Grants Search Results

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 in Memory of 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.

Based on progress to date, Dr. Almaliti was awarded a new grant in 2025 and 2026 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 and 2026 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.

Based on progress to date, Dr. Jones was awarded a new grant in 2023 to fund an additional year of this Scholar grant. Acute myeloid leukemia (AML) is a difficult to treat cancer that is associated with death in nearly 4 out of 10 children who are diagnosed with this disease. We know that there are multiple factors that contribute to poor outcomes in these patients, however, researchers don't fully understand all of them. Dr. Jones will gain a greater understanding of the resistance associated with a specific type of AML that is particularly difficult to treat. She hopes to gain clarity about this type of disease to find more specific therapies to target those resistance mechanisms in the cancer cells.

Neuroblastoma is one of the most common pediatric tumors, responsible for 12% of all cancer deaths in children under 15 years old. Only about 50% of patients with widespread neuroblastoma will live for ten years after diagnosis. A recent breakthrough in cancer treatments known as CAR T cell therapy reprograms a patient’s own immune cells to recognize tumor cells. While CAR T cell monotherapy works for some cancer types, several research studies using CAR T cells to treat neuroblastoma have been relatively unsuccessful. This is likely due to immune suppression caused by the tumor itself. Interestingly, it is known that if a person with cancer develops an infection, the infection can stimulate an immune response that will promote cancer remission. With this knowledge, Dr. Kudek and colleagues have pioneered an innovative technique to boost CAR T cell therapy response. They have shown that the cancer-destroying function of reprogrammed immune cells is boosted when a weakened infection is introduced into a tumor and found that this treatment combination in bladder cancer led to cure in most of the disease models. Encouraged by these findings, he is pursuing proof-of-principle studies to determine how this treatment approach can be best applied to neuroblastoma treatment.

This grant is named for the LukeStrong a Force Against Neuroblastoma Childhood Cancer Fund. When Luke was 5 years old, he was diagnosed with high-risk neuroblastoma. He is now in his teens and still in active treatment for relapsed neuroblastoma. Since 2014 Luke’s “Never tell me the odds” attitude has inspired his family and friends to shave their heads, fundraise with St. Baldrick’s, and help conquer childhood cancers.

Children with some kinds of blood cancers (leukemias) are not cured by regular chemotherapy and are at high risk of dying without better treatment options. Dr. Niswander is working to create new treatments that are more personalized for each child’s leukemia cells. The first treatment targets ‘miswired’ communication networks inside the leukemia cells that make them cancerous, and the second treatment uses the body’s own immune system to attack the leukemia cells. Each of these treatments is able to kill a patient’s cancer cells. But, eventually the leukemia cells develop changes that allow them to begin growing again despite the therapy, and the cancer comes back. These two therapies have never been combined together in patients. In this project, Dr. Niswander and colleagues are studying the best ways to combine these new treatments for two kinds of high-risk pediatric leukemias, since often two treatments that work in different ways are better than one. She is hopeful that by using patients’ own leukemia cells they will identify the best personalized treatments for future testing in pediatric patients to improve their chances of cure and living long and healthy lives.

For 2022, this grant is named for the Invictus Fund, a St. Baldrick’s Hero Fund created in memory of Holden Gilkinson. It honors Holden's unconquerable spirit in his battle with bilateral Wilms tumor by funding cures and treatments to mitigate side and late effects of childhood cancer.

In 2021, this grant was generously supported by Super Soph's Pediatric Cancer Research Fund, a St. Baldrick's Hero Fund. Sophie Rossi was diagnosed with AML at 3 months of age. Throughout her courageous battle, she was always smiling, always joyful. This fund was created to honor her spunky, sweet spirit by funding research to find cures for AML and all childhood cancers.

Diffuse intrinsic pontine glioma (DIPG) is a deadly pediatric brain cancer. Tragically, the majority of children diagnosed with the disease die within 12 months because the most effective treatment, radiation, is palliative at best. Therefore, there is a significant need to develop new therapeutic strategies to improve the terrible outcomes for these patients. Looking at genes that are turned on or off in a cancer can be helpful to figure out what is causing cancer growth. DIPGs are known to have mutations in a group of genes called histones that intriguingly regulate whether other genes in the cell are turned on or off. While looking at genes that are turned on or turned off in DIPG, Dr. Tsai found a gene called FOXR2 that is turned on in a subset of these tumors. FOXR is not usually present in the normal brain, but it has high levels in a subset of DIPGs. This is exciting because if researchers can target FOXR2 with new therapies, only tumor cells would be affected, sparing the normal cells in the brain. The goal of this project is to figure out exactly how FOXR2 makes DIPGs grow and to identify strategies that can be used in new treatments to target FOXR2.

A portion of this grant is generously supported by Griffin's Guardians, a St. Baldrick's partner. Griffin's Guardians was created by the Engles in memory of their son, Griffin. Their mission is to provide support and financial assistance to children battling cancer in Central New York, raise awareness about pediatric cancer and provide funding for research.

Based on progress to date, Dr. Moghimi was awarded a new grant in 2023, 2024, and 2025 to fund an additional year of this Scholar grant. In recent years, a very successful immunotherapy strategy to modify a patient's immune cells (called T-cells) to attack cancer has been developed for children with leukemia (cancer of blood cells). These modified immune cells are called Chimeric Antigen Receptor T cells (CAR-T cells). These CAR-T cells are very potent and do a better job than any chemotherapy at killing cancer. However, this life-saving tool has been available only to a small group of patients and for a handful of cancers. This is because most cancers don't have the targets those CAR-T cells aim for, or they have a target that can also be found on normal organs. As a result, these CAR-T cells could harm normal organs as collateral damage, a significant adverse effect of treatment that clinicians would want to avoid. In this proposal, Dr. Moghimi is striving to build the next generation of CAR-T cells that solely react to a combination of targets. These cells recognize a tumor only if they have both targets in sight and will not otherwise attack normal organs. CAR-T cells that operate based on a combination of two targets are more accurate than other targeting cells. Using this new generation of CAR-T cells, researchers would be able to significantly expand the availability of this powerful treatment to many more patients. Dr. Moghimi and colleagues will develop these special CAR-T cells for patients with Acute Myeloid Leukemia (AML), another common form of leukemia with a higher mortality rate for children. These results will provide pre-clinical evidence that could quickly translate to new clinical trials for children with relapsed AML through the Therapeutic Advances in Childhood Leukemia and Lymphoma (TACL) consortium, an international collaboration headquartered at CHLA.

In Africa, the majority of children who get cancer die from their disease. This happens in many cases because the patients do not get a correct diagnosis. Without a precise and correct diagnosis, these children cannot benefit from the newest curative treatments. To help prevent this from happening, Dr. Lutwama will develop and test a strategy to diagnose pediatric cancer correctly in a manner that is affordable, reliable, and within a shorter time frame in resource-limited settings.

In Africa, the majority of children who get cancer die from their disease. This happens in many cases because the patients do not get a correct diagnosis. Without a precise and correct diagnosis, these children cannot benefit from the newest curative treatments. To help prevent this from happening, Dr. Lutwama will develop and test a strategy to diagnose pediatric cancer correctly in a manner that is affordable, reliable, and within a shorter time frame in resource-limited settings.

Based on progress to date, Dr. Wu was awarded a new grant in 2023 and 2024 to fund an additional year of this Scholar grant. Diffuse midline glioma (DMG) is a fatal brain cancer in children and there are no effective treatments. The brain's natural barrier prevents drugs from reaching the tumor. Focused ultrasound (FUS) uses sound waves to temporarily open the blood brain barrier to increase drug delivery to the protected tumor cells in the brain. Dr. Wu will be using panobinostat, a promising drug tested in cancer cells in the laboratory to examine if FUS can increase its delivery and whether the addition of radiation can further improve the outcomes. This grant was awarded at Columbia University Medicine Center and transferred to Virginia Tech.

The 2023 and 2024 years of this grant is co-funded by the Focused Ultrasound Foundation.

The 2021 and 2022 years of this grant are 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.”

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.

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.

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. 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.

For a child is diagnosed with a diffuse intrinsic pontine glioma so called DIPG, the options for treatment are scarce and so are the chances for survival. This aggressive brain tumor generally strikes children who are 6 years old and younger, with most surviving less than a year after diagnosis. The only known effective treatment is the use of radiation. Yet, even with radiation therapy most children show tumor progression within the year after radiation therapy. Given this reality, there is a desperately need to identify the drug that increase the anti-tumor activity of radiation, as a mean to improve treatment outcome for these children. DNA damage is thought to be the most toxic effect caused by radiation, and Dr. Hashizume and others showed that the majority of the DNA damage caused by radiation are repaired within 24 hours of treatment. This DNA damage repair is possibly responsible for the tumor progression observed in DIPG after radiation therapy, thereby ultimately taking no survival benefits to the patients. As the recipient of the Just Do It...and be done with it St. Baldrick's Research Grant, Dr. Hashizume recently performed a genetic screening in DIPG cells collected from patient tumor and found specific therapeutic targets which is important for DNA damage repair. This research will study whether targeted inhibition of DNA damage repair increase DNA damage by radiation, leading to increased radiation toxicity in DIPG. Successful results from this research will find a new effective therapy which increases the anti-tumor activity of radiation, in turn, will ultimately leads to improved treatment outcomes for children with highly malignant and currently incurable cancer.

This grant is funded by and named for the “Just Do It…and be done with it” Hero Fund created in honor of Sara Martorano who was 4 when she was diagnosed with Stage IV Wilms tumor. Despite a grueling treatment protocol of surgeries, radiation and chemotherapy, Sara didn’t let anything dim her sparkle. Thanks to life-saving research, today she is cancer free. This fund celebrates the courage of all cancer kids enduring treatment and the support of their family and friends.

Awarded at Northwestern University and transferred to Ann & Robert H. Lurie Children's Hospital of Chicago.