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Showing 41-60 of 166 results

Lan Hoang-Minh Ph.D.

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Funded: 07-01-2020 through 02-17-2023
Funding Type: St. Baldrick's Scholar
Institution Location: Gainesville, FL
Institution: University of Florida affiliated with Shands Hospital for Children

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.

Benjamin Huang M.D.

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Funded: 07-01-2020 through 06-30-2022
Funding Type: St. Baldrick's Scholar
Institution Location: San Francisco, CA
Institution: University of California, San Francisco affiliated with UCSF Benioff Children's Hospital

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.

Melissa Mavers M.D., Ph.D.

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Funded: 07-01-2020 through 06-30-2023
Funding Type: St. Baldrick's Scholar
Institution Location: St. Louis, MO
Institution: Washington University in St. Louis affiliated with St. Louis Children's Hospital

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.

Ming-Ru Wu M.D., Ph.D.

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Funded: 07-01-2020 through 06-30-2022
Funding Type: St. Baldrick's Scholar
Institution Location: Boston, MA
Institution: Dana-Farber Cancer Institute affiliated with Boston Children's Hospital, Harvard Medical School

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.

Katie Greenzang M.D.

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Funded: 10-01-2019 through 09-30-2023
Funding Type: St. Baldrick's Scholar
Institution Location: Boston, MA
Institution: Dana-Farber Cancer Institute affiliated with Boston Children's Hospital, Harvard Medical School

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.

Ramon Sun Ph.D.

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Funded: 08-01-2019 through 07-31-2022
Funding Type: St. Baldrick's Scholar
Institution Location: Lexington, KY
Institution: University of Kentucky Research Foundation affiliated with Kentucky Children's Hospital

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.

David Libich Ph.D.

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Funded: 07-01-2019 through 06-30-2023
Funding Type: St. Baldrick's Scholar
Institution Location: San Antonio, TX
Institution: University of Texas Health Science Center at San Antonio

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.

Lara Davis M.D.

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Funded: 07-01-2019 through 06-30-2022
Funding Type: St. Baldrick's Scholar
Institution Location: Portland, OR
Institution: Oregon Health and Science University affiliated with Doernbecher Children's Hospital

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.

Paulina Velasquez M.D.

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Funded: 07-01-2019 through 06-30-2024
Funding Type: St. Baldrick's Scholar
Institution Location: Memphis, TN
Institution: St. Jude Children's Research Hospital

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.

Emily Johnston M.D.

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Funded: 07-01-2019 through 06-30-2023
Funding Type: St. Baldrick's Scholar
Institution Location: Birmingham, AL
Institution: University of Alabama at Birmingham affiliated with Children's of Alabama

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.

Diana Moke M.D.

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Funded: 07-01-2019 through 12-31-2021
Funding Type: St. Baldrick's Scholar
Institution Location: Los Angeles, CA
Institution: Children's Hospital Los Angeles

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.

Wendy Bottinor M.D.

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Funded: 07-01-2019 through 06-30-2023
Funding Type: St. Baldrick's Scholar
Institution Location: Richmond, VA
Institution: Virginia Commonwealth University affiliated with Children's Hospital of Richmond at VCU

Surviving cancer is an achievement of immeasurable magnitude, however for most survivors this achievement does not signify the end of cancer related health issues. The cardiovascular system is commonly affected by cancer treatment and cardiovascular disease is the second leading cause of death in childhood cancer survivors. As the To-morrow's Research Fund St. Baldrick's Scholar, Dr. Bottinor is helping childhood cancer survivors live healthier lives by using advanced cardiac imaging techniques to identify survivors with subclinical cardiovascular dysfunction before they develop overt heart disease. She plans to use echocardiographic imaging to detect cardiovascular disease at its earliest stages, when treatment is most likely to be efficacious.

Dr. Bottinor is analyzing cardiac screening studies collected in routine care to determine if subclinical abnormalities can predict which survivors are at risk for subsequent cardiovascular disease and therefore the most likely to benefit from early medical intervention. She believes these techniques will be helpful in childhood cancer survivors because previous work in adult patients has suggested that advanced cardiac imaging techniques can predict which patients with cancer on active treatment are at higher risk for developing subsequent cardiovascular disease.

This grant is named for To-morrow's Research Fund, a Hero Fund created to honor Becky Morrow who is a childhood cancer survivor. Becky was diagnosed with acute lyphoblastic leukemia when she was 12 and endured grueling treatments and its side effects. Today she is cancer free, a wife and a mom but suffers late effects. This fund supports survivorship research for safer treatments that help kids not only survive but thrive.

Awarded at Vanderbilt University and transferred to Virginia Commonwealth University.

Lynn Lee M.D.

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Funded: 07-01-2019 through 06-30-2022
Funding Type: St. Baldrick's Scholar
Institution Location: Cincinnati, OH
Institution: Cincinnati Children's Hospital Medical Center affiliated with University of Cincinnati College of Medicine

There is a type of leukemia which occurs mainly in infants, but also in children who have previously received chemotherapy for an unrelated cancer. This type of leukemia is extremely difficult to treat and often comes back despite chemotherapy. We have evidence that this leukemia relies on a protein called MBNL1. By disrupting MBNL1, leukemia cells cannot produce specific proteins they need to grow. As the Julia's Legacy of Hope St. Baldrick's Scholar, Dr. Lee, as proof of concept, has shown that MBNL1 can be blocked by a chemical inhibitor. By using computer models, testing large libraries of drug-like chemicals, and applying special chemistry techniques to improve how effective a drug is, Dr. Lee is looking for a way to block MBNL1 with a drug that can be used in humans. This will lead to a new therapy for this class of leukemias.

A portion of this grant is named for Julia's Legacy of Hope, a 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.

Benjamin Stanton Ph.D.

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Funded: 07-01-2019 through 12-31-2024
Funding Type: St. Baldrick's Scholar
Institution Location: Columbus, OH
Institution: The Research Institute at Nationwide affiliated with Nationwide Children's Hospital

Based on progress to date, Dr. Stanton was awarded a new grant in 2022 and 2023 to fund an additional year of this Scholar grant.
Rhabdomyosarcoma (RMS) is a highly aggressive and lethal pediatric cancer affecting children and adolescents and arises in the soft tissue and skeletal muscle of the extremities, head and neck, and reproductive organs. From the clinical perspective, although patient outcomes have improved in general, nevertheless survival rates for some RMS tumors remains at less than 30%. One particularly aggressive subtype is alveolar RMS which is driven by the occurrence of chromosomal translocations resulting in the generation of chimeric or fusion proteins between the PAX3 or PAX7 and the FOXO1 genes. These are known as fusion-positive RMS (FP-RMS) and are associated with reduced relapse-free survival and generally poorer outcomes. But researchers still have limited understanding of how the "fusion" gene itself is driving the tumor, and no subtype-specific therapies exist. Dr. Stanton aims to determine how the fusion gene works with a protein complex known as BAF, to alter the epigenetic state of the cell to keep them dividing and stop the cell from differentiating into mature muscle tissue. His team is exploring the mechanism of how the BAF complex regulates the epigenetic state and memory of the FP-RMS. Furthermore, using small-molecule drugs and genetic depletion strategies (CRISPR) they will determine if FP-RMS tumors are dependent on the BAF complex for survival. Finally, they are working to identify potential novel therapies for patients with aggressive and lethal FP-RMS, to improve their outcome.

The 2021 and 2022 portions of this grant is funded by and named for the Aiden's Army Fund. When he was 8 years old, Aiden Binkley was diagnosed with Stage IV rhabdomyosarcoma. He had a huge tumor in his pelvis and the cancer had metastasized to his lungs. But this bright, funny and courageous boy believed he got cancer so he could grow up to find a cure for it. Aiden’s story has inspired so many people and his vision to cure cancer is being carried on by Aiden’s Army through the funding of research. They will march until there is a cure!

The 2020 and 2023 portions of this grant is funded by and named for by Berry Strong, a St. Baldrick’s Hero Fund, established in honor of Caroline Berry. Diagnosed with alveolar rhabdomyosarcoma when she was 14, Caroline endured a two-year battle with courage and determination. Throughout treatments of radiation and chemotherapy and undergoing six surgeries, Caroline was a beacon of hope, unselfishly raising awareness and funding for research so no child would have to endure what she did. After a brief remission, scans revealed Caroline had relapsed and she passed away on Thanksgiving in 2018. Caroline is remembered as a bright light, creative, intelligent, funny and feisty who was always eager to share a smile with others. She continues to be an inspiration through the Berry Strong Hero Fund which will continue her legacy and her passion to raise awareness and fund the most promising childhood cancer research.

Yangming Ou Ph.D.

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Funded: 07-01-2019 through 06-30-2025
Funding Type: St. Baldrick's Scholar
Institution Location: Boston, MA
Institution: Boston Children's Hospital affiliated with Dana-Farber Cancer Institute, Harvard Medical School

Based on progress to date, Dr. Ou was awarded a new grant in 2022 and 2023 to fund an additional year of this Scholar grant. Due to the tumor and treatment damaging the developing brain, 60-80% of pediatric brain tumor survivors experience long-term neurocognitive impairment. There are two possible paths to improve outcomes: intervene the adverse brain development after treatment, or further optimize radiotherapy dose distribution in the brain before treatment. For the former, the question is to find at-risk patients to intervene after treatment. For the latter, the question is to find target brain regions, where changing radiation doses can potentially change outcomes. Both questions have been studied on the population-level, not on the individual level. This project aims to push our knowledge in these two fronts to the individual level. Dr. Ou is using data from 3 just finished clinical trials to find target patients and find target brain regions for radiation dose optimization. Compared to studies that consider one risk factor a time, Dr. Ou will consider a comprehensive set of risk factors to improve precision to the individual level. The results will allow him to design future larger-scale, multi-site retrospective replicative and eventually prospective clinical trials, to improve neurocognitive outcomes in this vulnerable population before and after treatment.

This grant is generously supported by the Grace for Good Fund, established in honor of Grace Carey and celebrates her survivorship from medulloblastoma. Her cancer journey began in 2007 when she was diagnosed at age 5 with a treatment regime that entailed surgery, proton beam therapy and chemotherapy. While Grace handled it all with minor setbacks, she now faces the physical, emotional and cognitive challenges wrought by the very medications and procedures that saved her life. This fund was inspired by Grace’s desire to help other kids with cancer and supports research of brain tumors and the multitude of challenges facing survivors post treatment.

Saba Ghassemi Ph.D.

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Funded: 08-01-2018 through 10-31-2021
Funding Type: St. Baldrick's Scholar
Institution Location: Philadelphia, PA
Institution: University of Pennsylvania affiliated with The Children's Hospital of Philadelphia

Adoptive immunotherapy has demonstrated unprecedented clinical success in the treatment of leukemia. In this therapy, T cells are isolated from a patient, expanded outside of the body, and genetically modified prior to reinfusion. The ability of these T cells to recognize and eliminate cancer cells is improved by expressing a protein (CAR) on the T cell surface. This protein increases "specificity," the ability to recognize cancer cells, and "function," the ability to destroy those cancer cells. An important challenge in cellular immunotherapy is to minimize the manipulation of patients' T cells outside the body. Prolonged culture protocols trigger functional exhaustion and compromises their efficacy upon return to the body. A critical issue involves the pre-requisite "activation" step necessary for CAR expression on the cell surface of T cells. By optimizing culture conditions, Dr. Ghassemi developed approaches to express CAR in dormant T cells without the need for activation. Importantly, her preliminary findings show that elimination of this activation step retains effector function and potency of CAR T cells in models of the pediatric cancer ALL. This project is providing insight into the regulatory components influencing CAR expression in dormant T cells which will lead to superior CAR T cells for cellular immunotherapies against ALL.

This grant is supported by TEAM ABBY Gives, a St. Baldrick's Hero Fund. Abby was diagnosed with Pre-B ALL when she was almost five years old. She had a successful bone marrow transplant, but battle battled graft vs. host disease (GVHD) for years. Abby and her treatment team worked hard over many years to keep the GVHD in check. Sadly, Abby passed away on October 19, 2021. This fund unites the incredible support of family and friends in Abby's memory and inspires others to join the fight for cures and better treatments.

Masanori Hayashi M.D.

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Funded: 07-01-2018 through 06-30-2021
Funding Type: St. Baldrick's Scholar
Institution Location: Denver, CO
Institution: University of Colorado affiliated with Children's Hospital Colorado

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.

Robbie Majzner M.D.

Funded: 07-01-2018 through 06-30-2021
Funding Type: St. Baldrick's Scholar
Institution Location: Palo Alto, CA
Institution: Stanford University affiliated with Lucile Packard Children’s Hospital

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.

Michel DuPage Ph.D.

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Funded: 07-01-2018 through 06-30-2021
Funding Type: St. Baldrick's Scholar
Institution Location: Berkeley, CA
Institution: University of California, Berkeley

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.

Nickhill Bhakta M.D.

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Funded: 07-01-2018 through 06-30-2021
Funding Type: St. Baldrick's Scholar
Institution Location: Memphis, TN
Institution: St. Jude Children's Research Hospital

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.