5th Annual Golf for a Cure Classic  


Monday, October 06, 2008
Curt I. Civin, M.D.
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Dr. Curt Civin
Johns Hopkins University
Baltimore, Maryland

"Transduction of Human Hematopoietic Adult Stem Cells"

Bone marrow transplants have become an important treatment option for many kinds of cancer. In this procedure, high doses of chemotherapy or radiation are administered to help destroy cancer cells. However, these powerful treatments also decimate bone marrow, requiring a transplant so the patient's body can produce new, healthy blood cells.

By identifying the parent blood cell, called a stem cell, responsible for the formation of all types of blood and immune system cells, researchers and physicians have been able to improve the effectiveness and reduce the side effects of traditional bone marrow transplants.

In fact, when parent stem cells from the blood, rather than from the bone marrow, are used for the transplant, they regenerate the blood supply faster for the best possible results. In addition, the use of purified stem cells reduces the likelihood that there will be a dangerous immune system rejection of the transplanted marrow, making this procedure more available to patients with a range of cancers and immune system disorders.

Isolating this parent blood cell has other benefits, too. Most important of all, it allows researchers to study how these cells divide to re-create new copies of themselves. This opens the possibility that these cells can be manipulated or altered to correct and prevent serious diseases. Called gene therapy, this technique may provide a way to switch off cancer and other diseases before and even after they take hold.

Project Director Expertise

In 1982, Dr. Curt Civin, an oncologist at Johns Hopkins Hospital in Baltimore, Maryland, discovered a way to identify stem cells, isolating them in a series of laboratory experiments. His groundbreaking stem cell research has resulted in new tools in use today for diagnosing leukemia and purifying stem cells for research and clinical stem cell transplantation

NFCR Research Overview

Once he had isolated stem cells, Dr. Civin hypothesized they might have a unique property on its surface, called an antigen. To identify this antigen, he developed an antibody, or cellular defense mechanism, that would target the antigen when it was introduced into the cell.

NFCR Research Findings

Dr. Civin's current work as an NFCR Project Director focuses on developing a better understanding of how stem cells function. By learning how to stimulate the renewal process of the stem cell, Dr. Civin hopes to develop a way to produce a significant supply of donor stem cells for cancer patients in need of specialized bone marrow transplants.

This new and potentially life-saving form of transplant, called a stem cell graft, allows a physician to administer higher and more effective doses of chemotherapy or radiation, while reducing the threat that the new marrow will be rejected by the patient's immune system.

As an NFCR researcher, Dr. Civin has also determined that stem cells can be used to correct genetic diseases of the immune system, such as severe combined immunodeficiency disease (SCID), a genetic disorder caused by a missing enzyme that shuts down the body's ability to combat disease. A stem cell graft that includes donor stem cells and the missing enzyme can replace the defective cells with healthy cells that will repopulate the patient's blood supply.

Future Research Goals

Dr. Civin is working to pinpoint the moment when stem cells divide by studying what triggers the cell renewal process. He also is researching what genes serve as triggers to fight disease, in the hope that he can insert selected genes into the dividing cell. This would allow him to program stem cells to accomplish specific tasks, such as disabling the process that causes the human cell to become cancerous.

Furthermore, these genetically engineered stem cells could enable molecular surgery, a technique that would allow physicians to direct genes to fight disease. For example, physicians could boost the immune system to fight cancer or replace defective genes to alter the course of a blood-based genetic disease. This would reduce the need for chemotherapy and radiation, which causes many side effects and destroys healthy tissue. Initial experiments in test tubes and with laboratory mice show promise.

Impact on Cancer Prevention, Treatment, or Cure

Dr. Civin's work has far-reaching implications for cancer patients. First and foremost, Dr. Civin's research provides hope for more effective treatments with fewer side effects. Meanwhile, the stem cell grafts he has pioneered minimize the risk of reintroducing cancerous tumor cells that have metastasized or spread to the bone marrow. This is often the case in solid tumors in children, breast cancer, small cell lung cancer, lymphoma, and ovarian cancer.

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