Grants Search Results

This institution is a member of a research consortium which is being funded by St. Baldrick's: Reducing Ethnic Disparities in Acute Leukemia (REDIAL) Consortium. For a description of this project, see the consortium grant made to the lead institution: Baylor College of Medicine, Houston, TX.

This institution is a member of a research consortium which is being funded by St. Baldrick's: Reducing Ethnic Disparities in Acute Leukemia (REDIAL) Consortium. For a description of this project, see the consortium grant made to the lead institution: Baylor College of Medicine, Houston, TX.

The goal of precision medicine is to identify specific genetic changes in individual cancer cells to inform cancer therapy. The Precision-based Therapy for Childhood Leukemia Consortium conducted the first pediatric leukemia precision medicine, multi-institutional clinical trial in the United States. The goal of the consortium is to identify genetic changes in the patient’s leukemia that could be targeted with novel therapies. This group is now expanding efforts to identify genetic changes in patients enrolled on the trial. Additionally, they are using a combination of a chemistry and biology approach to model new genetic changes. The combination of these studies is bringing precision medicine to pediatric patients with relapsed leukemia, with the ultimate goal of cure. Fund administered by Dana-Farber Cancer Institute. This grant is funded by and named for Julia's Legacy of Hope, a Hero Fund that honors her positive, courageous spirit and carries out her last wish: "no child should have to go through what I have experienced". Diagnosed at 16 with Ewing sarcoma, Julia fought cancer and survived only to be stricken by a secondary cancer as a result of treatment. Her family hopes to raise awareness and funds for research especially for Adolescent and Young Adult (AYA) patients.

This institution is a member of a research consortium which is being funded by St. Baldrick's: Precision–based Therapy for Childhood Leukemia. For a description of this project, see the consortium grant made to the lead institution: Dana–Farber Cancer Institute, Boston, MA.

This institution is a member of a research consortium which is being funded by St. Baldrick's: Precision–based Therapy for Childhood Leukemia. For a description of this project, see the consortium grant made to the lead institution: Dana–Farber Cancer Institute, Boston, MA.

This institution is a member of a research consortium which is being funded by St. Baldrick's: Precision–based Therapy for Childhood Leukemia. For a description of this project, see the consortium grant made to the lead institution: Dana–Farber Cancer Institute, Boston, MA.

Beckwith-Wiedemann Syndrome (BWS) is a cancer predisposition syndrome and patients with BWS have a significantly increased risk of developing hepatoblastomas. The same genetic changes on chromosome 11 that cause BWS are found in 40% of hepatoblastomas. Dr. Kalish has previously shown that noncancerous liver and HB tissue from patients with BWS have distinct molecular signatures, suggesting the events that set up patients with BWS for HB are due to these changes on chromosome 11. Using the largest BWS collection of tissues worldwide, Dr. Kalish and colleagues will study the specific features of BWS and nonBWS liver cells and HB cells to determine how the changes of chromosome 11 lead to HB. Cell models derived from liver tissue will be used to test how these changes cause the transition from normal liver to HB. This work is a critical step in developing targeted therapies for patients with HB.

This institution is a member of a research consortium which is being funded by St. Baldrick's: Treehouse Childhood Cancer Project. For a description of this project, see the consortium grant made to the lead institution: University of California, Santa Cruz, Santa Cruz, CA.

This institution is a member of a research consortium which is being funded by St. Baldrick's: Treehouse Childhood Cancer Project. For a description of this project, see the consortium grant made to the lead institution: University of California, Santa Cruz, Santa Cruz, CA.

This institution is a member of a research consortium which is being funded by St. Baldrick's: Treehouse Childhood Cancer Project. For a description of this project, see the consortium grant made to the lead institution: University of California, Santa Cruz, Santa Cruz, CA.

Atypical teratoid/rhabdoid tumors (AT/RT) are the most common malignant brain tumors of infancy. Standard therapies lead to severe toxicities and poor overall survival. Dr. Raabe aims to identify novel therapies to reduce toxicities and improve survival. Dr. Raabe and colleagues found that cancer cells rely on activation of the integrative stress response (ISR) to maintain cell equilibrium and survival. However, if the ISR is activated too intensely or for too long, cells undergo apoptosis and die. Paxalisib and gemcitabine are medications that induce considerable cell stress, further activating the integrative stress response, and extending survival in models of AT/RT. Dr. Raabe is investigating how these medications act together to target cell stress pathways and their impact on survival in models of AT/RT. The findings will translate directly through the International Pacific Pediatric Neuro-Oncology Consortium (PNOC) into a new clinical trial treating children with relapsed or refractory AT/RT. This grant is named for Hannah’s Heroes, a St. Baldrick’s Hero Fund created in honor of Hannah Meeson and pays tribute to her fight by raising awareness and funding for all childhood cancers because kids like Hannah “are worth fighting for.”

Each year, approximately 1000 Americans aged 20 years or younger are diagnosed with acute myeloid leukemia (AML). Currently, even the most effective targeted drug BCL2 inhibitor-venetoclax (VEN) cannot eradicate all leukemia cells. The remaining cells cause disease recurrence and result in a very low overall survival rate for AML patients. In preliminary studies, Dr. Li found that targeting an enzyme called ADSS2 promotes pediatric AML cells sensitivity to VEN induced mitochondrial apoptosis, resulting in a synthetic lethality. This study will ask how ADSS2 preserves AML cells fitness and test the effectiveness of a first-in-class ADSS2 inhibitor combined with VEN or other BCL2 family protein MCL1 inhibitor in models of AML. If successful, this could lead to a clinical trial with potential impact for childhood AML patients.

Pediatric Burkitt Lymphoma (BL) arises from errors during immune (B cell) development. Treatment failure is associated with dismal outcomes, and many pediatric BL survivors will suffer long-term toxicities from therapy, highlighting the need to explore opportunities to identify patients who may be cured with less intense therapies. Little is currently known about the biology of pediatric BL and clinical implications of specific mutations. Therefore, systematic analysis of tissue from children treated on clinical trials represents a unique opportunity to gain insights from valuable specimens to inform biologic risk-based chemotherapy and identify potential targeted therapeutic strategies. Dr. Allen will characterize intrinsic and acquired genetic factors that underlie pathogenesis and predict response to therapy in patients with pediatric BL who have completed treatment on COG clinical trials. This grant is funded by and named for Jack's Pack - We Still Have His Back, a St. Baldrick's Hero Fund. Jack Klein was a ten year old who loved life, laughing and monkeys. During his illness, his community of family and friends near and far rallied around him under the moniker "Jack's Pack". Their slogan was "We have Jack's Back". After Jack succumbed to Burkitt's Lymphoma, his "pack" focused their energy and efforts to funding a cure...just as Jack would have wanted.

Children receiving bone marrow transplant can have serious complication such as bloodstream infections and graft versus host disease and some children die of these complications. Alteration of the bacteria in the gut by treatments including antibiotics is an important cause of these complications. In a previous study Dr. Davies and colleagues have tested the use of human milk to help keep gut bacteria healthy in very young children and found that this treatment worked. They are now studying a purified sugar from human milk, 2-FL that can be given easily as a medicine. Dr. Davies will also test a novel rapid urine test and a blood test to assess health of the gut bacteria during the study. Current tests require a stool sample and can take a long time. This trial will generate the data needed to perform a large-scale multi-center randomized clinical trial that will best prove how well this treatment works. This grant is generously supported by the Rays of Hope Hero Fund which honors the memory of Rayanna Marrero. She was a happy 3-year-old when she was diagnosed with Acute Lymphoblastic Leukemia (ALL). She successfully battled ALL, but a treatment induced secondary cancer claimed her life at age eight. Rayanna had an amazing attitude and loved life. She, like so many kids facing childhood cancer, did not allow it to define who she was. This Hero Fund aspires to give hope to kids fighting cancer through research.

Leukemia, a cancer of the blood and bone marrow, is the most common cancer in kids. Over the last 60 years, great strides have been made in treating children with leukemia, and today, most leukemias are curable. However, certain leukemias are difficult to treat and have a poor prognosis. In order to better treat cancers, researchers seek to better understand the pathways by which cancer cells develop in order to identify medicines that target proteins in these pathways. Dr. Aumann and colleagues study the fusion protein CALM-AF10 which is found in some leukemias, and found that these leukemias have increased expression of a protein called SIX1. Dr. Aumann is studying how the SIX1 protein makes blood cells turn into leukemia cells, and is using two small molecule inhibitors in combination with other chemotherapy as potential new treatments for this and other leukemias. The hope is that the these studies will clarify the role of SIX1 in CALM-AF10 and other leukemias.

There is an urgent need for novel targeted therapy for osteosarcoma (OS). Molecular targeted therapy has yielded stunning response rates of >90% for specific targets such as NTRK gene fusions. In contrast, the mainstay of therapy for osteosarcoma has remained the same for more than 30 years and few targeted agents have successfully translated to the clinic for OS patients. The challenge to develop targeted therapies for osteosarcoma is that the tumor has different driver mutations in different patients. However, 12-39% of tumors share a common amplification in a gene called MYC. In this project, Dr. Grohar and colleagues will consider this subset of osteosarcoma as a distinct entity. They are characterizing the role of MYC in the pathogenesis of osteosarcoma and determining how MYC makes osteosarcoma aggressive. Ultimately, they will identify compounds that will serve as clinical candidates for MYC-driven osteosarcoma. They will then determine if they are best translated to the clinic as single agents, in combination with chemotherapy, or as metastasis-targeted therapy. Dr. Grohar has assembled a team with the necessary expertise. Chand Khanna is a disease expert in osteosarcoma, the Grohar lab has expertise in drug development for bone sarcomas, the Neamati and O'Keefe labs are experts in drug discovery/chemical biology/natural products. Filemon Dela Cruz is an expert in preclinical drug modeling, Ted Laetsch and Rashmi Chugh are experts in sarcoma clinical trial design, and Ethos (led by Chand Khanna) and Vuja De (led by David Warshawsky) are companies that will aid in the development of these compounds for the clinic. Together they will identify compounds that are specific and effective for MYC amplified osteosarcoma. To make a significant impact for kids fighting osteosarcoma, five funding partners have banded together with St. Baldrick’s to support this grant – The Fight Osteosarcoma Together (FOT) Super Grant supported by Battle Osteosarcoma, CureSearch for Children’s Cancer, Michael and April Egge, The Osteosarcoma Collaborative, and the Zach Sobiech Osteosarcoma Fund of Children’s Cancer Research Fund.

Diffuse intrinsic pontine glioma (DIPG) is a deadly pediatric brain cancer, and there is a dire need to develop new therapeutic strategies to improve the terrible outcomes for these patients. Looking at genes that are turned on or off in a cancer can be helpful to figure out what is causing cancer growth. While looking at genes that are turned on in DIPG, Dr. Tsai found a gene called FOXR2 that is turned on at very high levels in a subset of DIPGs. FOXR2 is usually turned off, and turning on FOXR2 makes tumors grow very quickly. FOXR2 is actually capable of turning on an entire set of genes that are called ETS transcription factors (TFs). This is surprising as these genes have never been shown to be activated in DIPGs. Others have shown that ETS TFs can turn on the MAPK signaling pathway. Dr. Tsai also has found that FOXR2 is able to activate MAPK signaling. The goal is to determine exactly how FOXR2 and ETS TFs cooperate together to turn on MAPK signaling to make DIPGs grow. This grant was awarded at Dana-Farber Cancer Institute and transferred to Children's Hospital of Los Angeles. A portion of this grant is generously supported by Griffin's Guardians, a St. Baldrick's partner. Griffin's Guardians was created by the Engles in memory of their son, Griffin. Their mission is to provide support and financial assistance to children battling cancer in Central New York, raise awareness about pediatric cancer and provide funding for research.

Acute Lymphoblastic Leukemia (ALL) are aggressive cancers of B- and T- immune cells. ALL is most common in children but also affects adolescents and young adults. 90% of childhood ALL is curable. However, ~10% of children and ~30% of adolescents and young adults with ALL are not cured. To combat hard-to-treat ALL, Dr. Swaminathan will harness the body’s natural anti-cancer defense mechanism: a type of immune cell called a natural killer (NK) cell. He will also find defective NK cells in children with ALL. Those with fewer defective NK cells tend to survive longer and spend more of their lives free from disease compared to patients with high levels of abnormal NK cells. These findings will inform the development of NK cells as affordable therapies to cure pediatric ALL.

This institution is a member of a research consortium which is being funded by St. Baldrick's: Precision–based Therapy for Childhood Leukemia. For a description of this project, see the consortium grant made to the lead institution: Dana–Farber Cancer Institute, Boston, MA.

Genes instruct cells to do their jobs through making specific proteins. In our body, all cells store this what-to-do manual in a set of higher-order structures called chromosomes. When chromosomes break off, the broken pieces sometimes exchange their places to build new chromosomes. These errors, known as translocations, could have no effect on our bodies, but in many cases they might cause problems as severe as cancer. Dr. Su's research focuses on learning how chromosomal translocations promote tumor formation in children and young adults, as well as looking for clinically useful approaches to correct their pathogenic activities and cure these deadly diseases.