Grants Search Results

Limited progress has been made over the last 30 years against kid brain tumors, especially those in the thalamus and the pons (Diffuse Intrinsic Pontine Glioma, DIPG), a specific location in the brain. Radiotherapy (RT) is the only treatment available that can prolong the life of children with the most aggressive form of brain tumors. Recently, RT is recognized to activate the immune system against multiple tumors. However irradiated kid brain cancers always regrow which suggest that RT is not activating immunity against these tumors. Understanding why this phenomenon is happening is critical to develop strategies that will exploit the immune stimulation from RT to control and cure brain cancer. The activation of cancer-associated fibroblasts (CAFs) by RT can be responsible for treatment resistance and the lack of immune stimulation of kids brain cancers. Dr. Vanpouille-Box's initial results show that stopping the immunosuppression of CAFs with a fibroblast activating protein alpha (FAP) blocker re-activates the immune system against irradiated pediatric brain tumors. Thus, blocking CAF emerges as a novel approach to prevent brain cancer regrow and to activate immunity in irradiated brain cancer. She proposes to: 1) Define the role of CAF in mice models of pediatric brain cancer 2) Determine the efficacy of CAF and EGFR blockade in irradiated pediatric brain cancer. Dr. Vanpouille-Box and colleagues hope to find that: - CAF stop the immune stimulation of irradiated pediatric brain tumors - blocking CAF immunosuppression works well to reactivate immunity against irradiated brain cancer, especially on the context of epidermal growth factor receptor therapy. 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.

Based on progress to date, Dr. Moghimi was awarded a new grant in 2023 and 2024 to fund an additional year of this Scholar grant. In recent years, a very successful immunotherapy strategy to modify a patient's immune cells (called T-cells) to attack cancer has been developed for children with leukemia (cancer of blood cells). These modified immune cells are called Chimeric Antigen Receptor T cells (CAR-T cells). These CAR-T cells are very potent and do a better job than any chemotherapy at killing cancer. However, this life-saving tool has been available only to a small group of patients and for a handful of cancers. This is because most cancers don't have the targets those CAR-T cells aim for, or they have a target that can also be found on normal organs. As a result, these CAR-T cells could harm normal organs as collateral damage, a significant adverse effect of treatment that clinicians would want to avoid. In this proposal, Dr. Moghimi is striving to build the next generation of CAR-T cells that solely react to a combination of targets. These cells recognize a tumor only if they have both targets in sight and will not otherwise attack normal organs. CAR-T cells that operate based on a combination of two targets are more accurate than other targeting cells. Using this new generation of CAR-T cells, researchers would be able to significantly expand the availability of this powerful treatment to many more patients. Dr. Moghimi and colleagues will develop these special CAR-T cells for patients with Acute Myeloid Leukemia (AML), another common form of leukemia with a higher mortality rate for children. These results will provide pre-clinical evidence that could quickly translate to new clinical trials for children with relapsed AML through the Therapeutic Advances in Childhood Leukemia and Lymphoma (TACL) consortium, an international collaboration headquartered at CHLA.

Based on progress to date, Dr. Wu was awarded a new grant in 2023 and 2024 to fund an additional year of this Scholar grant. Diffuse midline glioma (DMG) is a fatal brain cancer in children and there are no effective treatments. The brain's natural barrier prevents drugs from reaching the tumor. Focused ultrasound (FUS) uses sound waves to temporarily open the blood brain barrier to increase drug delivery to the protected tumor cells in the brain. As the Hannah's Heroes St. Baldrick's Scholar, Dr. Wu will be using panobinostat, a promising drug tested in cancer cells in the laboratory to examine if FUS can increase its delivery and whether the addition of radiation can further improve the outcomes. The 2023 year of this grant is co-funded by the Focused Ultrasound Foundation. The 2021 and 2022 years of this grant are funded by and named for Hannah’s Heroes, a St. Baldrick's Hero Fund established to honor Hannah Meeson. At age six she was diagnosed with anaplastic medulloblastoma. After a relapse and several additional months of 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.”

Based on progress to date, Dr. Jones was awarded a new grant in 2023 to fund an additional year of this Scholar grant. Acute myeloid leukemia (AML) is a difficult to treat cancer that is associated with death in nearly 4 out of 10 children who are diagnosed with this disease. We know that there are multiple factors that contribute to poor outcomes in these patients, however, researchers don't fully understand all of them. Dr. Jones will gain a greater understanding of the resistance associated with a specific type of AML that is particularly difficult to treat. She hopes to gain clarity about this type of disease to find more specific therapies to target those resistance mechanisms in the cancer cells.

Unfortunately, kids with cancer suffer from symptoms related to their cancer and cancer treatments. These symptoms are often not fully appreciated by their health care providers and hence may be undertreated. Doses of cancer treatments are decreased or cancer treatments are even stopped when symptoms from cancer treatments aren't well controlled. Improving symptom assessments for kids with cancer will enhance health care providers' ability to track and manage kids' symptoms, to identify symptom trends, and could even prevent changes in cancer treatments due to poorly controlled symptoms. The Patient Reported Outcome Common Terminology Criteria for Adverse Events (Pediatric PRO-CTCAE) is a recently-developed survey that allows kids with cancer and their parents to directly report the type and severity of kids' symptoms to health care providers. This tool includes 130 questions about 62 different symptoms. However, since there are so many questions in this tool, the symptoms that are asked about are pre-selected by researchers or health care providers. Pre-selecting the questions prevents kids and parents from reporting all of a kid's symptoms, risking symptoms being missed. The overall objective of Dr. Crane's research is to refine and pilot test a novel web-based interface for the Pediatric PRO-CTCAE that will allow kids and parents to systematically and easily report all of a kid's symptoms on a routine basis, but without having to answer 130 questions.

Although rates of cure for childhood cancer have greatly improved in recent years, thousands of children continue to suffer from advanced, incurable cancer. Healthcare professionals bear a responsibility to ensure that the care of children with advanced cancer meets the goals and wishes of patients and their families. However, we do not know whether we fulfill this aim in pediatric cancer care. Dr. Ananth's prior research reveals intensive healthcare use near the end of life for children with advanced, incurable cancer. This includes lengthy stays in the intensive care unit and common use of interventions like breathing tubes. Yet, healthcare professionals worry that intense care toward the end of life for children with cancer may increase child and family suffering. In adults with cancer, quality measures have been developed to evaluate where care is most intense, or poor quality. This has consequently allowed researchers to develop interventions to improve the quality of care for adults with incurable cancer. Unfortunately, there are no comparable measures or standards for what constitutes good, or high quality, end-of-life care for children with cancer. Dr. Ananth seeks to address this problem. The overall objectives of this research are to (1) refine a list of potential measures of high quality end of life for children with cancer, and (2) develop an innovative questionnaire to systematically evaluate whether patients are receiving high quality end-of-life care. She hopes that, through this work that is distinctly family-centered, she can develop interventions to enable healthcare teams to provide optimal, compassionate care for children who have incurable cancer.

Based on progress to date, Dr. Bertrand was awarded a new grant in 2022, 2023, and 2024 to fund an additional year of this Scholar grant. Ependymoma is an aggressive pediatric brain tumor that is treated with surgery and radiation, but is resistant to chemotherapy. Ependymoma can be divided into different groups by location and biology. One type of ependymoma is driven by a fusion cancer-causing protein RELA-fusion. There are currently zero available drug therapies that target this protein, and we have a poor understanding of its function in cancer. Dr. Bertrand's research seeks to understand how this protein induces cancer in cells and models so that we can devise new treatments. The 2024 portion of this grant is funded by and named for Hannah’s Heroes, a St. Baldrick's Hero Fund established to honor Hannah Meeson. At age six she was diagnosed with anaplastic medulloblastoma. After a relapse and several additional months of 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.” Awarded at Baylor College of Medicine and transferred to St. Jude Children's Research Hospital.

Based on progress to date, Dr. Bonifant was awarded a new grant in 2022 and 2023 to fund an additional year of this Scholar grant. There is a clear need for new treatment options for pediatric relapsed or refractory acute leukemia. Patients receiving standard chemotherapy regimens continue to suffer a poor prognosis. Immunotherapy has the potential to fulfill this need through its precise tumor targeting and novel anticancer biology. Recently, a type of immune cell, the T cell, has been genetically modified to specifically recognize leukemia cells through a specialized receptor (CAR). Infusion of CAR-T cells has shown stunning anti-leukemia activity in clinical trials. However, this approach has key limitations. Importantly, CAR-T cell persistence in each patient is critical to ensure continued disease remission. Alterations in leukemia surface protein expression have also been reported as a mode of CAR-T evasion. These may lead to disease relapse. Dr. Bonifant and her colleagues believe they can introduce new elements to existing chimeric receptors to promote improved CAR-T survival. They also believe they can alter these cells to target more than one surface antigen -- to prevent antigen loss and CAR-T escape, while enhancing specific disease targeting. The overall rationale for this proposal is the confidence in CAR-T cells as a powerful addition to anti-leukemia therapy. Dr. Bonifant would like to facilitate continued improvement of this therapeutic modality in order to ultimately attain durable cures. The 2021, 2022, and 2023 portions of this grant are 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. The 2020 portion of this grant is funded by and named for the Rally for Ryan Fund, a St. Baldrick's Hero Fund. Ryan was diagnosed with high risk ALL when he was seven years old. He endured 3 ½ years of often harsh treatments with smiles, laughter and a brave acceptance that this was his fight to win. And Ryan did prevail—he took his last chemo pill in January 2016 but relapsed at the end of the year. He endured CAR T-cell therapy and a bone marrow transplant and is once again cancer-free. This fund honors Ryan’s commitment to help make a difference for kids with cancer.

Based on progress to date, Dr. Chavez was awarded a new grant in 2022 to fund an additional year of this Scholar grant. Researchers have found that some very aggressive cancers produce extra pieces of DNA that are located outside of our 23 chromosomes and form circles. This is why we call them circular extrachromosomal DNA, or ecDNA. These ecDNAs are thought to be a fundamental driver of cancer growth. However, very little is known about ecDNA in childhood brain tumors. This is why researchers have now looked for ecDNA in medulloblastoma- a cancerous brain tumor that starts in the lower back part of the brain, called the cerebellum. Medulloblastoma can occur at any age, but most often occurs in young children. Though medulloblastoma is rare, it's the most common cancerous brain tumor in children. And indeed, we have observed that there are very specific types of ecDNA in medulloblastoma tumors, especially in those tumors that are very aggressive and difficult to treat. As the Hannah's Heroes St. Baldrick's Scholar, Dr. Chavez would like to learn more about ecDNAs in medulloblastoma and hopes that this will lead to a scientific revolution in how some of the most difficult-to-treat childhood brain tumors are understood and treated. This grant is named for Hannah’s Heroes, a Hero Fund established to honor Hannah Meeson. At age six she was diagnosed with anaplastic medulloblastoma. After a relapse and several additional months of 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.” This grant was awarded at the University of California, San Diego, and transferred to Sanford Burnham Medical Research Institute.

Acute myeloid leukemia (AML) is an aggressive pediatric cancer associated with poor outcomes. Current therapies are toxic and result in a high incidence of late effects; including infertility, heart failure, and second cancers. Therefore, distinguishing who will be cured with chemotherapy alone from those who require more intensive therapies is critical to improving cure rates in AML while limiting treatment related late effects. The presence of small numbers of persisting leukemia cells after chemotherapy has become an important predictor of leukemia relapse. However, current assays used to detect residual leukemia have limited sensitivity and many patients with "no detectable leukemia" still go on to relapse. This underscores the need to identify and develop more accurate and sensitive leukemia detection assays for AML. This project aims to develop a novel assay that harnesses "best in class" technologies to enable detection of one leukemia cell for every one million normal cells -- a sensitivity that eclipses the current standard of care by more than one hundred-fold. Additionally, unlike many other novel methods for detecting leukemia, this assay will be universally applicable to every patient diagnosed with AML. Finally, this assay will reveal not simply whether or not leukemia cells are present, but the exact genetic code comprising the remaining leukemia cells. Successful validation of Dr. Huang's assay will therefore fill a critical unmet need in the field of AML, and the resulting product will be an optimized test ready for clinical use. A portion of this grant 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.

Many children with cancer cannot be cured with chemotherapy alone and must undergo stem cell transplantation (sometimes known as bone marrow transplantation). But this potential cure can also cause a very bad complication called graft-versus-host disease (GVHD) which can make children suffer miserably or even cause death. Studies have shown that a special type of white blood cell called an invariant natural killer T cell (or iNKT cell) is important in preventing GVHD. However, there are many different kinds of iNKT cells, some of which may be good to prevent GVHD and some of which may be ineffective or even cause harm. As the Rays of Hope St. Baldrick's Scholar, Dr. Mavers' research will study these different iNKT cells to identify ways we can separate out the good cells and use them to prevent GVHD, as well as ways we can modify the iNKT cells to make them even better at this job. The results from this project can help make stem cell transplantation a safer way to cure cancer and give survivors long, healthy lives. This grant is funded by and named for the Rays of Hope Hero Fund that honors the memory of Rayanna Marrero by giving hope through research funding. She is remembered for her infectious smile and energetic spirit that continue to inspire so many. This grant was awarded at Stanford University and transferred to Washington University, St. Louis.

Cancer immunotherapy has demonstrated great potential for treating cancer. However, challenges such as 1) the lack of ideal targetable tumor antigens; 2) severe toxicity due to off-target interactions; and 3) tumor-mediated immunosuppression are limiting the success of immunotherapies to be broadly applicable. To potentially overcome these challenges, Dr. Wu and his colleagues have developed a programmable synthetic gene circuit platform that enables tumor-localized therapeutic payload production, for recruitment and activation of immune cells: Tumor Immuno-therapy by Gene-circuit Engineered Response (TIGER). This strategy makes use of the body's own immune system to kill tumor cells. Gene circuits (highly engineered DNA sequences that work together), delivered systemically, will be turned on by the presence of two cancer-specific signatures, therefore only be activated within cancer cells and not normal cells. Cancer cells will be forced by the activated gene circuits to produce immunomodulators. Dr. Wu has demonstrated that TIGER mediates robust therapeutic efficacy in vivo in solid tumors. They have also identified sensors that can distinguish high-grade stem-like glioma cells from non-stem-like glioma cells. To further accomplish clinical translation of this platform, several advances are required: 1) identification of tumor sensors that efficiently detect highly heterogeneous primary patient tumors, to optimize tumor-targeting efficiency and specificity; and 2) optimization of therapeutic output combinations for achieving maximal efficacy. This project will focus on advancing the above two aspects to facilitate clinical translation of TIGER to treat pediatric high-grade glioma and overcome existing barriers to effective immunotherapy. The first year of this grant is funded by and named for the Kai Slockers Pediatric Cancer Research Fund. Kai was diagnosed at 2½ with Atypical Teratoid Rhabdoid Tumor (ATRT), a rare and very aggressive brain cancer. Within two weeks of diagnosis, he passed away, a mere 3 months shy of his third birthday. When Kai took his last breath, the cloudy sky opened up with a bright ray of sun that streamed through the windows of his hospital room – the darkness of the disease was replaced with the light of hope and the peace of no more suffering. Whenever the sun is out, his family thinks of him, assured that his legacy of hope shines on. In his brief life, Kai shared his warmth, energy, goofy sense of humor, and caring heart with all those he met. This Hero Fund was created in his memory and will support research to help other kids with cancer have a better chance to fight and survive. It has a special focus on brain tumor research, specifically treatments that could minimize the harsh effects of brain tumor treatment. The Slockers family hopes to continue his legacy of light and hope through the funding of childhood cancer research. A portion of this grant is generously supported by the Derick the Defeater Fund, a St. Baldrick's Hero Fund. Derick was a hero in so many ways. Diagnosed with medulloblastoma at the age of six, he endured 2 years of treatment with determination and a positive spirit. He inspired friends and family with his smile and charisma, even advocating for other children fighting cancer and teaching everyone what it meant to be brave. Derick’s courage lives on in a comic book his friends created called “Derick the Defeater” about a superhero who fought evil villains that looked like cancer cells. This Hero Fund honors his legacy of helping others through the funding of childhood cancer research.

Based on progress to date, Dr. Hoang-Minh was awarded a new grant in 2022 to fund an additional year of this Scholar grant. Brain tumors are the most common cause of cancer-related deaths in children. The current treatments are often associated with lifelong mental and motor deficits, and the tumors often recur. Therapies that specifically and efficiently target the tumors and minimize toxicity to the body are critical to improve clinical outcomes for children affected by these deadly diseases. As the Pray for Dominic St. Baldrick's Scholar, Dr. Hoang-Minh's research is exploring a powerful method that uses the children's own immune system to destroy their brain tumors, known as immune cell therapy. This therapy has emerged as a very effective and safe treatment for blood cancers and several types of solid tumors. It uses powerful immune cells, called T cells, to specifically kill the brain cancer cells and has already shown promising results in preclinical and clinical studies conducted at our institution. This project investigates novel approaches to make this immune therapy even more effective and safer. Dr. Hoang-Minh will also follow the fate of therapeutic T cells using a new, non-invasive imaging technology called magnetic particle imaging. The results of these studies are important as they could improve clinical protocols using immune cell therapies for childhood brain tumors and extend or save the lives of children afflicted with those very aggressive cancers. 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.

Thanks to remarkable scientific advances, over 80% of children with cancer will become long-term survivors, but most survivors experience long-term side effects of treatment. Our research has found that parents want early information about long-term side effects of treatment starting at diagnosis, but unfortunately most parents do not receive the information they need. In this project, Dr. Greenzang and colleagues are building a website to help parents understand the long-term effects of their treatment choices. They will then use the website with parents who are making new treatment decisions to evaluate whether parents find the website clear and useful, and to assess if using it can improve parents' understanding of medical information, help parents make decisions about treatment, and help prepare parents for their children's long-term care.

In the new era of personalized medicine, Ewing sarcoma still relies on decades-old chemotherapy options, where aggressive treatments are met with poor disease outcomes. Ewing sarcoma is a devastating disease that affects children and young adults age 5-16. Based on treatment outcome and patient qualities of life, Ewing sarcoma is in desperate need of research and development of new therapeutic options. One of the key observations of Ewing sarcoma made back in the 1930s is the accumulation of a large amount of glycogen. Glycogen is a sugar molecule that our body uses to store energy; only specific organs such as the liver and muscle are capable of producing glycogen. The ability of Ewing sarcoma tumors to store large amount of glycogen has been forgotten until now. Dr. Sun aims to understand the reason behind large glycogen accumulation in Ewing sarcoma and exploit the glycogen deposits as a possible drug target for the treatment of Ewing sarcoma. The successful completion of this project will bring new hope to this century-old disease and facilitate the development of the next generation of novel therapeutics specifically for Ewing sarcoma. A portion of this grant is funded by and named for Julia's Legacy of Hope, a St. Baldrick's Hero Fund that honors her positive and courageous spirit and carries out Julia's 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.

Based on progress to date, Dr. Johnston was awarded a new grant in 2022 to fund an additional year of this Scholar grant. Hospice (home-based end of life care focusing on pain control and emotional support) leads to better quality of life for the dying person and easier grief for families when an adult dies of cancer. We do not know if the same is true in children. We do not know rates of hospice use in children dying of cancer, what inequalities exist, nor how families perceive hospice, especially in minorities. Dr. Johnston aims to better understand US pediatric hospice use with a focus on minorities. Her team will determine rates of and inequities in hospice use in children dying of cancer nationally and then interview families that had a child die of cancer and pediatric oncology and hospice teams to determine if the inequities are in line with child and family preferences. Early palliative care in adults leads to better quality of life and more hospice and home death. It is unknown if the same is true in children with cancer. Dr. Johnston and colleagues will offer early palliative care to children with brain tumors to determine if early palliative care is acceptable to families and providers and if a randomized control trial would be possible. After this project, she will be able to use information and skills gained to design a trial aimed at improving hospice access for those that desire hospice that builds on the lessons learned in this study. Researchers will then better understand hospice in children with cancer, necessary to ensure all children dying of cancer have high quality end of life care.

Ewing sarcoma is an aggressive bone and soft tissue cancer that primarily affects children and adolescents. Patients often suffer severe side-effects from treatment and there are no second-line therapies for relapsed tumors. It is critical that we develop new and less toxic treatments for this cancer. Ewing Sarcoma is caused by a rearrangement of DNA that fuses pieces of two different proteins together to form a new protein. This new protein, called EWS-FLI1, can turn on genes that should not be on, leading to the transformation of the cell into an Ewing Sarcoma tumor. This fusion protein has features that make it very difficult to study, it sticks to itself and does not have a structure, a good analogy is that it behaves like a piece of cooked spaghetti. As the Shohet Family Fund for Ewing Sarcoma Research St. Baldrick's Scholar, Dr. Libich is utilizing his background in working with similar proteins that do not have structure. He is using NMR (nuclear magnetic resonance) which works just like MRI, to peer into the protein to understand exactly how it functions. This information is critical for designing molecules (drugs) that will be able to only affect the function of EWS-FLI1 and thus open new ways of attacking Ewing's sarcoma. This grant is funded by and named for the Shohet Family Fund for Ewing Sarcoma Research. In his freshman year of college, Noah was diagnosed with Ewing sarcoma. He endured many months of chemotherapy and had limb salvage surgery. Able to return to school, Noah had no evidence of disease for 2½ years until April 2018 when routine scans revealed he had relapsed. He passed away in May 2021 at the age of 25. Noah and his family were always passionate about the need for curative treatments for diseases of the AYA population. The Shohet family intends to raise funds for this Hero Fund in Noah's memory to find cures for Ewing sarcoma and to carry on his legacy of possibilities and hope.

Survivors of cancer have a higher risk of health problems because of the severity of the chemotherapy and radiation treatments they received. As survivors of childhood cancer age, they increasingly succumb to the "late effects" of their cancer treatment (such as second cancers and heart and lung disease). After 10-15 years, these late effects become the leading causes of death in this population. Adolescents and young adults (AYAs, aged 15-39) are a subgroup of cancer patients that are defined as high risk because they: more commonly suffer from toxicities and side effects of their cancer treatment; have unique barriers to accessing health care; and suffer specific psychosocial concerns because of their life stage transitioning into adulthood. To date, little research has been done on the factors that influence long-term health outcomes in the population of survivors of AYA cancer. Dr. Moke is working to explore how cancer and its treatments affect health later on in life in survivors of AYA cancer, identify the causes of death in this population, and determine what factors and cancer treatments are associated with these specific life threatening health problems. This study will provide the baseline data needed to design ways to decrease the severity of and death from these late effects, and thus be an important step in promoting long and healthy lives in survivors of AYA cancer.

Based on progress to date, Dr. Velasquez was awarded a new grant in 2022 and 2023 to fund an additional year of this Scholar grant. Childhood acute myeloid leukemia (AML) is a blood cancer that is very difficult to treat in children and adolescents. T cells are one component of the patient's own immune system that helps defend against infections. New cancer treatments use modified T-cells that can ‘see' and kill cells that have CD123, a molecule present on AML, and have shown promising results in studies in the laboratory. Dr. Velasquez is testing if these specific T-cells are safe and can kill leukemia cells in children that have AML that came back after initial treatment. She also wants to see what happens to these T-cells after they have been given to the patient and how it affects the tumor. This information from the treated patients and the study of their T-cells and tumor cells will be useful in finding a cure for AML.

Osteosarcoma is a cancer of bone that happens in young people. Dr. Davis and colleagues are trying to find ways to help the immune system fight off tumor cells, which may help us find a cure. They are examining all of the different type of immune cells in over 100 osteosarcoma tumor samples to identify how patterns in the cells match with other characteristics, such as how well a patient does with standard osteosarcoma treatment. They are also looking at biopsies from patients before and after immune therapy and will try to boost responses to immune therapy with a targeted drug. By understanding the way the immune system "sees" (or doesn't see) osteosarcoma, they will be able to predict which patients will benefit from different types of immune therapy and who will need other drugs added to their treatment regimen. A portion of this grant is generously supported by the Sweet Caroline Fund, a St. Baldrick's Hero Fund, created to honor the memory of Caroline Richards who was diagnosed in 2014 with osteosarcoma in her right arm when she was 11 years old. She persevered through rigorous treatments with a giving spirit and a contagious smile, always thinking of how to make others happy or laugh. Caroline sadly lost her battle a year later but this fund pays tribute to her compassion for others by supporting osteosarcoma research to help kids with cancer.