Showing 1-20 of 119 results
Corey Falcon M.D.
Funded: 07-01-2017 through 06-30-2019
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 06-30-2018
Funding Type: St. Baldrick's Fellow
Institution Location: Palo Alto, CA
Institution: Stanford University affiliated with Lucile Packard Children’s Hospital

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 06-30-2018
Funding Type: St. Baldrick's Fellow
Institution Location: San Francisco, CA
Institution: University of California, San Francisco affiliated with UCSF Benioff Children's Hospital

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.

Gordon Cohen M.D., M.P.H.
Funded: 07-01-2016 through 06-30-2018
Funding Type: St. Baldrick's Fellow
Institution Location: Baltimore, MD
Institution: Johns Hopkins University School of Medicine affiliated with Johns Hopkins Children's Center

Pediatric Philadelphia Chromosome positive (Ph+) acute lymphoblastic leukemia (ALL) has a poor prognosis despite the introduction of Ph+ targeted drugs (TKIs). And for patients that fail to respond or relapse after TKIs, the prognosis remains grim. Dr. Cohen is studying why pediatric Ph+ ALL patients relapse (or fail to respond) to TKI treatment. Dr. Cohen has shown in early studies that combinations of TKIs and another new class of drugs can overcome TKI resistance. Because many of the drugs Dr. Cohen and his team are testing are commercially available, promising data from their research may move into future clinical trials.

Gina Ney M.D., Ph.D.
Funded: 07-01-2016 through 06-30-2018
Funding Type: St. Baldrick's Fellow
Institution Location: Ann Arbor, MI
Institution: University of Michigan affiliated with C.S. Mott Children’s Hospital

Many cancers include groups of cells that are relatively inactive, meaning that they divide less frequently than other cells and use fewer nutrients from their environment. This inactivity often makes these cells less susceptible to chemotherapy because these cells do not take in chemotherapy drugs due to their slow growth. As a result, these cells can remain present after treatment, potentially leading to disease recurrence. Dr. Ney is studying these inactive cells and their behavior, to more fully understand cancer and how to better treat it. This grant is made with generous support from Tough Like Ike, which was created in honor of cancer fighter Issac “"Ike" ”Yarmon. The organization raises awareness and funds for leukemia and childhood cancer research, as it impacts many lives. Through the support of Ike's friends, family and neighbors, the goal is to someday find a cure.

Michael Deel M.D.
Funded: 07-01-2016 through 06-30-2018
Funding Type: St. Baldrick's Fellow
Institution Location: Durham, NC
Institution: Duke University Medical Center affiliated with Duke Children's Hospital & Health Center

Rhabdomyosarcoma is a childhood cancer of the muscle that has two major subtypes. Children with the alveolar type do very poorly, as these are frequently recurrent and metastatic. Alveolar rhabdomyosarcoma cells have a specific fusion protein that is a powerful cancer driver and that does not yet have a pharmaceutical treatment. Dr. Deel and his team recently found that the Hippo pathway, normally functions to suppress tumor growth, is not correctly regulated in alveolar rhabdomyosarcoma, which leads to TAZ (a co-activator) activating pro-tumoric gene transcription. Dr. Deel is studying the interaction between the protein and the co-activator TAZ as a novel therapeutic target.

Melissa Mathias M.D.
Funded: 07-01-2016 through 06-30-2017
Funding Type: St. Baldrick's Fellow
Institution Location: New York, NY
Institution: Memorial Sloan Kettering Cancer Center

Children's cancer can spread through the body by hiding from the body’s immune system. There are certain cells, called regulatory T cells, that make it easier for cancer to hide by turning down the body’s immune system. Children with cancer who have a higher number of these cells seem to have a poorer outcome. Additionally, some cancers have these cells inside them. Dr. Mathias's lab has made a drug that can kill regulatory T cells, and will test this drug in a model to see the effects of killing the regulatory T cells, hopefully helping the body to improve the immune system's ability to kill cancer cells.

Arpan Sinha MBBS
Funded: 07-01-2016 through 08-31-2018
Funding Type: St. Baldrick's Fellow
Institution Location: Oklahoma City, OK
Institution: University of Oklahoma Health Sciences Center affiliated with The Children's Hospital at OU Medical Center

Myelodysplastic syndrome is a rare disease that affects the stem cells in the bone marrow, which causes decreased blood counts. It is also known to be a precursor to acute myeloid leukemia. Cellular mutations are present in majority of these cases, however the mechanisms of development of these mutations are still not clear. Dr. Sinha is studying these interactions, in order to identify new prognostic factors and therapeutic options for these patients. Awarded at Albert Einstein College of Medicine and transferred to University of Oklahoma Health and Science Center.

Samuel John M.D.
Funded: 07-01-2016 through 06-30-2018
Funding Type: St. Baldrick's Fellow
Institution Location: Dallas, TX
Institution: University of Texas Southwestern Medical Center at Dallas

Unfortunately, even with intensive treatment, only 60% of children with acute myeloid leukemia (AML) will survive the disease. Intensifying standard therapies have failed to improve survival rates, so a new approach is needed. Dr. John is creating a novel receptor that will specifically target a marker on AML cells. He will put this new receptor on T-cells of the body, which will then target and kill the leukemia cells. Through this research, Dr. John hopes to improve outcomes for pediatric AML.

Daniel Zinn M.D.
Funded: 07-01-2016 through 06-30-2018
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

Langerhans cell histiocytosis (LCH) is a rare cancer of the immune cells that can cause a wide range of symptoms, ranging from a rash to lethal multi-organ disease. Dr. Zinn hypothesizes that a patient’s symptoms are determined by a combination of the patient’s specific mutation and the specific immune cell that becomes mutated. Dr. Zinn is investigating the causes of LCH in order to develop the most effective and safe therapies for each patient.