Stem Cell Research

What we are attempting to create with primordial stem cell technology are cells which have the capacity to restore damaged cells in the body, including the following:

cardiac muscle cells to treat heart attack victims and degenerative heart disease;

skin cells to treat burn victims;

spinal cord neuron cells for treatment of spinal cord trauma and paralysis;

neural cells for treating those suffering from neurodegenerative diseases;

pancreas cells to treat diabetes;

blood cells to treat cancer anemia, and immunodeficiencies;

neural cells to treat Parkinson’s, Huntington’s and Amyotrophic Lateral Sclerosis (ALS);

cells for use in genetic therapy to treat 5,000 genetic diseases, including Cystic Fibrosis, Tay-Sachs Disease, schizophrenia, depression, and other diseases;

blood vessel endothelial cells for treating atherosclerosis;

liver cells for liver diseases including hepatitis and cirrhosis;

cartilage cells for treatment of osteoarthritis;

bone cells for treatment of osteoporosis;

myoblast cells for the treatment of Muscular Dystrophy;

respiratory epithelial cells for the treatment of Cystic Fibrosis and lung cancer;

adrenal cortex cells for the treatment of Addison’s disease;

retinal pigment epithelial cells for age-related macular degeneration;

modified cells for treatment of various genetic diseases; and

other cells for use in the diagnosis, treatment and prevention of other deadly or disabling diseases or other medical conditions.

Many of us in the biomedical research community are excited about the prospects for this new generation of cell therapy. It promises to provide a new approach for treating human diseases, an approach that isn’t simply dependent on pharmaceutical intervention, but on living cells that can differentiate into blood, skin, heart, or brain cells and can potentially treat various cancers, spinal cord injuries, and heart disease and many other currently diseases where current treatments are unavailable or ineffective.

The types of cells that make up most of the human body are differentiated, meaning that they have already achieved some sort of specialized function such as blood, skin, heart or brain cells. The precursor cells that led to differentiated cells are commonly called in the medical research community "stem cells" because functions stem from them like the growth of a plant. Stem cells have the capacity for self-renewal, meaning that they can reproduce more of themselves, and differentiate, meaning that they can specialize into a variety of cell types with different functions.

Primordial stem cells are relatively undifferentiated stem cells that posses the power to differentiate into many different types of cells of incalculable value to medicine. Unfortunately, until recently human primordial stem cells have proven impossible to grow in the laboratory leaving medicine with no choice but to seek transplantable cells from aborted fetuses, cadavers, and so on. The advent of primordial stem cell technology promises to provide a means of manufacturing any human cell type in the industrial setting without the ethical problems associated with the need to continually source material from diseased individuals or other sources. Therefore, primordial stem cell technology could solve many of the ethical problems associated with sourcing material for transplantation.

In the last decade, scientists studying mice and other laboratory animals have discovered powerful new approaches involving cultured primordial stem cells. Studies of these cells obtained from mouse stem cells show that they are capable of differentiating, in vitro or in vivo into a wide variety of specialized cell types. Primordial stem cells have been derived by culturing cells of non-human primates. Promising efforts to obtain human stem cells have also recently been reported.

Let me distinguish primordial stem cells from other stem cells in the body. You may well have heard of hematopoietic stem cells, which can be extracted from the blood of an adult or child. These stem cells have differentiated to become a blood stem cell. There are many clinical trials underway to try to isolate and utilize these and other stem cells in the body for therapeutic purposes such as the treatment of cancer. Unfortunately, unlike the blood system, many tissues in the body do not contain stem cells. Therefore, injury to these tissues is permanent and disabling. For example, the loss of heart and neurons leads to a permanent loss of function.

By way of contrast a primordial stem cell has the potential to become literally any cell in the body and therefore it offers a solution to many untreatable diseases and conditions.

Primordial stem cell research has been hailed as the "[most] tantalizing of all" research in this field, because of the great promise of the technology to open new doors for the treatment of manifold diseases. In addition to the broad number of cell types that can be generated from primordial stem cells, they are especially well suited to be a source of gene therapy offering concrete therapeutic applications for the human genome sequencing project and similar efforts.

Using heart attacks as an example, we believe we will be able to replace damaged cardiac cells, with healthy stem cells, that could differentiate into cardiac muscle. Research using these primordial stem cells could lead to the development of "universal donor cells," and could be an invaluable benefit to patients. Primordial stem cell therapy could also make it possible to store tissue reserves that would give health care providers a new and virtually endless supply of the cells listed above. We could obviate the need to completely transplant an organ, a process which involves securing a donor and avoiding rejection (through the use of immunosuppressants which have side effects). Of course many individuals die for lack of a suitable organ for transplant. Repairing an organ from within the patient is obviously a tantalizing prospect. The generation of primordial stem cells to create these therapies would lead to great medical advances.

In addition this primordial stem cell research has the potential to develop and improve cell and tissue therapies in other tissue systems as well, including but not limited to spinal cord injury, diabetes and other diseases. It should be clear why BIO and I are so concerned about the pending legislation’s impact on biomedical research. We urge the Congress to ensure that this legislation concerning human cloning would not in any way obstruct this promising research.