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Statement of Harold Varmus, M.D., Director, National Institutes of Health, Before the Senate Appropriations Subcommittee on Labor, Health and Human Services, Education and Related Agencies, January 26, 1999 on research applications of stem cells.

The isolation and culturing of human pluripotent stem cells opens certain avenues of research for the first time. Let me mention just three potential applications of human pluripotent stem cells. The first is research focused on how stem cells differentiate into specific types of cells. The goal is to identify the genetic and environmental signals that direct the specialization of a stem cell to develop into specific cell types. Studying normal cell and tissue development will provide an understanding of abnormal growth and development which, in turn, could lead to the discovery of new ways to prevent and treat birth defects and even cancer.

A second and more practical application of research using these cells is in pharmaceutical development. Use of human pluripotent stem cells could allow researchers to study the beneficial and toxic effects of candidate drugs in many different cell types and potentially reduce the numbers of animal studies and human clinical trials required for drug development.

The third and most obvious potential application of these human pluripotent stem cells is to direct the specialization of the cells into cells and tissues that could be transplanted into patients for the purpose of repairing injury and pathological processes. A number of such examples are described in my December testimony, but two are worth mentioning here.

(i) Transplantation of healthy heart muscle cells could provide new hope for patients with heart disease. The hope is to develop heart muscle cells from human pluripotent stem cells and then transplant them into the failing heart muscle in order to augment the function of the heart. Preliminary work in mice and other animals has demonstrated that healthy heart muscle cells transplanted into the heart successfully repopulate the heart tissue and integrate with the host cells. These experiments show that this type of transplantation is feasible.

(ii) In many individuals with Type I diabetes, the production of insulin in the pancreas by specialized cells called islet beta cells is disrupted. There is evidence that transplantation of either the entire pancreas or isolated islet cells could mitigate the need for insulin injections. Islet cell lines derived from human pluripotent stem cells could be used for this critical research and, ultimately, for transplantation.

Because human pluripotent stem cells continue to replicate robustly, stem cells derived from a few embryos or from a few fetuses could potentially be used in hundreds of individual research protocols.

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