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Children with high-grade gliomas, such as glioblastoma multiforme, have few therapeutic options and usually die of their disease. CAR T cells recognize protein targets on cancer cells and kill those cells. Many brain tumors express target proteins on only some of their cells and therefore cannot be efficiently treated with a CAR T cell that recognizes only one target. Therefore, Dr. Majzner aims to make T cells that can recognize up to four targets. He is exploring the best way to achieve specificity (the narrowness of the range of substances with which an antibody or other agent acts or is effective) for four antigens including using gene editing in order to make CAR T cells that can come from a healthy donor but be used in any patient.

A portion of this grant is funded by and named for the Be Brooks Brave Fund. Despite his diagnosis at age 5 with inoperable brain and spinal tumors, Brooks taught so many people what life is truly about--love. He was BRAVE beyond his years with an inspiring “faith over fear” attitude. This Hero Fund hopes to raise money for high-grade glioma research so no other family will hear the words, “there is no cure”.

A portion of this grant was also generously co-supported by the McKenna Claire Foundation, a St. Baldrick's partner and the Living for Luker Brain Tumor Research Fund, a St. Baldrick's Hero Fund. The McKenna Claire Foundation was established by the Wetzel family in memory of their daughter, McKenna. Their mission is to cure pediatric brain cancer by raising awareness, increasing community involvement and funding research. The Living for Luker Brain Tumor Research Fund was established in memory of Luke's love for life and caring for others. He was diagnosed at age 10 with Diffuse Intrinsic Pontine Glioma, a rare, uncurable cancer and never gave up hope throughout treatment.

There is a new and effective cancer treatment for some incurable pediatric blood cancers. The treatment involves programing a patient's own cells to destroy their tumor, a process called cellular immunotherapy. Despite great effort to use cellular immunotherapy to treat 'solid' tumors, which include tumors of the bones, muscles and other parts of the body, we have not been successful yet. One major reason is that the programmed patient cells are designed to recognize a single marker on a cancer cell. In some blood cancers, all of the tumor cells express the same marker increasing the likelihood that cellular immunotherapy can cure the patient. Solid tumors are more heterogeneous than blood cancers, meaning each solid tumor cell may express a different marker. Therefore, cellular immunotherapy is less likely to destroy all solid tumor cells and the chances of achieving a cure is much more difficult. A potential solution is to trigger the body's own immune system to destroy tumor cells that express many different markers, a process called "epitope spreading". Named as the David's Warriors St. Baldrick's Scholar, Dr. Leibowitz focuses his project on testing strategies to augment epitope spreading in pediatric solid tumors so that cellular immunotherapy may become an effective and viable treatment option in the future.

This grant is named for and generously supported by the David’s Warriors Hero Fund created in memory of David Heard who battled neuroblastoma and inspired his family and countless others to commit to raising money for research to fight pediatric cancer. This fund honors the amazing spirit in which he lived, embracing life until the very end.

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.

People always say we need to outsmart cancer to beat it. As the Hope with Hazel St. Baldrick's Scholar, Dr. DuPage is not sure we will ever be able to do this with simple chemicals or radiation beams. Not only are these strategies rarely specific for the cancer cells alone, often leading to severe side effects that can be lifelong, but cancers always find a way around these single agents and "relapse." What if we could use a "living drug" to treat cancer? A drug that was as wiley and adaptable as the cancer itself and would last for a lifetime? A smart drug! Our own immune systems are capable of fighting our own cancers with extreme precision if we can train them to do it. This research is focused on understanding how cells of our immune system interact with tumor cells and how we can train our own immune cells to fight our own cancers. It is called cancer immunotherapy, and for children with cancer, Dr. Dupage thinks there are no better patients because their young and healthy immune systems are perfectly suited to be trained to fight cancer, removing the need for procedures that can manifest dangerous side effects in adulthood, and safely protecting them for life.

This grant is named for the Hope with Hazel Fund in honor of Hazel Hammersley who was diagnosed with Stage III neuroblastoma at age 2. She endured treatments, surgeries and two relapses with an amazing ability to push through and live life to its fullest. She loved her family and her happy place was with them. Through Hazel, her family learned that no one can fight pediatric cancer alone. This fund remembers her love of life and is dedicated to raising awareness and funds for research to protect kids with cancer who are still in the fight.

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.

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.

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 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.

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. 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.

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.

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.

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 doctoral 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 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. 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.