Grants Search Results

Current therapies for neuroblastoma include the use of powerful chemotherapy, which weakens the immune system and can lead to life- threatening infections. As a result, these immune-compromised patients frequently require medication such as granulocyte colony-stimulating factor (GCSF), which helps the body produce white blood cells to help fight infection. In many adult cancers, GCSF has been found to increase the growth of cancer cells in a laboratory setting. Dr. Kim is studying how GCSF and GCSF-R enhance tumor growth, to clarify the appropriate use of GCSF in patients and determine whether GCSF- receptor may be a new therapeutic target in neuroblastoma.

Neuroblastoma is a solid tumor cancer of very young children, originating in the nerve tissue of the neck, chest, abdomen, or pelvis, but most commonly in the adrenal gland. About 45% of children diagnosed have advanced "high risk" disease, for which the survival rate is less than 40%. This project tests the new hypothesis that specific ceramide types and/or expression of sphingolipid enzymes control the growth and invasion of neuroblastoma. The role of a particular family of enzymes called ceramide synthases will also be examined. The goal is to develop new therapeutic strategies for the treatment of neuroblastoma.

Hodgkin lymphoma is a cancer of the immune system and is the most common cancer for children ages 15-19. It arises from Reed-Sternberg (RS) cells, which have two or more nuclei and often have gained or lost chromosomes. This research explores the workings of a protein called KLHDC8B, which is expressed during cell division and is altered in cases of familial Hodgkin lymphoma. Dr. Krem also uses blood and tissue samples from patients to find changes in proteins that are related to KLHDC9B. Those other proteins may be important for preventing onset of Hodgkin lymphoma.

Precursor B-lymphoblastic leukemia is a type of blood cancer in which too many immature white blood cells are found in the blood and bone marrow. It is the most common type of acute lymphoblastic leukemia (ALL). This project studies how specific B-precursor ALL cells are wired and how to disrupt this wiring by treating patients with specific and novel medications. The research improves our understanding of the biochemical mechanisms critical for the development of targeted therapies.

In 2009, the St. Baldrick's Foundation awarded a grant to the Pediatric Blood and Marrow Transplant Consortium (PBMTC) to construct a clinical trials infrastructure that would allow high-quality, appropriately monitored, multi-center pediatric trials. This grant,awarded in 2010, moves that project further towards its goals of: 1) increasing safety for pediatric patients after transplant by using a new medicine that has been shown in adults to treat leukemia with lower toxicity, 2) reducing relapse by giving immune therapy before and after transplant to leukemia patients, and 3) exploring the feasibility of new cellular therapy approaches aimed at preventing relapse.

Rhabdomyosarcoma, the most common soft tissue cancer in children, has two main forms; each behaves differently, so recognition is important for proper treatment. This research builds on previous studies that identified unique genes associated with one form. The proteins from these genes can be used to create special stains that are both affordable and accessible to hospitals that don't perform gene studies. This enhances our understanding of the biology of rhabdomyosarcoma, allow rapid identification of high risk patients who may benefit from specific therapies, and prevent overtreatment of patients with low risk tumors.

Pediatric patients who have bone marrow transplants have an impaired immune system, and the resulting infections can cause bacterial, viral and fungal infections and even death. Unfortunately, medicines to treat these infections are not adequate in many cases; an intact immune system is needed to achieve appropriate responses to infectious agents. The goal of this research is to expedite the immune recovery after transplant. This would result in better responses to infections and improvement in the lives and survival of patients. Dr. Stefanski was a St. Baldrick's Fellow and now has a faculty position.

A type of brain tumor called diffuse intrinsic pontine glioma (DIPG) has no known cure. Radiation therapy offers some temporary relief, but nearly all children die from this cancer within 1 year. A promising form of drug delivery, convection-enhanced delivery (CED), offers many benefits including allowing high concentration of drugs to reach the brain tumor. This study focuses on drug distribution following this new form of drug delivery. By relating drug distribution and radiation dose to tumor response, a better treatment can be designed. Resulting clinical trials for a new therapy may eventually cure DIPG.

Ewing sarcoma (ES) is one of the most common sarcomas in children, caused by a genetic abnormality called a "chromosomal translocation." This study has two goals. First, to understand the normal function of the protein EWS, to learn about what goes wrong in Ewings tumors. Second, to understand what other genetic events are required to turn normal human cells into Ewing sarcomas. Our approach is to try to combine EWS/FLI-1 expression with other genetic events in the type of human cell that we know gives rise to ES.

This grant is to the New Approaches to Neuroblastoma Therapy (NANT) consortium, to develop and test new therapies with high potential for improving survival. NANT links laboratory and clinical investigators to develop therapies that are tested at 15 North American neuroblastoma centers, supported by the NANT Operations Center at Children's Hospital Los Angeles. The strategy includes accurately evaluating response with "biomarkers" for tumor cells in blood and bone marrow combined with sophisticated imaging of tumors, essential for "personalized treatment" to predict if the treatment will benefit the patient. NANT studies enable definitive testing later in larger patient numbers. Funds Administered by the Children's Hospital Los Angeles.

Since the majority of children with acute lymphoblastic leukemia (ALL) will survive, understanding how to limit the acute and long-term toxicities of treatment is increasingly important. Toxicities of the bone, including fracture and osteonecrosis, are serious complications. Dr. Vrooman's project accesses how bone mineral density changes in children being treated for ALL, so future interventions may decrease skeletal morbidity in these children. She is funded by P.A.L.S. Bermuda with funds raised through the St. Baldrick's Foundation.

While current therapies have greatly improved the overall survival of children with leukemia, patients with acute myeloid leukemia (AML) still have a poor outcome. This research focuses on determining the role played by the CALM gene in the onset of leukemias. These studies improve our understanding of how CALM-rearranged leukemias develop and contribute to the development of novel therapeutic strategies to fight childhood leukemias.

Generously sponsored by Markit. Treatment for acute lymphoblastic leukemia can be particularly devastating to a child's developing brain, leading to deficient short-term memory and attention or more serious events like seizures or strokes. This research focuses on how the cough medicine dextromethorphan may help reverse severe, stroke-like neurotoxicity among children treated with chemotherapy drug methotrexate and could possibly also prevent such side effects before they occur. The most promising drugs will be rapidly advanced into clinical trials for children with leukemia, designed to decrease the neurotoxic effects of chemotherapy.

Cancer affects every part of patients and their families' lives. Self-image, thinking clearly, anxiety, depression, social isolation and fear of recurrence are all problems that teens and young adults with cancer face, while resuming normal development and being monitored for many problems caused by cancer and its treatment. This research is to create developmentally sensitive information and make it available through touch-screen technology, connecting teens and young adults with their health care team and community resources in real-time. This helps identify, communicate and maximize the benefits of clinical care while helping them reintegrate into school and work.

Fatigue is a pervasive, distressing symptom for children and teens with cancer. Decreasing fatigue and improving physical activity are important to provide energy for the normal activities of childhood that are important to ongoing development. A small, cost-effective device called the FitBit measures motion and provides daily feedback to the wearer. Children ages 6 to 18 in maintenance treatment for acute lymphoblastic leukemia use the FitBit, and researchers determine if it is accurate, if it helps childhood cancer patients to be more active, and if more active patients have less fatigue when getting steroids during treatment.

Little is known about the long-term impact of communication about prognosis on children with cancer and their families. The goal of this project is to increase parents' ability to make value-driven decisions for care of their children with cancer and to adjust to this life-changing event in the best way possible. A longitudinal observational study evaluates long-term outcomes, using questionnaire-based parent interviews to assess parental decision- making and psychological and medical outcomes of disclosure. The cohort involves 194 children diagnosed between April 2003 and May 2005, so long-term assessment of their outcomes is now possible. Dr. Mack is funded by P.A.L.S. Bermuda with funds raised through the St. Baldrick's Foundation.

This grant funds a first year medical student to work in a pediatric oncology research lab for the summer, perhaps encouraging the future choice of childhood cancer research as a specialty.

This grant helps fund a Presidential Symposium focused on medical education, an essential subject matter for physicians.

This grant funds a first year medical student to work in a pediatric oncology research lab for the summer, perhaps encouraging the future choice of childhood cancer research as a specialty.

This grant funds a first year medical student to work in a pediatric oncology research lab for the summer, perhaps encouraging the future choice of childhood cancer research as a specialty.