Washington Editor

WASHINGTON - The Obama administration Wednesday approved the first 13 human embryonic stem cell (hESC) lines eligible for use in government-funded research under new guidelines adopted in July.

National Institutes of Health Director Francis Collins said the 13 lines, which were developed by Children's Hospital Boston and Rockefeller University in New York, were derived from embryos that were donated under ethically sound informed consent processes.

"I want to emphasize just how careful NIH is being and how seriously we are taking the responsibility to be sure that these lines have been derived in a fashion that is ethically above reproach and that takes into account all of the issues related to informed consent," he told reporters Wednesday during a briefing. The 13 lines represent the first in what is expected to be a series of ongoing waves of approvals, Collins said.

He noted that an additional 96 lines have been submitted to the NIH for internal administrative review or consideration by an external working group, which presents its findings to a committee that makes recommendations to Collins.

That advisory panel is meeting Friday to review and debate 20 of those 96 lines.

"It is exciting to be able to say that after what has clearly been a time of some frustration on the part of the scientific community with an inability to gain access to federal funds to cell lines that investigators wish to utilize, that that is now changing, and as of today, has changed for these 13 specific lines," Collins said. "This is a significant day. This is a moment at which President Obama's strong desire to see a loosening up of what has been considered unduly tight restrictions on stem cell research is now coming to pass."

The president on March 9 issued an executive order lifting funding rules put in place by his predecessor. (See BioWorld Today, March 10, 2009, and March 11, 2009.)

Collins noted that there still is a ban against the creation of new stem cell lines with federal funds, based upon the Dickey-Wicker amendment, which remains in place.

The NIH in July issued final guidelines, which limited the funding to embryos donated for research created for reproductive purposes in excess of clinical need. The guidelines include strict rules, such as requiring written informed consent. (See BioWorld Today, July 7, 2009.)

Collins said the NIH has funded 31 grants so far this year totaling more than $21 million proposed for hESC research. However, those grants were restricted until the approved lines became available on the agency's registry.

"Those 31 grantees are now given the chance to look at the registry and to decide which of the lines there they would like to start work with, and as soon as they notify NIH of that, the restriction will be lifted," Collins said.

A number of NIH Challenge Grant applications also could be funded through the American Recovery and Reinvestment Act in fiscal year 2010, he added.

George Daley, a Harvard University professor and director of stem cell transplantation at Children's Hospital Boston, and his colleagues developed 11 of the 13 lines approved Wednesday, while Ali Brivanlou, head of the Laboratory of Molecular Vertebrate Embryology, and his colleagues at Rockefeller University developed two, Collins said.

The two institutions submitted information about the informed consent process for embryo donation to the NIH administrative review process, which confirmed that the submissions met the guidelines' specific requirements, he noted.

Brivanlou said his academic lab started on its hESC line research in 2002, a year after President Bush's order restricted federal funding to research involving 21 existing lines.

"We did it for very specific basic scientific reasons," he told BioWorld Today. "It became very clear to us from the early onset that the lines approved by the Bush administration were suboptimal for the kind of work we wanted to do."

Brivanlou noted that the 21 Bush-approved hESC lines were developed when the technology was still relatively new, "but the derivation was not done under the optimum conditions, and as we look at those cells more and more, we understand why things are going wrong."

He noted that some of the lines experienced a loss of chromosomes or had chromosomal abnormalities, which have made scientists "think twice about using them in any forum.

"You don't want to start working on something that already has some basic problems or issue that might affect the conclusions you reach on your basic studies," Brivanlou said. "You want to start on a canvas that is clean."

Rockefeller University was "very careful" to define the rules of engagement, which involved bioethics and legal deliberations, and therefore, it took a few years before Brivanlou's lab could get started, he said.

Brivanlou noted that sociopolitical implications imposed by the 2001 order left many academic researchers interested in hESC research out in the cold.

But, he said, "we are lucky at Rockefeller to have an administration that was supportive."

Brivanlou said his lab derived the two lines - RUES-1, a male XY line, and RUES-2 a female XX line - in 2005 using private funds provided by the Juvenile Diabetes Research Foundation.

"At the early stages, it was not easy to engage in this kind of work, but seeing these on the NIH registry warms my heart," Brivanlou said. "There is an inherent pleasure and satisfaction and honor derived from that."

Brivanlou said his lab will accommodate scientists interested in accessing the lines "as fast as we can."

He noted that since their derivation, the Rockefeller University lines have been distributed to scientists in the U.S. and abroad. But Brivanlou said he expects the requests for access to the lines to substantially increase now that NIH funding will be available to work with them.

"We welcome the demand," Brivanlou said.

Published  December 3, 2009