Westchester organization provides critical support and funding to world-renowned institutions
Westchester County, NY – (February 27, 2017) – Pediatric Cancer Foundation (PCF), the 46 year-old, Westchester-based nonprofit committed to fighting pediatric cancer, is proud to kick off 2017 with a spotlight on the work of its associated hospitals and researchers. Committed to its mission of Coming Together for a Cure, the local nonprofit is currently funding ground-breaking research being performed at six world-recognized institutions (in alphabetical order): Columbia University Medical Center, NewYork-Presbyterian Morgan Stanley Children’s Hospital; Comer Children’s Hospital, University of Chicago; Feinstein Institute of Medical Research – Northwell Health; Maria Fareri Children’s Hospital; Memorial Sloan Kettering Cancer Center; and The Stephen D. Hassenfeld Children’s Center for Cancer and Blood Disorders at NYU Langone Medical Center. PCF’s involvement with these leading pediatric cancer research facilities is helping far more children and families than many realize.
“Common challenges doctors and researchers face when studying pediatric cancer is a shortage of funds, support, and equipment,” explains Bonnie Shyer, President of PCF’s Board of Directors. “While the majority of cancer funding goes towards combatting adult cancers, we are dedicated to assisting hospitals making strides in pediatric cancer research. We’re incredibly proud of how we’ve been able to help our partner institutions and outstanding researchers make important strides in this arena.”
Through generous donations and the hard work of over 200 volunteers, PCF is proud to support the following efforts at their “family” of affiliated medical centers:
Columbia University Medical Center, NewYork-Presbyterian Morgan Stanley Children’s Hospital: The Pediatric Cancer Foundation Developmental Therapeutics Program (PCFDTP) at Columbia University Medical Center (CUMC) was established with the single-minded goal of offering hope and compassion to children and their families when cancer recurs or does not respond to treatment. The CUMC program is one of only 21 in the nation designated by the Children’s Oncology Group and the National Cancer Institute to offer early drug development trials to children with incurable cancer. The PCFDTP is also home to the Precision in Pediatric Cancer “PIPseq” initiative, helping guide patients to the therapies most likely to work against their disease through comprehensive DNA sequencing. Because of PCF funds, CUMC is able to evaluate more than one child per week with incurable cancer to provide clinical trial options and access to the newest anti-cancer agents. Since the program’s inception, 176 children have enrolled on clinical trials to gain access to drugs that are not available outside of a research study and many more have benefited from individual FDA compassionate use applications and CUMC research.
“It takes at least 40 hours of research staff work-time to convince the drug company and the FDA to release the drug for compassionate use,” says Judith Elkins, PCF Medical Liaison for CUMC. “The research staff efforts are paid for by PCF funds. They receive no other compensation for this patient driven work.” Additionally, Elkins says the program serves as an “incubator” for new clinical trials led by young investigators. There are five trials currently in development through which CUMC is mentoring the next generation of pediatric cancer clinical researchers.
Comer Children’s Hospital, University of Chicago: 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.”
Feinstein Institute of Medical Research – Northwell Health: Research funded by PCF at The Feinstein Institute has been directed towards studying and understanding Diamond Blackfan anemia.
“Through decades of support from PCF, my 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 Jeffery Lipton, head of Patient Orient Research 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 esophagus, stomach, colon, and rectum cancers.
According to Lipton, the biggest challenge in the way of pediatric cancer research is a large number of great scientific ideas put forward with not enough minds and funding to 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 microenvironment and the immune system itself to directly attack cancer,” says Lipton.
Maria Fareri Children’s Hospital: PCF provides necessary grant support for the execution of state-of-the-art translational and clinical research in children, adolescents and young adults with leukemia and lymphoma. Funds from PCF have gone towards the Hematological Malignancy Program in the Childhood and Adolescent Cancer and Blood Diseases Center at the Maria Fareri Children’s Hospital.
Last year, PCF was able to fund research that led to the treatment of 62 children at Maria Fareri. Treatments for patients with B Cell Non Hodgkin Lymphoma and Hodgkin Lymphoma had a 100 percent complete response rate, while Acute Leukemia treatments yielded a 90 percent response rate.
As a result of PCF funding and advanced research, Dr. Mitchell S. Cairo, Director of the Children and Adolescent Cancer and Blood Diseases Center at Maria Fareri, anticipates the introduction of targeted Chimeric Antigen Receptor (CAR) Natural Killer (NK) cell therapy for patients with refractory or relapsed hematological malignancies.
Memorial Sloan Kettering Cancer Center: 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.
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. 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. Funding from PCF was used to support production costs for a new antibody therapy, developed at MSK by Nai-Kong Cheung, MD, PhD, to treat neuroblastoma – a devastating pediatric cancer. 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.
The Stephen D. Hassenfeld Children’s Center for Cancer and Blood Disorders at NYU Langone Medical Center: PCF has funded the Carroll Lab at NYU Langone Medical Center and its goal of improving outcomes for the most common childhood tumor, Acute Lymphoblastic Leukemia (ALL).
“While cure rates have improved dramatically, there remains a ‘cost’ in terms of short- and long-term side effects. Between 10 to 15 percent of children will relapse, with their prognosis remaining dismal,” says Dr. William L. Carroll, Director of the Division of Pediatric Hematology/Oncology at NYU-Langone Medical Center. “Increasing the dose and intensity of retreatment, including stem cell transplant, has failed to improve outcomes over the past 20 years. Based on these issues, we have sought to discover the underlying biological pathways that lead to drug resistance and relapse. Our approach is based on new innovations in genetic technology that allows us to profile the genetic fingerprint of leukemia samples at diagnosis and relapse. For example, we were one of the first laboratories to discover the most common mutation present at relapse, and researchers are actively developing the first drug to attack the specific cause of relapse.”
NYU Langone researchers are now exploring the uncharted territory of the cancer epigenome. According to Dr. Carroll, the “dark space” of the genome has a clear impact on the way a cell behaves. An emerging group of new anticancer drugs are being developed to target the changes caused by the epigenome.