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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. 

Interesting stories this week on  full genome sequencing done on two individuals.   In Japan, a team from the RIKEN center used massively parallel sequencing technology to  analyze the complete genome of a Japanese individual, the first time this has ever been done.  While the work will be published in Nature Genetics, early story found at FierceBiotech.

The Institute of Medicine has released a report that calls for a national "War on Rare Diseases", similar to the 1970's "War on Cancer", reports the Wall Street Journal.   Rare diseases are those affecting fewer than 200,000 Americans (neurofibromatosis affects an estimated 100,000). As we have reported, in the last couple of years there has been significant progress in devoting resources to rare diseases, by the National Institutes of Health, by the Food and Drug Administration, and even by biotech and pharmaceutical companies. IOM now calls for a 'national strategy' to tackle rare diseases. This would be a sea change from the fact that for many years, finding treatments for rare diseases has largely been driven by advocacy groups and foundations like the Children's Tumor Foundation.

Though significant progress has been made in developing cancer treatments, some very large questions remain. Why do malignant tumors metastasize? Why do some tumors spread and others do not?  Dr. Harold Varmus, Director of the National Cancer Institute of the National Institutes of Health, is set to launch the  "Big Questions Initiative" which will solicit input from leading experts as to what these questions are and how to address them. This should be an interesting process, but for most researchers perhaps the biggest question of all is whether the government will commit any additional funding to the NIH in order to address new avenues of research. Read more.

Congress will be back in session on September 13th, and we expect the appropriations bill that includes funding for the Congressionally Directed Medical Research Program-NF Research Program (CDMRP-NFRP) to addressed in September.  We will be asking our supporters later this month to reach out to their Senators and Representatives.

To read a recent update and the history of this vital program please visit:

http://www.ctf.org/How-You-Can-Help/advocacy.html

Happy Labor Day

John