Grants Search Results

Based on progress to date, Dr. Gramatges was awarded new grants in 2016 and 2017 to fund additional years of this Scholar award. Acute myeloid leukemia (AML) is treated with intensive chemotherapy that results in treatment-related toxicities in 80% of patients, some so severe that the patient does not survive therapy. Dr. Gramatges's research investigates genetic markers characterizing the subpopulation of children and young adults with AML who are at risk for severe treatment-related toxicities. Validation of these markers may lead to upfront screening of individuals with newly diagnosed AML, and in cases where these markers are discovered, modifications to the treatment regimen and closer monitoring to reduce treatment-related morbidity and mortality in this disease.

Based on progress to date, Dr. Ladle was awarded a new grant in 2015 to fund an optional third year of this fellowship. While the body's immune system is capable of attacking cancer, many factors prevent this from happening. The goal of Dr. Ladle's research is to develop a vaccine to be given to patients that activates their own immune system to treat their cancer. The project focuses on how the body regulates the immune system to normally ignore cancer. New drugs are being developed that could help take the brakes off the immune system and allow it to recognize and attack cancer. Combining these drugs with a cancer vaccine could provide the boost needed for immune therapies to effectively treat pediatric cancers.

Based on progress to date, Dr. Schuback was awarded a new grant in 2015 to fund an optional third year of this fellowship. Dr. Schuback's research aims to improve treatment for children with Acute Myeloid Leukemia (AML), a type of blood cancer. This work focuses on characterizing the scope of mutations in a specific gene, ETV6, in children with AML. Preliminary work indicates that patients with such mutations are more likely to have a poor outcome. This project hopes to use the mutations in ETV6 as a marker to identify patients at high risk of relapse at the beginning of their treatment, in order to predetermine therapies that are most likely to succeed.

AML is a devastating form of leukemia. Therapy for AML is highly toxic and, still, only a minority of patients survive. This project aims to develop new, less toxic, and more effective therapies for AML. Dr. Salstrom hopes to use models to determine exactly which therapies will work best for which patients. This approach, called personalized medicine, allows researchers to treat each child's individual leukemia in the most effective and safest way possible.

Based on progress to date, Dr. Choo, the Tap Cancer Out St. Baldrick’s Fellow, was awarded a new grant in 2015 to fund an optional third year of this fellowship. Ewing sarcoma is a bone and soft tissue cancer that occurs in adolescent and young adults (AYAs). When the cancer spreads (metastasis), survival falls below 30% despite aggressive chemotherapy and surgery. Fortunately, promising data has identified certain genes that are specifically turned on in metastatic Ewing cells. By developing targeted therapy against these gene products, Dr. Choo hopes to effectively treat Ewing sarcoma. In addition, targeting this unique pathway may reduce the use of conventional toxic chemotherapy agents that can cause cancer themselves. Ultimately, this research may help reduce both morbidity and save countless children with metastatic Ewing sarcoma. This grant recognizes the partnership with Tap Cancer Out, a jiu-jitsu based 501(c)(3) nonprofit raising awareness and funds for cancer fighting organizations on behalf of the grappling community.

Based on progress to date, Dr. Velasquez was awarded a new grant in 2015 to fund an optional third year of this fellowship. Cancer treatments consisting of the infusion of T cells (one component of the patient's own immune system) that recognize CD19 (a molecule present on many blood cancers) have shown promise in early clinical studies. However, not all patients currently benefit from CD19-specific T-cell infusions. Dr. Velasquez's lab is conducting studies to optimize a new genetic approach with the ultimate goal of developing a clinical study to address this issue.

Based on progress to date, Dr. Fabbri was awarded new grants in 2016 and 2017 to fund additional years of this Scholar award. In neuroblastoma, a common childhood solid cancer, immune cells called tumor infiltrating macrophages (TAMs) promote neuroblastoma growth and spread. The mechanism for this process is unknown. Dr. Fabbri has shown in another type of cancer that cancer cells secrete special genes that induce TAMs to release a signal that promotes cancer growth. Dr. Fabbri is investigating how these genes and TAMs affect neuroblastoma growth, and testing what drugs can interrupt this process, with the goal of improving neuroblastoma treatment.

Based on progress to date, Dr. Norris was awarded new grants in 2016 and 2018 to fund additional years of this Scholar award. More than 70% of children diagnosed with cancer are cured of their disease, but today's therapies can have severe and life-long side effects, and too many children die from cancer. Cyclin dependent kinase-5 (Cdk5) inhibitors are a new type of therapy with the potential to treat childhood cancer. Dr. Norris, the Rebecca Alison Meyer Fund for Pediatric Cancer Research St. Baldrick’s Scholar, uses laboratory and computer models to determine how to optimize therapy with Cdk5 inhibitors, and how to combine Cdk5 inhibitors with current cancer treatments. Using this information, Dr. Norris studies Cdk5 inhibitors in adolescents with relapsed cancer, with the goal of developing new treatments for children with cancer. A portion of this grant is named for The Rebecca Alison Meyer Fund for Pediatric Cancer Research created to honor the memory of the joyful and spunky little girl who courageously battled brain cancer. Rebecca’s legacy lives on in the funding of promising glioblastoma research. Awarded at Rainbow Babies and Children's Hospital and transferred to Cincinnati Children's Hospital.

Based on progress to date, Dr. Crompton was awarded new grants in 2016 and 2017 to fund additional years of this Scholar award. Ewing sarcoma is an aggressive bone tumor affecting adolescents and young adults. Current treatment regimens fail to improve outcomes for patients with high-risk disease, and new therapeutic approaches are needed. Dr. Crompton's team recently identified a protein that is highly active in Ewing sarcoma and is targeted by drugs in clinical development. Dr. Crompton, the Team Clarkie Fund St. Baldrick’s Scholar, aims to demonstrate that these inhibitors warrant testing in clinical trials for patients with Ewing sarcoma, define the clinical indications for their use, and identify the most effective treatment combinations. Lastly, the project will develop a new screening effort to identify additional drug targets in Ewing sarcoma. A portion of this grant is named for the Team Clarkie Fund created to honor Clarkie Carroll and fund Ewing’s sarcoma research while stimulating greater awareness and inspiring others to believe pediatric cancer research can and will lead to a cure.

Based on progress to date, Dr. Maude was awarded new grants in 2016 and 2017 to fund additional years of this Scholar award. Acute lymphoblastic leukemia (ALL), a cancer of white blood cells, is the most common childhood cancer. Fortunately, most children with ALL are cured; however, 10-20% of children are not cured with standard chemotherapy. Recently, genetic tests identified abnormalities that may cause two types of ALL with poor survival rates. Dr. Maude's lab developed models of these leukemias to ask if new medicines that work specifically on the abnormal genes can improve the chance for cure. Dr. Maude, the Rally for Ryan Fund St. Baldrick's Scholar, hopes that these studies will find new treatments for these difficult to treat leukemias, giving these children a new chance for cure. This grant is named for the Rally for Ryan Fund. Ryan was diagnosed with high risk ALL when he was 7 years old. He endured 3 ½ years of often harsh treatments with smiles, laughter and a brave acceptance that this was his fight to win. And Ryan did prevail—he took his last chemo pill in January 2016 but sadly, relapsed later that year. He is currently in treatment and back in the fight. This fund honors Ryan’s commitment to help make a difference for kids with cancer by shaving for St. Baldrick’s and to raise funds for research.

Based on progress to date, Dr. Mizukawa was awarded new grants in 2016 and 2017 to fund additional years of this Scholar award. Acute myeloid leukemia (AML) arises from a subset of leukemia stem cells that are responsible for perpetuating the disease. Although most leukemia cells are readily killed by chemotherapy, if the leukemia stem cell escapes therapy, the child will eventually succumb to the disease. The leukemia stem cell has survival and self-renewal advantages provided by its supportive environment. The Cdc42 protein plays a central role in integrating environmental cues and promoting leukemia cell survival. Dr. Mizukawa is working to block Cdc42 and make the leukemia stem cell more sensitive to chemotherapy.

Based on progress to date, Dr. Lubega was awarded new grants in 2016 and 2017 to fund additional years of this International Scholar award. A third of cancers in children in Africa are due to Burkitt's lymphoma. Burkitt's lymphoma seems to arise from the body's attempt to fight Epstein-Barr virus and malaria infections. This study measures infection-fighting proteins in children with and without Burkitt's lymphoma. The goal is to discover if these infection-fighting proteins in blood or saliva can be used as specific indicators of Burkitt's lymphoma. These proteins can be developed into clinical tests for early detection and monitoring treatment for children with cancer.

Rhabdomyosarcoma (RMS) is the most common soft tissue sarcoma of childhood and some subtypes are highly aggressive and spread to different organs. Current treatment strategies include surgery, radiation and chemotherapy, and clinical trials combining these modalities still result in only 30% survival. Advances in cancer genome studies have identified several genetic changes that are crucial for aggressive tumor growth and spread of the disease. Some of these genetic changes result in display of specific proteins on the tumor cell surface. Development and preclinical evaluation of monoclonal antibodies against such tumor-specific molecules would open the door for a variety of targeted therapeutics and novel treatment options for these patients.      

Human synovial sarcoma is uniformly driven by a precise genetic lesion (change to our heritable material, or DNA), which converts a normal protein into one that functions abnormally and promotes cancer development. This research aims to identify molecules which prevent this conversion and halt synovial sarcoma growth.

Although radiation is a known carcinogen whose effects are most pronounced in children, it is ubiquitous in modern life. By studying survivors of pediatric Hodgkin lymphoma, Dr. Onel's team discovered that genetic variants regulating one gene are both very common and strongly associated with increased risk for radiation-induced cancers. Dr. Onel and his team are working to determine how radiation activates this gene, how the gene directs the response to radiation, and how variants alter this response. Dr. Onel hopes that these results will lead to new ways to identify children at risk for radiation-induced cancers, or new drugs to prevent this devastating late effect of radiation exposure.

Medulloblastoma is the most common malignant brain tumor. There is an urgent need to develop novel therapies for children with medulloblastoma. Dr. Van Meir and his team are studying the importance of the loss of tumor suppressor BAI1 in medulloblastoma. Such new knowledge has the potential to reveal new ways to treat this disease.

Neuroblastoma is a common childhood cancer. Cancers happen because of mutations (mistakes) in the genetic code within them, and knowing which specific mutation happened in each particular cancer should help doctors improve their treatments. Dr. Hogarty's team discovered that some neuroblastomas have mutations in a specific gene, ARID1, and that these tumors are especially difficult to cure. Dr. Hogarty is studying this gene more since it determines how nerve cells behave, and neuroblastoma arises from mutated nerve cells. This may give us insight into new ways to treat neuroblastoma.

Neuroblastoma is a pediatric tumor that is very difficult to treat, even with surgery and chemotherapy, because certain genes in these cancer cells are over-expressed. Dr. Malkas and her team have identified a protein that is uniquely expressed by these cancer cells, and discovered that a small portion of this protein can be used as a decoy to bind-up other proteins to selectively kill cancer cells. She is working to determine how this protein kills tumor cells, and how to make the cells more sensitive to chemotherapy.

Medulloblastoma is the most common malignant brain tumor in children. These tumors are caused by or associated with two proteins which cannot be directly attacked with drugs. However, these proteins rely on other proteins involved in the translation (the process of making more proteins) to cause cancer. Currently researchers can alter translation with drugs in clinical trials for adult cancers. Dr. Weiss's team is trying to determine how these two proteins rely on these translational proteins in medulloblastoma, and how to modulate them with currently available drugs, to halt tumor growth and destroy tumor cells.

Rhabdoid tumors are highly aggressive cancers that strike young children, for which a cure still remains elusive. In nearly all cases of rhabdoid tumors a specific tumor gene (SNF5) is mutated. But how this mutation drives rhabdoid tumor formation remains largely unknown. Dr. Wang's research investigates how this mutation eventually predisposes to cancer formation, with the ultimate goal of translating these findings to find potential therapies for this aggressive pediatric cancer. This research is funded by P.A.L.S. Bermuda with funds raised through the St. Baldrick's Foundation.