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Childhood cancer kills more children each year in the US than any other disease. Upstate Golisano Children's Hospital's mission is to conquer childhood cancer through superior care, research, and comprehensive support. Upstate Golisano Children's Hospital provides the medical expertise for children, adolescents, and young adults in the central New York area. They do this by participating in national treatment trials sponsored by the Children's Oncology Group (COG). This grant supports a Clinical Research Associate to ensure that more kids can be treated on clinical trials, often their best hope for a cure.

Malignant rhabdoid tumor (MRT) and atypical teratoid/rhabdoid tumor (AT/RT) are rare but devastating childhood cancers. Most children diagnosed with MRT and AT/RT are under the age of two, and most will die from their disease despite intensive treatment interventions. New insights into what causes these cancers, and new therapies, are desperately needed. Genetically, MRT and AT/RT are simple cancers, caused by loss of just one gene called SMARCB1. If we are to understand and treat MRT and AT/RT, therefore, we need to understand how loss of SMARCB1 causes cancer.

As the recipient of the Oh Danny Boy I Love You So: The Danny O'Brien Rhabdoid Tumor Research Fund St. Baldrick's Research Grant, Dr. Tansey is working on an innovative molecular mechanism through which loss of SMARCB1 causes MRT and AT/RT. He proposes that these mutations drive cancer by stimulating the activity of a known pro-tumorigenic gene called MYC. Dr. Tansey further proposes that MRT and AT/RT can be effectively treated by drugs that block the actions of MYC, currently being developed by us and others. He is testing this model and exploring its therapeutic implications. Completion of this work has the potential to transform the understanding of how MRT and AT/RT form and how they can be treated. Danny O’Brien was five months old when he was diagnosed with a rare malignant rhabdoid tumor on his liver. Despite treatment to shrink the tumor, he passed away at the tender age of 9 months. Fortunately, he knew nothing but love and affection for all of his short life. This fund honors Danny’s courage and remembers his unconditional love in the midst of his battle with cancer.

We can now manipulate the immune system with remarkable precision and efficacy to fight certain cancers. However, tumors adapt to reduce immunotherapy efficacy. This is true for solid tumors including osteosarcoma. Therapy-refractory metastatic osteosarcoma represents a pressing unmet need, as the outcome has not improved for over 3 decades. This multi-institutional collaborative proposal explores tumor-extrinsic pathways by which pulmonary metastatic osteosarcoma evade immunity. Dr. Huang’s team is focusing on key molecules responsible for such evasion, against which existing or emerging therapeutic agents are available currently or in the very near future. Therefore, uncovering these pathways could realistically offer multiple new clinical trials for pediatric and AYA metastatic osteosarcoma in the next 3 years. This Osteosarcoma Collaborative St. Baldrick's Grant to Cure Osteosarcoma is funded through the generosity of the Osteosarcoma Collaborative.

Despite the use of multimodal treatments and the implementation of several clinical trials worldwide, pediatric cancers survival rate has come to a standstill for the last decade. Moreover, intensive therapies are not devoid of long-term side effects, notably increasing lifetime risk for secondary malignancies. The duty of the pediatric oncologist is to propose the most adequate treatment to cure pediatric patients with the best quality of life for a long time. Therefore, understanding the biological underpinnings of pediatric malignancies is crucial to develop new therapeutic paths adapted to the specificities of a young organism. A major pitfall is the lack of adequate experimental models. To overcome this problem, Dr. Broutier is developing patient-derived 3D-organoid models (mini-tumor growing in a dish) of pediatric cancers. Beside their broad interest for research community, she will use them to identify mechanisms involved in cell death resistance in pediatric cancers, as a key step towards development of new targeted therapies adapted to children and adolescents.

Nearly half of all children diagnosed with Acute Myeloid Leukemia (AML) will suffer a relapse after initially successful treatment. Whereas therapy efficiently clears the bloodstream of leukemia cells, frequent evidence of residual drug resistant disease points to a leukemia protective role of the bone marrow microenvironment. The mechanism by which the bone marrow acquires these protective abilities is not clear. Dr. Kurre recently observed that so called stroma cells, that provide bone marrow structure and support are functionally altered. Pilot studies conducted by Dr. Kurre also identified a new mechanism by which AML changes these stroma cells. In this project Dr. Kurre is studying pediatric AML samples to understand how the altered stroma protects leukemia cells from the effect of drugs commonly used to treat children with AML. The long term goal is to develop treatment approaches that reduce the burden of relapse, by maintaining initial remissions in children with AML, and without further escalating drug toxicity. Awarded at Oregon Health and Science University, and transferred to The Children's Hospital of Philadelphia.

Adoptive immunotherapy has demonstrated unprecedented clinical success in the treatment of leukemia. In this therapy, T cells are isolated from a patient, expanded outside of the body, and genetically modified prior to reinfusion. The ability of these T cells to recognize and eliminate cancer cells is improved by expressing a protein (CAR) on the T cell surface. This protein increases "specificity," the ability to recognize cancer cells, and "function," the ability to destroy those cancer cells. An important challenge in cellular immunotherapy is to minimize the manipulation of patients' T cells outside the body. Prolonged culture protocols trigger functional exhaustion and compromises their efficacy upon return to the body. A critical issue involves the pre-requisite "activation" step necessary for CAR expression on the cell surface of T cells. By optimizing culture conditions, Dr. Ghassemi developed approaches to express CAR in dormant T cells without the need for activation. Importantly, her preliminary findings show that elimination of this activation step retains effector function and potency of CAR T cells in models of the pediatric cancer ALL. This project is providing insight into the regulatory components influencing CAR expression in dormant T cells which will lead to superior CAR T cells for cellular immunotherapies against ALL.

This grant is supported by TEAM ABBY Gives, a St. Baldrick's Hero Fund. Abby was diagnosed with Pre-B ALL when she was almost five years old. She had a successful bone marrow transplant, but battle battled graft vs. host disease (GVHD) for years. Abby and her treatment team worked hard over many years to keep the GVHD in check. Sadly, Abby passed away on October 19, 2021. This fund unites the incredible support of family and friends in Abby's memory and inspires others to join the fight for cures and better treatments.

Ewing Sarcoma (ES) is a type of cancer growing in or around bones in children and young adults. A protein called CD99 is present on all ES cells and inhibition of CD99 by different means kill ES cells. As of today none of these methods of CD99 inhibition is available as a clinical tool. Dr. Uren's team recently discovered that an FDA approved drug, clofarabine, can do the same and kill ES cell by directly binding and blocking CD99. Since clofarabine is already FDA approved, it can be tested on children with ES immediately in a Phase II clinical trial. Clofarabine is currently used in leukemia patients in the clinic due to its ability to inhibit different proteins in the cell. Since his findings suggest that there is a novel mechanism that was not known before, it is critical to establish how exactly inhibition of CD99 in ES cells lead to their death. That knowledge is the key to initiate a Phase II clinical trial with ES patients. This project will provide the missing information and accelerate design of new clinical trials based on CD99 inhibition.

There is a new and effective cancer treatment for some incurable pediatric blood cancers. The treatment involves programing a patient's own cells to destroy their tumor, a process called cellular immunotherapy. Despite great effort to use cellular immunotherapy to treat 'solid' tumors, which include tumors of the bones, muscles and other parts of the body, we have not been successful yet. One major reason is that the programmed patient cells are designed to recognize a single marker on a cancer cell. In some blood cancers, all of the tumor cells express the same marker increasing the likelihood that cellular immunotherapy can cure the patient. Solid tumors are more heterogeneous than blood cancers, meaning each solid tumor cell may express a different marker. Therefore, cellular immunotherapy is less likely to destroy all solid tumor cells and the chances of achieving a cure is much more difficult. A potential solution is to trigger the body's own immune system to destroy tumor cells that express many different markers, a process called "epitope spreading". Named as the David's Warriors St. Baldrick's Scholar, Dr. Leibowitz focuses his project on testing strategies to augment epitope spreading in pediatric solid tumors so that cellular immunotherapy may become an effective and viable treatment option in the future.

This grant is named for and generously supported by the David’s Warriors Hero Fund created in memory of David Heard who battled neuroblastoma and inspired his family and countless others to commit to raising money for research to fight pediatric cancer. This fund honors the amazing spirit in which he lived, embracing life until the very end.

Medulloblastoma is one of the most common malignant brain tumors in children. The Group 3 subgroup tumors have the poorest outcomes due to dissemination of tumor cells to distant sites in the central nervous system. As the recipient of the Benicio Martinez Fund for Pediatric Cancer Research St. Baldrick's Research Grant, Dr. Pei has identified a subpopulation of tumor cells that contribute to the metastasis after radiotherapy. He is determining whether targeting these cells can eliminate or prevent metastasis of Group 3 medulloblastoma, thereby improving the outcome of patients with this disease. Weeks after being the top fundraiser in his 6th grade class and shaving his head at his school’s event, Benny was diagnosed with medulloblastoma. Despite complications from treatment and setbacks, Benny has an amazing can-do attitude and is battling the cancer with determination.

This grant is funded by the Hero Fund that honors Benny’s fight and supports cures and better treatments for kids like him.

Some childhood cancers do not respond to chemotherapy, surgery, or radiation. For these patients, researchers are developing a new set of treatments that use their own immune system to attack the cancer. To turn on these defenses, they need to bolster the DNA in 200 million immune cells, efficiently and safely. Unlike other strategies, these cells do not need to come from the patients, who are already weakened. Dr. Weiss has invented an engineering solution to do so and is testing it so that he can make this treatment widely available to patients and their doctors soon.

Phthalates are chemicals added to many products that we use every day, including some common medications. Phthalates interfere with hormone systems in our bodies, which might cause cancer. Dr. Ahern wants to know if phthalate exposure while in the womb or during childhood increases the risk of childhood cancer. It would usually be time-consuming and expensive to answer this question scientifically. However, Dr. Ahern's team has developed a way to measure phthalate exposure using electronic pharmacy records that is both fast and inexpensive. This technique works because phthalate exposure from medications dwarfs exposure from other products. He will use this technique on existing pharmacy and cancer data from the entire population of Denmark. Dr. Ahern will measure phthalate exposure in pregnant women and in their children, and calculate whether that exposure increases a child's chances of developing cancer. If he finds that it does, we could prevent childhood cancer cases by limiting the amount of phthalates used in consumer products.

Children with high-grade gliomas, such as glioblastoma multiforme, have few therapeutic options and usually die of their disease. CAR T cells recognize protein targets on cancer cells and kill those cells. Many brain tumors express target proteins on only some of their cells and therefore cannot be efficiently treated with a CAR T cell that recognizes only one target. Therefore, Dr. Majzner aims to make T cells that can recognize up to four targets. He is exploring the best way to achieve specificity (the narrowness of the range of substances with which an antibody or other agent acts or is effective) for four antigens including using gene editing in order to make CAR T cells that can come from a healthy donor but be used in any patient.

A portion of this grant is funded by and named for the Be Brooks Brave Fund. Despite his diagnosis at age 5 with inoperable brain and spinal tumors, Brooks taught so many people what life is truly about--love. He was BRAVE beyond his years with an inspiring “faith over fear” attitude. This Hero Fund hopes to raise money for high-grade glioma research so no other family will hear the words, “there is no cure”.

A portion of this grant was also generously co-supported by the McKenna Claire Foundation, a St. Baldrick's partner and the Living for Luker Brain Tumor Research Fund, a St. Baldrick's Hero Fund. The McKenna Claire Foundation was 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. The Living for Luker Brain Tumor Research Fund was established in memory of Luke's love for life and caring for others. He was diagnosed at age 10 with Diffuse Intrinsic Pontine Glioma, a rare, uncurable cancer and never gave up hope throughout treatment.

Based on progress to date, Dr. Ch'ng was awarded a new grant in 2020 to fund an additional year of this Fellow award. 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.

Based on progress to date, Dr. Summers was awarded a new grant in 2020 to fund an additional year of this Fellow award. 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. Funded as the Emily Beazley's Kures for Kids Fund St. Baldrick's Fellow, this grant 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.

At the age of 8, Emily was diagnosed with Stage III T-cell lymphoblastic non-Hodgkin’s lymphoma and battled through three relapses. Her family prayed for a miracle but discovered Emily herself was the miracle, inspiring a community to come together to show love and change lives. She had a dream of starting a foundation to fund research and named it “Kures for Kids”. Today, Emily's family and friends carry on her dream and her mission in her memory.

In the U.S., children with a blood cancer called Hodgkin lymphoma (HL) are usually treated successfully. Some of these children will suffer health problems several years later because of the treatment they received. Because of this, doctors use powerful imaging tools to identify patients who are likely to do well or not. Those who are likely to do well require less treatment and those who are less likely to do well can receive more treatment. But in low-income countries like Malawi, these tools are unavailable, and the children there often receive treatment that may be unnecessary. Scientists have found unique abnormalities in adults with HL that can tell us who is less likely to do well. Here, Dr. Ozuah is testing whether these abnormalities are present in children and could be used to decide how best to treat children with HL in low-middle income countries

Although many children being treated for cancer initially respond to therapy, cancer cells often become resistant to chemotherapy drugs. Drug resistance is a major cause of cancer relapse, recurrence, and treatment failure. Dr. Gordon's goal is to identify new approaches to block, or reverse, resistance to an important class of cancer drugs. He has already identified one approach to reverse resistance in the laboratory, which he is now testing in models of cancer. Dr. Gordon is also testing a large number of additional drugs for the ability to prevent or reverse resistance.

Juvenile myelomonocytic leukemia (JMML), a fatal childhood blood malignancy, has limited therapeutic options. Relapse remains the main cause of treatment failure, most likely due to the persistence of leukemic stem cells (LSCs), a small population of self-renewing precursor cells that give rise to the bulk of tumor cells. Dr. Qu is exploring an innovative approach to eradicating LSCs in a subset of JMML that is caused by genetic mutations in Ptpn11. The information gathered from this study may yield a novel strategy for the treatment of this particular type of JMML.

Pediatric undifferentiated sarcomas are highly aggressive cancers that typically affect soft tissues of young children. Due to their uncertain classification and lack of molecular signature there are no standard criteria for diagnosis or treatment. With the Alan's Sarcoma Research Fund St. Baldrick's Research Grant, Dr. Antonescu is applying state of the art genomic methods to provide a detailed genetic characterization in these orphan cancers and investigating driving chromosomal translocations or mutations involved in their growth. These results will establish an objective classification of these tumors based on their genetic abnormalities and will provide potential therapeutic targets for further novel therapies. Furthermore these findings will inform the generation of faithful models for studying sarcoma formation and new drug development.

This grant is funded by and named for the Alan's Sarcoma Research Fund, a St. Baldrick's Hero Fund. Alan Sanders was diagnosed with a rare sarcoma in his hip at 17 months. He had an indomitable spirit and throughout his 4 ½ year battle with cancer, he was joyful, upbeat and pressed on courageously in spite of surgery and treatments. Today his family and friends carry on his legacy and his rallying cry, “Fight’s on!” in the battle against childhood cancer by funding sarcoma research.

Based on progress to date, Dr. Osborn was awarded a new grant in 2022 and 2023 to fund an additional year of this Scholar grant. Abnormal growth of B-cells can result in leukemia, and a cutting-edge treatment option is immunotherapy with T-cells. T-cells can be engineered to express a chimeric antigen receptor (CAR) that is a 'seek and destroy' molecule for the CD19 protein on B-cells. CAR T-cells are the first FDA approved gene therapy and some stunning therapeutic responses have been observed. However, the T-cell activity can be so robust that they cause a massive cytokine storm that can be lethal. Furthermore, normal and cancerous B-cells express the CD19 protein targeted by the CAR, so normal B-cell loss occurs resulting in an impaired immune system. These side effects represent a significant hurdle in the safe and effective treatment of B-cell leukemia. To address this, Dr. Osborn, will express the CAR in a specialized subset of cells called T-regulatory (Treg) cells. Tregs have the same potent killing ability as T-cells but accomplish it without healthy tissue collateral damage. Additionally, he will engineer functional B-cells that are invisible to the CAR. This will allow for normal B-cell numbers and an intact immune system. Dr. Osborn will conduct these studies that are structured to resolve an unmet need, are highly novel, and are poised to make an immediate impact on childhood leukemia. 

The 2022 portion of this grant is named for the Rays of Hope Hero Fund which honors the memory of Rayanna Marrero. She was a happy 3-year-old when she was diagnosed with Acute Lymphoblastic Leukemia (ALL). She successfully battled ALL, but a treatment induced secondary cancer claimed her life at age eight. Rayanna had an amazing attitude and loved life. She, like so many kids facing childhood cancer, did not allow it to define who she was. This Hero Fund aspires to give hope to kids fighting cancer through research.

A portion of this grant was funded by and named for the Mighty Mimi Hero Fund. Mimi Enyon was diagnosed with acute lymphoblastic leukemia at the age of 3. Her courage in the fight was unparalleled and she became “Mighty Mimi” to all those she inspired on her way to remission. This fund was established to share Mimi’s cancer journey in an effort to raise awareness and funding for childhood cancer research for kids like her.

A portion of this grant is generously supported by the Stanley Kuzmickas Feeney Fund for Pediatric Cancer Research. It was Christmas 2015 when Stanley was diagnosed with acute lymphoblastic leukemia at 13 months old. He courageously endured treatments for 3½ years. Today, he is in remission and eagerly started school in fall 2019. In his honor, Stanley’s family has organized a head-shaving event each year since July 2016 called “StoshyStrong." With the funds raised, the Feeney family created this Hero Fund to support research in new discoveries, genomics and other biological therapies for the treatment of ALL. Their goal is to one day see personalized treatments for every child.

Osteosarcoma is a cancer of bone that typically affects teenagers and young adults. Tumor spread (or metastasis) to the lungs is common, and up to 40% of patients with osteosarcoma will eventually experience a cancer recurrence (or relapse). Unfortunately, no therapies have shown benefit following relapse and these patients have a very poor prognosis. The ability of cancer to control and hide from the body’s immune system is important for tumor growth and metastasis, so preventing these functions is an important treatment strategy. Recent work, including a canine osteosarcoma trial, has shown that currently available medications may work together to block some of the effects that cancer has on the immune system, reducing tumor growth and the ability to spread. Dr. Faulk will conduct a clinical trial which will combine these drugs (losartan and sunitinib) in children and young adults with relapsed osteosarcoma to test the safety of the new combination, see how the drugs are broken down by the body, and determine how the drugs affect the immune system and the growth of the tumor.