Innovative Science Programs: Stem Cell and Regenerative Medicine
Leader: Mark Noble, Ph.D.
An exciting innovative science program is stem cells, where cellular approaches to treat diseases and replace tissues offer great opportunity. The advent of antibiotics, the use of vaccinations – these are developments whose effects have resounded across the world, improving the lives of hundreds of millions of people. We could well be at the threshold of a similar leap forward with the use of stem cells to understand, prevent, and treat or cure a wide array of diseases, including several types of cancer, Parkinson’s disease, heart attacks, and even bone fractures. The area is already moving quickly from the research laboratory to the clinic, and clinical trials at URMC of new stem-cell therapies for fatal diseases within two years are feasible.
Stem cells are essentially early-stage cells capable of generating all the different tissues found in the body. They provide a potential bounty of cells that could be customized to treat, in theory, almost any disease. More than 200 people – many considered world leaders in their field – in in approximately 40 laboratories already work on stem cells – many making the research area one of the most pervasive at the Medical Center. Exciting programs exist in neuromedicine for treating Parkinson’s disease and spinal cord injury; in cancer, including leukemias and prostate malignancies; and within musculoskeletal specialties which are looking to regenerate bone and cartilage. In addition to existing resources, several additional top investigators will be recruited as part of a new Stem Cell and Regenerative Medicine Institute.
URMC researchers are at the forefront of developing the tools needed to pinpoint such cells, control their development, and customize their use for several diseases. Our researchers are among the few who have discovered stem cells and worked out their complicated lineage. They are also among the first to display the capability of manipulating them to the great extent necessary to treat disease effectively. A symphony of molecular signals normally directs their development, and URMC scientists are teasing out the myriad chemicals, proteins, nutrients, and other biochemical signals that affect the process.
Within the nervous system, the most provocative research currently underway at URMC is aimed at a group of fatal children’s diseases known as pediatric leukodystrophies, where myelin – a coating around nerve cells – is damaged or missing. Scientists have shown that with careful manipulation, stem cells can evolve into cells that make new myelin, offering a potential method to treat diseases that are currently untreatable, including Krabbe’s and Tay-Sachs diseases. In related work, URMC scientists have used neural stem cells to replace the precise types of cells lost in patients with Parkinson’s and Huntington’s diseases, as well as to repair nerves damaged as the result of spinal cord injury.
Cancer research is another area where stem cells both hold tremendous promise and have already realized important scientific developments. In one project at URMC, scientists have learned how to exploit their knowledge of stem cells to learn how drugs commonly used to treat cancer can actually damage the brain; the scientists use stem cells as a sort of early warning system to monitor the effects of drugs and other compounds on the body. Other URMC scientists are developing the first cancer therapy that specifically targets the stem cells that play a role in causing leukemia; that therapy is expected to be tested in patients with lymphoma and multiple myeloma as well. And another team has identified for the first time a specific type of stem cell in the brain that is at the root of some brain tumors. Such work opens the possibility of stopping cancer at its roots, as well as improving the care of patients who already have the disease. Many clinical trials in this area are expected to occur over the next two years.
Beyond these applications, stem cell and regenerative medicine permeates research and treatment options in many areas. Stem cells hold the potential for actually growing new blood vessels, a treatment that goes to the core of conditions like heart failure and coronary artery disease. Our researchers have made several discoveries about the way that stem cells create the blood, potentially leading to new ways to expand the supply of cells such as red blood cells in patients. Their colleagues have discovered a crucial role for stem cells in bone healing and regeneration by pursuing in the laboratory the finding that increased levels of lead interfere with fracture healing.Stem cell research draws upon several existing strengths at the Medical Center and is central to several other initiatives highlighted in the current strategic plan. Stem cell and regenerative medicine has direct, immediate relevance to diseases like cancer, heart disease, conditions of the skeletal system, and diseases of the brain and spinal cord. Our growing knowledge about stem cells complements developments in other scientific areas, such as imaging and nanotechnology, where the institution also has particular strengths.
Stem Cell and Regenerative Medicine
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