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Dear Neurofibromatosis Community Member:

In 2006, the Children’s Tumor Foundation (CTF) asked ourselves “what areas of neurofibromatosis research need attention and funding to advance us towards improved therapeutics and better care and outcomes for those living with NF1, NF2 and schwannomatosis?” We issued a Request for Ideas (RFI) to the neurofibromatosis community – researchers, clinicians and lay persons.  The responses contributed to our 2006 process of Strategic Planning for Neurofibromatosis Research, a process which resulted in five recommended areas on which CTF should focus over the years that followed:

1.    NF preclinical drug screening

2.    Establishment of a national neurofibromatosis clinic network

3.    Establishment of a neurofibromatosis tissue bank and patient registry

4.    Identification of new biomarkers for neurofibromatosis

5.    Funding of neurofibromatosis pilot clinical trials

Through investments totaling more than $7 million, the Foundation has initiated programs in each of these areas. Our research portfolio today includes the NF Preclinical Consortium and Drug Discovery Initiative for preclinical drug screening; a national 43-site NF Clinic Network; a Clinical Trial Awards program; and an NF Registry & BioBank. This is in addition to our long-running Young Investigator Award program; ad hoc funding programs including Schwannomatosis Awards; and support of the annual NF Conference and topic-focused consensus workshops.

How to respond to this RFI

·         Provide your name and contact info (optional)

·         State your relationship to neurofibromatosis (scientist, clinician, individual with NF, etc.)

·         Please review and respond to the attached THREE questions.

·         Keep each question response to half a page (box shown).

·         You are encouraged to think as broadly as possible!

Pleas click here to download the RFI as a Word document.  Please click here to download the RFI as a pdf. 

Children's Tumor Foundation Clinical Program Coordinator  Kathleen Berentsen is at the Child Neurology Society Annual Meeting this week in Providence, Rhode Island.  Kathleen is hosting a CTF information booth to inform  physicians and clinical staff in attendance about the CTF NF Clinic Network and in-development Patient Registry and BioBank, and share CTF's informational brochures for families , covering  all aspects of living with NF1, NF2 and schwannomatosis.  Kathleen will attend some of the sessions including tomorrow's special session on neurofibromatosis, and we hope to bring you a report from the meeting next week!

 

The National Institutes of Health have announced a potential commitment of up to $50 million OVER 5 YEARS to form a Tumor Microenvironment Network (TMEN). This U54 opportunity proposes to assemble up to TWELVE Centers to 1) generate a comprehensive understanding of composition of the stroma in normal tissues as well as its roles in tumor initiation, progression, and metastasis; 2) delineate mechanisms of tumor-stroma interactions in human cancer; and 3) identify the mechanism(s) by which tumor stroma may affect responses to treatment. Primary brain tumors are of interest as well as peripheral nervous system tumors. The program is co-funded by National Cancer Institute and National institute for Neurological Disorders and Stroke. An applicant may request a project period of up to 5 years and a budget up to $700,000 per year (direct costs).

Given the established role of the microenvironment in NF tumors, this program offers a terrific opportunity for neurofibromatosis research centers to apply. Letters of Intent are due December 20, 2010 and if invited full applications by January 20, 2011.  Anticipated earliest start date of funding: September 2011. For more information click here.  (Request for Applications RFA-CA-021).

 

New Food and Drug Administration funding could provide an opportunity for neurofibromatosis clinical trial applications to be supported. FDA Orphan Products Development Grants support the clinical development of products for use in rare diseases or conditions for which no current therapy exists or when the proposed product would be superior to the existing therapy. This would include neurofibromatosis, and the Children's Tumor Foundation encourages NF researchers to apply. Phase 1 clinical trials are eligible for grants of up to $200,000 per year for up to 3 years. Phase 2 and 3 clinical trials are eligible for grants up to $400,000 per year for 4 years. Dollar caps represent ALL costs (i.e. direct plus indirect). 

Next application deadline is February 2, 2011. Questions: contact Katherine Needleman at Katherine.needleman@fda.hhs.gov or 301-796-8660. To read the request for applications click here.

This week saw a group of bipartisan senators - Sherrod Brown (D-Ohio), Sam Brownback (R-Kan.) and Al Franken (D-Minn.) - introducing the Creating Hope Act: legislation to encourage pharmaceutical and biotechnology companies to focus more intently on cures for rare diseases that affect children and which today have no treatments. A rare disease is one that affects fewer than 200,000 Americans. Neurofibromatosis affects 100,000, and has no treatment available, so could stand to benefit from this bill. The bill aims to offer incentives to industry such as faster approval at the Food and Drug Administration, and expands on a 2007 law offering "priority review vouchers" to companies that developed drugs for tropical diseases (this has also traditionally been a neglected area).  

This is another development in a series of promising changes that we have seen this year both in the government, and in industry, in terms of increasing interest in finding treatments for rare diseases.  Click here for article in 'The Hill'.