Could Nobel Herald Boost for Stem Cells?

Oct. 9, 2007 — -- With many scientists concerned that the United States may be falling behind other countries in the race to exploit the potential of stem cells, some hope the Nobel Prize accorded to two scientists from the United States and one from the United Kingdom for a critical gene manipulation technique could jump-start political progress in the field.

The trio of scientists that developed a technique called gene targeting was awarded the Nobel Prize in Medicine on Monday.

Sir Martin J. Evans of Cardiff University in Wales, one of the scientists, was the first to discover embryonic stem cells in the body — a discovery that some say launched the stem cell revolution the world over.

Today, countries including Australia, Belgium, China, India, Israel, Japan, Singapore, South Korea, Sweden, the United Kingdom and others are moving forward with research on human embryonic stem cells — research that some believe is already showing promise in the treatment and cure of a host of diseases and conditions.

Indeed, the first therapies based on human neural stem cells have already entered clinical trials — scientists expect therapies based on the more controversial human embryonic stem cells to enter clinical trials within the next couple of years.

But thus far, the restrictive stance on funding and research of human embryonic stem cells adopted in the United States has largely prevented this branch of stem cell study from progressing in this country.

"Our group and others have already shown that we can treat Parkinson's in animals using human dopamine neurons derived from human embryonic stem cells," said Dr. Curt Freed, director of the University of Colorado School of Medicine Neurotransplantation Program for Parkinson's Disease in Denver.

"Provided that the political and funding climates become more favorable than during the current Bush administration, treatment of Parkinson patients with cells derived from human embryonic stem cells should take place in less than five years in the United States, and perhaps earlier elsewhere," Freed said.

Political Forces Holding the U.S. Back?

Helen Blau, director of the Baxter Laboratory for Genetic Pharmacology at Stanford University, says the United States is "lagging badly" in stem cell research when compared to other countries. She adds that she hopes the award will make a difference in this trend.

"Perhaps the U.S. government will note that foreign countries are being honored in this arena and that we had better get our act together in this country, or be left in the dust," Blau said. "It is tragic that U.S. science is losing its long-term advantage in the development of novel therapies."

"The prize may draw political attention to the importance of stem cell research," Freed said.

However, some researchers feel the award will have little, if any, impact on human embryonic stem cell research — ironically, a field of study to which the gene targeting advance has greatly contributed.

"The drivers for public policy of stem cell research has never had much to do with science and I can't imagine this well-deserved recognition of superb scientific discovery will have any substantial impact on the religious perception of research with embryonic cells and the connection drawn to fetal life," said Dr. Daniel Salomon, associate professor at The Scripps Research Institute in the Department of Molecular and Experimental Medicine in La Jolla, Calif.

At the heart of this problem, many say, is a lack of funding.

"Politicization of embryonic stem cell work has been detrimental and has scared some of the public, but the biggest obstacle has been lack of funding," said Roy Ogle, professor of medicine, biomedical engineering and plastic surgery at the University of Virginia in Charlottesville. "We would be seven years closer to treatments than we are now if the Bush administration had supported scientific research adequately."

Some Potential Being Realized

Despite what many see as funding shortfalls for stem cell research, U.S. scientists have already started to unlock the therapeutic potential of nonembryonic stem cells.

"We are very close to seeing treatments for diseases of bone marrow, blood and the immune system, using cell replacement therapies, employing stem cells from bone marrow, peripheral blood and cord blood," Ogle said.

"We have proof of principle studies in animals and limited clinical trials that suggest adult stem cells can be used to regenerate, repair or replace musculoskeletal tissues — bone, cartilage, muscle, tendon, et cetera — blood vessels, skin and peripheral nerves."

While progress has been made, other researchers agree that the current stranglehold of a federal ban on funding for most human embryonic stem cell research continues to stymie advances.

However, others note that current challenges in gene research have less to do with politics and funding, and much more to do with the current boundaries of knowledge.

"The barriers to the therapeutic use of [embryonic stem cells] are primarily scientific, not political," said Dr. Markus Grompe, professor of medical and molecular genetics at Oregon Health and Science University in Portland. "Work on the NIH [National Institutes of Health] approved cell lines is progressing with federal funding."

Tim McCaffrey, professor and vice chair of biochemistry and molecular biology at the George Washington University Medical Center in Washington, D.C., agrees.

"While it is interesting, and entertaining, to frame the stem cell field in terms of 'political barriers,' the true legal barriers are quite minimal, while the immunological and technical barriers to using stem cells therapeutically are quite substantial, " McCaffrey said.

"It is crucial to understand that stem cell research is producing knowledge, which can translate into therapeutic advances within the next few years," he said. "But, it is quite possible that stem cells, themselves, may or may not be part of the therapy."

Still, Monday's announcement, if nothing else, may serve to hearten those involved in groundbreaking work in genetics — a promising high note for those in the field.

"I would argue that these basic studies have already contributed to saving lives, through insights into disease and drug development," said Dr. George Daley, associate professor of medicine at Children's Hospital Boston.

"I believe that human embryonic stem cells will likewise translate into important biomedical advances. It will just take time."