Grants Search Results

AML is the most common type of blood cancer. Normal blood stem cells have the capacity to produce all types of blood cells. However, impaired blood stem cells, as a consequence of genetic changes, play a central role in the initiation and progression of AML. Dr. Qian is researching how an alteration of expression of a critical gene, which is required for normal function of blood stem cells, causes AML. This study also aims to identify new therapeutic approaches to cure childhood leukemia by targeting the impaired blood stem cells.

Children who are successfully treated for cancer sometimes develop a second cancer due to treatment with radiation and chemotherapy drugs. Leukemia is one of the most common types of secondary cancer, and treatment-induced leukemia is extremely aggressive and very hard to cure. Chromosome 7 is often deleted in this type of leukemia. Dr. Shannon recently created models with deletions similar to those found in children with treatment-induced leukemia. With the Kenneth and Mary Ellen Wilson St. Baldick's Research Grant, Dr. Shannon is using these models to understand how these leukemias develop, why they are so hard to treat, and to test new therapies.

This grant is named in memory of Kenneth and Mary Ellen Wilson, parents of Todd and Jason Alonzo. Their legacy of giving and generosity lives on in the service and dedication of their children.

Rhabdomyosarcoma is the most common soft tissue sarcoma in children. Dr. Shern's group recently discovered that mutations in 10 genes drive this disease. This research grant is determining whether these mutations can be used as markers of prognosis or response to therapy. Dr. Shern hopes to develop a tool that can be used to better detect and treat rhabdomyosarcoma. If successful, the developed tool can immediately be integrated into clinical trials to improve the current therapy.

This grant is made with generous support from the PFP Fund for Cancer Research, a St. Baldrick's Hero Fund created in memory of Peyton Arens and honors his fighting spirit by supporting research that will bring about cures and less toxic treatments.

Better therapies that specifically target cancer cells while leaving normal cells undamaged will lead to therapies with fewer short or long term side effects. Cancer, specifically Ewing sarcoma, changes how proteins are made. New proteins occur by rearranging the messages that come from DNA. The rearranged messages turn into proteins that keep the cancer growing. Dr. Toretsky's research aims to find out what the rearranged messages are and how the new proteins could be targeted with new medicines.

This grant is made with generous support from the Team Clarkie Fund, a St. Baldrick's Hero Fund created to honor Clarkie Carroll. It will fund Ewing sarcoma research while stimulating greater awareness and inspiring others to believe pediatric cancer research can and will lead to a cure.

Improving the outcome of aggressive childhood leukemias depends on developing new targeted treatments. With the NetApp St. Baldrick's Research Grant, Dr. Wechsler is studying a mutant gene called CALM-AF10 that causes high-risk leukemias in children. They determined that the leukemia-causing properties of this gene depend on its interaction with a partner protein called CRM1. This research is studying the mechanisms by which CRM1 enables CALM-AF10 to cause leukemias, and the ability of drugs to inhibit CRM1 to stop leukemia growth.

This grant is named for the NetApp team, whose offices around the world have raised more than $5.8 million since 2007 for life-saving research through the St. Baldrick's Foundation.

While many children with leukemia experience good outcomes on modern therapies, there are a large number of children who still relapse and die of their disease. Using new methods, Dr. Willman identified a new form of leukemia, called Ph-like ALL, which has a variety of gene mutations which code for enzymes. Currently, drugs that inhibit these enzymes are available for clinical trials. Dr. Willman is testing these drugs in children with high risk leukemia. This research aims to extend Dr. Willman's current studies to include children with standard risk leukemia in order to also improve their chances of survival.

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.      

T-cell acute lymphoblastic leukemia (T-ALL) is an aggressive cancer that requires treatment with highly intensive chemotherapy. The prognosis of patients with relapsed and refractory (treatment resistant) T-ALL is very poor. Dr. Ferrando and his team have identified that specific mutations in the NT5C2 gene lead to chemotherapy resistance in 20% of relapse T-ALL cases. The goal of this research is to generate a model of chemotherapy resistance driven by mutant NT5C2 genes and utilize this model to develop new tailored therapies for the treatment of relapsed T-ALL.      

Hypodiploid acute lymphoblastic leukemia (ALL), in which the leukemic cells have lost multiple chromosomes, is associated with poor outcome. Dr. Mullighan and his team identified multiple new gene mutations that have not previously been recognized in this disease. Dr. Mullighan is investigating the impact of the identified mutations on leukemia formation, and investigating therapeutic alternatives for this high-risk leukemia. PI was initially Dr. Linda Holmfeldt.

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.

Alveolar rhabdomyosarcoma is an aggressive childhood cancer that often arises in muscle. It contains a DNA error which drives cells to divide when they shouldn't, resulting in cancer. Dr. Linardic has discovered one targetable protein that is controlled by this DNA error. Her work aims to understand how this protein and the DNA error associated with Alveolar Rhabdomyosarcoma are related, and whether the protein she discovered will be a useful drug target.

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.

Many currently used chemotherapeutic drugs have severe toxic side effects and a significant number of cancer patients do not respond well to chemotherapy. Thus, developing more effective and less harmful new anticancer drugs remains significant and challenging. Dr. Zhou's team has discovered several small molecule chemical compounds that are stronger to kill cancer cells, and less toxic to normal cells than currently used chemotherapeutic drugs. Dr. Zhou is studying the mechanism by which these compounds kill cancer but not normal human cells, and how to develop these compounds as effective and safe therapeutic drugs for treating refractory cancer patients.

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.

Recurrence of childhood Acute Myeloid Leukemia (AML) is all too frequent after initially successful treatment. The underlying drug resistance is partly related to the protective role of the bone marrow microenvironment, where leukemia cells grow. Dr. Kurre's team has recently discovered that AML cells release small amounts of material in the bone marrow microenvironment that cause changes to promote leukemia progression. Dr. Kurre is working to better understand these changes and how these changes can reprogram the leukemia bone marrow to protect residual AML cells and lead to relapse.

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.

NUT midline carcinoma is a deadly cancer of children and adolescents. This cancer is caused by a cancer gene called BRD4-NUT, but it cannot work without the help of other cancer genes. BRD4-NUT itself cannot be targeted very effectively with known cancer drugs. Dr. French is working to identify the cancer genes that are helping BRD4-NUT so that we can effectively treat NUT midline carcinoma with drugs that interfere with these cancer genes.