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Showing 21-40 of 172 results
Christopher Kuo M.D.
Funded: 07-01-2023
through 06-30-2026
Funding Type: St. Baldrick's Fellow
Institution Location:
Los Angeles, CA
Institution: Children's Hospital Los Angeles
Based on the progress to date, Dr. Kuo was awarded a new grant in 2025 to fund an additional year of this Fellow grant.
Ewing sarcoma (EwS) is a malignant cancer of bone and soft tissues that occurs mainly in children, adolescents and young adults. If the tumors spread, fewer than 1/3 will survive. For some pediatric cancers, recent progress has led to new treatments that use one's own immune system to target cancer cells. However, immunotherapy has not been successful for EwS because we don't know enough about how EwS tumor cells evade the immune system. The tumor microenvironment (TME) is an intricate ecosystem consists of cancer cells and the host's immune system. Dr. Kuo's project will focus on dissecting the TME of EwS, to understand how tumors develop. Using EwS tumors removed from pediatric patients during their cancer diagnosis and treatment, Dr. Kuo will use newly-developed techniques to map the TME and use a genetic model of EwS developed at CHLA to examine tumor/immune cell interactions in living tissue. The long-term goal of this work is to identify new treatment options for children with EwS.
Years 1 and 2 of this grant are 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.
Year 3 of this grant is made possible through funding from D-Feet Cancer, The Dalton Fox Foundation and named in memory of Dalton, who is the inspiration for the Foundation that is lovingly led by his parents and sister. Dalton loved to tell good jokes and had a contagious sense of humor that lit up any room he walked into. Even on his worst days, he inspired his community and family with his positive outlook. He loved baseball, swimming, fishing and watching Marvel movies and while his time here was much too short, his memory continues to motivate so many. The Foundation is focused on fueling more research for Ewing sarcoma.
Robert Lindquist M.D., Ph.D.
Funded: 07-01-2023
through 06-30-2026
Funding Type: St. Baldrick's Fellow
Institution Location:
San Francisco, CA
Institution: University of California, San Francisco
affiliated with UCSF Benioff Children's Hospital
Based on the progress to date, Dr. Lindquist was awarded a new grant in 2025 to fund an additional year of this Fellow grant.
Children with the brain tumor ependymoma have high relapse rates and poor long-term survival. Treatment options for ependymoma are limited and there is no known effective chemotherapy. Dr. Lindquist is working to make a new model of this tumor, to study how the tumor forms and grows, and to test new therapies in this model and patient-derived tumors. The ultimate goal is to identify new therapies that will extend the lives of children with ependymoma.
Crystal Wang M.D.
Funded: 07-01-2023
through 06-30-2025
Funding Type: St. Baldrick's Fellow
Institution Location:
Palo Alto, CA
Institution: Stanford University
affiliated with Lucile Packard Children’s Hospital
Cancer cells compete with the body for food. Some cancer cells use fat to grow, spread, and hide in the brain. When cancer cells hide in the brain, it is hard for chemotherapy reach them due to the blood brain barrier, which allows cancers to come back when they come out of hiding. Dr. Wang and colleagues are investigating how childhood leukemia uses fat to survive in the brain and how drugs that starve leukemia of fat can kill leukemia cells hiding in the brain.
The second year of this grant is generously supported by Rhys’ Pieces of the Cure, a Hero Fund created to honor Rhys Goldman and his journey with cancer. He was diagnosed with pre-B acute lymphoblastic leukemia just 2 weeks before his 6th birthday and endured treatment for three years. Rhys missed a lot of school and life during those years but since marking the end of treatment in July 2018, he has been enjoying swimming, singing in a boys’ choir, chess tournaments, playing with his dogs and going to school. Rhys’ Pieces for the Cure was created to ensure more research is funded for the treatment of pediatric cancer that is specifically focused on less toxic cures for kids.
Marc Schwartz M.D., Ph.D.
Funded: 07-01-2022
through 06-30-2024
Funding Type: St. Baldrick's Fellow
Institution Location:
Boston, MA
Institution: Dana-Farber Cancer Institute
affiliated with Boston Children's Hospital, Harvard Medical School
One of the most exciting recent developments in cancer treatment is the growing ability to use the body's own immune system to directly fight tumors. However, these treatments still do not work on most patients, and we think it is critical to understand how each cancer type avoids the immune system. Dr. Schwartz is investigating how neuroblastoma, one of the most common pediatric solid tumors, escapes destruction by the immune system. To accomplish these goals, he will use cutting-edge technologies to dissect the immune biology in a model of neuroblastoma, with a particular focus on studying an important type of cancer-killing cell called a 'CD8 T cell'. Dr. Schwartz thinks him and his colleagues have identified an important new way that neuroblastoma evades these T cells. Their preliminary results suggest that neuroblastoma directly causes T cell death, limiting the ability of T cells to survive and kill enough tumor cells. He is trying to learn how neuroblastoma causes the death of T cells and find ways to block this immune evasion strategy. Most importantly, he predicts that combination treatment designed to block neuroblastoma's ability to kill T cells along with existing immune therapies will drastically improve the ability of the immune system to eradicate neuroblastoma.
A portion of this grant is funded by and named for the Oliver Wells Fund for Neuroblastoma, a St. Baldrick's Hero Fund. From the moment he was born, Ollie was the center of the Wells family with a contagious smile and a sparkle in his eyes. As the youngest child, it was devastating when they learned the 15 year old toddler had cancer. Oliver was diagnosed with high risk neuroblastoma and spent the next 13 months bravely enduring chemotherapy and radiation, more than a dozen surgeries and a bone marrow transplant. But Ollie persevered and smiled through it all. It was an unfair fight from the beginning and in July 2018, Ollie passed away. The Oliver Wells Fund for Neuroblastoma was established in his memory to raise funds to find cures and give hope to other kids facing the same fight. In this way, the Wells family intends to share Oliver’s joy for life and use his story to help find a cure.
Betsy Young M.D.
Funded: 07-01-2022
through 12-31-2025
Funding Type: St. Baldrick's Fellow
Institution Location:
San Francisco, CA
Institution: University of California, San Francisco
affiliated with UCSF Benioff Children's Hospital
Based on the progress to date, Dr. Young was awarded a new grant in 2024 to fund an additional year of this Fellow grant.
Osteosarcoma is a bone tumor that usually occurs in children and young adults and can be deadly especially when the tumor spreads to other body parts. The treatment strategy for this disease has not seen significant improvement in over 30 years, and there is no specific treatment for tumors that have spread throughout the body. In this project, the major goal is to identify factors that control the spread of osteosarcoma in order to develop new therapies to extend the lives of patients. Currently, Dr. Young is investigating whether osteosarcoma cells block the activation of one part of the patient's immune system, protecting the cancer cells from an immune attack and allowing them to spread throughout the body. This work has the potential to uncover new treatments to harness the immune system to fight this devastating disease.
This grant is named for the Team Jackson Hero Fund. The fund was established in honor Jackson Schmitt who died six days after his diagnosis with osteosarcoma from a stroke. Jackson’s story was told worldwide and his legacy lives on through funding life-saving osteosarcoma research.
Ruyan Rahnama M.D.
Funded: 07-01-2022
through 08-31-2025
Funding Type: St. Baldrick's Fellow
Institution Location:
San Francisco, CA
Institution: University of California, San Francisco
affiliated with UCSF Benioff Children's Hospital
Based on the progress to date, Dr. Rahnama was awarded a new grant in 2024 to fund an additional year of this Fellow grant.
Acute Myeloid Leukemia (AML) is a blood cancer that affects children. While there have been important advances in treatment and care of pediatric patients with AML, 20-40% relapse and have poor outcomes. Novel therapies are urgently needed to combat this disease. One treatment modality under investigation involves manipulation of the body's immune system by reprogramming immune cells with inherent anti-leukemia properties to specifically target AML cancer cells. Dr. Rahnama is focused on the study of natural killer (NK) cells as immune cells of interest. NK cells can be engineered to express Chimeric Antigen Receptors (CARs) that recognize specific proteins on leukemia cells in order to attack and kill them. The site where the CAR-modified NK cell and the target leukemia cell come together is known as the immunological synapse (IS). The IS is highly organized and plays a key role in activating the NK cell. Dr. Rahnama aims to better understand the interaction between CAR-modified NK cells and target leukemia cells by studying the biology of the IS as related to how tightly the two cells interact. Her goal is to improve CAR-NK cell design for ultimate use as pediatric AML treatment. This grant was awarded at Johns Hopkins University School of Medicine and transferred to the University of California, San Francisco.
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!
LaShanale Wallace Ph.D.
Funded: 01-01-2022
through 12-31-2024
Funding Type: St. Baldrick's Fellow
Institution Location:
Memphis, TN
Institution: St. Jude Children's Research Hospital
Myelodysplastic Syndrome (MDS) are clonal stem cell disorders characterized by abnormal cell growth and shape, lack of mature blood cells, and increased risk of acute myleoid leukemia (AML) development. Approximately 10,000 new cases are diagnosed every year in the United States. Survival ranges from months to years, and bone marrow transplantation remains the only cure. To identify new drug targets associated with more specificity and less morbidity and mortality, it is essential to understand the molecular course of MDS. Unbiased sequencing studies have identified over 45 recurrent somatic mutations in MDS patient samples. Of these pathways, splicing factor and epigenetic regulator mutations are the most common. Point mutations in splicing factor 3b subunit 1 (SF3B1) are found in less than 25% of MDS patients. Dr. Wallace and colleagues have shown that mutations in SF3B1 lead to an altered function, name upstream cryptic 3 splice site selection. Epigenetic regulators, including the de novo DNA methyltransferase, DNMT3A, are the second most common class of mutations identified in MDS. Studies on the effect of loss of DNMT3A expression have primarily been limited to the effects of altered methylation in regions outside of the gene body, such as enhancer accessibility. Although sequencing studies have shown that both splicing factor and epigenetic regulator mutations commonly co-occur as early mutations in MDS pathogenesis, it is unknown how altered DNA methylation and aberrant mRNA splicing can cooperate to promote MDS progression. Using cell line and animal model systems, Dr. Wallace will determine whether the cooperation of epigenetic regulator and splicing factor mutations lead to a more aggressive form of MDS. This grant is funded through a partnership between the St. Baldrick’s Foundation and the American Cancer Society.
Jessica Tsai M.D., Ph.D.
Funded: 07-01-2021
through 06-30-2023
Funding Type: St. Baldrick's Fellow
Institution Location:
Boston, MA
Institution: Dana-Farber Cancer Institute
affiliated with Boston Children's Hospital, Harvard Medical School
Diffuse intrinsic pontine glioma (DIPG) is a deadly pediatric brain cancer. Tragically, the majority of children diagnosed with the disease die within 12 months because the most effective treatment, radiation, is palliative at best. Therefore, there is a significant need to develop new therapeutic strategies to improve the terrible outcomes for these patients. Looking at genes that are turned on or off in a cancer can be helpful to figure out what is causing cancer growth. DIPGs are known to have mutations in a group of genes called histones that intriguingly regulate whether other genes in the cell are turned on or off. While looking at genes that are turned on or turned off in DIPG, Dr. Tsai found a gene called FOXR2 that is turned on in a subset of these tumors. FOXR is not usually present in the normal brain, but it has high levels in a subset of DIPGs. This is exciting because if researchers can target FOXR2 with new therapies, only tumor cells would be affected, sparing the normal cells in the brain. The goal of this project is to figure out exactly how FOXR2 makes DIPGs grow and to identify strategies that can be used in new treatments to target FOXR2.
A portion of this grant is generously supported by Griffin's Guardians, a St. Baldrick's partner. Griffin's Guardians was created by the Engles in memory of their son, Griffin. Their mission is to provide support and financial assistance to children battling cancer in Central New York, raise awareness about pediatric cancer and provide funding for research.
Lisa Niswander M.D., Ph.D.
Funded: 07-01-2021
through 06-30-2024
Funding Type: St. Baldrick's Fellow
Institution Location:
Philadelphia, PA
Institution: The Children's Hospital of Philadelphia
affiliated with University of Pennsylvania
Children with some kinds of blood cancers (leukemias) are not cured by regular chemotherapy and are at high risk of dying without better treatment options. Dr. Niswander is working to create new treatments that are more personalized for each child’s leukemia cells. The first treatment targets ‘miswired’ communication networks inside the leukemia cells that make them cancerous, and the second treatment uses the body’s own immune system to attack the leukemia cells. Each of these treatments is able to kill a patient’s cancer cells. But, eventually the leukemia cells develop changes that allow them to begin growing again despite the therapy, and the cancer comes back. These two therapies have never been combined together in patients. In this project, Dr. Niswander and colleagues are studying the best ways to combine these new treatments for two kinds of high-risk pediatric leukemias, since often two treatments that work in different ways are better than one. She is hopeful that by using patients’ own leukemia cells they will identify the best personalized treatments for future testing in pediatric patients to improve their chances of cure and living long and healthy lives.
For 2022, this grant is named for the Invictus Fund, a St. Baldrick’s Hero Fund created in memory of Holden Gilkinson. It honors Holden's unconquerable spirit in his battle with bilateral Wilms tumor by funding cures and treatments to mitigate side and late effects of childhood cancer.
In 2021, this grant was generously supported by Super Soph's Pediatric Cancer Research Fund, a St. Baldrick's Hero Fund. Sophie Rossi was diagnosed with AML at 3 months of age. Throughout her courageous battle, she was always smiling, always joyful. This fund was created to honor her spunky, sweet spirit by funding research to find cures for AML and all childhood cancers.
Matthew Kudek M.D.
Funded: 07-01-2021
through 06-30-2023
Funding Type: St. Baldrick's Fellow
Institution Location:
Milwaukee, WI
Institution: Children's Hospital of Wisconsin
affiliated with Medical College of Wisconsin, Midwest Children's Cancer Center
Neuroblastoma is one of the most common pediatric tumors, responsible for 12% of all cancer deaths in children under 15 years old. Only about 50% of patients with widespread neuroblastoma will live for ten years after diagnosis. A recent breakthrough in cancer treatments known as CAR T cell therapy reprograms a patient’s own immune cells to recognize tumor cells. While CAR T cell monotherapy works for some cancer types, several research studies using CAR T cells to treat neuroblastoma have been relatively unsuccessful. This is likely due to immune suppression caused by the tumor itself. Interestingly, it is known that if a person with cancer develops an infection, the infection can stimulate an immune response that will promote cancer remission. With this knowledge, Dr. Kudek and colleagues have pioneered an innovative technique to boost CAR T cell therapy response. They have shown that the cancer-destroying function of reprogrammed immune cells is boosted when a weakened infection is introduced into a tumor and found that this treatment combination in bladder cancer led to cure in most of the disease models. Encouraged by these findings, he is pursuing proof-of-principle studies to determine how this treatment approach can be best applied to neuroblastoma treatment.
This grant is named for the LukeStrong a Force Against Neuroblastoma Childhood Cancer Fund. When Luke was 5 years old, he was diagnosed with high-risk neuroblastoma. He is now in his teens and still in active treatment for relapsed neuroblastoma. Since 2014 Luke’s “Never tell me the odds” attitude has inspired his family and friends to shave their heads, fundraise with St. Baldrick’s, and help conquer childhood cancers.
Jeremy Rubinstein M.D., Ph.D.
Funded: 07-01-2020
through 06-30-2022
Funding Type: St. Baldrick's Fellow
Institution Location:
Cincinnati, OH
Institution: Cincinnati Children's Hospital Medical Center
affiliated with University of Cincinnati College of Medicine
Bone marrow transplantation is a highly effective treatment for relapsed and difficult to treat forms of pediatric leukemia, but unfortunately has a high risk for dangerous side effects. Viral infections are a major problem in the weeks and months after bone marrow transplant while children's immune systems are still immature. These infections can be debilitating and even deadly while also being very difficult to treat since available antiviral medications frequently do not work. Over the last few years, researchers have had great success in combating these viral infections by taking T-cells (a type of infection fighting cell that is part of the immune system) donated by children's personalized stem cell donors and engineering them to attack and kill certain viruses. Additionally, the rates of side effects using this therapy have been incredibly low. Dr. Rubinstein now intends to offer this therapy as a preventative measure, with the hope that this strategy will decrease the number of patients suffering from dangerous viral infections after bone marrow transplant. This clinical trial has the potential to decrease the number of pediatric cancer survivors who die from infection while also shortening hospitalizations and decreasing the need for other anti-viral medications.
This grant is generously supported by the Rally for Ryan Fund, a St. Baldrick's Hero Fund. Ryan was diagnosed with ALL when he was 7 years old and began treatment immediately. Initially labeled “high risk” due to a poor response, he completed 3½ years of a difficult treatment protocol before relapsing 11 months later. After his third relapse and an unsuccessful immunotherapy trial, Ryan had a bone marrow transplant in December 2020. He is currently fighting graft vs. host disease but is doing well and is optimistic for a good response. The Campanaros created this Hero Fund to celebrate Ryan’s courageous spirit and knowing firsthand the importance of research, to raise funds to find better treatments for kids with cancer.
Zachary Reitman M.D., Ph.D.
Funded: 07-01-2020
through 06-30-2023
Funding Type: St. Baldrick's Fellow
Institution Location:
Durham, NC
Institution: Duke University Medical Center
affiliated with Duke Children's Hospital & Health Center
Based on progress to date, Dr. Reitman was awarded a new grant in 2022 to fund an additional year of this Fellow award. Brainstem gliomas are deadly brain tumors that affect children. The only effective treatment is radiation therapy, but despite this treatment all children with this disease eventually experience growth of the tumor and eventually death. As the Emily Beazley's Kures for Kids Fund St. Baldrick's Fellow, Dr. Reitman will test if treatments that enhance the efficacy of radiation therapy can improve survival in the laboratory. This could lead to new clinical trials aimed at helping children with brainstem gliomas to survive longer.
This grant is funded by and named for Emily Beazley's Kures for Kids 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.
Lei Peng M.D.
Funded: 07-01-2020
through 06-30-2022
Funding Type: St. Baldrick's Fellow
Institution Location:
Baltimore, MD
Institution: Johns Hopkins University School of Medicine
affiliated with Johns Hopkins Children's Center
Over-expression of HOXA9 protein in acute leukemias, which are cancers of the blood, is associated with worse outcomes. This over-expression occurs in more than 50% of acute myeloid leukemia (AML) cases and in approximately 75% of infant acute lymphoblastic leukemia (ALL) cases. In the laboratory setting, decreasing the level of HOXA9 in AML cells has been shown to reduce their growth. This project aims to develop a way to target HOXA9 in AML and infant ALL using short segments of DNA called oligonucleotides designed to decrease HOXA9 protein or prevent its function. The use of oligonucleotides as drugs has recently been successful in the treatment of various disorders. The goal of these studies is to eventually lead to the use of oligonucleotides as novel therapeutic agents in a clinical trial setting for treatment of AML and infant ALL.
Anya Levinson M.D.
Funded: 07-01-2020
through 06-30-2022
Funding Type: St. Baldrick's Fellow
Institution Location:
San Francisco, CA
Institution: University of California, San Francisco
affiliated with UCSF Benioff Children's Hospital
Leukemia is the most common form of childhood cancer. While most children with leukemia can be cured, patients whose leukemia comes back after an initial response to therapy are very difficult to treat and often die of their disease. As the Ty Louis Campbell Foundation St. Baldrick's Fellow, Dr. Levinson studies one of the classes of medicines used to treat leukemia called "glucocorticoids" (a type of steroid), in a type of leukemia called T-cell ALL. Though glucocorticoids are usually very good at killing leukemia cells, some patients have been found to not respond (or be "resistant") to glucocorticoids, while others develop resistance over time, making their disease far more difficult to treat. Dr. Levinson's research is focused on understanding how and why such resistance develops in an effort to identify ways to overcome it and, ultimately, increase the percentage of children with T-cell ALL who can survive their disease.
This grant is funded by and named for the Ty Louis Campbell Foundation, a St. Baldrick's partner, created in memory of Ty Louis Campbell who lost his battle with brain cancer at the age of five. The Foundation seeks less toxic, more effective treatments that are specifically designed for children fighting cancer. Their ultimate mission is to help fund the intelligence and technology that will uncover new ways to cure children with cancer.
Stephanie Dixon M.D.
Funded: 07-01-2020
through 06-30-2022
Funding Type: St. Baldrick's Fellow
Institution Location:
Memphis, TN
Institution: St. Jude Children's Research Hospital
Most children diagnosed with cancer today will survive but will develop late complications of their cancer treatment. Childhood cancer survivors have almost twice the risk of diabetes compared to other adults. Diabetes is known to increase the risk of heart disease among survivors, and heart disease is the leading cause of non-cancer death among survivors. Prediabetes is easily diagnosed and begins months to years before diabetes. However, little is known about prediabetes risk-factors and prevention in survivors, despite reports that up to 1 in 3 survivors have prediabetes. Using treatment information and recent assessment of over 3,500 adult survivors of childhood cancer, this research will identify the extent of prediabetes among survivors, characterize what cancer-treatments increase risk, and determine how quickly these survivors develop diabetes. Research will then establish if a medication and lifestyle intervention to prevent diabetes in prediabetic survivors is safe and achievable. This will inform a future diabetes intervention trial with the goal of improving long-term survival and quality of life for childhood cancer survivors.
Shannon Conneely M.D.
Funded: 07-01-2020
through 06-30-2023
Funding Type: St. Baldrick's Fellow
Institution Location:
Houston, TX
Institution: Baylor College of Medicine
affiliated with Vannie E. Cook Jr. Children's Cancer and Hematology Clinic, Texas Children's Hospital
Based on progress to date, Dr. Conneely was awarded a new grant in 2022 to fund an additional year of this Fellow award. Acute myeloid leukemia (AML) is the second most common blood cancer in children and is difficult to cure. About one quarter of children with AML have a form of the disease called core binding factor (CBF) AML. Despite intense therapy, cancer will come back in one out of three children with CBF-AML. We want to find new ways to treat this common form of AML by learning how the specific combination of mutations in the cancer cells affect their ability to grow and survive. Some patients with CBF-AML have unique mutations that can stop cells from correctly fixing damage, allowing them to grow too quickly. The project will study how these mutations contribute to CBF-AML cells' development, growth, and survival, affecting the cancer cells' ability to grow using cancer cells with these unique mutations. This will help in understanding how this type of AML develops, and may lead to new ways to treat children with this disease.
This grant is generously supported by Double Deckers Destroy AML, a St. Baldrick's Hero Fund. Joel and Seth were not only identical twins but best friends. In an ironic twist of fate, both boys were diagnosed with Acute Myeloid Leukemia just three months apart. With the overlapping diagnoses and treatments, the family was separated for months at a time and looked forward to days when they could be together at home. Joel and Seth both received bone marrow transplants and endured complications from the procedures. Sadly, both boys relapsed. Surrounded by their loving family, Joel died in November 2017 at the age of three, followed by Seth in May, 2019 when he was four years old. The twins were named as 2020 Ambassadors for St. Baldrick's so their story can continue to inspire many. The Double Deckers Destroy AML Hero Fund was established because the Decker family strongly believes more research is needed for AML, especially when the disease has relapsed. They want to support research so other families won’t have to say goodbye too soon.
Erica Braverman M.D.
Funded: 07-01-2020
through 03-31-2023
Funding Type: St. Baldrick's Fellow
Institution Location:
Pittsburgh, PA
Institution: Children's Hospital of Pittsburgh
affiliated with University of Pittsburgh
There are new cancer therapies in which a patient's own immune system is retrained to fight against their cancer. In one of these therapies, known as CAR-T cells, a patient's immune cells are removed from the bloodstream and reprogrammed to target and attack their cancer when the cells are returned to the body. While this therapy has shown great promise, there are still situations, especially with very high-risk cancers, where it does not work. One significant issue that exists with this treatment is that the retrained immune cells do not always stick around after being given back to the patient, which allows the cancer to outlast the therapy and come back. We know that once cancers have resisted a treatment once, it is difficult to use the same treatment again. This projects aims to find ways to alter tumor-targeting immune cells to make them last longer when they are given back to patients, ultimately allowing for a long-term cure for their cancer without the need for further treatment.
This grant is generously supported by the TeamConnor Childhood Cancer Foundation. TeamConnor Childhood Cancer Foundation's mission is to raise funds for national childhood cancer research programs, to build awareness that only a fraction of the NIH’s annual funding supports childhood cancer research, and to support inpatient programs. Founded in 2008 in honor and memory of Connor Cruse, TeamConnor has funded over $4M in pediatric cancer research grants across the United States.
Angela Steineck M.D.
Funded: 07-01-2019
through 06-30-2023
Funding Type: St. Baldrick's Fellow
Institution Location:
Milwaukee, WI
Institution: Medical College of Wisconsin
affiliated with Midwest Children's Cancer Center, Children's Hospital of Wisconsin
Clinical trials have been essential to the great progress that has been made toward curing childhood cancer. New personalized therapies in current clinical trials promise to be more effective and less toxic than drugs in the past. But, to truly understand what a child finds most bothersome and provide the best quality of life possible, we need to ask the child directly. No one has done this for children receiving personalized therapies for cancer that has returned or not responded to chemotherapy, a group where quality of life is especially important. To answer this question, a team from Seattle Childrens Hospital/University of Washington and Boston Childrens Hospital/Dana Farber Research Institute designed this study. Dr. Steineck and colleagues are using surveys, especially made for children, to learn what children feel when they are treated on a clinical trial and what bothers them the most. This will help doctors find better ways to recognize and treat these symptoms, alleviate suffering, and improve how children view their quality of life. Knowing this is important for families to understand what their child may experience with treatments used on todays clinical trials and guide them in their very important, but difficult decision about the care their child receives.
This grant is funded by and named for Friends for Hope, a St. Baldrick's Hero Fund created to honor Morgan Loudon and celebrate her strength and determination as a cancer survivor. Morgan was 9 years old when she was diagnosed with a rare rhabdoid tumor and today she has no evidence of disease. With this fund, the Loudon family hopes to rally family and friends to “battle on” in the search for cures and better treatments.
This grant was awarded at Seattle Children's and transferred to Medical College of Wisconsin.
Micah Maxwell M.D., Ph.D.
Funded: 07-01-2019
through 09-09-2021
Funding Type: St. Baldrick's Fellow
Institution Location:
Baltimore, MD
Institution: Johns Hopkins University School of Medicine
affiliated with Johns Hopkins Children's Center
Neuroblastoma is a common solid tumor in children, accounting for 1 in 10 new cancer diagnoses. Approximately half of the children with the high-risk form of the disease will die, and the survivors will bear a lifelong burden from the intensity of therapy. We are desperately in need of novel treatment approaches. The most aggressive neuroblastomas have extra copies of a gene called MYCN, which causes neuroblastoma cells to have different metabolism from normal cells. As the Mighty Micah's Mission Fund St. Baldrick's Fellow, Dr. Maxwell is investigating the abnormal metabolism of neuroblastoma in order to uncover new potential therapies. He has found that the amino acid, asparagine, is critical to the growth and survival of neuroblastoma, and has identified two medications (called DON and asparaginase) that, when combined, reduce the levels of this critical nutrient and effectively kill the most aggressive neuroblastomas. This work could serve as the basis for new clinical trials with this drug combination in children with neuroblastoma. Dr. Maxwell aims to exploit neuroblastoma's metabolic Achilles' heel in order to improve outcomes for children who suffer from this devastating disease. This approach holds great promise for future targeted therapies to treat not only neuroblastoma, but many other cancers that rely on abnormal metabolism.
This grant is named for Mighty Micah's Mission Fund, a St. Baldrick's Hero Fund. Diagnosed when he was 15 months old with high risk neuroblastoma, Micah was in treatment for nearly 7 years and survived two relapses. Thanks to research supported by St. Baldrick’s and the development of a new drug that is less toxic and more effective, Micah has no evidence of disease today. He has been named a 2020 Ambassador for St. Baldrick’s and as a science fan who hopes to become a doctor one day, Micah is grateful to the researchers who strive to find cures: “Those medicines save kids’ lives and one of them saved mine.” This fund honors Micah’s cancer journey and supports neuroblastoma research to find better treatments and cures for kids with this disease.
Lisa Force M.D.
Funded: 07-01-2019
through 12-30-2021
Funding Type: St. Baldrick's Fellow
Institution Location:
Memphis, TN
Institution: St. Jude Children's Research Hospital
Children and adolescents everywhere in the world get cancer and both the type of cancer, and perhaps more importantly, where they live in the world, factor into whether they live or die. This is due to major disparities between countries in access to optimal treatment, early abandonment of therapy despite the potential for cure, and availability of quality supportive care. Acute lymphoblastic leukemia (ALL), the most common childhood cancer, is mostly curable in countries with strong health systems, like the United States. However, we do not know the exact number of children and adolescents who develop and die from ALL worldwide, because many countries with limited resources also lack quality health registration systems. Identification of context-appropriate strategies to prevent future deaths in children with ALL are necessary, and when combined with improved burden estimates, can guide policy decisions more effectively. Knowing that the majority of countries in the world have limited resources, this project will determine what the best interventions are to improve outcomes for children and adolescents with ALL now, while testing ways to improve estimates of the number of children with ALL who are currently not correctly diagnosed or do not reach healthcare. Awarded at St. Jude Children's Research Hospital and transferred to University of Washington.