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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: 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.

Katherine Tarlock M.D.

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Funded: 07-01-2017 through 06-30-2021
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

Based on progress to date, Dr. Tarlock was awarded a new grant in 2020 to fund an additional year of this Scholar grant. 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.

This grant is generously supported by Rhys’ Pieces of the Cure, a Hero Fund created to honor Rhys Goldman and his journey with cancer. He was diagnosed with pre-B acute lymphoblastic leukemia just 2 weeks before his 6th birthday and endured treatment for three years. Rhys missed a lot of school and life during those years but since marking the end of treatment in July 2018, he has been enjoying swimming, singing in a boys’ choir, chess tournaments, playing with his dogs and going to school. Rhys’ Pieces for the Cure was created to ensure more research is funded for the treatment of pediatric cancer that is specifically focused on less toxic cures for kids.

Sam Behjati Ph.D, BMMCh., B.A.

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Funded: 10-01-2016 through 09-30-2019
Funding Type: Robert J. Arceci International Innovation Award
Institution Location: Cambridge, United Kingdom
Institution: Wellcome Trust Sanger Institute

The seeds of many childhood cancers are likely to be laid during human development before a child is born. Therefore, one may view childhood cancer cells as being stuck in development. Dr. Behjati aims to define at what stage tumour cells are stuck and to find ways of stimulating cancer cells to resume development. He envisions that this will provide a way of maturing cancer cells into harmless, non-cancerous cells. The St. Baldrick's Robert J. Arceci International Innovation Award is given in honor of the late Dr. Robert Arceci. A pioneer in the field, this award reflects Dr. Arceci's values including creativity, collaboration, and commitment to early- to mid-career scientists.

Reshmi Parameswaran Ph.D

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Funded: 09-01-2016 through 08-31-2022
Funding Type: St. Baldrick's Scholar
Institution Location: Cleveland, OH
Institution: Case Western Reserve University

Based on progress to date, Dr. Parameswaran was awarded new grants in 2019 and 2020 to fund additional years of this Scholar grant. 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.

This grant is generously supported by Rays of Hope, a St. Baldrick's Hero Fund created in memory of Rayanna Marrero. She was a happy 3 year old when she was diagnosed with Acute Lymphoblastic Leukemia. She battled ALL and won but a treatment induced secondary cancer claimed her life at age eight. Rayanna had an amazing attitude and loved life. She, like so many kids facing childhood cancer, did not allow it to define who she was. This Hero Fund aspires to give hope to kids fighting cancer through research.

James DeGregori Ph.D.

Funded: 07-01-2016 through 06-30-2017
Funding Type: Research Grant
Institution Location: Denver, CO
Institution: University of Colorado affiliated with Children's Hospital Colorado

Progress in improving therapy for Acute myeloid leukemia (AML) has been slow and survival rates for patients remain quite low. Thus, there is a great need for more effective and less toxic therapies for AML. Dr. DeGregori has identified a new molecule called MCJ, the loss of which is associated with the resistance of cancers to therapies. Dr. DeGregori has developed novel drugs that can restore MCJ function in cancer cells, and is investigating whether these MCJ activating drugs can be used improve therapies for AML.

Yves DeClerck M.D.

Funded: 07-01-2016 through 06-30-2018
Funding Type: Research Grant
Institution Location: Los Angeles, CA
Institution: Children's Hospital Los Angeles

Neuroblastoma is the second most common solid tumor in children, and is a cancer that frequently metastasizes to the bone marrow. Dr. DeClerck is studying how neuroblastoma cells "teach" bone marrow cells to promote tumor growth.

William Weiss M.D., Ph.D. 

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Funded: 07-01-2016 through 06-30-2017
Funding Type: Research Grant
Institution Location: San Francisco, CA
Institution: University of California, San Francisco affiliated with UCSF Benioff Children's Hospital

Targeted therapy works by attacking an abnormal gene product that is specific to the cancer type. Only a minority of neuroblastoma types show genetic drivers, which makes it difficult to develop targeted therapy. Most neuroblastomas show too many or too few copies of large chromosomal regions, called CNAs. Dr. Weiss is studying the connection between CNAs and neuroblastoma, to determine if it CNA is a possible candidate for targeted therapy. Dr. Weiss is engineering CNAs to create CNA-driven models of neuroblastoma, which he will then use to identify CNA-specific therapies to treat neuroblastoma.

Michael Eck M.D., Ph.D.

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Funded: 07-01-2016 through 06-30-2017
Funding Type: Research Grant
Institution Location: Boston, MA
Institution: Dana-Farber Cancer Institute affiliated with Boston Children's Hospital, Harvard Medical School

Childhood brain tumors are frequently quite different than those of adults. Dr. Eck's For the Love of Jack St. Baldrick''s Research Grant aims to find new 'targeted' therapies for low-grade astrocytomas (a type of brain tumor) in children that are caused by a mutation in a protein called BRAF. BRAF mutations are common in cancer, and drugs have been developed that are effective in some tumors caused by one type of BRAF mutation. Unfortunately, these drugs do not work on the BRAF mutation found most often in pediatric brain tumors. Dr. Eck is using detailed information about the molecular structure of the BRAF mutation found in pediatric brain tumors to discover new drugs that specifically target this cause of brain tumors in children. Jack Tweedy was diagnosed with brain and spinal cancer when he was two. Since then he has endured multiple surgeries and 270 weeks of chemotherapy but never fails to uplift those around him. Together with his family, he inspires others to help fund the best research to ensure that all cancer warriors have better treatment options.

Michael Deel M.D.

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Funded: 07-01-2016 through 09-30-2019
Funding Type: St. Baldrick's Fellow
Institution Location: Durham, NC
Institution: Duke University Medical Center affiliated with Duke Children's Hospital & Health Center

Based on progress to date, Dr. Deel was awarded a new grant in 2018 to fund an additional year of this Fellow award. Rhabdomyosarcoma is a childhood cancer of the muscle that has two major subtypes. Children with the alveolar type do very poorly, as these are frequently recurrent and metastatic. Alveolar rhabdomyosarcoma cells have a specific fusion protein that is a powerful cancer driver and that does not yet have a pharmaceutical treatment. Dr. Deel and his team recently found that the Hippo pathway, normally functions to suppress tumor growth, is not correctly regulated in alveolar rhabdomyosarcoma, which leads to TAZ (a co-activator) activating pro-tumoric gene transcription.

As the Aiden's Army St. Baldrick's Fellow, Dr. Deel is studying the interaction between the protein and the co-activator TAZ as a novel therapeutic target. Aiden Binkley 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!

Le Su Ph.D.

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Funded: 07-01-2016 through 06-30-2017
Funding Type: Research Grant
Institution Location: Huntsville, AL
Institution: HudsonAlpha Institute for Biotechnology

Genes instruct cells to do their jobs through making specific proteins. In the human body, all cells store these "instructions" in the chromosomes. When chromosomes break off, the broken pieces sometimes change places and create new chromosomes. These changes are called chromosomal translocations. Dr. Su is studying how chromosomal translocations cause deadly diseases in children and young adults, and more importantly, is investigating possible clinical options to correct these abnormal conditions.

Alan B Cantor M.D. Ph.D.

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Funded: 07-01-2016 through 06-30-2017
Funding Type: Research Grant
Institution Location: Boston, MA
Institution: Boston Children's Hospital affiliated with Dana-Farber Cancer Institute, Harvard Medical School

Juvenile myelomonocytic leukemia (JMML) is an aggressive blood cancer of young children. The only current curative treatment is bone marrow transplantation. Yet, about 50% of children do not survive despite this very aggressive therapy. An improved understanding of the mechanisms that cause JMML is critical to developing new treatments. Dr. Cantor is studying a protein called GATA2, which normally turns on and off white blood cell genes, to determine if it is incorrectly activated in JMML. If so, the results of this project will support development of medicines to inhibit GATA2 for the treatment of JMML.

Charles G. Mullighan M.D.

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Funded: 07-01-2016 through 06-30-2020
Funding Type: Robert J. Arceci Innovation Award
Institution Location: Memphis, TN
Institution: St. Jude Children's Research Hospital

Acute lymphoblastic leukemia is a tumor of white blood cells that normally fight infection. Changes in DNA, or mutations, are important in driving the development of ALL. Mutations in genes that control the reading of DNA are particularly common in leukemia cells that don't respond to treatment. Dr. Mullighan is studying engineered ALL cells and tumors to understand how these mutations result in resistance to therapy, and to develop new ways of treating ALL. The St. Baldrick’s Robert J. Arceci Innovation Award is given in honor of the late Dr. Robert Arceci. A pioneer in the field, this award reflects Dr. Arceci’s values including creativity, collaboration, and commitment to early- to mid-career scientists.

Samuel John M.D.

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Funded: 07-01-2016 through 06-30-2021
Funding Type: St. Baldrick's Fellow
Institution Location: Dallas, TX
Institution: University of Texas Southwestern Medical Center at Dallas

Based on progress to date, Dr. John was awarded a new grant in 2018 to fund an additional year of this Fellow award. Unfortunately, even with intensive treatment, only 60% of children with acute myeloid leukemia (AML) will survive the disease. Intensifying standard therapies have failed to improve survival rates, so a new approach is needed. Dr. John is creating a novel receptor that will specifically target a marker on AML cells. He will put this new receptor on T-cells of the body, which will then target and kill the leukemia cells. Dr. John hopes to improve outcomes for pediatric AML with this grant.

This grant is generously supported by Super Soph's Pediatric Cancer Research Fund. Sophie Rossi was diagnosed with AML at 3 months of age. Throughout her courageous battle, she was always smiling, always joyful. This fund was created to honor her spunky, sweet spirit by funding research to find cures for AML and all childhood cancers.

Roderick O'Sullivan Ph.D.

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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.

Ryan Roberts M.D., Ph.D.

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Funded: 07-01-2016 through 06-30-2020
Funding Type: St. Baldrick's Scholar
Institution Location: Columbus, OH
Institution: Nationwide Children's Hospital affiliated with The Research Institute at Nationwide

Based on progress to date, Dr. Roberts was awarded a new grant in 2019 to fund an additional year of this Scholar grant. 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.

The Hannah's Heroes St. Baldrick's Consortium Grant: Pediatric LEukemiA Precision-based Therapy (LEAP) Consortium

Funded: 07-01-2016 through 12-31-2023
Funding Type: Consortium Research Grant
Institution Location: Boston, MA
Institution: Dana-Farber Cancer Institute affiliated with Boston Children's Hospital, Harvard Medical School

This consortium is conducting the first multi-institutional, genomics-based precision medicine trial for children with relapsed, refractory, and very high-risk leukemias. They are deploying new technologies to sequence all currently known cancer-promoting genes in an individual child's leukemia. Next, guided by a multi-disciplinary group of leukemia experts, they will make treatment recommendations based upon the genomic findings in the child's leukemia and the availability of a relevant targeted drugs for children. Results from this project will define the types and frequency of specific mutations in children with relapsed/refractory leukemia, and assess the impact of the treatment recommendations on their clinical care. The consortium will also perform laboratory-based studies to determine the response to therapy in lab models derived from the patient's leukemia cells, to identify new drug targets for these children. Data generated in this proposal will inform future genomically-based targeted therapy trials for children with leukemia with the long-term goal of precise, safe, and more efficacious therapies for children with these diseases. Funds administered by Dana-Farber Cancer Institute.

This grant is named for Hannah’s Heroes, a Hero Fund established to honor Hannah Meeson. At age 6 she was diagnosed with anaplastic medulloblastoma. After a relapse and additional treatment, Hannah currently shows no evidence of disease. Throughout her treatments, Hannah never complained and remained positive and happy. This fund pays tribute to her fight by raising awareness and funding for all childhood cancers because kids like Hannah “are worth fighting for.”

Pediatric LEukemiA Precision-based Therapy (LEAP) Consortium Member

Funded: 07-01-2016 through 12-31-2023
Funding Type: Consortium Research Grant
Institution Location: Seattle, WA
Institution: Seattle Children's Hospital affiliated with Fred Hutchinson Cancer Research Center, University of Washington

This institution is a member of a research consortium which is being funded by St. Baldrick's: Pediatric LEukemiA Precision-based Therapy (LEAP) Consortium. For a description of this project, see the consortium grant made to the lead institution: Dana-Farber Cancer Institute, Boston, MA.

Pediatric LEukemiA Precision-based Therapy (LEAP) Consortium Member

Funded: 07-01-2016 through 12-31-2023
Funding Type: Consortium Research Grant
Institution Location: San Francisco, CA
Institution: University of California, San Francisco affiliated with UCSF Benioff Children's Hospital

This institution is a member of a research consortium which is being funded by St. Baldrick's: Pediatric LEukemiA Precision-based Therapy (LEAP) Consortium. For a description of this project, see the consortium grant made to the lead institution: Dana-Farber Cancer Institute, Boston, MA.

Pediatric LEukemiA Precision-based Therapy (LEAP) Consortium Member

Funded: 07-01-2016 through 12-31-2023
Funding Type: Consortium Research Grant
Institution Location: Chicago, IL
Institution: The University of Chicago affiliated with Comer Children's Hospital

This institution is a member of a research consortium which is being funded by St. Baldrick's: Pediatric LEukemiA Precision-based Therapy (LEAP) Consortium. For a description of this project, see the consortium grant made to the lead institution: Dana-Farber Cancer Institute, Boston, MA.

Pediatric LEukemiA Precision-based Therapy (LEAP) Consortium Member

Funded: 07-01-2016 through 12-31-2023
Funding Type: Consortium Research Grant
Institution Location: Denver, CO
Institution: University of Colorado affiliated with Children's Hospital Colorado

This institution is a member of a research consortium which is being funded by St. Baldrick's: Pediatric LEukemiA Precision-based Therapy (LEAP) Consortium. For a description of this project, see the consortium grant made to the lead institution: Dana-Farber Cancer Institute, Boston, MA.