stem Cells, which allows for a broadening of the number of lines available for use in federally funded research and the ability to add new lines to the NIH Registry moving forward. By July, the NIH had published its Guidelines for Human Stem Cell Research and implemented the executive order, opening the door for researchers to apply to the NIH for grants to conduct research with human embryonic stem cells derived both before and after August 9, 2001 that meet the criteria described in the new guidelines. The NIH also announced an application process for institutions interested in making stem cell lines derived using non-federal funds available for federally funded research, which should lead to a new NIH Registry that contains a much larger and more varied number of human embryonic stem cell lines.

According to Amy Wilkerson, associate vice president for research support, the university started the application process for the two lines in mid-September. “We filed under the procedure for administrative review, which requires that the provenance of the lines meets all requirements of the current NIH guidelines for research with human embryonic stem cells,” says Ms. Wilkerson.

As part of the review process, the university submitted information about the process used to obtain informed consent from embryo donors; the NIH administrative review process confirms that the submissions met specific requirements regarding informed consent.

The process leading up to this approval began in February 2002, when Acting President Thomas P. Sakmar initiated a working group to explore conducting human embryonic stem cell research at Rockefeller. Ms. Wilkerson and Ms. Imhoff, along with Kathleen Denis, associate vice president for technology transfer, and the Rockefeller general counsel’s office, played key roles in establishing the early strategy for supporting human embryonic stem cell research at Rockefeller.