Grants Search Results

NUT midline carcinoma (NMC) is a deadly cancer that affects children and young adults, with a survival of less than 7 months. NMC is caused by a protein called BRD4-NUT that changes the structure of DNA in such a way that the DNA drives expression of cancer-associated genes that promote growth of NMC. Dr. French proposes to determine what is actually happening to the structure of the DNA that allows it to express the cancer-driving genes. There are two protein types he suspects are helping BRD4-NUT distort the DNA conformation; these are called HDACs and HATs. Dr. French's team will use state-of-the-art inhibitors that target specific HDACs and HATs to determine their respective roles and help identify novel therapeutics to treat this incurable disease.

Leukemia is a blood cell cancer that frequently affects children. Despite the advances in treatment options, children with certain subtypes of leukemia are resistant to current therapy. Novel therapy for childhood leukemia is urgently needed. Dr. Fang's team recently found a protein, whose name is GPR68. They found that the levels of GPR68 were increased in blood cells of leukemia patients. When they decreased GPR68 levels, leukemia cells died, suggesting that increased GPR68 helped leukemia cells survive. Interestingly, normal blood cells with reduced levels of GPR68 were normal, suggesting that only leukemia cells need GPR68. Dr. Fang's findings suggest that lowering GPR68 levels or limiting its function may help cure leukemia without injuring normal blood cells. As the recipient of the Emily Beazley's Kures for Kids Fund St. Baldrick's Research Grant, she will be working to understand the function of GPR68 in leukemia cells and normal blood cells, and test drugs that could cure leukemia by inhibiting GPR68 function.

This grant is 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.

Neuroblastoma is a cancer formed in certain types of nerve tissue and is the most common pediatric solid tumor outside of the brain. It is the leading cause of cancer-related death in children under five years old. Those patients who do survive often develop long-term side effects from intensive chemotherapy and radiation therapy. Thus, we need to develop better, safer alternative therapies for neuroblastoma. Dr. Cripe is currently studying the use of genetically modified herpes viruses. These viruses, which include the recently FDA-approved herpes virus T-VEC, can selectively infect and kill cancer cells without harming normal cells. In addition, these viruses are also able to enhance the patient's immune response against the cancer cells, potentially leading to a systemic and long-lasting protective immunity against cancer dissemination and recurrence. In the course of his studies, Dr. Cripe found that tumors infected with virus induce a counter measure by attracting cells that suppress immunity. In this study, he will test if he can improve therapy by interfering with that counter measure. If successful, these results may lead to a novel clinical trial for neuroblastoma patients.

Current cancer therapy is very toxic and does not always work. We have developed a way to deliver much more drug to the tumor, and much less to the patient, by packaging the drug in properly designed nanomedicines. These delivery systems take advantage of the fact that most aggressive tumors have leaky blood vessels, so our nanomedicines can pass through into the tumor, but they bypass most normal tissues. Using these formulations, we can deliver 10-100 times as much drug to the tumor, so we can use less total drug and still get better results. In addition, Dr. Brodeur is using a novel drug called SN22. Although SN22 is related to a commonly used chemotherapy agent called irinotecan, it is an active drug, and unlike irinotecan it does not have to be activated by the liver. It is not only much more potent but also harder for the tumor cells to get rid of. These features make SN22 much more therapeutically effective. The carrier Dr. Brodeur is using to make this nanomedicine can deliver four molecules of SN22 within each “packet” that enters the tumor. Because he can use less total drug, and because the nanomedicine can circulate for a long time with the drug attached, there is much less exposure to the rest of the body, so side effects are dramatically reduced. As the recipient of the Invictus Fund St. Baldrick's Research Grant, Dr. Brodeur's goal is to develop more effective but less toxic therapy to treat children with cancer, and he can accomplish that goal with this approach using nanomedicine-based drug delivery. The nanomedicines he is developing should be effective against many different solid tumors in children or adults and he hopes to bring them forward to Phase 1 clinical trials.

This grant is funded by and named for the Invictus Fund, a St. Baldrick's Hero Fund created in memory of Holden Gilkinson and honors his unconquerable spirit in his battle with bilateral Wilms tumor as personified in the poem “Invictus” by William Ernest Henley. His family hopes to fund cures and treatments to mitigate side and late effects of childhood cancer.

Neuroblastoma is a cancer of the nervous system that causes aggressive disease in infants and young children, and the overall survival rate of high-risk (stage IV) patients is low. Ornithine decarboxylase (ODC) is a validated target in several cancers and we advanced the ODC inhibitor DFMO into neuroblastoma clinical studies. While promising, large quantities of DFMO are needed for patient treatments because about 80% of the drug is released into the urine. To improve the retention of DFMO in the blood, this study explores the combination of DFMO with an FDA-approved adjuvant. We expect that DFMO in the presence of this adjuvant will (a) increase the DFMO concentration in the blood and (b) induce more potent anti-tumor effects in neuroblastoma tumor-bearing mice. Since both DFMO and the adjuvant are FDA-approved drugs, this new regimen could rapidly advance to neuroblastoma clinical studies.

Pediatric undifferentiated sarcomas are highly aggressive cancers that typically affect soft tissues of young children. Due to their uncertain classification and lack of molecular signature there are no standard criteria for diagnosis or treatment. With the Alan's Sarcoma Research Fund St. Baldrick's Research Grant, Dr. Antonescu is applying state of the art genomic methods to provide a detailed genetic characterization in these orphan cancers and investigating driving chromosomal translocations or mutations involved in their growth. These results will establish an objective classification of these tumors based on their genetic abnormalities and will provide potential therapeutic targets for further novel therapies. Furthermore these findings will inform the generation of faithful models for studying sarcoma formation and new drug development.

This grant is funded by and named for the Alan's Sarcoma Research Fund, a St. Baldrick's Hero Fund. Alan Sanders was diagnosed with a rare sarcoma in his hip at 17 months. He had an indomitable spirit and throughout his 4 ½ year battle with cancer, he was joyful, upbeat and pressed on courageously in spite of surgery and treatments. Today his family and friends carry on his legacy and his rallying cry, “Fight’s on!” in the battle against childhood cancer by funding sarcoma research.

Phthalates are chemicals added to many products that we use every day, including some common medications. Phthalates interfere with hormone systems in our bodies, which might cause cancer. Dr. Ahern wants to know if phthalate exposure while in the womb or during childhood increases the risk of childhood cancer. It would usually be time-consuming and expensive to answer this question scientifically. However, Dr. Ahern's team has developed a way to measure phthalate exposure using electronic pharmacy records that is both fast and inexpensive. This technique works because phthalate exposure from medications dwarfs exposure from other products. He will use this technique on existing pharmacy and cancer data from the entire population of Denmark. Dr. Ahern will measure phthalate exposure in pregnant women and in their children, and calculate whether that exposure increases a child's chances of developing cancer. If he finds that it does, we could prevent childhood cancer cases by limiting the amount of phthalates used in consumer products.

In the U.S., children with a blood cancer called Hodgkin lymphoma (HL) are usually treated successfully. Some of these children will suffer health problems several years later because of the treatment they received. Because of this, doctors use powerful imaging tools to identify patients who are likely to do well or not. Those who are likely to do well require less treatment and those who are less likely to do well can receive more treatment. But in low-income countries like Malawi, these tools are unavailable, and the children there often receive treatment that may be unnecessary. Scientists have found unique abnormalities in adults with HL that can tell us who is less likely to do well. Here, Dr. Ozuah is testing whether these abnormalities are present in children and could be used to decide how best to treat children with HL in low-middle income countries

Osteosarcoma is the most common primary bone tumor in childhood. The survival rate remains dismal, mainly due to ineffective therapeutic approaches for the relapsed/metastatic patients. One major obstacle of treating osteosarcoma is lack of suitable preclinical models. Dr. He's studies have established the first cultured osteosarcoma tissue model (an organoid). Dr. He aims to establish the first biobank of osteosarcoma organoids from patients as an open resource for the field, and utilize this organoid biobank to evaluate a novel class of therapeutics targeting key signaling pathways in osteosarcoma cells. This study will provide a powerful platform for predicting clinical treatment responses and developing new therapeutics for treating osteosarcoma.

Cancer cells rely on specific nutrients for growth and survival, rendering nutrient restriction as a potential therapeutic strategy. Along this line, acute lymphoblastic leukemia (ALL) cells have been found to be dependent on exogenous supply of asparagine, a nonessential amino acid, for protein synthesis. As a result, depletion of asparagine in the blood stream by L-asparaginase, a chemo-agent, has been successfully used to treat pediatric ALL for 40 years. However, ALL patients can develop resistance to the continuous application of this chemo-agent. Dr. Zhang is determining how ALL cells become resistant to L-asparaginase treatment, and therefore to provide experimental evidence of novel therapeutic targets that can potentially improve the outcome in pediatric ALL patients.

Sarcomas are tumors of the bone and soft tissues that comprise up to 20% of cancer diagnoses in children. Despite dismal outcomes for patients with recurrent or metastatic disease, treatment regimens have remained largely unchanged for decades – intense non-specific chemotherapy combined with surgery or radiation. Dr. Tulpule studies Ewing’s sarcoma (ES), a bone tumor caused by a unique genetic change that creates a tumor-specific protein EWS-FLI1. To date, no drug has been identified to directly block the cancer causing EWS-FLI1 protein. His research takes a different approach to combating ES by asking a fundamental question: can we identify a targetable weakness in ES tumors that is caused by the EWS-FLI1 protein? Using a cutting-edge screening technology called CRISPR interference, Dr. Tulpule's team identified a specific vulnerability in ES cells’ capacity to repair damage to their DNA. Normal cells have many backup systems in place to repair DNA damage, but they have shown that EWS-FLI1 causes ES cells to become overly reliant on a single pathway, known as homologous recombination (HR) repair, such that blocking HR is an effective and specific way to kill ES. Dr. Tulpule is building a detailed understanding of why ES cells are so vulnerable to HR pathway blockade and then applying that knowledge towards developing less toxic and more effective treatments for ES patients.

Despite improvements in outcome for patients with acute lymphoblastic leukemia (ALL), up to 25% of children and 40% of adults fail frontline therapy and their prognosis is dismal, especially for high-risk and relapsed leukemia. Cure rates for ALL patients that are relapsing on therapy is approximately 20% and currently there are no targeted therapies. Dr. Tsirigos' goal is to address this significant clinical need by studying how DNA, our genetic material, is organized inside the nucleus of the cells, i.e. how it is folded in three-dimensional space. Over the past decade, seminal studies have demonstrated that DNA folding is of fundamental importance in that it allows the cell to properly perform its function and retain its identity (e.g. a liver cell versus a blood cell). Very recent studies have demonstrated that disruptions of DNA folding may cause various diseases, such as developmental defects and cancer. However, no study has addressed the question of disruptions of DNA folding on the genome-wide scale in cancer or how such disruptions may be exploited for more effective treatments. Dr. Tsirigos is attempting to answer two key questions. First, are cancer-promoting genes (“oncogenes) capable of disrupting normal DNA folding to transform normal cells into malignant ones? And, second, can drug treatment restore DNA folding and thereby also restore normal cell function?

This grant is made with generous support from the Rally for Ryan Fund. Ryan was diagnosed with high risk ALL when he was 7 years old. He endured 3½ years of treatments with a brave acceptance that this was his fight to win. He recently relapsed and is in the fight again. This fund honors Ryan’s perseverance and his commitment to make a difference for kids with cancer by shaving for St. Baldrick’s and raising funds for research.

Based on progress to date, Dr. Osborn was awarded a new grant in 2022 and 2023 to fund an additional year of this Scholar grant. Abnormal growth of B-cells can result in leukemia, and a cutting-edge treatment option is immunotherapy with T-cells. T-cells can be engineered to express a chimeric antigen receptor (CAR) that is a 'seek and destroy' molecule for the CD19 protein on B-cells. CAR T-cells are the first FDA approved gene therapy and some stunning therapeutic responses have been observed. However, the T-cell activity can be so robust that they cause a massive cytokine storm that can be lethal. Furthermore, normal and cancerous B-cells express the CD19 protein targeted by the CAR, so normal B-cell loss occurs resulting in an impaired immune system. These side effects represent a significant hurdle in the safe and effective treatment of B-cell leukemia. To address this, Dr. Osborn, will express the CAR in a specialized subset of cells called T-regulatory (Treg) cells. Tregs have the same potent killing ability as T-cells but accomplish it without healthy tissue collateral damage. Additionally, he will engineer functional B-cells that are invisible to the CAR. This will allow for normal B-cell numbers and an intact immune system. Dr. Osborn will conduct these studies that are structured to resolve an unmet need, are highly novel, and are poised to make an immediate impact on childhood leukemia. 

The 2022 portion of this grant is named for the Rays of Hope Hero Fund which honors the memory of Rayanna Marrero. She was a happy 3-year-old when she was diagnosed with Acute Lymphoblastic Leukemia (ALL). She successfully battled ALL, but a treatment induced secondary cancer claimed her life at age eight. Rayanna had an amazing attitude and loved life. She, like so many kids facing childhood cancer, did not allow it to define who she was. This Hero Fund aspires to give hope to kids fighting cancer through research.

A portion of this grant was funded by and named for the Mighty Mimi Hero Fund. Mimi Enyon was diagnosed with acute lymphoblastic leukemia at the age of 3. Her courage in the fight was unparalleled and she became “Mighty Mimi” to all those she inspired on her way to remission. This fund was established to share Mimi’s cancer journey in an effort to raise awareness and funding for childhood cancer research for kids like her.

A portion of this grant is generously supported by the Stanley Kuzmickas Feeney Fund for Pediatric Cancer Research. It was Christmas 2015 when Stanley was diagnosed with acute lymphoblastic leukemia at 13 months old. He courageously endured treatments for 3½ years. Today, he is in remission and eagerly started school in fall 2019. In his honor, Stanley’s family has organized a head-shaving event each year since July 2016 called “StoshyStrong." With the funds raised, the Feeney family created this Hero Fund to support research in new discoveries, genomics and other biological therapies for the treatment of ALL. Their goal is to one day see personalized treatments for every child.

Children with high-grade gliomas, such as glioblastoma multiforme, have few therapeutic options and usually die of their disease. CAR T cells recognize protein targets on cancer cells and kill those cells. Many brain tumors express target proteins on only some of their cells and therefore cannot be efficiently treated with a CAR T cell that recognizes only one target. Therefore, Dr. Majzner aims to make T cells that can recognize up to four targets. He is exploring the best way to achieve specificity (the narrowness of the range of substances with which an antibody or other agent acts or is effective) for four antigens including using gene editing in order to make CAR T cells that can come from a healthy donor but be used in any patient.

A portion of this grant is funded by and named for the Be Brooks Brave Fund. Despite his diagnosis at age 5 with inoperable brain and spinal tumors, Brooks taught so many people what life is truly about--love. He was BRAVE beyond his years with an inspiring “faith over fear” attitude. This Hero Fund hopes to raise money for high-grade glioma research so no other family will hear the words, “there is no cure”.

A portion of this grant was also generously co-supported by the McKenna Claire Foundation, a St. Baldrick's partner and the Living for Luker Brain Tumor Research Fund, a St. Baldrick's Hero Fund. The McKenna Claire Foundation was established by the Wetzel family in memory of their daughter, McKenna. Their mission is to cure pediatric brain cancer by raising awareness, increasing community involvement and funding research. The Living for Luker Brain Tumor Research Fund was established in memory of Luke's love for life and caring for others. He was diagnosed at age 10 with Diffuse Intrinsic Pontine Glioma, a rare, uncurable cancer and never gave up hope throughout treatment.

There is a new and effective cancer treatment for some incurable pediatric blood cancers. The treatment involves programing a patient's own cells to destroy their tumor, a process called cellular immunotherapy. Despite great effort to use cellular immunotherapy to treat 'solid' tumors, which include tumors of the bones, muscles and other parts of the body, we have not been successful yet. One major reason is that the programmed patient cells are designed to recognize a single marker on a cancer cell. In some blood cancers, all of the tumor cells express the same marker increasing the likelihood that cellular immunotherapy can cure the patient. Solid tumors are more heterogeneous than blood cancers, meaning each solid tumor cell may express a different marker. Therefore, cellular immunotherapy is less likely to destroy all solid tumor cells and the chances of achieving a cure is much more difficult. A potential solution is to trigger the body's own immune system to destroy tumor cells that express many different markers, a process called "epitope spreading". Named as the David's Warriors St. Baldrick's Scholar, Dr. Leibowitz focuses his project on testing strategies to augment epitope spreading in pediatric solid tumors so that cellular immunotherapy may become an effective and viable treatment option in the future.

This grant is named for and generously supported by the David’s Warriors Hero Fund created in memory of David Heard who battled neuroblastoma and inspired his family and countless others to commit to raising money for research to fight pediatric cancer. This fund honors the amazing spirit in which he lived, embracing life until the very end.

Ewing sarcomas are bone cancers that impact many adolescents and young adults. Despite the use of intensive traditional chemotherapy combined with advanced surgical techniques, 30-40% of patients still die after the disease eventually spreads to other organs, such as the lungs and bone marrow. Dr. Hayashi's team believes the key to overcoming this problem lies in the identification of “Circulating Tumor Cells (CTC)”. These are cells that break away from the original tumor and travel through the blood stream, eventually taking root in another organ to form what is called metastatic disease, meaning the cancer has spread from where it started into different areas of the body. These cells undergo multiple changes in order to leave the original tumor and survive in the harsh environment of the blood stream, eventually leaving the blood stream to invade another organ where they multiply and grow. This project aims to dissect each of these complicated steps with the goal of unveiling which element of this devastating process can be targeted to disrupt it.

People always say we need to outsmart cancer to beat it. As the Hope with Hazel St. Baldrick's Scholar, Dr. DuPage is not sure we will ever be able to do this with simple chemicals or radiation beams. Not only are these strategies rarely specific for the cancer cells alone, often leading to severe side effects that can be lifelong, but cancers always find a way around these single agents and "relapse." What if we could use a "living drug" to treat cancer? A drug that was as wiley and adaptable as the cancer itself and would last for a lifetime? A smart drug! Our own immune systems are capable of fighting our own cancers with extreme precision if we can train them to do it. This research is focused on understanding how cells of our immune system interact with tumor cells and how we can train our own immune cells to fight our own cancers. It is called cancer immunotherapy, and for children with cancer, Dr. Dupage thinks there are no better patients because their young and healthy immune systems are perfectly suited to be trained to fight cancer, removing the need for procedures that can manifest dangerous side effects in adulthood, and safely protecting them for life.

This grant is named for the Hope with Hazel Fund in honor of Hazel Hammersley who was diagnosed with Stage III neuroblastoma at age 2. She endured treatments, surgeries and two relapses with an amazing ability to push through and live life to its fullest. She loved her family and her happy place was with them. Through Hazel, her family learned that no one can fight pediatric cancer alone. This fund remembers her love of life and is dedicated to raising awareness and funds for research to protect kids with cancer who are still in the fight.

Today, over 80% of children diagnosed with cancer in high income countries like the United States will survive. Considered a miracle of modern science, these gains are unfortunately not reflective of outcomes for the 90% of children with cancer who live in low- and middle-income countries (LMIC). Yet, as many LMIC continue their epidemiological transition away from overwhelming infectious disease to a greater non-communicable disease burden, cancer care has recently become a major global focus. As policy-makers now begin the cancer control and prioritization planning process to meet this challenge, estimates of global and national cancer-related disease burden are a critical piece of data required when making decisions. However, current efforts meant to generate global pediatric cancer burden data such as incidence, mortality and long-term morbidity measures are not ideally suited for this purpose as they are optimized to measure adult cancer burden and do not incorporate key pediatric-specific data sources. Instead, a pediatric cancer specific analysis is needed since children are sufficiently different from adult cancers such that new methods, which account for small numbers of cases, the lack of etiologic risk factors, histology-based classification codes, and the long-term effects of treatment, are required. As the Friends for Hope Fund St. Baldrick's Scholar, Dr. Bhakta will address this critical gap by producing comprehensive pediatric cancer-related burden estimates using the most current data sources and rigorous statistical estimation methods. It is anticipated the results of this study (to be published and made publicly available) will provide global stakeholders and policymakers key outcomes data to cure more children with cancer globally.

This grant is named for the Friends for Hope Fund created to honor Morgan Loudon and celebrates her strength and determination as a cancer survivor. Diagnosed when she was 9 with a rhabdoid tumor, today she has no evidence of disease. Through this fund, Morgan's family and friends hope to 'battle on' in the search for cures and better treatments.

The lives of children surviving cancer are cut short by life-threatening infections. Most pathogens causing these infections come from the gut. Healthy children have good bacteria in their gut that protect them from pathogens. Chemotherapy and antibiotics harm good bacteria in the gut that protect children from pathogens. Without good bacteria, pathogens increase in the gut. These pathogens can get into the bloodstream and cause serious infections. Prior studies have repeatedly shown that replacing healthy stool with good bacteria in the gut is the best treatment and prevention of gut infections. Dr. Andersen's team is developing a new test that detects the good bacteria and pathogens in the gut using a stool sample. This new test will allow earlier identification of children with cancer at the greatest risk for a serious infection and the children most likely to benefit from replacing healthy stool with good bacteria back in their gut. They believe that replacing good bacteria in the gut can prevent serious infections from pathogens, including those with limited antibiotic treatment options. This new test will also identify the specific good bacteria in the gut that prevent infection for developing new probiotics to prevent serious infections in children with cancer.

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