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CHLA 2016 Impact Report
Read the 2016 CHLA Press On Impact Report
Press On Announces Research Partnership with GRU
$2.5 Million over 5 Years
September 3, 2015
On Thursday, September 3, 2015, Press On announced a $2.5 million gift to establish the Press On Translational Pediatric Oncology Program at the Georgia Cancer Center. This extraordinary act of generosity and goodwill will prove vital in the Cancer Center’s research and treatment of pediatric cancer, the leading cause of disease death in American children.
The Press On support for this novel program ensures that researcher and clinician opportunities to explore new drug development and treatment options – options that will form future standards of pediatric oncology care.
Translational research, also known as “bench-to-bedside,” harnesses the knowledge from basic sciences to produce new drugs, devices and treatment options for patients – even the youngest of them.
Press On and Rising Tide Foundation for Clinical Cancer Research award Grant for $800,000 for a Pediatric Neuroblastoma Trial
$100,000 from Press On
June 5, 2015
Atlanta, GA: The Press On Fund and the Rising Tide Foundation for Clinical Cancer Research (RTFCCR) are pleased to announce their first collaborative grant in the amount of $800,000 to Children’s Hospital of Los Angeles (CHLA), Children’s Healthcare of Atlanta (CHOA), and the University of Southern California, to initiate the New Approaches to Neuroblastoma Therapy (NANT) consortium’s Precision Clinical Trial.
The grant will allow Co-Principal Investigators Shahab Asgharadeh, M.D. of CHLA and Kelly Goldsmith, M.D. of CHOA to enrich bone marrow samples from children with relapsed neuroblastoma for the purpose of identifying specific genomic alterations leading to tumor progression and therapy resistance.
The primary aim of the study is to identify potentially targetable genetic and immunologic biomarkers in relapsed neuroblastoma.
The study will also assess a novel method for enriching tumor cells from bone marrow aspirates to support gene sequencing, which could potentially allow a much larger group of relapsed neuroblastoma patients to access future personalized medicine trials. A corroboration of this methodology could lead to a wider application in other adult or pediatric solid tumors.
Neuroblastoma is the most common solid tumor of the central nervous system in children. High-risk neuroblastoma is highly lethal and is responsible for 15% of childhood cancer related deaths. The five-year survival rate for high risk neuroblastoma stands at only 30% and recurrent neuroblastoma almost always fatal.
This grant was made possible by a strategic collaboration between Press On and the Rising Tide Foundation for Clinical Cancer Research in Switzerland. RTFCCR is dedicated to empowering and supporting pioneering scientists and clinical investigators to make critical headway in cancer research. With the cooperation of the CSRA Community Foundation, Press On Strives to leverage its research dollars with other cancer research oriented foundations and non-profits, like Rising Tide, and as evidenced in its Genomic Research Study with St Jude and Wash U, and its
The Rising Tide Foundation for Clinical Cancer Research is an entrepreneurial, private non-profit organization established in Switzerland in 2010. It is committed to empowering and collaborating with global research excellence centers and scientists to advance novel strategies and treatments to help cancer patients improve their quality of life and win the fight against cancer. RTFCCR is funding translational and clinical cancer research with the highest potential for near-term patient impact. Press On is a field of interest fund administered by the Community Foundation of the CSRA, which is located in Augusta, GA. Press On was founded by Atlanta residents Stephen and Erin Chance after their son, Patrick, was diagnosed with high risk neuroblastoma. The Chances joined forces with Tara and Turner Simkins of Augusta when their son, Brennan, was diagnosed with acute myeloid leukemia, an extremely deadly form of childhood cancer. Patrick died on his ninth birthday after fighting for almost six years. Brennan is alive and doing well after a groundbreaking treatment regimen including four bone marrow transplants.
The NANT consortium brings together a multidisciplinary team of laboratory and clinical scientists from 14 pediatric hospitals and institutions in the US and Canada with complementary expertise in genetics, biology, immunology, chemistry, pathology, biostatistics, clinical investigations, and imaging all with a single focus on finding better treatments for children with high-risk neuroblastoma.
Press On Funds CAR Study at CHOP
Augusta, GA: Many children with acute myeloid leukemia (AML), like Brennan Simkins have cancers that are labeled “incurable” with multi-agent chemotherapy and radiation. Through the collaboration between the Press On Fund and Dr. Richard Aplenc at the Children’s Hospital of Philadelphia (CHOP), it is this team’s goal to benefit these children from alternative therapies. Rapid progress has recently been made with adoptive immunotherapy approaches using human T cells engineered with synthetic chimeric antigen receptors (CARs) against tumor antigens for a variety of human cancers. Press On believes that AML should be no exception.
As evidenced in Brennan’s case, over one-third of children with AML relapse or are resistant to current best available therapies. Relapsed or chemotherapy-resistant AML accounts for more than 50% of childhood leukemia-related deaths. New treatments are needed to prevent relapses and to improve long-term cures. However, drug discovery research for childhood AML has made little progress to date in bringing new treatments to the clinic. The Hematologic Malignancies team at the Children’s Hospital of Philadelphia (CHOP) has recently published tremendous success with a novel T cell immunotherapy called CART19 for children with relapsed acute lymphoblastic leukemia, and is working to develop similar treatment approaches for children with AML. In earlier studies, CHOP created a new immunotherapy for AML called CART123, which rapidly killed human AML cells in specialized mouse models. However, CART123 treatment also caused serious side effects upon normal blood-forming cells, which could limit its usefulness in treating patients with AML and may require development of alternative approaches. With a $100,000 Press On Dr Richard Aplenc at CHOP will leverage his team’s grant clinical expertise in high-risk pediatric leukemias and their experience with immunotherapy development in the laboratory and in the clinic to conduct these research studies. Their research focuses on: (1) development and laboratory testing of a new CART38 AML immunotherapy that may decrease side effects upon normal blood cells and (2) identification of other pediatric AML proteins for future targeting with new T cell immunotherapies. Results from this work will help improve our understanding of the biology of childhood AML and to develop innovative therapies to advance to the clinic for testing in children with AML who otherwise have no remaining treatment options.
Pioneering Bone Marrow Transplantation for Neuroblastoma
January of 2014
*Note: the original commitment was $450,000 over three years, but two installments totaling $304,193.82 were sufficient to complete the research and no further contributions were required.
Augusta, GA: The Press On Fund invested $150,00 toward a three year, $450,000, commitment to a pioneering study that provides an immuno-therapy strategy for relapse-neuroblastoma patients. With this investment, Dr. Wing Leung, and his research team at St. Jude Children’s Research Hospital, are developing a novel 3 pronged approach to attack neuroblastoma, an approach that can be added to current treatment options with relatively little anticipated toxicity. This study uses Natural Killer ( or NK cells; the NK Cell study was also funded by Press On) or stem cells from parental donors to treat neuroblastoma. The parental donor, or haplo transplant, is a stem cell transplant protocol similar, but not identical, to the 3rd/4th transplants of Brennan Simkins, which were also pioneered at St. Jude. This study is now open to include neuroblastoma patients, and is being pursued in the honor of Patrick Chance, Press On’s inspiration in the fight against neuroblastoma. The Press On team believes this investment was appropriately spawned from both the Chance and Simkins families first-hand experience in the fight for their sons.
MIBG Cancer Therapy Center at Aflac Cancer Center
(Children’s Healthcare of Atlanta)
Press On has funded a new radiation therapy program at the Aflac Cancer Center of Children’s Healthcare of Atlanta with a $200,000 donation. This funding provided for all construction and material costs of the specialty radiation room, named in honor of Patrick Chance, and other aspects of the MIBG service. There are currently only a small number of centers around the country who currently offer MIBG treatment.
MIBG therapy is a treatment that uses radioiodine labeled metaiodobenzylguanidine (I-131 MIBG) to target certain tumors such as neuroblastoma and pheochromocytoma and delivers a much higher dose of radiation directly to the tumor. During this therapy, patients need to be treated in a special lead-lined room that prevents exposure to others. The MIBG therapy service will allow all children in Georgia to be treated in their home state and will allow for the Alflac Cancer Center to serve as a referral center for the southeastern United States.
Genome Study AML 7q deletion (Washington University, St; Louis & St. Jude Children’s Research Hospital)
$303,420 over 2 years
Next-generation DNA sequencing technology will be used in the St. Jude Children’s Research Hospital – Washington University Pediatric Cancer Genome Project to sequence the genomes of 600 pediatric cancer patients. The Press On Fund has committed to a two year, $200,000 funding for the sequencing of the rare subtype of AML, called AML 7q deletion (which is Brennan Simkins specific subtype of leukemia)
As part of the new project, DNA will be isolated from both the cancer cells and a normal, healthy tissue sample from the same patient. The healthy cells give the scientists a reference DNA sequence to which they can compare genetic alterations in the patient’s tumor cells. The scientists look for genetic differences in a patient’s cancer genome compared with his or her normal genome.
Typically, hundreds of mutations may be linked to the cancer, but the challenge for researchers is to sift through massive amounts of genetic data to distinguish the dozen or so “driver” mutations—those that are thought to initiate and contribute to tumor growth—from the “passenger” mutations, which are random, background mutations that are not relevant to the disease.
The advantage of the whole-genome approach is that scientists can move beyond a list of genes that have been previously associated with cancer to explore the entire genome and find meaningful cancer-causing mutations. Such a project holds enormous potential for improving the diagnosis and treatment of childhood cancers.
NK Cell Study (St. Jude Children’s Research Hospital)
In 2011, The Press On Fund dedicated $100,000 in seed monies to help initiate and secure the pilot study of Natural Killer Cell infusions for leukemia at St. Jude Children’s Research Hospital. This protocol particularly provides new hope to children with relapse AML (like Brennan Simkins), who historically have experienced one of the lowest survival rates of all pediatric cancers. This study will determine how long these NK cells work and survive in participants and will glean knowledge about the effectiveness of expanded use of NK cells against this disease. Dr. David Shook of St. Jude is the primary research physician leading this study, who had been one of Brennan’s caregivers during transplants 2, 3 & 4 at St. Jude.
MABG (Children’s Hospital of Philadelphia)
Neuroblastoma is known to be sensitive to radiation, thus our funding of the MIBG service at CHOA. However, MIBG does not target isolated tumor cells. Thus, researchers at Children’s Hospital of Philadelphia and the University of Pennsylvania are designing MABG, which is specifically intended to target disseminated disease.
Press On funded this research and development effort with a $50,000 grant. There has been significant progress towards the two specific aims of the project: 1) synthesizing high specific activity and 2) successfully creating a preclinical mouse model to study in vivo biodistribution and therapeutic trials.
Press On has received a grant request to further this project with the intent of applying for an NIH grant.
LMO1 (Children’s Hospital of Philadelphia)
Researchers at CHOP have discovered that a specific oncogene, LMO1, is associated with the most aggressive forms of Neuroblastoma. Press On funded research with a $50,000 grant to define the mechanism by which LMO1 drives Neuroblastoma progression. Additionally, in collaboration with researchers at Harvard, CHOP has developed a transgenic model of Neuroblastoma based on LMO1 overexpression. Third, the Press On grant allowed researchers to define the frequency of Neuroblastoma patients impacted by LMO1 gene mutation.
The development of this model allows for the manipulation of pathways and surveying for druggable targets that are upregulated by LMO1. Several potential targets are already identified as candidates. This genetic approach will set a new paradigm for targeted treatments of human cancers.
Based on this work, Dr. Maris at CHOP and Dr. Look at Harvard submitted a new multi PI-RO1 application for NIH funding. Despite enthusiasm from the peer review committee and an outstanding score, the grant was not funded dues to a scarcity of funds available for childhood cancer research. Because of this, Press On has received a grant request for bridge funding to continue the remarkable work.
PI3 Kinase Inhibitor (Children’s Healthcare of Atlanta)
Press On has initiated over $200,000 to Children’s Healthcare of Atlanta for Dr. Donald Durden’s research of a novel PI3 Kinase Inhibitor and Targeted Therapies for neuroblastoma.
Immunotherapy: Hu3F8, Turbo3F8, and a new Bi-Specific Neuroblastoma Anti-Body (Memorial Sloan Kettering Cancer Center, New York)
$20,000 from Press On
The Press On Fund recently partnered with the Band of Parents, The Isabella Santos Foundation, Arms Wide Open Foundation & Brooke’s Blossoming Home for Childhood Cancer Foundation (Partners) in committing $2,000,000 to Dr. Nai-King Cheung at Memorial Sloan-Kettering Cancer Center (MSKCC) for the development, manufacturing, and clinical testing of a new bi specific anti-body for the treatment of Neuroblastoma. This new antibody attaches to Neuroblastoma cells as well as T cells, thereby causing a much better tumor kill. Earlier iterations of the monoclonal antibody 3F8 relied upon the immune response from NK cells while T cells sat on the sidelines because they did not recognize Neuroblastoma as the enemy.
Press On’s collaboration with these other parent driven organizations creates the opportunity to expedite the development and testing of this important discovery. Unlike other monoclonal antibodies for the treatment of Neuroblastoma, the possibility exists that this bi-specific antibody will be near painless, capable of home administration, and could be used indefinitely for maintenance.
Press On’s partnership with these organizations, Dr. Cheung, and MSKCC has been in place for years. Press On funded the development and research of Hu3F8, which is now in the clinic at MSKCC, and “Turbo 3F8.” During the work on Turbo 3F8 the bi-specific antibody was developed and is so promising that it has been moved up front so that we can treat kids as soon as possible with this less toxic, more effective immunotherapy.
Monoclonal antibodies attach to Neuroblastoma cells and signal a child’s immune system to attack and kill neuroblastoma. Since first used in 1987, 3F8 treatment has greatly improved survival without lasting side effects.