Grants Search Results

Do you ever get a cold sore on your lip, or know someone who does? That sore is caused by a virus that destroys the cells in your lip. As the virus spreads, the sore gets bigger. Viruses are great at killing cells and spreading. But, the sore eventually goes away because the immune system attacks the infected cells, killing them and stopping the viral infection, allowing your lip to heal. Imagine if we could get both the virus and the immune system to kill cancer cells instead of lip cells! Previously Dr. Haworth's team used a safe version of the cold sore virus to infect a common type of hard-to-treat childhood cancer cells. The virus directly killed cancer cells and caused the immune system to attack the cancer cells that the virus missed. Dr. Haworth's team is testing ways to make the virus and immune system work better together. Dr. Haworth is infecting model tumors with the virus, and giving immune cells designed to attack the tumor, hypothesizing that giving both virus and immune cells will cure the tumor. Awarded at The Research Institute at Nationwide and transferred to St. Jude Children's Research Hospital.

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 immune system not only fights infection, but can also fight cancer cells. Recently, doctors have been able to use patients' own immune cells to help treat their cancer. These immune cells can also attack the patient's normal tissues, which is harmful. Dr. Richman is working to learn how normal tissues might be protected while still allowing the immune cells to effectively kill the cancer cells.

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.

Based on progress to date, Dr. Ladle was awarded a new grant in 2020 to fund an additional year of this Scholar grant. As the Aiden's Army Fund St. Baldrick's Scholar, Dr. Ladle is using the body's own immune system to destroy cancer - specifically a class of cancer in children originating from connective tissues called sarcomas. Using fire as an analogy, Dr. Ladle seeks to build an intense flame of a powerful immune response which will specifically kill the cancer cells. To create this fire, one must follow specific steps. The kindling, which must be easily burned, is protein targets on the cancer cells (termed tumor antigens) recognized by the immune system. Next, the spark to ignite the kindling is initial inflammation in the tumor against these tumor antigens. Finally, to feed the fire, fuel or lighter fluid can be added in the form of recently approved immune modulator drugs which, when infused into patients, bind to immune cells residing in the tumor and activates them to kill the tumor cells. Each ordered step is essential in building an effective fire. This project addresses each of these key aspects for generating a successful immune response to treat sarcomas and creating new tumor antigens, adding inflammation to jump start the immune response against these antigens, and combining with new immune modulators allowing the immune cells to be active in destroying sarcomas.

This grant is funded by and named for the Aiden's Army Fund, a St. Baldrick's Hero Fund. When he was 8 years old, Aiden Binkley was diagnosed with Stage IV rhabdomyosarcoma. He had a huge tumor in his pelvis and the cancer had metastasized to his lungs. But this bright, funny and courageous boy believed he got cancer so he could grow up to find a cure for it. Aiden’s story has inspired so many people and his vision to cure cancer 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: 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.

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 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.

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.

Cancer cells are hard to defeat because they are so similar to normal cells. Most current methods that kill cancer cells impose collateral damage on normal cells that lead to immune suppression, hair loss, and gastro-intestinal damage. Dr. Whitehurst's research focuses on identifying therapies that will only kill tumor cells but leave normal cells unharmed. Here, she is focused on a tumor type that impacts adolescents: Ewing Sarcoma. She has identified a pathway, called TNFa, which is “mis-wired” in these cancer cells. Instead of dying when this pathway is activated, the cancer cells keep growing. Importantly, she has identified inhibitors of the pathway that can kill these tumor cells. Dr. Whitehurst is working to understand how this pathway is mis-wired in cancer cells and the consequences of its inhibition. The end goal would be the identification of chemical inhibitors that could be used in the clinic as a less toxic and more effective treatment option.

Diffuse intrinsic pontine glioma, also referred to as brainstem glioma, is a pediatric cancer that accounts for the majority of deaths from brain tumors in children. Although radiation therapy is the standard of care for brainstem gliomas, the median survival of children with this tumor type is less than one year from diagnosis. In order to improve the treatment of these patients, Dr. Kirsch's team is using a model of brainstem glioma that can be used to evaluate the effectiveness of new therapies. Using this model, they are testing whether removing a protein called ATM, which is the target of drugs now entering clinical trials, will enhance radiation sensitivity in brainstem gliomas. They hypothesize that deleting this target, when given in combination with radiation therapy, will increase the number of tumor cells killed by radiation and will therefore improve survival in brainstem gliomas when they have a specific gene mutation commonly found in this childhood brain tumor. If successful, these studies will inform the design of future clinical trials testing this strategy in children with brainstem gliomas.

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.”

Many cancer treatments kill both normal and cancer cells. Drugs used in standard cancer treatments have long term effects in children, such as causing developmental delays or second cancers later in life. Dr. Blackburn's team is working to find new drugs that kill cancer cells, but do not affect normal cells. By discovering characteristics that are unique to cancer and finding a drug that recognizes that specific characteristic, they will be able to selectively kill cancer cells. Their research goal is to improve cancer treatments so that children can live long, normal lives after their cancer is cured.

Based on progress to date, Dr. Winters was awarded a new grant in 2019 to fund an additional year of this Fellow award. Dr. Winters' research involves developing more effective and more targeted therapies for children with acute myeloid leukemia (AML), a type of leukemia that continues to have poor outcomes. The therapy for pediatric AML has not changed much in 20-30 years, and many children who receive this therapy relapse. There is a protein on many AML cells called CD123, which marks the earliest leukemia cells. In adults there are drugs that target this protein which are being studied in clinical trials. However, no one has studied whether CD123 is a useful target in pediatric AML. Dr. Winters is looking at CD123 protein expression in AML samples from pediatric patients, as well as investigating whether expression of CD123 marks the primitive leukemia cells in these patients - that is, those that give rise to the leukemia and cause relapse. She is also testing some of the same drugs that are being used in adult clinical trials on these pediatric samples in a laboratory setting, to see if they may be useful in pediatric patients. These studies are expected to generate new therapy options for children with difficult-to-treat AML.

Half of neuroblastomas are high-risk neuroblastoma, with poor survival. Understanding abnormalities that drive high-risk neuroblastoma (drivers) enables development of therapies against specific drivers. Until 2015, we had identified drivers for half of high-risk neuroblastomas. Recently, most remaining high-risk neuroblastomas were shown to have high levels of TERT, a protein that helps chromosomes replicate. It is still not clear how a protein that helps chromosomes replicate could drive cancer. Perhaps TERT is needed for neuroblastoma tumors to grow, but is not driving the tumor. To distinguish these possibilities, Dr. Weiss is testing whether TERT can drive neuroblastoma in human stem-cell models. In Dr. Weiss' system, stem cells generated from normal human blood or skin cells, are differentiated to form a cell type called neural crest, from which neuroblastoma is derived. He is introducing known drivers into these cells to generate a model for neuroblastoma. Some known drivers (MYCN) lead to neuroblastoma, while others (ALK) do not. Dr. Weiss is using this model to test whether TERT is a driver, or is required for neuroblastoma in the context of other drivers (ALK). Successful completion will generate a model to evaluate whether therapy directed against TERT could help children with neuroblastoma.

This grant is generously supported by the Amanda Rozman Pediatric Cancer Research Fund created in memory of Amanda Rozman and honors her courageous battle with neuroblastoma by funding promising new to improve the efficacy and number of treatments available for relapsed and refractory neuroblastoma.

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!

Synovial sarcoma is a soft-tissue cancer in adolescents and young adults. More than half of patients develop metastasis, or spread of the cancer to the lungs. Once it has metastasized, synovial sarcoma is fatal in nearly all patients. Dr. Jones' team has developed a model of synovial sarcoma and found that when the tumor spread to the lungs many white blood cells begin to infiltrate the tumors. He is studying whether these particular white blood cells from the immune system are trying to fight the tumor or are helping the tumor grow and spread to the lungs. This team is testing if the presence of these immune cells in a large panel of human synovial sarcomas are associated with the same patients developing clinical spread of disease.

Based on progress to date, Dr. Mulama was awarded a new grant in 2020 to fund an additional year of this Scholar grant. Kaposi sarcoma-associated herpesvirus is a virus that causes cancer known as Kaposi sarcoma, which is very common in HIV+ children, especially in Africa and sometimes in individuals who get an organ transplant. Dr. Mulama is designing and testing a vaccine that prevents and treats the viral infection, as well as antibodies to detect infection in people. He will also test the vaccine so that one day it can be used as a treatment to prevent Kaposi sarcoma-associated herpesvirus infection and Kaposi sarcoma in more than 40,000 patients worldwide each year.

High grade gliomas (HGG) are a vicious subtype of pediatric brain tumors that remain the leading cause of death among children with cancer. New therapeutic strategies are urgently needed to combat this scourge. By mining genomic datasets from HGGs, Dr. Walensky's team has identified a unique susceptibility profile based on retention of wild-type p53 status and dual expression of the negative regulators HDM2 and HDMX. Whereas p53 can be mutated or deleted to avoid cell cycle arrest or apoptosis, a frequent alternative mode of p53 suppression relies on overexpression of HDM2 and HDMX. Small molecules have been developed to target HDM2 specifically, but co-expression of HDMX causes resistance. Only a stapled peptide modeled after the critical p53 transactivation helix is capable of blocking both HDM2 and HDMX, a feature that has prompted its advancement to Phase I/II clinical trials in adult cancers.

As the recipient of the St. Baldrick’s Research Grant with generous support from the Team Campbell Foundation, Dr. Walensky is testing a novel therapeutic strategy for pediatric HGG based on a dual-targeting stapled peptide inhibitor of HDM2/HDMX. He believes that the proof-of-concept data to emerge could provide a compelling rationale for conducting a clinical trial in these otherwise rapidly fatal pediatric brain cancers. The Team Campbell Foundation was created in memory of Campbell Hoyt who passed away from Anaplastic Ependymoma. Their mission is to improve the lives of families facing a childhood cancer diagnosis through raising awareness, funding research and providing psycho-social enrichment opportunities.

Recently the Meshinchi lab discovered that mesothelin, a cancer-specific antigen, is highly expressed in a subset of childhood AML cases, a result that both highlights the distinct genetic differences between adult and pediatric cancers and opens the door for the development of more targeted therapies. Dr. Kolb is developing novel combinations of bispecific T-cell engaging antibodies, called SMITEs (Simultaneous Multiple Interaction T-cell Engagers) that will co-target mesothelin and the AML marker CD33. These T-cell engaging protein pairs physically link cancer cells to cytotoxic T-cells resulting in more potent and selective killing than single agents alone.