Children’s Brain Tumor Project Lab Update: Spring 2015

Posted in Research, Updates

By Dr. Mark Souweidane and Dr. Jeffrey GreenfieldCo-Directors, Children’s Brain Tumor Project This lab update is more like a chorus of “On the Road Again,” as we have both been traveling so much this spring. As reluctant as we always are to leave our work here, that travel has been invaluable in the sharing of information and the enhancing of our worldwide collaborations. The DIPG Workshop in Barcelona in February brought together experts from all across the United States and Europe, from cities including Amsterdam, Helsinki, and London—not to mention institutions such as the NIH, Duke, Dana Farber, Northwestern, and Weill Cornell. The workshop is made possible through the efforts of the Alicia Pueyo Fund, a family foundation dedicated to finding treatments for brainstem gliomas and encouraging collaboration among researchers worldwide. We are honored to participate alongside other international leaders who are striving to defeat DIPG, and grateful to the dedicated families who are at the forefront of the effort, always refusing to take no for an answer. March meant Paris, for the first-ever International Gliomatosis Cerebri Conference, organized and driven by families affected by GC. That groundbreaking meeting created a framework for important collaborations as we go forward. (Click here to read more about both conferences.) In April, we look forward to the annual meeting of the DIPG Collaborative in Chicago. The 2015 Symposium promises to be filled with new research and development about this tumor, which is one of the prime targets of our research efforts here at the...

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News From Barcelona and Paris

Posted in Research, Updates

In February, Dr. Souweidane attended the Alicia Pueyo Workshop on DIPG in Barcelona, where he presented an update on the use of convection-enhanced delivery (CED) for DIPG to an international audience of scientists, clinicians, and family advocates. As Dr. Souweidane’s clinical trial approaches completion, other groups are highly interested in learning more about CED, as evidenced by the invitations he has received to present this innovative strategy: as a keynote speaker at the University of Alabama Birmingham/Children’s Hospital of Alabama for its annual Pediatric Neuro-oncology Symposium on May 1, at the DIPG Collaborative Symposium in April in Chicago, and as the 2015 E. Bruce Hendrick Visiting Professor at the University of Toronto/Hospital for Sick Children on May 22. In March, an unprecedented constellation of scientists and oncologists met in Paris to discuss the status of diagnosis, treatment, and scientific inquiry into gliomatosis cerebri (GC), a disease so close to many of us at the CBTP. Dr. Greenfield cohosted the two-day gathering, which took place at the Institut Curie. Developments included an agreement that Weill Cornell will host the International GC Registry with back-end analytic support from the DIPG community. The ICB in London and Weill Cornell in New York were also selected as the European and North American sites for sample collection and comprehensive genomic analyses. The panel discussed new scientific projects to focus on and the concept of a GC-specific clinical trial within two years. This cross-continental initiative is truly a remarkable example of the efforts that can result from collaboration and...

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Family Update: Spring 2015

Posted in From the CBTP Families, Updates

By Maria and Geoff Gratton As all couples have, we’ve experienced those defining moments in our life together, including our wedding day and the births of our children. While those continue to resonate with us, we now have another defining moment—one that reset our concept of “normal.” That defining moment came on December 1, 2012, at Connecticut Children’s Medical Center, when our son was diagnosed with an inoperable brain tumor. In less than five minutes our lives were forever changed, and we found ourselves desperately seeking answers in a world that we had never even known existed. In our small town we learned of another family who had earlier faced a pediatric brain tumor diagnosis. That family encouraged us to reach out to Weill Cornell and the wonderful doctors who specialize in these tumors. It was in early 2013 at Weill Cornell that we learned the name of what had originally been diagnosed as a low-grade tumor: gliomatosis cerebri (GC). In this dark and lonely moment, our random Internet search led us to a comforting web site, Elizabeth’s Hope. We quickly learned the frightening statistics, including the gross underfunding of federal monies for research and the lack of awareness about pediatric brain tumors. We needed to do something, anything, because doing nothing was not an option. That was the catalyst for the establishment of our organization, which we named Our Cure 4 Life. We held our first board meeting on January 25, 2015, with a mission to fund research, raise awareness, and provide support for those afflicted with rare, inoperable brain tumors. We quickly launched a web site (www.ourcure4life.org), created a Facebook page, and are now executing our mission. To that end, we have two major events planned in 2015: our first annual “Giving on the Green” golf tournament (scheduled for May 29 at The Golf Club at Windham) and the Our Cure 4 Life Gala in September 2015, which aligns with Pediatric Brain Cancer Awareness month. Today we live by a new set of rules about living in the present: The present moment is the only moment available to us, and it is the door to all moments. – Thich Nhat Hanh Together with our newfound family within the Children’s Brain Tumor Project, we will make great strides in our combined missions. We remain hopeful and encourage you to help us spread the word, provide support, and raise the funds necessary to find a cure—Our Cure 4...

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Family Update: Fall 2014

Posted in From the CBTP Families, Updates

By David Bernstein I read with great interest Cindy Campbell’s story in the previous issue of this newsletter about the devastating heartbreak her family endured during the loss of their beautiful 5-year-old son Ty. One can’t fathom why these deadly diseases take our children from us at such a young age, nor can anyone fully understand the toll it takes on the family and community throughout the entire journey of care. My family lost our beloved Zachary on March 12, 2014. He was diagnosed with a diffuse intrinsic pontine glioma (DIPG). The disease took our beautiful boy in nine months. He was 11 years old. As parents, we do everything we can to try to insulate and protect our beautiful children, and nurture them into the adolescents and adults we dream of. Unfortunately, for some of us there are those unforgiving moments in which we join the elite “club” (not by choice) of those who truly understand the emotional roller coaster, and who know what we would give up for “One More Day.” September was Childhood Cancer Awareness month, a time to honor and remember children and families affected by these rare diseases, and to help give kids with cancer better outcomes by supporting the foundations, charities, nonprofits, hospitals, and other organizations that share the mission of raising funds for research. After Zachary passed away, I had heart-to-heart conversations with our medical team. I asked them, “how far off are we from finding a cure for children with DIPG and other pediatric brain cancers?” To my surprise they all claimed we are four to six years away. My immediate question was “Why?” They all responded… “Lack of funding.” I was stunned to learn that as much progress as appears to have been made…we are still so far away from a cure. Consider this: 96 percent of federal funding for research is for adult cancers, leaving only 4 percent for childhood cancers. Yet kids make up 20 percent of our population. Funding from large cancer organizations doesn’t help very much; less than 1 percent of the American Cancer Society’s total donations are directed toward childhood cancer research Pharmaceutical companies fund 60 percent of all adult cancer research, but they do virtually no childhood cancer research because it’s not profitable. About 900 adult cancer drugs are in the drug development pipeline, but almost none for children’s cancers. After Zachary’s passing, my family was highly motivated to launch the Fly a Kite Foundation. The Fly a Kite Foundation is committed to children and families first. They are the primary reason for our existence. Our objectives are threefold: Creating art packages to provide a creative and therapeutic outlet for young brain tumor patients...

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Children’s Brain Tumor Project Lab Update: Fall 2014

Posted in About the CBTP, Research, Updates

By Sheng Li, Ph.D. Ty Louis Campbell Fellow I am so grateful for the opportunity to work with the Children’s Brain Tumor Project as the first Ty Louis Campbell Fellow. I thank Cindy and Lou Campbell and the TLC Foundation for funding this creative position. My passion is bioinformatics and computational biology, both of which play critical roles in decoding tumors such as DIPG, gliomatosis cerebri, and AT/RT—the cancer that claimed Ty’s life two years ago. After receiving a bachelor’s degree in biotechnology from Sun Yat-sen University in Guangzhou, China, I came to Weill Cornell in 2009. I’ve been here for the past five years as a Ph.D. candidate in computational biology, during which time I have published research articles on high-throughput sequencing data analysis for cancer biology in Nature Biotechnology, Nature Genetics, Journal of Clinical Investigation, and Genome Biology. I’ve also been a lecturer in cancer genome data analysis for courses at Weill Cornell Medical College and the Institute of Computational Biomedicine’s EpiWorkshop. I completed my doctoral dissertation on the topic of cancer epigenetic dysregulation, and I’ll be further pursuing that research in this fellowship. The science of epigenetics is relatively new, and it’s emerging rapidly. The term refers to changes in the ways genes are expressed that don’t change the DNA itself. Disruptions in the epigenome are thought to play a fundamental role in how cancer develops, but how those disruptions happen are only just now starting to be understood. Because they occur spontaneously, they cannot be predicted—which is why the rare and inoperable tumors that strike children seem to come from out of the blue. There are no known risk factors, no family history, no warning signs—just a child who is healthy one day and terminally ill the next. For answers, we must comb through a staggering amount of data that can now be produced through sequencing and other studies. Today’s high-throughput sequencing methods are faster and more accurate than they were even just a few years ago, but each tumor sequenced produces an avalanche of data that must be pored over and analyzed by a bioinformatics specialist in order to find answers and draw conclusions—or at least identify new pathways for future studies. I am proud to be playing a role in the Children’s Brain Tumor Project’s multi-pronged research efforts—banking tissue for future study, sequencing tumors from a wide range of patients, interpreting the data produced from sequencing those samples, and then testing new drugs and new delivery methods selected specifically for that individual tumor. The Department of Neurological Surgery is working with the Department of Pathology, the Cancer Center, and the Center for Precision Medicine here at Weill Cornell as well as collaborating with...

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Family Update: Summer 2014

Posted in From the CBTP Families, Updates

By Cindy Campbell My husband and I were introduced to the world of pediatric brain tumors when our perfect, beautiful little boy, Ty, was diagnosed with a rhabdoid tumor on the brainstem at 2 years and 10 months old. He died 13 days after his fifth birthday in October 2012 and we are left to pick up the pieces of our broken lives. Throughout Ty’s journey, and ever since his loss, Lou and I have been actively involved in getting to know other families like ours, and getting to know the doctors who are committed to helping these children through much needed, grossly underfunded research and clinical trials. We are in constant awe of the men and women who have devoted their lives to pediatric neuro-oncology and pediatric neurosurgery. Doctors like Jeff Greenfield and Mark Souweidane. Of course, there are thousands of scholars, researchers, and doctors who selflessly devote their time to helping others. Searching for a cure. But let me tell you why a career in pediatric neuro-oncology is so very remarkable to us both. First and most obviously, because we are talking about children. Little babies who don’t understand why magic kisses can’t make their boo-boos go away. Many of these veterans (including Jeff and Mark) have small children of their own, and instead of being turned off by how difficult it is to imagine their own children as patients, they are inspired to make sure these children are getting the best possible care and hope for a future. It takes a very special heart to be so devoted to a cause that may frighten others. Then there is the reality of pediatric brain tumors. When a brain or spinal tumor is involved, the detrimental effects are that much more severe. The sad reality of pediatric brain tumors is that they steal so much from the child. The ability to speak, eat, walk, play, and more can all be seen slowly slipping away as tumors progress. Brain and spinal cord tumors are the second most common of all cancers among children, and brain tumors include the deadliest of all types of childhood cancers (with survival rates of 0% in many cases). When a child is diagnosed with a brain tumor, the deterioration is like nothing you could imagine or prepare for. My son, for example, was 100% healthy before the dreaded diagnosis. He met all of his developmental milestones and at 2½ years old was enjoying the use of his improving vocabulary. His sense of humor was evolving and knock-knock jokes were becoming a new favorite (although I don’t think he truly understood the punchlines—at least not as much as his larger-than-life laughter would express). He loved...

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