Grants Search Results

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.

Children with cancer continue to succumb to their disease, many after receiving toxic therapies like chemotherapy and radiation. Also, surviving children face life long negative health consequences ranging from learning disabilities, to more severe effects such as a higher chance of getting another cancer in adulthood. Therefore, additional, rigorous scientific research needs to be performed to develop new and effective treatment options for these kids. Cancer growing inside the body hides in plain sight of the immune system. This is because cancer cells evolve to escape recognition by the immune cells. Therefore reawakening the immune system could be a very effective way of using a patients' own attacker cells to engulf cancer cells and get rid of the disease. Dr. Davare is working to discover and test new ways to reactivate immune cells for attacking cancer cells. For this project, she has developed an innovative method to identify synthetic molecules that will uncloak the cancer cell and make it visible to the immune system for destruction. This research strategy, in the long run, will open new doors and has the potential to not only increase survival of children with cancer, but their long term quality of life as well.

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.”

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.

This grant funds a doctoral student to complete work in pediatric oncology research for the summer. The experience may encourage them to choose childhood cancer research as a specialty.

Neurons are nerve cells that populate certain regions of the human body and are responsible for transmitting chemical signals back and forth to the brain to regulate critical bodily functions. A deadly form of pediatric cancer, known as neuroblastoma, occurs when a subset of these neurons start to proliferate uncontrollably. These cancer cells can migrate and spread throughout the body, making it very challenging to treat with currently available drugs. This highly aggressive form of neuroblastoma occurs in children who are older than 18 months. At such an early age, this disease can be quite devastating and there is an imperative need to better understand how this form of neuroblastoma develops. Recent work has identified pediatric cancer mutations in distinct specialized proteins that regulate chromatin (the complex of DNA and proteins), known as chromatin remodelers. One such protein, ATRX, was recently found to be mutated frequently in neuroblastoma tumors identified in adolescent and young adults, which have poor overall survival. Dr. Bernstein is exploring a novel therapy for neuroblastoma patients that harbor ATRX mutations thorough innovative and state-of-the-art approaches. Dr. Bernstein's team is comparing the cellular changes that occur in the presence of the drug in models of neuroblastoma.

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. 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.

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.

Cancer-related pain greatly compromises quality of life, and can increase disease morbidity, mortality, and healthcare costs by reducing children's compliance to medical procedures. The burden of cancer-related pain does not end when treatment concludes: many survivors of childhood cancer report cancer-related pain well into adulthood. Thus, there is a critical need for interventions that can reduce pain during and after children's treatments for cancer. Dr. Marusak is testing whether a martial arts therapy that centers around mindful breathing and meditative techniques can reduce pain and the underlying brain mechanisms in young cancer patients and survivors.

Based on progress to date, Dr. Wulff was awarded a new grant in 2019 to fund an additional year of this Fellow award. Ewing sarcoma (ES) is the second most common bone cancer in children. Approximately 25% of children with ES have metastasis, which are tumors that have spread to other parts of the body, such as the lungs. It is especially difficult to treat these children and more than 70% die within 5 years. Therefore, it is important to learn about what it is that allows these tumors to spread and hopefully develop new drugs to treat these patients. Certain proteins are expressed at much higher levels in metastatic lung tumors compared to the primary bone tumor, suggesting that these proteins play a role in allowing the tumor to spread. Dr. Wulff is studying the role of these proteins by increasing or decreasing them, and then testing how this affects the cancer's ability to grow and spread. Dr. Wulff's team thinks that the cancer's ability to spread can be decreased by decreasing a particular set of proteins. In addition, she is testing new drugs that inhibit the function of these proteins, with the hope to identify new therapies that will improve overall survival rates for patients with metastatic ES.

This is grant is generously supported by Team Clarkie, a St. Baldrick's Hero Fund. Clarkie Carroll was diagnosed with Ewing sarcoma in his upper right femur in 2013. He endured surgery and treatments with strength, positivity and a sense of humor. Today he has no evidence of disease.

A portion of this grant was also funded by this Hero Fund. It was created to honor Clarkie and ensure researchers have the resources to further Ewing’s sarcoma research as well as stimulate greater awareness and inspire others to believe pediatric cancer research can and will lead to a cure.

Only 45% of children with high-risk neuroblastoma (NB) are cured. The New Approaches to Neuroblastoma Therapy (NANT) consortium links laboratory and clinical investigators to develop therapies with high potential for improving survival and performs the first testing of these treatments at 14 neuroblastoma centers in North America and 3 in Australia, United Kingdom, and France. NANT studies aim 1) to enhance the ability of the immune system to eliminate NB by targeting both NB cells and surrounding normal cells that help tumor cells grow and resist treatments; 2) to improve treatment of NB using small molecule drugs that target NB gene abnormalities that “drive” tumor aggressiveness including abnormal/mutated ALK protein or abnormally increased MYCN protein; and 3) to use molecular biology to define new treatment targets in NB and normal cells that enhance NB growth and resistance to therapy to improve prediction of outcome with a “biomarker” test for NB cells in blood and bone marrow. The NANT consortium anticipates that these innovative studies will improve survival for children with high-risk neuroblastoma.

Children and adolescents with cancer and their families are at increased risk for psychosocial problems that can contribute to poorer health and quality of life, and it has been recommended that pediatric cancer centers develop programs to screen patients and families for psychosocial risk. The majority of pediatric cancers centers do not have practices in place to effectively and routinely screen all patients and families for psychosocial difficulties, with time and resources being acknowledged as barriers to implementation. Dr. Gilleland Marchak is developing a novel, patient-friendly technology to screen for psychosocial risk and evaluating its use at a large pediatric cancer center. Study outcomes will include data related to feasibility and acceptability of electronic screening, as well as efficacy in identifying families in distress and connecting them with family support team members to address problems in real time. By successfully leveraging technology to reduce barriers to universal psychosocial screening, we can improve communication between oncology providers and families regarding critical mental health, neurocognitive, and social issues that may negatively impact pediatric cancer treatment and health outcomes.

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.

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.

Dr. Deatrick is developing family support and education materials for maternal caregivers of young adult survivors of childhood brain tumors to improve their quality of life and quality of life of the survivors. Training in Problem Solving (TIPS) for Caregivers, leverages past research, eHealth, and Bright IDEAS family problem-solving intervention (an evidence-based intervention for caregivers of children newly diagnosed with cancer) to target challenges identified by maternal caregivers to their family management. TIPS is targeted to caregivers with “condition-focused FM” (family life organized around the special needs of the survivor). Using the prototype session developed in partnership with maternal caregivers, Dr. Deatrick will work with them to design the web-based intervention. She will develop other sessions of TIPS and adapt them to technology, which will be used “live” online with a health care provider and online with interactive homework sheets, videos, and other resources. Future research will involve fathers and other caregivers, survivors, and other family members.