Grants Search Results

Medulloblastoma is the most common malignant brain tumor in children. While multimodal therapy for medulloblastoma at diagnosis has resulted in improved outcomes, there are very few options at the time of relapse, with overall poor survival. Preclinical data have shown anti-tumor activity with liothyronine (T3, activated thyroid hormone) in medulloblastoma models. T3 has safety data and dosing which are available for children. Dr. Ronsley and colleagues will translate these preclinical findings into a clinical trial, which will treat children and adolescents with relapsed medulloblastoma with T3 in combination with chemotherapy and evaluate both safety and efficacy and the role for monitoring with liquid biopsy.

This grant is named for Hannah’s Heroes, a Hero Fund established to honor Hannah Meeson. At age 6 she was diagnosed with anaplastic medulloblastoma. After a relapse and additional 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.”

Bone marrow transplantation (BMT) is an effective treatment for patients with blood cancers, utilizing healthy donor cells to fight diseases. However, these donor cells contain specialized immune cells (T cells) that can also attack and damage the patient's healthy organs, known as graft-versus-host disease (GvHD). Because both GvHD and anti-cancer effect (GvL) are mediated by the same T cells, it is hard to separate GvHD from the beneficial GvL. Current treatments use global immune-suppressive drugs to prevent GvHD, but they also reduce the ability of T cells to fight cancer, increasing the risk of cancer recurrence. Dr. Kim and colleagues will investigate if targeting VLDLR-PPARd signaling selectively eliminates GvHD without compromising the GvL in preclinical models of BMT.

High-grade glioma (HGG) is an aggressive brain tumor treated with surgery and radiation/chemotherapy. With such aggressive treatment, most children are not cured. Infant-Type Hemispheric Glioma (IHG) has a better cure rate than other types of HGGs. Commonly diagnosed in patients younger than 1, IHGs are large tumors and occupy half of a baby's brain. Surgery is often complicated with life-threatening bleeding in the brain with severe adverse effects further compounded by chemotherapy. Therefore, though IHGs have favorable cure rates, they require more effective and less toxic therapy. IHGs have genetic defects called receptor tyrosine kinase fusion, which are targetable by medicines called tyrosine kinase inhibitors (TKI), commonly used in adults. When used in IHG, tumor size is reduced without surgery or chemotherapy. Dr. Bagchi's clinical trial will treat based on the tumor's genetic defects using TKI and integrate quality of life measures so children survive & thrive.

Osteosarcoma is a bone cancer that affects children and adolescents. Unfortunately, its treatment has remained the same since the 1980's, and once the cancer spreads to other organs, less than 40% of the patients survive despite treatment. Dr. Aquino Lopez and colleagues will use the immune system to eliminate tumor cells. During an illness with a virus, immune cells called virus specific T cells (VSTs) eliminate infected cells by recognizing "viral signals". Dr. Aquino Lopez will use an artificial virus combination called CAdVEC to modify cancer cells and make them look like they are infected with a virus. Doing so, will trigger immune cells to eliminate the cancer cells.

Ewing sarcoma is an aggressive bone tumor in children, adolescents, and young adults. New therapies with greater efficacy and less toxicity are urgently needed to save the lives of these young patients. Dr. Theisen and colleagues will identify a new vulnerability in the mitochondria (i.e. the powerhouse) of Ewing sarcoma cells as well as several possible drugs that target this pathway. Dr. Theisen and team will determine both the reason that Ewing sarcoma cells have this unique vulnerability and how best to target this pathway therapeutically. In the long term, this will lead to better ways to treat Ewing sarcoma.

This grant is named for Julia's Legacy of Hope, a Hero Fund that honors Julia's positive and courageous spirit and carries out her last wish: "no child should have to go through what I have experienced". Diagnosed at age 16 with Ewing sarcoma, Julia fought cancer and survived only to be stricken in college with acute myeloid leukemia, a secondary cancer as a result of treatment. Through this Hero Fund, her family hopes to raise awareness and funds for childhood cancer research especially for Adolescent and Young Adult (AYA) patients.

Brain tumors are the deadliest type of cancer that afflicts children. The ability of brain tumor cells to spread (metastasize) outside of the original tumor along the leptomeninges, the covering of the brain and spinal cord, is responsible for making many brain tumors so hard to treat. How cancer cells embedded in the leptomeninges survive, thrive, and resist best treatments is poorly understood. A better understanding of leptomeningeal metastasis is required to make new therapies that can meaningfully increase survival for children diagnosed with aggressive brain cancers. Dr. Reinecke and colleagues will create a way that can identify and screen potential therapies in a cell culture dish, thereby streamlining interventions they take to models of pediatric brain tumors. Dr. Reinecke and colleagues believe that establishing this preclinical platform has the potential to identify therapies that have a chance to positively impact the lives of children diagnosed with metastatic brain tumors.

Vincristine is a chemotherapy drug commonly used to treat cancer in children and young adults, but it can cause vincristine-induced peripheral neuropathy (VIPN), a side effect that leads to numbness, pain, weakness, and difficulty with balance. These symptoms can severely impact daily life and may require chemotherapy dose reductions or discontinuation, potentially affecting cancer treatment outcomes. Currently, doctors use a grading system to assess VIPN severity, but this method is not sensitive enough to detect early symptoms and is difficult to implement consistently. Therefore, there is a need for a more reliable and accessible way to identify VIPN early. Dr. Belsky will utilize a potential solution of a blood test to measure neurofilament light chain (NfL), a substance released during nerve damage. Dr. Belsky and colleagues will explore whether NfL levels can detect VIPN in children and young adults to improve the ability to monitor nerve damage, enabling doctors to adjust treatments earlier, optimizing cancer care.

The most common source of pain when treating children is needles. Avoiding painful procedures is a cause of stopping treatment for children with cancer in Low and Middle-Income Countries (LMICs). Dr. McNeil and colleagues will utilize an evidence-based care bundle to reduce needle pain based off of past results used in hospitals with LMICs with high patient and parent satisfaction which reduced pain by a significant amount. Keeping in mind the large differences in resources and cultures between the different hospitals, Dr. McNeil and colleagues will be able to understand the key features of using the care bundle in different hospitals. Dr. McNeil's team will identify different adaptations each hospital uses and study the ability of a hospital to continue to implement care.

Neuroblastoma (NB) is a deadly childhood cancer. Children with NB are classified as high-risk (HR) due to genetics or metastatic disease. Metastatic disease occurs when tumor cells (TC) leave the original tumor and travel to distant organs and develop into new tumors by a complex set of steps. Before tumor cells can form metastasis, they often go through a long period of dormancy (rest) to evade therapy. In NB, the bone marrow is the most common site of metastasis and relapse. The MYCN_TT is a unique model of NB, that spontaneously metastasizes to distant sites such as kidney marrow (equivalent to bone marrow). Dr. Anderson and colleagues will utilize the MYCN_TT model in understanding the molecular and cellular mechanisms underlying metastasis and dormancy, which will inform the development of novel therapeutic strategies for HR-NB.

One in three children with newly diagnosed cancer has unmet resource needs such as food, housing, transportation, or utilities. These social needs increase during cancer care and are linked to worse outcomes for children and their parents. Government benefits such as the Supplemental Nutritional Assistance Program (SNAP or food stamps) improve child and maternal health outcomes. Dr. Umaretiya will conduct a pilot study using ASSIST, (a benefits navigator intervention to help families enroll and stay on government benefits such as SNAP) among 40 families of children with newly diagnosed cancer and test whether it is feasible and acceptable to families, allowing to identify barriers and facilitators to use ASSIST intervention.

Ewing sarcoma is a devastating childhood bone cancer. Doctors treat these pediatric patients with toxic chemotherapies, radiation, and surgery. Dr. Langdon and colleagues will develop targeted combination therapies to safely and effectively kill Ewing sarcoma cells. Dr. Langdon finds changing where proteins are normally found in cancer cells create potential new targetable vulnerabilities for Ewing sarcomas. Each vulnerability is thought of as a new "Achilles heel" for these cancers. Dr. Langdon and colleagues believe that combining two drugs - one which changes where proteins are normally found and one which targets the new vulnerability - will kill Ewing sarcomas. Dr. Langdon's team will look to why these drugs work so well against Ewing sarcomas and determine if they are truly safe and effective.

Acute kidney injury (AKI) commonly occurs during therapy for acute lymphoblastic leukemia (ALL). Small studies in pediatric ALL have suggested that AKI increases the chance of dying or the treatment not working. AKI may also lead to permanent chronic kidney disease (CKD) in survivors. This has never been investigated in a large population of children with ALL. This project will use data from the multicenter Leukemia Electronic Abstraction of Records Network to investigate how different types of AKI impact survival from ALL and the development of CKD. Dr. Bravo and colleagues will enroll children who have completed ALL therapy into a study to assess markers of kidney function over the subsequent year. This study will be critical to inform recommendations for how doctors screen for kidney-related problems in childhood ALL survivors. This is only a first step; once completed, Dr. Bravo and team can then expand these efforts to understand kidney damage from treatments for other types of cancer too.

This grant is named for To-morrow's Research Fund, a Hero Fund created to honor Becky Morrow who is a childhood cancer survivor. Becky was diagnosed with acute lymphoblastic leukemia when she was 12 and endured grueling treatments and its side effects. Today she is cancer free, a wife and a mom but suffers late effects. This fund supports survivorship research for safer treatments that help kids not only survive but thrive.

Allogeneic cell therapy is a new approach to cancer treatment that harnesses living cells from healthy donors to fight tumors. To do so, immune cells are isolated from blood and incubated outside the body to expand subsets capable of killing cancer. Dr. Jonus and colleagues have shown that gamma delta (gd) T cells expanded from healthy adults help to eradicate neuroblastoma grown in models. Based on this,Dr. Jonus and team are performing a first-in-child clinical trial of gd T cells for patients with neuroblastoma. Going forward, Dr. Jonus's findings show an opportunity to make gd T cell therapy more effective by expanding a new type of gd T cell, Vd1, with unique properties that should improve both the cell therapy's fitness and its ability to infiltrate into solid tumors. In parallel for a potent second-generation therapy, Dr. Jonus will engineer Vd1 gd T cells to 1) express receptors that help them better recognize neuroblastoma and 2) evade immune recognition so that the therapy is not killed after being infused into a patient.

The first year 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.

Diffuse midline glioma (DMG), previously known as diffuse intrinsic pontine glioma (DIPG), is a deadly childhood tumor with no effective treatments. Dr. Li's project seeks to understand the genetic and epigenetic dysregulation of DMGs. Through cutting-edge single-cell analyses and advanced AI models, researchers aim to map the tumor's epigenetic landscape, identify key regulatory elements, and predict the function of risk mutations. This knowledge could pave the way for new targeted therapies and improve DMG outcomes.

This grant is funded by and named for #Joe Strong 71, a St. Baldrick’s Hero Fund created in memory of Joe Purdue. Joe was a talented football player and cherished friend and son. He was diagnosed with DIPG shortly after graduating from high school, cutting short his plans to attend college. He is remembered for determination as he battled the most lethal form of brain cancer. #Joe Strong 71 carries on Joe's legacy by funding research for DIPG.

Children everywhere in the world get cancer but their chances of surviving differ based on where they live. Disparities in childhood cancer diagnoses and survival have been described by sex and age, but there are gaps in this literature from countries with limited resources. The first goal of Dr. Force's project is to analyze how childhood cancer diagnoses and survival differ by sex, age, and world region, using data from the most comprehensive international collection of hospital cancer registries, and to assess potential underlying drivers of these disparities, which would be beneficial in identifying interventions to improve equity in childhood cancer outcomes. The second goal of Dr. Force's project is to compare childhood cancer data from hospitals and population-based cancer registries, to determine whether hospital data could be used to supplement information on childhood cancer burden where data is currently lacking in global models, better illuminating the disparities that exist globally.

Acute lymphoblastic leukemia (ALL) is the most common type of childhood cancer with more than 3000 children/adolescents under the age of 20 diagnosed with ALL each year in USA. ALL affects a type of white blood cells called lymphocytes that help the body fight infection and disease. ALL can be broadly divided into either B-ALL or T-ALL. B-ALL affects a type of lymphocytes called B-lymphocytes whereas T-ALL affects T lymphocytes. Historically children with T-ALL have worse prognosis than B-ALL. B-ALL also have better therapeutic options whereas children with T-ALL are limited to therapies with well documented long-term negative effects like chemotherapy, radiation therapy. In this proposal, Dr. Thansapani and colleagues aim to evaluate a new therapeutic approach of nutrient deprivation to treat T-ALL grounded on their strong preliminary finding that T-ALL cells need high levels of the nutrient valine for their growth and survival. Dr. Thandapani's project investigates different avenues exploiting this vulnerability.

Whilst it is well known that damage to our DNA can cause cancer, it is still not fully understood what causes such DNA damage in many childhood cancers. Dr. Wang and colleagues recently made a breakthrough by discovering that our own body produces a natural toxin called formaldehyde that causes DNA damage and an aggressive blood cancer in children. This was a shocking discovery as it had previously been thought that formaldehyde mainly came from industrial chemicals found in factories. Dr. Wang's overall aim in this research proposal is to unravel exactly where formaldehyde toxins are made in our body. This knowledge can help to identify children at risk of developing blood cancers, and to develop strategies to modulate the production of formaldehyde as novel therapies against blood cancers.

The first year of this grant is 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.

Acute myeloid leukemia (AML) is the second most common type of leukemia in children. Despite treatment advancements, over 30% of children with AML cannot be cured. In AML cell populations, the leukemia stem cells (LSCs) make up a small part of the total, but are specially important: they provide a steady supply of new AML cells and are unfortunately very resistant to killing with drugs. Dr. Sui and colleagues believe that if they are able to kill the LSCs, they could cure patients with AML. Dr. Su has found that an enzyme called METTL1 is important in allowing LSCs to safely stay anchored in the bone marrow and identified a drug that inhibits METTL1 and eliminates LSCs. Dr. Su's study explores why METTL1 is important for LSCs and investigates how Dr. Su and team could best use their in-house developed METTL1 inhibitor to treat childhood AML using model systems. If successful, this research could pave the way for a clinical trial, offering hope for improved outcomes for childhood AML patients.

This grant is generously supported by Double Deckers Destroy AML, a St. Baldrick's Hero Fund. Joel and Seth were not only identical twins but best friends. In an ironic twist of fate, both boys were diagnosed with Acute Myeloid Leukemia just three months apart. With the overlapping diagnoses and treatments, the family was separated for months at a time and looked forward to days when they could be together at home. Joel and Seth both received bone marrow transplants and endured complications from the procedures. Sadly, both boys relapsed. Surrounded by their loving family, Joel died in November 2017 at the age of three, followed by Seth in May, 2019 when he was four years old. The twins were named as 2020 Ambassadors for St. Baldrick's so their story can continue to inspire many. The Double Deckers Destroy AML Hero Fund was established because the Decker family strongly believes more research is needed for AML, especially when the disease has relapsed. They want to support research so other families won’t have to say goodbye too soon.

Acute myeloid leukemia (AML) is a blood cancer that affects children. AML treatment involves intensive chemotherapy that requires over 140 days in the hospital. This places immense financial burden on families including medical bills, transportation costs, childcare, and missed days from work. This burden and resulting distress are called financial toxicity. Dr. Zheng's research is focused on measuring financial toxicity and trying to figure out what can be done about it. One important idea to consider is that many parents quit their jobs or reduce their hours to care for their child. Dr. Zheng plans to use surveys and interviews to gain a clearer picture of how work disruptions develop over AML treatment and lead to financial toxicity. Dr. Zheng wants to identify what work arrangements and policies could offer the most support. Ultimately, it could lead to advocating for more flexible work schedules, remote work options, or other accommodations that could make a real difference for these families.

Acute Lymphoblastic Leukemia (ALL) are aggressive cancers of B- and T- immune cells. ALL is most common in children but also affects adolescents and young adults. 90% of childhood ALL is curable. However, ~10% of children and ~30% of adolescents and young adults with ALL are not cured. To combat hard-to-treat ALL, Dr. Swaminathan will harness the body’s natural anti-cancer defense mechanism: a type of immune cell called a natural killer (NK) cell. He will also find defective NK cells in children with ALL. Those with fewer defective NK cells tend to survive longer and spend more of their lives free from disease compared to patients with high levels of abnormal NK cells. These findings will inform the development of NK cells as affordable therapies to cure pediatric ALL.