See What We’ve Been Up to
The Press On Fund has made a 4-year, $1 million pledge in the first study to examine the efficacy of a drug that inhibits an enzyme certain pediatric brain tumors use to protect themselves from a natural immune response. This study will benefit children with recurrent brain tumors or newly diagnosed DIPG, a particularly aggressive brain tumor.
Drs. Theodore Johnson and David Munn, pediatric oncologists at the Medical College of Georgia and Georgia Cancer Center are the lead investigators on this exciting clinical trial; Press On and the CAM FUND previously invested $150,000 in an earlier phase of the study, which is exploring whether an inhibitor of the enzyme indoleamine 2,3 dioxygenase, or IDO, will allow for lower doses of chemotherapy and radiation, better quality of life during treatment, and still achieve improved survival for these children.
The idea is to unleash a natural immune response to attack the tumor. “We also have evidence that the addition of an IDO inhibitor to these children’s treatment will help us reduce the toxicity often associated with aggressive cancer treatment, improve quality of life during treatment and in some cases offer at least the potential for cure,” Johnson says. Standard therapies cure about 60 percent of children with these brain tumor types, and the investigators hope their new approach can help the 40 percent who have recurrent disease.
Adding the IDO inhibitor Indoximod to the mix helps standard treatments work better — even at a lower dose — by setting the immune system free to respond as it should.
For example, children in the study will be taking a low-dose, once daily chemotherapy pill known to have relatively modest side effects. In their first-in-humans phase 1 trial, which focused more on safety than efficacy, the investigators saw children finish high school, take their SATs and start and do well in college while on the protocol. “You wouldn’t be able to do that if you were getting high dose, intravenous chemotherapy,” Munn says.
The trial also will provide frontline treatment to 30 children with newly diagnosed diffuse intrinsic pontine glioma, or DIPG, a brain tumor for which there is no standard therapy considered curative. For these children, the trial pairs the Indoximod and radiation followed by maintenance therapy with chemotherapy and Indoximod, with the goal of improved overall survival compared with current approaches, which typically produce only a short-term response.
Our donation supports the LLS PedAL clinical trial, a global precision medicine master clinical trial run by a team of renowned pediatric oncologists with the goal of matching children with relapsed acute leukemia to a targeted therapy based on the specific abnormalities driving their cancer. The goal of the trial is to test multiple targeted therapies simultaneously at up to 200 clinical sites worldwide.
LLS PedAL is a key component of The LLS Children’s Initiative, a comprehensive $100 million multi-year endeavor to take on children’s cancer from every direction, including new research grants to advance novel therapies, enhanced free education and support services for children and their families, and renewed policy and advocacy efforts.
Augusta, GA – Press On has granted $150,000 to a pediatric brain tumor study at St. Jude Children’s Research Hospital run by Dr. Giles W. Robinson. The study intends to perform extensive molecular analysis on pediatric brain tumors of patients that are actively receiving therapy or about to receive therapy. The goal is series of trials that tailor and evaluate therapy relative to molecular features.
Thanks to technological advances in recent years, it is now recognized that brain tumor categorization is much more complex than previously thought. Pediatric brain tumors are being re-categorized and sub-categorized based on their many different cellular signals. Says Dr. Robinson, “the primary failure of treating pediatric brain tumors remains an inability to exploit and target the biologic errors that have led to the disease.” If cancer is traffic control gone wrong, “current cancer therapy with surgery, radiation and cytotoxic chemotherapy is akin to forcefully removing the entire congested city block. If successful, the source of congestion is removed but what remains will be damaged, scarred, and rarely reusable.” Instead, Dr. Robinson intends to find a targeted solution to the problem.
$300,000 over 2 Years
New York, NY – Press On is donating $300,000 toward a clinical trial led by Memorial Sloan Kettering Cancer Center (MSK) evaluating a promising new vaccine to counter neuroblastoma. This gift has been matched by The Band of Parents, a group of parents and friends of children with the rare pediatric nervous system cancer.
Until recently, only a small percentage of children with advanced neuroblastoma live two years past a second remission, but with the vaccine clinicians have seen a high percentage of patients respond well. These results prompted the MSK team to investigate whether vaccinating earlier builds a child’s immunity and improves outcomes even more dramatically.
Augusta, GA – Press On has granted $50,000 to a study developing immunotherapy treatments for Medulloblastoma, Neuroblastoma, and other solid tumors at Medical University of South Carolina Children’s Health. The grant was a match of a $50,000 donation made by the South Carolina band The Blue Dogs, bringing the total to $100,000. Run by Dr. Jacqueline Kraveka, the study that received the grant is now in clinical trials.
“Support from the Press On Fund has been crucial for MUSC Children’s Health to open and conduct six neuroblastoma and early phase clinical trials,” reports Dr. Kraveka’s team. Three of those trials are Phase I trials – MUSC Children’s Health is the only facility in South Carolina to offer Phase I pediatric cancer trials! Another trial, an exciting pilot study titled the PEDS-PLAN, is the first to incorporate precision medicine into upfront therapy for newly diagnosed high-risk Neuroblastoma. The survival rate for high-risk Neuroblastoma is currently 60% – something Press On and Dr. Kraveka’s team would like to change. There are currently 14 patients enrolled across the six trials, with goals to increase their clinical trial portfolio and enroll more patients in 2018.
Children’s Hospital of Georgia · $150,000
Augusta, GA, – Press On Fund, with the addition of CAM FUND, a like-minded nonprofit, has contributed to a Children’s Hospital of Georgia clinical trial for pediatric brain tumors run by Dr. Ted Johnson. The funding will allow for the enrollment of new patients and for the inclusion of DIPG patients in this clinical trial for cutting-edge immuno-chemo-radiotherapy for progressive brain tumors. DIPG is a highly aggressive and difficult-to-treat brain tumor that is currently “a massive unmet need as a pediatric orphan disease.”
“This is just the first of many collaborated efforts we expect to bring to medical research to help find a cure for childhood brain cancer. The innovative work that Dr. Johnson is doing shows great promise to getting us there. Together we are stronger for the voices that have been silenced too soon,” said Mark and Terri Rettig, founders, CAM FUND.
“With contributions and ongoing support from wonderful organizations such as the CAM FUND and Press On, medical research is able to further its reach in finding a cure,” said Dr. Ted Johnson, Associate Professor of Pediatrics, at the Medical College of Georgia.
Final $130,806.18 of $450,000
Augusta, GA – The Press On Fund has completed a 2014 pledge of $450,000 with an investment of $130,806.18 in a pioneering study that provides the world’s first Bone Marrow Transplant immunotherapy strategy for relapsed and high-risk Neuroblastoma patients. Pioneered by Wing Leung, M.D., Ph.D., and continued under the leadership of Brandon Triplett, M.D., this St. Jude Children’s Research Hospital project aims to change the current poor prognosis for high-risk and relapsed Neuroblastoma.
The project has two specific aims: the development of Natural Killer cell (NK cell) therapy; and the development of parental stem cell transplantation (haplo-transplant), both for Neuroblastoma. Over the past three years, this study has resulted in three clinical trials, all showing promise with two still ongoing.
St. Jude Children’s Research Hospital is one of the world’s premier centers for the research and treatment of pediatric cancer and other catastrophic childhood diseases. It was there that Brennan Simkins, whose parents Tara and Turner are among the leaders of the Press On Fund, received two life-saving haplo-transplants for his Acute Myeloid Leukemia. Those haplo-transplants resemble but are not identical to the very stem cell transplantation being studied in this project, this time for Neuroblastoma.
This study is being pursued in honor of Patrick Chance, who remains Press On’s inspiration to study Neuroblastoma, even after his passing in 2012. 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 and continues Patrick’s legacy as his “healing hands on earth.”
Augusta, GA – Press On Fund is combining forces with TARGET Pediatric AML (TpAML) to establish the largest ever collaborative research initiative to cure pediatric Acute Myeloid Leukemia (AML). Press On’s $250,000 investment in TpAML represents its first commitment to this nationwide project, endorsed by the Children’s Oncology Group as the “highest potential, greatest need” for pediatric AML research.
TpAML’s goal is to speed personalized, precision medicine to young AML patients with a two-pronged approach: using comprehensive target discovery across all AML types to maximize the use of all available targeted agents, even those designed originally for other diseases; and providing clinical genomic testing to all active pediatric AML patients treated at COG-affiliated institutions.
This initiative fills the gaps in the Leukemia and Lymphoma Society’s multi-million dollar “Beat AML” campaign, which currently only focuses on AML patients 60 years of age and older.
From Dr. Peter C. Adamson, COG Chair: “The Children’s Oncology Group is honored to have the support of the Press On Fund for the TARGET Pediatric AML Initiative. The Press On Fund’s support of this pioneering initiative will help us build a unique platform for discovery that will lead to better treatments and improve the outcome for all children with acute myeloid leukemia.”
Read the 2016 CHLA Press On Impact Report
$100,000 from Press On
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.
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.
St. Jude Children’s Research Hospital · $450,000
Augusta, GA: The Press On Fund invested $150,00 toward a three-year, $450,000, commitment to a pioneering study that provides an immunotherapy strategy for relapse and high-risk neuroblastoma patients. With this investment, Dr. Wing Leung and his research team at St. Jude Children’s Research Hospital are developing a novel three-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.
Children’s Healthcare of Atlanta · $200,000
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.
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.
St. Jude Children’s Research Hospital · $100,000
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.
Children’s Hospital of Philadelphia · $50,000
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.
Children’s Hospital of Philadelphia · $50,000
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.