Grants Search Results

Rhabdomyosarcoma (RMS) is the most common malignant soft tissue tumor in childhood. Despite intensification of aggressive therapy involving combination chemotherapy, radiation and surgery, the overall outcome of RMS is among the least improved in childhood cancer. Dr. Huang and colleagues aim to explore a novel concept of applying a clinical available technique of tumor-reduction cryoablation, whereby tumors are damaged by ultra-cold argon gas or liquid nitrogen to release endogenous immune adjuvants, to enhance an efficacious systemic anti-tumor immunity against distant RMS metastasis. He seeks to procure preclinical efficacy and mechanistic data that will enable a rapid translational clinical trial targeting metastatic sarcoma within 3 years.

Alveolar rhabdomyosarcoma is one of the most aggressive and difficult to treat tumors in children. If not caught early, metastatic disease has a dismal 5-year survival of less than 5%, even after the most intensive chemotherapy possible. Even in the rare circumstances when these children do well, the long-term side effects of the intensive chemotherapy are debilitating. We can, and must, do better. We have known for some time that the cause of alveolar rhabdomyosarcoma in 60% of the most aggressive cases is a specific genetic abnormality. This genetic mistake creates a new gene, and Dr. Hiebert will determine how this new gene causes cancer and determine what would happen to these sarcoma cells if we had a drug specific for this new gene. To do this, he has engineered alveolar sarcoma cells grown in the lab so that this cancer gene can be quickly turned off by an existing drug. This allows, for the first time, the treatment of these sarcoma cells with a specific drug to define all of the events that occur in the first few minutes to several days of drug treatment to establish that inhibition of this new cancer gene is a viable therapeutic strategy.

This grant is generously supported by Rachael Chaffin’s Research Fund, a Hero Fund created in memory of a young girl who loved life. Rachael loved people, animals and the outdoors. It was heartbreaking when she was diagnosed with Rhabdomyosarcoma in the summer of 2013 at the age of 11. With a positive attitude and determination, Rachael began her long battle with cancer. She truly believed she would beat cancer so she could go on to help others. In 2014, Rachael organized a team of family and friends called “Kicking Cancer with Ray Ray” to raise funds for St. Baldrick’s and they continue the tradition today. This Hero Fund honors Rachael’s passion to find a cure for kids’ cancer and carries on her legacy of increasing awareness of childhood cancer to find better treatment options and cures through research.

For a child is diagnosed with a diffuse intrinsic pontine glioma so called DIPG, the options for treatment are scarce and so are the chances for survival. This aggressive brain tumor generally strikes children who are 6 years old and younger, with most surviving less than a year after diagnosis. The only known effective treatment is the use of radiation. Yet, even with radiation therapy most children show tumor progression within the year after radiation therapy. Given this reality, there is a desperately need to identify the drug that increase the anti-tumor activity of radiation, as a mean to improve treatment outcome for these children. DNA damage is thought to be the most toxic effect caused by radiation, and Dr. Hashizume and others showed that the majority of the DNA damage caused by radiation are repaired within 24 hours of treatment. This DNA damage repair is possibly responsible for the tumor progression observed in DIPG after radiation therapy, thereby ultimately taking no survival benefits to the patients. As the recipient of the Just Do It...and be done with it St. Baldrick's Research Grant, Dr. Hashizume recently performed a genetic screening in DIPG cells collected from patient tumor and found specific therapeutic targets which is important for DNA damage repair. This research will study whether targeted inhibition of DNA damage repair increase DNA damage by radiation, leading to increased radiation toxicity in DIPG. Successful results from this research will find a new effective therapy which increases the anti-tumor activity of radiation, in turn, will ultimately leads to improved treatment outcomes for children with highly malignant and currently incurable cancer.

This grant is funded by and named for the “Just Do It…and be done with it” Hero Fund created in honor of Sara Martorano who was 4 when she was diagnosed with Stage IV Wilms tumor. Despite a grueling treatment protocol of surgeries, radiation and chemotherapy, Sara didn’t let anything dim her sparkle. Thanks to life-saving research, today she is cancer free. This fund celebrates the courage of all cancer kids enduring treatment and the support of their family and friends.

Awarded at Northwestern University and transferred to Ann & Robert H. Lurie Children's Hospital of Chicago.

Children with acute lymphoblastic leukemia (ALL), a cancer of the blood, who receive modern therapy have a high likelihood of being cured. A side effect of treatment in some survivors of ALL is lifelong troubles with learning and memory. One part of therapy that may increase the chance of having learning and memory problems is many exposures to anesthesia. Treatment for ALL includes painful procedures for which children routinely receive anesthesia. Understanding the connections between specific anesthesia medicines, their doses, and the length of time they are given and the chances of having learning problems later on will be important for patients, families and clinical teams in making the best choices for anesthesia use.

As the recipient of the Jack's Pack - We Still Have His Back St. Baldrick's Research Grant, Dr. Eischen is focused on researching Burkitt lymphoma, a blood cancer that predominately develops in children and young adults. The goal of this proposal is to investigate a novel approach to eliminate Burkitt lymphoma cells, and particularly difficult to treat relapsed and refractory to treatment Burkitt lymphoma. Although five-year survival rates for Burkitt lymphoma is 85-90%, treatment is toxic with associated complications, and children that relapse or that are resistant to treatment have poor survival rates even with additional therapy. Therefore, more research and new treatments are needed for Burkitt lymphoma. This project stems from a paradigm-shifting discovery she recently made and will use an innovative approach that includes testing newly designed compounds to target a specific protein called Mdm2 in Burkitt lymphoma cells causing their death. This approach should also cause the death of Burkitt lymphoma cells that contain mutations in a gene that make them resistant to many current therapies and that reduces patient survival. Completion of the research will result in increased understanding of the role of Mdm2 in human Burkitt lymphoma cell survival, testing of new compounds that target Mdm2, and pre-clinical tests with the compounds on human Burkitt lymphoma cells. The long-term goal of these studies is to have an improved, more effective treatment approach for non-Hodgkin's lymphomas, and particularly those lymphomas that are resistant to current therapies.

This grant is funded by and named for Jack's Pack - We Still Have His Back, a St. Baldrick's Hero Fund. Jack Klein was a ten year old who loved life, laughing and monkeys. During his illness, his community of family and friends near and far rallied around him under the moniker "Jack's Pack". Their slogan was "We have Jack's Back". After Jack succumbed to Burkitt's Lymphoma, his "pack" focused their energy and efforts to funding a cure...just as Jack would have wanted.

The human immune system is astonishing in its ability to eliminate cells and organisms that give rise to disease. This process of immune surveillance is one of the last lines of defense that protect both adults and children from cancer; however, researchers have found that dysfunctional immune responses can permit cancerous leukemia cells to grow uncontrollably in the body. In this work, Dr. Dreaden will improve upon a drug that attempts to restore immune elimination to leukemia by redirecting a subset of immune cells, so-called T cells, to bind with and kill cancerous cells. By tethering such drugs with molecules that stimulate T cells to multiply, and possibly enable these cells to recognize leukemia cells again at a much later date, he aims to further improve both the strength and durability of responses to this promising class of immuno-therapy. Already, Dr. Dreaden and colleagues have made and screened more than 45 of these unique, multi-functional therapies and aim here to study the precise mechanics by these drugs act on immune cells, as well as their ability to impart memory-like immune responses to leukemia. Given the modular nature of this treatment approach, it could be rapidly extended to a range of other cancer cells, immune cells, and immune stimulating factors in the future.

Precise understanding of the basic mechanisms by which childhood cancers develop is essential to design tailored and superior treatments for cancer patients. These treatments are expected to cure and avoid long-term complications in cancer survivors. In many instances, we turn to models to reproduce human cancers, but the success of this strategy depends on how accurately we can unravel the origin of the disease. This project is based on Dr. Dominguez-Sola and colleagues recent findings on the origins and cellular basis of Burkitt lymphoma, a most aggressive form of childhood lymphoma with little treatment alternatives. This project will use unprecedented models of this cancer type to expand our understanding of the mechanisms of disease and identify therapeutic strategies that are less toxic, more effective, and superior to those currently available in the clinic.

Pediatric cancers are often comprised of mixtures of cells with different characteristics. Some of the most important differences relate to chromosomal changes, with some cells having a normal or nearly normal chromosome profile, others having altered numbers of intact chromosomes, and yet others having extra or missing copies of one or more chromosome segments. Prior studies have shown that cancers with more segmental changes are usually more aggressive and therapy-resistant, but the specific effects associated with the different chromosomal changes are unknown. Here Dr. Cobrinik and colleagues will define the effects of such changes in two pediatric cancers -- retinoblastoma and neuroblastoma -- by isolating individual cells within the tumors that either have or lack specific chromosome changes, comparing their overall gene expression and cell signaling profiles, and identifying the critical changes that increase malignancy. The study involves three investigators with expertise in neuroblastoma, retinoblastoma, and a novel single cell sequencing approach that enables us to distinguish and characterize the chromosomally distinct cells within individual tumors in unmatched detail. This study is expected to reveal the most central features that distinguish more versus less aggressive cancers, as a critical step towards targeting and subduing the more aggressive and lethal cells within individual tumors.

Acute lymphoblastic leukemia (ALL) is the most common cancer of children, and although treatment is considered largely successful, in many cases leukemic cells stop responding to chemotherapy and re-emerge. As a consequence, ALL relapse remains a leading cause of childhood cancer-related death. Dr. Aifantis will test the possibility that the bone marrow microenvironment surrounding the leukemia supports the growth of disease and protects leukemia cells from chemotherapy. Together with colleagues he generated the first map of the ALL immune cell microenvironment allowing identification of novel players within the remodeled leukemic bone marrow that promote leukemia survival. They found that high levels of a specific cell type, known as non-classical monocytes, in ALL patient blood and bone marrow correlates with inferior patient survival. They demonstrated that depletion of leukemia-supporting monocytes enhances killing of leukemic cells with specific ALL therapies. In this project Dr. Aifantis will investigate the processes giving rise to monocytes capable of supporting leukemia survival. Further, he will use novel model systems to test whether targeting monocytes enhances responses to a range of existing ALL therapies as well as emerging approaches, such as Chimeric Antigen Receptor (CAR) T-cell therapy, that utilize a patient's own immune system to kill leukemic cells.

Based on progress to date, Dr. Das was awarded a new grant in 2022 to fund an additional year of this International Scholar grant. When a cell divides, the DNA duplicates. However there may be errors in this process. Most are corrected by an in-built replication repair mechanism. If not corrected, this may lead to mutations. The repair mechanism itself may be faulty in some children with an inherited condition. They develop cancers in the brain, intestines and blood, with very high number of mutations. These cancers are difficult to diagnose and do not respond to standard chemotherapy and radiation. Dr. Das and colleagues have developed cutting edge yet inexpensive genomic tool, called 'signatures' which will help better diagnose this disease. The tool will also predict which children will benefit from a new, promising treatment known as immunotherapy. It will also help diagnose other family members before they develop cancers and initiate surveillance to improve their chances for survival. The condition is more prevalent in the developing world where the custom of marrying within one's community is prevalent. Hence validation of the utility of this tool and developing local capacity to use this will benefit large number of children and their families in underserved areas across the globe.

The 2022 portion 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 Team Campbell Foundation. The Foundation was established in memory of Campbell Hoyt, who courageously battled anaplastic ependymoma, a rare cancer of the brain and spine, for five years before passing away in August of 2014 at the age of eight. Its mission is to improve the lives of families facing a childhood cancer diagnosis through raising awareness, funding research and providing psycho-social enrichment opportunities.

Based on progress to date, Dr. Das was awarded a new grant in 2022 to fund an additional year of this International Scholar grant. When a cell divides, the DNA duplicates. However there may be errors in this process. Most are corrected by an in-built replication repair mechanism. If not corrected, this may lead to mutations. The repair mechanism itself may be faulty in some children with an inherited condition. They develop cancers in the brain, intestines and blood, with very high number of mutations. These cancers are difficult to diagnose and do not respond to standard chemotherapy and radiation. Dr. Das and colleagues have developed cutting edge yet inexpensive genomic tool, called 'signatures' which will help better diagnose this disease. The tool will also predict which children will benefit from a new, promising treatment known as immunotherapy. It will also help diagnose other family members before they develop cancers and initiate surveillance to improve their chances for survival. The condition is more prevalent in the developing world where the custom of marrying within one's community is prevalent. Hence validation of the utility of this tool and developing local capacity to use this will benefit large number of children and their families in underserved areas across the globe.

The 2022 portion 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 Team Campbell Foundation. The Foundation was established in memory of Campbell Hoyt, who courageously battled anaplastic ependymoma, a rare cancer of the brain and spine, for five years before passing away in August of 2014 at the age of eight. Its mission is to improve the lives of families facing a childhood cancer diagnosis through raising awareness, funding research and providing psycho-social enrichment opportunities.

This institution is a member of a research consortium which is being funded by St. Baldrick's: Consortium for Childhood Cancer Predisposition. For a description of this project, see the consortium grant made to the lead institution: Emory University, Atlanta, GA.

This institution is a member of a research consortium which is being funded by St. Baldrick's: Consortium for Childhood Cancer Predisposition. For a description of this project, see the consortium grant made to the lead institution: Emory University, Atlanta, GA.

This institution is a member of a research consortium which is being funded by St. Baldrick's: Consortium for Childhood Cancer Predisposition. For a description of this project, see the consortium grant made to the lead institution: Emory University, Atlanta, GA.

This institution is a member of a research consortium which is being funded by St. Baldrick's: Consortium for Childhood Cancer Predisposition. For a description of this project, see the consortium grant made to the lead institution: Emory University, Atlanta, GA.

This institution is a member of a research consortium which is being funded by St. Baldrick's: Consortium for Childhood Cancer Predisposition. For a description of this project, see the consortium grant made to the lead institution: Emory University, Atlanta, GA.

Cancer remains the leading cause of illness-related death in children. Researchers now know that inherited genetic abnormalities contribute to cause cancer in many more children than previously understood. Children with Cancer Predisposition Syndromes (CPS) have a high risk of cancer, and we have a unique opportunity to prevent or detect cancer at early stages to improve their survival, as well as to better understand mechanisms of cancer development for all pediatric tumors. However, these CPS are rare and difficult to study in individual institutions. Thus, the Consortium for Childhood Cancer Predisposition was created, through which researchers aim to better understand the tumor risks in these patients and their distinct biology. Also, they will study surveillance and prevention strategies efficiently, by utilizing coordinated efforts to collect data and samples from patients with CPS (through a registry and biorepository). The Consortium currently comprises seven institutions, is led by experts who have already collaborated to have major impacts in this field, and has engaged syndrome specific advocacy groups to accelerate patient-centered advances. The impact of this Consortium will be improved outcomes for children at highest risk of cancer through better identification, early tumor detection, optimized psychosocial support and cancer prevention. Funds administered by Emory University.

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.

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.

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.