Grants Search Results

Childhood acute myeloid leukemia (AML) is the second most common leukemia in children, but causes the majority of leukemia related deaths. Current therapies do not adequately treat the disease and more than half of all children diagnosed with AML eventually relapse and die of their disease. Dr. Meshinchi recently performed a comprehensive genetic analysis of pediatric AML and identified mutations in CSF3R gene, the first pediatric-specific mutation in AML. This JJ's Angels St. Baldrick's Research Grant aims to study the function of this mutation to learn how it might be used as a therapeutic target. This grant is named for the JJ's Angels Hero Fund created in memory of Julianna LaMonica to honor her joyful spirit and strength even in the midst of her battle with cancer. Her story continues to inspire so many to support the cause.

Hepatoblastoma is the most common childhood liver cancer. Dr. Prochwnik is using models to investigate two mutant genes, SIRT3 and SIRT4, which normally work to balance metabolism and energy stores, that are commonly found in hepatoblastoma. This work aims to identify novel targets for cancer therapies in hepatoblastoma and other cancers.

Juvenile myelomonocytic leukemia (JMML) is a fatal childhood myeloid malignancy with limited therapeutic options. Relapse remains the main cause of treatment failure, most likely due to the persistence of leukemic stem cells, a small population of self-renewing precursor cells that are responsible for long-term maintenance of leukemia growth and drug resistance. This research tests for the therapeutic effects of Stat5 inhibition by pimozide, a clinically used antipsychotic drug, in a subtype of JMML caused by mutations in Ptpn11.

Acute Myeloid Leukemia is an aggressive form of childhood cancer for which the therapy causes many side effects and the survival is 60%. Dr. Sakamoto's lab has found that a protein known as CREB is overproduced in AML cells and is associated with a worse prognosis. This research aims to study the protein RSK, which increases CREB activity in AML cells, and find ways to block RSK as a new approach to treat AML.

Ewing sarcoma is the second most common bone tumor in children and adolescents, and overall survival is dismal for patients with recurrent disease. Dr. Shelat recently identified an "Achilles’ heel" in this cancer, and showed that exploiting this weakness using a three drug cocktail cured the disease more than 80% of the time in models. This project is to validate this weakness as a marker for sensitivity to the drug cocktail in Ewing sarcoma and other pediatric cancers, and to find new drug combinations that better target this weakness.

Dr. Sondel has been using an immune-based therapy to treat children with cancer, and is seeing that it is clearly helping some patients. He recently found the presence of an antibody seems to predict which patients will do best with this treatment. This research aims to understand what this antibody is recognizing, and then to determine how it is helping the treatment to work better.

The most common childhood brain tumors are called gliomas. Despite advances in research, the prognosis for the aggressive pediatric gliomas remains poor. Dr. Song is studying how genes important in normal brain development are hijacked to cause brain cells to grow into a tumor and why these tumors are so resistant to treatment. This research aims to increase understanding of the biology of pediatric malignant gliomas and help develop new treatments that will ultimately improve outcome of these fatal tumors. This grant is made with generous support from the McKenna Claire Foundation, a St. Baldrick's partner established by the Wetzel family in memory of their daughter, McKenna. Their mission is to cure pediatric brain cancer by raising awareness, increasing community involvement and funding research.

Ewing sarcoma is a type of cancer that mostly affects children and your adults. This cancer is caused by a type of mutation in DNA called a translocation. Dr. Sweet-Cordero's Team Clarkie St. Baldrick’s Research Grant aims to understand how this translocation, called EWS-FLI1, causes cancer in order to find better therapies for children with this disease. This grant is named for the Team Clarkie Fund created to honor Clarkie Carroll and funds Ewing’s sarcoma research while stimulating greater awareness and inspiring others to believe pediatric cancer research can and will lead to a cure.

Medulloblastoma is the most aggressive brain tumor in children. Finding new therapies depends upon a better understanding of the biological mechanisms of medulloblastoma formation. As the recipient of the Hannah's Heroes St. Baldrick's Research Grant, Dr. Van Meir is evaluating the role of a tumor suppressor in medulloblastoma tumorigenesis. Understanding the role of this suppressor could lead to novel therapeutic prospects for children with 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.

Rhabdomyosarcomas are the most common soft-tissue cancers in children and adolescents. Current treatments are often ineffective, and researchers need new ideas to treat this cancer. Dr. Hettmer has developed a new model of pleomorphic rhabdomyosarcoma, and is using this to show that a protein (Gremlin 1) produced by rhabdomyosarcoma tumors could be important. She is investigating how Gremlin 1 changes rhabdomyosarcoma behavior and whether it can serve as a drug target. Awarded at Children's Hospital Boston and transferred to Charité Universitätsmedizin Berlin.

Most children dying of cancer have tumors that have developed “resistance” to conventional treatments like chemotherapy and radiation therapy. Scientists know very little about what is occurring in cancer cells to cause this, and therefore we have few options on how to cure such patients. Dr. Hogarty's team recently discovered that resistant cancer cells have altered the way their mitochondria, a key part of all cells, respond to the stress of cancer treatments; this leads to their inability to die. This research aims to explore this discovery further and find out opportunities to exploit it.

The most common childhood cancer, acute lymphoblastic leukemia is a cancer of the bone marrow. Exciting new research shows that healthy normal bone marrow cells can protect leukemia cells from cancer drugs. Dr. Mullen's research aims to find ways to prevent normal marrow cells from protecting leukemia cells and thus make cancer drugs more effective.

Children with a rare brain cancer called glioblastoma rarely survive. The treatment of this cancer has not advanced for years because of difficulty accessing and growing the tumor cells. Recently, the tumor cells were found to harbor changes in a gene that is strongly conserved through evolution. Dr. Partridge has modeled the same genetic mutations in simple yeast cells to ask how the mutations impact the growth of cells and to find ways to effectively kill cells bearing the mutations. This research will then ask if similar therapies can be used to treat children with glioblastoma. This grant is made with generous support from the "Henry Cermak Fund for Pediatric Cancer Research" created in memory of Henry Cermak and dedicated to his wish that "no one gets left out".

AML is the most common type of blood cancer. Normal blood stem cells have the capacity to produce all types of blood cells. However, impaired blood stem cells, as a consequence of genetic changes, play a central role in the initiation and progression of AML. Dr. Qian is researching how an alteration of expression of a critical gene, which is required for normal function of blood stem cells, causes AML. This study also aims to identify new therapeutic approaches to cure childhood leukemia by targeting the impaired blood stem cells.

Children who are successfully treated for cancer sometimes develop a second cancer due to treatment with radiation and chemotherapy drugs. Leukemia is one of the most common types of secondary cancer, and treatment-induced leukemia is extremely aggressive and very hard to cure. Chromosome 7 is often deleted in this type of leukemia. Dr. Shannon recently created models with deletions similar to those found in children with treatment-induced leukemia. With the Kenneth and Mary Ellen Wilson St. Baldick's Research Grant, Dr. Shannon is using these models to understand how these leukemias develop, why they are so hard to treat, and to test new therapies. This grant is named in memory of Kenneth and Mary Ellen Wilson, parents of Todd and Jason Alonzo. Their legacy of giving and generosity lives on in the service and dedication of their children.

Rhabdomyosarcoma is the most common soft tissue sarcoma in children. Dr. Shern's group recently discovered that mutations in 10 genes drive this disease. This research grant is determining whether these mutations can be used as markers of prognosis or response to therapy. Dr. Shern hopes to develop a tool that can be used to better detect and treat rhabdomyosarcoma. If successful, the developed tool can immediately be integrated into clinical trials to improve the current therapy. This grant is made with generous support from the PFP Fund for Cancer Research, a St. Baldrick's Hero Fund created in memory of Peyton Arens and honors his fighting spirit by supporting research that will bring about cures and less toxic treatments.

Better therapies that specifically target cancer cells while leaving normal cells undamaged will lead to therapies with fewer short or long term side effects. Cancer, specifically Ewing sarcoma, changes how proteins are made. New proteins occur by rearranging the messages that come from DNA. The rearranged messages turn into proteins that keep the cancer growing. Dr. Toretsky's research aims to find out what the rearranged messages are and how the new proteins could be targeted with new medicines. This grant is made with generous support from the Team Clarkie Fund, a St. Baldrick's Hero Fund created to honor Clarkie Carroll. It will fund Ewing sarcoma research while stimulating greater awareness and inspiring others to believe pediatric cancer research can and will lead to a cure.

Improving the outcome of aggressive childhood leukemias depends on developing new targeted treatments. With the NetApp St. Baldrick's Research Grant, Dr. Wechsler is studying a mutant gene called CALM-AF10 that causes high-risk leukemias in children. They determined that the leukemia-causing properties of this gene depend on its interaction with a partner protein called CRM1. This research is studying the mechanisms by which CRM1 enables CALM-AF10 to cause leukemias, and the ability of drugs to inhibit CRM1 to stop leukemia growth. This grant is named for the NetApp team, whose offices around the world have raised more than $5.8 million since 2007 for life-saving research through the St. Baldrick's Foundation.

While many children with leukemia experience good outcomes on modern therapies, there are a large number of children who still relapse and die of their disease. Using new methods, Dr. Willman identified a new form of leukemia, called Ph-like ALL, which has a variety of gene mutations which code for enzymes. Currently, drugs that inhibit these enzymes are available for clinical trials. Dr. Willman is testing these drugs in children with high risk leukemia. This research aims to extend Dr. Willman's current studies to include children with standard risk leukemia in order to also improve their chances of survival.

Rhabdomyosarcoma (RMS) is the most common soft tissue sarcoma of childhood and some subtypes are highly aggressive and spread to different organs. Current treatment strategies include surgery, radiation and chemotherapy, and clinical trials combining these modalities still result in only 30% survival. Advances in cancer genome studies have identified several genetic changes that are crucial for aggressive tumor growth and spread of the disease. Some of these genetic changes result in display of specific proteins on the tumor cell surface. Development and preclinical evaluation of monoclonal antibodies against such tumor-specific molecules would open the door for a variety of targeted therapeutics and novel treatment options for these patients.