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Dr. Dionysiou and team are studying a small molecule naturally found in the body called miR-21. This molecule could make a treatment called allogeneic hematopoietic cell transplantation (allo-HCT), which cures children with aggressive leukemia, safer and more effective. This treatment uses immune cells from a donor, but it can cause a serious problem called graft-versus-host disease (GVHD), where the donor cells attack the patient's healthy tissues. The challenge is to stop this attack without weakening the donor cells' ability to fight the cancer. By understanding how miR-21 controls the immune response, Dr. Dionysiou hopes to find ways to prevent GVHD while allowing the donor cells to attack the cancer, making this life-saving treatment safer and more effective.

This grant is generously supported by the Rays of Hope Hero Fund which honors the memory of Rayanna Marrero. She was a happy 3-year-old when she was diagnosed with Acute Lymphoblastic Leukemia (ALL). She successfully battled ALL, but a treatment induced secondary cancer claimed her life at age eight. Rayanna had an amazing attitude and loved life. She, like so many kids facing childhood cancer, did not allow it to define who she was. This Hero Fund aspires to give hope to kids fighting cancer through research.

In the past decade, new therapies that train the immune system to recognize and kill cancer cells have revolutionized cancer care. Unfortunately, cancers arising from connective tissue like bone have not responded to these immunotherapies. Despite almost four decades of trialing/testing progressively more intensive chemotherapy, outcomes for osteosarcoma (the most common bone tumor) remain dismal once it has spread beyond the initial site. Dr. Smith wants to understand why these immunotherapies have failed by studying a model closely resembling human osteosarcoma. Based on his findings, Dr. Smith will test novel immunotherapies to prioritize the next generation of osteosarcoma human clinical trials.

Teens with leukemia go through tough treatments that make them feel tired and weak, so they spend a lot of time sitting and lying down, which can make side effects worse and put them at risk for chronic diseases like diabetes. Dr. Ivory is testing ReSeT, a program she developed for teenagers getting leukemia treatment to interrupt sitting time with short exercise breaks that will likely improve their lifestyles, heart health, and quality of life. Over 10 weeks, each teenager will use a Fitbit, health coaching, and an app-based support group to slowly increase their activity. After testing ReSeT in 30 teenagers to see if they can do it and what they think, she will fine-tune ReSeT and test it again in 10 more teenagers and compare how they do with 10 teenagers who didn't get the program to see if the program works. The goal is to use small behavior changes to help teenagers with cancer be more active during and after treatment to improve their lifelong health.

This grant funds a student to complete work in pediatric oncology research for the summer. Dr. Satake and colleagues are studying a rare and aggressive childhood kidney cancer called malignant rhabdoid tumor of the kidney (RTK). Children with RTK have extremely poor outcomes (survival rate 25%) despite lots of different treatments since tumors continue to grow even with treatment and tend to relapse. They believe that the investigational drug called OTS964 may be a new potential treatment. They also believe that OTS964 may be even more effective when used with navitoclax, a drug which has a different mechanism of killing cancer cells. In this project They plan to test the new treatment using these drugs in a human RTK mouse model, and to study the mechanism of actions, with the goal of finding a new treatment for RTK patients. This work is being completed under the mentorship of Dr. Noriko Satake.

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.

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.

Children diagnosed with a brain tumor called diffuse midline glioma (DMG) have no options for a cure. Dr. Ramakrishna and team at Stanford Medicine have developed a new treatment, called CAR T cells, for these children by training their immune system to find and kill cancer cells. Excitingly, children treated on a clinical trial with these CAR T cells have improved symptoms and reduced tumor sizes. Unfortunately, for some patients, tumors grew after treatment, suggesting a need to understand how to improve the treatment. An immune cell, called a myeloid cell, was identified surrounding treatment of the first patients. Dr. Ramakrishna and colleagues will seek to understand these myeloid cells in the context of CAR T cell activity in patients. Modeling these myeloid cells in the lab, Dr. Ramakrishna and team will test approaches to improve CAR T cell activity against DMG cells. This project will improve understanding of CAR T cells in patients and develop new treatments for children with these devastating brain tumors.

Sneha Ramakrishna, MD, is a pediatric hematology-oncologist at Stanford Medicine Children’s Health and an assistant professor - University Medical Line in Pediatrics - Hematology & Oncology at Stanford Medicine.

This grant is named for the Pray for Dominic Hero Fund. The fund was established in honor of Dominic Liples who lived with joy. He is remembered for compassion and determination while he faced his own difficult battle with a rare and aggressive brain cancer. The Pray for Dominic fund carries on Dominic's legacy of joy and hope by funding research for high-grade gliomas.

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.

Children with a brain tumor diagnosis face the daunting prospect of surgery, radiation and chemotherapy. While these treatments can be successful, some brain tumors continue to grow and spread in the brain, which can make them impossible to treat. In addition, current treatments can adversely impact a child's cognitive, emotional and physical well-being. New treatments against the most aggressive brain tumors to reduce the burden of tumors in children and increase their quality of life are needed. Dr. Stewart and colleagues will develop new technologies and test new drugs that will allow to rapidly transplant patient tumor cells into models to make hundreds of model avatars that can then be treated with a panel of drugs to identify the most effective treatments for a child's specific tumor, focusing on very aggressive tumors first. Dr. Stewart and team expects this information will be used to help guide doctors decisions on the best treatment options to eliminate the child's tumor and minimize side effects.

Dr. MacDonald and colleagues have developed a new test that detects cancer cells in the blood and other fluids of patients with brain tumors. They have discovered that children with a particular type of brain tumor often have cancer cells in their blood. They can also use their test to follow how treatment changes the number of cancer cells in the blood to find out if the treatment being given is working to kill all the remaining cancer cells in the brain. This is the first time such a test has been able to do this using a simple blood draw. Dr. MacDonald and colleagues will use their test for children with other types of brain tumors. Dr. MacDonald and team will study the cancer cells in the blood to see why and how these brain cancer cells are still alive after treatment. This will help to identify the "steps" in which the cancer cells take to avoid being killed by the treatments being given and ultimately will then lead to new treatments targeting these steps to cure childhood brain tumors.

Dr. Stanton and colleagues are developing new approaches to understand how DNA is organized for gene expression in a lethal childhood tumor called rhabdomyosarcoma (RMS). In RMS, the standard of care therapies haven't changed substantially in 40 years and patient outcomes haven't improved greatly during this time. New approaches are desperately needed. There is a lack of a fundamental understanding of the mechanisms for how the cancer-causing genes function in RMS. Dr. Stanton and his team are integrating cutting edge approaches in synthetic biology, modeling, and genomics to understand how and why RMS forms, with the granularity of single gene targets for mechanism studies. Through Dr. Stanton's studies, the community will gain an understanding of how the cancer-causing genes are altering the organization of DNA in RMS cells.

This grant is funded by and named for the Aiden's Army Fund, a St. Baldrick's Hero Fund. Aiden Binkley who was diagnosed with Stage IV rhabdomyosarcoma at age 8. This bright, funny and courageous little boy believed he got cancer so he could grow up to find a cure for it. His vision is being carried on by Aiden’s Army through the funding of research. They will march until there is a cure!

Each year, approximately 1,000 Americans aged 20 years or younger are diagnosed with acute myeloid leukemia (AML). Currently, no drugs can eradicate all AML cells in pediatric patients, and cells remaining after treatment often cause disease recurrence and poor survival. Dr. Li and colleagues have found that two mitochondrial enzymes that function in energy metabolism, known as DHODH or SDH, shield leukemia cells from eradication by immune cells. Dr. Li will use models relevant to pediatric AML to ask how these factors block anti-cancer immune responses and test effectiveness of a first-in-class leukemia cell-specific DHODH inhibitor combined with existing immune therapy in eradicating AML. If successful, this study will lead to development of new anti-leukemia drugs that could approach a cure for childhood AML.

A subset of deadly pediatric cancers, known as sarcomas, are caused by a mysterious, mutated protein called CIC::DUX4. Treating this disease is very difficult, in part because little is known about its biology. The teams of Dr's. Wood and Hendrickson have assembled the world's best collection of CIC::DUX4 laboratory models, using them to discover that CIC::DUX4 sarcomas require a protein called COP1 for their survival, whereas normal tissues in bodies do not. Dr. Wood and colleagues will perform studies to understand how well future drugs targeting COP1 will work as therapies for this deadly disease while defining why this disease needs COP1 in the first place. Together, these studies will identify a highly promising new therapeutic strategy for CIC::DUX4 sarcoma while also revealing insights that could lead to additional new types of drugs for this disease.

Brain cancer is the leading cause of cancer-related deaths in children, highlighting the urgent need for more effective treatments. While chimeric antigen receptor (CAR) T-cell therapy has transformed outcomes for children's blood cancers, it has shown limited success in brain tumors. Dr. Huang and colleagues will initiate a phase I trial in children with HGG and DIPG to assess safety and immune effects utilizing the understanding that CD70, a protein driving tumor growth, is a promising CAR T-cell target for high-grade gliomas (HGG) and diffuse intrinsic pontine gliomas (DIPG).