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Reducing Ethnic Disparities in Acute Leukemia (REDIAL) Consortium Member

Funded: 07-01-2017 through 06-30-2022
Funding Type: Consortium Research Grant
Institution Location: Dallas, TX
Institution: University of Texas Southwestern Medical Center at Dallas

This institution is a member of a research consortium which is being funded by St. Baldrick's: Reducing Ethnic Disparities in Acute Leukemia (REDIAL) Consortium. For a description of this project, see the consortium grant made to the lead institution: Baylor College of Medicine, Houston, TX.

Taryn Allen Ph.D.

Funded: 07-01-2017 through 12-30-2021
Funding Type: Supportive Care Research Grant
Institution Location: Bethesda, MD
Institution: National Cancer Institute, National Institutes of Health affiliated with NIH Clinical Center

Children diagnosed with brain tumors and their parents have many challenges when there is little hope for a cure. Research shows that children who have an incurable brain tumor and their parents are stressed, less happy with their lives, and in poorer health when compared to healthy children and their parents, yet no known helpful coping strategies have been developed for this brain tumor population. Mindfulness exercises are a type of coping tool that help people pay attention to the present moment and handle difficult emotions that come up when facing very stressful life events. In particular, mindfulness exercises have been shown to improve quality of life in children and adults with terminal illness.

With the MaxLove Project Fund St. Baldrick's Supportive Care Research Grant, Dr. Allen is evaluating a new coping program using age-appropriate mindfulness activities to improve the quality of life of children diagnosed with an incurable brain tumor and their families. Coping interventions are greatly needed for this population. The MaxLove Project Fund honors the survivorship journey of Max Wilford who was diagnosed with a brain stem tumor at the age of four. Despite several surgeries and an intense treatment protocol, Max is now able to be a “regular” kid due in large part to integrative therapies he received.

Beth Winger M.D., Ph.D.

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Funded: 07-01-2017 through 12-31-2020
Funding Type: St. Baldrick's Fellow
Institution Location: San Francisco, CA
Institution: University of California, San Francisco affiliated with UCSF Benioff Children's Hospital

Based on progress to date, Dr. Winger was awarded a new grant in 2019 to fund an additional year of this Fellow award. The goal of this project is to test a promising new drug called PLX9486 to treat pediatric cancers. In some cancers, a protein called "KIT" acts as an engine to drive growth. In comparison to existing treatments, PLX9486 is able to stop KIT in a unique way. Therefore, it is expected that this new drug will be very effective against cancers that are driven by KIT. However, over time cancer cells figure out ways to bypass drugs, leading to drug resistance. In addition to testing the effectiveness of PLX9486 against cancer cells, Dr. Winger is also studying how KIT might bypass the drug to develop resistance. Understanding the potential causes of drug resistance will allow her to develop strategies to overcome this resistance. This project will systematically evaluate a new drug that has the potential to transform the treatment of pediatric cancers driven by KIT.

Reducing Ethnic Disparities in Acute Leukemia (REDIAL) Consortium Member

Funded: 07-01-2017 through 06-30-2022
Funding Type: Consortium Research Grant
Institution Location: Ft. Worth, TX
Institution: Cook Children's Medical Center

This institution is a member of a research consortium which is being funded by St. Baldrick's: Reducing Ethnic Disparities in Acute Leukemia (REDIAL) Consortium. For a description of this project, see the consortium grant made to the lead institution: Baylor College of Medicine, Houston, TX.

Heather Wilson-Robles DVM

Funded: 07-01-2017 through 03-31-2019
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, a St. Baldrick's Hero 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.

Grant Rowe M.D., Ph.D.

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Funded: 07-01-2017 through 12-31-2020
Funding Type: St. Baldrick's Fellow
Institution Location: Boston, MA
Institution: Boston Children's Hospital affiliated with Dana-Farber Cancer Institute, Harvard Medical School

Based on progress to date, Dr. Rowe was awarded a new grant in 2019 to fund an additional year of this Fellow award. Leukemia that develops in infants under one year of age is difficult to cure and has poor long-term outcomes compared to leukemia developing in older children or teenagers. The therapies used for infant leukemia are highly toxic with long-term adverse effects and are not particularly effective at curing the disease. To identify more tolerable and more effective treatments, we need better models to study infant leukemia in the laboratory. We could use such a model to identify the genes that drive infant leukemia and make it so aggressive and challenging to treat, and to develop drugs to target these genes. Dr. Rowe has developed a new model of infant leukemia and is using this system to identify the key genes that make this form of leukemia so aggressive.

Reducing Ethnic Disparities in Acute Leukemia (REDIAL) Consortium Member

Funded: 07-01-2017 through 06-30-2022
Funding Type: Consortium Research Grant
Institution Location: El Paso, TX
Institution: Texas Tech University Health Sciences Center - El Paso

This institution is a member of a research consortium which is being funded by St. Baldrick's: Reducing Ethnic Disparities in Acute Leukemia (REDIAL) Consortium. For a description of this project, see the consortium grant made to the lead institution: Baylor College of Medicine, Houston, TX.

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.

Joanna Pierro D.O.

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

Based on progress to date, Dr. Pierro was awarded a new grant in 2019 to fund an additional year of this Fellow award. While outcomes for childhood leukemia have improved dramatically, the prognosis for children who relapse remains poor making relapsed leukemia one of the main causes of cancer death in children. Discovering the underlying pathways that lead to chemotherapy resistance and relapsed disease is therefore a top priority. To prevent relapse and improve treatment response, Dr. Pierro's laboratory has focused on discovering genetic mutations responsible for relapse and chemotherapy resistance. Mutations in a gene known as MMSET have been identified as one of the most common mutations in relapsed leukemia in children. This mutation in other cancers imparts a poor prognosis which suggests it has a role in drug resistance. Dr. Pierro's team has developed leukemia cell lines with and without the MMSET mutation and is treating the lines with chemotherapy to test this theory. He is also identifying the pathways controlled by this gene to identify the mechanism by which it protects the cells from the effects of chemotherapy. This information could be used to develop targeted therapy to prevent relapse and restore sensitivity to chemotherapy thereby improving outcomes.

Reducing Ethnic Disparities in Acute Leukemia (REDIAL) Consortium Member

Funded: 07-01-2017 through 06-30-2022
Funding Type: Consortium Research Grant
Institution Location: San Antonio, TX
Institution: Children's Hospital of San Antonio

This institution is a member of a research consortium which is being funded by St. Baldrick's: Reducing Ethnic Disparities in Acute Leukemia (REDIAL) Consortium. For a description of this project, see the consortium grant made to the lead institution: Baylor College of Medicine, Houston, TX.

William Weiss M.D., Ph.D. 

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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.

Michael Koldobskiy M.D., Ph.D.

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

Based on progress to date, Dr. Koldobskiy was awarded a new grant in 2019 to fund an additional year of this Fellow award. Acute lymphoblastic leukemia (ALL) is the most common cancer in children. Despite dramatic improvements in treatment outcome in recent decades, relapsed and resistant disease remains a leading cause of childhood death from cancer. Dr. Koldobskiy studies the ways in which leukemia cells rely on "epigenetic" modifications, or chemical marks that modify the expression of genes without a change in the genetic sequence itself. Variability of epigenetic marks allows leukemia cells flexibility in turning genes on and off, and may account for resistance to treatment. By dissecting the mechanisms of epigenetic modification in childhood ALL, he aims to identify new targets for treatment.

Reducing Ethnic Disparities in Acute Leukemia (REDIAL) Consortium Member

Funded: 07-01-2017 through 06-30-2022
Funding Type: Consortium Research Grant
Institution Location: McAllen, TX
Institution: Vannie E. Cook Jr. Children's Cancer and Hematology Clinic affiliated with Baylor College of Medicine, Texas Children's Hospital

This institution is a member of a research consortium which is being funded by St. Baldrick's: Reducing Ethnic Disparities in Acute Leukemia (REDIAL) Consortium. For a description of this project, see the consortium grant made to the lead institution: Baylor College of Medicine, Houston, TX.

Loren Walensky M.D., Ph.D.

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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.

University of Texas MD Anderson Summer Fellow

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Funded: 06-19-2017 through 08-11-2017
Funding Type: St. Baldrick's Summer Fellow
Institution Location: Houston, TX
Institution: University of Texas M.D. Anderson Cancer Center

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

Johns Hopkins University Summer Fellow

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Funded: 06-15-2017 through 08-15-2017
Funding Type: St. Baldrick's Summer Fellow
Institution Location: Baltimore, MD
Institution: Johns Hopkins University School of Medicine affiliated with Johns Hopkins Children's Center

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

University of Tennessee Summer Fellow

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Funded: 06-05-2017 through 11-30-2017
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. The experience may encourage them to choose childhood cancer research as a specialty.

University of Oklahoma Summer Fellow

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Funded: 06-05-2017 through 08-04-2017
Funding Type: St. Baldrick's Summer Fellow
Institution Location: Oklahoma City, OK
Institution: University of Oklahoma Health Sciences Center affiliated with The Children's Hospital at OU Medical Center

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

Indiana University Summer Fellow

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Funded: 06-01-2017 through 07-31-2017
Funding Type: St. Baldrick's Summer Fellow
Institution Location: Indianapolis, IN
Institution: Indiana University affiliated with Riley Hospital for Children, IU Health Proton Therapy Center

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

Washington University in St. Louis Summer Fellow

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Funded: 06-01-2017 through 07-31-2017
Funding Type: St. Baldrick's Summer Fellow
Institution Location: St. Louis, MO
Institution: Washington University in St. Louis affiliated with St. Louis Children's Hospital

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