UPDATE: NF Clinical Trials for the Summer 2014 Quarter

We’ve updated the NF Clinical Trials section of our website via clinicaltrials.gov. Studies that have recently been added include:

Internet Support Group for Parents of a Child With Neurofibromatosis Type 1Cabozantinib for Plexiform Neurofibromas (PN) in Subjects With NF1 (16 Years+)MEK Inhibitor PD-0325901 Trial in Adolescents and Adults With NF1

Study of Imatinib Mesylate in Neurofibromatosis Type I Patients Aged 2 to 21 With Plexiform Neurofibromas

Transformation of Plexiform Neurofibromas to Malignant Peripheral Nerve Sheath Tumors in Neurofibromatosis Type 1

A Trial of Ganetespib Plus Sirolimus: Phase 1 Includes Multiple Sarcoma Subtypes and Phase 2 MPNST

Ophthalmological Screening and Follow-up of Optic Pathway Gliomas in Children With Neurofibromatosis Type 1

Clinical trials are an exciting and important opportunity for people with NF1 and NF2 to make a difference in their own lives as well as the lives of others affected with NF1 and NF2. When you participate in a clinical trial or study you are:

  • Receiving the most advanced care.
  • Giving to future generations of people living with NF1 and NF2.
  • Helping to change the way we practice medicine.

If you are interested in learning more about the importance of participating in clinical trials or about clinical trials in general, please visit NF Clinical Trial Facts.

Courtney’s Corner: Making Moments Matter

Club NF Cooks

As the school year begins, it can feel as if the pace of life switches to fast forward. Work schedules, school schedules, activities, sports and homework all need to fit in a finite amount of time. If you are also coping with school struggles, whether it is with social interactions, academics, attention or motor skills, activities that should only take a few minutes to complete often snowball into full-night events.

As a physical therapist and a mom of two school-aged children, I try to modify activities so I can accomplish two goals at once, rather than just one. Yes, realistically this sometimes means that the original activity takes a little longer to complete. However, in the end, the minor changes usually keep my kids more excited about the task and enable them to continue to build a foundation of strength and balance, which allows for mastery of other skills. Here are a few of my favorite tricks of the trade!

  • Consider having your child sit on a therapy ball while doing homework. Not only does allowing a child to bounce while sitting help them stay focused on the task longer, it also addresses core strength and balance.
  • Try practicing spelling words by creating the words out of play dough. This is not only excellent for finger strength and dexterity, but it also allows children who struggle with handwriting to practice spelling in a different way.
  • Find simple recipes for children to make their afternoon snack all by themselves. This is an excellent way for them to practice reading, following directions and maintaining attention. Additionally, it involves an end product they are invested in obtaining to satisfy an immediate need; if they do not complete the snack, they will remain hungry!
  • Consider purchasing a core strengthening disk (available online via Amazon, Target, Walmart and a variety of other specialty stores), and have your child stand on it while brushing their teeth. This works on balance and ankle strength for at least 2 minutes, twice a day.
  • If you choose to take the elevator, have your child practice standing on one leg while the elevator is in motion. Making this a family challenge will certainly add a little excitement to your elevator trips!
  • If you spend a lot of time in the car with your children, consider listening to CDs that review math facts. Multiplication facts are much easier for some children to learn when they are set to music.
  • If you have a child working on reading out loud, suggest that they read books to toddler-aged neighbors or relatives. This begins to develop a sense of responsibility for the older child as well as strengthen their own reading skills.
  • And finally, my personal favorite, look at chores needing completion around the house and decide which ones address motor skills, strength, problem-solving or organization skills. Assign those chores to your child! Sock sorting works on matching skills, carrying trashcans to the curb addresses strength, and sorting the recycling works on problem-solving. Chores also give children a sense of responsibility and can address many aspects of development.

Nicole’s Nook: More Apps for Heading Back to School

As we approach the beginning of another school year for our children, I thought that I would highlight a few apps to optimize their school performance. Below are five especially useful apps for your consideration.

JotNot Scanner Pro, $1.99

  • This universal app takes a picture of a worksheet. You can then crop it and turn it directly into a PDF.  From there, you can email, print, fax or save it directly into your photo library.  Perhaps the most remarkable feature of this app is that you can actually open the document in a variety of other apps, including iAnnotate.
  • This app is also beneficial in the classroom, allowing students to get started on their work immediately.  The student, aide or teacher can take the photo, eliminating the need to wait for the work to be scanned or emailed.  You can also scan multiple pages and even export them to iBooks, so you can use the accessibility features.

iAnnotate, $9.99

  • This iPad-only app allows you to use a variety of tools to make notes on a PDF or document.  These tools include, but are not limited to, a colored pen, a highlighter, a typewriter, stamps and a microphone, as well as strike-through, caption and underline functions.  You can also customize multiple toolbars for frequently used tools.
  • If you are using this app in the classroom, you can make notes and complete a worksheet using your finger!  If you have an iPad 3 or higher, you can also use the built-in dictation feature to speak your answers in the text boxes.
  • Your finished product can be shared via email, Dropbox or Google Drive.  It can also be viewed and edited in most external PDF readers.

Co-Writer, $17.99

  • This universal app creates documents and features word prediction, text to speech and spelling support.  It uses a standard QWERTY keyboard, which cannot be customized. It includes a main dictionary, which can be adjusted, as well as topic dictionaries. Some topic dictionaries are built into the app, but Co-Writer also has the ability to create custom dictionaries, involving a web search on a specific topic and the subsequent creation of the topic dictionary.
  • There are a variety of popular sharing options for the created documents.

Explain Everything, $2.99

  • This app is an interactive whiteboard, which allows you to annotate and narrate slides. You can create dynamic lessons, activities and tutorials, as well as create slides with color and text.  It records on-screen drawings, annotations and object movement. It also captures audio using the iPad microphone.
  • You can import and export easily, including photographs, as well as PDFs, PowerPoint and Keynote files from Dropbox, Evernote, email and camera roll.  It does not require creating an account to use.

myMemoir, $1.99

  • This is a journaling app that boasts a simple user interface.  The finished product is easy to export and can include photos or videos.

Also don’t forget to use some of the great built-in features and apps available on the iPad.

  • The Calendar can be used to track assignments, send reminders, and assist with overall organization.
  • Enabling Speak Selection will allow text to speech whenever the text is highlight. This can be particularly helpful to students with auditory processing and reading difficulties.
  • Set a Locked Timer by creating a Passcode Lock and Setting the Timer for the amount of time you want your student or child to play.  Then select STOP PLAYING, SET and START.  Now go to the app you want to play.  When the time is up, the iPad automatically goes to the lock screen.  This can be a great way to terminate those preferred games and apps, and redirect your children in a functional way!

Here’s to another great school year!!

Nicole Weckherlin, OTR/L

A Message from the Director – Summer 2014

Dr. Gutmann Riverfront Times

With thoughts of children returning to school and pleasant fall weather in St. Louis, I wanted to update you on developments and events happening in the Washington University NF Center.

On May 16, 2014, we hosted the second Washington University NF Center Research Symposium in the Eric P. Newman Education Center on the campus of the Washington University School of Medicine. This wonderful event drew over 180 clinicians, researchers, and families, and was widely regarded as an overwhelming success. We were so fortunate to showcase Dr. Sean Morrison (UT-Southwestern) and Dr. Jonathan Epstein (University of Pennsylvania) as our two keynote speakers.

We also launched the first full Beat NF session, a jazz music therapy-based program for toddlers. This unique partnership with Phil Dunlap from Jazz St. Louis and Dr. Cynthia Briggs from Maryville University Department of Music Therapy ran for six consecutive Friday mornings in June. The feedback was extremely positive, and we are currently expanding the program to include new music and activities.

There have been several exciting research advances to report. First, based on new findings by researchers in the Washington University NF Center, a large international clinical consortium confirmed the link between patient gender and the risk of vision loss in NF1. Second, Dr. Courtney Dunn and her colleagues published a manuscript on evaluating low muscle tone in children with NF1. Third, Cynthia Garcia, a graduate student in my laboratory, was successfully awarded a PhD degree for her work to identify new drug treatments for NF2-associated spinal cord tumors. Lastly, the NF Clinical Trials Consortium reported their initial findings using a new class of medication to treat NF1-associated plexiform neurofibroma.

To stay up to date with progress in the world of NF, I invite you to visit our website regularly and to join us on Facebook.

With best wishes for the school year,

David H. Gutmann, MD, PhD
Donald O. Schnuck Family Professor
Director, Washington University NF Center

Back to School: Communicating with Your School

The 2014 – 2015 school year is upon us!

NF1 is the most common disorder you’ve probably never heard of–that is what we tell people the first time they learn about NF1. Because NF1 is not a commonly known disorder, it is likely that the teachers and school administrators at your school also have not heard about it. In these cases, it can be challenging for both parents and children to receive the supports they need in order to make the school year run smoothly. Here are some tips on how to communicate effectively with your school in order to help your child have a successful year.


The more you can educate teachers and administrators about what NF1 is, the better equipped they will be to handle any issues your child may encounter. We provide a range of educational brochures covering features of NF1 from birth through adulthood. We highly recommend that families share these pamphlets with their teachers and administrators as a way to introduce them to NF1. Feel free to download and print them out or to email them.

Know Your Rights

The public school system is required to provide developmental screening to any family who requests it. Developmental screening is necessary if you want your child to receive services such as speech therapy or preferential seating in the school setting. Sometimes the school will say a child does not qualify for therapy services because the screen conducted did not reveal any significant delays. In the event that your school says your child does not qualify for services, you have the right to file a formal complaint with the Missouri Department of Special Education (phone number 573.751.4212) to pursue further testing. To learn more, please read our handout.

Know Your Goals

Take time to work with your child and his or her teachers and administrators to make it clear what your goals for your child are. Having clear goals will help educators guide your child throughout the year. In the case that your child has an Individualized Education Plan (IEP), your child’s team should schedule regular meetings to ensure that goals are being set and met on a regular basis.

Back to School: Peer Relationships

The 2014 – 2015 school year is upon us!

Friendships are an integral part of life and incredibly important to the school experience; however, children with NF1 sometimes struggle with making friends. Having even just one good friend can have a significantly positive effect on academic success. Below are some suggestions for ways to help your child build friendships this year.

Get Involved

Getting your child involved in an activity that he or she loves is an excellent way to help him or her meet friends. Remember to seek an activity your child truly enjoys. It might be a sport or could be chess club. Maybe it’s the Science Olympiad Team or choir. When your child is involved in activity that he or she truly enjoys, he or she will have the opportunity to meet like minded peers. Having similar interests and goals is a perfect starting place for a friendship.

Practice Social Skills

Some children with NF1 may experience difficulty picking up on social cues which might cause trouble in building and maintaining friendships. If you notice your child is not recognizing common social cues try pointing out social cues in every day life. For example, “Did you notice when the woman looked at her watch? I ended my conversation because that was her cue she was running late.” If you feel like your child could benefit from more regular practice, consider obtaining the app Social Express designed to teach children how to think about and manage social situations. The better your child is at navigating social situations, the easier it will be for him or her to build lasting relationships.

Educate Others about NF1

Some children with NF1 may be at greater risk for experiencing bullying because their peers may perceive them as “different.” If you find that your child is experiencing bullying consider asking your school if you or your child can talk to the other students about what NF1 is. Children are less likely to bully others when they understand the situation. Check out our educational brochures for both teens and adults on how to tell others about NF1. For more information on how to address bullying, please view our handout.

Remember also to check out Club NF and Beat NF. They are wonderful opportunities for your child to build friendships with peers living with NF1 and going through similar experiences.

Washington University NF Center Family Celebrates Final Day of Chemotherapy

Children with NF1 are prone to develop a type of brain tumor that affects the nerve that carries vision from the eyes to the brain, known as an optic pathway glioma (OPG). In some instances, these tumors cause progressive vision loss, and require treatment with chemotherapy.Ally is one of our children with NF1 and a progressive OPG. A six-year-old girl now preparing for first grade, Ally has been battling her OPG since August 2011. After four different types of chemotherapy and one surgery, we are happy to report that Ally’s tumor is stable.

On August 4, 2014, Ally received her final dose of chemotherapy. As is the tradition for children cared for by the Pediatric Neuro-Oncology team, Ally celebrated her big day by ringing the bell in the oncology wing at St. Louis Children’s Hospital. Friends, family, physicians, nurses, and Team NF were there to cheer her on. Even Ronald McDonald made a special appearance.

“It was a beautiful moment.” said Team NF Coordinator Kirsten Brouillet, “Seeing the family together celebrating and smiling really puts everything into perspective.”

Please join us in congratulating Ally and her family on this momentous occasion. To see Ally’s big day for yourself, please watch our video Ally Rings the Bell.

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Club NF Swims!

On August 2, 2014, the Washington University NF Center in collaboration with the St. Louis Children’s Hospital Foundation hosted their August 2014 Club NF Event! The program focused on gross motor development and learning organizational skills.

Club NF met at the Des Peres Lodge. The morning began with the children working on motor skill development with physical therapist Courtney Dunn, PT DPT. The children had the opportunity to practice balancing and played many fun gym games including crab-walk soccer.

While the children played in the gym, parents met with Julie Hough, owner of the local company The Ordered Home, to discuss how to create an organized system for the new school year. Julie provided advice on topics such as how to handle incoming papers and assignments and how to get out the door each morning with less stress.

After Julie’s talk, families came back together to make responsibility charts. The goal of the charts is to provide children with a visual cue to remind them of what tasks they need to complete throughout the day. The charts can also help children view completing tasks in a positive light.

After the hard work of the morning, families headed to the pool to relax and play.

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For more pictures, please visit our Facebook page.