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Showing 101-120 of 160 results
Clinton Carroll M.D.
Funded: 07-01-2011
through 06-30-2013
Funding Type: St. Baldrick's Fellow
Institution Location:
Nashville, TN
Institution: Vanderbilt University Medical Center
affiliated with Monroe Carell Jr. Children's Hospital at Vanderbilt
To treat cancer effectively we must first understand why cancer develops. The body is constantly producing cells which make up our tissues. Often, when a cell develops chromosomal damage, the cell is fixed by multiple proteins referred to as the DNA Damage Response (DDR), or the cell is eliminated through a process called apoptosis. Unfortunately, some damaged cells escape these processes because the DDR does not work properly, and they go on to divide and form cancer. This project focuses on understanding one aspect of the DDR, a protein called SMARCAL1, to gain insight into why normal cells become genetically unstable and cancerous, and to uncover proteins and pathways that might serve as novel targets for cancer drugs, to help eventually eradicate of childhood cancer.
Tiffany Chang M.D.
Funded: 07-01-2011
through 06-30-2014
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. Chang was awarded a new grant in 2013 to fund an optional third year of this fellowship. Children with Neurofibromatosis Type 1 (NF1) are strongly predisposed to Juvenile Myelomonocytic Leukemia (JMML), a relentless form of cancer. Only 50% of children with JMML survive beyond 5 years, and hematopoietic stem cell transplantation currently offers the only potential for cure, although transplant-related mortality is high. Therapies targeted to specific molecular abnormalities in JMML may offer a better alternative. Ras is a protein involved in normal cellular growth as well as malignant transformation. Understanding the therapeutic effects of inhibiting Ras effector pathways will inform novel treatment strategies.
Rishikesh Chavan M.D.
Funded: 07-01-2011
through 06-30-2012
Funding Type: St. Baldrick's Fellow
Institution Location:
Houston, TX
Institution: Baylor College of Medicine
affiliated with Vannie E. Cook Jr. Children's Cancer and Hematology Clinic, Texas Children's Hospital
Cancer is caused by alterations (mutations) in the genetic material of tumor cells. The identification of these mutations has allowed the development of treatments specifically targeted at the mutated genes, resulting in remarkable clinical advances for a few specific cancer types. This proposal uses state-of-the-art sequencing technologies to analyze hepatoblastoma, the most common liver cancer of children. The goal of this project is to dramatically shift current research and treatment paradigms by directing investigators to the most relevant genes causing hepatoblastoma, which are currently largely unknown.
Rachel Thienprayoon M.D.
Funded: 07-01-2011
through 06-30-2013
Funding Type: St. Baldrick's Fellow
Institution Location:
Dallas, TX
Institution: University of Texas Southwestern Medical Center at Dallas
Pediatric palliative care is a comprehensive system of care aimed at preventing or relieving symptoms and suffering caused by a life-threatening illness. At the end of life, hospice is an important provider of palliative care. The goal of this study is to identify factors that are associated with hospice use in pediatric oncology, about which little is known. Although cure rates have dramatically increased in pediatric cancer, palliative care and hospice are an integral part of caring for the patients who do not survive. A better understanding of why parents and patients choose hospice can help improve the care of pediatric oncology patients at the end of life.
Jennifer Elster M.D.
Funded: 07-01-2011
through 06-30-2013
Funding Type: St. Baldrick's Fellow
Institution Location:
Pittsburgh, PA
Institution: University of Pittsburgh
affiliated with Children's Hospital of Pittsburgh
A growing tumor requires a blood supply, and in some tumors, such as neuroblastoma, the number of blood vessels in a tumor correlates with metastases and mortality. The formation of new blood vessels is called angiogenesis. Anti-angiogenic drugs designed to stop these blood vessels from forming have proved disappointing, so far. The lab in which Dr. Elster is working has identified one reason for this. In this project, known pharmacologically active compounds are screened to find what may be the backbone for the next class of anti-angiogenic drugs.
David Gass M.D.
Funded: 07-01-2011
through 06-30-2013
Funding Type: St. Baldrick's Fellow
Institution Location:
New York, NY
Institution: Columbia University Medical Center
affiliated with Morgan Stanley Children’s Hospital, New York-Presbyterian
Several recent studies have identified point mutations IDH1 and IDH2 in gliomas. IDH are key enzymes involved in cell metabolism. These mutations occur frequently (50-93%) in diffusely infiltrating astrocytomas and oligodendrogliomas, as well as in some glioblastomas. These studies suggest that IDH1 mutations are an early event in the formation of specific types of diffusely infiltrating gliomas, and this project uses a virus to deliver genes to determine if mutant IDH1 can induce brain tumors in models.
Michael Angelo Huang M.D.
Funded: 07-01-2011
through 06-30-2013
Funding Type: St. Baldrick's Fellow
Institution Location:
Hershey, PA
Institution: Pennsylvania State University
affiliated with Penn State Hershey Children's Hospital
Neuroblastoma is the second most common solid cancer in childhood. Half of patients are at an advanced stage at diagnosis, difficult to treat successfully even with aggressive therapy. Ferritin is the body's storage form of iron, and elevated levels have been linked to worse outcomes in patients with neuroblastoma, but this phenomenon is still poorly understood. This research is investigating how genetic mutations in HFE, the gene responsible for the iron overload disorder, promote the development and spread of neuroblastoma cells. By identifying which specific iron metabolism pathways are involved, we can come up with new therapeutic strategies that are more specific and less toxic.
Andres Morales M.D.
Funded: 07-01-2011
through 06-30-2013
Funding Type: St. Baldrick's Fellow
Institution Location:
Boston, MA
Institution: Dana-Farber Cancer Institute
affiliated with Boston Children's Hospital, Harvard Medical School
Neuroblastoma can range from spontaneous regression to relentless progression. Clinical and biological prognostic factors are used to classify patients as low-, intermediate-, or high-risk of relapse, to determine the intensity of treatment necessary. Recently, several investigators have published "genetic signatures" that correlate with outcome in children with high-risk neuroblastoma in retrospective studies, but due to expense, timing and availability, these signatures are not currently used. This project aims to circumvent these limitations, improving the care of the 40% of high-risk patients who are destined to fail current therapies. Awarded at The University of Chicago and transferred to Dana-Farber Cancer Research Institute.
Youmna Othman M.D.
Funded: 07-01-2011
through 06-30-2017
Funding Type: St. Baldrick's Fellow
Institution Location:
Cleveland, OH
Institution: Rainbow Babies and Children's Hospital
affiliated with University Hospitals of Cleveland
Significant challenges remain in the treatment of leukemia that has infiltrated into the central nervous system (CNS). The CNS serves as a sanctuary site for leukemic cells which can relapse and spread to other organs. In particular, T-cell ALL, a sub-type of acute lymphoblastic leukemia (ALL), has a strong propensity to infiltrate the CNS. Dr. Othman's research focuses on a recently identified target protein, CDK5, which has been implicated in the migration of immune cells. These potentially paradigm-shifting investigations promise the development of new biological agents or immune-mediated therapies against CNS leukemia and other devastating childhood tumors of the brain.
Edward Allan Sison M.D.
Funded: 07-01-2011
through 06-30-2013
Funding Type: St. Baldrick's Fellow
Institution Location:
Baltimore, MD
Institution: Johns Hopkins University School of Medicine
affiliated with Johns Hopkins Children's Center
Leukemia, a cancer of the white blood cells, is the most common cancer in children. While a majority can expect to be cured with chemotherapy, a significant number either never go into remission, or relapse. One theory as to why certain leukemias do this is that normal, non-cancerous cells in the bone marrow can help small populations of leukemia cells evade chemotherapy-induced death, leading to relapse. This research project focusing on a protein called CXCR4, is to find a way to make chemotherapy more effective and may lead directly to clinical trials in children with high-risk leukemias that will improve cure rates.
Jodi Skiles M.D.
Funded: 07-01-2011
through 06-30-2014
Funding Type: St. Baldrick's Fellow
Institution Location:
Indianapolis, IN
Institution: Indiana University
affiliated with Riley Hospital for Children, IU Health Proton Therapy Center
Based on progress to date, Dr. Skiles was awarded a new grant in 2013 to fund an optional third year of this fellowship. Vincristine is one of the core anticancer agents used in many childhood cancers. It is associated with cumulative dose-dependent neurotoxicity, often making it necessary to reduce chemotherapy dosage, compromising effective treatment. There is substantial variability in the way vincristine is metabolized from one person to the next. For example, Caucasians develop neurotoxicity much more frequently than African-Americans, probably as a result of genetic differences in a specific drug-metabolizing enzyme. This is of particular importance in African populations where the treatment outcome is poorer than in U.S. populations. Currently, drug dosing recommendations used in Kenya are based on dosing schemas derived in primarily Caucasian patient populations. This study helps develop individualized vincristine dosing parameters based on genetic predictors, which may lead to improved cure rates, as well as less neurotoxicity in long-term cancer survivors both in the U.S. and in Kenya.
Batul Suterwala M.B.B.S.
Funded: 07-01-2011
through 06-30-2013
Funding Type: St. Baldrick's Fellow
Institution Location:
Los Angeles, CA
Institution: Children's Hospital Los Angeles
Bone marrow transplantation is an important and potentially curative treatment for children with relapsed leukemias. Infections due to a delay in the recovery of the immune system after transplantation are a major cause of complications and even death in these children. T-cells are a type of immune cells that fight infection. Of the various components of the immune system, the T-cells are the one that recover the poorest after bone marrow transplantation. The aim of this project is to understand how VEGF improves T cell production, with the ultimate goal of improving cure rates for children with relapsed leukemias.
Ilanit Brook M.D.
Funded: 07-01-2010
through 06-30-2012
Funding Type: St. Baldrick's Fellow
Institution Location:
Los Angeles, CA
Institution: University of California, Los Angeles
affiliated with Mattel Children's Hospital
With every new pediatric cancer diagnoses, a family is given the most unexpected and stressful news of their lives. This kind of stress makes people more susceptible to post-traumatic stress and depression, and also diminishes the immune system, allowing the body to become sick more often. This project examines the level of chronic stress felt by the parents of children and the factors involved. It is vital that we work to improve the outcomes for both patients and their families.
Stuart Cramer D.O.
Funded: 07-01-2010
through 06-30-2011
Funding Type: St. Baldrick's Fellow
Institution Location:
Birmingham, AL
Institution: University of Alabama at Birmingham
affiliated with Children's of Alabama
Neuroblastoma is a cancer that develops in the nervous tissue and most cases occur in children younger than two years old. For patients with high-risk disease overall survival remains poor, thus there is a tremendous need to develop new treatments. This research will allow us to improve the clinical development of a new drug, MLN 8237, which has shown promise in early pediatric clinical trials, and could improve survival in this devastating disease and provide additional targets for therapeutic intervention.
Suzanne McGoldrick M.D.
Funded: 07-01-2010
through 06-30-2012
Funding Type: St. Baldrick's Fellow
Institution Location:
Seattle, WA
Institution: Fred Hutchinson Cancer Research Center
affiliated with University of Washington, Seattle Children's Hospital
Bone marrow transplantation is a treatment option for children with leukemia. When no bone marrow donor is available, cord blood transplantation is an alternative. This research focuses on two poorly understood aspects of the results of bone marrow transplants: First, the prolonged recovery of the recipient's new immune system. Second, the dominant cord in double cord blood transplantation, in which a patient receives cells from two cords instead of one. Understanding both of these immune processes is key to improving the care and survival of these patients.
Agne Petrosiute M.D.
Funded: 07-01-2010
through 12-31-2014
Funding Type: St. Baldrick's Fellow
Institution Location:
Cleveland, OH
Institution: Rainbow Babies and Children's Hospital
affiliated with University Hospitals of Cleveland
Based on progress to date, Dr. Petrosiute was awarded a new grant in 2013 to fund an optional third year of this fellowship. Medulloblastoma, the most common malignant brain tumor of childhood, often exhibits an aggressive growth pattern and causes high morbidity and mortality despite aggressive therapy. This project studies the role of a target protein, CDK5, and related molecules in controlling the invasion and spread of medulloblastoma. These potentially paradigm-shifting investigations promise the development of new biological agents or immune-mediated therapies against medulloblastoma and other devastating brain tumors of childhood.
Navin Pinto M.D.
Funded: 07-01-2010
through 06-30-2012
Funding Type: St. Baldrick's Fellow
Institution Location:
Chicago, IL
Institution: The University of Chicago
affiliated with Comer Children's Hospital
African-American children with neuroblastoma treated with chemotherapy die or relapse more often than Caucasian children. This research aims to find the genetic factors that may be involved. Our lab gives chemotherapy to white blood cells from healthy volunteers from all over the world. The entire genome for each of these cells is known. By comparing how sensitive or resistant these cells are to chemo against their genetic code, we can find genetic changes that are associated with chemotherapy resistance. This information will help us to personalize therapy and eventually improve cure rates.
Jennifer Pope M.D.
Funded: 07-01-2010
through 06-30-2012
Funding Type: St. Baldrick's Fellow
Institution Location:
Cincinnati, OH
Institution: Cincinnati Children's Hospital Medical Center
affiliated with University of Cincinnati College of Medicine
Normal genetic variations are responsible for differences in ability to fight infection, metabolize medications, and repair damage to our DNA. One cause of DNA damage is exposure to oxidants and free radicals, which may increase with exposure to radiation, food, and chemicals. This research is to determine some of the genes that make children with Down syndrome 10-20 times more likely to develop leukemia than other children. This could help identify children at risk and help develop new treatments. It may also determine if these genetic changes are present in children without Down syndrome who develop leukemia.
Rachel Rau M.D.
Funded: 07-01-2010
through 06-30-2012
Funding Type: St. Baldrick's Fellow
Institution Location:
Baltimore, MD
Institution: Johns Hopkins University School of Medicine
affiliated with Johns Hopkins Children's Center
Despite intense treatment, only approximately 50% of children with AML (acute myeloid leukemia) will survive. Many cases of AML have genetic abnormalities that likely contribute to the development of leukemia and impact the outcome of the patient. Two such mutations happen to occur together frequently in AML, mutations in a gene called nucleophosmin and a gene called Flt3. This research studies the relationship between these two gene mutations, to gain insight into the cause of leukemia and how best to treat patients who have these two common genetic mutations.
David Shyr M.D.
Funded: 07-01-2010
through 06-30-2011
Funding Type: St. Baldrick's Fellow
Institution Location:
Orange, CA
Institution: Children's Hospital of Orange County
To continue improving the treatment of childhood leukemia, we must broaden our understanding of cancer biology at the molecular level so we can better exploit the vulnerabilities of cancer cells. Cancer occurs when mutations disrupt the normal cellular machinery that controls cell growth. For example, the production of a protein called TCF-1 is dramatically increased during the critical phase of leukemia development. A better understanding of specific growth regulating proteins such as TCF-1 may allow development of more targeted therapies in the future.