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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

A Night to Shine On November 10th, the Foundation's Annual New York City Benefit Dinner raised over $1 million. This is the largest event in the 32year history of the Foundation and speaks strongly to the advances we are making and the successes we have had. Alec Baldwin was truly the Master of Ceremonies as the evening’s host and we are so grateful for his suppor t of the cause. From assuming auctioneering responsibilities to chiding our Chairman (and his boss!), Alec commanded the room and helped us raise funds that will improve NF care. A big thank you and congratulations to our evening’s corporate honoree, Alexandria Real Estate Equities, our medical honoree, Dr. Mark Phillips, and our NF Ambassador Rose Match Suna. All have contributed significantly to improving the lives of those affected by NF

IN THIS ISSUE The Neurofibromatosis Year Ahead: What Can We Expect in 2011............. 2 Lastest Drug Discovery Initiative Awardees.............................................. 4 Give The Gift of Hope............................. 5 National Programs............................. 6 Regional News..................................... 11 Snapshots From The Evening................. 15 NF Forum 2011..................................... 16

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and we are a better Foundation because of their commitment to our cause. Also a big thank to all who were in attendance, your suppor t of the Foundation fuels our mission and fur thers our resolve. We hope you had a wonderful evening. The money raised will help fund clinical trials and preclinical testing to advance future clinical trials. It will help suppor t our NF Clinic Network which improves care for individuals with NF. And it will help ensure our annual NF Conference continues to bring together the best and brightest minds in research. The theme of the Benefit was “A Night to Shine” and we are confident that the light from the evening will help guide us toward treatments for NF. continued on page 15

Alec Baldwin with 2010 NF Ambassador Rose Match Suna

Foundation Names New President The Children’s Tumor Foundation is pleased to announce that George T. Orfanakos has been named the new President of the Foundation. Orfanakos will assume the role at the beginning of the new year. “George has been critical to the advances we have made in raising funds for NF research, promoting public awareness, and bettering clinical care for individuals living with NF,” said Stuart Match Suna, the Chairman of the Foundation’s Board of Directors. “His focus and dedication have helped the Foundation reach a larger audience and better more lives; we expect that this new, expanded role will mean even greater things as we work to find treatments and a cure for NF.”

Current President John Risner added "George has brought not only new ideas on how to fulfill our mission, but as importantly the planning and execution to accomplish them. I look forward to the progress he and his team will continue to make in our shared cause of ending neurofibromatosis." continued on page 14

Help Us Help You

Please take our one-time survey today and help us better tailor our materials to your needs. Visit Survey ends January 15! Through January 15, the Foundation is conducting an online survey of individuals affected by NF, family members, friends, researchers, clinicians, donors… really anyone who is involved with CTF or NF in any way. This survey is designed to help the Foundation better understand the NF community so that we may provide meaningful information, ensure proper clinical care, and assess the needs of the clinical and research communities. Please visit

NF News • Children’s Tumor Foundation | 1

research news

The Neurofibromatosis Year Ahead: What Can We Expect in 2011? The past five years have seen significant advances in neurofibromatosis research, and both NF1 and NF2 clinical trials are well underway – heartening news for those with NF. In 2006, we activated a “Strategic Plan for Neurofibromatosis Research,” focusing on key areas in need of funding in order to accelerate NF research to the clinic. The Strategic Plan recommendations were a result of i) analyzing the state of NF research at the time, and ii) an advisory summit of experts from NF research and clinical care, biotech and pharmaceutical companies, government and other Foundations. In January, CTF will review the accomplishments of the 2006 Strategic Plan and host another expert summit to identify NF research priorities for the next five years. Below, we summarize some of the progress made since 2006, and look ahead to some anticipated highlights of 2011.

Preclinical Drug Testing – Finding Drug Candidates for Clinical Trials In 2006, preclinical drug testing was identified to be a major gap in NF research. Today, it is CTF’s largest area of focus through our NF Preclinical Consortium (NFPC) and Drug Discovery Initiative (DDI) Awards. The major NF trials of the past few years had their origins in positive findings from preclinical testing – including Lovastatin for NF1 learning disabilities trials and both lapatinib and Rapamycin for NF1 plexiform tumors trials. CTF’s NF Preclinical Consortium took on a major challenge: to test drugs in parallel in models of different NF1 and NF2 tumors. Guided by an Advisory Board heavy in industry expertise, the NFPC Centers have conducted a sophisticated level of drug testing that, if positive, will translate to the clinic. NFPC has built up a growing pipeline of candidate drugs and established collaborations with companies including Novartis, Genentech and Avila Therapeutics. Building on these accomplishments, in November 2010 CTF announced the intent to support expansion (to seven sites) and continuation (for two more years) of NFPC with an addition $3.75 million commitment through 2013. This will be a competitive expansion: both new and existing Centers may apply. The DDI Awards program, offering funding for preclinical drug testing, continues to flourish. We have now funded over 35 DDIs, 2 |

an investment of $900,000. DDI success is measured by a few key parameters: whether industry collaborations are established (20 to date), whether followon funding is secured from NIH, DOD or elsewhere ($4 million to date), and whether the studies are published (18 to date). DDI has jump-started a number of ideas that are new and unusual, and at such an early stage they not likely secure funding elsewhere. DDI also helps investigators access drugs from industry through our

hope to see some of these ideas advancing to the clinic, and to support advancement of these projects to the clinic through our Advanced DDI Awards and Clinical Research Awards programs.

Clinical Trials – Where Are We and What’s Next? Today there is a pipeline of ongoing or planned NF clinical trials for almost all of the manifestations of NF1 and NF2, from vestibular schwannomas to learning

NFPC Centers have conducted a sophisticated level of drug testing that, if positive, could be translated to the clinic. DDI Toolbox. For example, through our ad hoc Schwannomatosis Awards program, we funded Dr. Larry Sherman (Oregon Health Sciences University) to develop the first mouse model of schwannomatosis pain, helped access appropriate drugs for testing from AstraZeneca and Pfizer, and through DDI Award funding we support the drug Dr. Larry Sherman testing. DDI Awards will continue into 2011. We

disabilities. These include large-scale, multicenter trials funded by the DOD Phase II NF Clinical Trials Consortium, and much smaller trials limited to one or two sites, such as those funded by the CTF Clinical Trial Award program. The Clinical Trial Award program was launched in 2007 offering $125,000 to support small pilot trials to advance new ideas emerging from preclinical drug testing quickly into small scale clinical trials. We received only a modest number of applications for this program, and funded only two Clinical Trial Awards. In contrast, we had many inquiries from the NF research community seeking funding for studies adjunct to clinical trials

research news – such as developing biomarkers to allow for early detection of drug response, utilizing imaging as a trial tool, and other resources that will improve future Dr. Scott Plotkin trial design and implementation. As a result in fall 2010 we remodeled the Clinical Trial Award program into $100,000 Clinical Research Awards. These may still fund a pilot clinical trial, but have a broader remit to allow funding of adjunct research studies. We have had a high level of interest in this program and look forward to announcing up to four Clinical Research Awards in early 2011. In retrospect, the Clinical Trial Award program was premature in anticipating a large number of pilot clinical trials as the preclinical research pipeline still needed to mature. The Foundation has responded to this need for funding to improve clinical trial design. Looking ahead to 2011, we anticipate that Clinical Research Award mechanism will continue to fill this role as well as funding pilot trials.

NF Clinic Network – Improving Care; Building a Patient Registry and BioBank In 2007, CTF’s Clinical Care Advisory Board set out to establish guidelines for what an NF Clinic should be and to recognize clinics meeting those guidelines by recognizing them as Affiliate Clinics of the NF Clinic Network (NFCN). Today, there are 43 NFCN Affiliate Clinics. As NFCN grows, our goal is to promote communication and sharing of information via the recently introduced NFConnect ListServ for clinics. Through annual reporting, NFCN is providing a picture of how NF care is delivered across the country, and how many patients are being seen. Looking ahead we hope that NFCN will provide consensus on NF clinical care issues, ultimately leading to improved care for individuals living with NF.

In 2011, NFCN will play a vital role in the launch of the CTF NF BioBank and Registry. Developed in partnership with Genetic Alliance, the CTF NF BioBank and Registry is the first national bank for NF. It will be piloted at five NFCN sites in early 2011 and we hope will be open NFCN-wide in mid-2011. An NF BioBank and Registry will be a vital link in the chain connecting patients to research. The BioBank will collect and store tumor tissues, blood and other samples donated from patient surgeries and will provide a valuable resource for researchers to discover new biological findings about NF that may lead to new drug targets and new treatments. The Registry will also provide a wealth of information. While it is exciting to see the number of clinical trials increasing, recruiting sufficient patients can be challenging, and this will only grow harder as the number of trials grow, and patients have more options. Also, there is a lot of enthusiasm now among patients to participate in trials but it’s possible this will

continuing to fund earliest stage research since new discoveries will identify the drug targets of the future. CTF continues to support early stage research – as well as encouraging new researchers into NF – through our longrunning Young Investigator Award program. The work of current YIA recipient Dr. Wei Li (Memorial Sloan-Kettering Cancer Center) to unravel NF2 merlin protein signaling was published in Cell in 2010. CTF also has a tradition of jump-star ting new areas of research, by responding quickly to new ideas with summits for researchers to develop priorities in that area, in some cases followed by special targeted funding programs. Finally, a key role that CTF plays is organizing summit workshops of leading experts in specific areas of NF research to drive the development of new ideas. In the past couple of years these summits have led to the publication of CTF-driven consensus papers authored by leading experts. In 2011, CTF will host

CTF has a tradition of jump-starting new areas of research ‘burn out’ especially if early trials are not successful. A Registry will help to identify patients for potential trial participation, and to notify them about pertinent trial opportunities. In particular a Registry will be a vital resource in encouraging biotechnology and pharmaceutical companies to support NF clinical trials as it is a clear indicator of the number of patients who are eligible for particular trials.

Discovery Research - The Source of New Ideas Every drug that makes its way to the clinic has its roots in discovery research. NF clinical trials are now well underway, but we cannot forget the critical importance of

summit workshops on bone dysplasia (February), schwannomatosis (June) and learning disabilities (June). Each of these summits will result in publication of consensus papers, and some of them result in new CTF funding mechanisms. For example, CTF has had a significant role in advancing schwannomatosis research over the past few years. Since the candidate schwannomatosis gene INI1/SmarcB1 was identified in 2007, CTF has invested $500,000 in schwannomatosis research, including the creation of the first mouse models of schwannomatosis pain, as described above. We anticipate funding further new schwannomatosis research on the heels of the June 2011 summit.

NF News • Children’s Tumor Foundation | 3

research news Latest Drug Discovery Initiative Awardees: New Models and Drug Targets to Find NF Therapies Since its inception in 2006 the Drug Discovery Initiative Awards program has funded more than 35 projects to accelerate progress to finding effective therapies for NF1, NF2 and schwannomatosis. Offering fairly modest dollar amounts of $15,000 and $30,000, the attraction of DDI for researchers is rapid review (many investigators have funding in hand within six weeks of application deadline, versus the more typical six months for government funding) and limited requirements – DDI will support good ideas even if there is no preliminary data. Importantly too, DDI links help to translate basic biological discoveries into research that could identify drug therapies.

could have a much broader application. In the DDI Award-funded study Dr. Kalamarides will examine the effects of the drug Rapamycin on meningioma growth. Rapamycin, a recently approved drug therapy for Tuberous Sclerosis, and already in clinical trials for NF1 plexiform neurofibromas, targets the cell signalling element mTOR which was recently shown to be abnormal in NF2 meningioma cells. This therefore presents a rational therapeutic approach that could rapidly advance to the clinic since the safety profile of the drug in patients is well understood.

DDI links help to translate basic biological discoveries into research that could identify drug therapies

A Zebrafish Chemical Approach to NF1 Pathogenesis Thomas Look, M.D., Dana Farber Cancer Institute Zebrafish have, over the past 20 years, cemented themselves as an invaluable part of researching the genetics underpinning of specific diseases. Unlike mice, zebrafish reproduce quickly and therefore zebrafish carrying specific genetic mutations – in this case, the Nf1 gene can be developed in a short time. These can be used to test drug therapies and in a short time figure out if that drug can restore normal tissue growth even in the absence of a functioning NF1 gene. This can serve as an initial ‘screen’ for drugs that may then be of value to advance to the mouse models where researchers can therefore acquire more detailed information about the likelihood of drug action in humans. Dr. Look’s group will use the NF1 zebrafish model to test an array of drugs already approved by the Food and Drug Administration, this means if any look promising they could advance quickly to the clinic.

Through this innovative approach, DDI has jump-started a number of exciting research ideas and helped investigators garner enough results to be able to secure larger grants from CTF or elsewhere. Our second DDI deadline of 2010 was in August. From a number of high quality applications four were selected for funding, two focused on NF1 and two focused on NF2.

In Vivo DDI Award

In Vitro DDI Award In Vivo DDI Award Preclinical testing of the mTORC1 inhibitor Rapamycin in NF2 meningioma mouse models Michel Kalamarides, M.D., Ph.D., House Ear Institute The Foundation would like to congratulate Dr. Kalamarides on his recent joint appointment with the House Ear Institute in Los Angeles. This will allow him to expand his translational research collaborations with Dr. Marco Giovannini and colleagues at HEI. For the first time this DDI Award brings a mouse model of meningioma to preclinical testing. Dr. Kalamarides has been carefully refining this model to be as representative as possible of the human state and therefore the most meaningful drug testing model. Meningiomas are the second major tumor type that affect persons with NF2. Clinically they are very difficult to manage, there are no effective drug therapies, and one-third will become malignant. In the general population meningiomas account for one-third of all brain tumors so drugs developed for NF2 meningioma 4 |

Small molecule screen to identify inhibitors of NF2- mediated Yap1 activation Morvarid Mohseni, Ph.D., Children’s Hospital Boston Dr. Mohseni’s research hopes to identify drug treatments for NF2 through a new approach: by focusing on the recently identified Hipposignalling pathway. The Hippo pathway was first discovered in the research model Drosophila (fruit flies) but homologous pathways were then found in mouse and human. Of particular interest within the Hippo pathway is the protein named Yap1 which is a downstream signalling element of NF2 protein in the cell and could be driving tumor growth. Dr. Mohseni hopes to intervene in this signalling pathway by testing an array of up to 100,000 small molecule compounds on human-derived schwannoma cells. This is a high throughput study, where responses can be rapidly monitored through a genetic-based ‘reporter’ which allows for rapid evaluation of such a large number of agents for a fairly modest cost.

research news In Vivo DDI Award Preclinical trial of translation read-through drugs in human NF1 Xenografts Margaret Wallace, Ph.D., University of Florida This research is a continuation of Dr. Wallace’s successful previously CTF-funded in vitro DDI Award. NF1 is a dominant genetic condition, which means that in a patient’s cells, there is one normal copy of the NF1 gene, and one copy which no longer works because of a mutation. If such a cell has random mutation event (called a second hit), the normal gene copy can also become mutated. These cells have virtually no functioning NF1 neurofibromin protein and as a result the tumors and other manifestations of NF1 can occur. About a quarter of all NF1 gene mutations are called ‘nonsense’ mutations. This class of drugs that will be tested has been effective in studies of mice with cystic fibrosis and Duchenne muscular dystrophy (due to nonsense mutations), and a drug, Ataluren, developed by PTC Therapeutics, is now in clinical trials for those conditions. When tested on cells from neurofibromas, read-through drugs help them make neurofibromin again. Here Dr. Wallace advances these findings to in vivo studies. Human-derived neurofibromas will be transplanted into mouse nerves, where they would normally grow, and treated with read-through drugs. Positive results would suggest that these drugs might be clinical candidates for NF1.


Congratulations Dr. Susan Lindquist, 2010 National Medal of Science Recipient



CTF Awarded 'Innovator' Presentation at Partnering For Cures 2010


12 Give the Gift of Hope Around the holidays, many people appreciate the opportunity to give a different kind of gift - one that can make a lasting difference in the lives of loved ones and other in need. In this difficult economic climate a thoughtful gift goes a lot further than an expensive one. This year, consider giving a gift to the Children’s Tumor Foundation in honor of family members and friends. For each gift of $25 or more that you give, we will mail your special honoree a card acknowledging your generous donation on their behalf. Simply visit the Foundation’s website at and click on “Donate Online” to make an “Honor Someone” donation. If mailing a check, please include your honoree’s name and address - and the holiday or other special occasion that you are celebrating.

Dr. Susan Lindquist of the Whitehead Institute has been awarded the National Medal of Science this week in a ceremony at the White House. Dr. Lindquist has been the recipient of a Children’s Tumor Foundation Drug Discovery Award, and she gave the opening Keynote Lecture at the 2010 NF Conference in Baltimore, MD in June. The National Medal of Science is given annually to a small number of select scientists by the National Science Foundation. The 2010 Award recognizes Dr. Lindquist’s overall focus on understanding how proteins fold, which could have broad reaching applications in medicine and biotechnology. Her neurofibromatosis-focused work has looked at heat shock proteins and their potential role in NF1.

The Children's Tumor Foundation has been selected to take the stage as an 'Innovator' at Partnering for Cures, the Milken Institute forum that aims to transform the medical research system and accelerate cures by providing a partnering venue for foundations, industry and investors. Partnering for Cures had its inaugural meeting in 2009 and we are honored to be a selected speaker for the second year in a row. CTF's 'Innovator Presentation' will showcase progress to date in our Neurofibromatosis Preclinical Consortium (NFPC), CTF's collaboration of six research centers conducting drug trials on models of different NF1 and NF2 related tumors. NFPC, CTF's biggest investment to date at $4M+, has generated a number of exciting findings to date and has established collaborations with Novartis, Genentech, Avila Therapeutics and other industry entities that we hope will advance progress to NF clinical therapies.

Tough Times Ahead for NIH Research Grants



At the American Society of Human Genetics meeting last week, National Institutes of Health head Dr. Francis Collins reported that NIH could now be facing a period where only 10% of all NIH applications will be funded. This would mark a historic low for NIH and a halfing of the current success rate of applications. NIH, whose budget for 2010 was $31 billion, is the leading funder of basic disease research as well as a major supporter of clinical research in the US. While foundations such as the Children's Tumor Foundation can afford to seed-fund new ideas in neurofibromatosis research, the majority cannot afford to support multi-million dollar grants over many years. It is absolutely vital for researchers to be able to transition to NIH funding to provide the financial backbone to keep driving forward with new discoveries and then translatiing these to clinical treatments.

NF News • Children’s Tumor Foundation | 5

national programs

All of us at the NF Endurance office want to wish you a healthy and happy holiday season. We suggest you eat a lot of cookies, ice cream, candy canes, turkey, and stuffing. Really. Go ahead, cram it in. We’ll be here for you in the New Year when it comes time to make that resolution.

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We’re all excited about 2011 and aim for it to be our best year ever. Please check our website at to see a complete listing of our schedule. Among others, we’re excited to add the Big Sur Marathon and the Alcatraz Triathlon to our schedule this year, along with another Race Across America Team and an expedition up Mount Rainier in Washington. And, as we travel the country visiting our established existing events, it really is like a big family reunion each year. NF Endurance is about finding out what is inside you. It’s about reaching beyond your expectations and accomplishing things you didn’t think were possible. It’s also about providing hope and inspiration for the millions of people in the world living with NF. This team is a perfect fit to honor the struggle and hurdles life throws at the NF family. As a rule, we are not athletes, nor are we scientists or researchers (in most cases). We’re moms, dads, brothers, sisters, uncles, aunts and friends of our NF heroes and we can run or ride a long way to bring attention to this dreadful and devastating disorder. A couple interesting statistics for your amusement. Last year, as a team, we received about 14,000 donations that averaged about $100 each. That’s about 38 per day, 365 days per year, or about $27,000 per week. How ‘bout that!! I think that’s rather impressive. Oh, here’s one more – as best as I can figure, about three times every hour there is another child born with NF. There are still no treatments and there isn’t a cure. We’ve a long way to go, my friends, but I’m glad I’m on this journey with all of you. It’s in you. It’s possible. Join us!

03/20 NYC HALF MARATHON New York City, NY

Images from the New York City Marathon

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national programs NOWN OPE

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moments of challenge – the commitment to train, the courage to set far-reaching personal goals, the physical effort and determination to finish – well this gets the attention of your friends and family. Add to the spirit of endurance the desire to RUN FOR A REASON and you have a powerful story to tell, a story of hope that people will listen to and support. Your success is not determined by your performance but rather by your passion, your desire to make a difference: you are striving to “be the change you wish to see in the world.” When you bike/swim/run for a cause, “It’s not the speed that matters but the purpose of the journey. The world, and the NF community in particular, need all the help we can give by way of cheerful, optimistic, inspiring thought, and personal example. NF Endurance offers this opportunity in a meaningful and fulfilling way. Let us help you “tell your story,” using our fundraising support and together the NF cause will be stronger. You will change lives, maybe even your own. - Bob Skold, Jr. (Bob works for NF Endurance in team communications and support. In October, he participated in the 2,500th running of the original marathon in Athens, Greece. In January, Bob will complete his 50th marathon running for our team in Yuma, Arizona.)

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INSPIRATION Participating in an endurance event with its sense of adventure and

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wanted to share some success stories in regards to fundraising. I never really thought I would do that well in fundraising and just always thought I could count on some close friends and family to make a donation and sort of always had low expectations and felt bad in asking for money. Let me tell you I was so wrong!! I put one of my letters in the mailbox of a neighbor who I had spoken with while walking with my daughter in the stroller. She took my letter and showed it to all of her other neighbors who I did not give a letter to. I received a $5,000 donation from a neighbor down the street whom I didn't even know! This neighbor also made another $5,000 donation this year. Never would have imagined in my wildest dreams this could ever happen. - Dan L.

My name is Janine and this is my first marathon. A little over four

years ago, I wouldn't have been able to walk 26 blocks yet I dreamed of running in this marathon. After losing 200 pounds and finding the dedication someone needs to train for a marathon, I think I'm ready (slow and steady is my motto). Along my journey, a dear friend introduced me to NF and the Children's Tumor Foundation. While I do not know anyone with NF, I felt a strong connection to the Foundation... life is full of possibilities for a kid with NF; I wanted to help someone realize his dream as I will accomplish mine tomorrow. Thank you for providing me with the opportunity to take part in such an amazing event. - Janine R. NF News • Children’s Tumor Foundation | 7

national programs

T he Racing4Research team would like to wish you and

your family a happy, healthy and safe holiday season!

NF Hero Inspiration A lmost 200 guests attended the inaugural Kevin Martin Charity Casino

Night in New Rochelle, New York on October 23 in support of Kevin’s Racing4Research fundraising efforts. The evening also marked the first local appearance of the promotional Racing4Research Porsche which provided a dramatic talking piece and photo opportunity for arriving guests. Kevin was proud to point out his name on the side of the car along with the names of many other local NF Heroes. While 12-year-old Kevin did not take to the casino tables, he did spend the evening meeting and greeting the many friends, family and community members who turned out in support of his efforts, including New Rochelle’s Mayor Noam Bramson, New Rochelle City Councilman Barry Fertel, and Westchester County Clerk Tim Idoni. Kevin also selected the winning raffle numbers as the evening drew to a close. It was truly an “evening of fun, food, friends and giving,” with just over $20,000 being raised through the contributions of attendees, sponsors and online supporters. Especially moving was a video about Kevin and his life with NF1. Many people commented that they had known of Kevin’s medical issues for a while, but until seeing Kevin’s video, they never really understood NF or realized all that Kevin has been confronted with since first being diagnosed at age two. They came away better informed about NF, and inspired by a boy who remains vibrant and enthusiastic about life, with an ongoing desire to give back to CTF and his local community.

More than 200 guests attended the Ladybug Benefit at the Glen Cove

Mansion on October 7 to help raise money for Racing4Research on behalf of NF Hero, Julia "Ladybug" Perfetti. With the Benefit Dinner growing momentum in the Glen Cove community, Team Ladybug raised almost $40,000 in its third year. The event was surely one to remember with dinner, cocktails, live music, fabulous gifts, and of course, the beautiful Julia Ladybug, enjoying the festivities and greeting guests who came out to show their support for Team Ladybug and Children’s Tumor Foundation. In a heartfelt moment from Julia’s parents Laura and Anthony Perfetti, Laura thanked everyone for their support and said, "Our Ladybug is realizing that neurofibromatosis is going to be part of her life – a difficult realization for an 8-year-old. Our fundraising has given us hope in this situation." Team Ladybug’s stellar fundraising effort and positive enthusiasm is a great asset to the Racing4Research team and our mission to find a cure could not be possible without them.

The big race is quickly approaching and our whole team is confident that 2011 will be the best year yet for Racing4Research - both on the track and off. We've partnered with the winningest team in Grand-Am GT racing, TRG, and we have a fantastic line-up of amazing drivers — including NASCAR driver Kenny Wallace — ready to pilot the CTF Porsche to its best finish yet (I'm thinking podium!) While we've got a great team on the track, we're even more proud of our team off the track — our amazing NF Heroes. The NF Heroes family continues to expand — we're expecting to host the largest group yet in Daytona 2011 — welcoming all new friends to the excitement and hope provided by the Racing4Research program. Racing4Research offers children and families something outside the oftentimes painful world of NF, a vehicle of hope if you will. Our goal is provide a positive experience for our families that can be carried with them throughout the year as they continue their struggle with NF. It's a once-in-a-lifetime experience for everyone involved, from the families to the team. In past years, we've watched as the drivers and team have undergone a transformation — it is clear that this is not just a race to them. It is about our NF Hero families and the hope we are building together. They come to race and find a cause. Join us. I promise the Racing4Research experience is like no other — you will come away changed, inspired, and ready to fuel the cure for NF!

MARK YOUR CALENDARS! 2011 Rolex 24 Hours at Daytona

January 29-30 • Green flags drops at 3:30 PM EST Live TV broadcast begins at 3:00 (check your local listings.) Stay tuned for news about our live webcast!

Visit to find a Race Day Party near you!

11/25 - 12/26 R4R PROMO CAR: Short Hills Mall, NJ

01/29 RACE DAY PARTY: Los Angeles, CA • San Diego, CA • Tucson, AZ

01/29 - 01/30 ROLEX 24 HOURS AT DAYTONA: Daytona, FL

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national programs Racing4Research Promotional Car Tours Tristate Area

Kevin Martin Charity Casino Night • NY

Wireless Zone Headquarters • CT 2009 NF Ambassador Chris Bryka and George Orfanakos with Wireless Zone's Board of Directors

Trane / Wallwork Group HQ • NJ CEO Matt Sheeleigh takes a spin

Buffalo Wild Wings / CT Post Mall 10% of all BWW sales on October 29th were donated to CTF

CTF Annual NYC Benefit • NY Paul Miller Porsche's Paul Gavel

Neiman Marcus / Short Hills Mall • NJ NY Jets kicker Nick Folk and long snapper Tanner Purdum

Grover Cleveland Elementary • NJ

The Racing4Research promotional car has been made possible through the generosity of:

NASDAQ • NYC NF Hero Dylan Halliwell

NF News • Children’s Tumor Foundation | 9

national programs

NFWALK Our NF Walk motto “Every step makes a difference” holds true for the more than 2,400 walkers that participated in the 18 NF Walks organized across the country in 2010. With your support this year we raised over $280,000 for NF research. To everyone that participated in an NF Walk we'd like to say a huge THANK YOU for your role in putting the NF Walk program on the map and making 2010 our strongest year. Your feedback has been invaluable in helping us improve the program throughout the year. We hope to introduce new guides and tools to help make your Walk simpler and more fun to organize in the coming year. As we look ahead to 2011 our goal is to maintain the momentum and double the number of walks throughout the country. We continue to rely on your support to make this goal a reality.

Jupiter NF Walk


On November 6 in Jupiter, Florida over 150 people showed up on a chilly morning for the first NF Walk in Florida. Between a 5K walk for NF, cupcake decorating, face painting, lots of delicious food and special guests like clowns and various cartoon characters no one wanted to go home when the day was over. Congratulations to the Brain Family who organized this walk in honor of their son Dylan, who lives with NF1, and all those who came out to support this NF walk that raised almost $5,000 for NF research!


San Diego NF Walk The Children's Tumor Foundation hosted the inaugural San Diego NF Walk on November 6 at the NTC Park at Liberty Station. The walk had over 140 walkers and 20 volunteers. The weather was beautiful and the view of the San Diego harbor was breathtaking. Outreach for Humanity shared their wonderful, energetic emcee Alan Bezeck. A Reason to Survive Inc. (ARTS) were there with an arts and craft table for the kids and local stores donated food, beverages, and raffle items. Local sororities, fraternities, cheerleaders, and a few Stormtroopers were on hand cheering on the walkers. The 2nd Annual San Diego NF walk will take place on November 5, 2011 at NTC Park. Check out more event photos on our Facebook page at "Neurofibromatosis California – Children’s Tumor Foundation."

Battery Park NYC NF Walk Raises $23,000

North Carolina

05/07 NF WALK NASHVILLE Tennessee

05/14 NF WALK LOS ANGELES California

05/21 NF WALK MONROE Michigan



The Children's Tumor Foundation was thrilled to bring, for the very first time, the NF Walk program to Battery Park City and share the moment with over 150 supporters. The day began with perfect autumn weather and visits from Cookie Monster to Dora the Explorer. The crowd fueled up with McDonald's apple pies, Dunkin' Donuts munchkins, and delicious Au Bon Pain coffee. The walk kicked off with some inspirational words and an energizing warm-up from award-winning Broadway choreographer Jerry Mitchell. As the sea of blue NF shirts made their way around Battery Park, the registration site was gearing up for the fantastic raffle to bring the NF Walk to a close. Raffle gifts ranged from an autographed NY Knicks Toney Douglas basketball to tickets to the new Broadway show Catch Me if You Can, and the lucky winners could not have been happier walking home not only with a great gift but also priceless memories.



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08/07 NF WALK POCONOS Pennsylvania



regional news

New York

On November 9, the Foundation’s 2010 Ambassador Rose Match Suna, her father, newly elected Chairman of the Board of the Foundation Stuart Match Suna, and NY Jets Kicker Nick Folk, along with 30 Foundation supporters rang the NASDAQ closing bell. The Foundation is grateful to NASDAQ, Nick Folk, the Match Suna family and all those who came out for their support and commitment to ending NF through research.

New York City, NY


The California Chapter hosted a Meet the Experts event on October 23 at Santa Clara Medical Center in San Jose. Pictured: Denise Carr; Diana Darcy, MS; Donna Wallerstein, M.D.; Robert Wallerstein, M.D.; Scott Rosethal, MS


It was a full house on November 6 at Blakely Hall in Issaquah where the Lynch family and a group of their friends hosted their very first fundraiser, ‘FuNFor A Cure’ to benefit their son and NF Hero 8-year-old Hayden Lynch. The event was a success and fundraised over $58,000 for NF research. The Lynch family, and their ten person committee, threw a fabulous Wine and Chocolate Evening where members of their community were able to show their encouragement for Hayden and their support for the Children’s Tumor Foundation. The Lynch’s put together a remarkable silent and live auction complete with a stay at an oceanfront condo in Maui and a luxurious get-away to the serene Halfmoon Bay in British Columbia. If fabulous gifts and delicious chocolates was not enough, a local Jazz and Blues band, Smoke and Honey, supported the cause and provided stellar music at the Lynch’s unforgettable fundraising event.

San Jose, CA


“Mummers Night for Michael / Strutting for a Cure” was an inaugural fund raising event hosted by the Zazula family and an overall wonderful night for NF awareness and raising funds for research. Held in South Philadelphia at the Mummers Museum, the event featured the support of local vendors and many local supporters who came out to help the Racing4Research campaign for Michael Zazula. The Zazula family was surprised and delighted to meet other NF families in the area who heard about the event and came out as well! The evening was a true success in every aspect and they could not be happier with how the event turned out. Plans are already in the works for next year! The Zazula’s are so happy and grateful for the community spirit and willingness to be part of something so special and important to their family and their son Michael. They are forever thankful to all who help to find a cure for NF! Pictured: Michael Zazula Jr.

Philadelphia, PA

NF News • Children’s Tumor Foundation | 11

regional news Florida

Orlando, FL

The Ehrli Family in Orlando outdid themselves once again by hosting their 7th annual Florida Golf Tournament at Bay Hill Country Club. A collection of 88 golfers were treated to a gorgeous, sunny day and raised more than $20,000 for NF research. Wireless Zone CEO Kevin Sinclair led the tournament off with great words of support and expressed Wireless Zone’s commitment to NF research. Arnold Palmer came out at lunch to say hello and show his support for all those living with NF. Pictured: Stephen J. Lewkowicz - Executive Vice President & CFO of Wireless Zone, Arnold Palmer, Ralph Lewkowicz


With the help of local mediums, psychics, and volunteers from the Spiritualist Chapel of Melbourne, Faye Eicholzer held a Psychic Fair FUNdraiser at the Holiday Inn Conference Center in Viera, FL on November 6. Two raffles prizes were given out every half hour so there were lots of winners! The grand prize was a romantic getaway at a bed & breakfast on Melbourne Beach. With a good turnout of vendors from area shops selling jewelry, essential oils, psychic paraphernalia, candles, books and tape, aura photography, tarot cards, henna art, and readings with several of the mediums. More than $1,800 was raised for NF research.


In October, the Markland family hosted a Haunt 4 A Cure - a Halloween costume party in Cleves, Ohio. Many people in the community got into the spooky spirit by going all out with their costumes to support the cause - the Chief of Police of Lawrenceburg, Indiana dressed up as Glenda the Good Witch and the Markland's dentist and friend rocked out an amazing Lady Gaga costume, both of their first attempts at women’s fashion. There was a hay ride throughout the grounds that was enjoyed by children and adults alike. Movies were playing, pumpkins were decorated, a costume contest, and about fifteen tables jam packed with silent auction and raffle items. More than $6,500 was raised through the $10 entry fee, raffles, silent auction, food/drinks, and costume voting. MUCH awareness was raised and Jill Markland was able to proudly announce that five years ago there were ZERO clinical trials but in June 2009 there were 28 clinical trials for NF treatments and that everyone in attendance has had a hand in making that happen while having so much fun and walking away with great prizes. Pictured: The Markland Family

North Carolina

Cleves, OH

Over 30 attendees spent their Saturday in Durham listening to the NF specialists from Duke University and the University of North Carolina at Chapel Hill. Special guest speaker, Dr. Stewart, from the National Institutes of Health provided insight on NF1 and personalized medicine. Heather Wray from the North Carolina Chapter made some great gift baskets to raffle and Suresh Nagappan, Sarah Jordan and their two beautiful children were on site for the day to promote their NF Walk in Greensboro. Thank you to all the doctors who presented and the NF Clinic at Duke for hosting this lovely day.


On a bright, sunny day in Oxford, more than 75 people, plus a university marching band, gathered together for an NF Walk. Raising awareness, raising funds for NF research, and bringing those together affected by NF was the purpose of the walk – and it was a great success. With an Elvis impersonator, Steve Stevens from 97.9 WVOK, Dora the Explorer, Ronald McDonald and many other extra guests – the NF Walk was truly a fun day. A big thanks to all the volunteers who came out to help and the local church who sponsored the cake walk which was especially delicious and helped raise funds for NF research. A great day had by all! 12 |

Workplace Giving Many employers throughout the United States have programs to match the charitable contributions of their employees. These programs can double and sometimes even triple your gift. Generally all gifts to the Foundation are eligible to be matched depending on your employers specific program. It usually only requires a simple form that should be sent to the Foundation along with your Membership dues or contribution. Check with your human resources or benefits office to find out if your employer will match your generosity. These gifts are extremely beneficial in supporting the work of the Foundation and are simple and convenient. For more information, please call the Development Office at 1-800-323-7938 or email us at info@

regional news Massachusetts

On October 24 nearly 200 people filled the lecture halls of the Simches Research Center at Massachusetts General Hospital for the annual Neurofibromatosis Symposium. This yearly NF Symposium alternates between Massachusetts General Hospital and Childrens Hospital Boston. The day long Symposium featured lectures, workshops, lab tours and discussion groups with Symposium attendees and presenters. Children’s Tumor Foundation was represented by Kathleen Berentsen and John Heropoulos who spoke with Symposium attendees and had a table featuring the Foundation’s National Programs and educational materials.

New Hampshire

CTF volunteers of New Hampshire met on November 16 to plan their second fundraiser. This newly established affiliate sponsored a successful spring fundraiser this year and is busy organizing another event for this upcoming year.

Boston, MA


Over 200 people filled the hall of St. Cecilia’s Church in Wilbraham, MA on October 16 for the 3rd Annual Oktoberfest to benefit the work of the Children’s Tumor Foundation. The Oktoberfest is sponsored by Anita and Larry LaBarbera and a group of dedicated volunteers who come together to make this event a terrific success in the name of their lovely daughter NF Hero Celia La Barbera. The committee, and other volunteers, work tirelessly to offer an evening of friendship, fun and loving support. The Oktoberfest features home cooked German foods, a silent auction and raffle. A spirit of love and friendship filled the hall as everyone came together to support the mission of the Children’s Tumor Foundation by raising more than $20,000. Pictured: Celia LaBarbera

Wilbraham, MA

Plans are under way for the national NF Walk Program to be extended into Rhode Island and Vermont. Currently, Massachusetts and Maine are sponsoring successful walks every year. If you are interested in helping bring this powerful tool for NF awareness and fundraising to these additional states in the New England Region please contact John Heropoulos at


The Affiliate hosted a golf tournament on Saturday, September 18 in honor of NF Hero Kaleb Grigg.


The 2nd Annual Brunswick NF Walk was held on the Androscoggin River Bicycle and Pedestrian Path and attended by 50 people, including 4 families directly affected by NF. Familiar faces joined many new faces - all were warmly welcomed. Due to the generosity of the walkers and the generosity of their families, friends and co-workers the Walk raised over $6,400 through pledges, in-kind donations and corporate match! There was an overwhelming sense of doing something meaningful by coming together to raise awareness about neurofibromatosis and the Foundation. Plans are being made for next year’s Walk to raise more awareness and more funding to help find treatments and a cure for NF. The 3rd Annual Brunswick NF Walk has already been scheduled for Saturday, September 17, 2011!

Brunswick, ME


Over 200 people gathered in West Des Moines on a beautiful fall day. There was food and fun and so much NF connecting. A big thank you to the NF Walk committee: Sheila Drevyanko, Kori Ensley, Carla Hinrichs, Kathy Van Gilder, Jodie Warth, Kim Walker, and Parker’s Team. A special thank you to all those who fundraised and helped us surpass our goal in raising over $13,000 for NF research. Plans have already begun for next year’s Walk!

NF News • Children’s Tumor Foundation | 13

foundation news Foundation Names New President Continued from page 1

Orfanakos is the Executive Director of the Foundation, having joined as Vice President in the spring of 2006. Orfanakos has initiated numerous programs including Racing4Research, a fundraising program centered around the Rolex 24 at Daytona, and the NF Forum, the country’s first national NF symposium for patients and families affected by the disorder. For 12 years prior to joining the Foundation, Orfanakos was a pastor in the Greek Orthodox Church serving parishes in New York and New Jersey. He holds a B.A. from Hellenic College in Brookline, Massachusetts and an M.Div. from Holy Cross Greek Orthodox School of Theology. Orfanakos has been highlighted in the New York Times, the Hallmark Channel and in many additional media outlets. Orfanakos will replace John W. Risner, whose fiveyear commitment to the Foundation expires at the end of the year when he will return to an advisory role. Orfanakos is the third president in the Foundation’s 32-year history.


y the time you read this we’ll be close to year’s end. We’ll also be close to the end of an era that has changed my life and the face and the direction of NF research in the world. John W. Risner has been the President of the Children’s Tumor Foundation and my boss for the past six years. I’ve watched John navigate the troubled waters that made up and make up the NF landscape. The politics, the scientists, the Clinic Network, the Foundation constituency, the Board of Directors, and most certainly, those of us who collect a paycheck from this Foundation, have all contributed to the creases in his face and the receding hairline we’ve witnessed over the past six years. Ending neurofibromatosis through research is not an easy task by any measure, and under his steady leadership and guidance, we’ve come such a long, long way. It’s been an honor to work with John and to witness the transformation that has occurred to the Children’s Tumor Foundation under his leadership. We will carry on, and I’m excited and hopeful about working for our new boss, George Orfanakos. Change is sometimes a difficult pill to swallow, but I’ve watched George for the past few years and I know we will make John proud. There are well over two million people in the world for whom John has provided hope and inspiration for a better future... a future with treatments and maybe even a cure for NF. For those two million, and for the one who has me wrapped around her little finger, I thank John from the bottom of my heart.

Steve Kendra

14 |

transition As you've seen in these pages, after a national search conducted

by our Board of Directors, George Orfanakos has been named the new President of the Children’s Tumor Foundation effective January 4, 2011. This leadership transition has been long planned. When I assumed the President’s role in 2005, after the retirement of our previous president, Peter Bellermann, I gave the Board a five-year commitment. While I am proud of the advances we have been able to make in all areas of NF in the last five years – not just NF research, but also in public awareness, patient support and NF Clinic’s - I am very excited with the future potential of our Foundation under George’s leadership. I have served as President, and you likely are reading this letter, because of our shared commitment to do everything we can advance progress in neurofibromatosis research. Probably the best thing I have been able to do here has been to hire great people that are committed to our mission. George, who has done an excellent job as Executive Director, typifies this, but he would be first to admit that our results have been due to the team we have brought together. Our Chief Scientific Officer, Dr. Kim Hunter-Schaedle, Chief Financial Officer Tom Malone, Sr. Vice President Steve Kendra, National Program Director Traceann Adams, and Communications Director Garrett Gleeson have all brought new ideas to address the challenges we face, hard work and dedication translate these ideas into action, and have engaged all of our staff in working together to launch, measure and review the many new programs implemented in the last five years. All of this is towards one vision – ending NF. Our research program is designed to build a pipeline of promising candidate compounds that can be brought into clinical trials for all the complications of NF. We have made great progress, and now embark on more fully engaging biotech and pharmaceutical companies to undertake the Phase II and Phase III trials needed to develop these compounds into FDA approved treatments. To accomplish this we need industry expertise on staff, and I have every confidence that George and Kim will be able to add this experience. Thank you to all of you who have been so supportive of the Foundation. I have seen many changes in the NF landscape in these last five years, but I have never been more positive on our work than I am now. Please know that I will continue to do everything I can for this Foundation, and trust that together with your continued support, we will achieve our shared goal of ending neurofibromatosis. - John W. Risner

foundation news 2010 Annual NYC Benefit Dinner:

a night to shine

snapshots from the evening

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). The mission of the Children’s Tumor Foundation, Inc. is to improve the wellbeing of patients and families affected by NF1, NF2, and schwannomatosis. The Foundation sponsors scientific research aimed at finding the causes and cures for the neurofibromatoses, promotes the development of clinical activities, works to create public awareness and provides patient support services. The Children’s Tumor Foundation is a founding member of the International Neurofibromatosis Association. Editor Garrett Gleeson, Creative Jill P. Beck,

Board of Directors Stuart Match Suna, Chairman Bruce R. Korf, M.D., Ph.D., Chairman, Medical Affairs Committee Allan E. Rubenstein, M.D., Director of Medical Affairs Emeritus Daniel Altman, Vice Chairman Laura Ganio Bona William Brooks Colin Bryar John Catsimatidis Suzanne Earle, Chairwoman Emeritus Mark Ebel Daniel Gilbert James. F. Gusella, Ph.D. Richard Horvitz Linda Martin, Secretary John McCarthy, Treasurer Steve McKenzie Denise Ogle Jason Pontin Robert Schaffer Tara Skirzenski Rachel Tiven David Viskochil, M.D., Ph.D. Nate Walker, Vice Chairman Peggy Wallace, Ph.D.

T hank You

95 Pine Street, 16th Floor New York, NY 10005 800.323.7938 | 212.344.6633 212.747.0004 F The Children’s Tumor Foundation is a 501(c)(3) non-profit organization and all donations are tax deductible. NF News • Children’s Tumor Foundation | 15


95 Pine Street, 16th Floor New York, NY 10005


01/29 FLORIDA: Racing4Research Rolex 24

06/11 WYOMING: NF Conference

07/29 MINNESOTA: NF Forum

16 |

NF News: Holiday 2010  

The 2010 Holiday Issue of the Children's Tumor Foundation's Newsletter

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