Showing 1-20 of 559 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.

Corey Falcon M.D.
Funded: 07-01-2017 through 06-30-2019
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.

New Approaches to Neuroblastoma Therapy (NANT) Consortium Member
Funded: 07-01-2017 through 06-30-2018
Funding Type: Consortium Research Grant
Institution Location: Atlanta, GA
Institution: Children's Healthcare of Atlanta affiliated with Aflac Cancer Center, Emory 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: Chicago, IL
Institution: Comer Children's Hospital affiliated with The University of Chicago

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: Boston, MA
Institution: Boston Children's Hospital affiliated with Dana-Farber Cancer Institute, Harvard Medical School

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.

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.