California may fund human gene editing research

Following last week's announcement that the UK is to allow researchers to edit the genes of human embryos, US state agency, the California Institute of Regenerative Medicine (CIRM), is considering whether to fund gene-editing in human embryos. It met last week to discuss guidelines and safeguards and will assess the issues and report to the full board within two months. Despite other state agencies supporting stem cell research, only California has publicly considered human embryo gene-editing. 

OAKLAND, Calif. — The California Institute of Regenerative Medicine was created in 2004 to fund stem cell research, after the federal government stopped paying for most experiments with human embryos. Now the state agency is considering underwriting another controversial use of embryos that the federal government won’t support — editing their genes.

Officials of the state agency, known as CIRM, discussed guidelines and safeguards for this type of research last week at a meeting of an internal committee that evaluates standards for research funding but made no decision about supporting such work. A new gene-editing technology called CRISPR-Cas9 has revolutionized biomedical research and is thought to hold great promise for eventually helping scientists cure hereditary ailments such as Parkinson’s or Huntington’s disease.

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Laws about embryo research are in flux around the globe, as nations struggle to keep up with quickly changing science. Following the first official government approval of an experiment that would alter human embryo DNA — in the United Kingdom last week — scientists there might soon use the cells during the first two weeks of embryonic development to study genetic factors in infertility.

This kind of research is legal in the United States, but the National Institutes of Health said last year that it won’t fund research involving gene editing of human embryos, eggs, or sperm. Changes to these “germline” genes are inherited by offspring.

So far, no researchers have publicly announced experiments to implant altered embryos in a woman’s uterus, let alone use them to create a fully developed baby. But finding the right balance of risks and benefits for genetic changes that can be passed on to future generations remains a central dilemma — and fear — for scientists and the public. In December, an international summit on human gene editing endorsed germline editing research in non-human animals and left open the door to modifying the genomes of early human embryos, eggs, or sperm as long as they’re not used to establish a pregnancy.

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The California stem cell agency has opened the prospect that state or private groups might take the lead in underwriting germline editing experiments in this country. CIRM spokesman Kevin McCormack said in an interview that his group’s standards panel will assess the issues and report to the full board within about two months. The agency might conclude that current rules are adequate to cover such work if the group chooses to fund it.

“The science is evolving so rapidly, even if we don’t make any changes to our (standards), it’s important to look and see if they are good enough, strong enough the way they are,” McCormack said.

CIRM, which already funds research using human embryos, is deciding whether to strengthen rules governing informed consent for embryo donors. Its standards experts are considering, for example, whether CRISPR can ethically be used on embryos obtained before the recent emergence of the gene-editing technology, or if it should be used only on newly donated biomaterials.

The agency is considering the use of gene-edited embryos for research that allows them to develop no longer than two weeks, as in the UK experiment.

California voters authorized $3 billion in bond sales to create CIRM, in response to a 2001 decision by President George W. Bush to sharply restrict federal funding for embryonic stem cell research. So far, CIRM’s board has committed about $1.9 billion to universities, individual researchers, and companies, although only $1.4 billion has actually been spent.

Among state agencies that support stem cell research — including in Texas, Connecticut, New York, and Maryland — only California’s has publicly contemplated human embryo gene editing. The Cancer Prevention and Research Institute of Texas and Bioinnovation Connecticut have not yet considered funding such experiments, their spokespersons said. The Maryland Stem Cell Research Fund has taken no position on this issue, and New York officials could not be reached for comment.

Some important private groups that support stem cell research also have moved cautiously. Russ Campbell, a spokesman for the Burroughs Wellcome Fund in Research Triangle Park, N.C., said “this is not an area that we are pursuing or supporting.”

Nancy Wexler, president of the New York-based Hereditary Disease Foundation, said her funds are currently committed to other priorities, and she does not anticipate supporting gene-editing of human embryos in the foreseeable future. But she called the recent move in the United Kingdom to promote this work “fantastic.”

“Their ability to do that is going to help all of us,” Wexler said, “so I say bravo.”