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Our Impact

    Home Our Impact

    We’re committed to showcasing the exceptional work your donation enables — from sponsoring talented physicians and researchers, to supporting pediatric inpatient units and purchasing state-of-the-art equipment. Below is an overview of some of the projects and progress our donors make possible.


    New York-Presbyterian Morgan Stanley
    Children’s Hospital of Columbia University Medical Center
    Pediatric Tumor Biology Lab, Division of Pediatric Surgery and Oncology, Columbia University Medical Center

    PCF Developmental Therapeutics Program (PCFDTP)
    RESEARCH LED BY DR. ALICE LEE & DR. STERGIOS ZACHAROULIS

    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 PCF 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 PCF for over 10 years of generous support that has allowed the program’s continued growth.

    READ MORE ABOUT PRECISION MEDICINE

    Precision in Pediatric Sequencing (PIPseq) Program Pediatric Oncology is continuing to lead the way in the development of precision cancer medicine at CUIMC. This first in class New York based precision medicine program is the linchpin for moving from a “one size fits all” approach to pediatric cancer management to more individually tailored care. The PCFDTP continues to be responsible for the implementation of the PIPseq program focusing on real-time data acquisition to benefit both the clinicians (identification of actionable variants) as well as laboratory investigators (discovery). Thus far, over 550 patient samples have undergone whole exome sequencing and/or gene expression profiling, or targeted panel sequencing at our institution through the PIPseq Program.
    Expansion of this enterprise is underway including: sequencing patients with newly diagnosed disease, warehousing data for future personalized treatment recommendations (in the event of relapse) and further discovery into the genetic causes of pediatric cancer. Dr. Jennifer Oberg is leading the collaborative effort within CUIMC to implement next generation pediatric precision cancer medicine into clinical practice. Specifically, whole exome and transcriptome sequencing is being used to improve the care of individual children with cancer at Columbia and beyond. With the help of the Sohn Conference Foundation, this initiative has been extended to children throughout the NY, NJ, CT tristate region. Thus far, 205 patients (55 in 2019) from 17 institutions (excluding CUIMC patients) have been sequenced and 14% of patients referred for testing were enrolled on an early phase trial at an outside institution.


    New York-Presbyterian Morgan Stanley
    Children’s Hospital of Columbia University Medical Center
    Pediatric Tumor Biology Lab, Division of Pediatric Surgery and Oncology, Columbia University Medical Center

    Clinical Research Pediatric Neuro-Oncology program
    RESEARCH LED BY DR. STERGIOS ZACHAROULIS
    TRANSLATIONAL MEDICINE

    The translational laboratories at CUIMC have a close relationship with the PCFDTP allowing for the rapid translation of pre-clinical research into biologically-informed trials. Initiatives currently underway or in development include: Feasibility of organotypic slice culture (OSC) drug screening as a tool to select therapy for children with relapsed brain tumors. Dr. Stergios Zacharoulis (PI) is collaborating with Dr. Peter Canoll, neuropathologist and Director of the Neuropathology service at CUIMC to evaluate the feasibility of using organotypic slice cultures to obtain real-time drug screening results for pediatric patients diagnosed with a recurrent or progressive brain tumor. Organotypic slice culturing is a novel methodology that permits freshly operated tumor to immediately undergo a culturing process to preserve the tumor with its micro-environment. Direct drug testing is subsequently performed within 2-3 days to select the most optimal therapy for patient care. In addition, the genomic and epigenetic landscape will be examined using single cell sequencing to identify novel pathophysiologic alterations in these tumors. OSC has high translational potential to inform single agent or combination therapy selection for future pediatric patients, as well as, identify pathophysiologic alterations that may represent novel therapeutic targets for drug development. The study received IRB approval on 6/11/19. Two patients have been accrued and their data are under review.

    A Feasibility Study Examining the Use of Non-Invasive Focused Ultrasound (FUS) for Intravenous Carboplatin Administration in Children with Relapsed Refractory Brain Tumors (Dr. Zacharoulis, PI). The primary endpoint of this study is to evaluate the feasibility of safely opening the blood brain barrier (BBB) in children with relapsed brain tumors using NgFUS with microbubbles and neuro-navigator-controlled sonication. The BBB opening will be evaluated by both imaging (MRI) and by measuring the intra-tumoral concentration of Carboplatin. The study is currently under review by the FDA. Once approved, the study will be submitted for IRB approval.

    READ MORE

    Targeting master regulator (MR) dependencies in high-risk osteosarcoma (OS): Osteosarcoma is a devastating disease that disproportionately affects adolescents and young adults and there has been no therapeutic progress over the past 25 years. A group of outstanding investigators from CUIMC (Lead institution; Drs. Darrell Yamashiro, PI; Andrea Califano, Co-I), Dana Farber Cancer Institute (Dr. Katherine Janeway, GAIN Consortium PI), Memorial Sloan Kettering (Drs. Andrew Kung; Julia Glade Bender) and the University of California, San Francisco (Dr. Alejandro Sweet-Cordero) are collaborating to elucidate the tumorigenic mechanisms of OS and demonstrate how MR proteins, independent of DNA mutational status, may be targeted to disrupt tumor equilibrium (i.e., homeostasis). This study is also being utilized as a platform to train next generation physician-scientists. Specifically, Dr. Pavisic is receiving training and guidance from Drs. Yamashiro and Califano throughout the conduct of the project.
    Targeting master regulator dependencies in diffuse intrinsic pontine glioma (DIPG): In collaboration with the Califano Laboratory, PIPseq Program, Dr. Oren Becher (Ann & Robert H. Lurie Children’s Hospital of Chicago, Feinberg School of Medicine) and Dr. Chris Jones (Institute of Cancer Research in London), Drs. Zacharoulis and Pavisic are embarking on a parallel study to the OS investigations described above to: 1) characterize the DIPG gene regulatory networks and extend and refine OncoTreat-based prioritization of clinically relevant inhibitors predicted to invert the activity of DIPG-specific MR proteins; 2) evaluate the in vivo therapeutic potential of compounds identified in Aim 1 by assessing their activity in PDX DIPG mouse models; and 3) prospectively assess the feasibility of OncoTreat-based therapy selection for DIPG patients. The protocol is currently under IRB review.


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

    Tissue Immune Response, Immunoregulation and Tumor surveillance
    Research led by Dr. Erica Fallon

    Dr. Fallon’s work focuses on incorporating fundamental studies on mouse models with novel translational approaches on human tissue samples to investigate tissue immune responses, including protective immunity, and compare tissues from pediatric solid tumors to detect differences, investigate mechanism of disease formation, and potentially identify new treatment strategies. They have a unique resource to obtain multiple tissues from research-consented organ donors, enabling novel investigation of human immunity throughout the body over age, disease type, and genetic diversity. They will additionally investigate immunoregulation and tumor surveillance and look to identify focused areas for targeted prevention and/or therapy.

    Northwell Health – Feinstein Institute for Medical Research
    Northwell Health-Feinstein Institute for Medical Research

    Dr. Lionel Blanc

    Dr. Lionel Blanc


    Dr. Jeff Lipton

    Dr. Jeff Lipton

    Osteosarcomagenesis (OS) in Diamond Blackfan Anemia patients
    RESEARCH LED BY DR. JEFFREY LIPTON & DR. LIONEL BLANC

    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 PCF, they would not have been able to accomplish this work. Our contribution will be acknowledged in Lyon.

    PCF 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 PCF.

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

    READ MORE

    “Through decades of support from PCF, our group has been able to understand the biology of Diamond Blackfan anemia, connect the disease to cancer predisposition and develop animal and cellular models that we are now able to use to understand the biology of cancer,” says Jeffrey Lipton, at The Feinstein Institute. Diamond Blackfan anemia is a rare childhood bone marrow failure syndrome that results in the inability to produce red blood cells. Lipton says the Feinstein research has linked the disease to an increased rate of myelodysplastic syndrome (MDS; pre-leukemia), osteogenic sarcoma, and gastrointestinal luminal cancers such as cancers of the rectum, colon, stomach, and esophagus.

    In addition to the inability to produce red blood cells, these patients suffer from skeletal defects, and research supported by PCF allowed Drs. Blanc and Lipton to engineer the first animal model presenting these defects. “Due to the predisposition to osteogenic sarcoma in these patients, this animal model becomes invaluable to study the link between skeletal defects and bone cancer in these patients. Generating this model exemplifies the high risk – high reward research that would have not been possible without the support from PCF,” says Blanc.

    According to Lipton, one of the greatest challenges in pediatric cancer research is that a large number of great scientific ideas are put forward but the lack of funding makes it difficult to explore the more innovative and risky ideas put the research into execution. Despite the difficulties the field faces, Lipton looks forward to an exciting year filled with new developments.

    “Advances in targeted therapy based upon a remarkable growth in our understanding of cancer genetics and genomics will result in the development of new and effective targeted therapies, both with small molecules targeting the mutations that result in cancer, as well as immunologic approaches that enhance and modify the tumor micro-environment and the immune system itself to directly attack cancer, but only by understanding the biology of each cancer can this be accomplished” says Lipton.

    “Deaths from childhood cancer continue to decrease as researchers at all of the institutions supported by PCF make major strides in our efforts to prevent or cure all of childhood cancer. I look forward to continued support from PCF until we can all declare the battle won!”

    – Jeffrey Lipton M.D., Ph.D, Director, Pediatric Hematology/Oncology and Stem Cell Transplantation, Feinstein Institute of Medical Research, Northwell Health Professor of Pediatrics and Molecular Medicine, Hofstra Northwell School of Medicine

    Maria Fareri Children’s Hospital Westchester Medical Center
    Maria Fareri Children’s Hospital Westchester Medical Center

    Mitchell S. Cairo, MD

    Mitchell S. Cairo, MD

    Hematological Malignancies Program
    RESEARCH LED BY DR. MITCHELL CAIRO

    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. PCF funding has made possible a Nurse Practitioner Coordinator to support, coordinate, and assist in clinical research.

    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.

    Memorial Sloan Kettering Cancer Center, Department of Pediatrics
    Memorial Sloan Kettering Cancer Center, Department of Pediatrics

    Specific targeting of protein (mTOR) in Rhabdomysarcoma
    RESEARCH LED BY DR. FILEMON DELA CRUZ

    Dr. Dela Cruz’s group previously showed that genetic mutations (for example, a mutation in a gene called MYOD1) can contribute to a tumor’s ability to resist chemotherapies, thereby increasing the chances of treatment failure and death from cancer progression in rhabdomyosarcoma (RMS). His team previously examined the genetic mutations of RMS and found that these tumors may rely on specific biological signals to survive. They have performed initial experiments demonstrating that drugs targeting a protein called mTOR may be effective in reducing cell growth in RMS harboring a mutation in the MYOD1 gene.

    Dr. Dela Cruz plans to further validate his group’s initial results described above in tumor mouse models to determine if specific targeting of the mTOR protein will be successful in halting tumor growth. The results, if validated further, would provide evidence to support a new way of effectively targeting these tumors that, so far, have resisted commonly used therapies.

    READ MORE

    Because of the rarity of pediatric cancers, pharmaceutical companies do not devote the resources to develop drugs for these diseases, while the National Cancer Institute only allocates 4% of its annual budget towards pediatric cancer.

    PCF has been partnered with Memorial Sloan Kettering Cancer Center (MSK) since 1997, providing funds that have allowed investigators at the largest pediatric oncology program in the United States to undertake new research initiatives. The close collaboration between our researchers and physicians makes it possible for us to quickly bring these lifesaving treatments from the lab bench to the patient bedside. Funding from PCF has allowed MSK’s Department of Pediatrics to build an infrastructure through which the latest technologies can be applied to develop new treatments to help children affected by cancer.

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

    Pediatric Hematology/Oncology Fellowship Training Program
    RESEARCH LED BY DR. BILL CARROLL AND DR. ELIZABETH ROMAN

    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. In fact, 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. There have been numerous identified genetic mutations within leukemia cells that can confer intrinsic resistance to chemotherapy agents. However, there are also changes to the environment surrounding the leukemia cells which may also facilitate their pathologic growth and survival. Dr. Robbins will work in the Carroll lab at the NYU Perlmutter Cancer Center, in collaboration with the Aifantis lab, to investigate the “microenvironment” surrounding leukemic cells, in order to identify changes in this niche that may facilitate resistance to both conventional chemotherapy as well as novel immunotherapeutic treatments (e.g. CAR T cells).

    University of Chicago Medicine Comer Children’s Hospital
    University of Chicago Medicine Comer Children’s Hospital

    Dr. Kandel
    Prevention of formation of metastases
    Research led by Dr. Jessica Kandel

    During the last year, Dr. Kandel’s work expanded their investigations of the effect of the “microbiome” – the bacterial environment contained in our bodies and on our skin. Their findings underscore the realization that this environment significantly impacts children with cancer. Bacterial toxins change the way that blood vessels that feed cancers behave. For example, these molecules can promote blood vessel leakiness that allows cancer cells to escape and circulate, and can also prepare the sites where they will grow as metastases. By dissecting these mechanisms, they are increasingly able to predict how to block and restrain the spread of cancers.

    These studies are an important advance, because this is what ultimately causes the loss of children with cancer. This is why they focus on these questions: they seek to prevent the formation of metastases, the distant spread of cancer that is its most deadly form. Their work resulted in important publications this year, and in recognition by the National Cancer Institute that this is a novel and important line of investigation.

    Groundbreaking partnership between U Chicago and Advocate Aurora

    Sue Cohn M.D., and Luca Vricella M.D. and Jessica Kandel M.D. – leaders of our new Children’s Surgery, Oncology, and Cardiovascular Service Lines for children. This new collaborative structure reflects a groundbreaking partnership between the University of Chicago and the largest healthcare entity in Illinois, Advocate Aurora, which allows our programs to reach children not only throughout our state but beyond our borders. On this day, we were discussing a novel approach to a very complex case, a child with a tumor involving the lung and heart, which we are collaborating to treat in the most cutting-edge way possible. We would not be able to do this work without the partnership of the Pediatric Cancer Foundation!

    Groundbreaking partnership between U Chicago and Advocate Aurora
    Groundbreaking partnership between U Chicago and Advocate Aurora
    Groundbreaking partnership between U Chicago and Advocate Aurora
    Click here to read about Dr. Kandel in the Chicago Sun-Times
    READ MORE

    At Comer Children’s Hospital, the PCF-funded medical team has been researching how blood vessel growth impacts the spread and growth of tumors.“Tumors cannot grow unless they receive oxygen and nutrients from the bloodstream. In addition, the bloodstream provides a path for travel of cancerous cells to other sites in the body,” says Jessica Kandel, Surgeon-in-Chief at Comer Children’s Hospital. “For these reasons, the growing understanding of tumors’ control of blood vessels has yielded cutting-edge advances in cancer therapy. Our lab has focused on cancers of childhood, which have been understudied compared with adult cancers. We study tumors such as neuroblastoma, which remain deadly to most children diagnosed after infancy, and for whom current treatments have significant toxicities.”

    According to Kandel, PCF supported research was critical in demonstrating that blocking certain blood vessel-stimulating molecules could suppress the growth of pediatric cancers. The research led to the successful clinical trials of groundbreaking drugs in children, and to increasing the array of options available to pediatric oncologists.

    “Importantly, these projects involved not only basic investigation, but the training of future pediatric cancer investigators – of whom there are not enough,” says Kandel. “None of this work would have been possible without the steadfast support of PCF.”

    Mount Sinai Health System
    Mount Sinai

    Supporting Pediatric Graft-versus-host Disease Research
    RESEARCH LED BY DR. JAMES FERRARA & DR. JOHN LEVINE

    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.

    READ MORE

    Mount Sinai Physician-Scientist Bios: Pediatric Graft-verus-host Disease

    James Ferrara, MD
    James Ferrara, MD
    Professor of Medicine, Hematology and Medical Oncology; Professor of Pediatrics, Hematology and Oncology; Professor of Oncological Sciences; Icahn School of Medicine at Mount Sinai

    James Ferrara, MD is a physician-scientist whose clinical and research career has focused on bone marrow transplantation (BMT), particularly its major complication graft-versus-host-disease (GVHD). He co-directs the Mount Sinai Acute GVHD International Consortium with Dr. John Levine.

    Dr. Ferrara graduated from Georgetown Medical School and completed his pediatric residency and fellowship at Boston Children’s Hospital and the Dana-Farber Cancer Institute. He directed the combined adult and pediatric BMT program at the University of Michigan. The Icahn School of Medicine at Mount Sinai recruited Dr. Ferrara in 2014 to become the Ward-Coleman Professor of Cancer Medicine and to direct the Center for Translational Research in Hematologic Malignancies.



    John Levine, MD
    Professor of Medicine, Hematology and Medical Oncology; Professor of Pediatrics, Hematology and Oncology; Icahn School of Medicine at Mount SinaiJohn Levine, MD is a physician-scientist who has developed and conducted clinical trials on patients who undergo BMT and suffer from GVHD. Dr. Levine is the Chair of the Children’s Oncology Group Cellular Therapy Committee and Co-Director of the Mount Sinai Acute GVHD International Consortium with Dr. James Ferrara.Dr. Levine graduated from Eastern Virginia Medical School and completed his pediatric residency at Children’s Hospital of Los Angeles and fellowship at Memorial Sloan-Kettering Cancer Center. He was the director of the Pediatric BMT Program at the University of Michigan before he was recruited to Mount Sinai in 2015 to direct the BMT clinical research program.

    Pediatric Cancer Foundation

    Pediatric Cancer Foundation (PCF) is a non-profit charitable organization dedicated to eradicating childhood cancer by raising funds for groundbreaking research and early phase clinical trials conducted by world renowned doctors at the hospitals we support. This critical early phase funding is a stepping stone to later stage, larger institutional funding and is instrumental in providing novel treatments and potential cures.

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