Understanding the Journey: A Parent’s Guide to DIPG Edited by Ruth I. Hoffman, MPH Copyright © 2012 by American Childhood Cancer Organization® (ACCO) Printed in the United States of America Published by American Childhood Cancer Organization® Cover and Interior Design: Ruth I. Hoffman MPH Dedicated to all of the children who have battled or will battle Interior Layout: Ruth I. Hoffman, MPH, Devon Harp DIPG, and to Andrew Smith—for the impact you left on my life. Printing History: May 2012 ISBN 978-0-9854593-0-7 “It is only in the darkness, we can see the brightest stars.” Library of Congress Control Number: 2012908135 Martin Luther King Jr. The American Childhood Cancer Organization’s® name and logo are registered trademarks of the national office of the American Childhood Cancer Organization®. Visit us on the web at: http://www.acco.org. This book is written to provide information about childhood cancer and should not be used as an alternative to receiving professional advice. Every effort has been made to ensure that the information in this book is accurate at the time of printing, however, there is no guarantee that the information will remain current over time. Always seek the advice of a trained professional. ® ACCO.ORG With the support of Table of Contents Preface vii Part I. Understanding the Diagnosis 1. Brain Tumors 101 1 Violette Renard Recinos, MD, George I. Jallo, MD 2. Typical History of DIPG 17 Eric H. Raabe, MD, PhD, Kenneth Cohen, MD, MBA 3. Pontine Anatomy and Function 27 Sven Hochheimer, MD, Javad Nazarian, PhD, Suresh N. Magge, MD 4. Imaging DIPG 43 Jonathan Finlay, MD, Girish Dhall, MD, John Grimm, MD, Stefan Bluml, PhD Part II. Treatment 5. Clinical Trials for DIPG 71 Adam Cohen, MS, MD, Howard Colman, MD, PhD 6. Surgery: What it Can and Cannot Offer DIPG 89 Michael H. Handler, MD 7. Radiation Therapy 101 Arthur Liu, MD, PhD 8. Radiosensitizers for DIPG 111 Roger J. Packer, MD 9. Chemotherapy and Biologics 123 David N. Korones, MD 10. The Use of Steroids in Patients with DIPG 139 Eric Bouffet, MD, Ute Bartels, MD Part III. Other Care Issues 11. Caring For Your Child At Home 153 Deborah Lafond, DNP, PNP-BC, CPON, CHPPN 12. Communication: When a Child Can No Longer Speak 193 Brownstone, MSW, RSW, Caelyn Kaise, MHSc, SLP (C), Reg. CASLPO, Ceilidh Eaton Russell, CCLS, MSc (candidate) Part IV. Research 13. Overcoming Research Hurdles in DIPG 223 Patricia Baxter, MD, Susan Blaney, MD 14. The Future of Genomics and Proteomics in DIPG 231 Mark W. Kieran, MD, PhD 15. Animal Models for DIPGs 239 Oren J. Becher, MD 16. Neural Stem Cells and DIPG 245 Michelle Monje, MD, PhD 17. Convection-Enhanced Delivery in DIPG 249 Zhiping Zhou, MD, PhD, Mark M. Souweidane, MD 18. Vaccine Treatment Strategies 269 Christopher Moertel, MD Part V. End of Life Decisions 19. DIPG and Tissue Donation 273 Cynthia Hawkins, MD, PhD, Eric Bouffet, MD, Ute Bartels, MD 20. Organ and Tissue Donation 285 Angela Punnett, MD, FRCPC 21. Integrating Palliative Care and Difficult Decisions 293 Justin N. Baker, MD, FAAP, FAAHPM, Adam J. Tyson, MD, Javier R. Kane, MD 22. Journey of Sadness and Hopes: A Letter to Parents 319 Tammy I. Kang, MD, MSCE, Chris Feudtner, MD, PhD, MPH Part VI. Appendices A. Sample Medications Form 333 B. Glossary of Terms 335 C. Resources 343 D. Research Articles 355 Preface The American Childhood Cancer Organization’s (ACCO) mission is to provide information and support for children and adolescents with cancer and their families; to provide grassroots leadership through advocacy and awareness; and to support research leading to a cure for all children diagnosed with this life-threatening disease. Since ACCO's founding in 1970, clinical research has increased the five-year survival rate of childhood cancer in the U.S. to approximately 80 percent. This improvement in survival brings hope to tens of thousands of families whose children are treated for cancer each year. In spite of the progress, however, too many families still endure the loss of their precious son or daughter to cancer. In the U.S. cancer continues to be the primary cause of death by disease in childhood. As a result, families whose children are currently fighting this disease need access to information to help them with the many treatment decisions they must make; and there is an acute need for an increase in pediatric oncology research funding that will lead to the development of new treatments for children diagnosed with cancer in the future. Among the many devastating childhood cancers, children who are diagnosed with diffuse intrinsic pontine glioma (DIPG) desperately need access to new treatments. Regarded as the most aggressive of all pediatric brain tumors, all of these children face a dismal prognosis. Currently, radiation therapy offers a short reprieve from a ravaging disease. The tragedy of this disease is best expressed in the following words from one parent's writing about her precious daughter. "Imagine that you had a cherubic, mischievous, energetic and moody two year old with flashing blue eyes, a brilliant smile and curly red hair. Imagine that each morning she got you up at 5:15 a.m. by standing up in her crib and shouting, "Maaamaaa, I'm awaaaake! Maaamaaa, where are you?" Imagine if when you went into her room she threw both her arms up towards you in a great big hug and chattered her way into the living room, telling you she wanted Cheerios for breakfast…with banana…and milk…and can we paint now…and watch Caillou. Imagine if when you tried to get her dressed Preface VII in the morning, she ran away from you laughing, no matter how big—painting with her mouth and visiting the pigs at the farm. And exasperated you got. Imagine if she insisted on picking out her own sometimes it's just being able to mouth the words "ice cream," and clothes, and you let her, rather than fight about it. Imagine if she then napping most of the day. But she is always true to herself, and could sing the entire theme song to "Golden Girls," could go down even though things are hard for her, she ignores the barriers of DIPG the slide on her own, could pee on the potty, catch a ball, dance and and chooses to forge her own path. Most importantly, she believes chase her friends. Imagine when you step off the subway after work that when life gives you a hundred reasons to cry, you need to find a and walk into her daycare room, all the kids turn to look at who has thousand reasons to smile…And in my own smiles, I have become entered the room, and when she sees you she flashes the most brilliant familiar with the bittersweet taste of getting to parent my precious smile and comes running with her arms up, saying "Mama! Mama! daughter—the best experience in the world, but like a spring day Mama!" Imagine if no matter how many times she had a tantrum that is much, much, too short." and demanded things from you and exhausted you, she ended each night with a snuggle and a kiss and you breathed in the smell of her As this parent so eloquently states, having a child diagnosed with DIPG is the curls and felt warm happiness all over. Imagine if you could never most difficult journey that any parent will ever endure. This book was written to love anything as much as you loved your first born child, your dream help parents understand that journey so that they are better equipped to make come true, your daughter. decisions regarding their child’s diagnosis, treatment, entry into clinical trials, palliative care and quality of life during this critical time. It was also written to Now imagine it is 9 months later. Imagine she is lying next to you in provide hope for a future when children diagnosed with DIPG will be cured your bed. She can't walk. She can't use her arms or hands. She can't of their disease and able to live long and healthy lives. hold her head up. She can't see the television. She can't tell you she loves you. She can't hug you. She is lying in the bed sound asleep, but coughing on her own saliva, which she is starting to choke on The Contributors because she can barely swallow. Imagine she was dying and there was In November 2009, I was introduced to Andrew Smith—a magical young boy nothing you could do to change it. Imagine if you knew that one who was battling DIPG. He was hospitalized at the National Institutes of Health day soon you would never get to see her again. Never see her smile, (NIH) in Bethesda, Maryland, where he and his family were making a decision feel her hand slip into yours, kiss her warm cheek, feel her sigh into regarding his participation in a clinical trial. It was Thanksgiving, and through your chest. Andrew's determination to communicate and the communication skills of his That is the simple reality of what we are living with. And it's hard. No parents with the "yes/no" questioning technique, we learned that the hospital matter how many good things happen to us, no matter how much menu did not include pumpkin pie—a "must have" item during the holiday we believe in a bright future for ourselves and a time of healing, we season. We also learned that Andrew (who was a "foodie") would love not just are being tortured.