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In a time of underfunded hospitals and scarce government grants, our board members and volunteers raise vital funds through a variety of events and campaigns that have allowed Pediatric Cancer Foundation to contribute over $20 million to the leading pediatric oncology institutions.
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Project: The Initiative for Drug Delivery Innovation is developing novel methods to improve drug delivery methods so that targeted therapies and immunotherapy become more effective.
Brain tumors remain the leading cause of death in children with cancer. Children with newly diagnosed Diffuse Intrinsic Pontine Glioma (DIPG) and glioblastoma, or relapsed malignant brain tumors (medulloblastoma, ependymoma, high-grade glioma), have an extremely poor prognosis. Despite advances in the treatment of adult cancers, researchers have failed to make significant progress in treating children with brain cancer. Therefore, as a team at Columbia University Medical Center, they have focused their efforts to discovering better methods of drug delivery that would ensure a more precise and targeted delivery while reducing systemic long-term effects for patients. Convection Enhanced Delivery (CED) is a method by which medicine can be infused directly into the brain tumor under controlled pressure so that maximum diffusion of the drug is achieved. And Focused Ultrasound with microbubbles (FUS) is a non-invasive method of “opening” the blood brain barrier with ultrasound micro-waves, allowing medicines to enter in sufficient concentrations.
Project: Pediatric Cancer Foundation Developmental Therapeutics Program (PCFDTP) at Columbia University Irving Medical Center (CUIMC)
Pediatric Cancer Foundation Developmental Therapeutics Program (PCFDTP) is to develop and test cutting-edge treatment strategies for children with incurable cancer, resistant to conventional therapies. The PCFDTP also serves as an incubator for precision cancer medicine to provide individually tailored therapies based on a patient’s molecular and genetic characteristics while also serving as a platform for laboratory investigations to identify the various abnormal molecular pathways underlying cancer growth and other molecular changes that may represent novel therapeutic targets for pediatric drug development. Columbia University Irving Medical Center is one of only 21 institutions in North America, and the only institution in the NY, NJ, CT tristate region, approved to offer early drug development trials from the National Cancer to children with relapsed/refractory cancer. Since the program’s inception, 352 patients have enrolled to PCFDTP associated studies. Approximately 75% of the patients receive treatment for solid tumors while the remaining 25% receive treatment for leukemia and lymphoma.
Project: Development of immunologic therapy for the treatment of pediatric solid organ malignancies, with a focus on Wilms tumor
Outcomes for childhood cancers have improved substantially over the past 4 decades due to dose intense multimodality and multi-agent interventions. This effect, however, remains largely restricted to localized tumors, leaving a vast treatment gap for advanced tumors as well as refractory and relapsed malignancies. Wilms tumor is the most common primary renal tumor and the third most common solid malignancy in children. Long-term survival now exceeds 90% yet despite favorable histology, tumor recurrence is observed in 15% of patients and survival rates are substantially lower. Approximately 50% of patients with anaplastic histology experience recurrence and/or advancement of disease, and with advanced disease, only 50% survive to adulthood. Additionally, toxicities derived from current multi-agent chemotherapy and radiation therapy regimens often result in chronic morbidity that has become more prevalent with improving survival. Treatment for Wilms tumor can result in long-term morbidity, including renal failure, infertility, cardiovascular disease, and secondary cancers. This research involves pediatric solid organ malignancies, currently focused on Wilms tumor, with evaluation of gene expression and immune responses in order to investigate mechanisms of disease, immunoregulation, and the effects of chemotherapy to identify novel treatment strategies for patients with advanced, recurrent, and/or metastatic disease.
Project: Development of bone and blood cancers in mouse models of Diamond Blackfan anemia
Diamond Blackfan anemia is a rare inherited bone marrow failure characterized by the inability to make red cells, birth defects and a predisposition to cancer. These cancers include osteogenic sarcoma (bone cancer) and MDS/AML (pre-leukemia/acute myeloid leukemia). With funding from Pediatric Cancer Foundation, they have developed 2 mouse models that replicate the human disease, restricting the known gene mutation to bone and most recently and never before done successfully in DBA, to blood forming cells; we are characterizing both models to understand the cause of bone cancer and leukemia.
Project: Pediatric Hematology/Oncology Fellowship Research Support
Pediatric Low-Grade Gliomas are the most common group of central nervous system tumors in children. Treatment traditionally includes surgery, old fashioned chemotherapy, and/or radiation therapy. Both the tumors and traditional therapy have adverse effects on children. These tumors harbor certain mutations that can be targeted with new small molecule drugs. These small molecule therapies are effective but need to be given daily and for long periods of time. The drugs can have adverse side effects, have unknown toxicities when given chronically to growing children, and the tumors can eventually become resistant. Using animal tumor models, their goal is to help determine optimal short-term treatment schedules/doses of these small molecule drugs, either when given alone or in combination with old fashioned chemotherapy. Their studies have the potential to guide clinical trials by identifying the optimal treatments, schedules, and duration.
Project: Using human stem cells to model neuroblastoma development and identify new drug targets
Through investigating the factors that drive normal cells to develop into neuroblastoma, they hope to identify new targets for treatment of this disease. They have derived a unique living model of this process in stem cells and by studying the mechanisms that initiate disease, they hope to gain insight into how to treat neuroblastoma in the future. This work may have broad relevance to the treatment of other pediatric cancers with similar mechanisms of development.
Project: Reducing the Burden of Oncologic Chemotherapy And Radiation Exposure Utilizing Targeted Immunotherapy in Children, Adolescents and Young Adults with Lymphoma
As Chief of Pediatric Hematology, Oncology and Stem Cell Transplantation at Maria Fareri Children’s Hospital, Dr. Mitchell Cairo leads a multidisciplinary team of researchers developing treatments to battle Hematologic Malignancies in children and adolescents through targeted immunotherapies, reduced intensity conditioning and allogeneic stem cell transplantation, novel chemotherapy for reinduction therapy, Human Derived Placental Stem Cell (HDPSC) therapy, haplo identical stem cell transplantation, and single or double umbilical cord blood transplantation in children and adolescents who are at a high risk of Hematological Malignancies. The promise of unlocking treatments by harnessing the power of the patient’s immune system renews the efforts of Dr. Cairo’s team to lead advances that save lives while minimizing unwanted side effects both now and in the future. Overall, the program is equipped to design and deliver customized and personalized therapy for each child and adolescent in the cancer center diagnosed with a hematological malignancy.
Project: Improving efficacy and reducing toxicity of childhood neuroblastoma therapy
Neuroblastoma is a common and frequently deadly cancer of childhood. Even with the most intensive treatment, which is very toxic, about half of children with this cancer fail to respond. We are developing a novel strategy combining ultrasound to push special microbubbles containing novel formulations of FDA-approved drugs into the cancers, thus increasing their effects while reducing toxicities.
Project: Phase II Study Combining Cryoablation Therapy and Dual Checkpoint Inhibition in Relapsed/Refractory Pediatric Solid Tumors
While immune checkpoint inhibitors such as nivolumab and ipilimumab are safe in children, they are not effective treatment alone in pediatric solid tumors, likely because pediatric tumors are “cold” with low levels of T-cell infiltration and antigen presentation. Cryoablation therapy, which is a minimally invasive treatment that freezes tumor cells has been found to stimulate T-cells and other white blood cells to enter a tumor and stimulate a systemic anti-tumor immune response. We hypothesize that cryoablation therapy will enhance the efficacy of checkpoint inhibition against pediatric solid tumors. Therefore, we propose a phase II clinical trial for patients with relapsed or refractory pediatric solid tumors and more than one site of disease that will combine cryoablation therapy with nivolumab and ipilimumab with goal of disease response.
Project: Infrastructure Support for Pediatric Graft-versus-Host Disease Research
Bone marrow transplants can cure leukemia, but there is a risk of graft-vs-host disease (GVHD), a complication where immune system cells from the donor attack the healthy tissue of the patient. GVHD does not always respond to treatment with immune suppressing medications and, after relapse, is the leading cause of death after a bone marrow transplant. The Mount Sinai Acute GVHD International Consortium (MAGIC) collects clinical data and research samples for a multicenter natural history study of graft-vs-host disease (GVHD). The goal of this study is to increase our understanding of GVHD in children and use the knowledge learned to design innovative clinical trials to improve GVHD outcomes in this vulnerable population. Research funding supports the MAGIC Data Coordinating Center (DCC) which oversees the enrollment and monitoring of children across 12 international study sites, improving outcomes for children worldwide that have undergone bone marrow transplant.
Project: RNA Therapeutics for the Treatment of Pediatric Sarcomas
Pediatric Low-Grade Gliomas are the most common group of central nervous system tumors in children. Treatment traditionally includes surgery, old fashioned chemotherapy, and/or radiation therapy. Both the tumors and traditional therapy have adverse effects on children. These tumors harbor certain mutations that can be targeted with new small molecule drugs. These small molecule therapies are effective but need to be given daily and for long periods of time. The drugs can have adverse side effects, have unknown toxicities when given chronically to growing children, and the tumors can eventually become resistant. Using animal tumor models, their goal is to help determine optimal short-term treatment schedules/doses of these small molecule drugs, either when given alone or in combination with old fashioned chemotherapy. Their studies have the potential to guide clinical trials by identifying the optimal treatments, schedules, and duration.
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