Lurie’s Quarterly Impact Update – December 2019
The generous support of Cal’s Angels is enabling cancer biologist Xiao-Nan Li, MD, PhD, to break new ground and accelerate progress for children and young adults battling cancer. We appreciate your commitment and are pleased to provide an update on recent activities made possible as a result of your generosity.
As Director of Pediatric Xenograft Modeling at Lurie Children’s Stanley Manne Children’s Research Institute, Dr. Li is working to develop more precise and effective treatments to combat pediatric cancer. His expertise in creating animal tumor models that parallel the unique attributes of individual tumors that develop in humans (patient-derived orthotopic xenograft or PDOX models) truly sets apart our Precision Medicine Oncology initiative from all others.
Dr. Li has transferred to Chicago frozen xenograft cells stored in liquid nitrogen from most of the original models (> 120) he created at his previous institution, Texas Children’s Hospital. In his new laboratory in the Louis A. Simpson and Kimberly K. Querrey Biomedical Research Center, Dr. Li is working to recreate his mouse models of tumor biology derived from patient tumor tissue and begin testing a variety of approved and investigational therapies. He has built a team of four accomplished researchers to oversee this work and is he process of recruiting up to five additional postdoctoral fellows/research technicians.
Dr. Li’s PDOX modeling work continues to advance the development of novel treatments for the most aggressive childhood brain cancers. While he remains focused on this important work, his team is now extending their studies to other pediatric cancers such as leukemia and solid tumors. The research group has implanted five leukemia samples from Lurie Children’s biorepository in animal models and, most recently, 60 mice with malignant brain tumors in the new animal facility in the middle of August 2019. The mice are being closely monitored for evidence of tumor formation. Dr. Li’s lab is actively expanding the cohorts of their special strain of immune-deficient animals through breeding and is now starting implantation of new childhood cancers tumors, including nearly 21 solid tumors/brain cancers and 33 leukemias. He expects to implant 50-70 new patient tumors in 2019, and steadily increase the number to 80-120 per year after 2020. With a tumor take rate of 50-60 percent, this should result in 20 new models by 2020, and more than 40 new models after 2021.
Generous support provided through Cal’s Angels will accelerate Dr. Li’s work by giving him the tools to lead the way in advancing truly personalized medicine. He is in the process of acquiring an advanced high-throughput robotic drug screening system that will give him the capacity to study thousands of potential new treatments with greater speed and efficiency than ever before. The Echo Liquid Handling technology and Access Laboratory Workstation are powerful tools to support drug discovery, allowing us to simultaneously test thousands of approved anti-cancer drugs as well as new compounds in different combinations. A robotic arm moves droplets of candidate drugs, in miniscule amounts, from storage tubes into wells on assay plates, preventing the risk of cross contamination from human contact. Tumor cells are added to the wells, and the robot monitors for promising drug responses. Because the technology works 24 hours a day, seven days a week, Dr. Li hopes to be able to screen thousands of drug combinations. Those that show the greatest success will be rapidly advanced for additional studies in his PDOX models. This equipment has been ordered and he is now awaiting delivery. Additionally, Dr. Li plans to expand his current drug library from 8,000 to 110,000 therapies by including more investigational agents against human cancers. Ultimately, his goal is to translate the most promising therapies into future clinical trials for children.
We are deeply grateful for your investment that has allowed Dr. Li to integrate this cutting-edge technology into his research. Your support is advancing the pursuit of new discoveries that have the power to change and improve young lives.
One Million to the Cal’s Angels Drug Discovery Fund – August 2019
We are so excited to announce that with our most recent donation to Ann & Robert H. Lurie Children’s Hospital of Chicago, the Cal’s Angels Drug Discovery Fund has reached the ONE MILLION dollar mark! These additional funds will advance pediatric cancer research in the following ways:
• The purchase of the first Echo 550 robotic drug screening system, which works 382 times faster than a human.
• The acquisition of a new and updated drug library, increasing drug options to over 11,000, to support the work of Dr. Xiao-Nan Li. This is a critical path as the drugs are needed to even begin a clinical trial.
• Salary support for research staff.
In 2017 we advanced our mission and commitment to pediatric cancer research and we will NOT stop until a cure is found. However, this is the next step to finding more treatment options with less toxicity and eradicating the horrific side effects associated with pediatric cancer. Every child deserves more!
It is through the generous support of our donors and volunteers that this is possible, so a huge heartfelt THANK YOU to all of you!
Innovative Research Leading to Drug Discovery – January 2019
Ann & Robert H. Lurie Children’s Hospital of Chicago
Cancer biologist Xiao-Nan Li, MD, Ph.D., Director of Pediatric Xenograft Modeling at the Stanley Manne Children’s Research Institute at Lurie Children’s, has pioneered the development of clinically relevant mouse models of pediatric cancers for drug screening. Specifically, these models are made by taking tumor tissues from patients and implanting them into the anatomic locations in mice reflective of the child’s tumor, where they are tested with specific drugs to determine the best course of therapy. Because the mouse model tumors behave like human tumors, this method produces the most accurate recreation of how tumors in children will respond to specific therapies. With 130 models of different brain tumor subtypes, Dr. Li is recognized as having developed the largest number of tumor models in the world. He is expanding this successful approach to create models for all types of cancers, not only at diagnosis but also at recurrence and at the time of death to better understand tumor invasion and test new drug combinations that can significantly prolong survival times. Our goal is to move to personalized clinical drug screening models that would directly benefit each patient. Cal’s Angels’ support serves as the catalyst to push this important work forward.
Lurie’s Quarterly Impact Update – September 2017
Thank you for your support to accelerate discovery of new oncology therapies for young people treated at Ann & Robert H. Lurie Children’s Hospital of Chicago. Your dedication has been essential as we fully implement our Precision Medicine initiative, including the pending hire of a research technician to expedite research and support the talent and resources we have brought to Lurie Children’s over the last few months.
Lurie Children’s launched its Precision Medicine Initiative in early 2017—an imperative to bring the necessary talent and resources together if we are to find the best therapies that will be most effective against the unique characteristics of each child’s cancer. In recent months, we have made considerable progress in this initiative:
• Our oncology team, together with researchers at our Stanley Manne Children’s Research Institute, are now using an individual’s genetic sequencing and other advanced technologies to analyze biological data, and identify disease biomarkers and potential drug targets. Most tumors arise following mutations in critical genes that lead to uncontrolled cellular growth. In some patients, identifying these genes may aid in tumor diagnosis and define the most appropriate therapy. Other tumors may acquire additional mutations during or following therapy that result in relapse. These examples are just the beginning of what we are now understanding through Precision Medicine. Extensive research is required to identify additional genes that mutate and guide us to target those pathways.
• Our Precision Medicine initiative has also introduced a comprehensive biobanking policy for collecting tissues that are crucial for research, implemented a Predisposition Clinic for patients and families, and recruited critical personnel, including a genetics counselor and a clinical research professional.
As a testament to the growing strength of our research and clinical care, two new globally acclaimed physician-scientists have joined our team. They will provide critical value by developing orthotopic xenograft models (PDX). These orthotopic models are made by taking tumor tissues from patients and implanting them into the anatomic locations in mice reflective of the child’s tumor, where they are tested with specific drugs to determine the best course of therapy. This method is difficult to conduct, but produces the most accurate recreation of how tumors in children will respond to drugs.
• Oren Becher, MD, recently came from Duke University School of Medicine and has established his laboratory. Dr. Becher has extensive experience developing models of pediatric brain tumors, particularly Diffuse Intrinsic Pontine Glioma. These models enable us to study genes and mutations that influence cancer development and evaluate novel therapies that could translate into new clinical trials for children. His work will also be informed by the range of clinical trials available at Lurie Children’s and the multi-institution trials we participate in.
• Xiao-Nan Li, MD, PhD, will join our program from Baylor University Texas Children’s Hospital in January 2018. His team will develop animal models in all pediatric cancers, not only at diagnosis, but also at recurrence and at the time of death. Dr. Li’s team includes Research Assistant Professor Xingding Zhang, PhD, who joined our faculty in late spring to establish Dr. Li’s lab and transfer cell lines and breeding colonies from Baylor to Lurie Children’s to begin drug screening of molecular-targeted agents.
Your funding will bring a vital link to our oncology team by allowing these transformational initiatives and world-class investigators to speed up research and ultimately bring better therapies to children sooner. I look forward to updating you on our progress as we continue our search for a research technician with the right skill set to partner with these scientists.
Lurie’s Quarterly Impact Update – June 2017
We remain deeply grateful for Cal’s Angels’ leadership gift to the Division of Hematology, Oncology, Neuro-Oncology and Stem Cell Transplantation at Ann & Robert H. Lurie Children’s Hospital of Chicago. Your support will accelerate our efforts in pediatric drug discovery, making it possible to bring new therapies from the laboratory to human clinical trials in a more efficient and cost-effective way. As the focus of pediatric cancer research shifts to precision medicine and the development of targeted therapies based on the molecular signatures of specific types of cancer, rather than the site of origin, the work made possible through your support promises to pave the way for less toxic and more individualized therapies that will improve outcomes for our patients and all children with cancer.
Specifically, we plan to utilize a portion of your $525,000 investment to hire a new research technician to support pre-clinical laboratory studies of new drug compounds. It will also cover costs associated with acquiring various therapies for testing, including molecularly targeted therapies that form the foundation of Lurie Children’s new Precision Medicine Initiative.
Launched earlier this year, Lurie Children’s Precision Medicine Initiative is revealing insights into the biological, environmental and lifestyle factors affecting each child’s disease and using them to create individually tailored treatment plans. Working in close alignment with investigators at the Stanley Manne Children’s Research Institute, our oncology team will use genetic sequencing and other advanced technologies to analyze biological data and identify biomarkers of disease and potential drug targets. Patients will receive personalized recommendations for treatment according to the assessment of their tumor. Information from each child will be fed back into the lab to fuel discovery of increasingly more precise diagnostics and therapies. Our research teams will use laboratory models to study new drug combinations and solve the significant problem of drug resistance that limit the effectiveness of cancer therapies. As we gather data from increasingly larger numbers of patients who undergo advanced sequencing, this will inform new research questions that will form the basis for future studies to develop more targeted and effective treatments. The impact of our Precision Medicine Initiative will be felt across the spectrum of cancer care – from early diagnosis and treatment to strategies to improve long-term outcomes.
Fueling New Collaborations
Significant resources are needed to advance drug discovery in pediatric oncology. Your gift provides crucial funding to recruit a research technician who will partner with investigators across the Stanley Manne Children’s Research Institute as well as Northwestern University Feinberg School of Medicine. The ideal candidate will have a chemistry background and experience partnering with the pharmaceutical industry to acquire compounds for single and/or combination testing in established mouse models. Examples of two studies that will benefit from the research technician’s support include:
•Rintaro Hashizume, MD, PhD, has created an animal model to examine the effectiveness of delivering various chemotherapy agents intranasally to children with aggressive brain tumors, particularly diffuse intrinsic pontine glioma (DIPG), which has a very poor survival rate. Dr. Hashizume’s goal is to complete pre-clinical therapeutic testing in the next 3-5 years and then proceed to clinical trials.
•In January, world renowned researcher Oren Becher, MD, officially joined Lurie Children’s from Duke University. Dr. Becher has extensive experience developing novel mouse models of pediatric brain tumors. He has developed a genetically engineered mouse model of DIPG that faithfully replicates the human disease in children and represents an important platform with which to study the biology of DIPG and evaluate new therapeutics.
The addition of a new research technician will speed efforts to develop clinical trials and increasingly more effective therapies.
Improved diagnoses, earlier interventions and superior outcomes. These are the promises to our patients and families that drive all that we do at Lurie Children’s. Today, we are closer than ever to achieving our vision. Cal’s Angel’s investment will be a powerful catalyst to accelerate research discovery and transform the lives of children.
Lurie’s Quarterly Impact Update – December 2016
As we start a new year, I would like to express my appreciation for the support and thoughtfulness Cal’s All-Star Angel Foundation has demonstrated in order to provide brighter futures for children and their families. Your investment is enabling us to obtain the necessary personnel and resources to pursue breakthrough research that will produce increasingly more individualized and effective treatments for our patients.
Early in the coming year, we will begin the search for a skilled research technician to coincide with the arrival of internationally recognized cancer biologist Xiao-Nan Li, MD, PhD. As you may recall from our last report, Dr. Li will assume the new role of Director of Pediatric Xenograft Modeling at the Stanley Manne Children’s Research Institute in January. He is a world leader in creating novel mouse models, called patient-derived orthotopic xenograft (PDOX) models, for preclinical drug screening and biological studies of pediatric tumors. Dr. Li actively participates in National Cancer Institute-sponsored research consortia, including serving as Chair of the High Grade Glioma Committee of the Pediatric Brain Tumor Consortium. His expertise and substantial research funding will allow us to advance our Precision Medicine initiative. The pending hire of a research technician will help to accelerate the work of this accomplished investigator and bring the most effective personalized drug combinations to patients at a rapid pace.
I am pleased to share that our first joint project with Dr. Li is already in the planning stages. This project aims to develop PDOX models from surgical samples of various types of pediatric brain tumors obtained from patients treated at Lurie Children’s and Texas Children’s Hospital. The PDOX models will faithfully replicate the biology of recurrent brain tumors and provide us with a much-needed and ample supply of recurrent tumor tissues for detailed genomic analysis to understand mechanisms of therapy resistance and identify new targets. Mice bearing recurrent brain tumors will be ready-made for testing novel drug combinations. Our ultimate aim is to secure certification for this novel system so that preclinical drug testing results can be applied to guide clinical treatment of children with malignant brain tumors.
Research has the best potential to transform pediatric health care and ultimately, to improve children’s lives. Your partnership is invaluable to our work. I look forward to updating you on our progress as we begin our search for a research technician. I would be pleased to arrange for members of Cal’s Angels to meet with Dr. Li and tour his laboratory once he settles in to his new role. Feel free to contact T.J. Johnson, Assistant Vice-President of Major Gifts, at email@example.com or (312) 227-7251.
With deepest gratitude,
Stewart Goldman, MD
Division Head, Hematology, Oncology, Neuro-Oncology & Stem Cell Transplantation
Meryl Suzanne Weiss Distinguished Professor in Hematology, Oncology and Stem Cell Transplantation
Professor of Pediatrics, Northwestern University Feinberg School of Medicine
Director, Center for Clinical Trials Research
Co-director, Falk Brain Tumor Center, Stanley Manne Children’s Research Institute
Announcement: Cal’s Angels funds Lurie Children’s groundbreaking children’s cancer research
Cal’s Angels partners with Lurie Children’s Hospital and Northwestern University Feinberg School of Medicine to create new treatments for children’s cancers and save more children’s lives!
Although significant advances in care and research have been achieved over the last half-century, cure rates for certain forms of childhood cancers have seen little to no improvement. Moreover, aggressive therapies currently used to treat these cancers can lead to life-long complications such as learning disabilities, heart disease, secondary cancers, and more.
While the need to develop safer, more effective therapies has never been greater, this comes at a time when there is a growing crisis in pediatric drug discovery as fewer new drugs are being developed and approved for use in children. The pharmaceutical industry has approximately 900 adult cancer drugs in the development pipeline and almost none for children’s cancers. The development and testing of new agents for pediatric populations has been hit particularly hard because the diseases that affect children are different, have a smaller potential market, pose greater regulatory obstacles and offer a limited return on investment. As a result, there is a critical imperative for the medical community to initiate, develop and evaluate new therapies specifically formulated for children and adolescents.
Cal’s Angels and Lurie Children’s are committed to moving research forward as quickly as possible to save more children’s lives. Working collaboratively with researchers at Northwestern University Feinberg School of Medicine, Lurie Children’s will initiate a drug discover program, sponsored by Cal’s Angels, for the repurposing of existing drug compounds and ongoing testing of new medications. Specifically, the program will use patient derived xenografts – where a sample of a child’s own cancer, either bone marrow or solid tumor, is used to grow a tumor in mice – to speed development of less toxic and more effective therapies for pediatric cancer in a more cost effective and efficient way. Accountability and quarterly reporting will occur throughout the trial.
Find out how you can be a part of this groundbreaking research and help save the lives of children with cancer.