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                As neurofibromatosis clinical trials increase in number, the clinicians leading them are keen to design the trials to be as effective and meaningful as possible. A key part of this effort is developing the right trial endpoints - measures and metrics that can be used to determine if a drug or intervention is effective or not.  To tackle this area, a team of neurofibromatosis clinicians and researchers has formed  a working group called Response Evaluation in Neurofibromatosis and Schwannomatosis (REiNS). Spearheaded by Dr. Scott Plotkin (MGH) and Dr. Brigitte Widemann (NCI) and first convened in June at the Children’s Tumor Foundation 2011 NF Conference, around 30 leading NF clinicans and researchers participating in the REiNS met in Boston to continue their planning and discussions.

                Past endpoints for neurofibromatosis clinical trials have included changes in maximum tumor dimension on MRI scans or changes in cognitive function on neuropsychological assessment (for learning disabilities).  Looking ahead, more advanced endpoints under discussion (and already being piloted in some cases) include volumetric tumor analysis and whole body MRIs; and the use of biomarkers – biological indicators in the blood or other body fluids to determine if a drug is working. REiNS members have organized into groups to focus on different measures and plan to meet every few months to continue advancing this project.

                The Children’s Tumor Foundation is delighted to be investing in endpoint development through our Clinical Research Awards program.  Currently our funded projects include developing a computerized test for more accurate assessment of learning disabilities trials; identifying a blood biomarker of NF1 status; and developing measures of response for optic pathway glioma trials. We will be announcing more funded awards in early 2012. 

                Clinicians or researchers interested in participating in REiNS can contact Vanessa Merker at MGH: vmerker@partners.org. 

Following a competitive application process, CTF was selected for the second year in a row to deliver an ‘Innovator’ presentation at the 2010 Partnering for Cures Meeting  - the second annual meeting organized by the Milken Institute to bring together medical research leaders and decision-makers, innovators, and advocates from across sectors, to find workable solutions that will expedite the medical research and development process. Over 800 attendees participated including incoming CTF President  George Orfanakos, who met personally with Milken Institute founder, Michael Milken, to share CTF’s goals and programs; and CTF Chief Scientific Officer Dr. Kim Hunter-Schaedle, who presented ‘The Neurofibromatosis Preclinical Consortium’ as an example of an innovative collaboration between foundations, universities and industry.  The Foundation also met one-on-one with a number of potential partners from the investment, biotech and private sectors. A video of the presentation along with the slides can be viewed here: http://www.partneringforcures.org/2010_innovators/29_neurofib.html

For the second year in a row, the Children's Tumor Foundation neurofibromatosis research programs have been recognized and invited for presentation at the Partnering for Cures meeting, which takes place next week in New York. Chief Scientific Officer Dr. Kim Hunter-Schaedle will present in one of the invited "Innovator" slots on the morning of Wednesday December 15th.  Partnering for Cures is organized by FasterCures, an organization formed under the auspices of the Milken Institute to unite non-profit entities focused on medical research  in a forum for information exchange and building collaborations.   This is the second year of the Partnering for Cures meeting, which aims to drive collaboration between non-profit medical research foundations, the pharma and biotech  industry and federal agencies such as the FDA, and investors, in order to accelerate the identification of cures.  Uniquely, at Partnering for Cures, it is the medical research foundations that take center stage, a recognition of  the increasing central role of foundations in advancing research progress to the clinic. As well as two-days of presentations and panels, the meeting provides  an opportunity for one-on-one meetings between potential future collaborators and partners. The CTF Innovator presentation will showcase the Neurofibromatosis Preclinical Consortium (NFPC) in which candidate drugs are assessed in preclinical models of NF1 and NF2 tumor types. NFPC has led to collaborations with companies including Novartis, Genentech and Avila Therapeutics.  NFPC is CTF's biggest investment since 2008 at over $4M, and which we are set to expand for a further two years at $3.5M 2011-2013.  

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).

The Children's Tumor Foundation brings you the Summer Edition of neurofibromatosis highlights from scientific papers published over the past couple of months, including a few from CTF-funded research!  To read the Summer 2010 Neurofibromatosis Research Roundup click here.