Grants Search Results

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.

Retinoblastoma is a cancer of the eye in children that can cause blindness or death. New therapies are needed to combine with existing drugs to save vision, eyes, and lives. Dr. Corson has developed a new chemical that blocks abnormal blood vessel growth in the eye without side effects. By blocking new blood vessel formation, we could starve a growing retinoblastoma tumor of oxygen and nutrients and stop its growth. To determine this, Dr. Corson's research aims to test this new chemical in a model of retinoblastoma, both alone and in combination with an existing drug.

Medulloblastoma is the most common malignant brain cancer in children. Dr. Erdeich-Epstein's research aims to improve medulloblastoma treatment based on its biology. Dr. Erdeich-Epstein and colleagues have identified PID1 as a candidate tumor suppressor after finding that higher PID1 killed medulloblastoma cells and slowed medulloblastoma growth in models. This research is to examine PID1 further to help develop drugs to improve response of medulloblastoma to treatment.

Over the last decades the introduction of multi-agent chemotherapy protocols have resulted in major advances in the treatment of pediatric acute lymphoblastic leukemia (ALL). However the prognosis of patients whose leukemia relapses after an initial transient response to therapy remains highly unsatisfactory with cure rates of less than 40% despite intensive treatment. Dr. Ferrando's research aims to address fundamental questions on the role and mechanisms of genetic mutations associated with chemotherapy resistance to pave the way for the development of improved therapies for the treatment of relapsed ALL patients.

Dr. French's research focuses on a deadly cancer of children and young adults called NUT midline carcinoma. A while back Dr. French discovered the cancer protein that causes this disease, called BRD4-NUT. This discovery led to the development of inhibitors to BRD4 that are now being used to treat NUT midline carcinoma and this has expanded to the treatment of more common cancers in clinical trials. However, NUT midline carcinoma remains incurable. Recently, Dr. French discovered an additional and completely new protein that associates with BRD4-NUT, called a ZNF532, that helps BRD4-NUT cause this cancer. This research aims to understand how this new cancer protein contributes to the malignancy in this disease.

Alveolar rhabdomyosarcoma (aRMS) is a cancer of the soft tissues. This disease often responds to chemotherapy, but in many patients available treatments fail. Dr. Keller's lab identified how a cancer-causing gene called Pax3:Foxo1 leads to treatment failure. Remarkably, Pax3:Foxo1 can be silenced by entinostat, a drug recently granted FDA breakthrough designation for breast cancer. Dr. Keller has found that entinostat dramatically improves sensitivity to chemotherapy, but efficacy in relapsed disease is unknown. This research aims to identify whether entinostat makes relapsed aRMS sensitive to chemotherapy, paving the way for an evidence-based clinical trial that could save lives.

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.

The causes of relapse of acute lymphocytic leukemia (ALL) remain unknown and there are limited therapies for such children. Dr. Licht characterized a mutation of a gene present in 10-20% of children with relapsed ALL. A mutation in this gene that may cause relapse by reprograming cells to grow more rapidly and become chemotherapy-resistant. Using a new technique Dr. Licht's Do It for Dominic St. Baldrick’s Research Grant aims to determine how the mutation increases growth and causes drug resistance in order to devise new therapies for relapsed ALL.

The grant is named for the Do It for Dominic Hero Fund created in memory of Dominic Cairo. His family and friends continue to raise funds and support St. Baldrick’s to find cures for childhood cancers so no child ever has to go through what Dominic had to endure.

Rhabdomyosarcoma is an aggressive childhood cancer that often arises in muscle. Because of the poor survival, researchers are searching for genes underlying this cancer, with the hope of blocking them. A research scientist who studies how normal muscle forms found that a gene called MEST helps in defining muscle cell precursor identity. Dr. Linardic's lab had noticed that in rhabdomyosarcoma, MEST was highly expressed, suggesting that maybe MEST tricks cells into constantly growing. This project focuses on learning how MEST contributes to rhabdomyosarcoma, and how to block it.

Childhood acute myeloid leukemia (AML) is the second most common leukemia in children, but causes the majority of leukemia related deaths. Current therapies do not adequately treat the disease and more than half of all children diagnosed with AML eventually relapse and die of their disease. Dr. Meshinchi recently performed a comprehensive genetic analysis of pediatric AML and identified mutations in CSF3R gene, the first pediatric-specific mutation in AML. This JJ's Angels St. Baldrick's Research Grant aims to study the function of this mutation to learn how it might be used as a therapeutic target.

This grant is named for the JJ's Angels Hero Fund created in memory of Julianna LaMonica to honor her joyful spirit and strength even in the midst of her battle with cancer. Her story continues to inspire so many to support the cause.

Hepatoblastoma is the most common childhood liver cancer. Dr. Prochwnik is using models to investigate two mutant genes, SIRT3 and SIRT4, which normally work to balance metabolism and energy stores, that are commonly found in hepatoblastoma. This work aims to identify novel targets for cancer therapies in hepatoblastoma and other cancers.

Juvenile myelomonocytic leukemia (JMML) is a fatal childhood myeloid malignancy with limited therapeutic options. Relapse remains the main cause of treatment failure, most likely due to the persistence of leukemic stem cells, a small population of self-renewing precursor cells that are responsible for long-term maintenance of leukemia growth and drug resistance. This research tests for the therapeutic effects of Stat5 inhibition by pimozide, a clinically used antipsychotic drug, in a subtype of JMML caused by mutations in Ptpn11.

Acute Myeloid Leukemia is an aggressive form of childhood cancer for which the therapy causes many side effects and the survival is 60%. Dr. Sakamoto's lab has found that a protein known as CREB is overproduced in AML cells and is associated with a worse prognosis. This research aims to study the protein RSK, which increases CREB activity in AML cells, and find ways to block RSK as a new approach to treat AML.

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.

Dr. Sondel has been using an immune-based therapy to treat children with cancer, and is seeing that it is clearly helping some patients. He recently found the presence of an antibody seems to predict which patients will do best with this treatment. This research aims to understand what this antibody is recognizing, and then to determine how it is helping the treatment to work better.

The most common childhood brain tumors are called gliomas. Despite advances in research, the prognosis for the aggressive pediatric gliomas remains poor. Dr. Song is studying how genes important in normal brain development are hijacked to cause brain cells to grow into a tumor and why these tumors are so resistant to treatment. This research aims to increase understanding of the biology of pediatric malignant gliomas and help develop new treatments that will ultimately improve outcome of these fatal tumors.

This grant is made with generous support from the McKenna Claire Foundation, a St. Baldrick's partner 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.

Ewing sarcoma is a type of cancer that mostly affects children and your adults. This cancer is caused by a type of mutation in DNA called a translocation. Dr. Sweet-Cordero's Team Clarkie St. Baldrick's Research Grant aims to understand how this translocation, called EWS-FLI1, causes cancer in order to find better therapies for children with this disease.

This grant is named for the Team Clarkie Fund created to honor Clarkie Carroll and funds Ewing’s sarcoma research while stimulating greater awareness and inspiring others to believe pediatric cancer research can and will lead to a cure.

Medulloblastoma is the most aggressive brain tumor in children. Finding new therapies depends upon a better understanding of the biological mechanisms of medulloblastoma formation. As the recipient of the Hannah's Heroes St. Baldrick's Research Grant, Dr. Van Meir is evaluating the role of a tumor suppressor in medulloblastoma tumorigenesis. Understanding the role of this suppressor could lead to novel therapeutic prospects for children with medulloblastoma.

This grant is named for the Hannah's Heroes Hero Fund created in honor of Hannah Meeson and pays tribute to her fight by raising awareness and funding for all childhood cancers.