Grants Search Results

Based on progress to date, Dr. Gowda was awarded a new grant in 2020 to fund an additional year of this Scholar grant. Children with high risk B-cell leukemia, especially with loss or dysfunction of IKZF1 gene have very poor outcomes and high relapse rate. Every other child who relapses with high risk leukemia dies from the disease and there has not been much advancement in treatment for this group for the last 30 years. Dr. Gowda and team have found that a cancer promoting protein called casein kinase II (CK2) impairs the important functions of a protein that helps prevent leukemia. Inhibiting the CK2 protein will restore the ability of this protein to function properly and prevent leukemia. Dr. Gowda's team is testing if using a drug that inhibits CK2 protein along with the drugs that already are known to work in leukemia will have stronger anti-leukemia effect and improve the outcome. Using two agents that target same gene or pathway via different mechanisms will ensure effective shutdown of the particular pathway resulting in strong therapeutic effect. This strategy would also help lower the doses of each drug used and reduce their side effects and associated toxicity.

Based on progress to date, Dr. Agarwal was awarded a new grant in 2020 to fund an additional year of this Scholar grant. High-risk neuroblastoma is an aggressive cancer of very young children with less than 50% overall survival. Current therapy includes high-dose chemotherapy and radiation, which has long-term toxic side-effects. Despite these intensive therapies, neuroblastoma commonly relapse. This relapse is the primary cause of death from neuroblastoma due to disease spread, drug-resistance, and toxicity. As the Oliver Wells Fund for Neuroblatoma St. Baldrick's Scholar, Dr. Agarwal is focusing his research on developing effective therapeutic approaches to target those tumor cells which escape initial treatment and regenerate drug-resistant disease. Recently, Dr. Agarwal's team discovered a chemotherapy-resistant, highly tumorigenic sub-population of cells in neuroblastoma tumors. These cells escape initial therapy and may cause aggressive, drug-resistant relapsed disease. Furthermore, they found that specific epigenetic enzymes maintain this cell sub-population by activating key genes. These epigenetic modifiers can be successfully targeted with novel epigenetic inhibitors, currently under pre-clinical trials. These exciting findings suggest a new epigenetic therapeutic approach for high-risk neuroblastoma. This grant supports efforts to uncover the mechanisms controlling neuroblastoma tumorigenicity and relapse, and develop an effective targeted approach for high-risk neuroblastoma.

A portion of this grant is funded by and named for the Oliver Wells Fund for Neuroblastoma, a St. Baldrick's Hero Fund. From the moment he was born, Ollie was the center of the Wells family with a contagious smile and a sparkle in his eyes. As the youngest child, it was devastating when they learned the 15 year old toddler had cancer. Oliver was diagnosed with high risk neuroblastoma and spent the next 13 months bravely enduring chemotherapy and radiation, more than a dozen surgeries and a bone marrow transplant. But Ollie persevered and smiled through it all. It was an unfair fight from the beginning and in July 2018, Ollie passed away. The Oliver Wells Fund for Neuroblastoma was established in his memory to raise funds to find cures and give hope to other kids facing the same fight. In this way, the Wells family intends to share Oliver’s joy for life and use his story to help find a cure. 

A portion of this grant was also funded by and named for David's Warriors, a St. Baldrick's Hero Fund. The fund was created in memory of David Heard who battled neuroblastoma until his passing at the age of ten. David inspired his family and countless others to commit to raising money for research to fight pediatric cancer through the St. Baldrick’s Foundation. The Fund honors the amazing spirit with which he lived, embracing life until the very end.

Awarded at the Baylor College of Medicine, and transferred to St. John's University.

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.

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.

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.

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.

Neuroblastoma is a common type of childhood cancer that arises from nerve-like cells and causes tumors just outside the brain. Dr. George is studying tumor cells from patients to discover new molecular targets for treatment of neuroblastoma. Dr. George and his team are growing tumor cells from patients and examining their electrical properties in order to identify specific proteins that carry electrical current that may be new targets for treating neuroblastoma using existing non-cancer drugs.

Relatively little is currently known about the cellular and molecular mechanisms of medulloblastoma spread throughout the spinal fluid. Dr. Keller's preliminary findings show that medulloblastoma tumor cells actively migrate to the surface of the covering of the brain and spine, rather than arriving passively. Dr. Keller is investigating the efficacy of stopping the tumor cells from migrating to the brain and spine surfaces, which, if successful, will provide a rationale for future clinical trials.

Dr. Wechsler and his team study how normal white blood cells turn into leukemia cells. They have discovered that a protein called CRM1 is directly involved in turning on specific genes that cause leukemia to develop. Dr. Wechsler is studying this previously unrecognized function of CRM1 by identifying other proteins that are required for CRM1 to turn on. This research aims to allow Dr. Wechsler to design new drugs that can selectively target leukemia cells and increase cure rates.

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.

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.

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.

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.

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.

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!

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.

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.

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.

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.