Grants Search Results

This grant supports the Defeat Pediatric Brain Tumors Research Collaborative, focused on helping kids with high-grade gliomas. The Collaborative is made up of four teams that study new targets for medicines, search brain cancer biomarkers, and conduct clinical trials, all to accelerate scientific discovery in pediatric brain tumors.

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.

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.

Unrelieved symptoms lead to poorer quality of life for adolescents and young adults (AYAs) with cancer. Strategies are needed to help AYAs manage symptoms. Dr. Ameringer and her team have developed the Computerized Symptom Capture Tool (C-SCAT), a novel way to assess symptoms where AYAs create a picture of their symptoms using images and text on an app. Dr. Ameringer is testing whether use of the C-SCAT improves the self-management of symptoms and patient-provider communication about symptoms in AYAs with cancer. Dr. Ameringer's findings will inform a larger trial of the C-SCAT as a strategy to improve AYA symptom self-management, and in turn, quality of life.

Young age at diagnosis and intense therapy result in multiple late effects for Neuroblastoma survivors. The majority of High-Risk Neuroblastoma HR-NBL survivors have striking growth failure. Dr. Mostoufi-Moab is investigating the mechanism of growth failure in high-risk neuroblastoma survivors. Dr. Mostoufi-Moab is employing state-of-the art imaging measures of the growth plate to study and understand the mechanism of growth failure in high-risk neuroblastoma survivors, and target future intervention trials.

Adolescents and young adults (AYA) with cancer have an increased risk of developing secondary cancers, cardiovascular, metabolic and bone diseases as well as cognitive impairments, which can reduce their survival and quality of life. Furthermore, most AYA cancer survivors do not meet the recommended guidelines for physical activity. Dr. Nock is conducting a pilot study using 'cybercycling' (stationary cycling with interactive video gaming) to improve quality of life in AYA cancer survivors. She will also see if this exercise program improves their motivation to exercise, body composition (weight, body fat), fatigue, depression, and sleep habits.

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.

Drug resistance remains a major obstacle in acute lymphoblastic leukemia (ALL). Instead of targeting only the leukemia cells, Dr. Kim is studying the protective non-leukemia cells that are located in the bone marrow, creating a safe haven for drug-resistant ALL cells. Dr. Kim's team has identified a molecule in leukemia cells that allows leukemia cells to remain in the bone marrow and shelters them from the otherwise toxic effects of chemotherapy. Dr. Kim's Johnny Crisstopher Children’s Charitable Foundation St. Baldrick’s Research Grant is testing a novel inhibitor of this molecule to overcome drug resistance. The mission of the Johnny Crisstopher Children's Charitable Foundation is to raise awareness of pediatric cancer and provide funds for research, treatment, and - ultimately - a cure. Famed illusionist Criss Angel founded the foundation in 2008 for charitable causes but it has now become his life's mission since his son, Johnny Crisstopher was diagnosed with leukemia in 2015 at 20 months old.

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.

Medulloblastoma is formed by mutations that activate the Hedgehog signaling pathway. Dr. Vokes is investigating how the Hedgehog pathway controls the expression of genes through specific control regions of DNA. Dr. Vokes and his team are studying those DNA control regions in medulloblastoma cells, to determine if they can control the expression of target genes, thereby providing a possible therapeutic target for medulloblastoma.

Medulloblastoma (MB) is the most common malignant pediatric brain tumor, yet currently has no optimal treatment options. Medulloblastoma has been classified into 4 major subgroups, and Dr. Chen is targeting methylation mutations to develop improved therapeutics for two highly-aggressive subgroups of medulloblastoma. To facilitate this, Dr. Chen is establishing precision models of this disease to screen and test for therapeutics. To systematically identify protein targets required for survival of MB cells, Dr. Chen and colleagues are performing a genome screen to look for possible targets, in order to enhance understanding of this disease and lead to novel therapeutic routes.

Medulloblastoma (MB) is a highly aggressive pediatric brain tumor for which safer and more effective therapies are needed. Recent studies have identified four major forms of MB that differ in terms of molecular characteristics and patient outcomes. Dr. Wechsler-Reya is working to identify genes that drive Group 4 MB tumor formation, the most prevalent form of MB, to develop new strategies for treatment of this devastating disease.

Diffuse Intrinsic Pontine Glioma is a type of incurable brain tumor that affects young children. Despite treatment with radiation and chemotherapy, the tumor exhibits resistance to current treatments and grows back. Dr. Beroukhim is studying the tumors at a single-cell level to determine how they become resistant to treatments, which will help guide the development of combination therapies to improve outcomes.

This grant is made with generous support from the McKenna Claire Foundation established by the Wetzel family in memory of their daughter, McKenna. Their mission is to cure pediatric brain cancer by raising awareness, increasing community involvement and funding research.

Treatment of childhood acute lymphoblastic leukemia (ALL) using chemoradiation can be successful, but it is difficult to manage treatment-associated side events and secondary cancers. Furthermore, in relapsed/refractory patients, the overall prognosis remains dismal. Direct inhibition of the main proteins promoting cancer (the 'oncogenes') is not successful in ALL. Dr. Ntziachristos's "Just Do It...and be done with it" St. Baldrick's Research Grant will study certain oncogene-supporting mechanisms that might be specific to a diseased state, and not to a healthy state. Dr. Ntziachristos has selected one of these mechanisms to target in ALL models, and is assessing the anti-cancer activity that results. Such experiments could pave the way for clinical trials for high-risk disease.

This grant is named for the "Just Do It...and be done with it" Hero Fund created in honor of Sara Martorano who doesn''t let anything dim her sparkle and has a compassionate heart and smile. It also celebrates the courage of all cancer kids through treatment and the support of their family and friends.

The interaction between two important cancer-related proteins called MDM4 and TOP2A may cause cancer and contribute to disease progression. Dr. Zhou is studying the regulation of MDM4 and TOP2A to identify small-molecule inhibitors (agents) that can block the MDM4-TOP2A interaction, leading to inhibition of these two proteins. The results of these studies will provide important clues to help scientists develop novel methods and drugs to specifically and simultaneously target TOP2A and MDM4 for treatment of pediatric cancer patients.

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.

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.

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.

Ewing sarcoma is a cancer of bone and soft tissue in children and young adults, which is fatal if untreated. While often successful, therapies for Ewing sarcoma have severe long-term side effects for survivors. Dr. Lombard is testing a new means of treating Ewing sarcoma, by targeting the mitochondrion, the metabolic hub of the cell. Dr. Lombard and his team have early results to suggest that this new approach may provide a way to efficiently kill Ewing sarcoma cells, with little impact on normal cells and organs.

This grant is named for Love Your Melon, an apparel brand dedicated to giving a hat to every child battling cancer in America as well as supporting nonprofit organizations who lead the fight against pediatric cancer.

Osteosarcoma is the most common bone cancer in children. Typical treatment includes cancer-killing drugs for several weeks followed by surgery. These drugs work for some patients but not for others. Doctors need a way to identify which patients respond to treatment and which don’t. Dr. Roblyer is studying the efficacy of a new light-based technology to determine when and if patients respond to treatment. This technology is low-cost, fast, and measurements are taken with a hand-held or wearable probe, like a Fitbit for cancer. If successful, this research will provide doctors with a new and simple method to personalize and improve treatment for each child with osteosarcoma.