Past Recipients - 2002

Baylor College of Medicine
Houston, Texas
Jason Shohet, M.D., Ph.D.

Dr. Shohet's research focuses on one gene called MYCN found in great quantities in neuroblastoma cancer cells. Neuroblastoma, one of the most common solid tumor cancers in children, affects the nervous system and has a high death rate. The MYCN gene causes normal cells to become cancerous cells and is aided by a molecule called MCY7. This research hopes to better understand how the MYCN gene and MCY7 interact with one another to promote cancer development – information that may lead to development of more effective treatment targeted to this and other dangerous genes.

Columbus Children's Hospital and Children's Research Institute
Columbus, Ohio
Rachel Altura, M.D.

In this investigation, Dr. Altura is studying the gene called survivin. The gene appears to interfere in the treatment of cancer by making cancer cell growth response more susceptible to mutations and therefore more resistant to treatment. With a clearer understanding of survivin, it may be possible to design better chemotherapies for pediatric cancers that target these genes in tumor cells.

Children's Hospital of Los Angeles
Los Angeles, California
Sakurako Yamazaki, M.D (fellow) and C. Patrick Reynolds, M.D., Ph.D.

Drs. Reynolds and Yamazaki are investigating the effectiveness of a form of retinoic acid (a derivative of vitamin A) against neuroblastoma. Retinoic acid has been found to stop the growth of neuroblastoma cells in the laboratory and improves survival of patients when it is administered following chemotherapy. Some tumors, however, are resistant to retinoic acid. These researchers hope to understand the genetics of those more difficult to treat tumors – information that may lead to clinical trials of retinoic acid in conjunction with a frequently used drug called fenretinide.

Children's Research Institute
Children's National Medical Center
Washington, DC
Brian Rood, M.D.

The MGMT gene exists within childhood brain cancer cells, making these tumors resistant to one type of chemotherapy agent known as alkylators. The MGMT gene repairs damage to the cancer cell's DNA, which is caused by the drug, rendering the chemotherapy ineffective. Dr. Rood is examining a process called methylation – which turns-off genes. A better understanding of how the MGMT gene can be turned off or inactivated will make alkylators more effective in the treatment of brain tumors. The drug could reduce or delay the use of radiation, which can cause a number of side effects.

National Cancer Institute
Bethesda, Maryland
Javed Khan, M.D.

Alvelor rhabomyoscarcomas (ARMS) are cancers of the skeletal muscle that occurs in adolescents and is often diagnosed in its later stages. Researchers have discovered that some of the chromosomes in the genes of these cancers break apart and reattach themselves to create a brand new gene not present in nature. It's called the PAX3-FKHR gene and is believed to be critical in the development of the cancer. Dr. Khan's study hopes to better understand this process so that early detection and more effective therapies can be developed.

Ohio State University
Columbus, Ohio
Mauro Ferrari, Ph.D., M.S. and Constanino Benedetti, M.D.

In this investigation, researchers are developing a new non-injection delivery approach to pain relief for pediatric cancer patients. They propose the use of an oral medication and a patch applied under the tongue that contain not only the pain medication, but also special particles genetically engineered by nano-micro technology. The particles adhere to the wall of the small intestine and enhance and control the delivery of the pain medication. Two systems are under development. One type would be used for rapid and short-term pain relief for procedures such as spinal taps – relief would occur within 60 seconds and last just minutes. Another type would provide fast-acting relief that would be maintained over several hours.

University of Alabama
Birmingham, Alabama
David Crawford, M.D., Ph.D.

Dr. Crawford is studying the mechanism called mitosis that controls the division of cells. In normal mitosis, there are checks and balances that control the replication of cells with damaged DNA. But in cancer cells this regulatory process does not occur. In his research, Dr. Crawford wants to gain a better understanding of the process and why it becomes aborted in pediatric cancer.

University of Minnesota
Minneapolis, Minnesota
James G. Gurney, Ph.D.

While neuroblastoma is a devastating cancer that kills hundreds of children each year, some patients survive after a series of difficult and lengthy treatments. This study hopes to evaluate the long-term effects of the disease and therapy on children and families. This information and other survivor research data will be combined to better understand the impact of different therapies on the children's future health, quality of life and the dynamics of their families.

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