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“Dat” is Superbowl Champion Jon Stinchcomb holding THE Lombardi Trophy.  Jon is from Lilburn Georgia and cousin to the Leathers family.  He and his brother Matt (played for the Raiders in Superbowl XXXVII) have been amazing members of the Children's Tumor Foundation family, holding events like the Countdown to Kickoff and even running for the NF ENDURANCE team last March, helping to raise more than $50,000 for schwannomatosis research.

Jon, who comes in at 6’6” 325lbs, ran the first leg of a relay which was “longer than he’ll run all season.”  Matt and Jon became involved because their youngest cousin Drew suffers from schwannomatosis. However, that hasn’t stopped Drew from screamin “WHO DAT!” all season long.

 

Congratulations Jon and thank you for your amazing efforts.

[Chad Leathers & Jon at the ING Georgia Marathon]

Current Children’s Tumor Foundation Young Investigator Awardee Dr. Sutapa Banerjee (Washington University School of Medicine) is first author of a new publication* (links to abstract) in Cancer Research that identifies a new candidate drug target for treatment of NF1 tumors. Senior author Dr. David Gutmann is Dr. Banerjee’s mentor and also heads the optic pathway glioma site of the Children’s Tumor Foundation NF Preclinical Consortium.

The NF1 gene encodes neurofibromin, a tumor suppressor protein that suppresses the Ras cell signaling pathway. When the NF1 gene is mutated and functional neurofibromin protein is not made, these cell signals are hyperactivated. A number of these signals are being evaluated as candidate drug targets for NF1 treatments.  Dr. Banerjee’s study sought to identify additional new candidate drug targets. By screening a chemical drug library on NF1-deficient malignant peripheral nerve sheath tumor cells, the group found a natural product called cucurbitacin-I that inhibited growth. Research revealed this drug is targeting a cell signal called STAT3 which is hyperactive in NF1-deficient cells. The effects of the drug are independent of cell signals TORC1- and Rac1. This is important because sometimes blocking a drug target either blocks or stimulates another target which interferes with the effectiveness of drug.  This study however suggests STAT3 may be directly targeted with drugs to halt tumor growth without potential feedback to or interference from other cell signals.  

* Banerjee et al. (2010) The Neurofibromatosis Type 1 Tumor Suppressor Controls Cell Growth by Regulating Signal Transducer and Activator of Transcription-3 Activity In vitro and In vivo.  Cancer Res; 70(4); 1356-66.

 

Following a path blazed by Genzyme and now followed by a number of other biotechs and pharmas,   Glaxo Smith Kline has announced they will be opening a division dedicated to finding medical treatments for rare diseases.  

GSK has generously provided drug for the ongoing Children's Tumor Foundation-funded Phase Zero trial to assess lapatinib as a candidate treatment for  NF2 vestibular schwannoma.  The new GSK rare disease program comes in the wake of other program closures at GSK.  We will watch this closely.

 

Patient advocacy groups like the Children's Tumor Foundation play many roles from funding medical research, providing support, resources and a community 'voice' for patients.  At the Biotechnology Industry Organization 2009 Annual Conference, a forum for pharmaceutical and biotechnology companies, and more recently for patient advocacy groups to interact and partner with these entities, CTF's Chief Scientific Officer Dr. Kim Hunter-Schaedle  featured in a short video highlighting the role of patient advocacy groups. View this now at

http://biotech-now.org/biodigest-patient-advocacy-011790.html

 

 

Children's Tumor Foundation Chief Scientific Officer Dr. Kim Hunter-Schaedle has been invited by the Biotechnology Industry Organization (BIO) to present the Foundation's translational neurofibromatosis programs in preclinical drug screening and clinical trials at the 12th Annual BIO CEO and Investors Conference (www.ceo.bio.org) at the Waldorf-Astoria Hotel in New York on February 9th at 9am. BIOtech Now’s Tracy Krughoff talked to Dr. Hunter-Schaedle to preview the BIO meeting: listen to the podcast at http://www.bio.org/podcasts/HunterSchaedle.wma

A free lecture course  "The Science of Small Clinical Trials"  is now open for registration. The survey course covers methods to design and analyze clinical trials using small numbers of participants and is provided by The National Institutes of Health Office of Rare Diseases Research in conjunction with the Food and Drug Administration Office of Orphan Products Development.  This topic of how to design conduct and interpret smal lclinical trials is highly relevant to neurofibromatosis where small clinical trials - such as those funded by the Children's Tumor Foundation Clinical Trials Award - are likely to play an important part in advancing neurofibromatosis drug therapies.

The course comprises 7 x 2-hour lectures in February and March, and can be attended in person (NIH campus in Bethesda, MD) or online.  Remote  attendees will be able to submit questions to the speaker.  An optional self-administered open-book on-line examination concludes the course to qualify for a certificate from FDA's Office of Orphan Products Development. More information and registration:   http://small-trials.keenminds.org/

 

'What's New in Neurofibromatosis?' (links to abstract), the report from the Foundation's 2009 NF Conference, was published this week in the American Journal of Medical Genetics. Thanks to AJMG Editor-in-Chief Dr. John Carey  - we are excited to see this first publication of 2010 for the Children's Tumor Foundation on the heels of the two white papers published in 2009 (on NF2 clinical trials and bone dysplasia management).

This is the first in a series of articles outlining the agenda for the 2nd Annual NF Forum in Las Vegas from April 9-11.  The NF Forum is a patient and family medical symposium designed to educate individuals (of all ages) and their families about the NF disorders, management options, clinical trials, insurance concerns, social challenges and many other expressions of NF1 and NF2.  We welcome your comments and suggestions. 

How to be an advocate for your child: School Related Issues, will take place during the NF1 Specific Workshops section of the program (there will be concurrent NF2 Specific Workshops).  The presentation will be delivered by Dr. David Salsberg, a licensed clinical neuropsychologist and Clinical Instructor at The NYU School of Medicine. The presentation will highlight the main learning and attention issues associated with NF and related IEP/educational information.  Additionally there will be an information booth following the presentation where Dr. Salsberg will be available to discuss specific questions/concerns including:

·         Questions on Learning disabilities

·         Attention deficit disorder

·         Special Education

·         Advocating for services

·         Neuropsychological evaluations

·         Special Education Law

·         Psychological/emotional/behavioral issues

·         Sibling issues

For more information on the NF Forum please click here. 

 

 

Recently published NF papers cover a range of topics. These include:  the controversial issue of using radiotherapy to treat NF2 tumors; how a patient's NF2 genetic status might predict severity of the condition;   molecular advances in understanding the function of NF2 protein merlin (by recent CTF Young Investigator Awardee Timmy Mani);  a role for the Spred-1 (Legius Syndrome) gene in controlling brain development and patterning; new zebrafish models of NF1 that have vascular and cardiac valve abnormalities; speech charasteristics seen in NF1 patients; and the first case of germ line mosaicism in schwannomatosis.  For more on what’s new in NF publications, read the January Research Roundup.