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On March 15, 2012, Stanford Hospital announced the launch of a new program, led by Dr. Steven Chang, to treat rare tumor-multiplying diseases.

Stanford's program includes a team of 12 specialists in several fields: neurosurgery, epilepsy, neuro-ophthalmology, neuro-oncology, neuro-otology, neuro-interventional radiology, urology, and general surgery. 

"The goal," Chang said, "is to give cutting-edge care for conditions like Knodel's, neurofibromatosis 2 (NF2) and others including another form of neurofibromatosis, schwannomatosis tuberous sclerosis, von Hippel-Lindau disease, Sturge Weber syndrome and hemorrhagic telangiectasia."

Please click here to read more.

On February 18, 2012, a 10-year-old girl from Parker, Colorado was taken on her first “date,” one she will certainly never forget. It was with Denver Bronco’s quarterback and all-around "good guy" Tim Tebow. 
Presli Collins, who lives with NF1, was invited on this “date” by the Tim Tebow Foundation’s “Wish 15” program, which grants wishes to children with serious illnesses. Presli's mother, Lisa, says they couldn’t believe it when Tebow’s foundation called last week to arrange the trip. “She had just gone through a lot, and when we got the notice for this she was just completely shocked,” said Lisa.

Presli became an "NF Hero" when she was diagnosed with neurofibromatosis in 2008. Affecting 1 in every 3000 people, NF can lead to blindness, bone abnormalities, cancer, deafness, disfigurement, learning disabilities, and excruciating and disabling pain. Presli has bravely endured brain and back surgery to excise tumors, and anterior and posterior spinal fusion surgery to correct, as much as possible, scoliosis that has occurred due to NF.

While in Los Angeles, Presli was given true VIP treatment including a new dress and hairstyle, photos on the red carpet with Tebow, and "meet and greets" with celebrities such as Shaquille O'Neal, Nickelodeon star Nathan Kress, and the Disney Channel's China Anne McClain.

Presli's Dad remarked that he’s, "never seen her smile or be as happy as she was that weekend."  Presli's Mom continued, “I can’t even express how grateful we are for the opportunity she’s had [and] the joy she’s had."

"I got to meet Tim Tebow and he was a thousand times nicer than you think he is," said Presli. "In the middle of my dinner he took me to the chocolate fountain. [He also gave me an autographed football that] says, 'To Presli, number 15, Tim Tebow, God bless, go Broncos. Thank you for being an amazing date."

Presli is a happy, active, young girl, but the uncertainty of NF can be wearing. “I think the hardest part is not knowing how [NF will] manifest in her body,” says her mother. But the Collins's remain positive and optimistic, and encourage people to support the Tim Tebow Foundation, Children’s Hospital of Orange County, and the Children’s Tumor Foundation.

Some have said that Tim Tebow is the most exciting player in the NFL today. He is a Heisman Trophy winner, an All-American BCS champion, a first round draft pick, and a record setting professional Quarterback. He can now add "NF Hero's Hero" to his list of accolades, with the sincere gratitude and appreciation of the Children's Tumor Foundation and the millions of families worldwide who face NF everyday.

To learn more, click here for an article and video from The Huffington Post.

The Drug Discovery Initiative Awards program is focused on seed funding preclinical drug testing studies on neurofibromatosis in cell or animal models, and is one of the most successful Children’s Tumor Foundation programs to date. Since this program began in 2006, we have funded over 45 DDI Awards focused on preclinical testing of drug therapies for all aspects of NF1, NF2 and schwannomatosis, from learning disabilities to tumor growth to pain. This has been an investment for the Foundation of $1 million, but we have seen our investment leveraged, as awardees have gone on to secure over $5 million in follow-on funding from federal and other sources.

We are delighted to announce the recipients of the Drug Discovery Initiative Awards from our fall 2011 round. Dr. Toshifumi Tomoda of the Beckman Research Institute receives a $15,000 in vitro Award to study autophagy-inducing compounds as candidate therapeutics for NF2. Dr. David Little of the University of Sydney receives a $30,000 in vivo Award to study MEK inhibition as a therapeutic approach in mouse models of NF1 related tibial pseudarthrosis.

We are also pleased to announce that for 2012 we are significantly increasing DDI Award levels! We now offer $25,000 for in vitro DDI Awards, $50,000 for in vivo DDI Awards, and $75,000 for Advanced DDI Awards. There are two DDI Award deadlines in 2012: February 28th and August 31st. No preliminary data is required for DDI Award applications, and cutting-edge ideas are encouraged. If you have any questions about the DDI program, please contact Min Wong at mwong@ctf.org.

The neurofibromatosis clinical team at the Cincinnati Children's Hospital Medical Center is conducting a research survey to find out if people with neurofibromatosis type 1 (NF1) break their bones more often and have different types of broken bones (fractures) than those who do not have NF1. Adults 40-70 years old with NF1 and their spouses and/or siblings who do not have NF1 may be eligible to participate. The information learned from this research study may indirectly benefit other patients with NF1 in the future by providing a better understanding of NF1-associated bone disease.  

This study consists of completing a survey that is estimated to take a total of 20 minutes and contains questions about participants’ fracture history, physical activity, and diet.

For more information on the study, click here.  

                As neurofibromatosis clinical trials increase in number, the clinicians leading them are keen to design the trials to be as effective and meaningful as possible. A key part of this effort is developing the right trial endpoints - measures and metrics that can be used to determine if a drug or intervention is effective or not.  To tackle this area, a team of neurofibromatosis clinicians and researchers has formed  a working group called Response Evaluation in Neurofibromatosis and Schwannomatosis (REiNS). Spearheaded by Dr. Scott Plotkin (MGH) and Dr. Brigitte Widemann (NCI) and first convened in June at the Children’s Tumor Foundation 2011 NF Conference, around 30 leading NF clinicans and researchers participating in the REiNS met in Boston to continue their planning and discussions.

                Past endpoints for neurofibromatosis clinical trials have included changes in maximum tumor dimension on MRI scans or changes in cognitive function on neuropsychological assessment (for learning disabilities).  Looking ahead, more advanced endpoints under discussion (and already being piloted in some cases) include volumetric tumor analysis and whole body MRIs; and the use of biomarkers – biological indicators in the blood or other body fluids to determine if a drug is working. REiNS members have organized into groups to focus on different measures and plan to meet every few months to continue advancing this project.

                The Children’s Tumor Foundation is delighted to be investing in endpoint development through our Clinical Research Awards program.  Currently our funded projects include developing a computerized test for more accurate assessment of learning disabilities trials; identifying a blood biomarker of NF1 status; and developing measures of response for optic pathway glioma trials. We will be announcing more funded awards in early 2012. 

                Clinicians or researchers interested in participating in REiNS can contact Vanessa Merker at MGH: vmerker@partners.org.