Banner Year for DTRF Grants
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This past year has been a banner year for DTRF thanks to the fundraising efforts of hundreds of people, as well as large donations by several generous supporters. Thanks to all of you, our efforts to find a cure are accelerating rapidly. We're happy to note the projects below that we are currently funding-research by the top scientists in the world in this field.
To all of you we say -- please consider joining the cause and holding your own fundraiser. We are here to support you 100% in your event. Lynne Hernandez is specifically dedicated to developing and supporting fundraisers around the country. Please call Lynne to get started! It's easier than you might think.
And know that your efforts and
donations do make a difference
- we will find a cure!
Jeanne Whiting, President and Marlene Portnoy, Executive Director
2013 Grants
- Benjamin Alman, MD, Duke University, with collaborators Robert Maki, MD, PhD, Mt. Sinai Medical Center; Alexander Lazar, MD Anderson Cancer Center; and Alessandro Datti, PhD, Samuel Lunenefeld Research Institute, "Collaboration for a Cure: Identifying new therapeutic targets for desmoid tumors." This project will screen libraries of over 100,000 available drug agents to identify those which inhibit desmoid tumor cell growth but do not affect normal fibroblast cells.
- Matt van de Rijn, MD, PhD, Stanford University, "Next generation sequencing approach to desmoid tumors."
- Justin M. Cates, MD, PhD, Vanderbilt University, "Genetic determinants of local recurrence in desmoid-type fibromatosis."
- Mrinal Gounder, Memorial Sloan-Kettering Cancer Center, "A Phase III, double blind, randomized, placebo-controlled trial of sorafenib in desmoid tumors."
- Raphael Pollock, MD, Ohio State University Comprehensive Cancer Center, "A rational search for anti-desmoid therapies," investigating the molecular driving forces behind the development and progression of desmoid tumors.
- Aaron Weiss, DO, Maine Medical Center, "Deregulated mTOR in desmoid-type fibromatosis," a pilot study examining the role of sirolimus in the treatment of children and young adults with desmoid tumor that is deemed likely to recur following resection.
- Chiara Colombo, MD, Fondazione IRCCS Instituto Nazionale dei Tumori, "A high throughput genome study to identify predictors of aggressiveness in patients with sporadic desmoid tumor who undergo a wait and see approach."
- Nancy L. Cho, MD, Brigham and Women's Hospital, "Targeting Hyaluronic Acid in Desmoid Tumors."
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Desmoid Tumors are Featured
on The Doctors
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 Desmoid Tumors received more national exposure in a recent episode of The Doctors television show. The episode featured Casey Sonke, a desmoid tumor patient who has since raised over $50,000 for DTRF. The Doctors website tells Casey's story as follows: "About a year ago, Casey started experiencing the all too common symptoms of acid reflux. Married to her college sweetheart, Jeff, and mother to a 3-year-old, Casey says she knew something just wasn't right in her body. A few visits to the doctor's office later, and Casey had learned that she was pregnant and had a large soft tissue tumor, known as a desmoid tumor, growing in her abdomen. The tumor became intertwined with an artery that provided blood supply to her bowel and the placenta of the growing fetus, causing the mass to grow significantly larger. Casey and her husband then made the difficult decision to end the pregnancy and start fighting the inoperable tumor." See the clip of the episode � Casey's physician, Dr. Neeta Somaiah of the Sarcoma Medical Oncology Center, explained how a desmoid tumor develops and the potential health risks they pose. She said, "Though it appears benign in histology and lacks the  ability to metastasize - that is, spread to other organs - it can be very aggressive locally," explains Dr. Somaiah. "It can invade vital organs. It can cut off blood supply. It can grow pretty big and cause pain, and it also has the tendency to recur repeatedly." The episode emphasized an important take-away from Casey's story- to trust your instincts. "Always, always listen to your body," was the advice given that many desmoid patients can relate to.
Casey has been very open with her story to help others. She has now undergone six rounds of chemotherapy and has seen the tumor shrink from watermelon-sized to the size of a grapefruit. She stays active and continues to play tennis with her husband. DTRF is also grateful that she has been active in fundraising! "When I was told that the life expectancy for a patient like myself is only 5 years, I looked at my doctor and asked, 'How can I change this outcome?' My doctor said 'money needs to be raised so more research can be done to find a cure.' I left that appointment and immediately knew what I had to do. I emailed DTRF on June 10, 2013 and let them know my goal was to raise $50,000 before the race in September. I am happy to report that my goal was met and I can always look back knowing my efforts helped DTRF get closer to a cure." Casey concludes, "DTRF is the light at the end of a very dark tunnel. When you google Desmoid Tumors the first site that pops up is the Desmoid Tumor Research Foundation. As a patient it is my lifeline. When I was first diagnosed in 2012, it provided me with the answers to so many of my questions and my family's questions. I believe it will one day save my life as well as the lives of many other desmoid patients." |
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Need Travel Help?
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Financial Assistance for Travel to
Medical & Research Facilities
There are many wonderful organizations whose sole mission is to help fund patient travel to medical institutions.
We recently had conversations with the following organizations that provide free air transportation to/ from medical centers:
Angel Flight East (215-358-1900) is available to residents from Maine to Virginia and as far west as Ohio, who need to travel anywhere within the U.S., up to a maximum of 1,000 miles. However, if you do not live in this region they work with other flight networks to make the connection. This service is a way that you can visit the experts as well as travel to NIH for the desmoid clinical trial (as the first time is not covered) at no cost to you. They also told us that they will fly patients to Philadelphia for the patient meeting!
Corporate Angel Network (866-328-1313) is available for patients needing to travel for treatment, consultation, clinical trial or checkup. Patients can be accompanied by a family member/ companion. Financial eligibility is not required.
Mercy Medical Airlift (888-675-1405) can provide either air or ground transportation services for patients.
Angel Flights at NIH (301-451-9646) program of Mercy Medical Airlift, can provide flights for clinical trials at NIH. Financial need is imperative, but, in most cases, patients can bring an escort with them.
Lifeline Pilots (800-822-7972) will transport patient residents living between the Rocky Mountains and the East Coast. They will travel anywhere within the U.S., up to a maximum of 1,000 miles. Flights to our Patient Meeting would be considered "compassion"flights, so are not a top priority, but definitely worth requesting.
"I called them on a Wednesday, hoping to get from Pittsburgh to NYC by Friday am, in order to see Dr. as soon as possible. By the end of the phone conversation (giving all of the pertinent information to Corporate Angel representative), they had us booked on a round trip flight, transportation to and from....the Corporation we ended up flying with was the BEST experience you could imagine from the receptionist, to the pilots, to upper management." - Hillary Carrozza
For more information, call the National Patient Travel Help Line at 800-296-1217. Also, For more information on other flight services and other patient support services, visit our website.
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Assessing the Risk of Desmoid Tumor Recurrence
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 At our fall Patient Meeting, we were enlightened on the subject of assessing the risk of desmoid tumor recurrence after surgery in a presentation by Aimee Crago, MD, PhD. Dr. Crago is Assistant Attending Surgeon at Memorial Sloan-Kettering Cancer Center.
Historically, a recurrence rate of 20-50% has been reported in desmoid tumors treated with surgery. This leaves physicians with the difficult task of evaluating which patients are the best candidates for surgical versus medical managements. Dr. Crago reports, "We concluded that clinical risk factors can predict recurrence of a desmoid tumor after surgical resection."
Her research team developed a nomogram, i.e., a graphical calculating device, to predict the risk of recurrence after surgery. The nomogram can assist physicians in counseling patients regarding treatment options.
Dr. Crago explains, "To determine who is most likely to recur, we examined outcomes in 495 patients following surgery. After 5 years, 31% of these patients experienced a recurrence. To accurately assess risk, we integrated size, age and tumor site into a nomogram which can be used to estimate each individual patient's risk of recurrence after surgery.
"At our institution, for patients with high risk of recurrence, we often recommend a trial of alternate therapies such as sorafenib because aggressive surgery may not benefit patients if they will recur rapidly."
As examples of Dr. Crago's findings, the nomogram reveals the following:
- Patients with large, extremity tumors have a high risk of local recurrence and may benefit from systemic treatments as first line therapy as compared to those with abdominal wall tumors who are almost always cured with surgery.
- Patients with extremity and chest wall tumors had increased risk of recurrence as compared to patients with abdominal wall tumors (rectus sheath tumors) who were cured with surgery in over 90% of cases. Based on the nomogram, a 35-year-old woman with a small rectus sheath desmoid tumor has less than a 10% chance of recurrence.
- The younger the patients, the more likely they were to recur and the larger the tumor, the more often it was associated with recurrence after surgery.
Interestingly, Dr. Crago reports, "We did not see an association between microscopically positive margins after surgery and risk of subsequent recurrence." Dr. Crago concludes, "Future studies will examine the role of genetic markers in improving our ability to predict risk of post-operative recurrence."
The Powerpoint from Dr. Crago's presentation can be found here. Dr. Crago's office at MSKCC can be reached at 212-639-4807.
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2013 Wine Tasting Fundraiser Raises $170,000!
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Comedienne and media personality Rosie O'Donnell was the honored guest at our May 17, 2013 fundraiser in Piermont, New York. Rosie delighted the crowd with a stand-up comedy routine. Then she stunned everyone by offering to match all donation proceeds for the event. One by one enthusiastic donors stood to pledge donations which will go to help fund our Collaboration for a Cure drug screen project. With the match, a total of about $170,000 was raised- a huge jump-start to our fundraising campaign for this million dollar project.
Rosie shared both the lighter side and the poignant drama of the diagnosis of her wife Michelle's desmoid. "With a rare disease that only about a thousand people get every year in the United States, what were the odds of a celebrity being one of them?" she mused. But she noted that the positive side of this difficult journey is that she can use her celebrity to help raise funds for research for a cure so others will not have to suffer as Michelle has.
Rosie and Michelle graciously stayed another hour to greet people and speak with anyone who wanted to meet them. It was an evening of delicious food, wine and the best of company, as well as great feelings that come from helping a good cause.
SAVE THE DATE:
This Spring's event: May 13, 2014, 7:00-10:00 pm at the Crowne Plaza, Suffern, NY. "An Evening of Food, Wine & Song" featuring Broadway's Up and Coming with a special performance by Rosie's Theater Kids Come join the fun! Purchase tickets here.
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Welcome Lynne Hernandez
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 DTRF welcomes Lynne Hernandez in her new role as Director of Events, Operations and Outreach. In 2012 and 2013, Lynne brought her years of race management experience to act as Race Director for Running for Answers 5K and Kids' Fun Run. Moving forward, her role will encompass a number of things, but most importantly, she will be overseeing and supporting events throughout the U.S. in support of the Foundation. Contact Lynne to brainstorm about different ways you can plug into DTRF. Whether you'd like to create an event, volunteer, or share a special skill with us, she'd love to talk to you about the possibilities. Lynne can be reached at [email protected] or (215) 760-3977.
She says, "I'm inspired everyday, working with so many caregivers, patients and other volunteers who are empowered and energized by creating awareness and playing an active role in finding- and funding a cure."
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NCI Phase 2 Clinical Trial
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In November, we reached out to you about a Phase II trial funded by the National Cancer Institute (NCI) in Bethesda, MD. Dr. Shivaani Kummar, M.D, presented this study at our 2013 Patient Symposium in Philadelphia. Read more about the study in her Patient Meeting presentation here.
Since the beginning of the trial a number of patients have enrolled. The trial is still open and accepting up to 17 patients. Contacts for this trial are:
Ramya Parthasarathy: (301) 594-4949
Janelle Bingham: (301) 435-2715
Lemual Clayborn: (301) 451-0992
Visit the clinical trial page here.
There is no charge for medical care received at the National Institutes of Health (NIH) Clinical Center. Patients will be responsible for travel costs for their initial screening visits. In most cases, once patients are enrolled in a trial, the National Cancer Institute (NCI) will pay the transportation costs for all subsequent trial-related visits for patients who do not live in the local area. In addition, these patients will receive a small per diem to help offset the costs of meals and lodging if they are being treated as outpatients.
To find out if you are eligible for free travel to participate for clinical trials, or other medical treatments, please visit our Patient Resources or contact the National Patient Travel Help Line at 1-800-296-1217.
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An Inspiring 2013 Patient Meeting and Running for Answers
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Desmoid tumor patient Lynne Parks summed up our recent Philly weekend the best: "We had another amazing DTRF weekend full of wonderful people and information. As always, new bonds were made and new support given. It's validating and proactive. Patients, caregivers, organizers, volunteers, founders, doctors, sponsors, attendees, contributors and any combination thereof! We raised over $370,000 for research! We will find a cure!"  Patient Meeting 200 patients and their families and supporters gathered for a weekend of new information, new connections and hope at the 7th annual DTRF Patient Meeting held on Saturday, September 28 from 12:30-6:00 pm at the Sheraton Downtown Philadelphia. Our distinguished speakers included Aimee Crago, MD, PhD (Memorial Sloan-Kettering Cancer Center); Tony Ferlenda (CEO, Sarcoma Foundation of America); Suzanne George, MD (Dana Farber Cancer Institute); Mrinal Gounder, MD (Memorial Sloan-Kettering Cancer Center); Robert G. Maki, MD, PhD (Mount Sinai Hospital); Matt van de Rijn, MD, PhD (Stanford University Medical Center). Subjects discussed included: important overviews of current and promising new systemic treatments for desmoid tumors; surgery vs. wait-and-observe; the development of a prognostic nomogram for prediction of tumor recurrence; the continuing search to find genomic predictors of tumor behaviors; new technology that allows researchers to use paraffin-preserved tissues instead of needing frozen tissues (contact DTRF to donate yours!); information about the Nexavar trial being conducted in 100 hospitals nationwide; and, information about two desmoid clinical trials run by the National Cancer Institute's rare tumor iniative. We also loved hearing from Matt Zechmann and former Philadelphia Eagle Kevin Reilly who shared their desmoid tumor stories and inspired us with their courage, positive outlooks, and humor. Broadway entertainment from tony- nominated singer Orfeh and pianist Steven Jamail brought a bright mood at the end of the day. A wine and snacks hour allowed for closer connections and commiserating as we could share with each other and also ask informal questions of the physicians and researchers. It was an inspiring day for all as we became better informed and uplifted for our journeys ahead. Running for Answers The Patient Meeting was followed by our 4th annual Running For Answers. About 700 runners joined in the 5K race/walk and the Kids Fun Run through Fairmont Park on a beautiful sunny day. Scott O'Neil, new CEO of the Philadelphia 76ers, built enthusiasm with his rousing introductory remarks. Rosie O'Donnell, whose wife Michelle was diagnosed with a desmoid tumor over a year ago, contributed her characteristic humor to lift our spirits and inspire us on. Former Philadelphia Eagle, Kevin Reilly, ran several pre-race PSA's on local tv and radio stations. Interviews by local news media brought publicity and increased awareness. In our highest-grossing fundraising effort ever, the event raised over $370,000 for desmoid tumor research!! This met our goal of funding the first year of the Collaboration for a Cure research project that will start this fall (more info at www.dtrf.org). The funds were hard-earned by scores of patients and families who ran their own fundraising campaigns through their Firstgiving pages. You all came through when we needed you most to get this critical project underway! A thousand thank you's! This incredible weekend always leaves us renewed and invigorated in our mission to fund research and keep moving toward new treatments and an eventual cure. Thanks to everyone who donated and participated in any way! We hope you all plan to join us in Philadelphia on September 27-28, 2014. Save the date! For more information, visit runningforanswers.com.
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