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Thursday, September 21, 2006

Progress in stem-cell R&D

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Stem-cell therapy is advancing in ways that seemed unimaginable even just a few short years ago.These new developments suggest that life-saving technology that will revolutionize health care is right around the corner.And the most exciting thing is that these awesome changes can happen without bringing moral and ethical upheaval.

What are stem cells? Stem cells are the basic building-block cells found in humans that retain the ability to produce an identical copy of themselves when they divide and/or differentiate into other cell types. Because potentially these types of cells have the ability to act as a repair system for the body, medical researchers assume that this research has the potential to change the face of human disease.

And stem-cell research isn't new.In fact it is more than 40 years old.It was originally conceived by two Canadians, Ernest A. McCulloch and James E. Till. Starting in 1961, they had been working on experiments involving injecting bone-marrow cells into irradiated mice. By 1963 they had developed a theory regarding stem cells and published their results in Nature. That same year, the duo was able to demonstrate that these same marrow cells were capable of self-renewal -- the definition of what it means to be a stem cell.

Amazingly, at around the same time, 14-year-old Chauncey Sayre had a similar epiphany.When he thought about his father, who was much older than his mother, he wondered if the body might be able to repair itself to give people more time with their loved ones.He theorized that there must be a cell within the reproductive tract that would remain unaffected by the aging process. Otherwise, how else could humans give birth to normal, healthy children, even as they age?What he described in an essay at the time -- a treatise he still has to this day -- is a stem cell. More specifically, a germ-line stem cell.

Subsequently, the medical community has anticipated being able to use technologies derived from stem-cell research to treat cancer, spinal-cord injuries and other conditions.Up until now, the uncertainty and controversy associated with the therapy has led to mixed support from the public and the medical community. Many Americans rightly flinch at the thought of creating an identical human copy (cloning) or even destroying a fertilized egg in order to harvest its cells. Furthermore, while a number of current treatments already exist, the majority of them are not commonly used because they are either experimental, not cost-effective or simply too controversial.

But now there's been a convergence between a teen-ager's hypothesis and the world of science that doesn't come at the expense of society's morals or ethics.As a result, Chauncey Sayre and PrimeCell Therapeutics LLC, a California-based biotechnology company, are likely to confound much of the accepted science in this area. How?PrimeCell Therapeutics has achieved a significant breakthrough in the field of stem-cell research and cellular replacement therapies. Their revolutionary techniques will likely overcome any remaining obstacles in society or the medical community.

The firm has created what it dubs "primecells." They have managed to completely overwhelm the existing ethical and scientific debate involving stem cells by eliminating the controversies involving either the creation or destruction of human life forms. And perhaps most importantly, they are demonstrating that their techniques work. These primecells are the first stem cells capable of transforming into any cell type found in the body.

Their techniques do not involve the destruction of embryos, nor does it need or rely upon the creation of clones.Instead their breakthrough involves the use of adult-derived stem cells.And they have discovered how to transform these primecells into almost any cell type found in the body.PrimeCell's technique is to use the cells that normally become sperm and egg -- the most protected, genetically pure and most potent adult stem-cell source in the body to create their primecells.

And ingeniously the cell lines are "autologous" -- cell lines which come from you and are transplanted back into you for treatment. Imagine no longer worrying about the use of immunosuppressant drugs (which prevent the body from rejecting transplanted organs).Today these drugs must be taken for years or even for a lifetime even though they inhibit the body's natural ability to fight off disease. Since primecells come from you and then are used on you there is a remarkably reduced likelihood of infection following transplantation and there is no risk of rejection.

But the true breakthrough is that the primecells are pluripotent -- meaning they can develop into any of the three major tissue types: endoderm (interior gut lining), mesoderm (muscle, bone, blood) and ectoderm (epidermal tissues and nervous system). Thus these cells could be the basis for cellular replacement therapies to potentially treat Alzheimer's, heart disease, Parkinson's, diabetes, various autoimmune diseases and some forms of cancer, among other diseases.

And perhaps most importantly, they've actually been able to get this method to work repeatedly. They've successfully taken stem cells from one part of the body and turned them into cells from another part of the body. Remarkably, they have transformed the primecells into beating heart cells.

Actually, they've successfully transformed primecells into human heart, brain, bone and cartilage cells.The team's findings currently are undergoing peer review, and the company has introduced its model using mice and initial human results for PrimeCell at several global stem-cell conferences including most recently in Rome at a conference sponsored by the Vatican.

This cutting-edge science will revolutionize health care in ways that we can't imagine.And yet it all began with the musings of a child.

Horace Cooper is an assistant professor of George Mason University School of Law.

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