Showing 1-20 of 30 results
James Ch'ng M.D.
Funded: 07-01-2018 through 06-30-2020
Funding Type: St. Baldrick's Fellow
Institution Location: Los Angeles, CA
Institution: University of California, Los Angeles affiliated with Mattel Children's Hospital

Epstein-Barr virus (EBV) is a common viral infection that in the vast majority of people causes only minor or no illness. However, in some situations it can play a role in the development of certain forms of cancer, such as lymphoma. One way that it might contribute to the development of cancer is by affecting the way that cells use energy because viruses and cancers both require increased energy to support rapid growth. By studying how EBV changes the way that cells use energy, Dr. Ch'ng hopes to learn whether changes in cell energy use are a factor in the development of cancers associated with EBV and whether these changes can be targeted to treat these forms of cancer.

Kelly Faulk M.D.
Funded: 07-01-2018 through 06-30-2020
Funding Type: St. Baldrick's Fellow
Institution Location: Denver, CO
Institution: University of Colorado affiliated with Children's Hospital Colorado

Leukemia is a cancer of blood cells that can be caused by a variety of genetic changes. The leukemia cells of some children have a genetic change in which a gene (KMT2A) is broken and combined with other genes that typically do not interact with one another (this is called "rearranged"). This genetic rearrangement then alters how other genes are turned on or off in the cell, turning on genes that drive the development of leukemia. This leukemia can be seen in all ages but is most common in infants. Unfortunately, it is a very aggressive leukemia with a high risk of relapse and poor overall survival. A drug named pinometostat has been developed that directly targets the underlying changes that occur with this genetic rearrangement and specifically kills these leukemia cells. When pinometostat was tested by itself in these leukemia patients, it was proven safe and showed killing of leukemia cells in some patients. Dr. Faulk and colleagues have developed a new clinical trial that combines pinometostat with standard chemotherapy for kids and young adults with relapsed or resistant leukemia with a KMT2A gene rearrangement. This trial is testing the safety of pinometostat when it is given with other chemotherapy medicines, seeing how the drug is broken down by the body, and determining how the drug affects the body. The trial is also determining if pinometostat works better to fight cancer when given with other chemotherapy rather than alone. If proven safe, the drug may then continue in further testing to better understand how effective it is in this type of leukemia. The long-term goal is to improve the cure rate for this group of high-risk leukemia patients and provide these children with more treatment options that they urgently need.

Ryan Summers M.D.
Funded: 07-01-2018 through 06-30-2020
Funding Type: St. Baldrick's Fellow
Institution Location: Atlanta, GA
Institution: Emory University affiliated with Aflac Cancer Center, Children's Healthcare of Atlanta

Early T-precursor ALL (ETP-ALL) is a type of leukemia that is often difficult to treat with currently available chemotherapy. As a result, children with ETP-ALL have high rates of relapse of their leukemia and poorer survival rates than children with other types of ALL, and require more treatment with chemotherapy, often leading to long-term toxic side effects. For these reasons, new treatments for ETP-ALL are needed. MERTK is a protein that is found on the surface of some leukemia cells, including ETP-ALLs. Recently, Dr. Summers and colleagues developed a new medicine that has few toxic side-effects and can be used to kill leukemia cells that have MERTK on their surface. Funding from the St. Baldrick's Foundation will allow him to test whether and how this new medicine could be used to more effectively treat children with ETP-ALL, leading to improved outcomes and better quality of life.

Neekesh Dharia M.D., Ph.D.
Funded: 07-01-2018 through 06-30-2020
Funding Type: St. Baldrick's Fellow
Institution Location: Boston, MA
Institution: Dana-Farber Cancer Institute affiliated with Boston Children's Hospital, Harvard Medical School

Despite progress made in the treatment of pediatric cancers, several childhood cancers, such as high-risk neuroblastoma, Ewing sarcoma and rhabdomyosarcoma, continue to have poor survival rates. It is critical that we identify new therapies for these cancers, especially now that we are learning how cancers are driven by specific changes in proteins that bind DNA and control transcription. Researchers are beginning to identify potential vulnerabilities in cancers by systematically deleting almost every single gene in a cancer cell, and describing in greater detail the mutations and other events that occur in pediatric cancers. Dr. Dharia's team is integrating data from such approaches to discover specific vulnerabilities in high-risk neuroblastoma, Ewing sarcoma and rhabdomyosarcoma. Different types of cancer cells require different instructions or programs to survive, and Dr. Dharia proposes the identification of these programs will lead to new targets to treat these cancers. By identifying, validating and characterizing new targets for treatment of these childhood cancers, Dr. Dharia hopes to discover new therapies for cancer care. This research will take advantage of drugs that are already available and ideally identify completely new ways to treat these cancers.

Daniel Peltier M.D., Ph.D.
Funded: 07-01-2018 through 06-30-2020
Funding Type: St. Baldrick's Fellow
Institution Location: Ann Arbor, MI
Institution: University of Michigan affiliated with C.S. Mott Children’s Hospital

Bone marrow transplantation (BMT) is required to cure many childhood cancers. However, bone marrow transplantation is often complicated by severe and often fatal side effects. Both the beneficial anti-cancer effects and harmful side effects of bone marrow transplantation are due in part to the new immune system that the patient receives. Unfortunately, we do not know how to precisely fine tune this new immune system to make BMT safer for more children. Dr. Peltier's work seeks to further understand how a component of this new immune system is controlled by a recently identified class of genes called non-coding RNAs (ncRNA). These ncRNA genes do not make proteins like classic genes, but instead regulate the production and function of proteins made by classical genes. His early data shows that unique ncRNA genes from multiple classes of ncRNAs are turned on and off following BMT. However, it is not known if or how these unique ncRNA genes influence the new immune system after BMT. Dr. Peltier seeks to further understand the function of these ncRNAs following BMT, which may suggest ways of developing medicines to improve BMT.

Yamilet Huerta M.D.
Funded: 07-01-2018 through 06-30-2020
Funding Type: St. Baldrick's Fellow
Institution Location: Cleveland, OH
Institution: University Hospitals of Cleveland affiliated with Rainbow Babies and Children's Hospital

Leukemia is the most common type of cancer in childhood, and 20% of childhood leukemia has a myeloid origin. Acute myeloid leukemia (AML) is treated with intensive chemotherapy as standard of care. Unfortunately, despite chemotherapy and stem cell transplantation, the prognosis of a child with recurrent or refractory AML remains poor. T cells are part of our immune system, and when properly manipulated, can be highly effective in eradicating chemo-resistant tumor cells. Engager (ENG) T cells are genetically engineered T cells that are capable of binding specific target on AML cells and at the same time "engaging" neighboring T cells to mount an immune response and kill cancer cells. As the JJ's Angels Hero Fund St. Baldrick's Fellow, Dr. Huerta is investigating the mechanisms by which AML cells can be killed by this novel immunotherapy technique. This grant is named for the JJ's Angels Hero Fund which honors the memory of Juliana LaMonica and her courageous battle with AML. Diagnosed at the age of two, Juliana underwent a bone marrow transplant but passed away shortly after turning three. Her sweet spirit and charismatic personality continue to inspire people to support the funding of pediatric cancer research through Team JJ’s Angels.

Samara Potter M.D., M.B.A.
Funded: 07-01-2018 through 06-30-2020
Funding Type: St. Baldrick's Fellow
Institution Location: Houston, TX
Institution: Baylor College of Medicine affiliated with Texas Children's Hospital, Vannie E. Cook Jr. Children's Cancer and Hematology Clinic

Despite recent advances in technology, very little is known about many types of rare and high risk childhood cancers. Since the numbers of these patients are so small, it has been very difficult to study how best to take care of them. Dr. Potter is using technology to look at the genetic code of these rare tumors, in order to learn more about why and how they occur, as well as how they change over time. This knowledge will help to create tests to diagnose these patients, as well as to develop more effective, less toxic treatments.

Corey Falcon M.D.
Funded: 07-01-2017 through 06-30-2018
Funding Type: St. Baldrick's Fellow
Institution Location: Birmingham, AL
Institution: University of Alabama at Birmingham affiliated with Children's of Alabama

ALL is the most common blood cancer occurring in children. Great strides have been made in the treatment of this disease, but new less toxic therapies for high risk ALL are needed. A new effective therapy is chimeric antigen receptor T-cells (CAR-T) which involves altering a patient’s own cancer fighting cells (T-cells) to express a protein able to recognize a protein on ALL cells (CD19), thus promoting killing of ALL cells. This form of therapy is much less toxic than traditional chemotherapy, but it is still associated with unwanted side effects. Dr. Falcon is working on ways to eliminate anti-CD19 CAR-T if severe side effects occur. This will greatly enhance the safety of this promising treatment. A portion of this grant is generously supported by the Not All Who Wander Are Lost Fund which was named after Kiersten Dickson’s favorite quote from J.R.R. Tolkien and honors the memory of a free spirited, courageous young woman who battled a rare, incurable cancer. This fund hopes to advance cutting edge immunotherapy treatments for pediatric cancers.

Beth Winger M.D., Ph.D.
Funded: 07-01-2017 through 06-30-2019
Funding Type: St. Baldrick's Fellow
Institution Location: San Francisco, CA
Institution: University of California, San Francisco affiliated with UCSF Benioff Children's Hospital

The goal of this project is to test a promising new drug called PLX9486 to treat pediatric cancers. In some cancers, a protein called “KIT” acts as an engine to drive growth. In comparison to existing treatments, PLX9486 is able to stop KIT in a unique way. Therefore, it is expected that this new drug will be very effective against cancers that are driven by KIT. However, over time cancer cells figure out ways to bypass drugs, leading to drug resistance. In addition to testing the effectiveness of PLX9486 against cancer cells, Dr. Winger is also studying how KIT might bypass the drug to develop resistance. Understanding the potential causes of drug resistance will allow her to develop strategies to overcome this resistance. This project will systematically evaluate a new drug that has the potential to transform the treatment of pediatric cancers driven by KIT.

Amanda Winters M.D., Ph.D.
Funded: 07-01-2017 through 06-30-2019
Funding Type: St. Baldrick's Fellow
Institution Location: Denver, CO
Institution: University of Colorado affiliated with Children's Hospital Colorado

Dr. Winters' research involves developing more effective and more targeted therapies for children with acute myeloid leukemia (AML), a type of leukemia that continues to have poor outcomes. The therapy for pediatric AML has not changed much in 20-30 years, and many children who receive this therapy relapse. There is a protein on many AML cells called CD123, which marks the earliest leukemia cells. In adults there are drugs that target this protein which are being studied in clinical trials. However, no one has studied whether CD123 is a useful target in pediatric AML. Dr. Winters is looking at CD123 protein expression in AML samples from pediatric patients, as well as investigating whether expression of CD123 marks the primitive leukemia cells in these patients - that is, those that give rise to the leukemia and cause relapse. She is also testing some of the same drugs that are being used in adult clinical trials on these pediatric samples in a laboratory setting, to see if they may be useful in pediatric patients. These studies are expected to generate new therapy options for children with difficult-to-treat AML.

Robert Rowe M.D., Ph.D.
Funded: 07-01-2017 through 06-30-2019
Funding Type: St. Baldrick's Fellow
Institution Location: Boston, MA
Institution: Boston Children's Hospital affiliated with Dana-Farber Cancer Institute, Harvard Medical School

Leukemia that develops in infants under one year of age is difficult to cure and has poor long-term outcomes compared to leukemia developing in older children or teenagers. The therapies used for infant leukemia are highly toxic with long-term adverse effects and are not particularly effective at curing the disease. To identify more tolerable and more effective treatments, we need better models to study infant leukemia in the laboratory. We could use such a model to identify the genes that drive infant leukemia and make it so aggressive and challenging to treat, and to develop drugs to target these genes. Dr. Rowe has developed a new model of infant leukemia and is using this system to identify the key genes that make this form of leukemia so aggressive.

Michael Koldobskiy M.D., Ph.D.
Funded: 07-01-2017 through 06-30-2019
Funding Type: St. Baldrick's Fellow
Institution Location: Baltimore, MD
Institution: Johns Hopkins University School of Medicine affiliated with Johns Hopkins Children's Center

Acute lymphoblastic leukemia (ALL) is the most common cancer in children. Despite dramatic improvements in treatment outcome in recent decades, relapsed and resistant disease remains a leading cause of childhood death from cancer. Dr. Koldobskiy studies the ways in which leukemia cells rely on "epigenetic" modifications, or chemical marks that modify the expression of genes without a change in the genetic sequence itself. Variability of epigenetic marks allows leukemia cells flexibility in turning genes on and off, and may account for resistance to treatment. By dissecting the mechanisms of epigenetic modification in childhood ALL, he aims to identify new targets for treatment.

Nitya Gulati FAAP, M.B,B.S
Funded: 07-01-2017 through 06-30-2019
Funding Type: St. Baldrick's Fellow
Institution Location: New York, NY
Institution: Memorial Sloan Kettering Cancer Center

A new class of drugs called EZH2 inhibitors is currently in clinical trials for the treatment of patients with relapsed B-cell lymphomas, a common subtype of pediatric lymphoma. These drugs suppress the activity of the EZH2 enzyme, which is known to be critical to tumor growth. Over time, however, if the lymphoma cells become resistant to EZH2 inhibitors, they may lose their effectiveness. Dr. Gulati aims to understand the mechanisms through which lymphomas develop resistance to EZH2 inhibitors. This will extend the usefulness of these drugs and will help in the development of methods to overcome the resistance.

Joanna Pierro D.O.
Funded: 07-01-2017 through 06-30-2019
Funding Type: St. Baldrick's Fellow
Institution Location: New York, NY
Institution: New York University School of Medicine affiliated with NYU Langone Medical Center

While outcomes for childhood leukemia have improved dramatically, the prognosis for children who relapse remains poor making relapsed leukemia one of the main causes of cancer death in children. Discovering the underlying pathways that lead to chemotherapy resistance and relapsed disease is therefore a top priority. To prevent relapse and improve treatment response, Dr. Pierro's laboratory has focused on discovering genetic mutations responsible for relapse and chemotherapy resistance. Mutations in a gene known as MMSET have been identified as one of the most common mutations in relapsed leukemia in children. This mutation in other cancers imparts a poor prognosis which suggests it has a role in drug resistance. Dr. Pierro's team has developed leukemia cell lines with and without the MMSET mutation and is treating the lines with chemotherapy to test this theory. He is also identifying the pathways controlled by this gene to identify the mechanism by which it protects the cells from the effects of chemotherapy. This information could be used to develop targeted therapy to prevent relapse and restore sensitivity to chemotherapy thereby improving outcomes.

LaQuita Jones D.O.
Funded: 07-01-2017 through 06-30-2019
Funding Type: St. Baldrick's Fellow
Institution Location: Cincinnati, OH
Institution: Cincinnati Children's Hospital Medical Center affiliated with University of Cincinnati College of Medicine

Patients with acute myeloid leukemia (AML) that is associated with a specific type of mutation in a protein called FLT3, have a poor prognosis. When these patients relapse, they have been found to have a unique mutation in this protein that makes their leukemia very difficult to treat. Dr. Jones is studying the effects of a novel FLT3 inhibitor in patients who have developed exquisitely resistant AML.

Jade Wulff M.D.
Funded: 07-01-2017 through 06-30-2019
Funding Type: St. Baldrick's Fellow
Institution Location: Houston, TX
Institution: Baylor College of Medicine affiliated with Texas Children's Hospital, Vannie E. Cook Jr. Children's Cancer and Hematology Clinic

Ewing sarcoma (ES) is the second most common bone cancer in children. Approximately 25% of children with ES have metastasis, which are tumors that have spread to other parts of the body, such as the lungs. It is especially difficult to treat these children and more than 70% die within 5 years. Therefore, it is important to learn about what it is that allows these tumors to spread and hopefully develop new drugs to treat these patients. Certain proteins are expressed at much higher levels in metastatic lung tumors compared to the primary bone tumor, suggesting that these proteins play a role in allowing the tumor to spread. As the Team Clarkie Fund St. Baldrick's Fellow, Dr. Wulff is studying the role of these proteins by increasing or decreasing them, and then testing how this affects the cancer’s ability to grow and spread. Dr. Wulff's team thinks that the cancer’s ability to spread can be decreased by decreasing a particular set of proteins. In addition, she is testing new drugs that inhibit the function of these proteins, with the hope to identify new therapies that will improve overall survival rates for patients with metastatic ES. Clarkie Carroll was diagnosed with Ewing sarcoma in his upper right femur in 2013. He endured surgery and treatments with strength, positivity and a sense of humor. Today he has no evidence of disease. A portion of this grant is funded by the Team Clarkie Fund, created to honor Clarkie and ensure researchers have the resources to further Ewing’s sarcoma research as well as stimulate greater awareness and inspire others to believe pediatric cancer research can and will lead to a cure.

Fiorella Iglesias M.D.
Funded: 07-01-2017 through 06-30-2019
Funding Type: St. Baldrick's Fellow
Institution Location: Salt Lake City, UT
Institution: University of Utah affiliated with Huntsman Cancer Institute

Neuroblastoma is the second most common pediatric solid tumor. Patients with high-risk disease have only a 50% chance of survival. The immune system can be engineered to efficiently kill cancer cells while sparing healthy tissues. However, neuroblastoma has been shown to evade these treatments by downregulating their target structures and upregulating inhibitory proteins. Dr. Iglesias is developing immune cells that specifically recognize neuroblastoma cells and also circumvent the aforementioned treatment evading mechanisms by restoring the target structures and blocking the inhibitory proteins. Through this work Dr. Iglesias aims to develop a new treatment approach for patients with high-risk neuroblastoma.

Miriam Kim D.O.
Funded: 07-01-2017 through 06-30-2019
Funding Type: St. Baldrick's Fellow
Institution Location: Madison, WI
Institution: University of Wisconsin-Madison affiliated with American Family Children's Hospital

Children who have leukemia (a type of blood cancer) that is difficult to treat with just chemotherapy can be treated and even cured with transplants of blood stem cells from a donor. However, even when donor and patient cell types are carefully matched, immune system incompatibilities between a patient’s body and cells from a donor can cause many complications including graft-versus-host disease, which can be fatal in extreme cases. Results from this research will hopefully teach us a way to manipulate the immune system using something called “exosomes” so that the child receiving the stem cell transplant is less susceptible to attack from the donor’s cells and can have a successful cure. Through this research Dr. Kim hopes to be able to use exosomes to protect the child’s body from the donor cells that can cause harm, yet preserve the donor cells that can fight the leukemia.

Melissa Mavers M.D., Ph.D.
Funded: 07-01-2016 through 06-30-2019
Funding Type: St. Baldrick's Fellow
Institution Location: Palo Alto, CA
Institution: Stanford University affiliated with Lucile Packard Children’s Hospital

Based on progress to date, Dr. Mavers was awarded a new grant in 2018 to fund an additional year of this Fellow award. Many children with cancer cannot be cured with chemotherapy alone and must undergo bone marrow transplantation. This treatment permits very high doses of chemotherapy to cure the cancer then rebuilds the immune system, which is destroyed by such high chemotherapy doses. In many cases, rebuilding a new immune system causes the new immune cells to attack the body, which is called graft-versus-host disease. Graft-versus-host disease can damage many organs. Dr. Mavers is studying ways to use special cell types to stop graft-versus-host disease and make stem cell transplantation a safer way to cure cancer. This is made with generous support from the Rays of Hope Hero Fund that honors the memory of Rayanna Marrero by giving hope through research funding. She is remembered for her infectious smile and energetic spirit that continue to inspire so many.

Benjamin Huang M.D.
Funded: 07-01-2016 through 06-30-2019
Funding Type: St. Baldrick's Fellow
Institution Location: San Francisco, CA
Institution: University of California, San Francisco affiliated with UCSF Benioff Children's Hospital

Based on progress to date, Dr. Huang was awarded a new grant in 2018 to fund an additional year of this Fellow award. Cure rates for Acute myeloid leukemia (AML) are poor and current therapies are toxic. Dr. Huang is using accurate models of AML to test novel agents that target cancer specific dependences. The overall goal of Dr. Huang's research is to develop new therapeutic strategies in AML to enhance efficacy and reduce toxicity. This research will inform efforts to develop novel treatment combinations in children with AML.