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Consortium for Childhood Cancer Predisposition
Funded: 07-01-2020
through 06-30-2026
Funding Type: Consortium Research Grant
Institution Location:
Atlanta, GA
Institution: Emory University
affiliated with Children's Healthcare of Atlanta, Children's Healthcare of Atlanta at Egleston, Aflac Cancer Center
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.
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.
Prasanna Ananth M.D
Funded: 07-01-2020
through 06-30-2024
Funding Type: St. Baldrick's Scholar
Institution Location:
New Haven, CT
Institution: Yale University
affiliated with Yale-New Haven Children's Hospital
Although rates of cure for childhood cancer have greatly improved in recent years, thousands of children continue to suffer from advanced, incurable cancer. Healthcare professionals bear a responsibility to ensure that the care of children with advanced cancer meets the goals and wishes of patients and their families. However, we do not know whether we fulfill this aim in pediatric cancer care. Dr. Ananth's prior research reveals intensive healthcare use near the end of life for children with advanced, incurable cancer. This includes lengthy stays in the intensive care unit and common use of interventions like breathing tubes. Yet, healthcare professionals worry that intense care toward the end of life for children with cancer may increase child and family suffering. In adults with cancer, quality measures have been developed to evaluate where care is most intense, or poor quality. This has consequently allowed researchers to develop interventions to improve the quality of care for adults with incurable cancer. Unfortunately, there are no comparable measures or standards for what constitutes good, or high quality, end-of-life care for children with cancer. Dr. Ananth seeks to address this problem. The overall objectives of this research are to (1) refine a list of potential measures of high quality end of life for children with cancer, and (2) develop an innovative questionnaire to systematically evaluate whether patients are receiving high quality end-of-life care. She hopes that, through this work that is distinctly family-centered, she can develop interventions to enable healthcare teams to provide optimal, compassionate care for children who have incurable cancer.
Stacey Crane Ph.D.
Funded: 07-01-2020
through 03-31-2021
Funding Type: St. Baldrick's Scholar
Institution Location:
Houston, TX
Institution: University of Texas Health Science Center at Houston
Unfortunately, kids with cancer suffer from symptoms related to their cancer and cancer treatments. These symptoms are often not fully appreciated by their health care providers and hence may be undertreated. Doses of cancer treatments are decreased or cancer treatments are even stopped when symptoms from cancer treatments aren't well controlled. Improving symptom assessments for kids with cancer will enhance health care providers' ability to track and manage kids' symptoms, to identify symptom trends, and could even prevent changes in cancer treatments due to poorly controlled symptoms. The Patient Reported Outcome Common Terminology Criteria for Adverse Events (Pediatric PRO-CTCAE) is a recently-developed survey that allows kids with cancer and their parents to directly report the type and severity of kids' symptoms to health care providers. This tool includes 130 questions about 62 different symptoms. However, since there are so many questions in this tool, the symptoms that are asked about are pre-selected by researchers or health care providers. Pre-selecting the questions prevents kids and parents from reporting all of a kid's symptoms, risking symptoms being missed. The overall objective of Dr. Crane's research is to refine and pilot test a novel web-based interface for the Pediatric PRO-CTCAE that will allow kids and parents to systematically and easily report all of a kid's symptoms on a routine basis, but without having to answer 130 questions.
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.
Fred Hutchinson Cancer Research Center Summer Fellow
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.
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.
University of California, San Diego Summer Fellow
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 Pennsylvania State University Summer Fellow
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.
The Children's Hospital of Philadelphia Summer Fellow
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.
Case Western Reserve University Summer Fellow
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 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.
University of California, San Francisco Summer Fellow
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.
Virginia Polytechnic Institute and State University Summer Fellow
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.
Georgetown University Summer Fellow
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.
Children's Hospital, Los Angeles Summer Fellow
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.
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.
University of Vermont and State Agricultural College Summer Fellow
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.
University of Wisconsin-Madison Summer Fellow
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.