Past Recipients - 2004

Texas Children's Cancer Center
Baylor College of Medicine
Houston, TX
Malcolm K. Brenner, M.D., Ph.D.
Raphael Rousseau, M.D., Ph.D.

Drs. Brenner and Rousseau are developing a vaccine to fight leukemia in patients whose disease recurs or does not respond to conventional treatment. The vaccine is created in part from a sample of the patient's own skin. From this tissue sample, fibroblast cells are created. The researchers inject specialized genes into these cells to promote the production of natural cancer fighting substances. Then the patient's own leukemia cells – treated to prevent them from growing – are introduced into the mix and the vaccine is injected into the patient. The cancer cells help the vaccine recognize and destroy the tumors. Similar vaccines have shown to be effective and this research will help to develop a safe approach to fight difficult to treat childhood leukemia.

Texas Children's Cancer Center
Baylor College of Medicine
Houston, TX
Alison Ann Bertuch, M.D., Ph.D.

Osteosarcoma is the most common bone cancer in children and teens and just 30 percent of patients diagnosed are likely to survive long term. Dr. Bertuch is studying the molecular changes that lead to the disease, and she is focusing on telomeres—the very ends of chromosomes. Previous research indicates that telomeres may play a role in promoting osteosarcoma. She hopes this research will clarify why and how telomeres malfunction to promote osteosarcoma. This information may lead to better, more targeted treatment.

Children's Hospital of Philadelphia
Peter Adamson, M.D.
Jeffrey Skolnik, M.D. (fellow)

Actinomycine-D (Act-D) is a chemotherapeutic drug frequently used to treat childhood cancers, such as Wilms' Tumor and a tumor of muscle, called rhabdomyosarcoma. Little is know about how the drug works in the body. Although it is an effective treatment for some children, it can cause side effects including serious liver damage. Drs. Adamson and Skolnik are using new technology that allows them to study the drug's effect in minute quantities of blood. The information they obtain will improve dosing of Act-D in infants and young children, eliminating life-threatening side effects.

David Geffen School of Medicine at UCLA
J. Daniel Ozeran, M.D., Ph.D.

Dr. Ozeran is studying a specific abnormality in chromosomes that may be responsible for many cancers including Ewings Sarcomas, a family of cancers found in young children. Cancer results when a mutation in the genes causes the uncontrolled replication of cells. One type of mutation is called chromosomal translocation, in which the DNA of one chromosome breaks off and attaches to another chromosome. This causes the production of a protein that triggers the development of cancer. Dr. Ozeran is studying this action so that more effective drugs can be created to block this protein.

Duke University

Rhabdomyosarcoma is cancer of the muscle that affects children and teens. Previous research has shown that an unusual arrangement of DNA called the Pax3-FKHR gene seems to turn on the growth of muscle cells that should stop working in the fetus. It also causes the loss of an important protein known as p 16. Cancer often results from the loss of p16. In this research, Dr. Linardic is hoping to answer key questions about the protein and the gene—information that may lead to novel therapies for rhabdomyosarcoma.

Indiana University
Karen E. Pollok, Ph.D.

High dose chemotherapy is often the only way to treat childhood cancer of the central nervous system. But the chemotherapy can also destroy stem cells in bone marrow, which are critical in protecting cancer patients from anemia, bleeding disorders and life-threatening infections. Dr. Pollok is testing a chemotherapy-resistant gene that when introduced into stem cells can help protect them against harsh drug treatment. This has been proven to work in the lab and will now be tested in children with neuroblastoma and with children and adults with brain tumors.

Mayo Clinic/Rochester
Cynthia J. Wetmore, M.D., Ph.D.

Brain tumors are the most common solid tumor in children, yet little is known about how genetics contribute to their development. Treatment often leaves children with serious side effects such as developmental problems and seizures. And if the cancer recurs, most patients will not survive. Using a mouse model, Dr. Wetmore is studying the genetic mutations and biology of the cells that help promote tumor growth, so that future treatment will be more effective, with better long-term outcome for patients.

Memorial Sloan-Kettering Cancer Center
Neal Rosen, M.D., Ph.D.
Christine Pratilas, M.D. (fellow)

Cancer often results from the abnormal activity of proteins, which control the replication of cells. Two such proteins, Ras and Raf, are the targets of Drs. Rosen's and Pratilas' research. They hope to identify which tumors require Ras and Raf to grow and spread. A number of childhood cancers have mutations of these proteins. This research is examining drugs to inhibit their action, which may lead to clinical trials of these medications for pediatric cancers.

University of Hawaii
Randal K. Wada, M.D.
Matthew C. Tuthill, Ph.D. (fellow)

Neuroblastoma is often diagnosed after it has spread and when it can't be cured. Previous research has shown that patients treated with retinoic acid (RA) more than double their chance for survival. RA makes the cancer cells mature and turn into non-cancerous nerve cells. It appears that the RA shuts off a gene called N-myc that contributes to the cancer. This research is examining RA in cancer cells, as well as neuroblastoma cells that seem to resist RA. This information will help doctors better determine with patients will benefit from RA therapy and which may do better with other treatments.

Washington University
Joshua B. Rubin, M.D., Ph.D.

Brain tumors still kill many adult and children each year. Nearly 80 percent of pediatric patients with two cancers (medulloblastoma and glioblastoma), but long term side effects can be significant. This research is examining a protein called CXCR4, which appears to play a role in the growth and spread of these tumors. They hope to determine the role of CXCR4 in tumor development and see if it is a good target for treatment. They will also test a drug called AMD 3100 to determine if it can inhibit CXCR4 action and/or promote the natural death of tumor cells.

Washington University
William J. Grossman, M.D., Ph.D.

The body's own immune system can be an important weapon against disease including cancer. Dr. Grossman is investigating one type of immune cell called the T regulatory cell, which has been shown to be important in killing cancer cells. They also play a role in preventing a major complication of bone marrow transplantation. It is not yet clear how T regulatory cells work—and recent evidence indicates that there is more than one type. This research is examining these immune cells and how they may be manipulated or grown and used to fight various pediatric cancers.

Yale University
Xiaomei Ma, Ph.D.

Leukemia is the most common cancer in children, and acute lymphoblastic leukemia (ALL) is the affect 80 percent of pediatric leukemia patients. Understanding risk factors for ALL will help in the development of preventive interventions. Using data collected from a large epidemiological study of both leukemia patients and a control group (NCCLS), Dr. Ma is hoping to determine whether conditions related to the immune system (vaccinations, allergy, for example) or a specific group of genes are related to ALL.

City of Hope National Medical Center and Beckman Research Institute
Sunita Patel, Ph.D.

A childhood cancer diagnosis puts enormous strain on the entire families. Some families cope better than others, and those who have more difficulty are at greater risk for continuing stress many years past the diagnosis. This research examines a novel approach to identifying these stresses early and developing effective interventions in a practical clinical setting. Dr. Patel believes that one type of intervention will not work with all families and that a personalized, multidisciplinary approach will produce the best results. She will test this theory and study families' perceptions of its helpfulness so that better programs to meet the psychosocial needs of cancer patients and their families can be developed.

Children's Research Institute/Children's Hospital of Columbus
Kathryn J. Klopfenstein, M.D.

While many children survive cancer today, it still remains the number one killer of children by disease in the United States. Relief of pain and suffering — palliative care—is critical but is not at a high standard of quality across the country. This study is developing a team approach to palliative care to help guide patients and their families through a difficult process when they are faced with a terminal illness. The program includes training of physicians (interns, residents and fellows) whose previous education has not included palliative care. Through this program, a cadre of effective “patient advocates” will be developed whose primary responsibility is the well-being of the patient.

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