Grants Search Results

There has been little recent progress in treating Ewing sarcoma, a pediatric tumor involving bone. Dr. Stegmaier and colleagues have used a technology called CRISPR to identify urgently needed, new therapeutic targets for this disease. They prioritized a class of targets which are expressed in immature but not mature tissues. These proteins are often abnormally re-expressed in cancers such as Ewing sarcoma. Thus, drugs targeting these proteins would be expected to have minimal toxicity. The Stegmaier lab identified the target IGF2BP1 as a top selective gene dependency in Ewing sarcoma; deletion of IGF2BP1 was more deleterious to Ewing sarcoma than all other cancer types screened. Importantly, IGF2BP1 is not expressed in most normal human cells. Dr. Stegmaier will validate IGF2BP1 as a therapeutic target in Ewing and will determine the mechanisms by which Ewing sarcoma cells rely on IGF2BP1 for growth. With IGF2BP1 chemical inhibitors in development, this project has exciting translational potential for patients with Ewing sarcoma.

This grant is funded by and named for The Ben Brandenburg Fund for Ewing Sarcoma Research. Ben passed away at the age of 15. He is remembered for his quick wit, indomitable spirit and bravery. This fund is his lasting legacy and ensures that research is funded so fewer children will have to suffer from Ewing Sarcoma.

Adoptive immunotherapy has demonstrated unprecedented success in the treatment of pediatric leukemia. Extending its therapeutic potential to other pediatric malignancies such as glioblastoma (GBM) has proved challenging. In this therapy, T cells are isolated from a patient, expanded outside of the body, and genetically modified prior to reinfusion. The ability of these T cells to recognize and eliminate cancer cells is improved by expressing a protein (CAR) on the T cell surface. An important challenge is to minimize the manipulation of patients' T cells outside the body. Prolonged culture compromises their efficacy. Dr. Ghassemi developed approaches to generate CAR T cells in 1 day. These cells have increased potency. She is combining this recent development with a metabolic strategy to overcome the metabolic nature of tumor environment. This synthetic advancement combined with the production of CAR T cells in 1 day will lead to superior CAR T cells for cellular immunotherapies against pediatric GBM.

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”.

Philadelphia chromosome-like acute lymphoblastic leukemia (Ph-like ALL) is a high-risk type of B-ALL. Children and adolescents/young adults with Ph-like ALL have greater than 60% relapse risk and experience significant mortality with best-available chemotherapy. Emerging data suggest that patients with Ph-like ALL are more likely to develop immunotherapeutic resistance to treatments. Dr. Tasian, and Dr. Terry Fry at the University of Colorado, and their colleagues are testing whether a new CAR T cell immunotherapy that they developed will decrease the risk of relapse for patients with Ph-like ALL. This work will be the groundwork to translate findings into a phase 1 clinical trial. This grant is supported by The Ty Louis Campbell (TLC) Foundation. TLC was created in memory of Ty, who lost his 2-year battle with brain cancer just days after his fifth birthday. TLC funds innovative research and clinical trials specifically geared toward the treatment of the deadliest childhood cancers (including brain and spinal cord tumors). TLC seeks less toxic, more effective treatments that are specifically designed for children fighting cancer. Their ultimate mission is to help fund the intelligence and technology that will uncover new ways to cure children with cancer.

Based on the progress to date, Dr. Rahnama was awarded a new grant in 2024 to fund an additional year of this Fellow grant.

Acute Myeloid Leukemia (AML) is a blood cancer that affects children. While there have been important advances in treatment and care of pediatric patients with AML, 20-40% relapse and have poor outcomes. Novel therapies are urgently needed to combat this disease. One treatment modality under investigation involves manipulation of the body's immune system by reprogramming immune cells with inherent anti-leukemia properties to specifically target AML cancer cells. Dr. Rahnama is focused on the study of natural killer (NK) cells as immune cells of interest. NK cells can be engineered to express Chimeric Antigen Receptors (CARs) that recognize specific proteins on leukemia cells in order to attack and kill them. The site where the CAR-modified NK cell and the target leukemia cell come together is known as the immunological synapse (IS). The IS is highly organized and plays a key role in activating the NK cell. Dr. Rahnama aims to better understand the interaction between CAR-modified NK cells and target leukemia cells by studying the biology of the IS as related to how tightly the two cells interact. Her goal is to improve CAR-NK cell design for ultimate use as pediatric AML treatment. This grant was awarded at Johns Hopkins University School of Medicine and transferred to the University of California, San Francisco.

This grant is funded by and named for the Aiden's Army Fund. When he was 8 years old, Aiden Binkley was diagnosed with Stage IV rhabdomyosarcoma. He had a huge tumor in his pelvis and the cancer had metastasized to his lungs. But this bright, funny and courageous boy believed he got cancer so he could grow up to find a cure for it. Aiden’s story has inspired so many people and his vision to cure cancer is being carried on by Aiden’s Army through the funding of research. They will march until there is a cure!

Based on the progress to date, Dr. Young was awarded a new grant in 2024 to fund an additional year of this Fellow grant.

Osteosarcoma is a bone tumor that usually occurs in children and young adults and can be deadly especially when the tumor spreads to other body parts. The treatment strategy for this disease has not seen significant improvement in over 30 years, and there is no specific treatment for tumors that have spread throughout the body. In this project, the major goal is to identify factors that control the spread of osteosarcoma in order to develop new therapies to extend the lives of patients. Currently, Dr. Young is investigating whether osteosarcoma cells block the activation of one part of the patient's immune system, protecting the cancer cells from an immune attack and allowing them to spread throughout the body. This work has the potential to uncover new treatments to harness the immune system to fight this devastating disease.

This grant is named for the Team Jackson Hero Fund. The fund was established in honor Jackson Schmitt who died six days after his diagnosis with osteosarcoma from a stroke. Jackson’s story was told worldwide and his legacy lives on through funding life-saving osteosarcoma research.

Based on progress to date, Dr. Faulk was awarded a new grant in 2024 and 2025 to fund an additional year of this Scholar grant. Infant leukemia is an aggressive cancer with a very poor prognosis. The leukemia cells in most of these patients have a genetic change in which a gene (KMT2A) is broken and combined with other genes that typically do not interact with one another (this is called "rearranged"). A drug named SNDX-5613 has been developed that directly targets the changes that occur in a cell with a KMT2A rearrangement to specifically kill these leukemia cells, and it has shown promise in treating adult leukemia patients with a KMT2A rearrangement. An upcoming clinical trial will combine SNDX-5613 with traditional chemotherapy for children with leukemia with a KMT2A rearrangement that has come back (relapsed) or proven resistant to typical treatment (refractory). In addition to testing the safety and efficacy of SNDX-5613, studies will be done on patients’ blood and bone marrow samples to better understand how the drug functions in fighting leukemia. This trial represents the next step in evaluating this promising new targeted drug for these deserving patients, and the associated studies are key to helping us understand more about the biology of this leukemia and how to best target it.

This grant is named for the Oh Danny Boy, I Love You So: The Danny O'Brien Rhabdoid Tumor Research Fund. Danny O’Brien was just 5 months old when he was diagnosed with a malignant rhabdoid tumor on his liver. This cancer is extremely rare and aggressive. He endured chemotherapy to shrink the tumor for surgery, but the treatment was not effective. At the tender age of 9 months, Danny passed away. Fortunately, he knew nothing but love and affection all of his short life. This fund honors Danny’s courage and his unconditional love even in the midst of his battle with cancer.

Brain and spine tumors are the leading cause of cancer-related death in children and adolescents. While cure can sometimes be achieved with conventional chemotherapy, surgery, and/or radiation, prognosis is dismal for patients whose aggressive brain/spine tumors progress despite these treatments. There is a critical need to develop new effective, well-tolerated therapies for children, adolescents, and young adults with refractory high-grade brain/spine tumors. Lutathera is a targeted radiotherapy which binds to tumor cells that express somatostatin receptors, causing tumor cell death through localized release of radiation, with minimal side effects. Many pediatric and young adult high-grade brain/spine tumors express somatostatin receptors, making them ideal targets for this therapy. Dr. Lazow is conducting a clinical trial to assess the safety and effectiveness of Lutathera in children and young adults with recurrent high-grade brain/spine tumors. Within this trial, she will also 1) evaluate how somatostatin receptor expression varies across different brain/spine tumors and determine clinical, imaging, pathology, and genetic characteristics which correlate with that expression, 2) identify imaging and molecular biomarkers predictive of response to Lutathera and/or disease recurrence, and 3) perform radiation dosimetry to establish optimal dosing of Lutathera in children and young adults, ensuring adequate tumor penetration while minimizing toxicity. If Lutathera proves safe and effective in treating children and young adults with refractory brain tumors, further studies will be planned to expand to a larger patient population and eventually incorporate Lutathera into upfront treatment backbones for these aggressive diseases.

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.

High-risk medulloblastoma is a devastating childhood brain cancer that results in death in nearly 50% of patients. To improve future treatments for this disease, Dr. Prensner is studying a category of newly-discovered "dark proteins", which have been excluded from prior work due to their small size and unconventional locations in the human genome. He has found that a group of these dark proteins are critical for medulloblastoma cells to survive. This research will reveal how these dark proteins may point toward new approaches to treat medulloblastoma, which may be critical to define the next generation of anti-cancer therapies in this disease. This grant was awarded at Dana Farber Cancer Institute and transferred to the University of Michigan.

Dr. Sykes is developing new therapies for childhood leukemia and lymphoma. Specifically, he is looking at a type of leukemia that develops from abnormal T-cells and is named acute lymphoblastic leukemia (T-ALL). T ALL is a particularly deadly disease if it does not respond to therapy (refractory) or if it responds initially and then comes back (relapsed). When a normal T cell becomes a leukemia cell, it develops certain advantages and certain disadvantages. Therefore, one way to kill a leukemia cell is to identify these disadvantages and to exploit those using specific drugs. This research focuses on how leukemia cells make DNA and RNA building blocks called nucleotides. An enzyme called DHODH is essential to the process of making nucleotides within the leukemia cell. Drugs that inhibit this enzyme rapidly kill the leukemia cells and spare the life of normal cells. Researchers call this approach 'nucleotide starvation' because it starves the leukemia cells of these DNA and RNA building blocks. Normal cells have back-up systems to deal with periods of nucleotide starvation. Dr. Sykes believes that leukemia cells have lost these back-up systems and that is why they are so sensitive to starvation. So far his research has shown that this nucleotide starvation approach works extremely well in leukemia cells outside of the body and in leukemia cells in laboratory mouse leukemia models. The fact that many DHODH inhibitor drugs are already available and have already been tested in humans suggests that clinical trials are feasible and could begin in a timely manner. Dr. Sykes hopes that DHODH inhibitor therapy will be effective treatment for children with T ALL, especially those children who have run out of other good treatment options.

Limited progress has been made over the last 30 years against kid brain tumors, especially those in the thalamus and the pons (Diffuse Intrinsic Pontine Glioma, DIPG), a specific location in the brain. Radiotherapy (RT) is the only treatment available that can prolong the life of children with the most aggressive form of brain tumors. Recently, RT is recognized to activate the immune system against multiple tumors. However irradiated kid brain cancers always regrow which suggest that RT is not activating immunity against these tumors. Understanding why this phenomenon is happening is critical to develop strategies that will exploit the immune stimulation from RT to control and cure brain cancer. The activation of cancer-associated fibroblasts (CAFs) by RT can be responsible for treatment resistance and the lack of immune stimulation of kids brain cancers. Dr. Vanpouille-Box's initial results show that stopping the immunosuppression of CAFs with a fibroblast activating protein alpha (FAP) blocker re-activates the immune system against irradiated pediatric brain tumors. Thus, blocking CAF emerges as a novel approach to prevent brain cancer regrow and to activate immunity in irradiated brain cancer. She proposes to: 1) Define the role of CAF in mice models of pediatric brain cancer 2) Determine the efficacy of CAF and EGFR blockade in irradiated pediatric brain cancer. Dr. Vanpouille-Box and colleagues hope to find that: - CAF stop the immune stimulation of irradiated pediatric brain tumors - blocking CAF immunosuppression works well to reactivate immunity against irradiated brain cancer, especially on the context of epidermal growth factor receptor therapy.

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.

Based on progress to date, Dr. Yang was awarded a new grant in 2024 and 2025 to fund an additional year of this Scholar grant. Hepatoblastoma is a very rare liver tumor diagnosed mainly among children younger than five years of age. Since it is hard to collect enough cases to study, researchers have not fully evaluated germline risk factor, i.e., the genetic information inherited from parents. Dr. Yang and colleagues have generated the largest germline genetic dataset for hepatoblastoma in the world, with which they can study the genetic causes of both onset and survival. They aim to better understand these genetic mechanisms to facilitate early detection and possibly identify targets of therapy for hepatoblastoma.

This grant is named for Julia's Legacy of Hope, a Hero Fund that honors Julia's positive, courageous spirit and carries out her last wish: "no child should have to go through what I have experienced". Diagnosed at 16 with Ewing sarcoma, Julia fought cancer and survived only to be stricken by a secondary cancer as a result of treatment. Her family is raising awareness and funds for research for Ewing sarcoma, as well as issues impacting Adolescent and Young Adult (AYA) patients.

One of the most exciting recent developments in cancer treatment is the growing ability to use the body's own immune system to directly fight tumors. However, these treatments still do not work on most patients, and we think it is critical to understand how each cancer type avoids the immune system. Dr. Schwartz is investigating how neuroblastoma, one of the most common pediatric solid tumors, escapes destruction by the immune system. To accomplish these goals, he will use cutting-edge technologies to dissect the immune biology in a model of neuroblastoma, with a particular focus on studying an important type of cancer-killing cell called a 'CD8 T cell'. Dr. Schwartz thinks him and his colleagues have identified an important new way that neuroblastoma evades these T cells. Their preliminary results suggest that neuroblastoma directly causes T cell death, limiting the ability of T cells to survive and kill enough tumor cells. He is trying to learn how neuroblastoma causes the death of T cells and find ways to block this immune evasion strategy. Most importantly, he predicts that combination treatment designed to block neuroblastoma's ability to kill T cells along with existing immune therapies will drastically improve the ability of the immune system to eradicate 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.

Based on progress to date, Dr. Almaliti was awarded a new grant in 2025 to fund an additional year of this International Scholar grant. There is no nice way to tell someone they've got a brain tumor, and with a child it's unimaginable. In fact, brain tumors are the leading cause of solid tumor cancer death in children. Proteasome inhibitors are a recently discovered drug class that is effective in many types of cancer and have reduced side effects to normal cells. Dr. Almaliti aims to develop novel potent and selective proteasome inhibitors that will specifically kill brain cancer in children. This innovative approach should result in the discovery of new clinical leads for treating brain cancers in children.

This grant is funded by and named for Luke's Army Pediatric Cancer Research Fund. This Hero Fund was created in memory of Luke Ungerer who brought smiles and sunshine wherever he went with plenty to share with everyone. He battled a brain tumor with a positive spirit and inspired others with his courage in his short life. This fund intends to carry on Luke’s legacy of positivity with the hope that it will ripple across many lives for many years to come.

Cancer research allows scientists to modify specific immune cells to recognize and kill cancer. One type of immune cell is called the cytotoxic killer T cell. This T cell has a receptor (TCR) that is used to recognize a structure on the cancer cell's surface called a peptide-major histocompatibility molecules complex I (pMHC I). pMHC I complexes are diverse and are rarely shared amongst patients. This diversity prevents the use of a classic TCR across multiple patients to avoid tissue injury that known as graft versus host disease (GVHD). To bypass these limitations, Dr. Elsabbagh propose to develop T cells expressing a TCR that can target a protein called CD1d. Unlike MHC I, CD1d is not diverse and is well expressed on various childhood cancers including acute myeloid leukemia (AML), which has been known for high rates of treatment-related toxicity and disease recurrence. These modified cells will be pre-made and used universally in any AML or other children’s cancers that expresses CD1d. Dr. Elsabbagh and team will also attach a recent discovered enhancing protein called MyD88 to the created receptor to enhance their anticancer activity. They expect these modified T cells will be able to recognize and kill children AML cells.

Dr. Nguyen uses the patient's own immune T cells and armors them in the laboratory with a chimeric antigen receptor (CAR) to recognize neuroblastoma cells and kill them. Although she has demonstrated a robust anti-tumor effect of CAR T cells in models of neuroblastoma, she noticed that they can be overwhelmed by too many tumor cells and work less effectively. However, the CAR T cell function was restored when the cells were engineered to express tethered IL15 and -21 on their surface. Though these cytokines activated the CAR T cells and improved their function, models also showed signs of toxicity, which she hypothesizes to be caused by cytokine-driven CAR T cells. Therefore, this project will describe the manifestation and understand the cause of CAR T cell-associated toxicities in our neuroblastoma model. Furthermore, Dr. Nguyen and colleagues propose to reduce the side effects by engineering tethered cytokines that are predominantly expressed in the vicinity of tumor cells (conditional expression). The completion of this project will forge a new direction for the use of cytokines in CAR T cell therapy and render a new fourth-generation CAR T cell therapy safer for translation into the clinic. This honor award is without funding.

Based on progress to date, Dr. Almaliti was awarded a new grant in 2025 to fund an additional year of this International Scholar grant. There is no nice way to tell someone they've got a brain tumor, and with a child it's unimaginable. In fact, brain tumors are the leading cause of solid tumor cancer death in children. Proteasome inhibitors are a recently discovered drug class that is effective in many types of cancer and have reduced side effects to normal cells. Dr. Almaliti aims to develop novel potent and selective proteasome inhibitors that will specifically kill brain cancer in children. This innovative approach should result in the discovery of new clinical leads for treating brain cancers in children.

This grant is funded by and named for Luke's Army Pediatric Cancer Research Fund. This Hero Fund was created in memory of Luke Ungerer who brought smiles and sunshine wherever he went with plenty to share with everyone. He battled a brain tumor with a positive spirit and inspired others with his courage in his short life. This fund intends to carry on Luke’s legacy of positivity with the hope that it will ripple across many lives for many years to come.

Cancer research allows scientists to modify specific immune cells to recognize and kill cancer. One type of immune cell is called the cytotoxic killer T cell. This T cell has a receptor (TCR) that is used to recognize a structure on the cancer cell's surface called a peptide-major histocompatibility molecules complex I (pMHC I). pMHC I complexes are diverse and are rarely shared amongst patients. This diversity prevents the use of a classic TCR across multiple patients to avoid tissue injury that known as graft versus host disease (GVHD). To bypass these limitations, Dr. Elsabbagh propose to develop T cells expressing a TCR that can target a protein called CD1d. Unlike MHC I, CD1d is not diverse and is well expressed on various childhood cancers including acute myeloid leukemia (AML), which has been known for high rates of treatment-related toxicity and disease recurrence. These modified cells will be pre-made and used universally in any AML or other children’s cancers that expresses CD1d. Dr. Elsabbagh and team will also attach a recent discovered enhancing protein called MyD88 to the created receptor to enhance their anticancer activity. They expect these modified T cells will be able to recognize and kill children AML cells.

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 2022 SBF-AACR Award for Outstanding Achievement in Pediatric Cancer Research went to Dr. David Malkin at The Hospital for Sick Children (SickKids). Dr. Alanna Church at Boston Children's Hospital received the 2022 research grant. Dr. Church's research interests are in bringing molecular testing to the clinical care of children with cancer to improve diagnoses and treatments.

Although Hodgkin Lymphoma (HL) is largely curable, 10-20% of patients are resistant to treatment and difficult to cure. When a patient’s cancer comes back or does not respond to chemotherapy, it is often because the immune cells have become exhausted and unable to recognize the cancer cells in the body. The first goal of Dr. Bollard’s project is to determine if newer immunotherapy drugs called PD1 inhibitors, when given in combination with the administration of a novel cancer killing T-cell therapy, will produce long-lasting cures in patients with high-risk lymphoma with less side effects than conventional chemotherapy. Dr. Bollard and colleagues will take a patient’s immune cells and re-educate them in the laboratory to recognize antigens on the cancer cells and then give the T cells back to the patients to redirect them to the cancer cell. The second goal of this project is to genetically modify the cancer killing T cells and express a chimeric antigen receptor (CAR), which will enable the T cells to recognize a protein found in HL and to destroy the cancer cells more effectively. Dr. Bollard’s team proposes a novel approach of combining CAR technology with a tumor antigen specific T cells into a single “living drug” that will produce a robust response and provide a long-lasting immunity using the patient’s own immune system. This therapy has the potential to benefit not only children with HL, but other solid cancers such as neuroblastoma and brain tumors, where the environment surrounding the cancer cells make it difficult for T cells to infiltrate and kill. This grant is funded through a partnership between the St. Baldrick’s Foundation and the American Cancer Society.

More children die from brain tumors than any other type of cancer. Pediatric high-grade gliomas are the type of childhood brain tumor that is the hardest to treat and the most likely to result in death. Researchers have learned that pediatric high-grade glioma is actually several different types of tumors that are driven to grow by different genetic changes in the tumor cells. Almost all clinical trials have shown that chemotherapy doesn’t cure more kids and just leads to more side effects, however a clinical trial completed almost 10 years ago, used two chemotherapy medicines, in addition to surgery and radiation, and cured significantly more patients than previous therapy combinations. Dr. Green will use modern pathology tests to figure out the pediatric high-grade glioma subtype for all of the patients from that previous trial. Dr. Green and colleagues will look at the survival on each trial by subgroup to know which subgroup(s) showed better survival with the addition of one of the chemotherapy medicines to their treatment. Answering this crucial question will change the future of pediatric high-grade glioma treatment. With these results, current pediatric high-grade glioma patients will be able to be given the right standard treatment to maximize their chance of survival and minimize side effects. This grant is funded through a partnership between the St. Baldrick’s Foundation and the American Cancer Society.