Past Recipients - 2005

Columbus Children's
Columbus, OH
Rachel A. Altura, M.D.
Hugo Caldas, BSc, Ph.D. (fellow)

Drs. Altura and Caldas are evaluating survivin-based therapies in pediatric solid tumors. Survivin is a gene that is abnormally activated in tumor cells, but silent in most normal cells. Drs. Altura and Caldas hope to identify the portion of the survivin gene that acts as a switch to promote tumor cell growth – and to engineer a "suicide gene" with the power to induce cell death in tumor cells, without affecting these same pathways in normal cells.

Children's Healthcare of Atlanta
Atlanta, GA
Amy Lynn Baxter, M.D.

While pediatric pain has been well studied, nausea has not received the same attention. Research has found that given a choice most adults would choose limited pain over nausea. No one has tested this in children, in part because there is no scale to determine relative degrees of nausea like those scales available for pain. For pediatric patients receiving chemotherapy, the implications of nausea reach beyond suffering to threatening good nutrition and affecting compliance to therapy. Dr. Baxter is hoping to develop a tool to determine incremental differences in nausea levels. Once validated, this nausea scale will be available to prompt staff to assess for nausea, determine effectiveness of anti-nausea drugs, investigate the correlation between nausea and nutrition, and test whether different medications are more effective for different patients.

Medical College of Wisconson
Milwaukee, WI
Kristen Bingen, Ph.D.

Little is known about the long-term impact of pediatric hematopoietic stem cell (bone marrow) transplantation (HSCT)—a lengthy and intensive cancer treatment regimen that places physical and psychological demands on the child and family. Children who receive a HSCT are isolated from their friends and school for a long period of time. They also receive intensive chemotherapy and all over body radiation, which has shown to negatively impact learning and cognitive abilities for childhood leukemia and brain tumor patients. Dr. Bingen is studying the cognitive and psychosocial impact of HSCT in long-term survivors. She hopes to better understand why some patients are at risk for long-term cognitive or adjustment problems, and improve educational and psychosocial interventions for pediatric HSCT survivors.

Dana-Farber Cancer Institute
Boston, MA
Ranie E. George, M.D., Ph.D.

Neuroblastoma is a tumor of the developing sympathetic nervous system and is the second most common solid tumor of childhood after brain tumors. Treatment of advanced neuroblastoma in older children remains a major challenge. In the last decade treatment has improved survival; however relapse is common in older children and is nearly always fatal. Dr. George is taking a closer look at the factors that might predict early relapse and identify abnormalities that distinguish tumors that continue to grow from those that might stay in remission. This includes abnormalities that make some tumors drug resistant and tumors whose inactivation could lead to uncontrolled growth of cells.

Huntsman Cancer Institute
Salt Lake City, UT
Stephen L. Lessnick, M.D., Ph.D.

Little is known about the origin of Ewing's Sarcoma, a highly malignant pediatric solid tumor cancer. This cancer is characterized by specific genetic abnormalities, called chromosomal translocations, which fuse two chromosomes together. We know that this chromosomal fusion generates what is called EWS/FLI fusion proteins found only in the tumor, not in normal tissue. The protein turns other genes on and off inappropriately. This activity appears to be critical for Ewing's Sarcoma development. Dr. Lessnick is investigating the key factors that may turn EWS/FLI genes on and off to better understand how Ewing's Sarcoma develops, and how it can be more effectively treated.

Memorial Sloan-Kettering Cancer Center
New York, NY
Shahin Rafii, M.D.
Matthias A. Karajannis, M.D. (fellow)

In leukemia patients, the growth of leukemic cells is dependent on soluble growth factors, as well as direct cellular contact with non-leukemic cells, called stromal cells, in bone marrow and other tissues. Drs. Rafii and Karajannis are investigating the molecular mechanisms that regulate this environment and interactions between non-leukemic cells. This information may help researchers develop novel therapies that will specifically target and attack the environment that fosters growth of cancer cells, especially those leukemic cells that resist treatment, and for disease recurrence.

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

Dr. Rood's study is helping to clarify the role of HIC-1 in the formation of brain tumors, the most common cause of cancer-related death in children, and the most common solid tumor of childhood. HIC-1 is a tumor suppressor gene that plays an important role in childhood brain tumors, including medulloblastoma. Tumor suppressor genes normally control cellular production. When tumor suppressor genes are inactivated by various causes, normal growth controls are stopped, resulting in the development of tumors. This research will help to facilitate the identification of therapeutic targets and new drugs to stop specific genes or molecular receptors that conspire to inactivate HIC-1 in a large proportion of childhood brain tumors.

Texas Children's Cancer Center and Hematology Center
Houston, TX
Jason M. Shohet, M.D., Ph.D.
Andrew Slack, Ph.D. (fellow)

Neuroblastoma is the second most common pediatric solid tumor (after brain tumors) and represents a major therapeutic challenge. More than 50 percent of children with advanced disease still die despite aggressive, toxic chemotherapy regimens. Unlike pediatric leukemia, survival for advanced stage neuroblastoma has only marginally improved over the past decade. Drs. Shohet and Slack will examine the gene MYCN, which has been identified as a major prognostic factor for aggressive disease and poor survival. Better characterization of the proteins regulated by MYCN will provide both insight into the biology of this tumor, as well as provide preclinical data for development of novel 'molecular targeted' approaches to neuroblastoma therapy and improved outcomes.

Stanford University
Stanford, CA
Irving L. Weissman, M.D.
Daniel Kraft, M.D. (fellow)

Children with recurrent or aggressive cancers often undergo high doses of chemotherapy and\or radiation, followed by bone marrow transplantation (BMT) in an attempt to stop the cancer. Unfortunately, the most common cause of death for these children is relapse from their primary cancer. The disease often returns after BMT from cancer cells that have survived the transplant. This research examines strategies to improve the ability of the patient's new immune system to kill any residual cancer cells that survive the transplant and lead to relapse.

Children's Hospital & Research Center Oakland
Oakland, CA
Joseph Wiemels, Ph.D.
Patrick Chang, M.D. (fellow)

Although the majority of children with Acute Lymphocytic Leukemia (ALL) are cured, treatment of relapsed ALL remains a significant problem. Having the capability to categorize patients at low risk or high risk of relapse would improve treatment results. Drs. Wiemels and Chang are investigating FLT3, a genetic mutation that may be an indicator of poor prognosis in pediatric ALL. Recent studies have found that an FLT3 mutation does predict a poor prognosis for patients with Acute Myelogenous Leukemia (AML). Now this research will help clarify its significance in childhood ALL — information that may lead to the use of FLT3 inhibitor therapies that offer far fewer side effects than chemotherapy and directly targets cancer cells.

Vanderbilt University
Nashville, TN
Elizabeth Yang, M.D., Ph.D.
Viviana A. Lavin, M.D. (fellow)

Drs. Yang and Lavin are studying an abnormal protein called Shp2 that is found in pediatric patients with Juvenile Myelomonocytic Leukemia (JMML), a rare form of leukemia, which is almost always fatal. In recent studies, investigators have discovered that this protein has been found in patients with Noonan Syndrome, an inherited disorder that occurs in one out of every 1000 to 2500 children. Further understanding of the cause of this gene mutation will not only benefit in the treatment of JMML children, but could also help a wider spectrum of leukemias with mutations in this gene.

Texas Children's Cancer Center and Hematology Center
Jianhua Yang, Ph.D.

In aggressive neuroblastoma, a pediatric solid tumor cancer, growth characteristics of tumor cells are determined by a number of factors, including stop and go signals they receive from hormones and other biochemicals in the environment outside the cell. The focus of this project is to study the action of a signaling protein called neuroblastoma specific phosphatase (NSP) active in neuroblastoma tumor cells. Dr. Yang will evaluate the impact of disabling NSP in these tumor cells and explore the usefulness of blocking the action of NSP as a type of anti-cancer therapy.

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