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.
Our Impact
New York-Presbyterian Morgan Stanley
Children’s Hospital of 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.
New York-Presbyterian Morgan Stanley
Children’s Hospital of 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.
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
Dr. Lionel Blanc
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.
“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
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
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.
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
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!
Mount Sinai Health System
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.
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.
OUR FINANCIALS