Having arranged exclusive agreements with leading fertility centers to obtain embryos and eggs donated for stem cell research, having recruited a renowned expert in cloning procedures to lead its research endeavors and having ongoing collaborations with national and international experts in stem cell research, La Jolla-based Stemagen believes it is uniquely positioned to become a world leader in the most promising, yet challenging, area of embryonic stem cell research, "therapeutic cloning."
"The solid foundation we have in place certainly give us cause for a very optimistic outlook," said Stemagen CEO Samuel H. Wood, M.D., Ph.D.
Stemagen's optimism is due in part to the company's access to excess high-quality embryos. "Currently in the United States, fertility clinics are collectively storing over 400,000 embryos," Wood noted. "Many of these are 'left over' after a couple has completed their family. In some cases couples are willing to donate them to other infertile people, but only a small percentage do. Most often, couples will opt to have these excess embryos discarded, rather than continuing to pay storage fees."
Recently, however, couples have been offered another option: donating their left over embryos to stem cell research.
Although research in this area is still in its infancy, Wood said it is the widely reported potential of stem cells to treat a variety of presently incurable degenerative diseases that makes this option an attractive one for many couples.
"Increasingly, couples are choosing to donate their embryos for stem cell research rather than simply discarding them," Wood said.
"Having access to the embryos and oocytes necessary for this type of research is obviously crucial, but finding someone with the expertise to perform the very complicated procedures involved is equally important," said Wood. "There are only a handful of scientists in the world with the knowledge and expertise to move research in this area forward and we are fortunate to have one of the very best, Andrew French, Ph.D., as our chief scientific officer."
"Access to high quality embryos and oocytes (eggs) is essential for this type of research," said French. "Working exclusively with elite reproductive scientists with a long track record of outstanding skill in embryology, the probability of obtaining the very high-quality embryos needed for the generation of embryonic stem cell lines is markedly enhanced.
"For example," French continued, "under the direction of Catharine Adams, Ph.D., the embryology laboratory at the Reproductive Sciences Center, one of Stemagen's exclusive infertility center partners, has achieved the almost unheard accomplishment of obtaining three consecutive perfect scores on evaluations of its procedures on inspections conducted by independent experts in the field of reproductive biology."
French knows something about laboratory preparedness. He was the senior researcher at the Australian-based Monash Institute of Medical Research that generated some of the world's first cloned elite transgenic and non-transgenic dairy cows using a process called somatic cell nuclear transfer (SCNT).
It is the application of SCNT technology to the production of patient-specific embryonic stem cell lines, or therapeutic cloning, for which Stemagen has received IRB approval. While complicated, the process involves taking the genetic material out of an egg (oocyte) and replacing it with DNA of a selected individual.
For example, Stemagen could take an adult cell from a patient with Parkinson's disease and transfer it into an unfertilized egg that has had its genetic material removed in an attempt to create a stem cell line that could be used to better understand the disease or, perhaps in the future, used to treat that particular patient.
If successful, the company believes it will be able to replicate genetic-based degenerative diseases like Alzheimer's or Parkinson's in a petri dish for scientific analysis that may one day lead to treatments for diseases where today none currently exist.
Stemagen joins just two other entities in the United States -- Harvard University and University of California, San Francisco -- with independent institutional review board (IRB) approval to engage in SCNT/embryonic stem cell research.
Because Stemagen is a privately funded company and thus was not subject to federal restrictions on stem cell research that hamper many institutions, it was able to immediately begin its promising research projects, many of which are ongoing. Because Stemagen does not need to rely on government funding, Wood believes it is more nimble than larger institutions, and has been able to begin its work while others are only just now hiring staff and developing policies and procedures.
Indeed the science of stem cell derived transplantation is moving forward in other states. Scientists at the University of Washington last month announced the application of human embryonic stem cells in treating diseased retinal tissue in mice suffering from macular degeneration, the leading cause of vision loss in people over age 60.
"This is a very promising development for cell replacement in the retina," said Tom Reh, University of Washington professor of biological structure and leader of the research.
Reh said that if the stem-cell research at the university and other institutions continues to be successful, the first human tests of the technique could begin in about two years.
"The day draws ever closer where we will stop praising the potential of embryonic stem cells, and start lauding the treatments available as a result of their application," Wood said.
But Wood cautioned companies involved in this research need to maintain the highest levels of ethical standards and scientific review.
"While the majority of Americans support our research," he said, "we will lose that support if we make claims that don't hold up to scientific or ethical scrutiny."
Last year South Korean scientist Hwang Woo Suk published an article in the scientific journal "Nature" outlining claims that he had successfully cloned human embryonic stem cells, and could repeat the process efficiently, an achievement scientists consider a major step forward.
However, officials at Woo Suk's University later announced their scientist's claims to be false. The scandal dealt a significant, but fortunately temporary, blow to research in this area.
As a result, Wood said, any announcement now about any advancements in the field of therapeutic cloning are going to be closely scrutinized by the scientific community at large.
"That's why at Stemagen we adopted early on a process where every success we have is reviewed by an independent company for validation," he said. "You can be sure that every announcement about our research will be independently verified by leading scientists before it is publicly released."
Jimenez is director of corporate communications for Stemagen in San Diego, www.stemagen.com>