Grants Search Results

This grant funds a student to complete work in pediatric oncology research for the summer. It is named for the St. Baldrick's Foundation staff whose generous gifts have helped fund this opportunity and may encourage them to choose childhood cancer research as a specialty.

With increased survival for children with cancer, efforts that prevent long-term health problems are important for improving the quality of life and life expectancy of these children. Diet and fitness are two critical factors for healthy survivorship, but interventions for survivors of childhood cancer have had limited impact, focus almost exclusively on physical activity, and often exclude caregivers, the primary nutrition gatekeepers in the home. Although research supports a key role for the gastrointestinal (GI) microbiome in regulating weight and health outcomes, no studies have examined the “obesogenic” microbiome in the context of interventions for these survivors. Harvesting Hope for Kids (HH4K) is a unique, biobehavioral lifestyle intervention delivered over 8 weeks during the summer in a university-based, cancer survivor garden. It was adapted from a successful intervention for survivors of adult-onset cancer, with pilot data supporting its feasibility in children. In line with St. Baldrick’s mission to improve outcomes for children with cancer, this randomized controlled trial is evaluating the efficacy of HH4K to improve dietary and physical activity patterns in 40 survivors of pediatric cancer (i.e., ages 8-12; < 2 years off treatment). Results will support a larger, multi-institutional trial and improve survivorship care to prevent costly, long-term morbidity.

Some childhood cancers do not respond to chemotherapy, surgery, or radiation. For these patients, researchers are developing a new set of treatments that use their own immune system to attack the cancer. To turn on these defenses, they need to bolster the DNA in 200 million immune cells, efficiently and safely. Unlike other strategies, these cells do not need to come from the patients, who are already weakened. Dr. Weiss has invented an engineering solution to do so and is testing it so that he can make this treatment widely available to patients and their doctors soon.

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.

Sarcomas are tumors of the bone and soft tissues that comprise up to 20% of cancer diagnoses in children. Despite dismal outcomes for patients with recurrent or metastatic disease, treatment regimens have remained largely unchanged for decades – intense non-specific chemotherapy combined with surgery or radiation. Dr. Tulpule studies Ewing’s sarcoma (ES), a bone tumor caused by a unique genetic change that creates a tumor-specific protein EWS-FLI1. To date, no drug has been identified to directly block the cancer causing EWS-FLI1 protein. His research takes a different approach to combating ES by asking a fundamental question: can we identify a targetable weakness in ES tumors that is caused by the EWS-FLI1 protein? Using a cutting-edge screening technology called CRISPR interference, Dr. Tulpule's team identified a specific vulnerability in ES cells’ capacity to repair damage to their DNA. Normal cells have many backup systems in place to repair DNA damage, but they have shown that EWS-FLI1 causes ES cells to become overly reliant on a single pathway, known as homologous recombination (HR) repair, such that blocking HR is an effective and specific way to kill ES. Dr. Tulpule is building a detailed understanding of why ES cells are so vulnerable to HR pathway blockade and then applying that knowledge towards developing less toxic and more effective treatments for ES patients.

Based on progress to date, Dr. Osborn was awarded a new grant in 2022 and 2023 to fund an additional year of this Scholar grant. Abnormal growth of B-cells can result in leukemia, and a cutting-edge treatment option is immunotherapy with T-cells. T-cells can be engineered to express a chimeric antigen receptor (CAR) that is a 'seek and destroy' molecule for the CD19 protein on B-cells. CAR T-cells are the first FDA approved gene therapy and some stunning therapeutic responses have been observed. However, the T-cell activity can be so robust that they cause a massive cytokine storm that can be lethal. Furthermore, normal and cancerous B-cells express the CD19 protein targeted by the CAR, so normal B-cell loss occurs resulting in an impaired immune system. These side effects represent a significant hurdle in the safe and effective treatment of B-cell leukemia. To address this, Dr. Osborn, will express the CAR in a specialized subset of cells called T-regulatory (Treg) cells. Tregs have the same potent killing ability as T-cells but accomplish it without healthy tissue collateral damage. Additionally, he will engineer functional B-cells that are invisible to the CAR. This will allow for normal B-cell numbers and an intact immune system. Dr. Osborn will conduct these studies that are structured to resolve an unmet need, are highly novel, and are poised to make an immediate impact on childhood leukemia. 

The 2022 portion of this grant is named for the Rays of Hope Hero Fund which honors the memory of Rayanna Marrero. She was a happy 3-year-old when she was diagnosed with Acute Lymphoblastic Leukemia (ALL). She successfully battled ALL, but a treatment induced secondary cancer claimed her life at age eight. Rayanna had an amazing attitude and loved life. She, like so many kids facing childhood cancer, did not allow it to define who she was. This Hero Fund aspires to give hope to kids fighting cancer through research.

A portion of this grant was funded by and named for the Mighty Mimi Hero Fund. Mimi Enyon was diagnosed with acute lymphoblastic leukemia at the age of 3. Her courage in the fight was unparalleled and she became “Mighty Mimi” to all those she inspired on her way to remission. This fund was established to share Mimi’s cancer journey in an effort to raise awareness and funding for childhood cancer research for kids like her.

A portion of this grant is generously supported by the Stanley Kuzmickas Feeney Fund for Pediatric Cancer Research. It was Christmas 2015 when Stanley was diagnosed with acute lymphoblastic leukemia at 13 months old. He courageously endured treatments for 3½ years. Today, he is in remission and eagerly started school in fall 2019. In his honor, Stanley’s family has organized a head-shaving event each year since July 2016 called “StoshyStrong." With the funds raised, the Feeney family created this Hero Fund to support research in new discoveries, genomics and other biological therapies for the treatment of ALL. Their goal is to one day see personalized treatments for every child.

Cure rates for pediatric acute myeloid leukemia (AML) have remained at or below 60% for decades, and the largest reason for treatment failure is relapsed disease. Once relapse happens, it is very difficult to cure the disease. It is well known that interactions between AML cells and the non-cancerous supportive cells in the bone marrow, called stromal cells, can protect leukemia cells from chemotherapy. Dr. Redell's team reported that an enzyme in AML cells, called spleen tyrosine kinase (SYK), is turned on when AML cells contact stromal cells, and SYK helps AML cells survive chemotherapy. She will further investigate this enzyme, using a large panel of AML cells that come directly from pediatric patients. The drug company Gilead Sciences has given Dr. Redell a supply of their new SYK inhibitor, entospletinib, to test in her studies. To make sure that the results of the drug testing are related to blocking SYK, she also has made some AML cells that do not make SYK. Dr. Redell's team will do experiments to learn how SYK helps AML cells resist chemotherapy, and they will test entospletinib in AML models to determine if it might be a good drug to add to chemotherapy for patients.

Pediatric undifferentiated sarcomas are highly aggressive cancers that typically affect soft tissues of young children. Due to their uncertain classification and lack of molecular signature there are no standard criteria for diagnosis or treatment. With the Alan's Sarcoma Research Fund St. Baldrick's Research Grant, Dr. Antonescu is applying state of the art genomic methods to provide a detailed genetic characterization in these orphan cancers and investigating driving chromosomal translocations or mutations involved in their growth. These results will establish an objective classification of these tumors based on their genetic abnormalities and will provide potential therapeutic targets for further novel therapies. Furthermore these findings will inform the generation of faithful models for studying sarcoma formation and new drug development.

This grant is funded by and named for the Alan's Sarcoma Research Fund, a St. Baldrick's Hero Fund. Alan Sanders was diagnosed with a rare sarcoma in his hip at 17 months. He had an indomitable spirit and throughout his 4 ½ year battle with cancer, he was joyful, upbeat and pressed on courageously in spite of surgery and treatments. Today his family and friends carry on his legacy and his rallying cry, “Fight’s on!” in the battle against childhood cancer by funding sarcoma research.

Medulloblastoma is one of the most common malignant brain tumors in children. The Group 3 subgroup tumors have the poorest outcomes due to dissemination of tumor cells to distant sites in the central nervous system. As the recipient of the Benicio Martinez Fund for Pediatric Cancer Research St. Baldrick's Research Grant, Dr. Pei has identified a subpopulation of tumor cells that contribute to the metastasis after radiotherapy. He is determining whether targeting these cells can eliminate or prevent metastasis of Group 3 medulloblastoma, thereby improving the outcome of patients with this disease. Weeks after being the top fundraiser in his 6th grade class and shaving his head at his school’s event, Benny was diagnosed with medulloblastoma. Despite complications from treatment and setbacks, Benny has an amazing can-do attitude and is battling the cancer with determination.

This grant is funded by the Hero Fund that honors Benny’s fight and supports cures and better treatments for kids like him.

Current cancer therapy is very toxic and does not always work. We have developed a way to deliver much more drug to the tumor, and much less to the patient, by packaging the drug in properly designed nanomedicines. These delivery systems take advantage of the fact that most aggressive tumors have leaky blood vessels, so our nanomedicines can pass through into the tumor, but they bypass most normal tissues. Using these formulations, we can deliver 10-100 times as much drug to the tumor, so we can use less total drug and still get better results. In addition, Dr. Brodeur is using a novel drug called SN22. Although SN22 is related to a commonly used chemotherapy agent called irinotecan, it is an active drug, and unlike irinotecan it does not have to be activated by the liver. It is not only much more potent but also harder for the tumor cells to get rid of. These features make SN22 much more therapeutically effective. The carrier Dr. Brodeur is using to make this nanomedicine can deliver four molecules of SN22 within each “packet” that enters the tumor. Because he can use less total drug, and because the nanomedicine can circulate for a long time with the drug attached, there is much less exposure to the rest of the body, so side effects are dramatically reduced. As the recipient of the Invictus Fund St. Baldrick's Research Grant, Dr. Brodeur's goal is to develop more effective but less toxic therapy to treat children with cancer, and he can accomplish that goal with this approach using nanomedicine-based drug delivery. The nanomedicines he is developing should be effective against many different solid tumors in children or adults and he hopes to bring them forward to Phase 1 clinical trials.

This grant is funded by and named for the Invictus Fund, a St. Baldrick's Hero Fund created in memory of Holden Gilkinson and honors his unconquerable spirit in his battle with bilateral Wilms tumor as personified in the poem “Invictus” by William Ernest Henley. His family hopes to fund cures and treatments to mitigate side and late effects of childhood cancer.

This grant funds a medical student to complete work in pediatric oncology research for the summer. The experience may encourage them to choose childhood cancer research as a specialty.

The lives of children surviving cancer are cut short by life-threatening infections. Most pathogens causing these infections come from the gut. Healthy children have good bacteria in their gut that protect them from pathogens. Chemotherapy and antibiotics harm good bacteria in the gut that protect children from pathogens. Without good bacteria, pathogens increase in the gut. These pathogens can get into the bloodstream and cause serious infections. Prior studies have repeatedly shown that replacing healthy stool with good bacteria in the gut is the best treatment and prevention of gut infections. Dr. Andersen's team is developing a new test that detects the good bacteria and pathogens in the gut using a stool sample. This new test will allow earlier identification of children with cancer at the greatest risk for a serious infection and the children most likely to benefit from replacing healthy stool with good bacteria back in their gut. They believe that replacing good bacteria in the gut can prevent serious infections from pathogens, including those with limited antibiotic treatment options. This new test will also identify the specific good bacteria in the gut that prevent infection for developing new probiotics to prevent serious infections in children with cancer.

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.

This award allows Dr. Parsons the freedom to pursue discovery without the restrictions of a normal grant. The questions he is exploring include: What are the biologically and clinically-relevant genomic alterations in high-risk and rare pediatric cancers? What are the most useful and cost-effective clinical sequencing tests for childhood cancer patients? How can clinical genomics/precision oncology be most effectively implemented for diverse patients and families in varied clinical settings? What clinical benefit can precision oncology approaches and the use of molecularly-targeted therapies offer to childhood cancer patients? He is optimistic that a "team science" approach, bringing together investigators from diverse disciplines, departments, and institutions, will continue to yield critical data and discoveries that guide pediatric cancer research and clinical care in the next decade.

Despite improvements in outcome for patients with acute lymphoblastic leukemia (ALL), up to 25% of children and 40% of adults fail frontline therapy and their prognosis is dismal, especially for high-risk and relapsed leukemia. Cure rates for ALL patients that are relapsing on therapy is approximately 20% and currently there are no targeted therapies. Dr. Tsirigos' goal is to address this significant clinical need by studying how DNA, our genetic material, is organized inside the nucleus of the cells, i.e. how it is folded in three-dimensional space. Over the past decade, seminal studies have demonstrated that DNA folding is of fundamental importance in that it allows the cell to properly perform its function and retain its identity (e.g. a liver cell versus a blood cell). Very recent studies have demonstrated that disruptions of DNA folding may cause various diseases, such as developmental defects and cancer. However, no study has addressed the question of disruptions of DNA folding on the genome-wide scale in cancer or how such disruptions may be exploited for more effective treatments. Dr. Tsirigos is attempting to answer two key questions. First, are cancer-promoting genes (“oncogenes) capable of disrupting normal DNA folding to transform normal cells into malignant ones? And, second, can drug treatment restore DNA folding and thereby also restore normal cell function?

This grant is made with generous support from the Rally for Ryan Fund. Ryan was diagnosed with high risk ALL when he was 7 years old. He endured 3½ years of treatments with a brave acceptance that this was his fight to win. He recently relapsed and is in the fight again. This fund honors Ryan’s perseverance and his commitment to make a difference for kids with cancer by shaving for St. Baldrick’s and raising funds for research.

As the recipient of the Rosa and Francesco Romanello St. Baldrick's Research Grant, Dr. Lawlor is studying an aggressive tumor called Ewing sarcoma that occurs most often in teenagers. It usually starts in a bone and then can spread or metastasize throughout the body. Once it has spread, the chances of cure are very poor. She is studying how the tumor cells change the surrounding normal tissues to allow the tumor cells to leave the bone and spread to other sites in the body. Results so far have shown that the tumor cells and the normal tissues "talk to each other" and that this crosstalk is likely to be essential for the growth and spread of the tumor, both within the bone as well as in other tissues. Dr. Lawlor will decipher these messages, and the instructions they convey, so that new therapies can be developed that will intercept them and block tumor spread.

This grant is named in recognition of Salvatore Romanello for his decade of service as pro bono general counsel to the St. Baldrick's Foundation. He has chosen to name the grant in honor of his parents who instilled in him the values of generosity and caring for a greater cause.

The goal of Dr. Riehle's research is to find a cure for a rare form of liver cancer that occurs in children and young adults, called fibrolamellar hepatocellular carcinoma (FL-HCC). Unfortunately, surgery is currently the only effective treatment option for these patients, and once the disease has spread outside of the liver there is no chance for cure. Dr. Riehle's laboratory has spent the last few years trying to understand what changes within the liver cause healthy kids to get this cancer, and has developed a couple of new models of FL-HCC that can be used for drug screening. In this project she is using these models to test new treatment options and to try to understand how this cancer develops.

Based on progress to date, Dr. Ch'ng was awarded a new grant in 2020 to fund an additional year of this Fellow award. Epstein-Barr virus (EBV) is a common viral infection that in the vast majority of people causes only minor or no illness. However, in some situations it can play a role in the development of certain forms of cancer, such as lymphoma. One way that it might contribute to the development of cancer is by affecting the way that cells use energy because viruses and cancers both require increased energy to support rapid growth. By studying how EBV changes the way that cells use energy, Dr. Ch'ng hopes to learn whether changes in cell energy use are a factor in the development of cancers associated with EBV and whether these changes can be targeted to treat these forms of cancer.

Rhabdomyosarcoma (RMS) is a cancer with features of skeletal muscle, and the most common soft connective tissue cancer of childhood. The alveolar variant of RMS (abbreviated ARMS) is particularly hard to cure. If we could figure out which proteins in ARMS cancer cells work together to drive this cancer, we might also be able to figure out which are good drug targets. A common genetic error in ARMS is the mutant protein PAX3-FOXO1, which turns on cellular programs that cause ARMS cells to keep dividing. However, PAX3-FOXO1 is not a good drug target, and it does not work alone – it physically interacts with other proteins that carry out its cancer-causing instructions. Here, Dr. Linardic and colleagues will use a sophisticated new method to identify proteins in PAX3-FOXO1’s cellular neighborhood, a rapid screening technology to figure out which are most crucial to ARMS, then use models of ARMS to see which of the proteins might be the best drug targets. Importantly, this project will be carried out by three research teams with unique but complementary skills working together, united in a mission to find new therapies for this difficult-to-cure cancer.

Neuroblastoma is a cancer formed in certain types of nerve tissue and is the most common pediatric solid tumor outside of the brain. It is the leading cause of cancer-related death in children under five years old. Those patients who do survive often develop long-term side effects from intensive chemotherapy and radiation therapy. Thus, we need to develop better, safer alternative therapies for neuroblastoma. Dr. Cripe is currently studying the use of genetically modified herpes viruses. These viruses, which include the recently FDA-approved herpes virus T-VEC, can selectively infect and kill cancer cells without harming normal cells. In addition, these viruses are also able to enhance the patient's immune response against the cancer cells, potentially leading to a systemic and long-lasting protective immunity against cancer dissemination and recurrence. In the course of his studies, Dr. Cripe found that tumors infected with virus induce a counter measure by attracting cells that suppress immunity. In this study, he will test if he can improve therapy by interfering with that counter measure. If successful, these results may lead to a novel clinical trial for neuroblastoma patients.