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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

We mourn the loss of Congressman John Murtha, who passed away on Monday, February 8th, after complications from gall bladder surgery.

A former Marine Corps officer, Rep. Murtha was the first veteran of the Vietnam war elected to Congress in 1974, and focused on military affairs during his long and successful career in Congress.  As Chairman of the defense subcommittee of the House appropriations committee, he was a tireless advocate for our soldiers and their families.

Along with the breast, prostate, and ovarian cancer communities, the NF community owes Rep. Murtha a particular depth of gratitude.  Many of the key discoveries in NF have been made by projects supported by the Congressional Directed Medical Research Program (CDMRP). Rep. Murtha was a key figure in establishing this program in the 90's.  The program's  impact on the health of our military and their families has helped millions of others who have benefited from this work.

The CDMRP is an excellent example of what the federal government can accomplish - the funding has been very well invested and has a history of success that continues to build.   Please visit our Advocacy page (http://ctf.org/How-You-Can-Help/advocacy.html) to learn more and find out how you can help preserve this vital program.

John Risner

Patient advocacy groups like the Children's Tumor Foundation play many roles from funding medical research, providing support, resources and a community 'voice' for patients.  At the Biotechnology Industry Organization 2009 Annual Conference, a forum for pharmaceutical and biotechnology companies, and more recently for patient advocacy groups to interact and partner with these entities, CTF's Chief Scientific Officer Dr. Kim Hunter-Schaedle  featured in a short video highlighting the role of patient advocacy groups. View this now at

http://biotech-now.org/biodigest-patient-advocacy-011790.html

The congressionally funded Phase II Clinical Trials Consortium has just launched a terrific new public website. Three Consortium trials are now open: Lovastatin for learning disabilities; Rapamycin for plexiform tumors; and the newly added trial of Everolimus for optic pathway glioma. For more details on each trial, participating sites and entry criteria, as well as background information on the Consortium, please visit www.nfconsortium.org

The NF Consortium is dedicated to conducting clinical trials to improve the quality of life of persons with neurofibromatosis. The Consortium consists of nine clinical sites around the US and an Operations Center to coordinate Consortium activities at University of Alabama at Birmingham. It was formed in 2006 with funding secured through a Congressionally Directed Medical Research Program which has supported NF research since 1996, in part due to lobbying by individuals and organizations including the Children’s Tumor Foundation that represent the neurofibromatosis community.

Following on from our participation in December's investor-style forum for non-profit foundations, Partnering for Cures, we are delighted to announce that the Children's Tumor Foundation has been invited by the Biotechnology Industry Organization (BIO) to present our neurofibromatosis research programs and drug pipeline  at the 12th Annual BIO CEO and Investors Conference (www.ceo.bio.org) in New York on February 8-9. This meeting is attended by investors seeking investment opportunities with biotech companies.  In the past couple of years a small number of research foundations have also been invited to speak. For the Children's Tumor Foundation this provides us an  opportunity to showcase our programs in preclinical drug screening and clinical trials, and  potentially identify  industry drug collaborations and new sources of funding. More news to follow!

With healthcare reform taking center stage in DC, the defense appropriations bill was pushed well beyond the new fiscal year that began on October 1st.

This year the House included $25 million in their version of the defense appropriations bill which was announced back in June.    This amount was reduced to $13.75 million during the conference process last week to reconcile the House and Senate versions of the bill.  Shortly after the Senate approved the final version of the bill President Obama signed it into law.

While the original $25 million was not preserved, the $13.75 million is a very welcome increase over the $10 million in fy 2009.  This increase comes at a great time, as NF research has made significant gains in the last several years, and is poised to translate these discoveries into new clinical trials.

Please take the time to thank your Representatives and Senators for preserving this vital research funding.

John

The sands are shifting in the landscape of drug development. Large pharmas are merging and consolidating pipelines, quizzing more closely than ever the cost and rationale of clinical trials; some are moving research operations overseas where the work can be done faster and cheaper. Meanwhile small biotechs face a tough investor landscape and many are folding. And the future of healthcare and insurance is under hot debate.  In the face of this change, two federal stalwarts, the Food and Drug Administration (which regulates all US drug approvals) and the National Institutes of Health (the premier funder of medical research in the US) were under the microscope at last week’s Partnering for Cures meeting with discussion panels asking ‘can they do it better?’

Concepts like ‘personalized medicine’ (predicting from your genetic information whether a drug is likely to be effective) are potentially revolutionary but are challenging for the FDA, who have to incorporate evaluation of such new technologies in their review of drug protocols, all the while keeping a weather eye on drug safety. To paraphrase FasterCures founder Mike Milken, the FDA needs to be strengthened the US is to remain competitive in drug development.  To this end the FDA is streamlining its processes (e.g. moving from paper based to electronic document submission), regrouping and increasing staff. Meanwhile the NIH too faces change. Traditionally the 30+ NIH institutes have operated in ‘disease silos’ with institutes focused on one disease area (e.g. mental health) or organ system (e.g. eye). The emergence of genomics has turned this type of thinking on its head and looking forward we expect to see more cross-collaboration between institutes and promote innovative research that can be translated to healthcare such as early stage dug development, and the generation of biomarkers that will help accelerate clinical trials.  On this theme NIH is working in partnership with FDA on a special biomarkers program, building a bridge between the two agencies. This needs to go further, to develop better diagnostics, and rethink clinical trials to be faster and cheaper, all the while keeping a strong communication with the FDA. 

On a related note, some interesting comments emerged at Partnering for Cures about the healthcare system and the fact that (like the NIH) many hospitals operate on an organ based model.  Recent medical knowledge advances in suggest the need to move toward a mechanism based paradigm. Doctors should seek annual CME accreditation in genetics/genomics to be more comfortable in understanding and enrolling patients in clinical trials.

Meanwhile once again patients and foundations step to the fore. Many groups are already ‘driving the bus’ for their disease area (see MMRF in yesterday’s blog) but we can do more: by engaging with the NIH and FDA, educating them on our disease area and how we do clinical trials; and lobbying Congress for more funding and synchrony for the FDA, NIH and healthcare system.   

At last week’s Partnering for Cures meeting, Kathy Giusti (who helms the highly successful Multiple Myeloma Research Foundation) described her initial realization that if things were going to change for multiple myeloma patients, MMRF needed to ‘host the party’  - i.e. take the risk, invest the money, develop the research infrastructure - then call industry to the challenge.  MMRF have pioneered the use of scorecards to keep metrics (measures of progress) on their team of investigators– keeping them on their toes and rewarding success with more funding. It is a controversial concept for some academic researchers but those embracing the MMRF model have had the joy of actually moving drugs forward to the clinic.  

As Debi Brooks (Michael J. Fox Foundation) described, today, donors (or as they are now widely being termed ‘philanthropic investors’) are much more engaged in the day-to-day of research project oversight than in the past, and they too want to see metrics and understand how their dollars are making a difference.  However as there are few if any miracles in life, it is important for the philanthropic investor to understand that success in research is more often incremental (e.g. “now we understand why the drug does not work and have another drug in mind to test”) than mind-blowing (e.g. “drug works, disease cured”).  Today’s philanthropic investors may now have a better acceptance of this risk than in the past, and the fact that there is a possibility of failure as well as success.

An important role for a Foundation is to maintain a ‘Landscape’ of the current state of research in their area (as the Children’s Foundation does), who is investing in it and what has been accomplished, so they can show the philanthropic investor where their ‘investment’ fits into the picture and how it is helping research progress. The Helmsley Charitable Trust has embraced the concepts of risk, money, metrics and landscape to inform their investments in Type 1 diabetes, a disease area that already receives sizeable funding from the federal government and the powerhouse Juvenile Diabetes Research Foundation and has attracted some industry interest. The Helmsley group engaged in a landscape analysis that identified two areas of need that they are now supporting: a mouse model of Type 1 diabetes to truly recapitulate the human condition; and a universal Type 1 diabetes patient registry.

More on Partnering for Cures later this week. 

If you read my blog entries, you know I love to write. Nevertheless a conference that in 2 days inspires me to jot 60 pages of notes must be something. This happened at the first ever 'Partnering for Cures' at the NYC Millenium Broadway this week (www.partneringforcures.org). I have attended and presented at partnering meetings principally designed for biotech and pharma. But Partnering for Cures was the 'partnering meeting from a parallel universe' where the majority of presenters were from foundations - many participants living with the condition they are vying to find a cure for. WE - foundations -  were the main event - and biotech and pharma came to hear what we are accomplishing, most of us on limited budgets but all of us with the same commitment and drive.  This made for a very lively and interactive meeting with a packed agenda, little space to breathe (since over 600 attendees registered, at least half again as many as expected) but lots of new ideas to capture. 

The Children's Tumor Foundation presentation was well received with a decent sized audience (especially given the agenda's opportunity to be in at least 4 places at once!). It was taped so if possible we hope to make it available on our website soon.  We met with a number of new potential collaborators from both the biotech/pharma and other foundations. A number of the discussion panels were terrific, and there was a unique opportunity to hear back-to-back from 40+ other foundations the models they are implementing to find a cure for their disorder. The timing to learn from others is perfect for NF as we look back on the significant progress that both the Foundation and the NF community at large have made in the last couple of years, and as we map out future steps and goals. 

Given the amount of information that came out of Partnering for Cures, I will be doing a couple of blog pieces in the next week focused on different areas and topics - so watch this space.

In a unanimous vote (how often does that happen?) the Senate has confirmed President Obama's choice of Dr. Francis Collins to head the National Institutes of Health.  

Dr. Collins and his team at the Univ. of Michigan, along with Dr. Ray White at the Univ. of Utah, discovered the gene that causes NF1 in 1990.   The Foundation is proud to have funded this research through our Young Investigator Awards in the late 80's to Dr. Peggy Wallace (Univ. of Michigan) and Dr. Dave Viskochil (Univ. of Utah) who were post doc's in their respective labs.   In addition to NF1, he has helped discover the genes that cause cystic fibrosis, Huntington's disease and the M4 type of adult acute leukemia.

Dr. Collins succeeded Dr. James Watson as head of the National Human Genome Research Institute (NHGRI), in 1993, and held this post until 2008.  In that period, he led the Human Genome Project, which along with Dr. Craig Venter,  were the first to  sequence the entire  human genome.  In recognition of his leadership he was awarded the Presidential Medal of Freedom by President George Bush in 2007.

In addition to numerous published research articles, Dr. Collins is the author of the 2006 book The Language of God:  A Scientist Presents Evidence for Belief, which reconciles religious faith and scientific pursuit.

We could not agree more with HHS Secretary Kathleen Sebelius statement that  "Dr. Collins is one of our generation's great scientific leaders.  He will be an outstanding leader. Today is an exciting day for NIH and for science in this country."

John