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NF NEWS W I N T E R 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

Children’s Tumor Foundation Benefit Gala Breaks Fundraising Record The Children's Tumor Foundation held its Annual Benefit Gala on December 6, 2012 in New York City. The Gala, postponed from November 1, 2012 due to Superstorm Sandy, was one of the few major events in the New York area that persevered and rescheduled, just five short weeks after the original date. Despite all the obstacles, the event raised more than $2 million for neurofibromatosis (NF) research. With over 500 people from across the country in attendance, the atmosphere in the ballroom at Cipriani Wall Street was filled with cheers as the record breaking tally was announced.

CTF’s Ambassador, Mr. Drew Leathers, with his brothers Ben and Chad, stands for the first time in three years at the Gala.

The Foundation was honored by the participation of several special guests. World champion race car driver, TV personality, and Racing4Research partner, Justin Bell served

as master of ceremonies. Grammy-nominated Latin Jazz pianist Chuchito Valdes and his Quartet provided the musical entertainment for the evening, and auctioneer Nicholas Lowry, of Swann Galleries and Antiques Roadshow fame, led the live auction which included an opportunity to donate funds to CTF's Clinical Research Awards. Guests gave generously during this portion of the auction knowing that every dollar they contributed would be matched by an anonymous donor. Heartfelt speeches were delivered by the evening's honorees, Mr. Richard A. Horvitz and Dr. Kaleb Yohay, recipients of the Children's Humanitarian Award, and Mr. Drew Leathers, the 2013 Children's Tumor Foundation Ambassador. continued on page 12

Young Investigator Awardee Reports Major Advance in NF2 Research

Helen Morrison, PhD

The Children’s Tumor Foundation is pleased to announce that a study led by Helen Morrison, PhD and CTF grant recipient Alexander Schulz has discovered a previously unknown mechanism for the peripheral nerve damage seen in NF2 patients.

NF2 has long been known to cause nerve damage by harming Schwann cells, which wrap around nerves as Alexander Schulz part of the protective myelin sheath. Schulz and colleagues found evidence that axons, which are long nerve fibers that carry signals from nerve cells, are also harmed. While this finding is based on animal studies, similar changes in axons were seen in nerve tissue taken

from two human patients with NF2 and peripheral neuropathy (numbness in the hands or feet). As reported in the scientific journal Nature Neuroscience, damage to axons may explain the mystery of why some NF2 patients develop peripheral neuropathy, even if they have no tumors affecting these areas. This work was funded by a 2012 Children’s Tumor Foundation Young Investigator Award to Schulz, a medical student in the laboratory of Helen Morrison in the Leibniz Institute for Age Research in Jena, Germany.

“This report is groundbreaking because it gives us a new target for studying NF2,” explains Annette Bakker, PhD, Chief Scientific Officer at the Children’s Tumor Foundation. “If axons can be damaged as a result of NF2, we need to widen our view to include finding ways to address this damage, instead of being focused only on the Schwann cell changes in NF2.”

FROM THE PRESIDENT A note from Children’s Tumor Foundation President JoHN w. RISNER



The Children’s Tumor Foundation is pleased to announce

the creation of an online NF Registry, a new tool in the fight against neurofibromatosis. The purpose of the registry is to find people who may be eligible for clinical trials or other research studies being conducted in the field of neurofibromatosis, and to determine the commonality of specific NF characteristics.

Join today and help end NF! 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 Denise Pitzman, Chapter Council President Allan E. Rubenstein, MD, Director of Medical Affairs Emeritus

Daniel Graeff Steve McKenzie JoAnne Pastel Laura Perfetti Jason Pontin John W. Risner Robert Schaffer Rachel B. Tiven David Viskochil, MD, PhD Nate Walker Peggy Wallace, PhD

Daniel Altman Laura Ganio Bona William Brooks Colin Bryar John Catsimatidis Tracy Galloway Daniel Gilbert John Golfinos, MD

Honorary Richard A. Horvitz Michie Stovall O'Day Harold Ramis Alan Robbins, MD Doris Schnuck Carolyn E. Setlow Ed Stern, Pro Bono Counsel

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In past years, January and February were focused on developing our finance, operations, and research reports for the prior year, and finishing the plans for the current year’s events. Thanks to the growth of two relatively new programs, Racing4Research in late January, and Cupid’s Undie Run near Valentine’s Day, January and February are now very active months for the Foundation. Both of these programs are not only effective fundraising events, but, just as importantly, they are excellent vehicles for educating the public about neurofibromatosis (NF). Media coverage for both events reached new highs this year, helped by a wonderful weaving of NF into the plot of the FOX television show Bones on February 4, 2013. I recently met with the father of a child newly diagnosed with NF1. He said that the doctor counseled the family to “watch and wait.” They found this plan unacceptable. He and his wife were unwilling to wait for something to happen to their child, and felt a need to do all they could now. It struck me that this is also the feeling within the NF research community, at the Children's Tumor Foundation, and among all those affected by neurofibromatosis. There are many ways to get involved in the fight against NF, and our participation and fundraising will make a great difference in increasing the number of clinical trials in the field of neurofibromatosis. First, enroll in the NF Registry at Adults can enroll themselves, and parents can enroll their children. Getting as many people with NF as possible into this registry, even those with few or no current manifestations, is vital to attracting pharmaceutical and biotech investment in neurofibromatosis. Second, join our advocacy outreach. While this year the federal budget process is particularly complicated, please visit for information and updates. We will likely launch our annual advocacy campaign as soon as we get some clarity on Congress’s plans to deal with the FY13 budget. Third, get involved with one of our fundraising initiatives. From national programs like NF Walk, NF Endurance, Racing4Research, and Cupid’s Undie Run, to local symposia and special events, there are a wide variety of ways to participate in fundraising, public awareness, and patient support. We have a dedicated staff both in New York City and in satellite offices around the country that assist volunteers nationwide. Thank you for your continued support, and your dedication to the Children's Tumor Foundation's mission. I look forward to the coming year, and to the progress it will bring towards our goal of ending neurofibromatosis.

foundation news


The Children's Tumor Foundation Funds Three Clinical Research Awards The Children’s Tumor Foundation Clinical Research Awards fund small-scale clinical trials as part of an ambitious initiative to accelerate identification of NF drug therapies. In response to feedback from the research and clinical communities, the Foundation expanded the scope of the program so that Clinical Research Awards also fund adjunct studies to improve the design and implementation of clinical trials, making them faster and more meaningful. Below are the three most recent Clinical Research Awards funded by the Children's Tumor Foundation:

Here are a few events that may be of interest. For a complete listing or more information, please see 04/05 - Parsippany, NJ New Jersey Casino Night 04/28 - Foxboro, MA New England Yogathon 05/04 - Avondale, AZ Tea for NF

Kathryn North, The Children's Hospital at Westmead

A Phase II Randomized Placebo-Controlled Double-Blind Cross-Over Trial of Methylphenidate in Children with Neurofibromatosis Type 1 Dr. North’s research group in Australia will study whether children with NF1 and attention deficit hyperactivity disorder (ADHD) benefit from the use of methylphenidate (Ritalin, Concerta). It will try to determine the characteristics of those children most likely to be helped by this medication.

Nicole Ullrich, Boston Children's Hospital

Effect of Creating Visual Illness Narratives on Quality of Life in Children with Neurofibromatosis Type 1: A Pilot Intervention Study Children and adolescents with NF1 will be guided in creating their own videos about their experiences. The aim is both to teach their clinicians what they need to know about living with NF1, and to empower these young patients by having them share their stories and articulate what they think and feel to an audience that is eager to listen and understand.

05/04 - Altamonte Springs, FL Painting Party at Creative Canvas & Wine




Lei Xu, Massachusetts General Hospital

Effect of Anti-VEGF and Radiation on NF2 Vestibular Schwannoma


Radiation therapy is standard for NF2 vestibular schwannomas, but is associated with hearing loss in 50% of patients. Bevacizumab is a drug treatment that shows promise as an alternative to radiation, but is not as effective as could be desired. This project will test whether a combination of radiation and bevacizumab might improve effectiveness and lessen unwanted effects.

NF Research: Seeing the Big Picture and Promoting Collaboration It is extremely difficult for a single scientific laboratory or a single clinician to find a cure for NF. Therefore, the Children's Tumor Foundation is committed to fostering collaboration. Attempts to Find Effective Treatments for NF In 2012, CTF analyzed the current state of neurofibromatosis research and development and drug discovery. The issues and bottlenecks were identified, and efforts were made to solve the problems. In order to develop new treatments for NF, the different steps of drug discovery have to be freed from logistics problems; the first step is to get access to the material necessary for scientific testing. continued on page 4

Saturday, April 27, 2013 6:00 - 10:00 PM CBS Studio Center 4024 Radford Avenue Studio City

cocktails, buffet, live & silent auctions Cocktail Attire RSVP at Follow us online: @childrenstumor NF News • Children’s Tumor Foundation | 3

research news CLINICAL TRIALS: Cause for Hope In 2012, the NF Preclinical Consortium (NFPC) helped produce four out of the six clinical trials in NF1, and two in NF2. The Foundation succeeded in shepherding two-thirds of the medicinal compounds tested on patients to the clinic, which is an integral part of the clinical trials process. Collaboration, communication, and coordination of the different teams and hospitals involved in the testing is essential, and best facilitated by an independent non-profit such as CTF. For NF1

Participants are needed for both clinical and observational trials, a further step in the process. Observational trials involve collecting data on the natural course of a disease. Comparisons are made among gene carriers, patients at various stages of the disease, and those who do not have the disease gene.


For NF2

For the complete list of clinical trials open for recruitment in NF, please visit the Foundation's website at: What are Clinical Trials and Why Are They Important? Before a compound can be prescribed for patients, it must go through three phases of clinical trials. At the end of each phase, the compound is evaluated to see if it meets expectations and is ready to proceed to the next phase. In Phase I, a small number of subjects are given the drug and closely monitored to see whether the drug is safe and well-tolerated. Phase II involves more subjects and focuses on finding the correct dose. Safety and tolerability continue to be monitored. Phase III involves a still larger group of subjects - several hundred. Subjects are randomly assigned to either the treatment group or a control group that receives a placebo (a non-active compound, sometimes a sugar pill). The study is double blinded - neither the subjects nor the researchers assessing them will know who received the compound and who received a placebo until the end of the study. Phase III takes much longer because enough time must pass for differences in disease progression to emerge between the treatment group and the control group, assuming the treatment is effective.

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In NF2 CTF has signed a contract with Cenix and the University of Central Florida to follow up on the excellent Drug Discovery Initiative (DDI) results that Dr. Cristina Fernandez-Valle obtained in her NF2-/- cell screen. The cell screen is a model system for NF2. Dr. Annette Bakker proposed applying an advanced screening method from Cenix to gain a fuller understanding of how the selected compounds work. The compounds that will be tested include the confirmed “actives” from the NF2 cell screen as well as a new set of promising compounds selected by Marco Nievo of CTF. The new compounds have the same mechanism of action as the “actives” but have more potential as useful drugs. The data generated during this project will help us prioritize compounds for further preclinical development. Outcome of this pilot trial is expected in fall 2013. In NF1 In collaboration with the National Cancer Institute, Dr. Annette Bakker from CTF commissioned Cenix to use a powerful new technique called silencing RNA screening to study a substance identified by Karlyne Reilly and collaborators as having promising antiproliferative activity in NF1 models. Anti-proliferative agents affect the cell division that fuels the growth of neurofibromas. Focusing on a rational selection of approximately 1,000 high priority genes, this study is designed to identify those genes/pathways whose silencing (i.e. removal) either enhances or suppresses the compound's therapeutic effects, thereby providing important insights into the compound's cellular mechanism of action. This project also may find new predictive biomarkers that could be a basis for developing tests to monitor the activity of this eventual drug. Outcome of this pilot trial is expected within 2013. Following the successful completion of the pilot trials, a next round of partnerships between CTF, Cenix, and other possible entities will be proposed and discussed with the Foundation's Medical Advisory Committee (MAC).

New Discoveries in NF Research The Foundation is proud to announce that two studies funded by CTF resulted in high quality NF discoveries and were published in the prestigious scientific journal, Nature. In addition to Schulz's and Morrison's paper in Nature Neuroscience (please see page 1), a study on spinal meningiomas was published in Nature Genetics. Entitled,"Loss-of-function mutations in SMARCE1 cause an inherited disorder of multiple spinal meningiomas," this study was performed by CTF Young Investigator Award recipient Dr. Miriam Smith, under the direction of Dr. Gareth Evans at the Genetic Medicine, Manchester Academic Health Sciences Centre (MAHSC), St. Mary's Hospital, University of Manchester, UK. The discovery of this new gene opens the field of new targets to fight NF. YOUR FUNDRAISING MAKES A DIFFERENCE: See Where Your Donations Went The Children's Tumor Foundation recently added "disease maps" to its website so that it is possible to investigate the NF research landscape and learn where investments are made. Examining the disease maps will help you understand how many funds are directed toward learning disabilities, hearing issues, malignant peripheral nerve sheath tumors, and the many other manifestations of neurofibromatosis. To see the disease maps, please go to

programs for communication assistance with specific attention to the concerns of people with NF2 such as visual abnormalities and motor dysfunction, both of which can affect the use of the technology needed to run these programs (i.e. cell phones, iPads). A focus group will be held to demonstrate and evaluate several applications, or “apps,” for smartphones and tablets during the NF Forum in Nashville on Friday, April 19, from 2-4 p.m. If you have NF2 and would like to participate in this focus group, or for more information about this research, please contact Ms. Bergner at




Save the Da te!

Helping Patients in the Long and Short Term Since its inception in 1978, the Children's Tumor Foundation has been known for its leadership in NF research and development. While we remain committed to the long term goal of ending neurofibromatosis, we also want to assist patients living with NF today. To do this, we are expanding our mission. CTF has made the decision to increase its support of a variety of programs that assist families affected by NF in their daily lives. One CTF funded initiative currently underway is NF researcher Amanda Bergner's hearing loss aid project: The Use of Technology for Assisted Communication by People with NF2 and Hearing Loss. The principal investigator is Amanda Bergner from Johns Hopkins University and the co-investigator is Jennifer Smart from Towson University. Technology is available to assist with communication for people who have hearing loss. Amanda Bergner Programs that translate speech to text (to be read), text to speech (to be heard) and include pictures to help with both expressive and receptive communication needs exist but have not been evaluated for ease of use or effectiveness within the NF2 community. Ms. Bergner proposes to review and evaluate multiple available

June 8- 11, 2013 • Monterey, CA • Portola Hotel & Spa

2013 Co-chairs Alison Lloyd, Ph.D., University of College London, United Kingdom Scott Plotkin, MD, Ph.D., Massachusetts General Hospital/Harvard Medical School NF News • Children’s Tumor Foundation | 5

national programs

Racing4Research: Overcoming Adversity Both On and Off the Track Perfect weather greeted more than 150 NF Heroes, their families, and guests at this year's running of the Rolex 24 Hours At Daytona. The event marked the start of the sixth year of the Racing4Research fundraising program, and the team turned in a resilient performance worthy of the young NF Heroes they were racing to support. After running as high as second in the opening minutes of the day-long race, the team slipped into a race of mixed fortunes that saw the No. 73 Park Place Motorsports/Racing4Research Porsche GT3 team - drivers Daniel Graeff, Patrick Long, Patrick Lindsey, Spencer Pumpelly, and Jason Hart - encounter virtually every type of hardship the Rolex 24 At Daytona can throw at a team and drivers. Between a wheel issue on a pit stop, a three minute stop-and-hold penalty for an improper wave-around, and heavy contact in Daytona’s “Bus Stop,” the No. 73 was dealt a deck of cards that would ordinarily be difficult to overcome. Still, on every occasion, the team bounced back and made repairs to keep the car running and see the checkered flag in an eventual 16th place finish. “For us, it was all about finishing for these kids, our NF Heroes, who have had many days like we had and will have many more. So there’s a greater purpose here than just glory and fame. These kids ground you and humble you and remind you what it’s about, and it’s just great to be around them,” said Patrick Long, Porsche factory driver and first year Racing4Research driver. While results on track were mixed, off track the NF Heroes and their families enjoyed a wonderful weekend in sunny Daytona Beach,

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Florida. The program's traditional Friday Night Team & Family Dinner featured the drivers of the No. 73 and No. 72 teams and was hosted by Justin Bell, Racing4Research driver and SPEED on-air personality. Saturday started bright and early with breakfast at the suites along pit road, followed by a private meet-and-greet and photo session with the drivers and crew. The No. 73 car featured the names of more than 100 NF Heroes from around the country, and the Heroes in attendance signed the car prior to the race. Capping off the weekend was news that the 2013 campaign had raised more than $670,000 for NF research. NF Dad James Lee summed up the weekend best, "Going down to the Rolex 24 race in Daytona was amazing. My kids were extraordinarily excited about meeting the drivers and being so close to the cars. My favorite moments were seeing the drivers and crew stop, just a few hours before such an important event, to talk with the children. Not just sign autographs and move on, but really interact. I watched one of the crew sitting with a little boy, asking where he was from and how he was enjoying his vacation. Another driver was given a good luck coin by a little girl; he asked her to sign it and taped it to the car. Two hours before the race, my daughter asked if she could sit in the car; they let her in without hesitation. In addition, it is always great to meet the NF families, and see the CTF folks who have become an extension of our family over the years. It was wonderful to meet all of these people doing so much to help simply because they can. These are the people that made it a memorable trip, and reinvigorated me to fight another year."

national programs

Bones Episode Features Subplot on Neurofibromatosis Earlier this year, the FOX Television show Bones aired an episode featuring a subplot on neurofibromatosis and several NF Heroes in blue CTF t-shirts emblazoned with the Foundation's logo. Michael Peterson, a writer/producer for Bones, spearheaded the initiative to have NF included on the show. He was inspired to do so by his daughter. "Three years ago, my wife, Kelly, and I were blessed with our first child, Natalie," said Peterson. "Right away, we noticed that Natalie had a bowing of her left leg. Our pediatrician told us the leg would straighten in time.

When Natalie was six months old, a second pediatrician diagnosed her with neurofibromatosis." "Last year, my wife and I had an epiphany," Peterson continued. "Maybe the fact that I work on a 'bone show' and my daughter has an illness that has manifested exclusively in her tibia isn’t just some weird cosmic irony – maybe it’s an opportunity. I came to my bosses, Hart Hanson and Stephen Nathan, and they generously allowed me to pursue a story line that would help raise NF awareness."

NF Hero Cristina Hernandez with Bones stars David Boreanaz and Emily Deschanel.

That episode, entitled “The Doll in the Derby,” aired on February 4, 2013.

NF News • Children’s Tumor Foundation | 7

national programs NF ENDURANCE: iNFinite possibilities 03/17 ING New York City Half Marathon New York City, NY

The Children’s Tumor Foundation NF Endurance Team is Off and Running!

04/27 Kentucky derby marathon Louisville, KY

05/04 INDY Mini Marathon Indianapolis, IN

06/01 Ironman hawaii 70.3 Kohala Coast, HI

07/14 New York City Triathlon New York City, NY

07/21 Rock ‘n’ Roll Chicago Half Marathon Chicago, IL

07/28 Ironman Lake Placid

Meet Lauren Fritz The NF Endurance team is pleased to welcome Lauren Fritz to the Children's Tumor Foundation. Lauren will be the NF Endurance Manager for the Chicago area and will develop a volunteer committee that will meet monthly to cultivate a presence in The Windy City. She will also work on managing the three premiere Chicago events: the Chicago Rock ‘n’ Roll Half Marathon, the Chicago Triathlon, and the Bank of America Chicago Marathon. Lauren comes to us Lauren Fritz, NF Endurance from the Alzheimer's Association where she held the position of Specialist, Manager for Chicago Foundation Relations. She worked on their national gala, and also took the lead on planning their first National Alzheimer’s Dinner. Lauren brings experience with building volunteer committees as well as fundraising and constituent relationship management. Lauren is also a marathon finisher, and recently completed the Bank of America Chicago Marathon.

Are you a Super Mom? Motherhood is one of the most challenging jobs in the world. Add the dedication and perseverance required to train for an endurance event, and you have a SUPER MOM! This year we have hundreds of moms lacing up their sneakers in the early hours of the morning to sneak in as many miles as possible before their kids wake up. We want to take a moment to thank all of our moms for choosing to run with the NF Endurance team. You inspire and encourage us!

New Year's Resolution Contest Winners

Lake Placid, NY

09/15 ROCK 'N' ROLL PHILADELPHIA Philadelphia, PA

10/14 bank of america chicago marathon Chicago, IL

Cathy Leys

Kristina Rath

This year, the NF Endurance team offered two lucky athletes the chance to win entries into the exclusive 2013 Boston Marathon and the 2013 London Marathon. Entrants submitted their New Year's Resolutions, and the random drawing was held on January 16th. We are pleased to announce that the winners were Kristina Rath for the Boston Marathon and Cathie Leys for the London Marathon! These two women will be blogging about their training experiences weekly. You can follow their journey by going to

11/03 New York city marathon New York, NY

CHOOSE YOUR OWN EVENT: Run any local race of your choosing. For more information, or to register for an event, please go to 8 |

Ironman News • The Children’s Tumor Foundation will be the official charity partner of the 2014 IRONMAN Florida! The third-oldest North American race of its kind, IRONMAN Florida began in November 1999. It takes place in Panama City Beach which is known for pristine sand and moderate fall temperatures. It’s the perfect time of year to visit northwest Florida, and the event is as much a destination vacation as it is a race. • We will soon have news on how to secure one of our first-ever charity entries into the 2015 IRONMAN World Championships Kona. Stay Tuned!

"I run because it is a miracle that Caitlin can walk." Does the cold weather have you stuck on the couch? Read Laura Michele’s story and learn what inspires her to run every day.

"I run because it is a miracle that Caitlin can walk. My cousin Caitlin is a 19-year-old woman living with NF1, and her personal challenges include hearing loss, visual impairment, and learning disabilities such as dyslexia. Caitlin also wears a leg brace due to a tumor that runs the length of her foot and causes her intense pain. She used to be active in school sports Laura Michele and her such as cheerleading and basketball, but her tumors have grown so much cousin Caitlin that participation became too physically painful. Caitlin's doctors have also cautioned that should the tumor in her foot grow much larger, amputation will be necessary. In addition to the complications NF brings, Caitlin now has titanium rods and pins in both legs, and much of her spine was replaced with a titanium rod as well. No matter what challenges she faces, though, Caitlin is determined to pursue a career helping students with special needs. She is a constant source of positivity, and I am in awe of the strength of her spirit. "I ran the 2011 New York City Marathon in honor of Caitlin; it was my first road race ever. I am very inspired by my cousin and wanted to do something big to honor her fight. So I spent months training and turning myself into a runner, and completed the marathon! The day after the race, Caitlin was in a car accident on her way to school. Doctors were not sure she would live, let alone walk. But she survived and was walking within six months of the accident – most poignantly, across the stage to receive her high school diploma!! "I am thrilled to be running 13.1 miles through New York City on March 17th in honor of my hero, Caitlin, as a member of the Children's Tumor Foundation NF Endurance team. I am grateful to this organization for its commitment to making the lives of people with NF better."

Will Your Business Be Our Next Partner? Jonathan Byler, owner of ACME Bicycle Co., a professional bicycle fitting studio, has partnered with the Children’s Tumor Foundation for the 2013 IRONMAN Lake Placid. ACME Bicycle Co. is a custom bicycle specialty studio, and Jonathan has fitted bikes for over 1000 people from beginners to professionals. ( ACME has become an NF Endurance Community Partner, one of the first in New York City. ACME provides a discount to all NF Endurance participants and promotes CTF on social media and at its store. Through our Community Partners, the NF Endurance team is able to reach a broader audience and make new relationships in the endurance world. Jonathan has been racing for eight years and has decided to make his miles more meaningful by fundraising as part of the NF Endurance team. As an NF Endurance participant, Jonathan receives letters, videos, and cards from an NF Hero. Jonathan wrote “My NF hero is a seven-year-old boy from Kansas City with NF1 and Asperger's syndrome. I have a photo of him and a letter he wrote posted on my front door to give me a little extra motivation every time I head out for training. I am proud to be a Community Partner as well as an athlete for the Children's Tumor Foundation. I am a professional bike fitter and I am going to see to it personally that the entire NF Lake Placid team is fit perfectly to their bikes, which will help them reach their goals. I am really looking forward to this journey and to contributing to the Foundation. It certainly brings new meaning to a sport that has been a part of my life for so long.” To become an NF Endurance Community Partner, please email

The Children’s Tumor Foundation is pleased to announce the dates for this year’s NF Camp being held at Camp Kostopulos in Emigration Canyon, Utah. Now in its 17th year, NF Camp is a unique opportunity for teens and young adults, ages 12 to 21, facing the challenges of living with NF, to spend time with others who share similar issues, in a supportive, friendly environment. Camp gets more exciting every year with great activities and unique places to visit. Come join us this summer for a week of adventures, challenges, and friendship. Choose one of two sessions: Week 1: Saturday, July 13th – Friday, July 19th Week 2: Saturday, July 20th – Friday, July 26th

Camp tuition is $650 for the week, exclusive of transportation. Through the generosity of many of our supporters, tuition scholarships will be available again this year, and in cases of demonstrated need, the Foundation will also provide financial support for transportation to and from Salt Lake City. So mark your calendars, and start making plans. For more information about the camp and to download the application and scholarship request form, please go to or contact Patrice Pancza at Apply early – spots fill up very quickly, and you don’t want to miss out on this wonderful opportunity!!

NF News • Children’s Tumor Foundation | 9

national programs Sign Up for the Nashville, TN NF Walk Date:


The NF Walk Program: Supporting NF Research Every Step of the Way

April 21, 2013

The Children’s Tumor Foundation's NF Walk program hosted over 33 Walks, 5Ks, and Kids Runs in 2012. Because of the dedicated support of Walk organizers, committee members, volunteers, and participants, over $900,000 was raised for NF research. The Walk program would not be a success without your support – Thank you!

Public Square, Downtown Nashville


*Held in conjunction with the NF Forum!

NF Walk Donations Support Research Funds raised through the NF Walk program support Foundation grants such as Young Investigator Awards (YIA) at $100,000 each and Drug Discovery Initiatives (DDI) that range from $25,000 to $75,000 each. In addition, funds from the NF Walk program support the NF Clinic Network which helps provide up-to-date information and medical care for patients, as well as educational symposia for those affected by neurofibromatosis.

For more information: 212.344.6633 ext. 8543 or 8544

The Young Investigator Award is the Foundation’s cornerstone grant; it is designed to enhance the fundamental understanding of NF and its biology. In addition to establishing critical facts about the nature of the disorder, the YIA program introduces exceptionally talented scientists to the field of NF research. The Foundation has funded 103 YIA grants, and YIA awardees have gone on to receive 24 National Institutes of Health and Department of Defense grants. Many YIA recipients remain pioneers in NF research for years after receiving the award. Drug Discovery Initiatives test innovative theories and transform ideas into treatments, which can then be transferred from the lab to the patient. Now in its sixth year, 51 of these awards have been granted, and the DDI program has attracted the interest of industry, government, and other nonprofits, which collectively have invested more than $5.2 million in potential therapies that would not exist otherwise.

JOIN AN NF WALK IN YOUR AREA Spring April 20 - Staten Island, NY April 21 - Nashville, TN May 4 - Greenville, SC May 11 - Greensboro, NC May 18 - Tulsa, OK May 18 - Monroe, MI June 15 - Boston, MA

Summer June 22 - Philadelphia, PA June 23 - Cincinnati, OH June 29 - San Jose, CA July 28 - Portland, OR August 24 - Salt Lake City, UT September 14 - St. Louis, MO September 14 - Brunswick, ME September 14 - Biddeford, ME

Fall September 21 - Des Moines, IA September 22 - Addison Oaks, MI September 28 - Bowling Green, KY September 28 - Rochester, NY September 29 - Atlanta, GA October 5 - Dallas Fort Worth, TX October 5 - Mechanicsville, VA October 20 - Los Angeles, CA November 2 - Jupiter, FL November 2 - San Diego, CA

For more information, please visit, or call 212-344-6633, ext. 8543 or 8544. 10 |

YOUR STEPS SUPPORT A HERO Is there a hero you can support?

Grandparents Support NF Hero Jack Fink We would like to thank Steven and Jane Fink for travelling 1,114 miles from Philadelphia to participate in the Jupiter, FL NF Walk. Team Fink raised over $18,000 in support of their two-year-old grandson. Jack loves Lightning McQueen from the Pixar movie Cars, pulling every book off the bookshelf, and eating sushi. Jack also has NF1. Jack’s grandparents sent letters to all their friends and family to collect donations, and were one of our top teams at the Jupiter NF Walk. Thank you Team Fink! Celebrity Cruises Support NF Hero Brittney Clifton Every year, Celebrity Cruises selects a philanthropic organization to which to donate. In 2012, their Southeast and West regions chose the Children's Tumor Foundation because two of its employees' families are affected by neurofibromatosis; Scott Clifton's daughter, Brittney, was born with NF2, and Cindee Crain's sister, Cheri, lives with NF1. Their successful fundraising campaign raised over $16,000, mostly due to the motivating force of friendly competition. "We decided to make it a contest," said Scott, "Our Southeast and West sales regions formed two teams and competed to see who could raise the most money within the quarter's three month time period. It really boosted morale and unity within the offices!" Thank you Celebrity Cruises for supporting the NF Walk program.

Jack Fink

Brittney Clifton was born in 1991 with neurofibromatosis type 2 (NF2).Over the past ten years, Brittney has endured multiple surgeries to remove tumors in her spine, brain, and auditory canals. In 2008, Brittney was diagnosed with several cancerous spinal tumors (ependymomas) and has undergone chemotherapy and radiation treatments. Ependymomas are rare tumors accounting for less than 5% of all brain and spinal tumors. In 2010, Brittney underwent invasive spinal surgery to remove a tumor which had wrapped itself around her spinal cord. After removing the tumor, Brittney's spine was fused back together. She spent three weeks at Cedars-Sinai Medical Center and months in physical therapy to learn to walk again. Today, Brittney is completing her third year of nursing school at Seattle University, with the goal of working as a pediatric oncology nurse following graduation. Brittney wishes to thank everyone for their support, prayers, and encouragement. 5Ks and Kids Runs 5Ks and Kids Runs have become popular and enjoyable features of NF Walks. We encourage you to incorporate both in your 2013 events. Including a 5K or a Kids Run is a great way to involve everyone and provide a competitive environment for those training for marathons. Do you or someone you know want to help? The NF Walk team is looking for local volunteers to help organize and plan events, recruit participants, promote NF Walks in their communities, and help with day-of-Walk logistics. If you or someone you know is interested in volunteering, please email NF News • Children’s Tumor Foundation | 11

Meet The Board Children's Tumor Foundation Benefit Gala Breaks Fundraising Record Continued from page 1

Richard A. Horvitz, the evening's philanthropic honoree, is Chairman of Moreland Management Company and has made a lifelong commitment to improving the lives of children and young adults living with NF. He has served as Chairman of the Children's Tumor Foundation's Board of Directors and under his leadership, federal funding for NF research increased by $200 million. In addition, he has personally funded 11 Young Investigator Awards, illustrating the importance of supporting new research. At the Gala he reflected that years ago when NF was just being recognized, it was not always clear where research dollars should go, since at the time so much about the disorder was unknown. But all that has changed. "We know what we want to do. We know how to get there. We know the paths we want to take. We just need the resources," said Horvitz. Honoree Mr. Richard Horvitz

The evening's medical honoree, Dr. Kaleb Yohay, is a pediatric neurologist, NF physician, and Director of the Comprehensive Neurofibromatosis Clinic at the New York-Presbyterian/Weill Cornell Komansky Center for Children's Health. He has specialized in neurofibromatosis for over a decade, serving as a past Co-Director of The Johns Hopkins Hospital Comprehensive Neurofibromatosis Center, and publishing 14 papers on the subject. Dr. Yohay is beloved by his young patients due in large part to his philosophy of including them in discussions regarding their medical care. "The very first thing I do is talk to the child," said Dr. Yohay, "I think a lot of times there is a tendency to talk straight to the parent first when actually the child is the patient." This year's Children's Tumor Foundation Ambassador, Mr. Drew Leathers, was also honored during the Gala. Mr. Leathers, who has schwannomatosis, was diagnosed with NF at age 11. In 2009, after surgery to remove a tumor from his spinal cord, he became a quadriplegic, descended into unrelenting pain, spent 183 days at Johns Hopkins Hospital, and was confined to a bed for most of 2010. In 2011, treatments at Johns Hopkins led to his pain levels receding and the recovery of enough strength and movement to master a push wheelchair.

Honoree Dr. Kaleb Yohay with NF Hero Gina DiMartino

In his speech at the gala, Mr. Leathers stated, "The treatment I received was an investment in all of the patients who share my disorder, and in all of the families who share their burden. Our world will be forever changed because of the research that created this hope. But we are not done. Far from it! The opportunity is here, and tonight can offer a brighter tomorrow." With that, Drew was joined onstage by his brothers, Chad and Ben, who held his hands as he stood for the first time in three years. This emotional crescendo to the evening brought the audience to its feet, and emcee Justin Bell reflected in his closing remarks that the hope is to one day gather in celebration of a world in which effective treatments for NF have been found and people no longer suffer from neurofibromatosis.

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William Brooks Hometown: Frankfurt, KY Current Town: Belle Isle, FL Education: BS in Civil Engineering, University of Alabama Work with the Foundation: Within the Board of Directors, I serve as the Chair of the Government Affairs Committee, and am on the Volunteer Relations Committee and the Development Committee. I am also the Chair of Racing4Research, and the Race Director of the 2013 Cupid’s Undie Run in Orlando, FL. Favorite Memory with the Foundation: In January 2006, my family, including my wife, sons, and daughter Lilly Ann (who lives with NF1), accompanied a member of the CTF staff to the Rolex 24 Hours At Daytona, to meet with auto racing representatives concerning the possibility of raising NF research funds and awareness through the sport. The result of that visit is Racing4Research, CTF's successful national program led by Jill Beck. Every year, hundreds of CTF / NF families from across the country travel to Florida and cheer for our racecar. The Rolex 24 Hours At Daytona race is so much like the NF experience, an endurance event that presents challenges to our drivers and crew that they do not expect. Yet every year, they overcome those challenges; they may be battered, bruised, taped up, and hobbled, but they never quit until they make it to the finish line. One day they will win and so will we, in the battle against NF. Favorite Hobby: Who has time for a hobby? When we shut the doors at CTF because we have put ourselves out of business by developing a cure for NF, then I will find a hobby. Until then, I guess running in my underwear to raise research funds and NF awareness is my favorite hobby!

Meet The Staff The Power of the Pantsless: Cupid's Undie Run Raises Over One Million Dollars!

Pamela Knight Clinical Research Manager

Hometown: Philadelphia, PA Current Town: New York, NY Education: MS in Genetic Counseling from Sarah Lawrence College, 2012

"You don't have a permit for this! If I were you, I'd keep running!!!" yelled a police officer to Brendan Hanrahan, Bobby GIll, Tamara Forys, and Chad Leathers in February of 2010 as they led the firstever Cupid's Undie Run (CUR) around the U.S. Capitol building. Happening only in Washington, D.C. and attended by 650 people, CUR raised $10,000 for the Children's Tumor Foundation that year. This February, Cupid's Undie Run had over 9,000 participants in 18 cities (including Sydney, Australia) and garnered more than $1,000,000 for neurofibromatosis (NF) research. A funny event with serious results, Cupid's Undie Run is a "one mile-ish" fun run in which participants race in their underwear, outdoors, on Valentine's Day weekend. Although three out of the four CUR founders are located in D.C., this event has made an impact nationwide through the tireless efforts of its volunteer Race Directors and its newly-hired National Coordinator, Sarah Morley. The 2013 Cupid's Undie Run was sponsored nationally by Gimbal's Cherry Lovers Candy and Fathead, and featured on CNN, The TODAY Show, and Delta's Sky Magazine, as well as many local publications and television programs. Co-Founder Chad Leathers says, "It is simply awe-inspiring. These Race Directors pour their hearts into this event and the results are extremely impressive and greatly appreciated." This year's 18 events were organized by people with and without connections to neurofibromatosis. Greg Ruth, Co-Director of the Cupid's Undie Run in Minneapolis, said, "Even though we don't know anyone with NF, we saw an opportunity to help a lot of people who are enduring formidable obstacles. Hearing about their experiences, courage, and fortitude makes it very easy to put in 110%."

Work with the Foundation: My primary responsibilities include increasing the number of NF Registry participants, making the NF Biobank a reality, strengthening and broadening the NF Clinic Network, and helping to publicize the Children's Tumor Foundation’s efforts and accomplishments. I will also interpret scientific publications for a general audience, and coordinate initiatives that address the educational and social aspects of living with NF. Looking Forward To: Meeting clinic coordinators and patients, and being a part of the energetic, dedicated, and inventive team that is the Children's Tumor Foundation. Favorite Hobby: Reading

The Foundation is truly grateful to every volunteer and to all the Race Directors including: Karen Rabon and Krista Fredric in Austin; Hannah Thompson, Chris Griffith, and Alischia Linson in Atlanta; Katie Barry and Co-Founder Tamara Forys in Chicago; Amanda Petrak and the Cleveland Cavaliers in Cleveland; Justin Campbell and Carrie Skullie in Cincinnati; Sheila Cohoon, John Marcicky, and Quicken Loans in Detroit; Sarah Morley and her incredible team in Denver; Ben Leathers, January Clark, Kathleen Hatcher, and Kelly Mills in Los Angeles; Greg and Jessie Ruth in Minneapolis; Michael and Morgan Gaither in Nashville; Julie Pantoliano in New York; Bill Brooks in Orlando; Dan and Sandy Frenia in Philadelphia; Zach Bender and Leslie Peterson in San Francisco; Rebecca Zeller in Seattle; Heather and Pat Riley in St. Louis; Carrie Elton in Sydney, Australia; and, of course, Co-Founders Brendan Hanrahan, Bobby Gill, and CTF Chapter President Steve Lorberbaum in Washington D.C. Their efforts, dedication, and generosity were extraordinary and we cannot thank them enough. Many congratulations and much gratitude to all of the "cherubs" involved in Cupid's Undie Run 2013! To learn more or to get involved, please visit NF News • Children’s Tumor Foundation | 13

regional news

STORIES OF NF Siobhan Clarke

 Utah

Over 25 families braved inclement weather and snowy roads to attend the Third Annual NF Symposium in Salt Lake City on December 15, 2012. Included were seminars on neurological and bone issues as well as the latest research developments in NF1. Attendees also participated in a question and answer period with specialists in the field of neurofibromatosis.

Colorado 

On December 8, 2012, CTF's Colorado Chapter hosted their annual Gingerbread House Decorating Party in Denver. This event is always a big hit with kids, and over 50 people attended.

 Washington

Members of NF Endurance's Team Grace held a fundraiser at Blu Berry Frozen Yogurt in Spokane, in honor of NF Hero Grace Carney. Blu Berry Frozen Yogurt donated 20% of the evening's profits to the Children's Tumor Foundation. Team Grace fights NF by running the Portland Marathon every year.

Massachusetts 

The "For the Love of Celia Dinner Dance" took place on Saturday, February 2, 2013 in Wilbraham, MA. It was an evening of music, dancing, and fun that featured a silent auction and a "candy bar." This event raised funds in honor of eight-year-old Celia La Barbera who lives with NF2.

Remembrance of Cornelia Keller Cornelia F. Keller of Waterford, Va., loving mother and devoted friend, passed away January 16, 2013, at the age of 66. Cornelia lived with NF2 and was a vital supporter of research into treatments and management of neurofibromatosis. Her dedicated support of the Children's Tumor Foundation’s research programs helped fund groundbreaking advances including the discovery of the gene active in NF2, animal models that mimic the expression of the disorder, and preclinical research that is now being tested in clinical trials. In addition to her leadership role in advancing NF2 research, Cornelia was a human rights activist, philanthropist, conservationist, and 14 |

historic preservationist. She was a founding member of the Peter Paul Development Center in Richmond, and she was active in the formation of the Richmond Urban Institute. In the 1980s, she strove to reduce Cold War tensions through Peace Child International. Most recently, from her restored cottage in Waterford, Va., she promoted environmental conservation and historic preservation with the Journey Through Hallowed Ground Partnership, which she co-founded. Cornelia will long be remembered by her family and those that loved her. She will also be remembered by the NF community as a critical leader in the fight to find treatments and a cure for NF2. When an end to this disorder is found, it will be a part of her legacy along with the many other positive changes she made with her life. The Foundation shares its deepest sympathies with the Keller family and all those whose lives were touched by Cornelia.

"Looking at me one would think, 'Hey, there goes your average girl, a normallooking, free-spirited, strong, twentysomething woman.' It's true that I am free-spirited and a twenty-something, even pretty normal-looking, but I am far from average. "When I was four, I was diagnosed with NF1. I am legally blind in my left eye, and partially blind in my right eye due to an optic glioma. I also have a brain tumor next to my pituitary gland, bumps all over my body, and hundreds of freckles and café au lait spots. In my 28 years on earth, I have had 29 surgeries, all NF-related. Growing up was just awful. People were so mean to me because of the severity of my NF. I never really had friends because I shut myself out. I was so ashamed, strived to be normal, and did anything to be accepted. "That is, until I got on Facebook and started meeting others who had NF or knew someone with it. Since then, my life has changed. Not only am I now not ashamed, I have a whole new group of friends who can understand and/or relate to me. It's the best feeling ever. I no longer care that it took me 24 years to make friends from whom I don't have to hide anything. One of my best friends, Ann, also has NF1 like me and I am so grateful for her friendship and encouragement. She is the reason I wear bikinis; she popped my shame bubble! "Keep your eyes open because this is just the beginning. I am a short, tie-dye wearing, artist, metal-head, Irish, hippie chick who happens to enjoy bowling and making the world know about neurofibromatosis!"

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).

California 

On January 26, 2013, CTF's California Chapter hosted a Racing4Research race day party at BJ’s Brewhouse and Restaurant in Torrance, CA to watch the Rolex 24 At Daytona event and cheer on R4R's racing team.

 Rhode Island

The Ocean State NF Walk, which took place on September 16, 2012 with over 60 people in attendance, raised more than $12,000 for the NF Walk program. The 2013 Ocean State NF Walk will occur on May 19th in Providence, Rhode Island.

California 

On February 4, 2013, the FOX television show Bones aired an episode with a subplot involving NF. CTF's California Chapter hosted a viewing party for a few of the NF Heroes and their families that appeared in the episode wearing Children's Tumor Foundation t-shirts.

 Colorado

The Colorado Chapter of CTF would like to congratulate and welcome its new president, Shelly Pesta. The CO Chapter would also like to thank outgoing president Mark Ebel for his hard work and years of dedication to the Foundation.

California 

NF Hero David Groudas and his family and friends, collectively known as "Grudies Gang," participated in the San Diego, CA NF Walk on November 3, 2012 and raised over $1,500.

 Utah

CTF's Utah Chapter hosted numerous NF Heroes and their families for a Racing4Research viewing party in Salt Lake City on Jan. 26, 2013 to watch the Rolex 24 At Daytona race, R4R's premier event.

California 

CTF's California Chapter hosted a "Casino For A Cure" on January 19, 2013 in El Nido, CA and raised over $2,000 in honor of NF Hero Dakota Anderson. Special thanks to Brian Holloway for his Casino Company’s donations and support of this wonderful event.

Ohio 

The third annual Cruise-In for a Cure fundraiser was held in Kenton, OH in honor of NF Hero Bryce Gossard. The event featured a classic and hot rod car show, a live band, a Life Flight helicopter landing, fire trucks, games, raffles, an auction, clowns, a dunk tank, pony rides, and a variety of food stands. Plans for the fourth Cruise-In for a Cure event are underway.

New Children's Tumor Foundation Chapter in Alabama NF Moms Renie Moss and Nikki Robbins and CTF Volunteer Relations Coordinator Alison Désir are working together to form an Alabama Chapter of the Children's Tumor Foundation. An official "Neurofibromatosis Alabama-Children's Tumor Foundation" Facebook page has been established. Please "like" it in order to begin connecting with other NF families in Alabama and proposing ideas for the Chapter. This group will be working closely with the outstanding doctors and staff at the University of Alabama at Birmingham Neurofibromatosis Clinic, as well as CTF, to develop a calendar of NF related events and programs. If you are interested in being a part of the efforts to establish an Alabama Chapter of the Children's Tumor Foundation, please email

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 Mary Vetting, Creative Katherine Wu,

Foundation Staff John W. Risner, President Research Annette Bakker, PhD, Chief Scientific Officer 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 Lauren Fritz, NF Endurance Manager, Chicago Suzanne Cannon, NF Endurance Team Manager Alissa Marks, NF Endurance Manager, Marketing Bob Skold, NF Endurance Team Development 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, Program Coordinator Public Education and Patient Advocacy Simon Vukelj, Communications Director Mary Vetting, Communications Associate Katherine Wu, Design Associate Finance and Administration Judi Swartout, Chief Financial Officer Sarah Anderson, Accountant Eve Espinueva, Database Manager Monique Boucher, Database Supervisor Mohamed Amin, Database Assistant Rosa Amelia Perez, Project Coordinator Sarah Rosenberg, Executive Assistant Danielle Meyer, Tech Support, National Programs NF News • Children’s Tumor Foundation | 15

Non-Profit Org. U.S. Postage PAID New York, NY Permit #4238 95 Pine Street, 16th Floor New York, NY 10005

You Are Invited to the NF Forum!

For more information or to register, please visit, or contact Traceann Adams at 212-344-6633, ext. 6691, or

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The NF Forum is a weekendlong patient and family support meeting open to all people living with NF and their families. NF Forum attendees learn about the latest medical advancements in neurofibromatosis, get practical advice on NF-related issues, and learn about resources provided by the Children's Tumor Foundation.


NF News Winter 2013  
NF News Winter 2013  

The newsletter of the Children's Tumor Foundation