Showing 1-20 of 100 results
Kellie Haworth M.D.
Funded: 11-01-2017 through 10-31-2020
Funding Type: St. Baldrick's Scholar
Institution Location: Memphis, TN
Institution: St. Jude Children's Research Hospital

Do you ever get a cold sore on your lip, or know someone who does? That sore is caused by a virus that destroys the cells in your lip. As the virus spreads, the sore gets bigger. Viruses are great at killing cells and spreading. But, the sore eventually goes away because the immune system attacks the infected cells, killing them and stopping the viral infection, allowing your lip to heal. Imagine if we could get both the virus and the immune system to kill cancer cells instead of lip cells! Previously Dr. Haworth's team used a safe version of the cold sore virus to infect a common type of hard-to-treat childhood cancer cells. The virus directly killed cancer cells and caused the immune system to attack the cancer cells that the virus missed. Dr. Haworth's team is testing ways to make the virus and immune system work better together. Dr. Haworth is infecting model tumors with the virus, and giving immune cells designed to attack the tumor, hypothesizing that giving both virus and immune cells will cure the tumor. Awarded at The Research Institute at Nationwide and transferred to St. Jude Children's Research Hospital.

Justina McEvoy Ph.D.
Funded: 07-01-2017 through 06-30-2020
Funding Type: St. Baldrick's Scholar
Institution Location: Tucson, AZ
Institution: University of Arizona Medical Center

Rhabdomyosarcoma is a pediatric cancer of the developing skeletal muscle. The mechanisms that drive this tumor are poorly understood. From Dr. McEvoy's preliminary analysis, one possible mechanism is epigenetic deregulation of a group of long noncoding RNAs (lncRNA). This is exciting because lncRNAs play a role in tumorigenesis in other cancer types, including a subset of pediatric tumors. This presents a unique opportunity to develop novel therapeutic approaches for children with rhabdomyosarcoma. Dr. McEvoy's team hypothesizes that lncRNA deregulation is essential for rhabdomyosarcoma development. This study is working to understand the underlying mechanisms that drive this disease and identify potential new therapies. These results will have tremendous impact on patients, especially those with metastatic disease since only 20-40% will survive using current treatments.

Adam Green M.D.
Funded: 07-01-2017 through 06-30-2020
Funding Type: St. Baldrick's Scholar
Institution Location: Denver, CO
Institution: University of Colorado affiliated with Children's Hospital Colorado

High-grade gliomas (HGG) are aggressive brain cancers that affect both adults and children. Current treatment options are very limited, and the vast majority of patients die of their tumors within five years of diagnosis. One subtype of high-grade glioma that almost exclusively occurs in children, diffuse intrinsic pontine glioma (DIPG), is the last incurable childhood cancer, with zero percent long-term survivors. To address these tumors, Dr. Green and team have focused on a new field of cancer treatment called epigenetics, which literally means “above genetics” and refers to all changes to DNA that do not involve changes to the DNA sequence itself, but instead affect which genes are made into protein. Through prior work, Dr. Green's team has found a gene, BPTF, which controls the expression of many other genes and appears to drive HGG and DIPG growth. Dr. Green aims to determine how exactly BPTF drives growth by interacting with other genes, to measure how BPTF inhibition works with drugs called HDAC inhibitors and whether this strategy could work with current standard treatments, and to measure the effect of a new chemical that inhibits BPTF that could serve as a precursor to medicines targeting BPTF.

Brian Ladle M.D., Ph.D. 
Funded: 07-01-2017 through 06-30-2020
Funding Type: St. Baldrick's Scholar
Institution Location: Baltimore, MD
Institution: Johns Hopkins University School of Medicine affiliated with Johns Hopkins Children's Center

Dr. Ladle is using the body’s own immune system to destroy cancer - specifically a class of cancer in children originating from connective tissues called sarcomas. Using fire as an analogy, Dr. Ladle seeks to build an intense flame of a powerful immune response which will specifically kill the cancer cells. To create this fire, one must follow specific steps. The kindling, which must be easily burned, is protein targets on the cancer cells (termed tumor antigens) recognized by the immune system. Next, the spark to ignite the kindling is initial inflammation in the tumor against these tumor antigens. Finally, to feed the fire, fuel or lighter fluid can be added in the form of recently approved immune modulator drugs which, when infused into patients, bind to immune cells residing in the tumor and activates them to kill the tumor cells. Each ordered step is essential in building an effective fire. This project addresses each of these key aspects for generating a successful immune response to treat sarcomas – creating new tumor antigens, adding inflammation to jump start the immune response against these antigens, and combining with new immune modulators allowing the immune cells to be active in destroying sarcomas.

Hazel Nichols Ph.D.
Funded: 07-01-2017 through 06-30-2020
Funding Type: St. Baldrick's Scholar
Institution Location: Chapel Hill, NC
Institution: University of North Carolina at Chapel Hill affiliated with UNC Children's Hospital

Women who are diagnosed with cancer before 40 are often concerned about whether they will be able to have children in the future. Women can freeze their eggs or embryos (called fertility preservation) to help protect their fertility, but these services may not be widely available. Dr. Nichols has looked at how often women in North Carolina have children after cancer treatment and whether the health of their babies is different from women without cancer. She is further examining the use of fertility preservation after diagnosis and its association with birth rates and outcomes. This research will provide information to improve the long-term health of AYAs with cancer.

Luisa Cimmino Ph.D.
Funded: 07-01-2017 through 06-30-2020
Funding Type: St. Baldrick's Scholar
Institution Location: New York, NY
Institution: New York University School of Medicine affiliated with NYU Langone Medical Center

Vitamin C is essential for maintaining healthy hair, skin, immune system and heart function. In addition to these health benefits, Dr. Cimmino and team propose that vitamin C might be a non-toxic therapeutic for the treatment of patients with pediatric acute myeloid leukemia. Recently, it was discovered that vitamin C enhances the activation of a group of enzymes called TET proteins that are required for normal blood development. A significant fraction of children and young adolescents with acute myeloid leukemia have mutations in TET2, causing impaired TET2 activity and a block in normal blood cell formation. However, only one of the two copies of the TET2 gene is defective in these patients. Dr. Cimmino's team is working to determine if treatment with high-dose vitamin C could enhance the activity of the remaining, non-mutant, TET2 protein, kill leukemia cells and restore normal blood development. Alternative therapies such as treatment with vitamin C might provide a safe and effective strategy to improve outcome for pediatric leukemia patients.

Chandrika Gowda M.D.
Funded: 07-01-2017 through 06-30-2020
Funding Type: St. Baldrick's Scholar
Institution Location: Hershey, PA
Institution: Pennsylvania State University affiliated with Penn State Hershey Children's Hospital

Children with high risk B-cell leukemia, especially with loss or dysfunction of IKZF1 gene have very poor outcomes and high relapse rate. Every other child who relapses with high risk leukemia dies from the disease and there has not been much advancement in treatment for this group for the last 30 years. Dr. Gowda and team have found that a cancer promoting protein called casein kinase II (CK2) impairs the important functions of a protein that helps prevent leukemia. Inhibiting the CK2 protein will restore the ability of this protein to function properly and prevent leukemia. Dr. Gowda's team is testing if using a drug that inhibits CK2 protein along with the drugs that already are known to work in leukemia will have stronger anti-leukemia effect and improve the outcome. Using two agents that target same gene or pathway via different mechanisms will ensure effective shutdown of the particular pathway resulting in strong therapeutic effect. This strategy would also help lower the doses of each drug used and reduce their side effects and associated toxicity.

Sarah Richman M.D., Ph.D.
Funded: 07-01-2017 through 06-30-2020
Funding Type: St. Baldrick's Scholar
Institution Location: Philadelphia, PA
Institution: The Children's Hospital of Philadelphia affiliated with University of Pennsylvania

The immune system not only fights infection, but can also fight cancer cells. Recently, doctors have been able to use patients’ own immune cells to help treat their cancer. These immune cells can also attack the patient's normal tissues, which is harmful. Dr. Richman is working to learn how normal tissues might be protected while still allowing the immune cells to effectively kill the cancer cells.

Gary Kohanbash Ph.D.
Funded: 07-01-2017 through 06-30-2020
Funding Type: St. Baldrick's Scholar
Institution Location: Pittsburgh, PA
Institution: Children's Hospital of Pittsburgh affiliated with University of Pittsburgh

Brain tumors are the leading cause of cancer-related deaths in children, and ependymomas are the third most common kind. Recent studies have shown that “educating” the patient’s own immune system to fight cancers – immunotherapy – can be safe and effective. Dr. Kohanbash's team has identified three peptides that might activate immune cells to specifically fight one of the more lethal types of ependymoma. Dr. Kohanbash is testing these peptides in the lab. He is also looking at how immunotherapy could help fight all six types of ependymoma that affect kids, and thus is studying relevant characteristics in the largest-ever series of pediatric ependymoma tumors as well as in ependymoma patients already participating in a clinical trial of a vaccine based on another peptide. This grant is generously supported by the Henry Cermak Fund for Pediatric Cancer Research which was created in memory of a brave boy who had an amazing spirit throughout his battle with a brain tumor. This fund is dedicated to Henry’s wish that “no one gets left out.”

David McFadden M.D., Ph.D
Funded: 07-01-2017 through 06-30-2020
Funding Type: St. Baldrick's Scholar
Institution Location: Dallas, TX
Institution: University of Texas Southwestern Medical Center at Dallas

The EWSR1-FLI1 family of cancer genes causes Ewing sarcoma. However, no drugs currently exist that specifically block the action of EWSR1-FLI1 to cause cancer cells to grow. The McFadden Lab has engineered a "self-destruct button" into the EWSR1-FLI1 gene in Ewing sarcoma cells cultured in the laboratory, and these cells stop growing when the EWSR1-FLI1 gene is turned off. Dr. McFadden is using this laboratory tool to identify proteins that work with EWSR1-FLI1, and identify other genes it controls to cause Ewing sarcoma cells to grow. These studies will help identify new ways to stop the growth of Ewing sarcoma cells.

Saurabh Agarwal Ph.D
Funded: 07-01-2017 through 06-30-2020
Funding Type: St. Baldrick's Scholar
Institution Location: Houston, TX
Institution: Baylor College of Medicine affiliated with Texas Children's Hospital, Vannie E. Cook Jr. Children's Cancer and Hematology Clinic

High-risk neuroblastoma is an aggressive cancer of very young children with less than 50% overall survival. Current therapy includes high-dose chemotherapy and radiation, which has long-term toxic side-effects. Despite these intensive therapies, neuroblastoma commonly relapse. This relapse is the primary cause of death from neuroblastoma due to disease spread, drug-resistance, and toxicity. As the David's Warriors St. Baldrick's Scholar, Dr. Agarwal is focusing her research on developing effective therapeutic approaches to target those tumor cells which escape initial treatment and regenerate drug-resistant disease. Recently, Dr. Agarwal's team discovered a chemotherapy-resistant, highly tumorigenic sub-population of cells in neuroblastoma tumors. These cells escape initial therapy and may cause aggressive, drug-resistant relapsed disease. Furthermore, they found that specific epigenetic enzymes maintain this cell sub-population by activating key genes. These epigenetic modifiers can be successfully targeted with novel epigenetic inhibitors, currently under pre-clinical trials. These exciting findings suggest a new epigenetic therapeutic approach for high-risk neuroblastoma. This grant supports efforts to uncover the mechanisms controlling neuroblastoma tumorigenicity and relapse, and develop an effective targeted approach for high-risk neuroblastoma. This grant honors the memory of David Heard who battled neuroblastoma and pays tribute to the amazing spirit with which he lived, embracing life until the very end.

Katherine Tarlock M.D.
Funded: 07-01-2017 through 06-30-2020
Funding Type: St. Baldrick's Scholar
Institution Location: Seattle, WA
Institution: Seattle Children's Hospital affiliated with Fred Hutchinson Cancer Research Center, University of Washington

Acute myeloid leukemia (AML) is a cancer of white blood cells and almost half of children diagnosed with AML will not be cured, even with very intensive chemotherapy and in some cases bone marrow transplant. Many of the mutations in the leukemia that contribute to development of the cancer have been identified, but cannot be used for therapeutic benefit, especially in children. Dr. Tarlock and colleagues have performed genomic testing on the cells of many children diagnosed with AML and found that approximately 40% of children abnormally express the protein mesothelin on their leukemia cells. Dr. Tarlock and colleagues will develop a phase I clinical trial to test a new therapy strategy that uses principles of the immune system to deliver chemotherapy only to mesothelin-positive leukemia cells. They will develop a clinical assay for mesothelin detection in AML to identify children who will benefit from mesothelin-targeted therapy, and investigate methods to optimize disease response to mesothelin-targeting immune therapies.

Reshmi Parameswaran Ph.D
Funded: 09-01-2016 through 08-31-2019
Funding Type: St. Baldrick's Scholar
Institution Location: Cleveland, OH
Institution: Case Western Reserve University

Acute Myeloid Leukemia (AML) is the second most common acute leukemia in children, and current treatment strategies are inadequate to cure AML. Dr. Parameswaran is developing a new strategy using Natural Killer cells, which are a type of white blood cells with potential to kill cancer cells. Cancer cells often produce a protein that makes Natural Killer cells less active, which helps the cancer cells escape from NK cell-mediated killing. Dr. Parameswaran and her team are developing methods to stop this NK cell inactivation and thereby improve NK cell function to treat pediatric AML.

Liora Schultz M.D.
Funded: 07-01-2016 through 06-30-2019
Funding Type: St. Baldrick's Scholar
Institution Location: Palo Alto, CA
Institution: Stanford University affiliated with Lucile Packard Children’s Hospital

The human immune system is made up of a complicated network of cells including cells that help fight diseases such as cancer, and cells that prevent the immune system from fighting disease. Key cells that stop immune fighter cells from destroying cancer are called T regulatory cells (Tregs). Dr. Schultz is studying a new way to stop these Tregs and allow the good fighter cells to resume their ability to destroy cancer cells. This therapy will allow immune cells to put up a stronger fight against cancer and lead patients with cancer closer to cure.

Elias Sayour M.D., Ph.D.
Funded: 07-01-2016 through 06-30-2019
Funding Type: St. Baldrick's Scholar
Institution Location: Gainesville, FL
Institution: University of Florida affiliated with Shands Hospital for Children

For children affected by medulloblastoma, the development of more effective and specific therapies that will not add further toxicity to existing treatments is critical in improving clinical outcomes. Dr. Sayour, the Hannah'’s Heroes St. Baldrick'’s Scholar, is investigating methods to harness the immune system to destroy these tumors though the use of nanoparticle vaccines. Nanoparticles can deliver messages to the immune system teaching it to kill cancer cells. Dr. Sayour is studying possible immune targets for nanoparticles in medulloblastoma, identifying underlying tumor resistance mechanisms, and establishing an optimum vaccine approach which he will evaluate in medulloblastoma models. 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.

Grzegorz Nalepa M.D., Ph.D.
Funded: 07-01-2016 through 06-30-2019
Funding Type: St. Baldrick's Scholar
Institution Location: Indianapolis, IN
Institution: Indiana University affiliated with IU Health Proton Therapy Center, Riley Hospital for Children

Children continue to die from leukemia because malignant cells overcome chemotherapy by turning off normal genetic safety checkpoints. Dr. Nalepa has found that loss of the same checkpoints makes cancer growth dependent on other genes, which represent possible anti-cancer targets. Dr. Nalepa is investigating the weak points of the cancer cells, and testing these precision-medicine strategies in new models of childhood leukemia. Dr. Nalepa will use the results of this testing to generate personalized therapy driven by cancer-specific mutations that will be more effective and less toxic for children who suffer from leukemia.

Dhvanit Shah Ph.D.
Funded: 07-01-2016 through 06-30-2019
Funding Type: St. Baldrick's Scholar
Institution Location: Boston, MA
Institution: Brigham and Women's Hospital, Inc.

Finding a donor for a bone marrow transplant can be difficult for patients that are mixed-race or from an ethnic minority, as bone-marrow comes from donors that are genetically compatible. Dr. Shah is developing a method utilizing hemogenic endothelial cells (HEC) that will have the same effect, without requiring bone marrow from a matched donor. Dr. Shah is investigating FDA-approved drugs that stimulate the HECs to produce a novel and safe alternative to bone marrow-generated Hematopoetic stem cells, which will potentially treat childhood blood cancers.

Nicolas Llosa M.D.
Funded: 07-01-2016 through 06-30-2019
Funding Type: St. Baldrick's Scholar
Institution Location: Baltimore, MD
Institution: Johns Hopkins University School of Medicine affiliated with Johns Hopkins Children's Center

Osteosarcoma is a tumor that forms in the bones and is the most common bone tumor of childhood. Dr. Llosa is investigating how the immune system interacts with cancer cells from osteosarcoma tumors. Dr. Llosa's focus is on immunotherapy, a type of cancer treatment designed to boost the body's natural defenses to fight the cancer, and one of the most promising current approaches for treating tumors. Immunotherapy uses materials either made by the body or in a laboratory to improve or restore immune system function with the final goal of stopping the growth of tumors. Dr. Llosa is studying the immune microenvironment of metastatic osteosarcomas to assess their potential for checkpoint blockade (where immune responses are allowed through an checkpoint in malignant cells in order to fight the cancer) as a therapeutic option. This grant is made with generous support from the Ethan Jostad Foundation, established by Kim and Chris Jostad in 2011 in memory of their son, Ethan, who was taken by Alveolar Rhabdomyosarcoma at the age of nine. In addition to funding cutting-edge pediatric cancer research, the foundation'’s mission is to provide emotional and financial support to children and families impacted by the disease.

Ryan Roberts M.D., Ph.D.
Funded: 07-01-2016 through 06-30-2019
Funding Type: St. Baldrick's Scholar
Institution Location: Columbus, OH
Institution: Nationwide Children's Hospital affiliated with The Research Institute at Nationwide

Dr. Roberts is focused on developing new treatments for patients with metastatic bone tumors. These tumors, once they have spread to the lungs, are incredibly difficult to treat. Dr. Roberts and his team will study the pathways that proteins move between osteosarcoma cancer cells and lung tissues. These experiments are helping Dr. Roberts and his team to better understand the biology that lets those tumors grow in the lung and will evaluate treatments which might prevent metastases from growing, and make them treatable when they do.

Roderick O'Sullivan Ph.D.
Funded: 07-01-2016 through 06-30-2019
Funding Type: St. Baldrick's Scholar
Institution Location: Pittsburgh, PA
Institution: University of Pittsburgh affiliated with Children's Hospital of Pittsburgh

Telomeres are special sequences of DNA located at the ends of every chromosome, and are essential to maintaining proper cellular function. If telomeres are damaged or degraded, they may cause healthy cells to transform into cancer cells. Dr. O'Sullivan and his team have discovered a protein called RAD51AP1 that appears at high levels in neuroblastoma tumor cells, and they have determined that having less of this protein stops telomere damage in cells. Dr. O'Sullivan is investigating the consequences and impact of RAD51AP1 inhibition on the proliferation and survival of neuroblastoma tumor cells.