Grants Search Results

Based on progress to date, Dr. Gershon was awarded a new grant in 2014 to fund an additional two years of this Scholar award. Medulloblastoma strikes the cerebellum, a brain region that sees rapid growth after birth, as special cells called progenitors divide repeatedly, increasing the number of brain cells. Gene mutations that allow unrestricted progenitor growth cause medulloblastoma. Understanding which genes control progenitors, and how these genes work together, may lead to new medulloblastoma treatments. Dr. Gershon is investigating a previously unknown connection between the immune system, brain growth, and the formation of brain tumors, and a novel way to use developmental biology to treat medulloblastoma.  

Based on progress to date, Dr. Kim was awarded a new grant in 2014 to fund an additional two years of this Scholar award. Despite the recent advances in chemotherapy for acute lymphoblastic leukemia (ALL), drug resistance often results in relapse of ALL. Preclinical studies have shown that leukemia cells can evade chemotherapy as they are protected by their bone marrow environment. This study proposes to dislodge chemo-resistant cells from the protective bone marrow making them vulnerable to chemotherapy. We propose to study the functional role of CD49d in drug resistant leukemia and will validate it as a potential, novel target for treatment of recalcitrant childhood ALL.

Based on progress to date, Dr. Mullighan was awarded a new grant in 2014 to fund an additional two years of this Scholar award. Acute lymphoblastic leukemia (ALL) is the most common childhood cancer and still the most common cause of cancer related death in children. This project uses cutting-edge genetic profiling approaches to identify all genetic alterations contributing to the pathogenesis of high-risk childhood leukemia. This project uses detailed genomic analysis coupled with the development of experimental models of ALL that examine the role of newly identified genetic alterations in the development of leukemia, and response to therapy.  

Based on progress to date, Dr. Wei was awarded a new grant in 2014 to fund an additional two years of this Scholar award. Dr. Wei and his team are using a genetic screen to study a novel candidate drug molecule's mechanism of action as an inhibitor of NAMPT, a key protein that regulates cancer cell metabolism. Their findings show that the molecule is effective against patient leukemia cells. Dr. Wei is working to better understand how this molecule works to kill leukemia cells and identify what are the genes and pathways involved, in hopes that it can be used to treat and cure patients with acute lymphoblastic leukemia.

Based on progress to date, Dr. Wolfson was awarded a new grant in 2014 to fund an additional two years of this Scholar award. Cancer treatments for young children and older adults have made incredible progress. However, adolescents and young adults (AYAs) diagnosed at 15 to 39 years old have not seen these same improvements, leaving an AYA Gap. Within this AYA group, racial/ethnic minority patients fare poorly, as do patients not receiving care at a nationally recognized cancer center. This study tests the theory that the AYA Gap is largely due to disparities in access to quality cancer care. Ultimately, the aim of this project is to help develop strategies to reduce these disparities in outcome and eliminate the AYA Gap. Awarded at the City of Hope and transferred to University of Alabama at Birmingham.

Based on progress to date, Dr. Raabe, the Hannah's Heroes St. Baldrick's Scholar, was awarded a new grant in 2014 to fund an additional two years of this Scholar award. Medulloblastoma is the most common malignant pediatric brain cancer, and patients with high-risk medulloblastoma have a poor prognosis. Unfortunately, few models for aggressive medulloblastoma exist, severely limiting our ability to test new treatments for the patients who need them most. The goal of this proposal is to develop accurate models of high-risk medulloblastoma for pre-clinical therapeutic testing. This allows for rapid progress to be made, as researchers can use these models to screen for new drugs, test drug combinations, identify for factors conferring resistance to chemotherapy, and look for pathways that might be an "Achilles heel" for high-risk medulloblastoma. This grant is named for the Hannah's Heroes Hero Fund created in honor of Hannah Meeson and pays tribute to her fight by raising awareness and funding for all childhood cancers.

Based on progress to date, Dr. Braun was awarded a new grant in 2014 to fund an additional year of this Scholar award. Approximately one-third of human cancers harbor mutations in RAS genes, causing malignant cells to proliferate inappropriately. In pediatric oncology, RAS mutations are particularly common in leukemias. Unfortunately, the RAS protein is a difficult molecule to attack with drugs. An alternate approach is to target other proteins that are required partners for RAS to exert its control. Some types of cancer are dependent on rare "cancer stem cell" populations with unique properties. This research is to discover how cancer stem cells are affected by mutations that activate RAS, and to use this information to devise novel therapies.

Based on progress to date, Dr. Dallas was awarded a new grant in 2013 to fund an additional year of this Scholar award. Hematopoietic cell transplantation is a potential cure for various pediatric cancers. Approximately one-third of patients who require a transplant do not have a suitable matched donor, and umbilical cord blood transplants are an increasingly utilized alternative, with over 20,000 performed since 1988. One of the major complications is the increased risk for serious infection due to the prolonged period of time the patient's immune system is suppressed after transplantation. Dr. Dallas and her team have developed a novel method to generate cells that will hasten the time to recovery. Their goal is to translate these findings to pediatric patients and improve their survival.

Based on progress to date, Dr. De Oliveira was awarded a new grant in 2014 to fund an additional year of this Scholar award. New therapeutic approaches are needed for pediatric leukemia and lymphoma, because patients with refractory or relapsed disease still have a survival rate of less than 50% with current therapies. This research involves a novel cancer immunotherapy protocol, transferring a gene into the patient's own blood stem cells, giving rise to immune cells able to directly and specifically target a surface molecule that is present in more than 95% of leukemias and lymphomas. This project evaluates the cancer cell destruction by the modified immune cells, setting a basis for future clinical trials.

Based on progress to date, Dr. Diede was awarded a new grant in 2013 to fund an additional two years of this Scholar award. DNA methylation is a normal process used by cells to allow information to be passed on to successive generations of cells. Cancer cells can exploit this to silence genes that help prevent tumor formation. This has been extensively studied in adult cancers, but not in pediatrics. Given the relatively short time frame in which pediatric cancers develop, aberrant DNA methylation may play a very important role. This research is to better understand how it promotes the formation of pediatric rhabdomyosarcoma, and may open an exciting new area for treatment and provide valuable biomarkers for cancer detection, diagnosis, and risk assessment.

Based on progress to date, Dr. Gamper was awarded a new grant in 2014 to fund an additional year of this Scholar award with generous support from the McKenna Claire Foundation. Chemotherapy and radiation destroy both cancer cells and normal cells, with toxic effects on growing children during treatment and afterwards. Immunotherapy has the potential to destroy only cancer cells, but it has not lived up to its full potential because cancer cells can promote inappropriate immune responses or simply turn immune cells off. This research examines the function of T cells that lack the ability to methylate DNA; such cells may be better at killing tumors. This may help more patients with high-risk pediatric tumors, and decrease the risk of late-effects by reducing the need for more chemotherapy and radiation.

Based on progress to date, Dr. Rabin was awarded a new grant in 2014 to fund an additional year of this Scholar award. Children with Down Syndrome have a 20-fold increased risk of developing acute lymphoblastic leukemia (ALL), and suffer significantly more frequent and severe complications associated with chemotherapy, including life-threatening infections. This research involves abnormal activity of genes called JAK2 and CRLF2 and new drugs. It will also investigate whether gene variants that are associated with severe infection in the general population occur more frequently in those with Down Syndrome and ALL who suffer severe infectious complications. If so, patients identified as high-risk for infection could be identified ahead of time, to receive enhanced supportive care to prevent severe toxicity. This grant is made with generous support from the “Daniel the Brave Fund" created in memory of Daniel Gomez to honor his bravery and provide hope for those still in the fight.

Based on progress to date, Dr. Singh was awarded a new grant in 2013 to fund an additional two years of this Scholar award. Diamond Blackfan anemia (DBA) is an inherited condition that leads to anemia, birth defects and cancer. Over-expression of the p53 protein, which protects against tumor formation, may actually lead to DBA. One of p53's functions is to cause cell death in damaged or stressed cells. Chronic over-expression of p53 may lead to an environment that leads to cancer transformation and survival. Understanding the conditions that promote the formation and survival of cancer cells is vital to improve early diagnosis and treatment of childhood cancer.

Leukemia is cancer of white blood cells and accounts for about 25% of all childhood cancers. Human chromosomes are normally subjected to various environmental and developmental challenges that might cause breaks, called translocations. While almost all of those breaks are efficiently repaired by the DNA repair machinery, translocations do arise from very rare events when mistakes occur during the repair, increasing the risk for leukemia in children. This study involves basic questions about the cause and process of translocation to better understand the underlying causes of leukemia, potentially leading to the discovery of targets for new therapeutic strategies.

Based on progress to date, Dr. Engel was awarded a new grant in 2013 to fund an additional two years of this Scholar award. Approximately one thousand children are diagnosed with acute myeloid leukemia (AML) each year in the United States, and only about 50% survive. Dr. Engel's research is to gain a better understanding of how normal blood cell development is altered in AML and how we can overcome these alterations to regain control over blood cell growth and development. Dr. Engel began his research at Vanderbilt University Medical Center and moved to the Huntsman Cancer Institute in 2010.

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, 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.

Based on progress to date, Dr. Chang was awarded a new grant in 2012 to fund an additional two years of this Scholar award. One subtype of pediatric leukemia that continues to have a poor prognosis is Philadelphia chromosome positive Acute Lymphoblastic Leukemia (Ph+ALL). Dr. Chang is researching a unique protein called survivin, in hopes of developing it as a new target for future therapy for Ph+ALL patients.

Based on progress to date, Dr. Lee was awarded a new grant in 2012 to fund an additional two years of this Scholar award. Natural killer cells (NK cells), one of the white blood cells of our immune system, have the ability to kill several types of cancers in children, including AML, neuroblastoma, osteosarcoma, and Ewing's sarcoma. Dr. Lee's research involves more effective ways to use NK cells to fight childhood cancers.

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.