Our Impact

The Power of Research

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 has allowed Pediatric Cancer Foundation to contribute over $20 million to the leading pediatric oncology institutions. Pediatric Cancer Foundation proudly bridges funding gaps by providing seed grants for innovative research that holds great promise but does not yet qualify for federal grants or larger funding opportunities.

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Our Doctors and Researchers

Through the past fifty years, Pediatric Cancer Foundation has had an enormous impact on the advancements of both the technology and the medicine used to treat children with cancer. By funding ground-breaking research and early stage clinical trials, survival rates have increased from 58% up to just over 80%.

New York-Presbyterian Morgan Stanley  Children’s Hospital of Columbia University Medical Center

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, we have failed to make significant progress in treating children with brain cancer. Therefore, as a team we have focused our 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. In 2020, at the Pediatric Neuro-Oncology Program at Columbia University Irving Medical Center (CUIMC), we are launching the Novel Drug-Delivery Methods for Pediatric Brain Tumors Initiative to develop new methods that focus on drug delivery methods so that targeted therapies and immunotherapy become more effective. Convection Enhanced Delivery (CED) using an implanted drug-filled pump 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. This method has particular value for tumors that cannot be surgically removed. Also, we are investigating another non-invasive method, which “opens” the blood brain barrier using dynamic Focused Ultrasound (FUS) with microbubbles and separates the barrier cells through ultrasound micro-waves, allowing medicines to enter in sufficient concentrations. Selection of drugs is based on unique biological and bioinformatics redditive models. These models combine single cell tumor genomic sequencing, network RNA, and proteomic analysis to examine the changes that occur in all these systems after extensive drug testing in the biopsied/resected tumor tissue. We also use a novel technique called Organotypic Slice Cultures (OSC) that maintains slices of fresh tumor right after resection, to test unique combinations of drugs, and then conduct an in-depth analysis of them using single cell sequencing. This allows us to decide upon the best therapy for the patient within a short period of time.

Stergios Zacharoulis, MD
Herbert Irving Associate Professor of Pediatrics Chief of Pediatric Neuro-Oncology Columbia University Medical Center

Clinical Research Pediatric Neuro-Oncology program 

“With the very kind support of the Pediatric Cancer Foundation, we have been able to move very quickly establishing novel institutional clinical trials focusing on the way we deliver drugs directly into the brain tumors. The first worldwide trial using Convection Enhanced Delivery via subcutaneous pump in children with Diffuse pontine glioma has opened with impressive preliminary results.
We are also about to open the first ever in children Non-invasive Focused Ultrasound study that will allow open the blood brain barrier and drugs to reach the target in children with diffuse pontine glioma.
A huge thank you to Pediatric Cancer Foundation for the incredible support for all these efforts… “

Pediatric Cancer Foundation Developmental Therapeutics Program (PCFDTP)

The Pediatric Cancer Foundation Developmental Therapeutics Program (PCFDTP) is a keystone of the innovative care and clinical research within the Division of Pediatric Hematology, Oncology and Stem Cell Transplantation at Columbia University Irving Medical Center (CUIMC). Our longstanding partnership with the Pediatric Cancer Foundation has given us the tremendous opportunity to establish a robust translational research program that includes clinical comprehensive tumor sequencing, basic science research to define novel therapeutic targets, and clinical trials to bring innovative treatments directly to patients. Our program is one of only 21 COG Phase I (first-in-child) programs designated by the National Cancer Institute (NCI) to offer early drug development trials to children with incurable cancer, and is the only such program in the New York, Northern New Jersey and Connecticut tri-state region, servicing a population in excess of 20 million. We are deeply indebted to the Pediatric Cancer Foundation for over 10 years of generous support that has allowed the program’s continued growth.

Darrell J. Yamashiro, MD, PhD, Division Chief Columbia Pediatric Oncology Division Chief, Division of Hematology, Oncology, and Stem Transplantation

Pediatric Cancer Foundation Developmental Therapeutics Program (PCFDTP) 

“In these extraordinary times, the support of Pediatric Cancer Foundation has allowed us to continue to offer novel therapies to children with relapsed cancer, to begin ground-breaking trials for inoperable brain tumors, and to examine the immune microenvironment of pediatric solid tumors.”

Alice Lee, MD Columbia Pediatric Hematology-Oncology, Pediatrics

Pediatric Cancer Foundation Developmental Therapeutics Program (PCFDTP) 

Pediatric Tumor Biology Lab, Division of Pediatric Surgery and Oncology, Columbia University Medical Center

This study aims to evaluate and investigate immunotherapy as a potential novel treatment strategy for pediatric solid organ malignancies. Pediatric patients with solid organ masses concerning for malignancy will be considered for enrollment. Findings will help guide the potential future development of immunologic therapy for the treatment of pediatric solid organ malignancies, with a focus on Wilms tumor. Wilms tumor is the most common primary renal tumor and the third most common solid malignancy in children. Treatment for Wilms tumor can result in long-term morbidity, including renal failure, infertility, cardiovascular disease, and secondary cancers. New treatment modalities are needed to improve outcomes of the advanced, relapsed, and/or refractory tumors as well as lessen the burden of morbidity for children who survive treatment. Immunotherapy is a growing field of interest for targeting cancers, potentially providing a novel treatment strategy that eliminates the current toxic effects and further improves survival for patients with complicated disease.

Erica M. Fallon, MD
Assistant Professor of Surgery Morgan Stanley Children’s Hospital of NY-Presbyterian Columbia University Medical Center

Development of immunologic therapy for the treatment of pediatric solid organ malignancies, with a focus on Wilms tumor

“I’m one of the Pediatric Surgeons and the Director of Surgical Oncology at New-York Presbyterian Morgan Stanley Children’s Hospital. The Pediatric Cancer Foundation has been a resolute partner to and supporter of our Division for many years. We have depended on your organization’s commitment to eradicating pediatric cancers, raising awareness, and providing funding for the necessary work that aspires to achieve these goals. We greatly appreciate and sincerely thank you for your dedication and unwavering support. My research has focused on Wilms tumor, with the 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. We are extremely excited to be involved in this work, and look forward to continued collaboration and productivity.”

Northwell Health – Feinstein Institute for Medical Research

Drs. Lipton and Blanc’s work focuses on understanding how bone cancer develops in patients with a rare genetic disease. Diamond Blackfan anemia (DBA) is a rare inherited bone marrow failure syndrome characterized by red cell failure, congenital anomalies, poor linear growth and cancer predisposition. The overall goals of this research are (1) to understand the defects in bone development (poor linear growth, osteopenia, skeletal anomalies) as a consequence of RP haploinsufficiency (2) to acquire a fuller understanding of the etiology of OS in the context of DBA, and thus (3) to determine the role of ribosomal protein gene mutations in oncogenesis, using DBA as a model. Recently, their work has been accepted for an oral presentation at the International Society for Pediatric Oncology (SIOP) in Lyon, France in October, 2019. This project has taken almost 5 years to get to this stage. They now have a mouse model that is predisposed to develop osteogenic sarcoma. The next stage of the project is to map out the molecular changes that lead to tumorigenesis in the hope that they can identify molecular targets for therapy. As many know, therapy for osteogenic sarcoma has been stalled with no significant improvements in survival for decades. They are hopeful that this mouse model can be exploited by their group as well as other investigators to improve therapy for this cancer. There is no doubt that without the support of Pediatric Cancer Foundation, they would not have been able to accomplish this work. Our contribution will be acknowledged in Lyon.

Pediatric Cancer Foundation has been providing support to The Feinstein Institute for Medical Research and Cohen Children’s Medical Center for over 15 consecutive years. The team has garnered major grants from the NIH, DOD and the CDC, all based upon work initially supported by Pediatric Cancer Foundation.

Research funded by Pediatric Cancer Foundation at The Feinstein Institute has been directed towards studying and understanding Diamond Blackfan anemia.

Jeffrey Lipton, MD. Ph.D.
Chief, Hematology/Oncology and Stem Cell Transplantation Steven and Alexandra Cohen Children’s Medical Center of NY

“Pediatric Cancer Foundation has supported my science for over 20 years. In that time my Division has grown and we have spun off independent investigators. Pediatric Cancer Foundation has allowed us to start projects that have resulted in numerous NIH and DOD funded grants. Pediatric Cancer Foundation has provided the seed money that funds the acquisition of the necessary preliminary data without which we could not compete for federal funding. Of note Pediatric Cancer Foundation was not been deterred by the COVID19 pandemic. I am honored to have my research supported by Pediatric Cancer Foundation.”

Lionel Blanc, Ph.D.
Les Nelkin Memorial Endowed Chair of Pediatric Oncology Professor of Molecular Medicine and Pediatrics Zucker School of Medicine at Hofstra Northwell Head, Laboratory of Developmental Erythropoiesis Institute of Molecular Medicine The Feinstein Institutes for Medical Research

“Thank you so much for allowing us to continue our research in such difficult times! Pediatric cancer research cannot be put in quarantine! With your support, my lab is functioning at pre-COVID19 capacity.”

Maria Fareri Children’s Hospital Westchester Medical Center

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.

Mitchell Cairo, MD
Chief of Pediatric Hematology, Oncology & Stem Cell Transplantation at Maria Fareri Children’s Hospital

Hematological Malignancies Program

“We want to thank Pediatric Cancer Foundation for their generous grant support to our program at the Maria Fareri Children’s Hospital at Westchester Medical Center. This grant support from Pediatric Cancer Foundation in large part was critical to our success in our B-Cell Lymphoma and Hodgkin Lymphoma program in 2020 resulting in patients achieving 100 percent complete responses and survival with targeted antibody therapy treatments.”

Memorial Sloan Kettering Cancer Center, Department of Pediatrics

At MSKCC, an initiative was developed to prospectively enroll pediatric patients that enables genetic profiling of their tumors in addition to developing specific mouse tumor “avatars” for patients that may be useful in screening potential therapies for those patients. However, the development of PDX tumor mouse models requires technical knowledge in mouse tumor mode ling, as well as resources and infrastructure to support the generation of these unique experimental models. Preliminary work has begun, having developed a robust pediatric PDX program at MSK Kids and have a PDX model portfolio with over 130 unique PDX models encompassing 65 unique pediatric cancer types, such as Sarcoma, Neuroblastoma and renal tumors among the top cancer types. This collection represents one of the largest collections of pediatric PDX models to date. The primary objective is to fully establish PROXC and develop harmonized protocols for the conduct of multi-center mouse therapeutic trials of candidate drugs. To achieve this objective, a PROXC Research Program Manager will be funded by Pediatric Cancer Foundation.

Andrew Kung, MD
Chairman Dept. of Pediatrics at Memorial Sloan-Kettering

Development of pediatric cancer models to inform preclinical and clinical studies – PROXC (Pediatric Research in Oncology Xenografting Consortium)

“On behalf of MSK Kids, I am honored to have the opportunity to thank the Pediatric Cancer Foundation for its longstanding commitment to our institution. Since 1997, Pediatric Cancer Foundatino has provided vital funds that have allowed our investigators to pursue exciting new research initiatives with agility.

This year has been a challenging year for laboratory research because COVID-19 necessitated that we close our labs for 2 months. Thankfully, we have now resumed all research activity and now we are continuing to push forward with our search for new treatments for pediatric cancers. As the largest pediatric oncology program in the nation, we are delighted to have a dedicated partner in Pediatric Cancer Foundation as we strive to deliver the best available care for children with cancer today, while simultaneously doing the research that will improve outcomes for pediatric oncology patients in the future. In the face of a challenge affecting us all, thank you for your commitment to the cause of pediatric cancer.”

Filemon Dela Cruz, MD
Assistant Attending in Pediatrics Pediatric Sarcoma Service Department of Pediatrics, Memorial Sloan Kettering

Development of pediatric cancer models to inform preclinical and clinical studies – PROXC (Pediatric Research in Oncology Xenografting Consortium)

“Support from the Pediatric Cancer Foundation has enabled my group to translate our findings in the laboratory into novel approaches for treating children with high-risk cancers. “

Stephen D. Hassenfeld Children’s Center for Cancer and  Blood Disorders at NYU Langone Medical Center 

Training the next generation of bright, highly-motivated clinician scientists who are dedicated to the practice of patient-centered, compassionate, and research-driven care for children with malignancies and blood disorders. Led by Fellowship Director, Elizabeth Roman, MD, the NYU Langone Pediatric Hematology/ Oncology Fellowship Training Program is a three-year program, accredited by the Accreditation Council for Graduate Medical Education (ACGME), which provides the graduate with comprehensive training in hematology and oncology. Remarkable strides have been made in the treatment of newly diagnosed pediatric acute lymphoblastic leukemia (ALL) and acute myeloid leukemia (AML), yet a subset of children relapse and succumb to therapy-resistant disease. Relapsed leukemia remains a leading cause of cancer-related death in children. Identifying mechanisms of therapy resistance in relapsed leukemia is paramount to developing effective treatments that can overcome these barriers. to investigate the role of CD97 in pediatric acute lymphoblastic leukemia (ALL) in the Park laboratory. Immunotherapy for leukemia and other malignancies has drawn interest because of its non-overlapping toxicity with chemotherapy and radiation. The Fellow’s work will analyze the function of CD97 on a variety of cell processes and will also therapeutically target CD97 with monoclonal antibodies and assess treatment response. The goal is to translate laboratory findings in the hopes of developing a CD97 antibody that may be used in pediatric patients with relapsed or refractory leukemia in the future as an immunotherapy adjunct to standard chemotherapy. In addition, Pediatric Cancer Foundation has funded for two Vein Illuminators. Children receiving treatment for cancer and blood disorders have to undergo blood draws frequently to monitor their condition. Unfortunately, at times, it is hard to detect a vein due to the patient’s age, medical status and the treatment they are receiving. Vein Illuminators will allow our team to see a map of the veins underneath the skin’s surface with the goal of improving patient care, comfort and safety by increasing the accuracy and success rates of IV placement and blood draws.

William Carroll, MD
Division Chief, Pediatric Hematology/Oncology Medical Director, Hassenfeld Children’s Center Director, NYU Cancer Institute The Julie and Edward J. Minskoff Professor of Pediatrics

Pediatric Hematology/Oncology Fellowship Training Program 

“Support from the Pediatric Cancer Foundation has enabled my group to translate our findings in the laboratory into novel approaches for treating children with high-risk cancers. “

Elizabeth A. Raetz, MD

Division Chief, Pediatric Hematology/Oncology Medical Director, Hassenfeld Children’s Center Director, NYU Cancer Institute The Julie and Edward J. Minskoff Professor of Pediatrics

Pediatric Hematology/Oncology Fellowship Training Program 

“Generous support from Pediatric Cancer Foundation over many years has led to important discoveries in childhood leukemia. Our most significant accomplishments in 2020 were discoveries from the Carroll laboratory, where two postgraduate fellows are currently training, demonstrating the roles of both the epigenome and immune microenvironment in imparting resistance to conventional leukemia therapy. These discoveries will pave the way for new therapeutic interventions and current and past postgraduate fellows supported by Pediatric Cancer Foundation have actively participated in this work and are co-authors on recent 2020 publications.”

A quote from a former fellow:

As a fellow in Pediatric Hematology/Oncology with funding from Pediatric Cancer Foundation, the research I was able to perform really marked the beginning of my ability to make meaningful contributions to our field’s understanding of pediatric leukemia. This research helped further our understanding of the cellular and molecular mechanisms underlying disease relapse, and will hopefully lead to improved therapies for children. And for me personally, this funded fellowship was the foundational step in my career path – my “on ramp” to my current role in which I am able to continue investigating new therapies for children with cancer.

Another quote from a former fellow:

Pediatric Cancer Research receives only 4% of the national cancer budget from the NIH making organizations like Pediatric Cancer Foundation critical to our ability to advance the field of pediatric oncology. Additionally, pediatric oncology fellowships are only partially funded by the ACGME and institutions are often unable to support trainee salaries. Pediatric Cancer Foundation has been integral to filling both gaps. My Pediatric Cancer Foundation funding during fellowship allowed me to complete my training in pediatric oncology and jumpstart my career at NYU as a pediatric oncologist. It allowed me protected time in the lab to focus on my research and ultimately publish my work to help advance the field of pediatric cancer. I am forever grateful to Pediatric Cancer Foundation and their generous donors for allowing NYU to continue to train oncologists to carry on the legacy that came before us and deliver stellar care to children with cancer.

University of Chicago Medicine Comer Children’s Hospital

Neuroblastomas are highly aggressive pediatric cancers, currently causing 15% of cancer-related childhood mortality. They are the most common solid tumor of the abdomen in children. Despite the best available treatments, 50-60% of children with neuroblastoma develop recurrent disease, highlighting the critical need for better therapy. However, our ability to treat children with chemotherapy is limited by its severe toxicity; even the fortunate survivors who respond to intensive therapy have a high risk of long-term toxic effects. Thus, we seek to improve the current methods of delivering chemotherapy, with the goals of simultaneously enhancing treatment and reducing toxicity. In our studies, we are investigating what is termed “targeted chemotherapy” – novel systems which “home” treatment drugs selectively to the tumor, reducing exposure of normal tissues to toxic chemicals. These new strategies leverage cutting-edge biochemical and bioengineering techniques. The approach that we have employed involves design and synthesis of special microbubbles, which act as packaging for chemotherapy, and which can be detected and guided by ultrasound waves to penetrate tumor tissue. To explore this strategy, we have used doxorubicin, an effective but toxic molecule used to treat neuroblastoma, in a model of the disease. We also plan to test whether this same method can be applied to increase tumor uptake of other drugs, since children with neuroblastoma are typically treated with a multi-drug cocktail.in this proposal we will examine two methods of improving selective delivery of chemotherapeutics. we will test the effects of repurposing an existing drug, bevacizumab, to increase the ability of drugs to exit from blood vessels inside tumors. We will do this by increasing the permeability of tumor blood vessels.

Jessica J. Kandel, MD Mary Campau Ryerson Professor of Surgery, and Chief, Section of Pediatric Surgery  Surgeon-in-Chief, Comer Children’s Hospital  The University of Chicago Medicine & Biological Sciences

Improving efficacy and reducing toxicity of childhood neuroblastoma therapy  

“For more than 20 years, our lab’s work for children with cancer has been sustained by the Pediatric Cancer Foundation. Pediatric Cancer Foundation never let us forget that we are answering the call of mothers and fathers, grandparents, children, and families of children with cancer – and that together we have advanced treatment and saved lives. Together, we have not only forged new treatments, but trained a whole generation of young scientists with the same shared commitment.”

Mount Sinai Health System

Bone marrow transplantation or BMT (allogeneic hematopoietic cell transplantation) can cure many hematologic cancers and other childhood blood diseases. Every year, over 1,400 children undergo this lifesaving procedure in the United States and over 5,000 children receive a bone marrow transplant worldwide. Even more children would be able to receive a potentially life-saving bone marrow transplant were it not for the risk of graft-versus-host disease (GVHD), a deadly complication of BMT that develops in 40% of pediatric recipients. GVHD occurs when immune system cells from the bone marrow donor transplanted into the recipient mistake the normal healthy organs as invading foreign cells. When that occurs, the donor immune cells try to destroy the “foreign” tissue as they are programmed to do. Intensive immunosuppression can treat GVHD approximately half of the time. In the remaining cases, the patient either dies or develops a chronic form of the disease which can require years of treatment. In Mount Sinai Acute GVHD International Consortium (MAGIC), we collect clinical symptom and treatment data weekly to allow us to reconstruct a patient’s clinical course from diagnosis through treatment. MAGIC data coordinators are trained to recognize and question implausible patterns such as significant worsening in GVHD severity without escalation of treatment. As a result of this intensive effort, errors that affect other registries’ data are caught and corrected in the MAGIC database.

John E. Levine, MD, M.S. Professor of Internal Medicine and Pediatrics Director of BMT Clinical Research The Tisch Cancer Institute Mount Sinai Health System

Supporting Pediatric Graft-versus-host Disease Research

“Pediatric Cancer Foundation financial support allowed us to collect and analyze data from a large number of children who were at risk for severe graft-vs-host disease or GVHD, the major complication of bone marrow transplant. These analyses were a major accomplishment and showed that a blood test that we developed that predicts GVHD outcomes in adults also works for children.”

James L.M. Ferrara, MD, DSc. Ward-Coleman Chair in Cancer Medicine Professor and Director, Hematologic Malignancies Translational Research Center The Tisch Cancer Institute Icahn School of Medicine at Mount Sinai, New York

“Pediatric Cancer Foundation support allows us to understand the natural history of GVHD and to design the next generation of clinical trials to make BMT safer and more effective for pediatric patients.”

Our Impact

The Power of Research

Our Doctors and Researchers

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