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NF NEWS V ol u m e I I , 2013

T h e N e w s l e t t e r o f t h e C h i l d r e n ’ s Tu m o r F o u n d a t i o n

Join the NF Registry And Help End NF In 2012, the Children’s Tumor Foundation created an online NF Registry. The purpose of this registry is to find people who may be eligible for clinical trials or other research studies being conducted in the field of neurofibromatosis (NF), and to determine the commonality of specific NF characteristics. This past April, the NF Registry proved useful in finding patients for a specific study. David Stevenson, MD, of the University of Utah, is enrolling patients who have had tibial bowing, but never a tibial fracture. Tibial bowing is a rare, but serious, complication of NF1 in which improper growth of the tibial bone of the lower leg leads to bowing and a tendency to fracture. In just a few clicks, the NF Registry created a list of 50 patients who fit the study requirements. These patients were then sent an email describing the study and giving contact information for Dr. Stevenson. Of those who received the email, about 35% opened it and a handful

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contacted the researcher. While a 35% response rate is fairly typical of what you can expect, it highlights why we need to increase the number of people in the NF Registry. Please visit for more information, or contact Pamela Knight, Children’s Tumor Foundation Clinical Program Manager, at 212-344-6633, ext. 8555.

2013 NF Forum is largest ever In Nashville, TN on April 19-21, over 300 people attended the 2013 NF Forum which brings together patients and families to learn about the latest medical advancements in NF research, receive practical advice on NF-related issues, and learn about resources provided by the Children’s Tumor Foundation. This year’s Forum was themed, “A Family Comes Together: Celebrating the NF Robert Sebastian entertains at the NF Forum

Community” and it focused on the ways the Foundation is building relationships both in the world of research (amongst clinicians, researchers, and biotech) and within the population of those living with neurofibromatosis. Nashville is known as Music City, and attendees were treated to a unique opening reception on Friday evening as musician Robert Sebastian and his band kicked off the Forum with a rousing performance of songs new and old. Robert is an accomplished artist from Tennessee and has recorded several albums. He lives with NF2 and shared his experiences with the crowd, both through his music and with verbal interludes between songs. His notes were rockin’, his words were inspirational, and it was the perfect tone for the opening of this annual gathering. On Saturday morning, attendees were treated to a passionate opening keynote address by the current CTF Ambassador Drew Leathers. Drew is from Atlanta and was diagnosed with NF at age 11 and schwannomatosis in 2005. In 2009, Drew had surgery to remove a tumor that was obstructing his spinal cord and became a quadriplegic. Due to medical treatments and sheer tenacity, he has since regained function in his arms, and has become a bit of an continued on page 5

Children’s Tumor Foundation

board of directors

Stuart Match Suna Chairperson Linda Martin Vice Chairperson John McCarthy Treasurer Aram Fuchs Secretary Bruce R. Korf, MD, PhD, Chairperson, Medical Advisory Committee Suzanne Earle Chairperson Emeritus Jill Markland Chapter Council President Allan Rubenstein, MD Director of Medical Affairs Emeritus Daniel Altman Laura Ganio Bona William Brooks Colin Bryar John Catsimatidis Tracy Galloway Daniel Gilbert John Golfinos, MD Daniel Graeff Matthew Hay Steve McKenzie Lesley Oslica JoAnne Pastel Laura Perfetti Jason Pontin John W. Risner Robert Schaffer Rachel B. Tiven David Viskochil, MD, PhD Nate Walker Peggy Wallace, PhD Honorary Richard A. Horvitz Michie Stovall O'Day Harold Ramis Alan Robbins, MD Carolyn E. Setlow Ed Stern Pro Bono Counsel

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childrenstmrfdn @childrenstumor @childrenstumor children’s-tumor-foundation

Letter from the president A Note From Children’s Tumor Foundation President, John W. Risner

The first sequencing of the entire human genome was announced more than ten years ago. Yet it is only now that costs have dropped enough in sequencing, computer memory, and data storage that researchers can really begin to unlock the genomic code. Now we need to remove yet another roadblock: how to foster interdisciplinary collaboration and data-sharing, and properly acknowledge and reward team contributions to science. Why does Avastin shrink vestibular schwannomas in some NF2 patients, but not others? What drives Gleevec to shrink plexiform tumors in a limited number of NF1 patients, have no effect on others, and actually harm a small number of cases? Examples similar to these NF scenarios are seen in other genetic diseases as well, where drugs that bring major relief to some patients have no effect, or even harmful side effects, in others. We are at the dawn of the age of personalized or precision medicine. An individual’s genomic data set will help identify which specific complications they are at high risk for, which medicines will work on their particular case – and which will not. This promises tremendous benefits to patients and will make the overall healthcare system more efficient. NF researchers have not yet been able to identify a clear genotype (where the mutation occurs)/phenotype (what is the effect) correlation. Although in some cases increased risks are associated with certain mutations, most complications that occur in patients cannot be matched to mutations that occur on chromosome 17 for NF1, or chromosome 22 for NF2. Understanding this better will benefit care, as clinicians will be able to focus on looking for and treating certain complications. Last year Vertex Pharmaceuticals and the Cystic Fibrosis Foundation made an exciting announcement – FDA approval of Kalydeco, a new drug they developed that is a true breakthrough because it not only improves lung function, but also addresses the underlying cause of the disease. However, they were quick to note that it is effective in only 4% of CF patients - ones that carried a specific genetic mutation. This is still great news, and not just for this 4%, as this new path holds promise for the wider patient population. While still early days, this revolution is beginning now. For NF to be at the front of this advance we need to populate the NF Registry as fast as possible. Interest in rare disorders like NF is growing, but biotech and pharmaceutical companies need to know that the patient base exists, is engaged, and is interested in research. So if you have NF, and have not yet registered, please take ten minutes today to sign up at - not just for the personal benefits, but to advance our shared cause of ending neurofibromatosis. Thank you,

John W. Risner

research news

NF Research: Making Connections In January, the Children’s Tumor Foundation declared 2013 the year of collaboration. Therefore, in the first quarter, time and efforts were focused on connecting and partnering with industry, academic centers, publications, scientists, and other organizations in order to increase NF awareness and further research into neurofibromatosis. The NF Preclinical Consortium Delivers AGAIN:

The Story of AR-42 In early 2007, one of the gaps CTF identified as part of a strategic review was a need for greater preclinical testing of promising compounds. Before a drug can be approved for a clinical trial, extensive testing must be done in cell lines and animal models to first prove safety, and then establish efficacy. The pipeline for NF related compounds was thin, which drove the Foundation to establish the Drug Discovery Initiative (DDI). This new grant mechanism would provide relatively small amounts of funding to researchers to test compounds in NF cell lines and mouse models. One of the first DDI applications received was by Dr. Abraham Jacob, then at Ohio State University (OSU). He had been working with new compounds that he believed showed potential for treating NF related vestibular schwannomas. Dr. Jacob’s application for a $27,500 DDI grant to test OSU-03012 in an NF2 mouse model was approved and funded in 2007. The promising results led to follow-on funding of $40,000 from the Triological Society in early 2008 to test combinations of OSU-03012 and OSU-HDAC42. In late 2008, Ohio State licensed OSU-03012 and OSU-HDAC42 to Arno Therapeutics, a small biotech that saw promise in the compounds for NF2, other solid tumors, and blood cancers. The compounds were renamed AR-12 and AR-42 and work continued through Arno to optimize them. In 2010, Dr. Craig Hofmeister at The Ohio State University Comprehensive Cancer Center began a phase I clinical trial to establish safety in humans with various forms of blood cancer. AR-42 received further testing through the NF Preclinical Consortium (NFPC), which confirmed both the expected safe dosage levels and potential efficacy to treat NF2. Arno announced in early 2012 that AR-42 had been granted Orphan Drug designation by both the Food and Drug Administration (FDA) in the U.S. and the European Medicines Agency (EMA) in Europe. In December of 2012, Ohio State received a Clinical Trial Award from the Congressionally Directed Medical Research Program-NF Research Program for a Phase 0 proof of concept study trial of AR-42 in NF2. While it is still a long way from Phase 0 to FDA approval, the story of AR-42 proves that the DDI and NFPC are crucial programs in building the pipeline of drugs for treating NF, and demonstrates the importance of collaborations between academic labs, non-profits, and industry.

Adrienne Watson

CTF Investment Leads to Third Paper this Year Published in “Nature” The Children’s Tumor Foundation is pleased to announce that Adrienne Watson, a 2011 Young Investigator Awardee from the University of Minnesota, is the Second Author on a paper recently published in the journal “Nature Genetics” regarding malignant peripheral nerve sheath tumors (MPNST). This is the third time this year that a paper with funding from the Children’s Tumor Foundation has been published in “Nature.”

The Children’s Tumor Foundation Research Department Continues to Grow The Children’s Tumor Foundation is pleased to announce the appointment of Salvatore “Salvo” La Rosa as Director of Research and Development. Salvatore has a PhD in Medicinal Chemistry from the University of Strathclyde in Scotland (UK). Prior to joining the Children’s Tumor Foundation, Salvatore worked for various companies and biotechs across Europe. He Salvatore La Rosa has extensive experience as a project and group leader in rare disease drug discovery. Salvo is committed to applying this expertise into translating top research into clinical trials and thereby accelerating the path to new therapies for NF. 3

Dr. Margaret Anderson

research news CTF Connects to FasterCures FasterCures is a healthcare based nonprofit organization, within the Milken institute, led by Executive Director Dr. Margaret Anderson. Their goal is to clear the path to faster disease resolution by educating stakeholders about research barriers, amplifying solutions that could pave the way to progress, and creating opportunities for key leaders to come together and accomplish objectives. “This organization plays an instrumental ‘umbrella’ role in connecting foundations so that we can learn from each other’s business models and become stronger and faster in fighting disease,” says Annette Bakker, PhD, Chief Scientific Officer of the Children’s Tumor Foundation. CTF was recently highlighted by FasterCures’ “TRAIN Innovator Spotlight.” TRAIN, The Research Acceleration and Innovation Network, creates opportunities for medical research innovators to discuss the challenges that cut across diseases and promote their best practices to the rest of the community. On March 21, John Risner, President of the Children’s Tumor Foundation, and Annette Bakker interviewed Dr. Margaret Anderson. During the interview, Dr. Anderson mentioned that, “The successful disease foundations, even if they cover very different diseases have a lot in common; they keep their eye on what the patient needs and they empower their patients. They are not focused on just doing interesting research … they want to fix the problem.” The

lack of open data sharing among foundations and research institutions often stands in the way of this problem-solving. However, in terms of NF, Dr. Bakker is convinced that the Children’s Tumor Foundation is perfectly positioned to be the neutral facilitator amongst science and industry, driving the research that will develop efficacious therapies for neurofibromatosis. Dr. Anderson also shared that a “game changer” in recent years has been the patient revolution. “Patients are part of the process; they are not innocent bystanders anymore,” she said. Offering a registry is integral to fostering patient engagement. “It takes research from THEY to US,” Dr. Anderson stated. Registries allow the consolidation of data and open partnering opportunities with both industry and academia. The Children’s Tumor Foundation launched the NF Registry in 2012 and encourages participation amongst the neurofibromatosis community. To view the video of Dr. Anderson’s interview with Mr. Risner and Dr. Bakker, please visit .

CTF Partners with Thomson Reuters In 2012, CTF partnered with Thomson Reuters on two initiatives, disease maps and a drug repurposing project. The disease maps (see ) were developed to help donors visualize where funds raised for NF research are spent. The drug repurposing project was based on the observation that many pathways that are de-regulated in NF are often also de-regulated in other diseases such as cancer. These endeavors help the Foundation market NF to industry so that partnerships can be established. Thomson Reuters promoted the partnership in a joint press release with the Children’s Tumor Foundation, along with a detailed case study and a video recorded at the CTF offices in New York. Please visit for more information.


Online Grant Management System in Place at the Children’s Tumor Foundation In March 2013, the application and review process for the latest round of Young Investigator Awards (YIA) was moved entirely online for the first time. It is a great improvement over the previous system and has simplified and shortened the application process. This new online grant management system makes the procedure much easier for both applicants and reviewers, as well as CTF staff. The Foundation is currently in the process of moving the Drug Discovery Initiative application system online as well, and hopes to have all of the grant programs accessible via the Internet by the end of the year.

From Mice to Humans: The Story of Drug Combination Based on the high incidence of MPNSTs in patients with NF1, the limited treatment options, and the high mortality rate, there is a clear need for medical research into treatment of these cancers. Transgenic mouse models of MPNSTs for use in preclinical drug testing may have great utility in the rational clinical development of targeted agents. Exciting work from the laboratory of Karen Cichowski at Brigham and Women’s Hospital, a member of the NF Preclinical Consortium, demonstrated that using Hsp90 inhibitors coupled with the mTOR inhibitor sirolimus, a Pfizer product, led to dramatic tumor shrinkage in a transgenic MPNST mouse model. This work led directly to the development of a phase I/II trial of ganetespib, from Synta Pharmaceuticals, a potent small molecule inhibitor of Hsp90 with a favorable safety profile, in combination with the mTOR inhibitor sirolimus for patients with refractory MPNST. The multi-institutional clinical trial sponsored by the Sarcoma Alliance for Research through Collaboration (Principle Investigator A. Kim Aerang) has successfully obtained funding from the Department of Defense Clinical Trial Award program and is planned to be open for patients by the end of 2013.

CTF Funded Research Makes Progress

Results from Your Donations

• Christopher Maxwell, PhD, a 2012 Drug Discovery Initiative awardee from the University of British Columbia, has recently published in Oncotarget results from his sponsored study. Maxwell tested a small molecule inhibitor of Aurora A kinase in a xenograft mouse model of an NF1-MPNST. A significant reduction of tumor size and stabilization of growth resulted. This study opens new insights into the molecular mechanism and role of Aurora A kinase as a valid therapeutic target in MPNST tumors, especially if considered in combination therapies. • Filippo Giancotti, MD, PhD, who received a Drug Discovery Initiative award in 2012, also reported positive preliminary results on a novel small molecule in collaboration with a pharma company. The compound, which is already in Phase 1 clinical trials for an oncology target, showed selective efficacy in an NF2 mutant mesothelioma and schwannoma cell models. In the next six months the compound will be tested in an animal model and if the results are positive, a new clinical trial for NF2 could follow. • Joseph Kissil, PhD from Scripps Institute collaborated with CTF to evaluate a compound with a potentially novel mechanism of action against NF2-associated schwannomas in an NF2-relevant animal model. Preliminary results are encouraging showing a significant inhibition of growth of the NF2 tumors. Testing at different doses is ongoing and we hope to share more positive results soon.

2013 forum is largest ever continued from page 1 Internet hero in the NF community for his moving speech at the Foundation’s NYC Gala last December. Drew’s address in Nashville brought hope to those affected by NF, and was met with a tearful standing ovation. Saturday afternoon included several lectures on “The World of NF Research” from physicians and clinicians, followed by “Building a Strong Community” in which CTF staff took attendees through Foundation work in research funding, the NF registry, advocacy, and the use of social media to raise awareness and funds. Then, attendees broke up into specialized tracks for information concerning research in NF1, NF2, and schwannomatosis.

That evening, the Forum convened at the Country Music Hall of Fame where participants took a tour of the museum and ate dinner in the windowed atrium. It was a unique opportunity and the perfect cap to a full day of education and inspiration. This year’s Forum was chaired by Dr. Kaleb Yohay of New York-Presbyterian Hospital/Weill Cornell Medical Center and Dr. David Stevenson of the University of Utah. It also featured a meeting of the Children’s Tumor Foundation Chapter Council which represents the many CTF volunteers across the country. The Chapter Council selected Jill Markland from

Christopher Maxwell

Filippo Giancotti

Joseph Kissil

Lawrenceburg, IN as its new Chair, Connie Sorman from Pittsford, NY as Vice-Chair, and Karyn Zazula from Drexel Hill, PA as Secretary. In addition, the Forum held its first NF Clinics Meeting for clinic coordinators, nurse practitioners, genetic counselors, and other professionals involved in the care of NF patients. On Sunday morning, attendees closed out the Forum by gathering in Nashville’s Public Square and participating in the Nashville NF Walk. From there, it was planes, trains, and automobiles home, to spread hope and information to the entire NF community. * Save the Date: NF Forum 2014, Washington, D.C., June 6-8


national programs

Racing4Research Team Off to a Great Start! Early spring means that race season is well underway and the Racing4Research team has started off well with six podium finishes - three wins! - and more than 150 CTF friends and family cheering at the track. For the 2013 race season, the Children’s Tumor Foundation is represented in the Grand-Am Road Racing series with the Daytona 24 team of Park Place Motorsports. In April, drivers Patrick Long and Patrick Lindsey took home a hardearned second place finish at Road Atlanta, putting the CTF logo on the podium for the first time in the Grand-Am Rolex Series. In the Pirelli World Challenge series, CTF has found a home with


California-based GMG Racing onboard the No. 14 GMG / Children’s Tumor Foundation Audi R8. The No. 14 has had a “podium perfect” year-to-date, with driver James Sofronas putting CTF on the podium in each of the first five races of the season! Racing4Reearch has a number of events across the country that allow our NF Heroes and their families to join us at the track through tickets donated to CTF. Don’t know anything about racing? No problem. No experience required. Be prepared to be inspired!


national programs upcoming events July 14, 2013 NEW YORK CITY TRIATHLON New York, NY

July 21, 2013 CHICAGO ROCK’N’ROLL HALF Chicago, IL

July 28, 2013 IRONMAN LAKE PLACID Lake Placid, NY

August 24-25, 2013 CHICAGO TRIATHLON Chicago, IL *Kids race available

October 13, 2013 CHICAGO MARATHON Chicago, IL

November 1, 2013 IRONKIDS FLORIDA Panama City Beach, FL

November 3, 2013 NEW YORK CITY MARATHON New York, NY

January 11-12, 2014 DISNEY WORLD MARATHON Orlando, FL *Half, Goofy Challenge & Family 5K available

April 20, 2014 LONDON MARATHON London, England See a full list of events at

Share Your Photos 8

Is an Ironman race on Your Bucket List? The Children’s Tumor Foundation will be the exclusive charity partner for the 2014 Ironman Florida race. The event typically sells out in less than two minutes and the only way to get in will be through the CTF NF Endurance team. The Foundation is thrilled to be one of the first charities to partner with Ironman in such a capacity. CTF will also be the beneficiary of the first ever IronKids Florida events in 2013 and 2014. Open registration for Ironman Florida will be available to the public on November 1, 2013. The CTF NF Endurance website will be live on July 1, 2013 at which point you can reserve your spot on the team. If you have ever thought about doing an Ironman or know someone who may be interested, make sure to sign up by visiting . The fundraising minimum for the event will be $5,000 which includes the race registration fee and a virtual training plan from CTF’s certified coaches. Participants will also receive CTF gear and discounts from NF Endurance sponsors and partners, including TYR and Bike Transport.

The NF Endurance Kids Program is on the Move! Since the introduction of the NF Endurance Kids Program last December, children across the country have joined the fight to end NF. The NF Endurance Kids Program is designed to get young people involved in fitness and running, spread awareness about NF, and engage kids in the mission of the Children’s Tumor Foundation. All while having a lot of fun! Photo: John Parker

How does the program work? Kids collect pledges and donations for the Children’s Tumor Foundation, participate in short-distance races in their communities, (anything from a local 5K to a school-wide one mile fun run), and earn a variety of great fundraising prizes! On race day, our young team members look like pros as they cross the finish line in their free Children’s Tumor Foundation jersey, and receive an official NF Endurance finisher medal. A few weeks later, they receive their fundraising prizes in the mail which range from CTF branded hats, bags, and Frisbees, to gift cards and sports watches! For more information, to find an NF Endurance Kids team near you, or to start a team in your community, please visit .

Follow us on social media and share your photos by tagging us or using the hashtag #nfendurance. You could be featured on our website or in the next newsletter! |

In March, Gale Barnhart and Mary Schafer attended the NF Endurance program’s first semi-annual National Volunteer Summit in New York City. The next summit will be held in Chicago on October 11-13, 2013 (Chicago Marathon weekend). If you are interested in leading a volunteer committee and bringing an NF Endurance team to a race in your community, you may be eligible to attend. Please email for more information.

Cathie Leys ran in the 2013 London Marathon after winning her spot in this year’s NF Endurance New Year’s Resolution Raffle. NFE has guaranteed entries in this exclusive race for 2014. Make sure to secure your spot on the team today by visiting .

The team that participated in the 2013 Kentucky Derby Marathon and Mini Marathon, one of this year’s NF Endurance Community Events, raised over $44,000 for the Children’s Tumor Foundation. If you are interested in starting a new NFE event in your community, please email .



national programs Don’t Just Walk‌

Experience the NF Walk

NF Walk events are so much more than a stroll in the park. They are memories in the making. Kids Runs

Stroller Derbies A Celebration of NF Heroes

Food With Friends

5K Races

Fun Raffle Prizes

Face Painting Cupcake Decorating


Balloon Releases |

NF Champions Ken Janik and Erica Givens, Top Nashville NF Walk Fundraisers

Thank YOU! Huge thanks to Nashville, Tulsa, Greensboro, Greensville, Orlando, Staten Island, Rhode Island, and Monroe for hosting successful NF Walks this year! Much gratitude to the organizers, committees, participants, and donors, together you raised over S140,000 for NF research!

Ken Janik from Cheektowaga, NY and Erica Givens from Nashville, TN both live with NF2 and met through a Facebook support group. After emailing for a few months, they decided to attend the 2013 NF Forum and Nashville NF Walk and meet in person. Ken was one of the top fundraisers for the Nashville NF Walk and Erica was the Captain of Team Givens, one of the top teams. They enjoyed getting to know one another and are now a couple. “We are very happy,” says Erica. “Our relationship formed partially because of the NF Forum and Nashville Walk. So thank you very much, CTF, for helping bring us together!”

Live Music and Entertainment

Involve Your Local Business and Your Workplace in an NF Walk! There are many ways that businesses and places of employment can support the NF Walk program:

upcoming NF Walks August 24, 2013 Salt Lake City, UT

Sept.14, 2013 Biddeford, ME

Sept. 28, 2013 Putnam, CT

Sept. 8, 2013 Denver, CO

Sept. 21, 2013 Des Moines, IA

Sept. 29, 2013 Rochester, NY

Sept. 14, 2013 St. Louis, MO

Sept. 22, 2013 Addison Oaks, MI

September 29, 2013 Central Jersey, NJ

Sept. 14, 2013 Jacksonville, FL

Sept. 28, 2013 Milwaukee, WI

Sept. 29, 2013 Minneapolis, MN

Sept. 14, 2013 Brunswick, ME

Sept. 28, 2013 Bowling Green, KY

• Become a monetary sponsor • Match employees’ gifts • Start a corporate team and attend the NF Walk • Volunteer with co-workers • Donate goods or services for event day **If you or someone you know would like to provide a sponsorship for an NF Walk, please email or call 212-344-6633, ext. 8543 or 8544. 11

meet the staff

meet the board

Chrissie Connors

JoAnne Pastel

Hometown: I am from Valhalla, New York. Our only claim to fame is that Babe Ruth is buried there. Current Town: The Washington Heights neighborhood of Manhattan Education: I studied Political Science and Philosophy at Fordham University and I just finished taking a Spanish class at NYU. If you want to speak Spanish with me or talk contemporary French Philosophy, I’m all ears. Work with the Foundation: I am a Coordinator on the NF Walk Program with Jessica Beckerman and Traceann Adams. I work with local event Chairs to coordinate the details of organizing a walk and I help our participants reach their fundraising goals. My main objective is to ensure that everyone involved in an NF Walk has a great time! My favorite aspect of my job is talking to a participant who doesn’t think they can reach their fundraising goal. By event day, they have usually superseded their target by a mile! It’s wonderful to see people exceed their own expectations. Favorite Memory with the Foundation: I really enjoyed the NF Forum in Nashville this past April. It is amazing to meet all of the families who are determined to see an end to NF and to hear their stories. It keeps me motivated at work. Favorite Hobby: I love to cook while listening to jazz, soul, or funk records. My favorite things to cook are curry, pizza, and Sunday Gravy, and every weekend I make a huge meal. My hobby doesn’t really mesh well with the city I live in since New York kitchens are tiny! 12

Hometown: Minneapolis, Minnesota Current Town: I am currently living in Orono, Minnesota, a western suburb of the Twin Cities. Education: I have a Master’s Degree in Marketing from the University of St. Thomas located in St. Paul, Minnesota. Work with the Foundation: In May 2011, I became a member of the Board of Directors. Also, I was local Chair of the 2011 NF Forum held in Minneapolis, and Co-Chair of the 2011 inaugural Minneapolis NF Walk. In 2012, I served as Chair of the second annual Minneapolis NF Walk. Over $81,000 in corporate and individual donations were raised to support the efforts of the Children’s Tumor Foundation. Currently, I am Chairing the third annual Minneapolis NF Walk to be held at the Mall of America in September 2013. Last year, I also started an NF Awareness Night with the Minnesota Twins Baseball team. Someone from the Twin Cities NF community is asked to throw out the first pitch at this game. In addition, the Children’s Tumor Foundation is recognized on the scoreboard along with several facts about NF to help create awareness. Families affected by NF along with friends and other community leaders are in attendance. This past May was our second year as an event. Favorite memory with the Foundation: Meeting all of the families, friends, and members of the Board that are affected by NF along with the wonderful staff that work at the Children’s Tumor Foundation. When I first became involved with CTF, after my five-year-old daughter, Jacqueline, was diagnosed with NF1 at four months old, it made me feel like we were not alone in the battle against neurofibromatosis. Everyone is so supportive and embraces one another. I am thankful for my NF family! Favorite hobby: My kids, exercising, boating, and watching basketball, baseball, and soccer games.

CORRECTION A news item on page four of the Winter 2013 edition of NF News wrongly described radiation therapy as “standard for NF2 vestibular schwannomas.” It should have read, “Radiation therapy is standard for vestibular schwannomas.” Radiation may be used to treat NF2 vestibular schwannomas on an individual basis, taking into account the patient’s age and degree of functional impairment. CTF regrets the error, which occurred in a note about a CTF Clinical Research Award for “Effect of Anti-VEGF and Radiation on NF2 Vestibular Schwannoma.”

research news CTF Participates in National Brain Tumor Society/American Association of Cancer Research Systems Biology Think Tank Meeting In January 2013, Annette Bakker, Chief Scientific Officer of the Children’s Tumor Foundation, was invited by the co-chairs of the National Brain Tumor Society (NBTS)/American Association of Cancer Research (AACR) meeting, Dr. Andrea Califano (Columbia University, New York), Dr. Gordon Mills (MD Anderson, Houston), Mr. N. Paul TonThat (Executive Director, NBTS) and Margaret Foti (CEO, AACR) to participate in a think-tank discussion of the emergence of new disciplines in cancer biology. The specific focus of the meeting was to identify the critical goals and accomplishments that uniquely characterize the new field, as well as to identify possible impediments and roadblocks that may hinder its growth. CTF was privileged to be represented amidst a group of internationally recognized systems biologists for two days. A key distinguishing element that emerged from the discussion is the role of systems biology in generating computational models that are ultimately routed in cell regulatory processes to achieve a

CTF Holds First National Meeting for Neurofibromatosis Clinic Specialists at NF Forum Over 30 NF clinic coordinators, genetic counselors, and nurses attended a full-day meeting last April at the NF Forum in Nashville. The clinicians, who represented 21 of the 45 members of the NF Clinic Network, clearly appreciated the opportunity to hear inspiring and practical presentations and learn how different NF clinics approach their common goals. This event, known as the NF Clinics Meeting, offered presentations from experts in neurofibromatosis genetic testing, educational programs, diagnosis, and clinic

more mechanistic analysis and interpretation of experimental data. Many examples were identified as success stories from the use of kinetic models to elucidate response to combination therapy, to the use of regulatory networks to elucidate key drivers of tumor initiation, progression, and Dr. Andrea Califano chemo-sensitivity, to the initiation of clinical studies based on results originating from systems biology. A clear message from the meeting’s participants is that access to open, multimodal datasets is one of the most critical requirements for the success of these new approaches and that some creativity will be needed to deal with the current publication environment. The community also identified the need for grand challenges that can be addressed by the entire community, via crowdsourcing approaches, such as, for instance, predicting chemo-sensitivity to a specific panel of experimental drugs in pre-clinical tumor models. In this, systems biology approaches are unique as they have spearheaded the ability to integrate the results coming from a large number of labs into valuable sets of predictions that have a high likelihood of being experimentally validated.

management. Equally important, it gave the clinicians a chance to spend time with colleagues from all over the United States (and one from Canada). Attendees also had the opportunity to attend the NF Forum presentations and special events over the following days. Attendee evaluations were uniformly positive and there was a definite interest in convening similar meetings in years to come, which CTF plans to do. A demonstration by Kaleb Yohay, MD, director of the Comprehensive Neurofibromatosis Clinic at New York-Presbyterian Hospital/Weill Cornell Medical Center, of the database system he designed to track

patient care, led to strong interest by attendees in obtaining this database for use in their clinics. CTF is working with Dr. Yohay to develop this system for other clinics to use. Facilitating a continuing of the conversation among clinics is a key goal following the success of the NF Clinics Meeting. One idea that CTF is pursuing is holding a regular call-in meeting for all clinics. The Foundation is also currently assessing an online discussion group, NF Connect, which is now being used by a small group of testers before being opened to all NF clinicians. Check the CTF website ( ) to see when NF Connect goes live for all clinicians.


regional news

Find Your Local Children’s Tumor Foundation Chapter The Foundation has a presence in nearly all 50 states and facilitates local patient support groups, medical symposia, and fund raising events. Learn more about the Children’s Tumor Foundation Chapter in your area by visiting .

Georgia Approximately 120 golfers and over 30 volunteers participated in the 2nd Annual CureNFwithJack Golf Classic at the Alpharetta Athletic Club on April 15, 2013. Generous sponsors including Kroger, Verizon, UPS, Howe Roofing, and Roasters made the day possible. All involved felt that it was even bigger and better than last year! To learn more please visit or contact .

wisconson The Polak family hosted a threeon-three “Shoot for a Cure” basketball event in Oshkosh, WI on April 21, 2013 on behalf of their NF Hero, Owen.


south carolina

On Saturday, May 4, 2013, families came together in Amesbury, MA to take part in “Spring For A Cure: Molly’s Royal Faire.” Faire games, a silent auction, a raffle, and a royal buffet were all part of the fun. Guests were encouraged to wear their best royal garb with an award given to the Best Child and Best Adult costumes. Special thanks to Molly and the entire McBryan family for hosting this annual event!

The third annual Greenville, SC NF Walk took place on Saturday, May 4, 2013, in Conestee Park and raised nearly $10,000. Not letting the chilly winds deter them from their goal, individuals and teams covered the course and enjoyed the day. Many thanks to all who took part, especially Susan Luttrell, Becky Ward, Mary Meyers, Chris Hogan, Dee Risner, Carol Mohrmann, Dottie Risling and their families for the incredible amount of time and effort they put into organizing the event.

new jersey CTF’s New Jersey Chapter hosted their 7th Annual Casino Night on April 5, 2013. This event continues to be a success as it “Deals for a Cure” in support of NF research. With over 230 attendees, including Ottis Anderson, New York Giants Superbowl XXV MVP and two-time Superbowl Champion, the evening celebrated local NF Heroes and named Antonio Danino its 2013 Honoree.

florida NF Hero Lilly Ann Brooks organized a “Cashual Friday” among her fellow students at The Christ School in Orlando, FL. Those who donated $1.00 to the Children’s Tumor Foundation were allowed to dress casually on a designated Friday. Lilly Ann distributed flyers and gave a talk at her middle school to promote the fundraiser. Together the students and staff raised $600. Thank you Lilly Ann!

connecticut Welcome to Rhianna Curotto, new Chapter Representative for the State of Connecticut. Rhianna has already organized an NF Walk to take place in Putnam, CT on September 28, 2013, and a can drive fundraiser during May for NF Awareness Month.

colorado The 13th Annual NF Women’s Day Gathering, an opportunity for women affected by NF to share and support each other, was held on March 27, 2013 in Westminster, CO at Atlanta Bread Company. It was hosted by Brook Draper and Jane Cahn and featured speeches by Shelley Pesta and Cindy Lambrick.

arizona The Vogel family hosted a Tea For NF in Honor of NF Hero Hunter on May 4, 2013 in Avondale, AZ.

california San Diego NF Walk committee member Priscilla Cortez hosted a Party for A Cure on March 30, 2013 in La Jolla, CA. All proceeds benefitted Team Ruben who will be participating in the San Diego NF Walk on November 2, 2013.

Mary Vetting,

california The Children’s Tumor Foundation West Coast Benefit, “Lights, Camera, Action! Shining a Spotlight on NF,” was a big success. Held on April 27, 2013 at CBS Studio Center in Studio City, CA, the event featured veteran TV actor and Daytime Emmy Award nominee Dondre Whitfield as Master of Ceremonies, and New York Jets Placekicker Nick Folk signing autographs. Local NF Heroes in attendance received mini Oscar awards and Boy Scout Troop 84 served as ushers on the red carpet. The evening’s olive oil and cheese tasting, specialty cocktails, and make-your-own sundae bar made for a night to remember and the Benefit was enjoyed by all.


On April 12, 2013, the Cleveland Cavaliers hosted a Children’s Tumor Foundation Night. The evening included a chance for the kids to shoot a basketball on the court, and a good time was had by all.

The Children's Tumor Foundation is a 501(c)(3) not-for-profit organization dedicated to finding effective treatments for the millions of people worldwide living with neurofibromatosis (NF), a term for three distinct disorders: NF1, NF2, and schwannomatosis. NF causes tumors to grow on nerves throughout the body and can lead to blindness, bone abnormalities, cancer, deafness, disfigurement, learning disabilities, and excruciating and disabling pain. NF affects one in every 3,000 people, more than cystic fibrosis, Duchenne muscular dystrophy, and Huntington’s disease combined. The Children’s Tumor Foundation funds critical research into neurofibromatosis. In addition to benefiting those who live with NF, this research is shedding new light on several forms of cancer, brain tumors, bone abnormalities, and learning disabilities, ultimately benefiting the broader community. For more information, please visit . Editor

On March 21, 2013, Ponderosa High School in Parker, CO organized a Prom Fashion Show fundraiser. The school’s seniors modeled their prom dresses and tuxes and speakers included Shelley Pesta, President of CTF’s Colorado Chapter, Michelle Kroll, Ryan Kroll, Andy Collins, and Presli Collins. The show featured performances by a school band and a local dance studio, and the event raised $5,000 for CTF.


NF News is the official publication of the Children’s Tumor Foundation and is published quarterly. All issues are available on our website at . Please direct any questions or feedback to the editor (listed below).

Foundation Staff John W. Risner, President Research Annette Bakker, PhD, Chief Scientific Officer Salvatore La Rosa, PhD, Director of Research and Development Pamela Knight, Clinical Research Manager Daniel Aiese, Research Program Assistant Development and Volunteer Relations John Heropoulos, Senior Vice President Traceann Adams, NF Walk Program Director Jessica Beckerman, NF Walk Coordinator Chrissie Connors, NF Walk Coordinator Jill Beck, Racing4Research Program Director Sarah Coulam, NF Endurance Program Director Emily Phillips, NF Endurance Manager, Logistics/Marketing Lauren Fritz, NF Endurance Manager, Chicago Suzanne Cannon, NF Endurance Team Manager Alissa Marks, NF Endurance Manager, Marketing Angela Auzston, NF Endurance Community & Youth Events Mgr Chad Leathers, Program Director, Cupid’s Undie Run Patrice Pancza, Program Director, Grants and Foundations Garrett Gleeson, Program Director, Major Gifts Carolyn Castellano, Project Director, Major Events Kelly Mills, Volunteer Relations Regional Manager Julie Pantoliano, Volunteer Relations Coordinator Alison Désir, Volunteer Relations Coordinator Marissa Moscatello, Volunteer Relations Coordinator Kristine Poirer, Volunteer Relations Coordinator Sarah Morley, Special Event Manager Public Education and Patient Advocacy Simon Vukelj, Communications Director Mary Vetting, Communications Associate Finance and Administration Judi Swartout, Chief Financial Officer Sarah Anderson, Accountant Monique Boucher, Database Supervisor Mohamed Amin, Database Assistant Rosa Amelia Perez, Project Administrator Sarah Rosenberg, Program Assistant Danielle Meyer, Tech Support, National Programs

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Chapter Council Representatives at the NF Forum

Stories of Nf:

Jessica Contreras

“I was diagnosed with NF1 when I was 12. My family and I had never heard of it. Heck, I couldn’t even pronounce it! At 13, I had my first tumor removed. After that, I had multiple surgeries to remove tumors, mainly on my arms, hands, and legs. I lived a fairly normal life between my late teens and early 20’s. “When I was 27, I started noticing that I had to turn my head to the left to hear people. I scheduled a doctor’s appointment, and that visit changed my life. I was diagnosed with NF2 and told I had five brain tumors. I went to see a neurologist at Stanford, thinking we would discuss my brain tumors. I was shocked to learn that I also had a nine centimeter tumor at the top of my spine. I had it removed, and it left me paralyzed. Doctors weren’t sure if I would be able to walk again. A week after my surgery, I was transported to Santa Clara Valley Medical for physical therapy. With the love, prayers, and support of my family, six months later I was taking steps. “I will be participating in the San Jose NF Walk because I am thankful that I am able to walk, and I want to help fund research. I pray that one day I can tell my great-grandchildren, ‘Back in my day, there was no cure for NF.’ “Living with NF2 is just one of my many awesome qualities. I have a lot of scars but I call them my ‘strength badges.’ My journey with NF hasn’t been easy, but it’s made me appreciate the simple things in life. To my fellow Nf’ers, here’s a quote I live by from the movie, Joe Dirt, ‘Life’s a garden - dig it.’”

Children's Tumor Foundation Newsletter, Volume II 2013  
Children's Tumor Foundation Newsletter, Volume II 2013