Dennise Dalma-Weiszhausz, PhD

Stem Cell Applications

From Alzheimer’s to arthritis, blindness, burns, cancer, diabetes, heart disease, liver disorders, multiple sclerosis, Parkinson’s, spinal cord injury, stroke… Stem cells have been proposed as candidates to treat diseases and disorders of every organ of the human body.

Cell-based Therapies

Historically, donated organs have been transplanted and medical devices implanted to replace failing systems. In the case of the former, the need far outweighs the available supply and, in both cases, the risks and costs are high. Side effects may limit the effectiveness or feasibility of drug, radiation or surgical interventions. Advances in biotechnology have led to the identification and replication of specific substances — such as sugars, amino acids, neurotransmitters and hormones — that are deficient in some degenerative diseases. While administering these substances as medication can overcome some of the limitations of more traditional pharmaceutical products, such as lack of specificity, there is no synthetic technology that can deliver them to the precise sites of action under the appropriate physiological regulation and dosage, or for the duration required to cure the condition. Cells, however, do this naturally.

The concept of cell-based therapy (or simply cell therapy, as it is sometimes called) is to repair, replace or supplement damaged or diseased cells with healthy cells. The work of StemCells scientists has already generated the means to supply stem cells, which upon transplantation, can differentiate into healthy new cells or tissues, and which may thereby be capable of alleviating or potentially even curing a broad array of intractable conditions.

The introduction of healthy cells has been shown to protect remaining functionality, as in the company’s preclinical studies of the effects of neural stem cell transplantation in an animal model of AMD. Transplanted neural stem cells appear to be able to replace dysfunctional cells, such as the oligodendrocytes needed to alleviate myelination disorders, such as PMD. Where impaired cellular function is associated with the progressive decline commonly seen in degenerative diseases like NCL, stem cells can deliver specialized cells that secrete, metabolize or regulate essential substances. Stem cells may even be able to help make gene therapy a reality by delivering the truly long-term gene expression necessary for successful treatment of genetic disorders. The therapeutic programs currently underway and, in some cases, already in clinical trials at StemCells, Inc. are paving the way for a variety of promising new cell-based therapies.

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Tools for Research

Our understanding of human biology — the mechanisms and processes that allow humans to live, breathe, think and create — is still quite limited. It took nearly 200 years after discovery the of the cell in the mid 1600s, before it was proposed that all living things are made up of cells. And it wasn't until the late 1990s that science — in fact our own Scientific Advisory Board member Dr. Fred Gage — overturned the popular notion that the brain neurons with which we are born must last us a lifetime, by showing that adult brains can indeed generate new neurons — from stem cells. The identification and isolation of stem cells is a relatively recent scientific development. But, according to the UK Stem Cell Foundation, over 2,000 research papers on stem cells are now being published in reputable scientific journals every year¹. The fact is, we have much to learn from stem cells.

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Drug Discovery and Development

For every drug used clinically to treat a disease, more than 6,000 chemical compounds are synthesized and screened to uncover lead candidates; over 90 percent of the drugs that make it to clinical trials are not approved for use. On average, the process takes approximately 12 years from lead identification to market approval and costs nearly US$1 billion.^2,3 One reason for this high rate of failure may be inaccurate disease models. Stem cells can be induced to differentiate into cell types that retain more similarity to specific tissues than current models. They can even be genetically reprogrammed to produce cells emulating targeted genetic disorders, offering a more accurate and efficient platform for the drug discovery and development process. StemCells scientists have already developed stem-cell based disease models for use in screening and toxicity assays in vitro, as well as the corresponding in vivo models required for preclinical evaluation.

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