Grants Search Results

Medulloblastoma (MB) is an aggressive childhood brain cancer that is fatal in 40% of patients. MB shows abnormal activation of growth pathways in tumor cells which help them to grow. Dr. Ganesan's studies show that there are T cells (immune cells) within MB and they have potential to kill tumor cells. However, immunotherapy that boosts the activity of these T cells have not been successful and it is not known why. The goal of this project is to understand why immunotherapy has not worked in MB and whether immunosuppressive myeloid cells contribute to this restraint. Dr. Ganesan and colleagues will also study if combined treatment that inhibits the growth pathways (targeted therapy) and stimulates the T cells/immune system (immunotherapy) may together lead to greater tumor killing in MB mouse models. To test if combined therapy would work in humans, Dr. Ganesan will coculture a 3D version of patient’s brain tumor with their own T cells expanded from their tumor. If effective, these studies may lead to new treatments for MB.

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.

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. Hsiao 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. Hsiao 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.

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.

Whilst it is well known that damage to our DNA can cause cancer, is is still not fully understand 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 toxin is 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.

The ultimate goal of this project is to define the safest, most effective therapies for children and young adults with Langerhans cell histiocytosis (LCH), which aligns with St. Baldrick's mission to find cures for childhood cancers and give survivors long and healthy lives. LCH is a blood cancer most common in children that creates destructive inflammatory lesions that can be fatal. LCH is caused by mutations activating the MAPK growth pathway in developing blood cells. Current front-line therapy fails to cure over 50% of patients with disseminated disease, and safe and effective options for subsequent therapy is not known. High-dose chemotherapy can be effective, but is toxic. MAPK inhibitor therapy alone does not appear to be durable based on early trials. Dr. Allen and colleagues hypothesize that MAPK inhibition will make cells more sensitive to chemotherapy. Dr. Allen will therefore test safety and efficacy of a new approach of combining chemotherapy with targeted MAPK inhibitor therapy.

Ewing sarcoma (EwS), the 2nd most common childhood bone sarcoma, is an aggressive tumour that primarily affects children, adolescents, and young adults. When EwS tumor cells spread to other parts of the body, known as metastasis, survival is drastically diminished to only 15-20%, which has not changed for decades. Immunotherapy empowers a patient’s own immune system to attack cancer, which has tremendous promise as an alternative to chemotherapies that are often toxic, especially to a growing child. Dr. Sorensen and his team recently identified a protein that is highly expressed on the surface of EwS cells, while showing only minimal to absent expression in normal tissues, nominating IL1RAP as a very promising therapeutic target. With their collaborators at the University of Pittsburgh, they have identified specific antibodies binding to IL1RAP and have engineered these antibodies to be conjugated to a drug that kills EwS cells potently. In this project, they will perform the extensive validation of these compounds to enable the design of early clinical trials for the treatment of EwS. This Better Ewing Sarcoma Therapies (BEST) grant is supported by a unique partnership of funders through the St. Baldrick’s Foundation: D-Feet Cancer, The Faris Foundation, The Shohet Family Fund for Ewing Sarcoma Research, an anonymous donor, and the family and friends of Martha Riedel.

This grant supports a Clinical Research Associate to ensure that more kids can be treated on clinical trials, often their best hope for a cure.

This grant supports a Clinical Research Associate (CRA) to ensure that more kids can be treated on clinical trials, often their best hope for a cure. At Children's Hospital of Michigan many patients are of minority background, and historically minorities have not always had the same access to health care. Funding from St. Baldrick's Foundation will help the institution continue to have excellent CRA support in the clinical trials office for every patient.

This grant supports a Clinical Research Associate to ensure that more kids can be treated on clinical trials, often their best hope for a cure.

This grant supports a pediatric-focused Clinical Research Associate to ensure that more kids can be treated on clinical trials, often their best hope for a cure.

This grant supports a Clinical Research Associate to ensure that more kids can be treated on clinical trials, often their best hope for a cure.

Florida lacks a larger comprehensive cancer center for children, which makes it more difficult for children fighting cancer to receive cutting edge therapy. This grant supports a Clinical Research Coordinator to ensure that more kids can be treated on clinical trials, often their best hope for a cure.

The Loma Linda University Children's Hospital (LLUCH) services a four-county region (San Bernardino, Riverside, Inyo and Mono Counties). For the 1.3 million children living in this region, 25% come from families that live in poverty and are uninsured. This population represents 80% of the children treated here. This grant supports a Clinical Research Associate to ensure that more kids can be treated on clinical trials, often their best hope for a cure. The participation of this patient population in clinical trials is critical for the identification of therapies that can alleviate this health disparity and effectively treat all children.

The Pediatric Early Phase Clinical Trials Program at Montefiore Medical Center has grown significantly since it's creation 2 years ago. The goal of this program is to bring early phase clinical trials to patients with relapsed and refractory cancers in our underserved, resource poor community, as well as to patients across the NY-NJ-CT (tristate) area. This grant supports a Clinical Research Coordinator to ensure that more kids can be treated on clinical trials, often their best hope for a cure.

This grant supports a Clinical Research Associate to ensure that more kids can be treated on clinical trials, often their best hope for a cure.

This grant supports a Clinical Research Associate to ensure that more kids can be treated on clinical trials, often their best hope for a cure.