Grants Search Results

This grant supports a dedicated Adolescent and Young Adult (AYA) Clinical Research Coordinator to ensure that more kids can be treated on clinical trials, often their best hope for a cure.

This grant supports a Clinical Research Associate to ensure that more kids can be treated on clinical trials, often their best hope for a cure.

This grant supports a Clinical Research Associate to ensure that more kids can be treated on clinical trials, often their best hope for a cure.

The originality of Dr. Ayrault’s team lies on the characterization of the putative relationship existing between mechanisms implicated into cerebellar development and medulloblastoma formation in order to uncover signaling pathway alterations and generate novel therapeutic strategies. As many tumor cells closely resemble to normal cells at a precise stage of the development, Dr. Ayrault foresees that his study will not only reinforce this new conceptual view of tumor cell biology but might also be generalized in oncogenesis. Medulloblastoma is the most common malignant pediatric brain tumor. Medulloblastoma are divided in four subgroups. Two of them, Group 3 (G3) and Group 4 (G4), are still poorly understood. While genomic and transcriptomic analysis revealed key features of medulloblastoma subgroups, they have not totally revealed functional mechanisms implicated in G3/G4. Yet, a recent study from this group reveals that proteomic analysis may unveil unsuspected biological modifications in G3 and G4 medulloblastomas. Ultimately, by developing innovative technologies as well as investigating the crosstalk between developmental neurobiology and pediatric brain cancer, Dr. Ayrault hopes to fill the existing gap of knowledge in pediatric brain tumors and lead to major breakthroughs. The St. Baldrick’s Robert J. Arceci Innovation Award is given in honor of the late Dr. Robert Arceci. A pioneer in the field, this award reflects Dr. Arceci's values including creativity, collaboration, and commitment to early- to mid-career scientists.

The AACR-St. Baldrick's Foundation Award for Outstanding Achievement in Pediatric Cancer Research has been established to bring attention to major research discoveries to the pediatric cancer research community and to honor an individual in any sector who has significantly contributed to any area of pediatric cancer research, resulting in the fundamental improvement of the understanding and/or treatment of pediatric cancer. The recipient will nominate an emerging leader conducting research in the academic sector to receive a research grant. The 2020 SBF-AACR Award for Outstanding Achievement in Pediatric Cancer Research went to Dr. James Downing at St. Jude Children’s Research Hospital. Dr. Jarno Drost at Princess Maxima Center for Pediatric Oncology received the 2020 research grant. Dr. Drost's research interests are in Kidney and Rhabdoid Tumors.

This institution is a member of a research consortium which is being funded by St. Baldrick's: Consortium for Childhood Cancer Predisposition. For a description of this project, see the consortium grant made to the lead institution: Emory University, Atlanta, GA.

Therapies that only inhibit tumor cells but not normal cells are missing for the deadly childhood brain tumor medulloblastoma. As the recipient of the Miracles for Michael Fund St. Baldrick's Research Grant, Dr. Zhang has identified a promising drug target in medulloblastoma. This project aims to study the role of the target in medulloblastoma and evaluate the therapeutic potential of inhibiting this target using cutting-edge technologies and models.

This grant is funded by and named for the Miracles for Michael Fund, a St. Baldrick's Hero Fund created in memory of Michael Orbany who was diagnosed with medulloblastoma when he was 6 years old. After completing initial treatment, his cancer relapsed within a year and he passed away at the age of nine. Michael had unwavering faith and perseverance, wanting most of all to make others happy. This fund honors his tremendous strength to never ever give up.

The proto-oncogene MYCN is amplified in approximately half of patients with high-risk neuroblastoma. At relapse, tumors from high-risk patients typically activate a pathway called "MAP kinase signaling" through genetic mutations including loss of NF1, which normally dampens MAP kinase function. Since relapsed neuroblastoma is generally therapy resistant, these data suggest that MAP-kinase activation contributes to therapy resistance. Does MAP kinase signaling contribute to therapy resistance in MYCN-amplified neuroblastoma at diagnosis? Dr. Weiss proposes that dependence on increased MAP kinase signaling in MYCN-amplified neuroblastoma enables rare cells within this heterogeneous tumor to evade chemotherapy. This therapy-resistant population then undergoes selection for further activation of MAP-kinase signaling, reinforcing therapy resistance. How does MYCN drive MAP kinase? The NF1 tumor suppressor blocks MAP kinase signaling. Mis-splicing of the NF1 messenger RNA in neuroblastoma cells results in NF1-23a, a protein with decreased ability to block RAS. Inclusion of NF1 exon 23a is regulated by the RNA splicing proteins "T-cell intracellular antigen 1" (TIA1) and "TIA1 Like gene" TIAL1, both of which are MYCN target genes. If activation of TIAL and TIAL1 (TIA/L1) in MYCN-amplified neuroblastoma activates MAP-kinase signaling in primary tumors at diagnosis, does traditional treatment of these tumors select for further flux through MAP-kinase signaling, to enhance resistance at relapse? This is the issue that Dr. Weiss' proposal addresses. Successful completion clarifies the importance of MYCN-TIA/L1 axis as a driver of resistance in neuroblastoma, and suggests a a translational path to improve outcomes in neuroblastoma.

Dr. Weiss' grant is generously supported by the Arden Quinn Bucher Memorial Fund, a St. Baldrick's Hero Fund. Arden’s intelligence, empathy, and dynamic personality charmed everyone and is now her legacy. Before her neuroblastoma diagnosis on October 11, 2007 at age two, she happily played with boundless energy and imagination. Even throughout her difficult months of treatment, Arden bravely managed to keep smiling and learning. This fund supports St. Baldrick’s mission: funding the most promising research, wherever it takes place to provide kids fighting cancers less toxic, more effective treatments allowing them to live longer, healthier lives.

We know that mental health and physical health are closely connected. For example, teenage cancer patients who receive bone marrow transplants have high rates of anxiety, depression and other mental health issues, which have in turn been associated with relapse and even death rates. Researchers have recently discovered that the immune system may be an important link between the mind and the body -- psychological stress can create a specific pattern of molecular responses in immune cells, which causes inflammation and may produce poor outcomes in cancer. To see if the molecular response pattern indeed associates with altered immune cell function and with mental health symptoms, Dr. Taylor will study blood samples and quality-of-life surveys that are collected from teenage patients undergoing bone marrow transplant. If we can understand the biology of how a teen's mental state affects the cancer in their body, we can develop better ways to improve both psychological and medical outcomes in these vulnerable patients.

Brain tumors are one of the most common and deadliest forms of pediatric cancers. Those who do survive may experience late effects including cognitive, emotional, and social difficulties. Additionally, Columbia University Medical Center in New York, New York has some of the highest rates of COVID-19 cases in the U.S. Thus, the children and their families facing diagnoses of pediatric brain tumors, and the health care providers treating them, were faced with incredibly difficult decisions, at a time when the entire medical system was in a state of upheaval, and questions were being raised as to who should be treated and saved. In medicine shared decision making involving parents, children (when possible) and health care providers is recommended in situations where there is not a clear superior treatment, which may be the case for many pediatric brain tumors. This study aims to understand the factors parents, healthcare providers, and the children themselves considered when making health care decisions. This includes how they balanced potential short-term and long-term challenges with quality of life and longevity of life, what information informed their decisions, and how the COVID-19 pandemic influenced the decisions they were making. This information will provide important insight into how decisions are made, particularly in times of heightened crisis. This will make us better able to help families, patients, and children.

Over the past 10 years, we have made great strides in the diagnosis of Medulloblastoma, the most common malignant brain tumor of childhood. These advances have come from widely collaborative efforts to perform DNA sequencing on tumor specimens. This effort led to the identification of major subtypes of Medulloblastoma and a recognition that these subtypes are associated with differences in response to standard treatments and survival. Lagging behind, has been an understanding of the molecular mechanisms that drive relapse of Medulloblastoma. This occurs in 30-40% of Medulloblastoma patients and as yet, there are no curative options. As the recipient of the Thumbs Up Fund to Honor Brett Haubrich St. Baldrick's Research Grant, Dr. Rubin and his team members are proposing a novel clinical trial to address this pressing unmet need. Their trial, brings together what has been learned from sequencing Medulloblastoma and the recently developed ability to test the sensitivity of an individual patient's Medulloblastoma cells to hundreds of drugs simultaneously. The long-term goal is to use the combination of drug testing and DNA sequencing to design personalized treatments for relapsed Medulloblastoma patients. Success in this effort would not only provide new treatments for relapsed Medulloblastoma, but would also provide a new paradigm for personalized approaches to the treatment of all pediatric brain tumors.

A portion of this grant is funded by and named in honor of The Thumbs Up Fund to Honor Brett Haubrich, a St. Baldrick's Hero Fund. Brett is remembered for his kindness, his joy in making others happy and his faith even through his 3 ½ year battle with anaplastic astrocytoma, a difficult to cure brain cancer. Brett was diagnosed at the age of 11 and endured treatments and laser surgery which impacted his motor and speech functions. Yet he was always positive, often giving his signature “thumbs up” as a symbol of hope. In his honor, Team Brett began participating in St. Baldrick’s head shaving events in 2015 and each year, raised over $10,000. This Hero Fund hopes to raise funds for childhood cancer research for brain tumors like Brett’s so other families would have more options for cures.

Dr. Rowe is applying stem cell biology to understanding childhood leukemia. Overall, pediatric oncologists have made remarkable progress in treating children with leukemia with chemotherapy, but some children have forms of leukemia that don't respond well. Dr. Rowe is interested in better understanding what makes this subset of leukemias resistant to treatment. To do this, he is developing new models of these unfavorable forms of leukemia so that he can understand precisely how normal blood cells become leukemic blood cells. If Dr. Rowe can achieve this, then researchers can find new ways to more effectively treat these forms of leukemia.

Doctors have been testing ways to boost the immune system to fight cancer in clinical trials over the last ten years. Although these approaches have led to very impressive results in patients with blood cancers, they have not worked well in patients who have tumors in their solid organs. Solid tumors have specialized cells that act as bodyguards, protecting the cancer cells from the immune system. Dr. Parihar has developed a strategy to selectively remove these 'bodyguard' cells from tumors, which will then allow the immune system to enter tumors and kill the cancer cells. He will test a new and selective nano-medicine he has created to kill 'bodyguard' cells. If successful, the new nano-medicine can help the immune system of patients with a range of childhood solid tumors, including neuroblastoma, one of the most common extra-cranial solid tumors in children where response rates remain low.

Young children with Down Syndrome are at very high risk of developing cancer of the blood (leukemia) before the age of 4. These leukemias arise from abnormal blood cells that are first detected in newborns with Down Syndrome. Studies suggest that the abnormal blood cells arise in the early embryo before a permanent blood system is set up. Dr. Palis has developed a unique way to study the biology of these abnormal blood cells. Researchers can now study the path from abnormal blood cell to leukemia in a dish. Using this system, he will learn how certain genes drive the change from 'abnormal blood cell' to 'cancer blood cell' that occurs specifically in very young children with Down Syndrome. Long-term goals are to prevent leukemia from forming and to develop safer treatments for those children with Down Syndrome who develop leukemia.

A growing number of childhood cancer survivors have been treated with high-dose chemotherapy. This high-dose chemotherapy places the survivors at risk for cognitive problems, affecting their thinking skills, processing speed, attention, and memory. Downstream effects of cognitive impairment include difficulties with independent living as they transition into adulthood. Currently, there is limited information as to whether cognitive remediation interventions can improve cognitive functioning in childhood cancer survivors. Cognitive remediation is important for teaching effective decision-making and reasoning skills to enhance self-management and self-advocacy, which in turn could result in improvement in other areas, such as employment and independent living. In particular, given the barriers to accessing medical care (e.g., distance to medical center, reliable transportation), Dr. Murdaugh will use telemedicine to deliver this intervention. This study aims to assess the efficacy of a 4-week intervention delivered via telemedicine that teaches strategies to improve decision-making and reasoning skills in cancer survivors between the ages of 10 and 18 years old.

Pain is one of the most common and distressing symptoms that children with cancer face. Pain can persist between clinic visits but may not be reported unless the child is specifically asked. Dr. Linder's team co-designed a game-based symptom reporting app, Color Me Healthy, with elementary school-age children with cancer. In the initial test of the app among children receiving chemotherapy, more than half of children's symptom reports included a report of pain. Parents reported that the app helped them better understand their child's symptoms. Children and parents also gave important feedback to improve the app. During the first phase of the study, Dr. Linder will use feedback from children and parents to make important changes to the app to enhance how children report pain using the app and how children's data can be understood by parents and clinicians. In the second phase of the study, she will ask children to use the newly enhanced pain assessment feature and ask how parents and clinicians used children's data to help relieve their pain. Completing this study will allow Dr. Linder to use the app in a larger study to improve pain management and quality of life among children receiving cancer treatment.

Neuroblastoma is a pediatric cancer that originates from mistakes in nerve cell development. Limitations in our mechanistic understanding of disease onset and progression have led to inaccurate patient risk predictions and over-treatment of infants, with long-term side effects. Recently, Dr. Kulesa and colleagues developed a computational model to predict neuroblastoma disease outcome based on a network of six development genes of receptor tyrosine kinase signaling that is more accurate at early disease stages than any current gene list algorithm. What remains to be determined is whether this model can be refined to increase its predictive value and tested to simulate hypothetical treatment strategies with individual patient data. To address these questions, he will include MYCN into the network model, a proto-oncogene gene that is correlated with poor prognosis, and compare model and experiment results of network perturbations that simulate targeted treatments. Dr. Kulesa will take advantage of acquired human neuroblastoma cell lines and our ability to modulate these genes in culture, and patient data from large-scale neuroblastoma genomic databases and published studies. At the conclusion of our study, he will have a better understanding of the mechanistic basis of neuroblastoma disease progression and a refined computational model to more rapidly and accurately predict individual patient disease outcome.

Rhabdomyosarcoma is a common cancer in kids. It can be a very aggressive disease, especially a type that is caused by a genetic change that creates an abnormal cancer-driving protein in the cell, called "P3F". P3F-driven rhabdomyosarcoma shows a strong tendency to spread to other parts of the body, which is what typically leads to death from the disease. P3F is a very hard drug target. However, P3F works together with other machinery in the cell to cause rhabdomyosarcoma. Such machinery could be targeted to interfere with P3F effects, but is not well understood. Dr. Jedlicka and colleagues have recently found new parts of this machinery that help P3F cause rhabdomyosarcoma to spread to other parts of the body. In this project he will better understand how this new machinery works and how it could be targeted to interfere with rhabdomyosarcoma spread. This work could identify new ways to inhibit the aggressive nature of this disease and improve patient outcomes.

This grant is generously supported by Marlee’s Smile, a St. Baldrick's partner, founded in honor of 12-year-old, Marlee Pack. Diagnosed with alveolar rhabdomyosarcoma; she relapsed three times in four years. After the final relapse, Marlee had to make a decision no child should have to: continue painful, toxic treatments or enter hospice care. She passed away on February 23, 2019. Our mission at Marlee’s Smile is to change the lives of kids with cancer, one smile at a time in two ways. We give a custom Build-A-Bear to every child fighting cancer, as well as their siblings to honor Marlee’s giving heart as she knew the comfort of a furry friend. We fund targeted research of pediatric cancers, specifically sarcomas to honor Marlee’s dying wish that no child should have to suffer the pain and hopelessness of current cancer treatments.

Pediatric high-grade gliomas (pHGGs) are a fatal childhood cancer of the brain. Deregulation of specific histone modifications, both with and without a direct link to specific mutations, have been identified in these tumors. This project will investigate histone H3 post-translational modifications (PTMs) in pHGGs to advance our understanding of tumor development and understanding of biologic characteristics, and to promote the identification of effective therapies for improving the outcomes for patients with these tumors.

This grant is generously supported by The Benicio Martinez Fund for Pediatric Cancer Research, a St. Baldrick's Hero Fund created in honor of Benny's fight with cancer and supports cures and better treatments for kids like him. Weeks after being the top fundraiser in his 6th grade class and shaving his head at his school’s event, Benny was diagnosed with medulloblastoma. Since then he has had brain surgery, radiation and chemotherapy. Despite complications from treatment and setbacks, Benny has an amazing can-do attitude and is battling the cancer with courageous determination.

Dr. Resnick's research project focuses on how to cure one of the deadliest brain tumors in children called diffuse midline gliomas (DMGs), previously also known as diffuse intrinsic pontine gliomas (DIPGs). No available cancer treatments work against DMGs and children die from this lethal disease within 8-11 months of diagnosis. To improve survival and develop better treatment against DMGs, he assessed genes being turned on or off in DMG tumor cells. Together with colleagues, he has identified novel gene products common in multiple DMG tumors that arise when two unrelated genes join and become expressed as one novel protein entity. Here, he will study the role of these gene products, or gene fusions, in DMGs, specifically those involving a known cancer-causing gene called MET. He will test drugs that target the MET gene fusions in DMGs by performing experiments on models that accurately represent human DMG tumors. The results from this project will help identify new drug treatment strategies to target DMG tumors in children. Successful therapy options from this study will be made available to children with DMGs in real-time through our partnership with a clinical trial consortium that brings new treatments to children with brain tumors.