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I am pleased to announce that Congress has appropriated $16 million for NF research for fiscal year 2011.  This research, conducted through the Department of Defense, is critical both to those living with the challenges of NF and the brave men and women serving our country. 

As many of you know, research into treatments and cures for NF and its various manifestations has implications for those beyond the NF community.  Advancing our understanding of mast cells to stop tumor growth helps us understand how we may also speed wound healing for our war fighters.  Understanding the mechanisms of pain in NF can allow us to understand the signaling pathways that can block the pain our soldiers may face.  Imaging technologies advanced to understand NF tumor growth are proving essential in MRI volumetric imaging to the broader community, particularly our armed forces. 

We are grateful that those serving in Congress recognize the importance of NF research for those serving our country, and those living with NF.  We know that the importance of NF research validates that recognition. 

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

Welcome to the seventh in a series of "NF Bites" - providing snapshots of individual areas of neurofibromatosis research and how the Children's Tumor Foundation is advancing this. Over the coming days and weeks we will focus on different aspects of neurofibromatosis research. Today: in Part 2 on where are we with research progress in NF2, we focus on advances in preclinical drug testing and clinical trials.

NF2 research activity, especially in the area of testing candidate drug treatments has increased significantly in the past several years. This has been driven in part by our publication in 2009 of consensus documents on NF2 clinical trials and drug pipeline in Clinical Cancer Research, based on an expert workshop convened by the Foundation. We currently fund a significant amount of NF2 research ‘from bench to bedside’ – an indicator of the exciting progress made in NF2 recently in understanding the underlying biology and then applying this to finding effective drug therapies. Importantly CTF is hosting an expert workshop in May 2010 to evaluate progress to date in NF2 clinical trials and how these can be further accelerated.

·         The Foundation was a co-supporter of the groundbreaking pilot Phase II trial of Bevacizumab (Avastin) which caused vestibular tumor shrinkage, restored hearing and was published in New England Journal of Medicine in July 2009.

·         One of our first Clinical Trial Award recipients in 2009 was Jaishri Blakeley (Johns Hopkins University), receiving $125,000 to do a Phase Zero NF2 clinical trial of the drug Lapatinib, currently in progress.

·         Outside of CTF funding, a Phase II NF2 trial of note is underway at Massachusetts General Hospital to assess drug PTC-299 in patients. This trial is co-supported by CDMRP NFRP and PTC Therapeutics. 

·         Through the Foundation’s NF Preclinical Consortium, a $4M multi-year initiative to accelerate promising candidate drugs to the clinic, the laboratories of Andrea McClatchey (Harvard/ Massachusetts General Hospital) and Marco Giovannini (House Ear Institute) are testing a pipeline of NF2 drugs, in collaboration with Novartis, Genentech and Avila Therapeutics.

·         In one of our first Drug Discovery Initiative Awards, in 2006, Oliver Hanemann (University of Plymouth) received $11,000 to test the drug Sorafenib on human NF2 schwannoma cells in culture. Positive data emerging from this study has led to planning of a Phase II European clinical trial.

Look for the final instalments of NF Bites next week!

Welcome to the sixth in a series of "NF Bites" - providing snapshots of individual areas of neurofibromatosis research and how the Children's Tumor Foundation is advancing this. Over the coming days and weeks we will focus on different aspects of neurofibromatosis research. Today: where are we with research progress in NF1 learning disabilities?

Our understanding of NF1 learning disabilities has opened up significantly in the past few years in terms of understanding their basis and developing treatment strategies – but we still have much more to learn and CTF is supporting this area.

·         In 2005 Dr. Alcino Silva and his team at UCLA published a landmark finding. They showed that the cholesterol-modifying drug Lovastatin when given to a genetic mouse model of NF1 learning disabilities could reverse these deficits. Mice given Lovastatin had improved ability to respond in a test system called a water maze.

·         Dr. Silva’s findings advanced rapidly to the clinic and Lovastatin trials are now well underway around the world through the CDMRP NFRP Phase II Clinical Trials Consortium as well as other independent clinical trials. Lovastatin offers the first hopeful drug for those with NF1 learning disabilities, but has also advanced other areas of NF, for example Lovastatin also show promise as a treatment for bone dysplasia (reported in an earlier NF Bites’).

·           CTF Young Investigator Awardee Ana Oliveira (Duke University Medical Center) is examining the underlying changes that occur in brain cell in NF1 related learning & memory disabilities.  She has found that cells in the brains of individuals with NF1 are actually different in their appearance and function. 

·         CTF Young Investigator Awardee Weidong Li (UCLA) is assessing Ritalin in mice as a candidate treatment for NF1 cognitive defects. 

·         CTF Young Investigator Awardee Linnea Vose (New York Medical College) is testing learning disabilities candidate drug treatments including rapamycin and rolipram in NF1 fly models. 

·         One of the challenged of early clinical trials was how to measure a drug’s effectiveness on NF1 learning disabilities. To more quickly advance discoveries from mouse to human, Dr. Nicole Ullrich and her colleagues at Children’s Hospital Boston have developed a ‘humanized’ water maze which is a computerized test. It is anticipated this ‘Arena Maze’ will be able to quickly determine if a drug is improving NF1 learning disabilities.

·         In 2006 and 2007, CTF organized NF1 Learning Disabilities workshops to bring basic and clinical researchers in this area together. This helped in the planning of the ongoing Lovastatin clinical trials. Looking ahead we hope to again convene this community in 2011 to plan next steps. Of particular interest is the growing link between learning disabilities seen in NF1 and other genetic conditions. A next step is therefore to build on this common links and share data and ideas so that advancements can benefit not only those with learning disabilities in NF1 but also in the context of other genetic conditions.

Look for more NF Bites in the coming days!

The Department of Defense Congressionally Directed Neurofibromatosis Research Program (CDMRP NFRP) is now open for applications for Clinical Trial Awards, Investigator Initiated Awards and Investigator Initiated Focused Research Awards. For all programs, pre-applications are due April 29, 2010 and full applications May 20, 2010. A total of $13.75M is available in 2010. For more information and application forms: http://cdmrp.army.mil/funding/nfrp.htm