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Shannon Conneely M.D.

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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.

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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.

University of Hawaii Summer Fellow

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Funded: 07-01-2020 through 06-30-2021
Funding Type: St. Baldrick's Summer Fellow
Institution Location: Honolulu, HI
Institution: University of Hawaii Cancer Center

This grant funds an undergraduate student to complete work in pediatric oncology research for the summer. Raman spectroscopy (RS) is used to characterize different types of cancer tissue. Usually RS fingerprints are obtained when a slice of cancer tissue is examined under a microscope. With a new design as a portable hand-held RS probe, the St. Baldrick's Foundation Summer Fellow will use the probe to determine RS fingerprints in cancer cell cultures. If successful, the project results could be used to design uses of the probe in the clinic setting to detect cancer cells in blood or other fluids.

University of California, Davis Summer Fellow

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Funded: 07-01-2020 through 09-30-2020
Funding Type: St. Baldrick's Summer Fellow
Institution Location: Sacramento, CA
Institution: University of California, Davis School of Medicine affiliated with UC Davis Children's Hospital

This grant funds a medical student to complete work in pediatric oncology research for the summer. JMML is a rare type of childhood cancer that is really hard to cure. Right now, even our best treatments only stop this cancer for a year or so before it starts to come back. Cancers can be studied in specific models, which allow researchers to try out different drugs and treatments to see what works. The goal of this project is to use these models to find new treatments for JMML. This grant is named for the St. Baldrick's Foundation Staff whose generous gifts have helped fund this opportunity and may encourage students to choose childhood cancer research as a specialty.

Fred Hutchinson Cancer Research Center Summer Fellow

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Funded: 06-15-2020 through 09-14-2020
Funding Type: St. Baldrick's Summer Fellow
Institution Location: Seattle, WA
Institution: Fred Hutchinson Cancer Research Center affiliated with University of Washington, Seattle Children's Hospital

This grant funds a student to complete work in pediatric oncology research for the summer. There has been little success in curing high risk AML patients, with survival rates remaining at < 25%. This highlights our current reliance on highly intensive cytotoxic therapies and stem cell transplant, and their inadequacies. This project studies the combination of novel target discovery with state-of-the-art stem cell expansion technology. Protein science provides a unique opportunity to generate one of the most impactful therapeutic discoveries in childhood AML in the last 40 years, with minimal toxicity. The summer intern will assist in investigating the impact of drugs on cancer targets while minimizing toxicity toward healthy cells. Results will be used to help identify critical genes involved in cancer growth and disease resistance, and to leverage future work in drug development.

University of California, San Diego Summer Fellow

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Funded: 06-01-2020 through 08-31-2020
Funding Type: St. Baldrick's Summer Fellow
Institution Location: San Diego, CA
Institution: University of California, San Diego affiliated with Rady Children's Hospital San Diego

This grant funds an undergraduate student to complete work in pediatric oncology research for the summer. Children with aggressive neuroblastoma have poor cure rates despite intensive treatment, and new therapies are needed. Treatments that inhibit important proteins and pathways in neuroblastoma tumors are likely to be more effective with fewer side effects. Kinases are proteins that control signals in cancer cells, leading to cancer cell growth and spread. This study proposes to test a certain inhibitor to determine its effectiveness against neuroblastoma cells and tumors. The results of these studies will determine whether BLU-667 is effective against neuroblastoma, potentially leading to clinical trials using BLU-667 for treatment of children with neuroblastoma.

The Children's Hospital of Philadelphia Summer Fellow

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Funded: 06-01-2020 through 09-30-2020
Funding Type: St. Baldrick's Summer Fellow
Institution Location: Philadelphia, PA
Institution: The Children's Hospital of Philadelphia affiliated with University of Pennsylvania

This grant funds an undergraduate student to complete work in pediatric oncology research for the summer. Children diagnosed with leukemia are often effectively treated in the beginning, but later relapse with their disease. Scientists now feel that this is in part due to the sanctuary that the bone marrow (BM) provides the leukemia cells. This prevents complete elimination and can set children up for relapse. This study aims to understand how the BM protects leukemia cells. Once we have identified the mechanisms by which that happens we can then begin to develop drugs to prevent it. This lab has recently identified an inflammatory process by which leukemia cells change the BM function and think this is a root cause of disease persistence and relapse. The project will test this hypothesis and find out how to prevent the leukemia from changing the BM and causing relapse.

Children's Cancer Therapy Development Institute Summer Fellow

Funded: 06-01-2020 through 08-31-2020
Funding Type: St. Baldrick's Summer Fellow
Institution Location: Beaverton, OR
Institution: Children's Cancer Therapy Development Institute

This grant funds an undergraduate student to complete work in pediatric oncology research for the summer. This project will validate a drug for the medulloblastoma, a type of brain tumor, specifically tumors that spread from the original cerebellar location to the covering of the brain and spine (the meninges). This grant is named for the St. Baldrick's Foundation Staff whose generous gifts have helped fund this opportunity and may encourage students to choose childhood cancer research as a specialty.

The Pennsylvania State University Summer Fellow

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Funded: 06-01-2020 through 05-31-2021
Funding Type: St. Baldrick's Summer Fellow
Institution Location: Hershey, PA
Institution: Pennsylvania State University affiliated with Penn State Hershey Children's Hospital

This grant funds an undergraduate student and medical student to complete work in pediatric oncology research for the summer. T-cell acute lymphoblastic leukemia is a deadly childhood cancer that affects blood cells. The current treatment uses highly toxic medications. The goal of the proposed project is to test the efficacy of a novel, less toxic, targeted treatment for T-cell acute lymphoblastic leukemia. This award will train the student to perform experiments to test the efficacy of the novel treatment in T-cell leukemia and to determine the mechanisms of drug action against leukemia cells.

University of Colorado Summer Fellow

Funded: 05-26-2020 through 07-02-2020
Funding Type: St. Baldrick's Summer Fellow
Institution Location: Aurora, CO
Institution: Children's Hospital Colorado affiliated with University of Colorado

This grant funds a student to complete work in pediatric oncology research for the summer. This lab specializes in harnessing the power of a particular type of immune cells called macrophages and microglia which are the body's scavengers. This is done by blocking a "don't eat me" signal called CD47. The CD47 protein acts as a "don't eat me" signal to macrophages which normally engulf and devour cancer cells and other diseased and dying cells. It turns out that nearly every kind of cancer uses CD47 to evade these macrophages. Covering up the CD47 a "don't eat me" protein allows the immune cells to find and swallow cancer cells. Here we will test whether the ability of macrophages to eat tumor cells can be increased by blocking another immune dampening molecule called adenosine which is rapidly increased by tumor cells as they grow.

Case Western Reserve University Summer Fellow

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Funded: 05-26-2020 through 01-31-2021
Funding Type: St. Baldrick's Summer Fellow
Institution Location: Cleveland, OH
Institution: Case Western Reserve University

This grant funds a student to complete work in pediatric oncology research for the summer. Osteosarcoma (OS) is the most common and highly lethal bone cancer affecting children and adolescent populations. New therapies are desperately needed for this highly aggressive disease, as outcome for metastatic OS has not improved over the past few decades despite the utilization of aggressive combination chemotherapy. The summer fellow will focus on testing a novel CA-IX small molecule inhibitor using syngeneic OS tumors in vitro and in vivo. Activities generated through this Summer Fellowship grant will lay the foundation for pre-clinical data for the use of CA-IX inhibitor in future clinical trials.

University of California, San Francisco Summer Fellow

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Funded: 05-18-2020 through 08-17-2020
Funding Type: St. Baldrick's Summer Fellow
Institution Location: San Francisco, CA
Institution: University of California, San Francisco affiliated with UCSF Benioff Children's Hospital

This grant funds an undergraduate student and a medical student to complete work in pediatric oncology research for the summer. Neuroblastoma is a pediatric tumor in which a large subset has very poor survival. Researchers are trying to understand what makes this subset so deadly and have developed a system to test combinations of genes apart and together to determine how they could make certain neuroblastoma more aggressive. They will test whether certain mutations may make the neuroblastoma tumor cells more invasive and if these mutations could cause other critical gene expression changes in high risk neuroblastoma.

Creighton University Summer Fellow

Funded: 05-18-2020 through 02-28-2021
Funding Type: St. Baldrick's Summer Fellow
Institution Location: Omaha, NE
Institution: Creighton University

This grant funds two undergraduate students to complete work in pediatric oncology research for the summer. Tumors have extensive mutations in their DNA which play important roles in cancer development. Particular mutations that are frequently found in tumors are likely important for promoting cancer development. BubR1 is a protein that regulates the proper separation of DNA during cell division, and therefore plays an important role in suppressing cancer formation. A mutation in BubR1 (R249Q) is specifically observed in approximately 15% of pediatric cancers and is not found in adult cancers. Researchers will study this mutation and results may identify a unique mechanism of tumor development controlled by BubR1 specifically during developmental processes, uniquely promoting pediatric cancer. This project will provide an opportunity for these two students to spend the summer performing biomedical science research utilizing well-established and easy to learn techniques, to enhance their excitement in pediatric cancer research.

Georgetown University Summer Fellow

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Funded: 05-15-2020 through 10-30-2021
Funding Type: St. Baldrick's Summer Fellow
Institution Location: Washington, DC
Institution: Georgetown University affiliated with MedStar Georgetown University Hospital

This grant funds an undergraduate student to complete work in pediatric oncology research for the summer. Ewing sarcoma is a cancer that primarily occurs in children, adolescents, and young adults. While we don't know why certain people get Ewing sarcoma, we do know that most patients have the same problem with genes in their cancer cell. Just as genes affect your eye color, the Ewing sarcoma cells have a special gene, EWS-FLI1, that keeps the cancer growing. EWS-FLI1 is critical for Ewing sarcoma cells to survive. If you turn off EWS-FLI1, Ewing sarcoma cells die. This project will study exactly how YK-4-279, a chemical in a new drug in clinical trials, affects key survival processes, called transcription and splicing, to enable design of optimized drugs. This grant is named for the St. Baldrick's Foundation Staff whose generous gifts have helped fund this opportunity and may encourage students to choose childhood cancer research as a specialty.

Virginia Polytechnic Institute and State University Summer Fellow

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Funded: 05-15-2020 through 02-15-2021
Funding Type: St. Baldrick's Summer Fellow
Institution Location: Blacksburg, VA
Institution: Virginia Polytechnic Institute and State University

This grant funds a medical student to complete work in pediatric oncology research for the summer. Pediatric glioblastoma (p-GBM) is a lethal brain tumor that can affect children. This cancer is difficult to treat due to several factors, including the tumor's resistance to conventional therapies as well as the sensitivity of the surrounding healthy brain tissue. Children who undergo surgery to remove the brain tumor live an additional three to six years on average, though the quality of life may be low. Phosphoinositide 3-kinase (PI3K) is a protein family that normally regulates cell replication and survival. However, when it functions incorrectly, cells can experience unchecked growth and cause cancer. Inhibiting this protein family is a viable treatment option for cancer but blocking the whole PI3K family has severe side effects. It is imperative to understand each member of the PI3K family to better develop treatments that involve them.

University of Vermont and State Agricultural College Summer Fellow

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Funded: 05-01-2020 through 04-30-2021
Funding Type: St. Baldrick's Summer Fellow
Institution Location: Burlington, VT
Institution: University of Vermont and State Agricultural College affiliated with Vermont Children’s Hospital at the UVM Medical Center

This grant funds an undergraduate student to complete work in pediatric oncology research for the summer. Diffuse intrinsic pontine glioma (DIPG) is a type of children's brain tumor that currently has no cure or effective therapeutic options. This proposal aims to understand whether the target drug of ONC201, ClpP, can be targeted using novel compounds representing new potential therapeutics in DIPG.

New York University Summer Fellow

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Funded: 05-01-2020 through 04-30-2021
Funding Type: St. Baldrick's Summer Fellow
Institution Location: New York, NY
Institution: New York University School of Medicine affiliated with NYU Langone Medical Center

This grant funds an undergraduate student to complete work in pediatric oncology research for the summer. DIPG's are the worst type of brain cancer children can get; there is no cure. This project will try a new approach to change that. Using large publicly available datasets from large experiments, 4 drugs have been identified that theoretically can slow down the growth of DIPG tumors. Researchers will test these four drugs against several DIPG models generated from patients. If results are positive, this could lead to new treatments for this deadly disease.

The University of Tennessee Summer Fellow

Funded: 05-01-2020 through 09-30-2020
Funding Type: St. Baldrick's Summer Fellow
Institution Location: Memphis, TN
Institution: University of Tennessee Health Science Center

This grant funds a medical student to complete work in pediatric oncology research for the summer. This year it is estimated that 800 children will be diagnosed with osteosarcoma (bone cancer). The lab has identified a gene (WNT5B) that is too high in a subset of osteosarcomas. By making a cell line that removes WNT5B, the lab will compare its growth to the original cells and target this gene in those cancers that have it to design a specific targeted therapy.

Children's Hospital, Los Angeles Summer Fellow

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Funded: 05-01-2020 through 10-31-2020
Funding Type: St. Baldrick's Summer Fellow
Institution Location: Los Angeles, CA
Institution: Children's Hospital Los Angeles

This grant funds two students to complete work in pediatric oncology research for the summer. The experience may encourage them to choose childhood cancer research as a specialty. Project 1: Neuroblastomas are an enigmatic cancer of childhood with subtypes that have extremely good or poor survival. Poor prognosis neuroblastomas contain normal immune cells that help tumors grow. Important questions are 1) what is the repertoire of immune cells in neuroblastomas at time of diagnosis, 2) how the interplay between normal and tumor cells changes when tumors recur. The Summer Fellow will analyze images of tumors at recurrence and compare to the diagnosis images. These findings will provide insights into the types of immune cells that cancer cells rely on and may allow identification of new targets of therapy. Project 2: Decline in brain function may happen after irradiation to the brain in children. It is hard to predict the extent and speed by which it happens. There is suggestion that more rapid injury happens in areas with iron deposition. Using a novel MRI method that allows chemical identification and quantification of iron in the brain, the Summer Fellow will characterize the imaging changes in white matter of the brain in children who have been treated with radiation for their brain tumors. This will allow to then correlate the changes with future outcome of their cognitive function.

University of Wisconsin-Madison Summer Fellow

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Funded: 05-01-2020 through 12-31-2020
Funding Type: St. Baldrick's Summer Fellow
Institution Location: Madison, WI
Institution: University of Wisconsin-Madison affiliated with American Family Children's Hospital

This grant funds an undergraduate student to complete work in pediatric oncology research for the summer. High risk neuroblastoma remains a challenge to cure with only 50% survival, despite multi-modality treatment. Natural killer (NK) cells have been previously shown to have activity versus neuroblastoma but have not been consistently successful in clinical trials. In similar fashion to how people receive flu shots, this project will treat bone marrow transplant recipients with 3 doses of a vaccine, with or without anti-PD1 therapy to stimulate T and NK cells, to introduce their immune system to what neuroblastoma looks like, so in the event this tumor tries to grow, the immune system will stop it and kill it before making the patient sick. This grant is named for the St. Baldrick's Foundation Staff whose generous gifts have helped fund this opportunity and may encourage students to choose childhood cancer research as a specialty.