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As we have reported here, NIH's proposal to establish a National Center for Advancing Translational Sciences (NCATS) has been met by a mixture of enthusiasm and skepticism from scientists, lawmakers and NIH itself - as evidenced by over 1,100 comments submitted to the NIH website.  Despite these mixed feelings,  NIH director Dr. Francis Collins has announced NCATS will move forward in October witha proposed budget of $700M a year. NCATS will be committed to translational research - or taking basic scientific discoveries forward to develop clinical treatments - normally the domain of biotech and pharma companies rather than the federal government. Many scientists are concerned NCATS will decimate funds available for independent basic research, much of which might ultimately also lead to advancing clinical treatments.   Congress is currently in a 180 day comment period on the proposed reorganization of NIH that NCATS would require.
We will keep watching this story. Read a report from yesterday's Chronicle of Higher Education.

On February 28th 2011, the Foundation will be represented as a partner in this year's Rare Disease Day.

The date will mark the fourth International Rare Disease Day coordinated by EURORDIS and organised with rare disease national alliances in 25 countries. On that day hundreds of patient organisations from more than 40 countries worldwide will be organising awareness-raising activities and converging around the slogan “Rare but Equal”.

Hundreds of patient groups and their partners, coordinated by national alliances at the country level, are planning a multitude of events to draw attention to rare diseases and the millions of people who are affected by them. Awareness-raising activities are being planned across Europe, all the way to Russia, Georgia and Armenia, as well as in the US, Canada and as far as Australia, New Zealand, China and Japan!

for more info, goto: www.RareDiseaseDay.org

For the second year in a row the Children's Tumor Foundation has published the Proceedings from the Neurofibromatosis Conference in the American Journal of Medical Genetics.  The NF Conference is the biggest annual international gathering of NF researchers and clinicians; this meeting has been hosted by CTF for over 25 years. The 2010 meeting in Baltimore, MD was our biggest ever, of over 300 attendees. The 2010 meeting highlights reported in AJMG include the latest updates on NF clinical trials, diagnosis and care; as well as the latest discoveries coming through the pipeline. You can read the 2010 NF Conference publication here. The 2011 NF Conference will be held in Jackson Hole, WY June 11-14. NF researchers and clinicians wishing to attend should click here for more information.

The National Institutes of Health (NIH) proposal to create a new National Center for Advancing Translational Sciences (NCATS) - to be dedicated to accelerating the identification of drug treatments - has promoted media interest as well as questions on whether this will result in cannibalization of other centers at NIH.   NCATS could actually offer significant advantages for neurofibromatosis:  unlike e.g. cancer,  because its a multi-system disorder (affecting many tissues) NF doesn't have one focused NIH institute supporting NF research. Instead NF research funding is spread out across 9 or 10 institutes ranging from NINDS (neurological disorders) to NIAMS (muscular and skeletal diseases) which can have a diluting effect on the impact of any funds invested in NF.  In short -  a focused translational institute could really help advance the development of NF drug therapies. Now NIH is soliciting feedback on NCATS. We encourage you to take a look at the proposal and offer your comments on the Web site. NIH has engaged in a remarkably transparent process to share its thinking and solicit others’, and we know they are taking seriously the commentary they receive.

We hope you will take the time to be part of this important effort. You can start at http://feedback.nih.gov/.

For the second year in a row, the Children's Tumor Foundation is among a small group of foundations invited to speak at the Biotechnology Industry Organization CEO and Investors Conference, today at the Waldorf-Astoria in New York City.   The BIO conference is a forum for emerging biotechs to meet investors and form partnerships with larger pharmas; for CTF, it is a valuable opportunity to present the pipeline of candidate neurofibromatosis drugs currently in preclinical testing and clinical trials, and to engage new industry partners who have drugs that could be future neurofibromatosis treatments.  CTF Chief Scientific Officer Dr. Kim Hunter-Schaedle will present at 3pm. This terrific opportunity builds on CTF's recent invited presentation at the Milken Institute's Partnering for Cures conference which can be viewed here.

Today's New York Times has an interesting article on Louise Benge, a woman who -- along with her two sisters and two brothers -- suffered for years from an unknown debilitating disorder that left doctors baffled.  

Many of those in the NF community may see parallels to their own struggles to find answers for themselves or loved ones.  One of the things we work to do at the Foundation is inform the medical community about NF, diagnostic criteria, and management options, as well as clinics where they can refer patients for best care.  (Those looking for a clinician in their area can visit our Find a Doctor section.) 

But an even more important takeaway from the article is how the study of Ms. Benge's disorder provided scientists with important information that will inform research into treatments for heart disease, osteoporosis and other maladies which affect many more people.  This is true of research into neurofibromatosis as well.  From cancer to bone dysplasia, learning disabilities, deafness, blindness, pain, and the many other manifestations of NF, research the Foundation funds is unlocking critical information that will improve the lives of many individuals beyond those affected by the disorder.

That's not to say we've lost our focus on improving the life of those living with NF, that is our mission and will be until we find a cure, but it is an important concept to keep in mind as we promote, advocate, and work to advance NF research.  

[Times Article]

We are just two days away from the Rolex 24 Hours of Daytona and the chance to cheer on our #4 Children's Tumor Foundation car. It is hard to believe that this all started with two families and a couple of NF Heroes. This year we will have well over 200 people, 91 NF Heroes and will help raise more money then ever before for research. My thoughts tonight are also with the drivers and race team thanking them for their determination and commitment to our cause. A special thanks to all of our sponsors and to everyone who worked hard to increase public awareness of NF and the Foundation. 

Please be sure to check on the #4 car throughout the race and encourage friends and family members to TEXT CTF to 20222 to donate $10 towards NF research. We can all do our part.

This weekend the New York Times featured an article on the efforts of National Institutes of Health Director Francis Collins to set up a new center at NIH that is focused on developing drug treatments. The unit - to be called the National Center for Advancing Translational Sciences - could fill a valuable niche in advancing promising drug therapy ideas that come out of research labs, to a stage where industry might want to invest in taking these ideas to the market. In many ways this is a genius plan that could for conditions like neurofibromatosis build on what NIH foundations like CTF  have funded, but give them the extra fuel to propel them into the marketplace. The Center could also be a training ground for the increasing number of young scientists who want focussed careers on actually finding drug treatments for diseases.  

Many are asking why NIH, a federal agency,  would get into 'business'; and will the $1B investment anticipated divert funds that would otherwise go to academic researchers to pursue their research? The reality is this initiative is still to be fleshed out, so we don't yet have a full picture as to how it would function.  But with industry looking for new drug ideas, and with many Foundations like CTF endeavoring to bridge the gap to bring drugs to the clinic, this initiative could herald a welcome sea change at NIH that might bring benefit to many persons living with conditions which like neurofibromatosis that have no current treatments.

In December  the 111^th Congress could not agree on full appropriations bills, so they passed a continuing resolution (CR) to fund the government through March 4.   At the time our guidance was that this  CR would include funding for NF research in the current fiscal year of 13.75mln (equal to FY10).    Now with the  112^th Congress convening on January 5^th, we are being advised that all non-essential programs are not assured funding through the CR.   Our Government Relations Committee is monitoring the situation closely, and we will be announcing an advocacy plan to support continued funding of the CDMRP-NFRP this year.  Please visit our Advocacy page for more information.

For the second year in a row, the Children's Tumor Foundation will be among a small group of foundations invited to speak at the Biotechnology Industry Organization CEO and Investors Conference to be held Feb 14-15 in New York.   The BIO conference is a forum for emerging biotechs to meet investors and form partnerships with larger pharmas; for CTF, it is a valuable opportunity to present the pipeline of candidate neurofibromatosis drugs currently in preclinical testing and clinical trials, and to engage new industry partners who have drugs that could be future neurofibromatosis treatments.  CTF Chief Scientific Officer Dr. Kim Hunter-Schaedle will present at 3pm on February 14th. This terrific opportunity builds on CTF's recent invited presentation at the Milken Institute's  Partnering for Cures conference which can be viewed here.