Grants Search Results

This institution is a member of a research consortium which is being funded by St. Baldrick's: New Approaches to Neuroblastoma Therapy (NANT) Consortium. For a description of this project, see the consortium grant made to the lead institution: Children's Hospital Los Angeles, Los Angeles, CA.

The EWSR1-FLI1 family of cancer genes causes Ewing sarcoma. However, no drugs currently exist that specifically block the action of EWSR1-FLI1 to cause cancer cells to grow. The McFadden Lab has engineered a "self-destruct button" into the EWSR1-FLI1 gene in Ewing sarcoma cells cultured in the laboratory, and these cells stop growing when the EWSR1-FLI1 gene is turned off. Dr. McFadden is using this laboratory tool to identify proteins that work with EWSR1-FLI1, and identify other genes it controls to cause Ewing sarcoma cells to grow. These studies will help identify new ways to stop the growth of Ewing sarcoma cells.

Brain tumors are the most common solid tumor in children, and diagnostic imaging guides almost every step in the care of children with brain tumors. However, currently available imaging methods have limited accuracy. Dr. McConathy is using an amino acid tagged with radioactivity (FET) to detect abnormal metabolism in tumor tissue using positron emission tomography (PET) in combination with magnetic resonance imaging (MRI). He expects this new imaging technique to improve the ability to see brain tumors before and after surgery to help doctors better plan the treatment of children with brain tumors. In the long term, Dr. McConathy expects FET-PET/MRI to help select and plan the best therapies and increase the chance of achieving cures.

This institution is a member of a research consortium which is being funded by St. Baldrick's: New Approaches to Neuroblastoma Therapy (NANT) Consortium. For a description of this project, see the consortium grant made to the lead institution: Children's Hospital Los Angeles, Los Angeles, CA.

Rhabdomyosarcoma is a pediatric cancer of the developing skeletal muscle. The mechanisms that drive this tumor are poorly understood. From Dr. McEvoy's preliminary analysis, one possible mechanism is epigenetic deregulation of a group of long noncoding RNAs (lncRNA). This is exciting because lncRNAs play a role in tumorigenesis in other cancer types, including a subset of pediatric tumors. This presents a unique opportunity to develop novel therapeutic approaches for children with rhabdomyosarcoma. Dr. McEvoy's team hypothesizes that lncRNA deregulation is essential for rhabdomyosarcoma development. This study is working to understand the underlying mechanisms that drive this disease and identify potential new therapies. These results will have tremendous impact on patients, especially those with metastatic disease since only 20-40% will survive using current treatments.

Rhabdomyosarcoma is a deadly cancer when spread through the body. With the Aiden's Army Fund St. Baldrick's Research Grant, Dr. Li is combining drugs already FDA approved for adult cancers in a way that stops rhabdomyosarcoma tumor cells from creating new tumors elsewhere in the body. This approach is unique because Dr. Li not only aims to stop the tumor cells from growing, but will try to convert what is left to non-cancerous cells similar to what is found in normal muscle.

This grant is funded by and named for the Aiden's Army Fund, a St. Baldrick's Hero Fund. Aiden Binkley who was diagnosed with Stage IV rhabdomyosarcoma at age 8. This bright, funny and courageous little boy believed he got cancer so he could grow up to find a cure for it. His vision is being carried on by Aiden’s Army through the funding of research. They will march until there is a cure!

This institution is a member of a research consortium which is being funded by St. Baldrick's: New Approaches to Neuroblastoma Therapy (NANT) Consortium. For a description of this project, see the consortium grant made to the lead institution: Children's Hospital Los Angeles, Los Angeles, CA.

Based on progress to date, Dr. Green was awarded a new grant in 2020 to fund an additional year of this Scholar grant. High-grade gliomas (HGG) are aggressive brain cancers that affect both adults and children. Current treatment options are very limited, and the vast majority of patients die of their tumors within five years of diagnosis. One subtype of high-grade glioma that almost exclusively occurs in children, diffuse intrinsic pontine glioma (DIPG), is the last incurable childhood cancer, with zero percent long-term survivors. As the Luke's Army Pediatric Cancer Research Fund St. Baldrick's Scholar, Dr. Green and his team intend to address these tumors by focusing on a new field of cancer treatment called epigenetics, which literally means "above genetics" and refers to all changes to DNA that do not involve changes to the DNA sequence itself, but instead affect which genes are made into protein. Through prior work, Dr. Green's team has found a gene, BPTF, which controls the expression of many other genes and appears to drive HGG and DIPG growth. Dr. Green aims to determine how exactly BPTF drives growth by interacting with other genes, to measure how BPTF inhibition works with drugs called HDAC inhibitors and whether this strategy could work with current standard treatments, and to measure the effect of a new chemical that inhibits BPTF that could serve as a precursor to medicines targeting BPTF.

This grant is funded by and named for Luke's Army Pediatric Cancer Research Fund. This Hero Fund was created in memory of Luke Ungerer who brought smiles and sunshine wherever he went with plenty to share with everyone. He battled a brain tumor with a positive spirit and inspired others with his courage in his short life. This fund intends to carry on Luke’s legacy of positivity with the hope that it will ripple across many lives for many years to come.

This institution is a member of a research consortium which is being funded by St. Baldrick's: New Approaches to Neuroblastoma Therapy (NANT) Consortium. For a description of this project, see the consortium grant made to the lead institution: Children's Hospital Los Angeles, Los Angeles, CA.

Based on progress to date, Dr. Gowda was awarded a new grant in 2020 to fund an additional year of this Scholar grant. Children with high risk B-cell leukemia, especially with loss or dysfunction of IKZF1 gene have very poor outcomes and high relapse rate. Every other child who relapses with high risk leukemia dies from the disease and there has not been much advancement in treatment for this group for the last 30 years. Dr. Gowda and team have found that a cancer promoting protein called casein kinase II (CK2) impairs the important functions of a protein that helps prevent leukemia. Inhibiting the CK2 protein will restore the ability of this protein to function properly and prevent leukemia. Dr. Gowda's team is testing if using a drug that inhibits CK2 protein along with the drugs that already are known to work in leukemia will have stronger anti-leukemia effect and improve the outcome. Using two agents that target same gene or pathway via different mechanisms will ensure effective shutdown of the particular pathway resulting in strong therapeutic effect. This strategy would also help lower the doses of each drug used and reduce their side effects and associated toxicity.

Our immune system has a remarkable ability to eradicate a vast array of foreign invaders. With increasing knowledge of this remarkable organ system, we can now begin to manipulate our immune system to fight cancer with remarkable precision and efficacy. However; tumor cells adopt multiple ways to reduce the immunotherapy efficacy, especially for solid tumors. Dr. Huang's research program systematically studies ways by which cancer cells evade immune cell detection and his goal is to develop a comprehensive, multi-pronged approach to increase the overall effectiveness of immunotherapy, particularly in pediatric and adolescent and young adult brain tumors and solid tumors.

Ewing Sarcoma is an aggressive disease affecting children and young adults. Patients are treated with intensive chemotherapy. This helps some, but not all, with early disease, works poorly in those with advanced disease, and can have serious side effects. Searching for new and better therapies, Dr. Jedlicka's lab has found a new protein that works abnormally in Ewing Sarcoma and that could be a new target for treatment. Dr. Jedlicka is working to understand more about how this protein works and how best to block it, to see if it could be a useful new treatment.

This institution is a member of a research consortium which is being funded by St. Baldrick's: New Approaches to Neuroblastoma Therapy (NANT) Consortium. For a description of this project, see the consortium grant made to the lead institution: Children's Hospital Los Angeles, Los Angeles, CA.

Based on progress to date, Dr. Cimmino was awarded a new grant in 2020 to fund an additional year of this Scholar grant. Vitamin C is essential for maintaining healthy hair, skin, immune system and heart function. In addition to these health benefits, Dr. Cimmino and team propose that vitamin C might be a non-toxic therapeutic for the treatment of patients with pediatric acute myeloid leukemia. Recently, it was discovered that vitamin C enhances the activation of a group of enzymes called TET proteins that are required for normal blood development. A significant fraction of children and young adolescents with acute myeloid leukemia have mutations in TET2, causing impaired TET2 activity and a block in normal blood cell formation. However, only one of the two copies of the TET2 gene is defective in these patients. Dr. Cimmino's team is working to determine if treatment with high-dose vitamin C could enhance the activity of the remaining, non-mutant, TET2 protein, kill leukemia cells and restore normal blood development. Alternative therapies such as treatment with vitamin C might provide a safe and effective strategy to improve outcome for pediatric leukemia patients. Awarded at the New York University School of Medicine, and transferred to University of Miami.

Acute myeloid leukemia (AML) is a cancer of the immature cells in the bone marrow. One common chromosomal abnormality found in pediatric AML is the inversion of chromosome 16 (inv(16)). Current treatments for inv(16) AML are associated with significant toxicity, as well as serious long-term chronic effects. Therefore, there is a pressing need to develop new, more targeted treatments for children with inv(16) AML. Inv(16) generates a fusion gene called CBFB-MYH11. CBFB-MYH11 causes changes in gene expression, which are the first step in the development of leukemia. Because Cbfb-MYH11 is expressed in all inv(16) leukemia cells, it makes an attractive drug target. Currently, there are no CBFB-MYH11 inhibitors suitable for use in humans. However, it is possible that other proteins cooperate with CBFB-MYH11, some of which may be better drug targets. One potential co-factor is HDAC1. Dr. Hyde's team found that HDAC1 binds CBFB-MYH11 and is required for its activity. They also found that an HDAC1 inhibitor significantly blocks the growth leukemia cells in culture. In this project, Dr. Hyde is testing whether HDAC1 is an important co-factor of CBFB-MYH11 and if HDAC inhibitors effectively target Cbfb-MYH11+ leukemia cells in vivo. These results will have direct clinical implications for children with inv(16) AML.

This institution is a member of a research consortium which is being funded by St. Baldrick's: New Approaches to Neuroblastoma Therapy (NANT) Consortium. For a description of this project, see the consortium grant made to the lead institution: Children's Hospital Los Angeles, Los Angeles, CA.

Based on progress to date, Dr. Agarwal was awarded a new grant in 2020 to fund an additional year of this Scholar grant. High-risk neuroblastoma is an aggressive cancer of very young children with less than 50% overall survival. Current therapy includes high-dose chemotherapy and radiation, which has long-term toxic side-effects. Despite these intensive therapies, neuroblastoma commonly relapse. This relapse is the primary cause of death from neuroblastoma due to disease spread, drug-resistance, and toxicity. As the Oliver Wells Fund for Neuroblatoma St. Baldrick's Scholar, Dr. Agarwal is focusing his research on developing effective therapeutic approaches to target those tumor cells which escape initial treatment and regenerate drug-resistant disease. Recently, Dr. Agarwal's team discovered a chemotherapy-resistant, highly tumorigenic sub-population of cells in neuroblastoma tumors. These cells escape initial therapy and may cause aggressive, drug-resistant relapsed disease. Furthermore, they found that specific epigenetic enzymes maintain this cell sub-population by activating key genes. These epigenetic modifiers can be successfully targeted with novel epigenetic inhibitors, currently under pre-clinical trials. These exciting findings suggest a new epigenetic therapeutic approach for high-risk neuroblastoma. This grant supports efforts to uncover the mechanisms controlling neuroblastoma tumorigenicity and relapse, and develop an effective targeted approach for high-risk neuroblastoma.

A portion of this grant is funded by and named for the Oliver Wells Fund for Neuroblastoma, a St. Baldrick's Hero Fund. From the moment he was born, Ollie was the center of the Wells family with a contagious smile and a sparkle in his eyes. As the youngest child, it was devastating when they learned the 15 year old toddler had cancer. Oliver was diagnosed with high risk neuroblastoma and spent the next 13 months bravely enduring chemotherapy and radiation, more than a dozen surgeries and a bone marrow transplant. But Ollie persevered and smiled through it all. It was an unfair fight from the beginning and in July 2018, Ollie passed away. The Oliver Wells Fund for Neuroblastoma was established in his memory to raise funds to find cures and give hope to other kids facing the same fight. In this way, the Wells family intends to share Oliver’s joy for life and use his story to help find a cure. 

A portion of this grant was also funded by and named for David's Warriors, a St. Baldrick's Hero Fund. The fund was created in memory of David Heard who battled neuroblastoma until his passing at the age of ten. David inspired his family and countless others to commit to raising money for research to fight pediatric cancer through the St. Baldrick’s Foundation. The Fund honors the amazing spirit with which he lived, embracing life until the very end.

Awarded at the Baylor College of Medicine, and transferred to St. John's University.

The goal of this study is to develop new therapies for Ewing sarcoma by targeting a protein called EWS-FLI1. Many people believe that the key to improving outcomes for Ewing sarcoma patients is to develop new drugs that block EWS-FLI1. In order for this to be successful, there is a need to understand exactly what happens to the Ewing sarcoma cell when EWS-FLI1 is turned off. Dr. Grohar is using the latest technology to both characterize the consequence of EWS-FLI1 silencing and identify novel compounds that turn EWS-FLI1 off.

This institution is a member of a research consortium which is being funded by St. Baldrick's: New Approaches to Neuroblastoma Therapy (NANT) Consortium. For a description of this project, see the consortium grant made to the lead institution: Children's Hospital Los Angeles, Los Angeles, CA.

Survivors of childhood acute lymphoblastic leukemia (ALL) may have difficulty learning because of problems with attention and working memory caused by the medications they receive during the course of their leukemia treatment. The Georgia and the Peachy Keens St. Baldrick's Supportive Care Research Grant is determining if children would be willing to complete 25 computer-based training sessions (designed as games) over an 8-week period, at home, while they are taking oral chemotherapy medications. These computer games are designed to improve attention and working memory. If this study proves that children are willing to complete the training and participate in short (10- to 15-minute) evaluations of their attention and memory, Dr. Winick will then test this intervention with a larger number of children to see how well it works and how long any effects last.

This grant is named for the Georgia and the Peachy Keens Hero Fund which was created in honor of Georgia Moore’s 5th year past her cancer diagnosis. As a leukemia survivor, she inspires others to “just keep swimming” by raising awareness, hope and research dollars.