Grants Search Results

Over-expression of HOXA9 protein in acute leukemias, which are cancers of the blood, is associated with worse outcomes. This over-expression occurs in more than 50% of acute myeloid leukemia (AML) cases and in approximately 75% of infant acute lymphoblastic leukemia (ALL) cases. In the laboratory setting, decreasing the level of HOXA9 in AML cells has been shown to reduce their growth. This project aims to develop a way to target HOXA9 in AML and infant ALL using short segments of DNA called oligonucleotides designed to decrease HOXA9 protein or prevent its function. The use of oligonucleotides as drugs has recently been successful in the treatment of various disorders. The goal of these studies is to eventually lead to the use of oligonucleotides as novel therapeutic agents in a clinical trial setting for treatment of AML and infant ALL.

Based on progress to date, Dr. Reitman was awarded a new grant in 2022 to fund an additional year of this Fellow award. Brainstem gliomas are deadly brain tumors that affect children. The only effective treatment is radiation therapy, but despite this treatment all children with this disease eventually experience growth of the tumor and eventually death. As the Emily Beazley's Kures for Kids Fund St. Baldrick's Fellow, Dr. Reitman will test if treatments that enhance the efficacy of radiation therapy can improve survival in the laboratory. This could lead to new clinical trials aimed at helping children with brainstem gliomas to survive longer.

This grant is funded by and named for Emily Beazley's Kures for Kids Fund. At the age of 8, Emily was diagnosed with Stage III T-cell lymphoblastic non-Hodgkin’s lymphoma and battled through three relapses. Her family prayed for a miracle but discovered Emily herself was the miracle, inspiring a community to come together to show love and change lives. She had a dream of starting a foundation to fund research and named it “Kures for Kids”. Today, Emily's family and friends carry on her dream and her mission in her memory.

Bone marrow transplantation is a highly effective treatment for relapsed and difficult to treat forms of pediatric leukemia, but unfortunately has a high risk for dangerous side effects. Viral infections are a major problem in the weeks and months after bone marrow transplant while children's immune systems are still immature. These infections can be debilitating and even deadly while also being very difficult to treat since available antiviral medications frequently do not work. Over the last few years, researchers have had great success in combating these viral infections by taking T-cells (a type of infection fighting cell that is part of the immune system) donated by children's personalized stem cell donors and engineering them to attack and kill certain viruses. Additionally, the rates of side effects using this therapy have been incredibly low. Dr. Rubinstein now intends to offer this therapy as a preventative measure, with the hope that this strategy will decrease the number of patients suffering from dangerous viral infections after bone marrow transplant. This clinical trial has the potential to decrease the number of pediatric cancer survivors who die from infection while also shortening hospitalizations and decreasing the need for other anti-viral medications.

This grant is generously supported by the Rally for Ryan Fund, a St. Baldrick's Hero Fund. Ryan was diagnosed with ALL when he was 7 years old and began treatment immediately. Initially labeled “high risk” due to a poor response, he completed 3½ years of a difficult treatment protocol before relapsing 11 months later. After his third relapse and an unsuccessful immunotherapy trial, Ryan had a bone marrow transplant in December 2020. He is currently fighting graft vs. host disease but is doing well and is optimistic for a good response. The Campanaros created this Hero Fund to celebrate Ryan’s courageous spirit and knowing firsthand the importance of research, to raise funds to find better treatments for kids with cancer.

For the past 15 years, Dr. Northcott has devoted his entire scientific training and early independent career to studying the biological basis of medulloblastoma in large collections of patient tumors. From these efforts, his research has demonstrated that medulloblastoma is not a single disease, but rather a collection of different diseases referred to as subgroups, each of which is associated with distinct genetics, distinct age of onset, and distinct survival patterns. These findings have begun to directly impact how children afflicted with medulloblastoma are diagnosed and treated in the clinic. Currently, his lab consists of a team of basic scientists, computational biologists, and support staff that work collaboratively to understand fundamental questions related to the biological and clinical aspects of the different medulloblastoma subgroups. Leading a scientific program focused on medulloblastoma at St. Jude, Dr. Northcott has the privilege of being part of a collaborative research environment that facilitates working alongside pediatric neuro-oncologist’s leading world class clinical trials, allowing them to combine their expertise to determine why some children survive medulloblastoma and others do not. This group is currently pioneering and implementing innovative technical approaches to study extensive collections of medulloblastoma patient samples at the level of individual genes in single cancer cells. Information gained from these studies enables researchers to accurately model the different medulloblastoma subgroups in the lab and test new therapies before they are evaluated in clinical trials. Overall, his goal is to continue to make transformative discoveries related to the molecular, biological, and clinical characteristics of medulloblastoma subgroups that will improve treatments and outcomes for affected children and their families. 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.

Although rates of cure for childhood cancer have greatly improved in recent years, thousands of children continue to suffer from advanced, incurable cancer. Healthcare professionals bear a responsibility to ensure that the care of children with advanced cancer meets the goals and wishes of patients and their families. However, we do not know whether we fulfill this aim in pediatric cancer care. Dr. Ananth's prior research reveals intensive healthcare use near the end of life for children with advanced, incurable cancer. This includes lengthy stays in the intensive care unit and common use of interventions like breathing tubes. Yet, healthcare professionals worry that intense care toward the end of life for children with cancer may increase child and family suffering. In adults with cancer, quality measures have been developed to evaluate where care is most intense, or poor quality. This has consequently allowed researchers to develop interventions to improve the quality of care for adults with incurable cancer. Unfortunately, there are no comparable measures or standards for what constitutes good, or high quality, end-of-life care for children with cancer. Dr. Ananth seeks to address this problem. The overall objectives of this research are to (1) refine a list of potential measures of high quality end of life for children with cancer, and (2) develop an innovative questionnaire to systematically evaluate whether patients are receiving high quality end-of-life care. She hopes that, through this work that is distinctly family-centered, she can develop interventions to enable healthcare teams to provide optimal, compassionate care for children who have incurable cancer.

Based on progress to date, Dr. Bertrand was awarded a new grant in 2022, 2023, and 2024 to fund an additional year of this Scholar grant. Ependymoma is an aggressive pediatric brain tumor that is treated with surgery and radiation, but is resistant to chemotherapy. Ependymoma can be divided into different groups by location and biology. One type of ependymoma is driven by a fusion cancer-causing protein RELA-fusion. There are currently zero available drug therapies that target this protein, and we have a poor understanding of its function in cancer. Dr. Bertrand's research seeks to understand how this protein induces cancer in cells and models so that we can devise new treatments.

The 2024 portion of this grant is funded by and named for Hannah’s Heroes, a St. Baldrick's Hero Fund established to honor Hannah Meeson. At age six she was diagnosed with anaplastic medulloblastoma. After a relapse and several additional months of treatment, Hannah currently shows no evidence of disease. Throughout her treatments, Hannah never complained and remained positive and happy. This fund pays tribute to her fight by raising awareness and funding for all childhood cancers because kids like Hannah “are worth fighting for.”

Awarded at Baylor College of Medicine and transferred to St. Jude Children's Research Hospital.

Based on progress to date, Dr. Bonifant was awarded a new grant in 2022 and 2023 to fund an additional year of this Scholar grant. There is a clear need for new treatment options for pediatric relapsed or refractory acute leukemia. Patients receiving standard chemotherapy regimens continue to suffer a poor prognosis. Immunotherapy has the potential to fulfill this need through its precise tumor targeting and novel anticancer biology. Recently, a type of immune cell, the T cell, has been genetically modified to specifically recognize leukemia cells through a specialized receptor (CAR). Infusion of CAR-T cells has shown stunning anti-leukemia activity in clinical trials. However, this approach has key limitations. Importantly, CAR-T cell persistence in each patient is critical to ensure continued disease remission. Alterations in leukemia surface protein expression have also been reported as a mode of CAR-T evasion. These may lead to disease relapse. Dr. Bonifant and her colleagues believe they can introduce new elements to existing chimeric receptors to promote improved CAR-T survival. They also believe they can alter these cells to target more than one surface antigen -- to prevent antigen loss and CAR-T escape, while enhancing specific disease targeting. The overall rationale for this proposal is the confidence in CAR-T cells as a powerful addition to anti-leukemia therapy. Dr. Bonifant would like to facilitate continued improvement of this therapeutic modality in order to ultimately attain durable cures.

The 2021, 2022, and 2023 portions of this grant are funded by and named for Emily Beazley's Kures for Kids Fund, a St. Baldrick's Hero Fund. At the age of 8, Emily was diagnosed with Stage III T-cell lymphoblastic non-Hodgkin’s lymphoma and battled through three relapses. Her family prayed for a miracle but discovered Emily herself was the miracle, inspiring a community to come together to show love and change lives. She had a dream of starting a foundation to fund research and named it “Kures for Kids”. Today, Emily's family and friends carry on her dream and her mission in her memory.

The 2020 portion of this grant is funded by and named for the Rally for Ryan Fund, a St. Baldrick's Hero Fund. Ryan was diagnosed with high risk ALL when he was seven years old. He endured 3 ½ years of often harsh treatments with smiles, laughter and a brave acceptance that this was his fight to win. And Ryan did prevail—he took his last chemo pill in January 2016 but relapsed at the end of the year. He endured CAR T-cell therapy and a bone marrow transplant and is once again cancer-free. This fund honors Ryan’s commitment to help make a difference for kids with cancer.

Based on progress to date, Dr. Chavez was awarded a new grant in 2022 to fund an additional year of this Scholar grant. Researchers have found that some very aggressive cancers produce extra pieces of DNA that are located outside of our 23 chromosomes and form circles. This is why we call them circular extrachromosomal DNA, or ecDNA. These ecDNAs are thought to be a fundamental driver of cancer growth. However, very little is known about ecDNA in childhood brain tumors. This is why researchers have now looked for ecDNA in medulloblastoma- a cancerous brain tumor that starts in the lower back part of the brain, called the cerebellum. Medulloblastoma can occur at any age, but most often occurs in young children. Though medulloblastoma is rare, it's the most common cancerous brain tumor in children. And indeed, we have observed that there are very specific types of ecDNA in medulloblastoma tumors, especially in those tumors that are very aggressive and difficult to treat. As the Hannah's Heroes St. Baldrick's Scholar, Dr. Chavez would like to learn more about ecDNAs in medulloblastoma and hopes that this will lead to a scientific revolution in how some of the most difficult-to-treat childhood brain tumors are understood and treated.

This grant is named for Hannah’s Heroes, a Hero Fund established to honor Hannah Meeson. At age six she was diagnosed with anaplastic medulloblastoma. After a relapse and several additional months of treatment, Hannah currently shows no evidence of disease. Throughout her treatments, Hannah never complained and remained positive and happy. This fund pays tribute to her fight by raising awareness and funding for all childhood cancers because kids like Hannah “are worth fighting for.”

This grant was awarded at the University of California, San Diego, and transferred to Sanford Burnham Medical Research Institute.

Unfortunately, kids with cancer suffer from symptoms related to their cancer and cancer treatments. These symptoms are often not fully appreciated by their health care providers and hence may be undertreated. Doses of cancer treatments are decreased or cancer treatments are even stopped when symptoms from cancer treatments aren't well controlled. Improving symptom assessments for kids with cancer will enhance health care providers' ability to track and manage kids' symptoms, to identify symptom trends, and could even prevent changes in cancer treatments due to poorly controlled symptoms. The Patient Reported Outcome Common Terminology Criteria for Adverse Events (Pediatric PRO-CTCAE) is a recently-developed survey that allows kids with cancer and their parents to directly report the type and severity of kids' symptoms to health care providers. This tool includes 130 questions about 62 different symptoms. However, since there are so many questions in this tool, the symptoms that are asked about are pre-selected by researchers or health care providers. Pre-selecting the questions prevents kids and parents from reporting all of a kid's symptoms, risking symptoms being missed. The overall objective of Dr. Crane's research is to refine and pilot test a novel web-based interface for the Pediatric PRO-CTCAE that will allow kids and parents to systematically and easily report all of a kid's symptoms on a routine basis, but without having to answer 130 questions.

Based on progress to date, Dr. Hoang-Minh was awarded a new grant in 2022 to fund an additional year of this Scholar grant. Brain tumors are the most common cause of cancer-related deaths in children. The current treatments are often associated with lifelong mental and motor deficits, and the tumors often recur. Therapies that specifically and efficiently target the tumors and minimize toxicity to the body are critical to improve clinical outcomes for children affected by these deadly diseases. As the Pray for Dominic St. Baldrick's Scholar, Dr. Hoang-Minh's research is exploring a powerful method that uses the children's own immune system to destroy their brain tumors, known as immune cell therapy. This therapy has emerged as a very effective and safe treatment for blood cancers and several types of solid tumors. It uses powerful immune cells, called T cells, to specifically kill the brain cancer cells and has already shown promising results in preclinical and clinical studies conducted at our institution. This project investigates novel approaches to make this immune therapy even more effective and safer. Dr. Hoang-Minh will also follow the fate of therapeutic T cells using a new, non-invasive imaging technology called magnetic particle imaging. The results of these studies are important as they could improve clinical protocols using immune cell therapies for childhood brain tumors and extend or save the lives of children afflicted with those very aggressive cancers.

This grant is named for the Pray for Dominic Hero Fund. The fund was established in honor of Dominic Liples who lived with joy. He is remembered for compassion and determination while he faced his own difficult battle with a rare and aggressive brain cancer. The Pray for Dominic fund carries on Dominic's legacy of joy and hope by funding research for high-grade gliomas.

Acute myeloid leukemia (AML) is an aggressive pediatric cancer associated with poor outcomes. Current therapies are toxic and result in a high incidence of late effects; including infertility, heart failure, and second cancers. Therefore, distinguishing who will be cured with chemotherapy alone from those who require more intensive therapies is critical to improving cure rates in AML while limiting treatment related late effects. The presence of small numbers of persisting leukemia cells after chemotherapy has become an important predictor of leukemia relapse. However, current assays used to detect residual leukemia have limited sensitivity and many patients with "no detectable leukemia" still go on to relapse. This underscores the need to identify and develop more accurate and sensitive leukemia detection assays for AML. This project aims to develop a novel assay that harnesses "best in class" technologies to enable detection of one leukemia cell for every one million normal cells -- a sensitivity that eclipses the current standard of care by more than one hundred-fold. Additionally, unlike many other novel methods for detecting leukemia, this assay will be universally applicable to every patient diagnosed with AML. Finally, this assay will reveal not simply whether or not leukemia cells are present, but the exact genetic code comprising the remaining leukemia cells. Successful validation of Dr. Huang's assay will therefore fill a critical unmet need in the field of AML, and the resulting product will be an optimized test ready for clinical use.

A portion of this grant is generously supported by RowOn 4 A Cure, a St. Baldrick's Hero Fund. Rowan was a happy, spunky, funny, smart, and smiley little girl. With that same tenacity, she faced her cancer diagnosis of a rare form of acute myeloid leukemia when she was three. Despite intense chemotherapy and radiation and a successful cord blood transfusion, Rowan relapsed after a brief remission. The family relocated in search of another treatment option but before one could be found, Rowan sadly passed away. RowOn 4 A Cure was established to honor Rowan and continue her fight against AML by raising awareness and funds for research to find better options for treatment of relapsed AML and ultimately, a cure for the disease. Her family remembers Rowan’s perseverance during tough treatment days and intend to make an impact as they “Row On” to find a cure.

Cancer immunotherapy has demonstrated great potential for treating cancer. However, challenges such as 1) the lack of ideal targetable tumor antigens; 2) severe toxicity due to off-target interactions; and 3) tumor-mediated immunosuppression are limiting the success of immunotherapies to be broadly applicable. To potentially overcome these challenges, Dr. Wu and his colleagues have developed a programmable synthetic gene circuit platform that enables tumor-localized therapeutic payload production, for recruitment and activation of immune cells: Tumor Immuno-therapy by Gene-circuit Engineered Response (TIGER). This strategy makes use of the body's own immune system to kill tumor cells. Gene circuits (highly engineered DNA sequences that work together), delivered systemically, will be turned on by the presence of two cancer-specific signatures, therefore only be activated within cancer cells and not normal cells. Cancer cells will be forced by the activated gene circuits to produce immunomodulators. Dr. Wu has demonstrated that TIGER mediates robust therapeutic efficacy in vivo in solid tumors. They have also identified sensors that can distinguish high-grade stem-like glioma cells from non-stem-like glioma cells. To further accomplish clinical translation of this platform, several advances are required: 1) identification of tumor sensors that efficiently detect highly heterogeneous primary patient tumors, to optimize tumor-targeting efficiency and specificity; and 2) optimization of therapeutic output combinations for achieving maximal efficacy. This project will focus on advancing the above two aspects to facilitate clinical translation of TIGER to treat pediatric high-grade glioma and overcome existing barriers to effective immunotherapy.

The first year of this grant is funded by and named for the Kai Slockers Pediatric Cancer Research Fund. Kai was diagnosed at 2½ with Atypical Teratoid Rhabdoid Tumor (ATRT), a rare and very aggressive brain cancer. Within two weeks of diagnosis, he passed away, a mere 3 months shy of his third birthday. When Kai took his last breath, the cloudy sky opened up with a bright ray of sun that streamed through the windows of his hospital room – the darkness of the disease was replaced with the light of hope and the peace of no more suffering. Whenever the sun is out, his family thinks of him, assured that his legacy of hope shines on. In his brief life, Kai shared his warmth, energy, goofy sense of humor, and caring heart with all those he met. This Hero Fund was created in his memory and will support research to help other kids with cancer have a better chance to fight and survive. It has a special focus on brain tumor research, specifically treatments that could minimize the harsh effects of brain tumor treatment. The Slockers family hopes to continue his legacy of light and hope through the funding of childhood cancer research.

A portion of this grant is generously supported by the Derick the Defeater Fund, a St. Baldrick's Hero Fund. Derick was a hero in so many ways. Diagnosed with medulloblastoma at the age of six, he endured 2 years of treatment with determination and a positive spirit. He inspired friends and family with his smile and charisma, even advocating for other children fighting cancer and teaching everyone what it meant to be brave. Derick’s courage lives on in a comic book his friends created called “Derick the Defeater” about a superhero who fought evil villains that looked like cancer cells. This Hero Fund honors his legacy of helping others through the funding of childhood cancer research.

Cancer remains the leading cause of illness-related death in children. Researchers now know that inherited genetic abnormalities contribute to cause cancer in many more children than previously understood. Children with Cancer Predisposition Syndromes (CPS) have a high risk of cancer, and we have a unique opportunity to prevent or detect cancer at early stages to improve their survival, as well as to better understand mechanisms of cancer development for all pediatric tumors. However, these CPS are rare and difficult to study in individual institutions. Thus, the Consortium for Childhood Cancer Predisposition was created, through which researchers aim to better understand the tumor risks in these patients and their distinct biology. Also, they will study surveillance and prevention strategies efficiently, by utilizing coordinated efforts to collect data and samples from patients with CPS (through a registry and biorepository). The Consortium currently comprises seven institutions, is led by experts who have already collaborated to have major impacts in this field, and has engaged syndrome specific advocacy groups to accelerate patient-centered advances. The impact of this Consortium will be improved outcomes for children at highest risk of cancer through better identification, early tumor detection, optimized psychosocial support and cancer prevention. Funds administered by Emory University.

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.

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.

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.

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.

Acute lymphoblastic leukemia (ALL) is the most common cancer of children, and although treatment is considered largely successful, in many cases leukemic cells stop responding to chemotherapy and re-emerge. As a consequence, ALL relapse remains a leading cause of childhood cancer-related death. Dr. Aifantis will test the possibility that the bone marrow microenvironment surrounding the leukemia supports the growth of disease and protects leukemia cells from chemotherapy. Together with colleagues he generated the first map of the ALL immune cell microenvironment allowing identification of novel players within the remodeled leukemic bone marrow that promote leukemia survival. They found that high levels of a specific cell type, known as non-classical monocytes, in ALL patient blood and bone marrow correlates with inferior patient survival. They demonstrated that depletion of leukemia-supporting monocytes enhances killing of leukemic cells with specific ALL therapies. In this project Dr. Aifantis will investigate the processes giving rise to monocytes capable of supporting leukemia survival. Further, he will use novel model systems to test whether targeting monocytes enhances responses to a range of existing ALL therapies as well as emerging approaches, such as Chimeric Antigen Receptor (CAR) T-cell therapy, that utilize a patient's own immune system to kill leukemic cells.

Pediatric cancers are often comprised of mixtures of cells with different characteristics. Some of the most important differences relate to chromosomal changes, with some cells having a normal or nearly normal chromosome profile, others having altered numbers of intact chromosomes, and yet others having extra or missing copies of one or more chromosome segments. Prior studies have shown that cancers with more segmental changes are usually more aggressive and therapy-resistant, but the specific effects associated with the different chromosomal changes are unknown. Here Dr. Cobrinik and colleagues will define the effects of such changes in two pediatric cancers -- retinoblastoma and neuroblastoma -- by isolating individual cells within the tumors that either have or lack specific chromosome changes, comparing their overall gene expression and cell signaling profiles, and identifying the critical changes that increase malignancy. The study involves three investigators with expertise in neuroblastoma, retinoblastoma, and a novel single cell sequencing approach that enables us to distinguish and characterize the chromosomally distinct cells within individual tumors in unmatched detail. This study is expected to reveal the most central features that distinguish more versus less aggressive cancers, as a critical step towards targeting and subduing the more aggressive and lethal cells within individual tumors.

Precise understanding of the basic mechanisms by which childhood cancers develop is essential to design tailored and superior treatments for cancer patients. These treatments are expected to cure and avoid long-term complications in cancer survivors. In many instances, we turn to models to reproduce human cancers, but the success of this strategy depends on how accurately we can unravel the origin of the disease. This project is based on Dr. Dominguez-Sola and colleagues recent findings on the origins and cellular basis of Burkitt lymphoma, a most aggressive form of childhood lymphoma with little treatment alternatives. This project will use unprecedented models of this cancer type to expand our understanding of the mechanisms of disease and identify therapeutic strategies that are less toxic, more effective, and superior to those currently available in the clinic.

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.