Grants Search Results

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.

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.

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.

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.

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.

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 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.

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.

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.

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.

Genetic studies have identified the molecular causes of childhood cancers such as T-cell leukemia and neuroblastoma. A recurrent theme in these cancers is abnormal activation of the MYC gene. Accordingly, researchers like Dr. Wendel have spent much time and effort in trying to identify inhibitors of MYC as they believe these could be very powerful therapies for these childhood cancers. Dr. Wendel and his colleagues recently found a new way to block the production of MYC using a natural compound. The natural product is rare and hard to come by and therefore Dr. Wendel and his colleagues are exploring ways to generate synthetic drugs based on this plant product. With support from the St. Baldrick’s Foundation they are working to bring this new strategy to the clinic. They focus especially on heavily pre-treated and relapsed childhood leukemia because affected children have few options and they hope to make a difference.

The chemotherapy drugs used to treat cancer do not always work, because only a small amount of the drug ever gets to the tumor. Also, these drugs are very toxic to the patient. However, if we package the drugs into little packets called nanoparticles, we get a lot more drug into the tumor because their blood vessels are leaky. Also, the nanoparticles are too big to get into most normal tissues. Dr. Brodeur's Invictus Fund St. Baldrick’s Research Grant aims to find successful ways to give less total drug, have a much greater effect on the tumor, and have much less toxicity to the patient.

This grant is funded by and named for the Invictus Fund, a St. Baldrick's Hero Fund created in memory of Holden Gilkinson and honors his unconquerable spirit in his battle with bilateral Wilms tumor by funding cures and treatments to mitigate side and late effects of childhood cancer.

Leukemia is a form of blood cancer, and is the most common cancer in children. Overall, cure rates for children with some forms of leukemia is very high (almost 90%). However, when leukemia occurs in babies, the cure rate is only 40%. Dr. Brown has discovered that these leukemias may be harder to cure because the cancer cells have abnormal ways of organizing their DNA. Dr. Brown's research aims to understand this better to develop new treatments that will reverse this abnormal DNA organization and make the leukemias easier to cure.

Graft-versus-host Disease (GvHD) is a common devastating side-effect of bone marrow transplantation. More than 50% of children suffer from moderate to life-threatening GvHD after bone marrow transplantation. GvHD results when the primary leukemia-fighting cells in the transplants become overzealous and begin to attack not only the leukemia, but also the patient’s skin, intestines, liver, and mucosa. Dr. Choi has demonstrated that modulating a gene called interferon gamma receptor in these cells preserves the potent anti-leukemia activities while inhibiting GvHD. Dr. Choi's Rays of Hope St. Baldrick’s Research Grant aims to identify the mechanism underlying the actions of this gene to develop safe and efficient therapeutic strategies.

This grant is named for the Rays of Hope Hero Fund which honors the memory of Rayanna Marrero by giving hope through research funding. She is remembered for her infectious smile and energetic spirit which continue to inspire so many.

Rhabdomyosarcoma (RMS) is a devastating childhood cancer of muscle. Using models, Dr. Langenau recently identified NOTCH as a critical driver of RMS growth. This research aims to examine NOTCH inhitors for anti-tumor effects in RMS, providing rationale for moving these drugs into phase I clinical trials.

T-lymphoblastic leukemia is a blood cancer with a higher rate of chemo-resistance and central nervous system involvement than B cell leukemia. Dr. Letterio's research utilizes models and molecular biology techniques to test how a novel molecular target controls the development of acute T-lymphoblastic leukemia in the bone marrow and the central nervous system. Success in this new area of research will offer new hope in the development of novel therapies against T-cell leukemia.

Ewing sarcoma is the second most common bone tumor in children and adolescents, and overall survival is dismal for patients with recurrent disease. Dr. Shelat recently identified an "Achilles’ heel" in this cancer, and showed that exploiting this weakness using a three drug cocktail cured the disease more than 80% of the time in models. This project is to validate this weakness as a marker for sensitivity to the drug cocktail in Ewing sarcoma and other pediatric cancers, and to find new drug combinations that better target this weakness.