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Dr. Chris Moertel, Director of the Children's Tumor Foundation Neurofibromatosis Clinic Network Affiliate Clinic at the University of Minnesota, is making progress in what has largely been an elusive area for clinical researchers - the development of vaccines to treat brain tumors. Dr. Moertel pioneered these studies by successfully halting brain tumor growth in a dog which had been injected with cell matter harvested from its own tumor.  He has since advanced the trials into seven patients with malignant glioma who have been treated with vaccine made from their own tumor matter. It is very early days but these studies look promising and will continue.  Vaccines have been challenging to develop but if successful would reduce the need for using chemotherapy drugs.  The target is currently malignant cancer but looking ahead there may be potential to apply a similar approach to neurofibromatosis tumors.  View a short TV interview with Dr. Moertel talking about the work here.

Do you have a basic discovery that you think you can  translate to the clinic for the benefit of patients? The National Institutes of Health (NIH) Clinical Center has announced availability of funds for the 2012 Bench to Bedside Awards Program to support exactly this type of project. This unique program offers Awards of $135,000 over 2 years and supports clinical research - including in rare diseases such as neurofibromatosis. As part of its mission the program  faciliates access for researchers outside of the NIH to the institutes wealth of clinical research resources.  

A Letter of Intent is due on September 28, 2011.  More information about the program and the application process can be found at http://www.cc.nih.gov/ccc/btb/ or submit any questions by email to BenchtoBedside@mail.nih.gov.

NIH invites proposals for 2012 NIH Director's Pioneer Awards and New Innovator Awards for innovative approaches to major challenges in biomedical or behavioral research. 

• Pioneer Awards:  
  • Up to $2.5 million in direct costs over 5 years
  • Open to scientists at any career stage. 
•  New Innovator Awards: 
  • Up to $1.5 million in direct costs over 5 years
  • For early career stage investigators (ESI), defined as those who have not received an NIH R01 or similar grant and are within 10 years of completing their terminal research degree or medical residency.   

NIH expects to make at least 7 Pioneer Awards and at least 33 New Innovator Awards in summer 2012.  The deadline for submitting Pioneer Award applications is October 7, 2011. See the instructions in the Funding Opportunity Announcement RFA-RM-11-004.  
The deadline for submitting New Innovator Award applications is October 14, 2011. See the instructions in the Funding Opportunity Announcement RFA-RM-11-005.  

The National Institutes of Health (NIH) has a program called the Common Fund that supports exceptionally innovative and potentially high impact programs that are inherently high-risk but have the potential for high-payoff by catalyzing research across all of NIH and in the biomedical research community. These programs are managed by the NIH Office of the Director in partnership with the various NIH Institutes, Centers and Offices. Additional information about the NIH Common Fund can be found at http://commonfund.nih.gov.
New ideas for Common Fund programs are identified annually, and public comment is encouraged. NIH is now asking for YOUR input for 2013 programs.  Which ideas you think have the potential to fundamentally change how we think about, support, or do research in a specific field, or to create a new field all together?
Provide your input today:  http://commonfund.nih.gov/strategicplanning
The feedback period is open until Wednesday, September 14, 2011.

Schwannomatosis is the least well understood form of neurofibromatosis and also the rarest, affecting an estimated 1:40,000 persons. It causes tumors to grow on peripheral nerves throughout the body and severe, untreatable pain.  In 2007 the first candidate schwannomatosis gene was reported but there is much to learn and understand on the path to developing treatments. Since that time CTF has been at the forefront of leading schwannomatosis progress investing over $600,000 in projects including  genetics, animal model development, preclinical drug testing and establishing a Schwannomatosis International Database.  As well as funding research, CTF has accelerated progress and collaboration in this area by hosting a series of expert consensus meetings, the most recent in June 2011 from which a state-of-the-field report is being developed for publication.

As an outcome of the June consensus meeting, CTF announces a request for Applications for Schwannomatosis Awards up to $75,000. Funding may be requested for ANY research in any area of relevance to advancing schwannomatosis; areas of emaphasis are Genetics, Cell Biology & Translational Research; and Clinical Research. Applications must be received by Monday October 3^rd, 2011. For more information visit http://www.ctf.org/For-Scientists/schwannomatosis-awards.html

The Children’s Tumor Foundation is delighted to announce the funding of SIX Young Investigator Awardees from application received for our 2011 program. The recipients include three postdoctoral awardees and three graduate students; three focused on aspects of NF1 including tumors, bone dysplasia and learning disabilities; and three focused on NF2or schwannomatosis. Four awardees are US-based and two are international.    The 2011 Awardees represent an investment for CTF of around $500,000.
Young Investigator Awards provide the recipient with two years of salary support plus a $5,000 travel stipend to attend the NF Conference and other meetings.
2011 YIA recipients are as follows:
Miriam Smith, Ph.D., University of Manchester, United Kingdom. Project: Identification of novel genes predisposing to schwannomas and meningiomas by exome

Jonathan Payne, Ph.D., University of Sydney, Australia. Project: The Neural Basis and Treatment of Reading Disability in Children with NF1

Jianzhong Yu, Ph.D., Johns Hopkins University. Project: Molecular genetic characterization of the Merlin tumor suppressor protein complex

Alejandra Petrilli Guinart, University of Central Florida. Project: LIM kinase - a potential therapeutic target for NF2

Steven Rhodes, Indiana University School of Medicine. Project: Targeting the hematopoietic bone microenvironment in the treatment of NF1 pseudarthrosis

Adrienne Watson, University of Minnesota. Project: Understanding the Role of Wnt Signaling in Malignant Peripheral Nerve Sheath Tumors

For more information on these projects and past YIA recipients please visit:
http://www.ctf.org/For-Scientists/young-investigator-awards.html

This weekend the Children's Tumor Foundation participated with around 100 researchers,  clinicians and foundation representatives in the Second International Rasopathies Network Conference in Chicago.  The Rasopathies Network was conceived through a collaboration between physicians focused on, and families affected by, 'Rasopathies' - or rare diseases that affect signaling in the Ras pathway. Rasopathies include neurofibromatosis, Noonan's Syndrome, Costello Syndrome, Cardio-Facio-Cutaneous (CFC) Syndrome and Leopard Syndrome. These rare conditions share many common features, including learning disabilities, and skin and bone manifestations, with impact on quality and span of life. The goal of the Rasopathies Network is to drive collaboration between scientists working on the different rare diseases to accelerate research. The  2011 Rasopathies Network Conference was organized to occur at the end of a six-day schedule of family conferences and clinics for Noonan's, Costello and CFC families, which made for a busy and informative time.  Overall neurofibromatosis research has made significant advances compared to the other rare diseases and we  got some good words for our NF Clinic Network and preclinical programs.  But we can definitely learn from some of the approaches the other groups are taking, for example to develop patient health records, and to improve clinical care guidelines. As candidate drugs emerge, it is likely that they will have applicability across many of these rasopathies therefore ongoing communication is vital as the research moves forward.  At the close of the weekend CTF and the NF Network (formerly NF Inc) jointly participated in a small NF Symposium for local attendees where Alcino Silva (UCLA) and Maria Acosta (Children's National)  gave updates on learning disabilities research and trials; I presented some highlights from the recent 2011 NF Conference;  and Kim Bischoff (NF Network) gave a presentation on the importance of advocacy for NGF research funding. 
We looks forward to the 2013 meeting of this group, and to future collaborations with the Rasopathies Network and to both sharing our knowledge and learning a lot!

The goal of the Children's Tumor Foundation Drug Discovery Initiative is to fuel research that diversifies and advances the pipeline of candidate drug therapies for neurofibromatosis. Established in 2006, DDI Awards fills an important niche for the NF research community by providing modest 'seed' funds  - $15,000 for in vitro (cell) studies and $30,000 for in vivo (animal) studies to kick start new drug therapy ideas that might not have preliminary data, making it challenging to get initial funding elsewhere. The DDI Awards application process is easy and fast as only a 3 page research plan is required and  investigators can have the money 'in hand' as early as 6 weeks after the application deadline.  

Since 2006, DDI Awards has funded 45 drug testing studies that collectively cover NF1, NF2 and schwannomatosis manifestations including tumors, bone dysplasia, pain and learning disabilities.   To date CTF has invested around $1M in DDI Awards. We are delighted that we have been able to track this investment to a total of $5M in 'follow on' funding that the researchers have later secured from other sources such as NIH, DOD and industry.  In addition multiple industry collaborations have been forged by  NF researchers through DDI funding  and over 20 published scientific reports cite DDI funding. The next DDI Awards deadline is August 31st. In vitro ($15,000) and in vivo ($30,000) Awards are available.  Prior recipients of in vivo Awards with particularly promising data may be eligible to apply for a $50,000 Advanced DDI Award.   For application packet and information on past and current funded projects please click here.  General questions may be sent to Kim at khs@ctf.org

This weekend the Children's Tumor Foundation participated with around 100 researchers,  clinicians and foundation representatives in the Second International Rasopathies Network Conference in Chicago.  The Rasopathies Network was conceived through a collaboration between physicians focused on, and families affected by, 'Rasopathies' - or rare diseases that affect signaling in the Ras pathway. Rasopathies include neurofibromatosis, Noonan's Syndrome, Costello Syndrome, Cardio-Facio-Cutaneous (CFC) Syndrome and Leopard Syndrome. These rare conditions share many common features, including learning disabilities, and skin and bone manifestations, with impact on quality and span of life. The goal of the Rasopathies Network is to drive collaboration between scientists working on the different rare diseases to accelerate research. The  2011 Rasopathies Network Conference was organized to occur at the end of a six-day schedule of family conferences and clinics for Noonan's, Costello and CFC families, which made for a busy and informative time.  Overall neurofibromatosis research has made significant advances compared to the other rare diseases and we  got some good words for our NF Clinic Network and preclinical programs.  But we can definitely learn from some of the approaches the other groups are taking, for example to develop patient health records, and to improve clinical care guidelines. As candidate drugs emerge, it is likely that they will have applicability across many of these rasopathies therefore ongoing communication is vital as the research moves forward.  At the close of the weekend CTF and the NF Network (formerly NF Inc) jointly participated in a small NF Symposium for local attendees where Alcino Silva (UCLA) and Maria Acosta (Children's National)  gave updates on learning disabilities research and trials; I presented some highlights from the recent 2011 NF Conference;  and Kim Bischoff (NF Network) gave a presentation on the importance of advocacy for NGF research funding. 
We looks forward to the 2013 meeting of this group, and to future collaborations with the Rasopathies Network and to both sharing our knowledge and learning a lot!
 

The NF Forum officially commensed last evening following an exciting day of Chapter Council meetings.  Zachary, a young man who has taken the challenges of NF and made overcoming them an opportunity to be extraordinary, delivered the featured speech of the evening to a rousing response.  Mike Max, a Minnesota sports broadcaster and television personality, delivered the keynote and spoke of the courage and compassion of the NF community in a speech that was truly inspiring. 
And really that's what last night was all about: inspiration.  We are at an inspiring time in our search for treatments and a cure for NF.  Our community has come together in a way that had never before been the case.  Our national programs are connecting families from all over the country -- children and adults, individuals with NF1, NF2, and schwannomatosis --  to support one another and work toward treatments together, our Clinic Network continues to improve care for those battling the manifestations of NF, and, most importantly, our research programs are accelerating the development of treatments that will drastically improve the lives of those living with NF. 
In its third year, inspiration has always been a hallmark of the NF Forum, but never more than this year in Minneapolis.  Stay tuned all weekend as we bring you videos, pictures, and updates from the NF Forum.  Prepare to be inspired.