Showing 1-20 of 177 results
Jonathan McConathy M.D., Ph.D.
Funded: 07-01-2017 through 06-30-2018
Funding Type: Research Grant
Institution Location: Birmingham, AL
Institution: University of Alabama at Birmingham affiliated with Children's of Alabama

Brain tumors are the most common solid tumor in children, and diagnostic imaging guides almost every step in the care of children with brain tumors. However, currently available imaging methods have limited accuracy. Dr. McConathy is using an amino acid tagged with radioactivity (FET) to detect abnormal metabolism in tumor tissue using positron emission tomography (PET) in combination with magnetic resonance imaging (MRI). He expects this new imaging technique to improve the ability to see brain tumors before and after surgery to help doctors better plan the treatment of children with brain tumors. In the long term, Dr. McConathy expects FET-PET/MRI to help select and plan the best therapies and increase the chance of achieving cures.

Theresa Keegan Ph.D.
Funded: 07-01-2017 through 06-30-2018
Funding Type: Research Grant
Institution Location: Sacramento, CA
Institution: University of California, Davis School of Medicine affiliated with UC Davis Children's Hospital

Adolescent and young adult (AYA) cancer survivors have an elevated risk of medical problems that can impact the quality and length of their lives, but few studies have focused on the occurrence of late medical conditions in this population. Using data on nearly all AYA cancer survivors in California, the Rich and Weissman Family Lymphoma Survivorship Fund St. Baldrick's Research Grant is identifying how often specific late medical conditions occur and how the risk of these medical conditions vary by clinical and patient factors. The results of the study will identify subgroups of young patients at increased risk of serious medical conditions, information critical to improving survivorship care and outcomes. Jared Weissman is a Hodgkin’s lymphoma survivor thanks to a clinical trial made possible by research. This Hero Fund honors his survivorship and his grandparents, Terri and Barry Rich, by funding research for new treatment options for cures and less toxic after effects for survivors.

Kevin Shannon M.D.
Funded: 07-01-2017 through 06-30-2018
Funding Type: Research Grant
Institution Location: San Francisco, CA
Institution: University of California, San Francisco affiliated with UCSF Benioff Children's Hospital

Glucocorticoids, which are sometimes called "steroids", are a type of drug used to treat all children, adolescents, and adults with acute lymphoblastic leukemia (ALL). In fact, there is substantial evidence that glucocorticoids are the single most effective drugs used to treat ALL, and that relapse is frequently due to the fact that they stop working. Although glucocorticoids have been used for over 50 years, we still do not fully understand how they kill ALL cells and why some ALL cells become resistant and cause relapse. Dr. Shannon has developed a novel approach for generating, transplanting, and treating ALL in models that now provides an unprecedented opportunity to uncover mechanisms of drug response and resistance. The purpose of this research project is to study ALL cells that have become resistant to glucocorticoids during treatment in order to identify the underlying reasons and to use this knowledge to develop better ways of treating them.

William Weiss M.D., Ph.D. 
Funded: 07-01-2017 through 06-30-2018
Funding Type: Research Grant
Institution Location: San Francisco, CA
Institution: University of California, San Francisco affiliated with UCSF Benioff Children's Hospital

Half of neuroblastomas are high-risk neuroblastoma, with poor survival. Understanding abnormalities that drive high-risk neuroblastoma (drivers) enables development of therapies against specific drivers. Until 2015, we had identified drivers for half of high-risk neuroblastomas. Recently, most remaining high-risk neuroblastomas were shown to have high levels of TERT, a protein that helps chromosomes replicate. It is still not clear how a protein that helps chromosomes replicate could drive cancer. Perhaps TERT is needed for neuroblastoma tumors to grow, but is not driving the tumor. To distinguish these possibilities, Dr. Weiss is testing whether TERT can drive neuroblastoma in human stem-cell models. In Dr. Weiss' system, stem cells generated from normal human blood or skin cells, are differentiated to form a cell type called neural crest, from which neuroblastoma is derived. He is introducing known drivers into these cells to generate a model for neuroblastoma. Some known drivers (MYCN) lead to neuroblastoma, while others (ALK) do not. Dr. Weiss is using this model to test whether TERT is a driver, or is required for neuroblastoma in the context of other drivers (ALK). Successful completion will generate a model to evaluate whether therapy directed against TERT could help children with neuroblastoma. This grant is generously supported by the Amanda Rozman Pediatric Cancer Research Fund created in memory of Amanda Rozman and honors her courageous battle with neuroblastoma by funding promising new to improve the efficacy and number of treatments available for relapsed and refractory neuroblastoma.

Paul Jedlicka M.D., Ph.D.
Funded: 07-01-2017 through 06-30-2018
Funding Type: Research Grant
Institution Location: Denver, CO
Institution: University of Colorado affiliated with Children's Hospital Colorado

Ewing Sarcoma is an aggressive disease affecting children and young adults. Patients are treated with intensive chemotherapy. This helps some, but not all, with early disease, works poorly in those with advanced disease, and can have serious side effects. Searching for new and better therapies, Dr. Jedlicka's lab has found a new protein that works abnormally in Ewing Sarcoma and that could be a new target for treatment. Dr. Jedlicka is working to understand more about how this protein works and how best to block it, to see if it could be a useful new treatment.

E. Anders Kolb M.D.
Funded: 07-01-2017 through 06-30-2018
Funding Type: Research Grant
Institution Location: Wilmington, DE
Institution: Alfred I. Dupont Hospital for Children of the Nemours Foundation

Recently the Meshinchi lab discovered that mesothelin, a cancer-specific antigen, is highly expressed in a subset of childhood AML cases, a result that both highlights the distinct genetic differences between adult and pediatric cancers and opens the door for the development of more targeted therapies. Dr. Kolb is developing novel combinations of bispecific T-cell engaging antibodies, called SMITEs (Simultaneous Multiple Interaction T-cell Engagers) that will co-target mesothelin and the AML marker CD33. These T-cell engaging protein pairs physically link cancer cells to cytotoxic T-cells resulting in more potent and selective killing than single agents alone.

Jessica Blackburn Ph.D.
Funded: 07-01-2017 through 06-30-2018
Funding Type: Research Grant
Institution Location: Lexington, KY
Institution: University of Kentucky Research Foundation affiliated with Kentucky Children's Hospital

Many cancer treatments kill both normal and cancer cells. Drugs used in standard cancer treatments have long term effects in children, such as causing developmental delays or second cancers later in life. Dr. Blackburn's team is working to find new drugs that kill cancer cells, but do not affect normal cells. By discovering characteristics that are unique to cancer and finding a drug that recognizes that specific characteristic, they will be able to selectively kill cancer cells. Their research goal is to improve cancer treatments so that children can live long, normal lives after their cancer is cured.

Loren Walensky M.D., Ph.D.
Funded: 07-01-2017 through 06-30-2018
Funding Type: Research Grant
Institution Location: Boston, MA
Institution: Boston Children's Hospital affiliated with Dana-Farber Cancer Institute, Harvard Medical School

High grade gliomas (HGG) are a vicious subtype of pediatric brain tumors that remain the leading cause of death among children with cancer. New therapeutic strategies are urgently needed to combat this scourge. By mining genomic datasets from HGGs, Dr. Walensky's team has identified a unique susceptibility profile based on retention of wild-type p53 status and dual expression of the negative regulators HDM2 and HDMX. Whereas p53 can be mutated or deleted to avoid cell cycle arrest or apoptosis, a frequent alternative mode of p53 suppression relies on overexpression of HDM2 and HDMX. Small molecules have been developed to target HDM2 specifically, but co-expression of HDMX causes resistance. Only a stapled peptide modeled after the critical p53 transactivation helix is capable of blocking both HDM2 and HDMX, a feature that has prompted its advancement to Phase I/II clinical trials in adult cancers. As the recipient of the St. Baldrick’s Research Grant with generous support from the Team Campbell Foundation, Dr. Walensky is testing a novel therapeutic strategy for pediatric HGG based on a dual-targeting stapled peptide inhibitor of HDM2/HDMX. He believes that the proof-of-concept data to emerge could provide a compelling rationale for conducting a clinical trial in these otherwise rapidly fatal pediatric brain cancers. The Team Campbell Foundation was created in memory of Campbell Hoyt who passed away from Anaplastic Ependymoma. Their mission is to improve the lives of families facing a childhood cancer diagnosis through raising awareness, funding research and providing psycho-social enrichment opportunities.

Sriram Venneti M.D., Ph.D.
Funded: 07-01-2017 through 06-30-2018
Funding Type: Research Grant
Institution Location: Ann Arbor, MI
Institution: University of Michigan affiliated with C.S. Mott Children’s Hospital

Diffuse intrinsic pontine gliomas (DIPG) are lethal pediatric brain tumors with no treatments. In order to develop cures we need to understand their biology. Cancers survive on fuel to generate energy to support their uncontrolled proliferation. One of the fundamental nutrients that drive the energy production is the amino acid glutamine. How glutamine is taken up and metabolized by DIPG tumor cells is not know. Further it is not known if inhibiting cancer cells from taking up and metabolizing this fuel is therapeutic. To address this significant gap in our knowledge, Dr. Venneti is studying glutamine metabolism in DIPG cancer cells and evaluating inhibition of glutamine metabolism as a potential therapeutic strategy. This grant is made with generous support from the McKenna Claire Foundation 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.

Patrick Grohar M.D., Ph.D.
Funded: 07-01-2017 through 06-30-2018
Funding Type: Research Grant
Institution Location: Grand Rapids, MI
Institution: Van Andel Research Institute affiliated with Helen Devos Children's Hospital, Spectrum Health Hospitals

The goal of this study is to develop new therapies for Ewing sarcoma by targeting a protein called EWS-FLI1. Many people believe that the key to improving outcomes for Ewing sarcoma patients is to develop new drugs that block EWS-FLI1. In order for this to be successful, there is a need to understand exactly what happens to the Ewing sarcoma cell when EWS-FLI1 is turned off. Dr. Grohar is using the latest technology to both characterize the consequence of EWS-FLI1 silencing and identify novel compounds that turn EWS-FLI1 off.

David Kirsch M.D., Ph.D.
Funded: 07-01-2017 through 06-30-2018
Funding Type: Research Grant
Institution Location: Durham, NC
Institution: Duke University Medical Center affiliated with Duke Children's Hospital & Health Center

Diffuse intrinsic pontine glioma, also referred to as brainstem glioma, is a pediatric cancer that accounts for the majority of deaths from brain tumors in children. Although radiation therapy is the standard of care for brainstem gliomas, the median survival of children with this tumor type is less than one year from diagnosis. In order to improve the treatment of these patients, Dr. Kirsch's team is using a model of brainstem glioma that can be used to evaluate the effectiveness of new therapies. Using this model, they are testing whether removing a protein called ATM, which is the target of drugs now entering clinical trials, will enhance radiation sensitivity in brainstem gliomas. They hypothesize that deleting this target, when given in combination with radiation therapy, will increase the number of tumor cells killed by radiation and will therefore improve survival in brainstem gliomas when they have a specific gene mutation commonly found in this childhood brain tumor. If successful, these studies will inform the design of future clinical trials testing this strategy in children with brainstem gliomas. This grant is named for Hannah’s Heroes, a St. Baldrick’s Hero Fund created in honor of Hannah Meeson and pays tribute to her fight by raising awareness and funding for all childhood cancers because kids like Hannah “are worth fighting for.”

Katherine Hyde Ph.D.
Funded: 07-01-2017 through 06-30-2018
Funding Type: Research Grant
Institution Location: Omaha, NE
Institution: University of Nebraska affiliated with Children's Hospital & Medical Center, Nebraska

Acute myeloid leukemia (AML) is a cancer of the immature cells in the bone marrow. One common chromosomal abnormality found in pediatric AML is the inversion of chromosome 16 (inv(16)). Current treatments for inv(16) AML are associated with significant toxicity, as well as serious long-term chronic effects. Therefore, there is a pressing need to develop new, more targeted treatments for children with inv(16) AML. Inv(16) generates a fusion gene called CBFB-MYH11. CBFB-MYH11 causes changes in gene expression, which are the first step in the development of leukemia. Because Cbfb-MYH11 is expressed in all inv(16) leukemia cells, it makes an attractive drug target. Currently, there are no CBFB-MYH11 inhibitors suitable for use in humans. However, it is possible that other proteins cooperate with CBFB-MYH11, some of which may be better drug targets. One potential co-factor is HDAC1. Dr. Hyde's team found that HDAC1 binds CBFB-MYH11 and is required for its activity. They also found that an HDAC1 inhibitor significantly blocks the growth leukemia cells in culture. In this project, Dr. Hyde is testing whether HDAC1 is an important co-factor of CBFB-MYH11 and if HDAC inhibitors effectively target Cbfb-MYH11+ leukemia cells in vivo. These results will have direct clinical implications for children with inv(16) AML.

Emily Bernstein Ph.D.
Funded: 07-01-2017 through 06-30-2018
Funding Type: Research Grant
Institution Location: New York, NY
Institution: Ichan School of Medicine at Mount Sinai affiliated with Kravis Children’s Hospital at Mount Sinai

Neurons are nerve cells that populate certain regions of the human body and are responsible for transmitting chemical signals back and forth to the brain to regulate critical bodily functions. A deadly form of pediatric cancer, known as neuroblastoma, occurs when a subset of these neurons start to proliferate uncontrollably. These cancer cells can migrate and spread throughout the body, making it very challenging to treat with currently available drugs. This highly aggressive form of neuroblastoma occurs in children who are older than 18 months. At such an early age, this disease can be quite devastating and there is an imperative need to better understand how this form of neuroblastoma develops. Recent work has identified pediatric cancer mutations in distinct specialized proteins that regulate chromatin (the complex of DNA and proteins), known as chromatin remodelers. One such protein, ATRX, was recently found to be mutated frequently in neuroblastoma tumors identified in adolescent and young adults, which have poor overall survival. Dr. Bernstein is exploring a novel therapy for neuroblastoma patients that harbor ATRX mutations thorough innovative and state-of-the-art approaches. Dr. Bernstein's team is comparing the cellular changes that occur in the presence of the drug in models of neuroblastoma.

Brent Stockwell Ph.D.
Funded: 07-01-2017 through 06-30-2018
Funding Type: Research Grant
Institution Location: New York, NY
Institution: Columbia University Medical Center affiliated with Morgan Stanley Children’s Hospital, New York-Presbyterian

Neuroblastoma is one of the most common childhood cancers. There are different subtypes of Neuroblastoma; some have a very poor prognosis for the patient. Dr. Stockwell's team has identified a new aggressive subtype of Neuroblastoma, called “mesenchymal”, and sought new therapies that can specifically target this subtype. Since genetic markers that can identify patients with the mesenchymal subtype are know, a selective therapy will have a greater chance of success in the clinic. They recently discovered that a common type of cholesterol-lowering drug, called statins, are potent and selective killers of mesenchymal neuroblastoma cells in the lab. There are many different statins, and now Dr. Stockwell is determining which is the most potent drug and exploring why the mesenchymal subtype is so sensitive to statins. He is also testing these drugs in models of the disease to show that statins are effective at killing mesenchymal neuroblastoma cells. Since these drugs have a documented safety profile in children and well-studied pharmacological activity, these drugs can be brought through preclinical testing relatively quickly and developed as novel therapies for this aggressive pediatric cancer.

Guangheng Li M.D., Ph.D.
Funded: 07-01-2017 through 06-30-2018
Funding Type: Research Grant
Institution Location: Beaverton, OR
Institution: Children's Cancer Therapy Development Institute

Rhabdomyosarcoma is a deadly cancer when spread through the body. With the Aiden's Army Fund St. Baldrick's Research Grant, Dr. Li is combining drugs already FDA approved for adult cancers in a way that stops rhabdomyosarcoma tumor cells from creating new tumors elsewhere in the body. This approach is unique because Dr. Li not only aims to stop the tumor cells from growing, but will try to convert what is left to non-cancerous cells similar to what is found in normal muscle. This grant is named for Aiden Binkley who was diagnosed with Stage IV rhabdomyosarcoma at age 8. This bright, funny and courageous little boy believed he got cancer so he could grow up to find a cure for it. His vision is being carried on by Aiden’s Army through the funding of research. They will march until there is a cure!

Monika Davare Ph.D.
Funded: 07-01-2017 through 06-30-2018
Funding Type: Research Grant
Institution Location: Portland, OR
Institution: Oregon Health and Science University affiliated with Doernbecher Children's Hospital

Children with cancer continue to succumb to their disease, many after receiving toxic therapies like chemotherapy and radiation. Also, surviving children face life long negative health consequences ranging from learning disabilities, to more severe effects such as a higher chance of getting another cancer in adulthood. Therefore, additional, rigorous scientific research needs to be performed to develop new and effective treatment options for these kids. Cancer growing inside the body hides in plain sight of the immune system. This is because cancer cells evolve to escape recognition by the immune cells. Therefore reawakening the immune system could be a very effective way of using a patients' own attacker cells to engulf cancer cells and get rid of the disease. Dr. Davare is working to discover and test new ways to reactivate immune cells for attacking cancer cells. For this project, she has developed an innovative method to identify synthetic molecules that will uncloak the cancer cell and make it visible to the immune system for destruction. This research strategy, in the long run, will open new doors and has the potential to not only increase survival of children with cancer, but their long term quality of life as well. This grant is named for Hannah’s Heroes, a St. Baldrick’s Hero Fund created in honor of Hannah Meeson and pays tribute to her fight by raising awareness and funding for all childhood cancers because kids like Hannah “are worth fighting for.”

Andrei Thomas-Tikhonenko Ph.D.
Funded: 07-01-2017 through 06-30-2018
Funding Type: Research Grant
Institution Location: Philadelphia, PA
Institution: The Children's Hospital of Philadelphia affiliated with University of Pennsylvania

All proteins in our bodies are made using assembly instructions contained in messenger RNAs, or mRNA. mRNA molecules themselves are constructed from building blocks called exons. When exons are joined together, or “spliced”, out of order, the resulting protein code is scrambled. This is what causes several types of leukemias in older adults. We have discovered that incorrect splicing also occurs with high frequency in childhood leukemias originating in antibody-producing B-cells. Dr. Thomas-Tikhonenko is testing two ideas. The first is that incorrect splicing is needed to sustain uncontrolled multiplication of leukemic cells. The second is that restoring proper exon assembly with specific drugs would slow down or block cancerous growth. If successful, these studies could pave the way to new clinical trials and improved survival of children with leukemia.

Heather Wilson-Robles DVM
Funded: 07-01-2017 through 06-30-2018
Funding Type: Research Grant
Institution Location: College Station, TX
Institution: Texas A&M AgriLife Research

Cancer is a genetic disease in which a cell learns to take advantage of certain processes that allow that cell to grow and survive unchecked. Bone cancer is an aggressive disease in both children and pet dogs that can be painful and often leads to death of the patient even with aggressive surgery and chemotherapy. Most often these patients die because the tumor has spread to other areas of the body, not from the original bone tumor, which is often removed with surgery. Therefore, in order to better battle this disease, new therapies that target the cells that spread are needed. Preliminary work with a new drug that targets this process has shown promise as just such a therapy. The goal of The Ben's Green Drakkoman St. Baldrick's Research Grant is to more thoroughly investigate this drug for its ability to prevent or delay spread of the tumor cells using both human and dog bone tumor cells. This grant is named for the Ben's Green Drakkoman Fund, created to honor the memory of Ben Stowell who battled osteosarcoma with an inspiring determination to live life fully. The fund is named after a super hero Ben created named the Green Drakkoman who defeats his enemy, the Evil Alien.

Angelique Whitehurst Ph.D.
Funded: 07-01-2017 through 06-30-2019
Funding Type: Research Grant
Institution Location: Dallas, TX
Institution: University of Texas Southwestern Medical Center at Dallas

Cancer cells are hard to defeat because they are so similar to normal cells. Most current methods that kill cancer cells impose collateral damage on normal cells that lead to immune suppression, hair loss, and gastro-intestinal damage. Dr. Whitehurst's research focuses on identifying therapies that will only kill tumor cells but leave normal cells unharmed. Here, she is focused on a tumor type that impacts adolescents: Ewing Sarcoma. She has identified a pathway, called TNFa, which is “mis-wired” in these cancer cells. Instead of dying when this pathway is activated, the cancer cells keep growing. Importantly, she has identified inhibitors of the pathway that can kill these tumor cells. Dr. Whitehurst is working to understand how this pathway is mis-wired in cancer cells and the consequences of its inhibition. The end goal would be the identification of chemical inhibitors that could be used in the clinic as a less toxic and more effective treatment option.

Kevin Jones M.D.
Funded: 07-01-2017 through 06-30-2018
Funding Type: Research Grant
Institution Location: Salt Lake City, UT
Institution: University of Utah affiliated with Huntsman Cancer Institute

Synovial sarcoma is a soft-tissue cancer in adolescents and young adults. More than half of patients develop metastasis, or spread of the cancer to the lungs. Once it has metastasized, synovial sarcoma is fatal in nearly all patients. Dr. Jones' team has developed a model of synovial sarcoma and found that when the tumor spread to the lungs many white blood cells begin to infiltrate the tumors. He is studying whether these particular white blood cells from the immune system are trying to fight the tumor or are helping the tumor grow and spread to the lungs. This team is testing if the presence of these immune cells in a large panel of human synovial sarcomas are associated with the same patients developing clinical spread of disease.