PLEASE INVEST IN PROJECT IMPACT TO SAVE THESE KIDS

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Sophia Allison
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2 PLEASE INVEST IN PROJECT IMPACT TO SAVE THESE KIDS MORE THAN 4,600 AMERICAN CHILDREN HEAR THE WORDS BRAIN TUMOR UTTERED BY THEIR DOCTORS EVERY YEAR. FOR THOSE PATIENTS AND THEIR FAMILIES, THE IMPACT OF THIS DIAGNOSIS IS OVERWHELMING AND LIFECHANGING, FOREVER. PEDIATRIC BRAIN TUMORS TAKE MORE CHILDREN UNDER 19 YEARS OF AGE FROM US THAN ANY OTHER CANCER, INCLUDING LEUKEMIA. FOR PEDIATRIC HIGH-GRADE GLIOMAS, THE DIAGNOSIS IS PARTICULARLY GRIM. FOR ONE TYPE CALLED DIPG, MOST PATIENTS ONLY SURVIVE NINE MONTHS, OR ABOUT AS LONG AS THE TYPICAL SCHOOL YEAR. AND OVERALL, ONLY AROUND 25% OF ALL THOSE WITH HIGH-GRADE GLIOMAS SURVIVE MORE THAN 5 YEARS, RATES THAT TREND ON PAR WITH WHERE THEY WERE 40 YEARS AGO. THERE IS CURRENTLY NO STANDARD OF CARE FOR THESE KIDS. THIS IS COMPLETELY UNACCEPTABLE. The harsh realities of pediatric brain tumors, however, are set against the promising backdrop of advances and innovation in the research of pediatric cancers as a whole. Through the power of molecular biology and genetic sequencing, we now know more than ever before about the make-up and biology of these tumors. And by applying that knowledge, new therapies could be developed that not only improve a child s overall survival, but could lead to new treatments that could offer cures. These advances are clear signs that progress can be made for children with brain tumors. National Brain Tumor Society is committed to increasing the survival rates of children with high-grade gliomas and shining a bright spotlight into this dark corner of pediatric cancer. Through Project Impact: A Campaign to Defeat Pediatric Brain Tumors, the National Brain Tumor Society will drive the discovery of more therapeutic options for pediatric brain tumor clinical trials, and enable them to be delivered to patients more quickly. In doing so, the National Brain Tumor Society will enable the development of the first ever standard of care for pediatric high-grade gliomas. We ask you to please invest in this critical effort to help cure these kids. Your generous donations or legacy giving will help generate new research that could lay the path directly to better treatments and ultimately a care. Please join us now.

PROJECT IMPACT AND DEFEAT PEDIATRIC BRAIN TUMORS CREATING A STANDARD OF CARE FOR PEDIATRIC BRAIN TUMORS FOR THE FIRST TIME The Defeat Pediatric Brain Tumors is a powerful, unique global research and

gliomas the most lethal of pediatric cancers. The program infrastructure, researchers, and four interrelated cores that make up the program are immediately ready for deployment.

The program is an NBTS-led research collaborative designed to accelerate research through a platform that fosters collaboration as well as research data, information and materials sharing.

3 PROJECT IMPACT AND DEFEAT PEDIATRIC BRAIN TUMORS CREATING A STANDARD OF CARE FOR PEDIATRIC BRAIN TUMORS FOR THE FIRST TIME The Defeat Pediatric Brain Tumors is a powerful, unique global research and drug discovery program which aims to improve clinical outcomes for pediatric brain tumor patients and enable the development of the first-ever standard of care for treating pediatric high-grade gliomas the most lethal of pediatric cancers. The program infrastructure, researchers, and four interrelated cores that make up the program are immediately ready for deployment. All we need is funding. The program is an NBTS-led research collaborative designed to accelerate research through a platform that fosters collaboration as well as research data, information and materials sharing. The platform consists of four cores that work on critical areas of research simultaneously and in concert with one another to encourage sharing of findings and new discoveries in real time to aim toward a cure. Project Impact: A Campaign to Defeat Pediatric Brain Tumors will resource this program by initially providing it with $2.5 million in funding over one year, and $5 million total funding over five years. Funding will support work across four key areas of scientific focus, known as cores: Discovery Core (Molecular Diagnostics and Target Discovery): Catalyze the process of discovery to build off of new advances in genetic sequencing and expression. Biomarker Core (Identification & Validation): Identify and validate biomarkers for pediatric brain tumors across various modalities, including tumor tissue, liquid biopsy (plasma and other body fluids), imaging, etc. Preclinical Modeling & Drug Screening Core: Develop the critically needed, relevant mouse models and translate molecular and drug discovery in animal models into targeted therapies that improve patient survival and outcomes. Smart Trials Core (N-of-1 clinical trials): Drive the creation of biology-focused clinical trials, with new findings from the first three cores informing the design, target population, and testing of treatments. Human data from clinical trials will also provide information back to the preclinical cores and seed the development/refinement of new research.

of a new era in 2016. From that strong foundation, we have launched the Defeat Pediatric Brain Tumors Research Collaborative program.

4 of a new era in From that strong foundation, we have launched the Defeat Pediatric Brain Tumors Research Collaborative program. Pediatric brain tumors are the leading cause of cancerrelated death among children and young adults ages 19 and younger, surpassing leukemia. Over the past decades, little progress has been made in the treatment of pediatric brain tumors, while tremendous advances have been made across the rest of the pediatric cancer spectrum, as new discoveries in the lab have led to new treatments in the field that have significantly improved survival. For example, the most common form of childhood cancer, acute lymphoblastic leukemia, was generally diagnosed as fatal 50 years ago; today, cure rates for the disease reach close to ninety percent. However, for the 4,600 children diagnosed annually with a brain tumor, the scientific needle has barely moved, with only 25% of children suffering from a malignant brain tumor surviving five years rates that trend on par with where they were nearly forty years ago. But, never before in the history of mankind s battle with cancer has so much progress been made. Critical findings in recent years including many funded by the National Brain Tumor Society have positioned the field of pediatric brain tumors to enter a new era in treatment and survival. Among the most transformational of these findings was clear evidence that pediatric high-grade gliomas are biologically and molecularly distinct and separate from their adult counter-parts. These findings allow scientists to break from the norm of the past decades, and catalyze them to develop pediatric-specific treatments that finally move us away from using adult therapies on children, a strategy that simply has not worked. In 2009, NBTS the largest non-profit funder of brain tumor research in the country recognized the scientific opportunity to make a difference for patients and families, and committed one-third of its research funding to pediatrics. Since then, NBTS has built a track-record of success, playing a critical role in funding many of the field s crucial findings that have brought us to the threshold The structure of Defeat Pediatric Brain Tumors is what makes it unique. The research program is based on NBTS Defeat program model, which is a new business approach to science that facilitates collaborations and exchanges of real-time data to accelerate progress in R&D efforts and transform the way brain tumor research is funded and conducted. This approach is completely unique in its relationship to traditional funding of research by nonprofits, which typically rely on a standard RFA grant-making process, where checks are distributed to individual labs that apply for grant funding, but the research isn t managed and collaboration isn t fostered. The Defeat Pediatric Brain Tumors approach is beneficial because it: Was not built in a vacuum: the effort is based on input from all key stakeholders researchers, industry, government, and nonprofits/advocates to determine the real barriers to getting to more new treatments, Is the only effort in this field that is ambitious enough to tackle the challenges via an end-to-end approach, from basic science to the clinic, including powerful international collaborations and integrating with leading clinical trial consortium, the Pacific Pediatric Neuro-Oncology Consortium (PNOC); De-risks early entry of biopharmaceutical companies into pediatric brain tumor trials by producing industry-level data, requisite to beginning new clinical trials in humans; Provides 100% of the grant funding to the research: NBTS does not pay for hospital s indirect costs; Doubles the investment dollar: Collaborative agreements signed with partner research centers aim to lock in an agreement that for every dollar of grant investment the institution will match that dollar in cash or in-kind; Takes a unique approach to segments like Intellectual Property (IP) to allow breakthroughs to continue to fund the model and expanded research. 55 Chapel Street, Suite 200 Newton, MA Phone: (617)

LEADERSHIP THE DEFEAT PEDIATRIC BRAIN TUMORS PROGRAM EMPLOYS A TEAM OF RENOWNED RESEARCHERS AND DOCTORS WHICH MAKE UP OUR STRATEGIC SCIENTIFIC ADVISORY COUNCIL,

THIS COMMITTEE WILL BE OPEN FOR MEMBERSHIP TO VOLUNTEERS AND LEADERS IN THE BRAIN TUMOR COMMUNITY. SCIENTIFIC DIRECTOR Dr.

both the Gilbert Neurofibromatosis Institute and the Brain Tumor Institute of Children s National Health System; Professor of Neurology and Pediatrics at George

STRATEGIC SCIENTIFIC ADVISORY COUNCIL Susan Blaney, MD Deputy Director of the Texas Children s Cancer and Hematology Centers; Executive Vice Chair, Department of

Richard Gilbertson, MD, PhD Li Ka Shing Chair of Oncology and Director of the Cambridge Cancer Centre, England.

5 LEADERSHIP THE DEFEAT PEDIATRIC BRAIN TUMORS PROGRAM EMPLOYS A TEAM OF RENOWNED RESEARCHERS AND DOCTORS WHICH MAKE UP OUR STRATEGIC SCIENTIFIC ADVISORY COUNCIL, AND IS MANAGED BY A STEERING COMMITTEE CURRENTLY CONSISTING OF NBTS BOARD MEMBERS. THIS COMMITTEE WILL BE OPEN FOR MEMBERSHIP TO VOLUNTEERS AND LEADERS IN THE BRAIN TUMOR COMMUNITY. SCIENTIFIC DIRECTOR Dr. Roger Packer, MD Senior Vice-President, Center for Neuroscience and Behavioral Medicine, Gilbert Distinguished Professor of Neurofibromatosis, and is Director of both the Gilbert Neurofibromatosis Institute and the Brain Tumor Institute of Children s National Health System; Professor of Neurology and Pediatrics at George Washington University and Clinical Professor of Neurosurgery at the University of Virginia in Charlottesville. STRATEGIC SCIENTIFIC ADVISORY COUNCIL Susan Blaney, MD Deputy Director of the Texas Children s Cancer and Hematology Centers; Executive Vice Chair, Department of Pediatrics, Baylor College of Medicine (BCM); Vice President, Clinical and Translational Research, BCM. Richard Gilbertson, MD, PhD Li Ka Shing Chair of Oncology and Director of the Cambridge Cancer Centre, England. (A Cancer Research UK Major Centre and OECI Comprehensive Cancer Centre). Scott Pomeroy, MD, PhD Neurologist-in-Chief and Chairman, Dept. of Neurology, Boston Children s Hospital; Consultant, Pediatric Neuro- Oncology, Dana-Farber Cancer Institute; Bronson Crothers Professor of Neurology, Harvard Medical School. Raphaël Rousseau, MD, PhD Group Medical Director/Global Franchise Head, Pediatrics; Genentech, a member of the Roche Group.

STEERING COMMITTEE CO-CHAIRS Michael Nathanson, JD, LLM Michael Nathanson is the Chair of the National Brain Tumor Society Board of Directors.

He has been on the National Brain Tumor Society s Board of Directors since 2011. He has been named eight times by Barron s as one of the top 100 independent financial advisors in the nation. Mr.

He has been a practicing dentist in Bethesda, MD for over 25 years. Dr. Schlobohm became involved with the National Brain Tumor Society after losing his daughter to a brain tumor.

6 STEERING COMMITTEE CO-CHAIRS Michael Nathanson, JD, LLM Michael Nathanson is the Chair of the National Brain Tumor Society Board of Directors. He is also the Chief Executive Officer, President, and Chairman of The Colony Group, a leading independent wealth management company. He has been on the National Brain Tumor Society s Board of Directors since He has been named eight times by Barron s as one of the top 100 independent financial advisors in the nation. Mr. Nathanson has been living with a brain tumor since Cord Schlobohm, DMD Cord Schlobohm, DMD is the Vice Chair of the National Brain Tumor Society Board of Directors. He has been a practicing dentist in Bethesda, MD for over 25 years. Dr. Schlobohm became involved with the National Brain Tumor Society after losing his daughter to a brain tumor. He recently served as a patient advocate on the peer-reviewed Cancer Research Program of the Department of Defense, and has spoken on NPR on behalf of the National Brain Tumor Society. ABOUT THE STEERING COMMITTEE The Steering Committee has two co-chairs, and is organized into a series of solicitation committees, on which members of the Steering Committee will elect to serve. These committees are: Leadership Gifts Major Gifts Community Gifts LEARN MORE For more information about joining the Steering Committee, please contact Samantha Masterson at smasterson@braintumor.org or (617) , or visit

INVESTMENT BE PART OF THE IMPACT! Project Impact: A Campaign to Defeat Pediatric Brain Tumors provides multiple ways to get involved and invest in this revolutionary program.

7 INVESTMENT BE PART OF THE IMPACT! Project Impact: A Campaign to Defeat Pediatric Brain Tumors provides multiple ways to get involved and invest in this revolutionary program. Please consider donating today! RECOGNITION OPPORTUNITIES Special naming and commemoration opportunities will be integral to the Project Impact campaign, acting as critical tools in the successful solicitation of leadership and major gifts by providing incentives to donors to invest, while also providing high-level and public recognition opportunities in exchange for those investments. AMOUNT OPPORTUNITY $750,000 Endowment to permanently name a research core (four total) $500,000 Endowment to permanently name LLC s Scientific Director position $250,000 Sponsor a research grant (two-year-named term; eight total) $100,000 Sponsor a research core (two-year-named term; four total) $100,000 Sponsor a named Community Gifts matching gift fund $75,000 Sponsor LLC s Scientific Director position (one-year-named term) $50,000 Sponsor the sequencing of 50 genomes $25,000 Sponsor the sequencing of 25 genomes $10,000 Sponsor the sequencing of 10 genomes

GET INVOLVED Volunteer to advocate for policies that promote pediatric cancer and brain tumor research.

8 COMMUNITY & PERSONAL DONATION COMMITMENTS COMMUNITY EVENTS Host a community event to raise money for pediatric research. HOST INFORMATION SESSIONS Attend or host an informational session to raise awareness of the need for pediatric brain tumor research. GET INVOLVED Volunteer to advocate for policies that promote pediatric cancer and brain tumor research. PERSONAL AND LEGACY GIVING Invest in the campaign through a personal commitment, or consider legacy giving as part of your last will and testament. EMPLOYER MATCHING Find out if your employer has a matching gift program and direct that funding to pediatric research. LEARN MORE For more information, contact Samantha Masterson at smasterson@braintumor.org or (617) , or visit IT S AN EXCITING TIME IN INCREASING OUR UNDERSTANDING OF THIS DISEASE. WE NEED TO KEEP THE MOMENTUM UP - FROM FINDING MUTATIONS, TO UNDERSTANDING HOW THEY WORK AND HOW THEY IMPACT CLINICAL DECISIONS. THIS IS AN EXCITING TIME TO ADVANCE THIS PROJECT. - DR. SUZANNE BAKER, PHD ST. JUDE CHILDREN S RESEARCH HOSPITAL WE HAVEN T MOVED THE NEEDLE IN DECADES, AND THAT IS UNACCEPTABLE FOR KIDS AND FAMILIES. WHEN MY PATIENTS GET THE DIAGNOSIS, I MEET WITH THE FAMILY FOR THE FIRST TIME, AND WE RE ALREADY TALKING ABOUT END OF LIFE. THAT IS UNACCEPTABLE. WE NEED THE COURAGE TO MOVE TO PRECISION MEDICINE AND TAKE RISKS TO SAVE THESE KIDS. - DR. SABINE MUELLER, MD UNIVERSITY OF CALIFORNIA, SAN FRANCISCO

9 MAKING THE CASE USHERING IN A NEW ERA OF PROMISE FOR PEDIATRIC BRAIN TUMOR PATIENTS WHY THIS CAUSE? Over the past decades, little progress has been made in the treatment of pediatric brain tumors, while tremendous advances have been made across the rest of the pediatric cancer spectrum. For example, the most common form of childhood cancer, acute lymphoblastic leukemia, was generally considered fatal 50 years ago; today, cure rates for the disease reach close to 90%. For the more than 4,600 children diagnosed annually with a brain tumor, the scientific needle has barely moved. Five-year survival rates hover between 15-30%, yielding survival rates from diagnosis that span only the length of an average school year. Most treatments administered to children with brain tumors were not designed for the developing brain, often leaving survivors with a lifetime of neurological, cognitive, developmental, and other challenges. Lack of preclinical models for effectively testing drugs and small patient population size limit investment by pharmaceutical companies. Global regulatory barriers and a young knowledge base of these tumors inhibit the development of pediatric-specific treatments. WHY NOW? The confluence of ground breaking advances in our understanding of pediatric high-grade gliomas and the power of precision medicine leads us to a higher place from which we can launch a concerted fight against pediatric brain tumors. The time to act is now; the opportunity to succeed in improving the lives of children and their families is tremendous. Never before in the history of mankind s battle with cancer has so much research progress been made. Advances in molecular biology and genetic sequencing have unlocked new knowledge that is informing the development of new treatments and therapies. Through precision medicine, we are working to provide the right therapy for the right person at the right time. Critical findings in recent years including many funded by the National Brain Tumor Society have positioned the field of pediatric brain tumor research to enter a new era in treatment and survival. These findings allow scientists to break from the norm of the past decades, and catalyze them to develop pediatric-specific treatments that finally move us away from using adult therapies on children, a strategy that simply has not worked.

for pediatric brain tumor research and clinical trials. Project Impact, as a capital campaign, seeks to initially raise at least $2.

invest more than $5 million, from multiple sources, in the program.

10 WHY PROJECT IMPACT? Project Impact: A Campaign to Defeat Pediatric Brain Tumors is the National Brain Tumor Society s push to fund a major translational research and drug discovery program and create sustainable support for pediatric brain tumor research and clinical trials. Project Impact, as a capital campaign, seeks to initially raise at least $2.5 million in specialty gifts and funds over five years to resource the launch of scientific projects within the Defeat Pediatric Brain Tumors Research Collaborative, with an ongoing aim to ultimately invest more than $5 million, from multiple sources, in the program. Through this paradigm-shifting strategy, NBTS will fundamentally and permanently change the trajectory of pediatric high-grade gliomas by developing: A new standard of care that deploys effective drugs to dramatically extend the survival of children with high-grade gliomas A research platform that creates a system of tested and validated compounds to feed Phase I clinical trials A new methodology of conducting research differently breaking the old mold of single-investigator-driven projects and moving to highly collaborative teamscience where sharing data in real time is fueling faster research results New and trusted preclinical models for testing that incentivize the pharmaceutical industry to provide compounds and agents to researchers A collaborative research model that is scalable across the pediatric cancer spectrum that discovers new knowledge that can be applied in the fight against all pediatric brain tumors

11 LAUNCH Developmental Neurobiology Initiative to better understand factors that contribute to tumor origin COLLABORATION Lays groundwork for next investment in pediatric research by establishing the collaborative framework for the Defeat Pediatric Brain Tumor Research Collaborative LAUNCH Launching a $2.5 million capital campaign to fund research to permanently change the trajectory of this disease FUNDING St. Baldrick's Foundation signs on as founding partner LAUNCH RESEARCH Molecular profiling Discovered pediatric initiative to identify tumors unique and study the genetic and different from alterations that occur adult tumors within pediatric brain tumors RESEARCH Major analysis of the landscape to determine remaining barriers to progress and opportunities for research COLLABORATION Convenes the major stakeholders in pediatric brain tumors to fully understand what is holding back progress in research RESEARCH Announces members of the Strategic Scientific Advisory Council, comprised of leaders in the field who help guide the scientific efforts of our initiative COLLABORATION In partnership with the V Foundation for Cancer Research, invests in a new clinical trial for pediatric highgrade gliomas National Brain Tumor Society is fiercely committed to finding better treatments, and ultimately a cure, for people living with a brain tumor today and anyone who will be diagnosed tomorrow. This means effecting change in the system at all levels.

Chief Development Officer National Brain Tumor Society

Send Nominations or Cover Letter and Resume to: Lisa Vuona Vice President 617-262-1102 lvuona@lllsearches.com Chief Development Officer National Brain Tumor Society Newton, MA http://braintumor.org/ The