Grants Search Results

Many chemotherapy drugs used in childhood cancer have the potential to damage peripheral nerves and thus may impact a child’s ability to function. Early detection of this nerve damage is important to tailor treatment plans and initiate rehabilitation interventions. Currently, no measure of chemotherapy-induced peripheral neuropathy (CIPN) exists that is appropriate for use in children ages 1-4 years, an age group that is more commonly impacted by a number of childhood cancers. Thus, Dr. Gilchrist is finalizing the development of a scale of CIPN specifically tailored for young children that focuses on eliciting responses to test nerve function (such as tendon reflexes and strength of muscle groups) as well as developmentally appropriate measures of motor function (such as grasping an object or standing on one foot). She is also comparing results from 25 children ages 1-4 years being treated for cancer with neurotoxic treatments and 25 age and gender-matched controls to determine if the measure is both reliable and valid. If successful, she will be able to provide an objective measure for this common treatment side-effect that should positively impact both research and clinical care for these young children.

Juvenile myelomonocytic leukemia (JMML), a fatal childhood blood malignancy, has limited therapeutic options. Relapse remains the main cause of treatment failure, most likely due to the persistence of leukemic stem cells (LSCs), a small population of self-renewing precursor cells that give rise to the bulk of tumor cells. Dr. Qu is exploring an innovative approach to eradicating LSCs in a subset of JMML that is caused by genetic mutations in Ptpn11. The information gathered from this study may yield a novel strategy for the treatment of this particular type of JMML.

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

Millions of cells are formed every day in the developing brain of children. Medulloblastoma, a pediatric tumor, occurs when the proliferation of cells in the cerebellum (a lower part of the brain) becomes uncontrolled. The Notch pathway is a key mechanism that governs cell proliferation in many biological contexts. Aberrant up-regulation of Notch signals is associated with medulloblastoma. Re-gaining control of Notch could help cure medulloblastoma patients. As the recipient of the Hope for Daisy Research Fund for Pediatric Brain Tumors St. Baldrick's Research Grant, Dr. Rual's goal is to better understand the molecular mechanisms that control Notch signals in brain cells and, thus, to define novel therapeutic targets for the benefit of medulloblastoma patients. He recently identified the L3MBTL3 gene as a new modulator of Notch signals. Importantly, previous studies have shown that the L3MBTL3 genes is deleted in medulloblastoma patients. Dr. Rual hypothesizes that the L3MBTL3 deletions observed in medulloblastoma patients result in the aberrant regulation of Notch signals, thus supporting tumorigenesis. Dr. Rual's team will test this hypothesis by studying the extent to which inhibiting L3MBTL3 modulate medulloblastoma tumor progression in models of medulloblastoma. This study could offer critical mechanistic insights on the role of the L3MBTL3 in medulloblastoma that could be harnessed in the future for the therapeutic benefit of medulloblastoma patients.

This grant is funded by and named for the Hope for Daisy Research Fund for Pediatric Brain Tumors, a St. Baldrick's Hero Fund. Diagnosed with medulloblastoma at the age of six, Daisy Walsh met the challenge head on with joy, strength and laughter. Days before her eighth birthday, the tumor recurred and despite her brave battle, Daisy passed away in February 2020. This fund honors her courageous spirit by helping to raise awareness and funds for research to increase survival rates and hope for all children battling brain cancer.

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

This grant is funded by and named for Emily Beazley's Kures for Kids Fund, a St. Baldrick's Hero Fund. At the age of 8, Emily was diagnosed with Stage III T-cell lymphoblastic non-Hodgkin’s lymphoma and battled through three relapses. Her family prayed for a miracle but discovered Emily herself was the miracle, inspiring a community to come together to show love and change lives. She had a dream of starting a foundation to fund research and named it “Kures for Kids”. Today, Emily's family and friends carry on her dream and her mission in her memory.

Medulloblastoma is the most common malignant brain tumor of children. New approaches to treatment are needed, because current treatment can cause brain injury and fails too many patients. Some medulloblastomas are driven by excessive activity of a signaling pathway called SHH, and for these patients, SHH-pathway inhibitors may offer new hope. Drugs that target an SHH-pathway protein called SMO work against other cancers in other parts of the body. However, medulloblastomas rapidly become resistant when treated with SMO inhibitors.

As the recipient of the Miracles for Michael St. Baldrick's Research Grant, Dr. Sokolsky-Papkov will make SHH-targeted therapy newly effective for medulloblastoma using two innovations. She will use a new combination of two FDA-approved drugs, vismodegib and palbociclib. These inhibitors disrupt two different points in the pathway connecting SHH signaling to tumor growth, preventing resistance that can develop when either drug is administered alone. Furthermore, she has developed a method of packaging these drugs into tiny particles called nanoparticle micelles, which can deliver increased amounts of each drug into brain tumors. Dr. Sokolsky-Papkov hypothesizes that the combination of palbociclib and vismodegib, delivered for the first time in nanoparticle micelles, will advance brain tumor treatment and bring new effectiveness to medulloblastoma therapy.

This grant is named for the Miracles in Memory of Michael Fund created in memory of Michael Orbany who was diagnosed with medulloblastoma when he was six years old. Even through treatment and relapse, Michael had unwavering faith and perseverance, wanting most to make others happy. This fund honors his tremendous strength to never ever give up.

Despite cure after cancer, the majority of childhood cancer survivors are diagnosed with chronic health problems such as diabetes, hypertension, or heart disease within 30 years of cancer diagnosis and treatment. Hypertension and diabetes are important health problems associated with heart disease and result in early death compared to survivors without heart disease. Compared to siblings, childhood cancer survivors treated with radiation have a higher risk of developing diabetes at a young age. Even though important, the reasons for developing diabetes in cancer survivors are poorly understood. Insulin is an important hormone and affected by cancer treatment. Dr. Mostoufi-Moab is for the first time examining cancer treatment effects (radiation vs. chemotherapy) on making insulin and breaking down blood sugar in childhood cancer survivors, age 15-30 years. She will use tests to measure glucose breakdown, insulin secretion, and mathematical modeling to evaluate the body's handling of fat, all important steps for understanding the why cancer therapy causes diabetes in CCS. Understanding the steps leading to diabetes will help doctors chose the correct treatments for diabetes and for scientists to test options to prevent diabetes in cancer survivors. Achieving these goals will improve quality of life and avoid early death due to cancer treatment-related conditions in childhood cancer survivors.

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

Based on progress to date, Dr. Summers was awarded a new grant in 2020 to fund an additional year of this Fellow award. Early T-precursor ALL (ETP-ALL) is a type of leukemia that is often difficult to treat with currently available chemotherapy. As a result, children with ETP-ALL have high rates of relapse of their leukemia and poorer survival rates than children with other types of ALL, and require more treatment with chemotherapy, often leading to long-term toxic side effects. For these reasons, new treatments for ETP-ALL are needed. MERTK is a protein that is found on the surface of some leukemia cells, including ETP-ALLs. Recently, Dr. Summers and colleagues developed a new medicine that has few toxic side-effects and can be used to kill leukemia cells that have MERTK on their surface. Funded as the Emily Beazley's Kures for Kids Fund St. Baldrick's Fellow, this grant will allow him to test whether and how this new medicine could be used to more effectively treat children with ETP-ALL, leading to improved outcomes and better quality of life.

At the age of 8, Emily was diagnosed with Stage III T-cell lymphoblastic non-Hodgkin’s lymphoma and battled through three relapses. Her family prayed for a miracle but discovered Emily herself was the miracle, inspiring a community to come together to show love and change lives. She had a dream of starting a foundation to fund research and named it “Kures for Kids”. Today, Emily's family and friends carry on her dream and her mission in her memory.

Osteosarcoma is a cancer of bone that typically affects teenagers and young adults. Tumor spread (or metastasis) to the lungs is common, and up to 40% of patients with osteosarcoma will eventually experience a cancer recurrence (or relapse). Unfortunately, no therapies have shown benefit following relapse and these patients have a very poor prognosis. The ability of cancer to control and hide from the body’s immune system is important for tumor growth and metastasis, so preventing these functions is an important treatment strategy. Recent work, including a canine osteosarcoma trial, has shown that currently available medications may work together to block some of the effects that cancer has on the immune system, reducing tumor growth and the ability to spread. Dr. Faulk will conduct a clinical trial which will combine these drugs (losartan and sunitinib) in children and young adults with relapsed osteosarcoma to test the safety of the new combination, see how the drugs are broken down by the body, and determine how the drugs affect the immune system and the growth of the tumor.

Based on progress to date, Dr. Ch'ng was awarded a new grant in 2020 to fund an additional year of this Fellow award. Epstein-Barr virus (EBV) is a common viral infection that in the vast majority of people causes only minor or no illness. However, in some situations it can play a role in the development of certain forms of cancer, such as lymphoma. One way that it might contribute to the development of cancer is by affecting the way that cells use energy because viruses and cancers both require increased energy to support rapid growth. By studying how EBV changes the way that cells use energy, Dr. Ch'ng hopes to learn whether changes in cell energy use are a factor in the development of cancers associated with EBV and whether these changes can be targeted to treat these forms of cancer.

Based on progress to date, Dr. Huerta was awarded a new grant in 2020 to fund an additional year of this Fellow award. Leukemia is the most common type of cancer in childhood, and 20% of childhood leukemia has a myeloid origin. Acute myeloid leukemia (AML) is treated with intensive chemotherapy as standard of care. Unfortunately, despite chemotherapy and stem cell transplantation, the prognosis of a child with recurrent or refractory AML remains poor. T cells are part of our immune system, and when properly manipulated, can be highly effective in eradicating chemo-resistant tumor cells. Engager (ENG) T cells are genetically engineered T cells that are capable of binding specific target on AML cells and at the same time "engaging" neighboring T cells to mount an immune response and kill cancer cells. As the JJ's Angels Hero Fund St. Baldrick's Fellow, Dr. Huerta is investigating the mechanisms by which AML cells can be killed by this novel immunotherapy technique.

This grant is named for the JJ's Angels Hero Fund which honors the memory of Juliana LaMonica and her courageous battle with AML. Diagnosed at the age of two, Juliana underwent a bone marrow transplant but passed away shortly after turning three. Her sweet spirit and charismatic personality continue to inspire people to support the funding of pediatric cancer research through Team JJ’s Angels.

Based on progress to date, Dr. Peltier was awarded a new grant in 2020 to fund an additional year of this Fellow award. Bone marrow transplantation (BMT) is required to cure many childhood cancers. However, bone marrow transplantation is often complicated by severe and often fatal side effects. Both the beneficial anti-cancer effects and harmful side effects of bone marrow transplantation are due in part to the new immune system that the patient receives. Unfortunately, we do not know how to precisely fine tune this new immune system to make BMT safer for more children. As the Hope for Harper St. Baldrick's Fellow, Dr. Peltier seeks to further understand in his work how a component of this new immune system is controlled by a recently identified class of genes called non-coding RNAs (ncRNA). These ncRNA genes do not make proteins like classic genes, but instead regulate the production and function of proteins made by classical genes. His early data shows that unique ncRNA genes from multiple classes of ncRNAs are turned on and off following BMT. However, it is not known if or how these unique ncRNA genes influence the new immune system after BMT. Dr. Peltier seeks to further understand the function of these ncRNAs following BMT, which may suggest ways of developing medicines to improve BMT.

This grant is named for and generously supported by the Hope from Harper Fund created to honor Harper Wehneman who was diagnosed with Wilms tumor just before her 8th birthday. She fought valiantly throughout her cancer journey and is remembered for inspiring people to choose joy no matter the circumstance. This fund continues her legacy by giving hope to kids fighting cancer through research for stem cell transplant survival.

Despite recent advances in technology, very little is known about many types of rare and high risk childhood cancers. Since the numbers of these patients are so small, it has been very difficult to study how best to take care of them. Dr. Potter is using technology to look at the genetic code of these rare tumors, in order to learn more about why and how they occur, as well as how they change over time. This knowledge will help to create tests to diagnose these patients, as well as to develop more effective, less toxic treatments.

This grant is generously co-supported by the Invictus Fund and O Danny Boy I Love You So: The Danny O'Brien Rhabdoid Tumor Research Fund. The Invictus Fund was created to honor the memory of Holden Gilkinson who was diagnosed with Stage IV anaplastic Wilms tumor when he was 3 years old. Holden endured intense treatment and surgery, eventually losing both kidneys. He passed away just a few days shy of his 7th birthday. Through it all, Holden’s unconquerable spirit and love for life prevailed and is personified in the poem “Invictus” by William Ernest Henley. 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.

Based on progress to date, Dr. Dharia was awarded a new grant in 2020 to fund an additional year of this Fellow award. Despite progress made in the treatment of pediatric cancers, several childhood cancers, such as high-risk neuroblastoma, Ewing sarcoma and rhabdomyosarcoma, continue to have poor survival rates. It is critical that we identify new therapies for these cancers, especially now that we are learning how cancers are driven by specific changes in proteins that bind DNA and control transcription. Researchers are beginning to identify potential vulnerabilities in cancers by systematically deleting almost every single gene in a cancer cell, and describing in greater detail the mutations and other events that occur in pediatric cancers. As the Julia's Legacy of Hope St. Baldrick's Fellow, Dr. Dharia and his team are integrating data from such approaches to discover specific vulnerabilities in high-risk neuroblastoma, Ewing sarcoma and rhabdomyosarcoma. Different types of cancer cells require different instructions or programs to survive, and Dr. Dharia proposes the identification of these programs will lead to new targets to treat these cancers. By identifying, validating and characterizing new targets for treatment of these childhood cancers, Dr. Dharia hopes to discover new therapies for cancer care. This research will take advantage of drugs that are already available and ideally identify completely new ways to treat these cancers.

This grant is named for Julia's Legacy of Hope, a Hero Fund that honors her 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 hopes to raise awareness and funds for research especially for Adolescent and Young Adult (AYA) patients.

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

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

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

This grant funds a graduate student to complete work in pediatric oncology research for the summer. It is named for the St. Baldrick's Foundation Staff whose generous gifts have helped fund this opportunity and may encourage them to choose childhood cancer research as a specialty.

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

This grant funds a medical student to complete work in pediatric oncology research for the summer. It is named for the St. Baldrick's Foundation Staff whose generous gifts have helped fund this opportunity and may encourage them to choose childhood cancer research as a specialty.