Grants Search Results

Most cancer therapies have significant toxicity, thus new treatment strategies are needed. Pediatric patients with cancer are excellent candidates for immunotherapy because their immune system is more robust compared to adults. Due to our lack of understanding of how to best activate these specialized anti-cancer cells, progress in pediatric immunotherapy has lagged behind. This research focuses on how we can best activate specific T cells to defend the immune system against tumors, specifically gliomas (brain tumors) and advances the field of immunotherapy as a promising form of treatment for these children.

Rhabdomyosarcoma is the most common cancer that originates in the soft tissue of the body in children. There are two subtypes, embryonal (ERMS) and alveolar (ARMS), and children with ARMS have poorer response to conventional chemotherapy and radiation therapy, and much lower survival rates than those with ERMS. This research aims to discover chemical inhibitors of a gene called PAX3-FKHR and how "knocking down" that gene may help patients respond better to chemotherapy.The goal of this project is to establish a new approach for developing drugs to effectively treat ARMS.

The human body has an ability to detect and eliminate cancer through the immune system, but cancer cells can escape detection. Dr. Curran's research attempts to overcome this tumor escape via gene therapy mediated treatments. Pediatric leukemia is the most common childhood cancer, and patients with recurrent or resistant leukemia have limited options for treatment. Redirecting the immune system to eradicate resistant leukemia cells will provide a new possibility for a cure. Also, by specifically targeting cancer cells, we eliminate the long term complications associated with the conventional treatments of surgery, chemotherapy, and radiation. Dr. Curran was a St. Baldrick's Fellow and now has a faculty position.

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.

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.

Based on progress to date, Dr. Federman was awarded a new grant in 2012 to fund an additional two years of this Scholar award. We already have many powerful drugs to treat cancer but lack the means to deliver them directly to the intended targets (cancer cells), and as a result, cancer patients suffer significant side effects. Dr. Federman tests new ways of delivering anti-cancer treatments more directly using nanoparticles programmed to recognize particular cancer cells.

Based on progress to date, Dr. Mulcahy-Levy was awarded a new grant in 2011 to fund an optional third year of this fellowship. Understanding how cancer cells die is important in designing new therapies and improving the effectiveness of currently used therapies to cure childhood cancers. One element of cell death is called autophagy, a system the cell uses to break down as it dies, the focus of Dr. Mulcahy-Levy's project.

Based on progress to date, Oren Becher, M.D., AmWINS St. Baldrick's Scholar, was awarded a new grant in 2012 to fund an additional two years of this Scholar award. Brainstem glioma is a rare subtype of brain tumor found mostly in children, which cannot be cured with today's treatments. Dr. Becher’s research suggests that one major obstacle for progress in the treatment of these brain tumors is limited drug delivery due to the blood-brain-barrier, a protective mechanism that prevents the delivery of toxic chemicals into our brains (in this case- the cancer drugs). Dr. Becher is working with genetic models to find new ways to improve the delivery of cancer drugs to these brain tumors.

This grant is named for AmWINS, a wholesale insurance holding company, which has raised more than $1 million for childhood cancer research through the St. Baldrick's Foundation.

Acute myeloid leukemia (AML) is a potentially deadly form of childhood leukemia. Dr. Vaiselbuh is studying how AML cancer cells resist chemotherapy with the goal of finding a new strategy for treatment of childhood myeloid leukemia.

Based on progress to date, Dr. Volchenboum was awarded a new grant in 2012 to fund an additional two years of this Scholar award. Neuroblastoma strikes in many forms, some requiring little or no therapy, others deadly despite very aggressive treatment. Current tests that differentiate among these types can take weeks. Dr. Volchenboum is developing software to make real-time diagnosis possible, allowing therapy to be better tailored to the specific child. Once validated, these new technologies can be extended to other pediatric cancers.

This grant funds a cutting-edge research project to find new and better cures for childhood cancer.

This grant funds a cutting-edge research project to find new and better cures for childhood cancer.

Based on progress to date, Dr. Castellino was awarded a new grant in 2011 to fund an additional two years of this Scholar award. Medulloblastoma is the most common invasive brain tumor in children. Current treatments do not cure a lot of children and cause significant side effects. A better understanding of what causes this tumor to develop and to spread is needed in order to develop more effective therapies. A specific genetic alteration is frequently found in human medulloblastoma tumor samples which involves overexpression of a protein called WIP1, which is a negative regulator of cell death. Dr. Castellino's research on the inhibition of WIP1 may find a potential therapeutic approach to increase response to chemotherapy in medulloblastoma patients.