Showing 1-20 of 563 results
Ryan Summers M.D.
Funded: 07-01-2018 through 06-30-2020
Funding Type: St. Baldrick's Fellow
Institution Location: Atlanta, GA
Institution: Emory University affiliated with Aflac Cancer Center, Children's Healthcare of Atlanta

Early T-precursor ALL (ETP-ALL) is a type of leukemia that is often difficult to treat with currently available chemotherapy. As a result, children with ETP-ALL have high rates of relapse of their leukemia and poorer survival rates than children with other types of ALL, and require more treatment with chemotherapy, often leading to long-term toxic side effects. For these reasons, new treatments for ETP-ALL are needed. MERTK is a protein that is found on the surface of some leukemia cells, including ETP-ALLs. Recently, Dr. Summers and colleagues developed a new medicine that has few toxic side-effects and can be used to kill leukemia cells that have MERTK on their surface. Funding from the St. Baldrick's Foundation will allow him to test whether and how this new medicine could be used to more effectively treat children with ETP-ALL, leading to improved outcomes and better quality of life.

Daniel Peltier M.D., Ph.D.
Funded: 07-01-2018 through 06-30-2020
Funding Type: St. Baldrick's Fellow
Institution Location: Ann Arbor, MI
Institution: University of Michigan affiliated with C.S. Mott Children’s Hospital

Bone marrow transplantation (BMT) is required to cure many childhood cancers. However, bone marrow transplantation is often complicated by severe and often fatal side effects. Both the beneficial anti-cancer effects and harmful side effects of bone marrow transplantation are due in part to the new immune system that the patient receives. Unfortunately, we do not know how to precisely fine tune this new immune system to make BMT safer for more children. Dr. Peltier's work seeks to further understand how a component of this new immune system is controlled by a recently identified class of genes called non-coding RNAs (ncRNA). These ncRNA genes do not make proteins like classic genes, but instead regulate the production and function of proteins made by classical genes. His early data shows that unique ncRNA genes from multiple classes of ncRNAs are turned on and off following BMT. However, it is not known if or how these unique ncRNA genes influence the new immune system after BMT. Dr. Peltier seeks to further understand the function of these ncRNAs following BMT, which may suggest ways of developing medicines to improve BMT.

Yamilet Huerta M.D.
Funded: 07-01-2018 through 06-30-2020
Funding Type: St. Baldrick's Fellow
Institution Location: Cleveland, OH
Institution: University Hospitals of Cleveland affiliated with Rainbow Babies and Children's Hospital

Leukemia is the most common type of cancer in childhood, and 20% of childhood leukemia has a myeloid origin. Acute myeloid leukemia (AML) is treated with intensive chemotherapy as standard of care. Unfortunately, despite chemotherapy and stem cell transplantation, the prognosis of a child with recurrent or refractory AML remains poor. T cells are part of our immune system, and when properly manipulated, can be highly effective in eradicating chemo-resistant tumor cells. Engager (ENG) T cells are genetically engineered T cells that are capable of binding specific target on AML cells and at the same time "engaging" neighboring T cells to mount an immune response and kill cancer cells. As the JJ's Angels Hero Fund St. Baldrick's Fellow, Dr. Huerta is investigating the mechanisms by which AML cells can be killed by this novel immunotherapy technique. This grant is named for the JJ's Angels Hero Fund which honors the memory of Juliana LaMonica and her courageous battle with AML. Diagnosed at the age of two, Juliana underwent a bone marrow transplant but passed away shortly after turning three. Her sweet spirit and charismatic personality continue to inspire people to support the funding of pediatric cancer research through Team JJ’s Angels.

Samara Potter M.D., M.B.A.
Funded: 07-01-2018 through 06-30-2020
Funding Type: St. Baldrick's Fellow
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

Despite recent advances in technology, very little is known about many types of rare and high risk childhood cancers. Since the numbers of these patients are so small, it has been very difficult to study how best to take care of them. Dr. Potter is using technology to look at the genetic code of these rare tumors, in order to learn more about why and how they occur, as well as how they change over time. This knowledge will help to create tests to diagnose these patients, as well as to develop more effective, less toxic treatments.

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.

Corey Falcon M.D.
Funded: 07-01-2017 through 06-30-2018
Funding Type: St. Baldrick's Fellow
Institution Location: Birmingham, AL
Institution: University of Alabama at Birmingham affiliated with Children's of Alabama

ALL is the most common blood cancer occurring in children. Great strides have been made in the treatment of this disease, but new less toxic therapies for high risk ALL are needed. A new effective therapy is chimeric antigen receptor T-cells (CAR-T) which involves altering a patient’s own cancer fighting cells (T-cells) to express a protein able to recognize a protein on ALL cells (CD19), thus promoting killing of ALL cells. This form of therapy is much less toxic than traditional chemotherapy, but it is still associated with unwanted side effects. Dr. Falcon is working on ways to eliminate anti-CD19 CAR-T if severe side effects occur. This will greatly enhance the safety of this promising treatment. A portion of this grant is generously supported by the Not All Who Wander Are Lost Fund which was named after Kiersten Dickson’s favorite quote from J.R.R. Tolkien and honors the memory of a free spirited, courageous young woman who battled a rare, incurable cancer. This fund hopes to advance cutting edge immunotherapy treatments for pediatric cancers.

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.

David Mulama Ph.D.
Funded: 07-01-2017 through 06-30-2020
Funding Type: International Scholar
Institution Location: Duarte, CA
Institution: Beckman Research Institute of the City of Hope

Kaposi sarcoma-associated herpesvirus is a virus that causes cancer known as Kaposi sarcoma, which is very common in HIV+ children, especially in Africa and sometimes in individuals who get an organ transplant. Dr. Mulama is designing and testing a vaccine that prevents and treats the viral infection, as well as antibodies to detect infection in people. He will also test the vaccine so that one day it can be used as a treatment to prevent Kaposi sarcoma-associated herpesvirus infection and Kaposi sarcoma in more than 40,000 patients worldwide each year.

New Approaches to Neuroblastoma Therapy (NANT) Consortium
Funded: 07-01-2017 through 06-30-2018
Funding Type: Consortium Research Grant
Institution Location: Los Angeles, CA
Institution: Children's Hospital Los Angeles

Only 45% of children with high-risk neuroblastoma (NB) are cured. The New Approaches to Neuroblastoma Therapy (NANT) consortium links laboratory and clinical investigators to develop therapies with high potential for improving survival and performs the first testing of these treatments at 14 neuroblastoma centers in North America and 3 in Australia, United Kingdom, and France. NANT studies aim 1) to enhance the ability of the immune system to eliminate NB by targeting both NB cells and surrounding normal cells that help tumor cells grow and resist treatments; 2) to improve treatment of NB using small molecule drugs that target NB gene abnormalities that “drive” tumor aggressiveness including abnormal/mutated ALK protein or abnormally increased MYCN protein; and 3) to use molecular biology to define new treatment targets in NB and normal cells that enhance NB growth and resistance to therapy to improve prediction of outcome with a “biomarker” test for NB cells in blood and bone marrow. The NANT consortium anticipates that these innovative studies will improve survival for children with high-risk neuroblastoma.

Reducing Ethnic Disparities in Acute Leukemia (REDIAL) Consortium Member
Funded: 07-01-2017 through 06-30-2018
Funding Type: Consortium Research Grant
Institution Location: Orange, CA
Institution: Children's Hospital of Orange County

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.

New Approaches to Neuroblastoma Therapy (NANT) Consortium Member
Funded: 07-01-2017 through 06-30-2018
Funding Type: Consortium Research Grant
Institution Location: Palo Alto, CA
Institution: Lucile Packard Children’s Hospital affiliated with Stanford University

This institution is a member of a research consortium which is being funded by St. Baldrick's: New Approaches to Neuroblastoma Therapy (NANT) Consortium. For a description of this project, see the consortium grant made to the lead institution: Children's Hospital Los Angeles, Los Angeles, CA.

New Approaches to Neuroblastoma Therapy (NANT) Consortium Member
Funded: 07-01-2017 through 06-30-2018
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: New Approaches to Neuroblastoma Therapy (NANT) Consortium. For a description of this project, see the consortium grant made to the lead institution: Children's Hospital Los Angeles, Los Angeles, CA.

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.

New Approaches to Neuroblastoma Therapy (NANT) Consortium Member
Funded: 07-01-2017 through 06-30-2018
Funding Type: Consortium Research Grant
Institution Location: Aurora, CO
Institution: Children's Hospital Colorado affiliated with University of Colorado

This institution is a member of a research consortium which is being funded by St. Baldrick's: New Approaches to Neuroblastoma Therapy (NANT) Consortium. For a description of this project, see the consortium grant made to the lead institution: Children's Hospital Los Angeles, Los Angeles, CA.

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.

Amanda Winters M.D., Ph.D.
Funded: 07-01-2017 through 06-30-2019
Funding Type: St. Baldrick's Fellow
Institution Location: Denver, CO
Institution: University of Colorado affiliated with Children's Hospital Colorado

Dr. Winters' research involves developing more effective and more targeted therapies for children with acute myeloid leukemia (AML), a type of leukemia that continues to have poor outcomes. The therapy for pediatric AML has not changed much in 20-30 years, and many children who receive this therapy relapse. There is a protein on many AML cells called CD123, which marks the earliest leukemia cells. In adults there are drugs that target this protein which are being studied in clinical trials. However, no one has studied whether CD123 is a useful target in pediatric AML. Dr. Winters is looking at CD123 protein expression in AML samples from pediatric patients, as well as investigating whether expression of CD123 marks the primitive leukemia cells in these patients - that is, those that give rise to the leukemia and cause relapse. She is also testing some of the same drugs that are being used in adult clinical trials on these pediatric samples in a laboratory setting, to see if they may be useful in pediatric patients. These studies are expected to generate new therapy options for children with difficult-to-treat AML.

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.

David Mulama Ph.D.
Funded: 07-01-2017 through 06-30-2020
Funding Type: International Scholar
Institution Location: Kakamega, Eldoret
Institution: Masinde Muliro University of Science and Technology

Kaposi sarcoma-associated herpesvirus is a virus that causes cancer known as Kaposi sarcoma, which is very common in HIV+ children, especially in Africa and sometimes in individuals who get an organ transplant. Dr. Mulama is designing and testing a vaccine that prevents and treats the viral infection, as well as antibodies to detect infection in people. He will also test the vaccine so that one day it can be used as a treatment to prevent Kaposi sarcoma-associated herpesvirus infection and Kaposi sarcoma in more than 40,000 patients worldwide each year.