This year’s Nobel Prize for Medicine was awarded to three scientists who figured out how to target gene mutations in mice by using embryonic stem cells. Those three elements – gene therapy, embryonic stem cells, and the use of animals in laboratory experiments – are among the most controversial topics in research today.
The laureates were Mario Carpecchi, of the University of Utah; Martin Evans, of Cardiff University, in Wales; and Oliver Smithies, of University of North Carolina-Chapel Hill. Their discoveries led to the creation of a “knockout mouse”, so called because individual genes could be knocked out to determine their function. “Such gene ‘knockout’ experiments have elucidated the roles of numerous genes in embyronic development, adult physiology, aging and disease,” said a Nobel Foundation press release.
The award is likely to stir some debate among scientists and observers. Gene therapy testing in humans has so far failed to produce an effective therapy and has caused the death of at least one person, 19-year old Jesse Gelsinger, who died at a University of Pennsylvania trial in 1999. An Illinois woman, Jolee Mohr, died in July during a gene therapy clinical trial sponsored by Seattle-based Targeted Genetics, but whether the experimental drug contributed or not to the death is still to be determined (the company, and many gene therapy experts, believe it did not.) It may be a coincidence, but Targeted Genetics – which designs therapies based on targeted gene mutation or replacement – has seen its stock rise 7.29 percent to $2.06 in the wake of the Nobel announcement.
The use of human embryonic stem cells, which can become any type of cell, they sit in the midst of a heated political debate in the U.S., where the government currently opposes the use of federal funds in any research that could harm human embryos.
As for mice, many oppose the use of the little rodents in research. But the Nobel emboldened those who support experimenting on mice.
“Now would be a good time for animal rights leaders to step away from their opposition to animal studies and demonstrate support for humane and responsible research that benefits both people and animals,” said Jacquie Calnan, president of Americans for Medical Progress, an organization that supports biomedical research, in a statement.