BBDF Blog

By FasterCures

Published October 8, 2014

WASHINGTON, DC–(Marketwired – Oct 8, 2014) – Thirty of the most creative, cross-sector collaborations in medical research will present their models to potential partners and funders at FasterCures’ sixth annual Partnering for Cures meeting, November 16-18 in New York City. Selected through a competitive proposal process, these partnerships are aimed at reducing the time and cost of getting new medical solutions from discovery to patients.

“The quality of our over 120 applicants — more than twice the submissions of previous years — was terrific and made for a particularly difficult decision-making process,” said FasterCures’ Executive Director Margaret Anderson. “These collaborations address some of the thorniest issues in medical research using models that can be scaled and translated across diseases and sectors.”

From re-imagining clinical trial infrastructure to improving and expanding data sharing to creating the tools and resources needed to translate basic science into cures, these innovators are accelerating the path from lab to market for novel — and needed — therapies.

For example, 2014 presenters include:

• The first-ever public-private partnership focused on advancing the regulatory science of medical devices
• An effort that harnesses the power of high-performance computing to “hack” brain disease
• One of the first clinical trials to address coverage with evidence development
• A collaboration that blazes a trail for successful reimbursement of disruptive technologies
• An outcome assessments consortium that identified and validated a new measure of disability for use as a primary endpoint in multiple sclerosis trials
• And many more…

With nearly one-third of the 120 applicants indicating that their collaborations were either established or enhanced as the result of participating in past Partnering for Cures, the meeting remains a key venue for innovators across the ecosystem to unite and share ideas.

We invite you to attend these presentations and glimpse the future of medical R&D. By engaging patients as trusted partners in the drug development process and by working together — across labs, companies, borders and sectors — these partnerships provide a shining example of transformation in action.

Contact Cecilia Arradaza (carradaza@fastercures.org) for a complimentary media registration code and join us in NYC.

2014 Innovator Presentations at Partnering for Cures

1. Addario Lung Cancer Medical Institute: Remote clinical trials – The genomics of young lung cancer study
2. ALD Connect
3. Alzheimer’s Association: Amyloid Imaging Coverage with Evidence Development Workgroup
4. Boston Biomedical Innovation Center: NIH Centers for Accelerated Innovation
5. Brave Bosom: Free the Data
6. Catalent Institute: Non-invasive Macromolecule Delivery Consortium
7. Christopher & Dana Reeve Foundation: The Big Idea
8. CureDuchenne: Accelerating Access to Treatments of Duchenne Muscular Dystrophy
9. Focused Ultrasound Foundation: Blazing a trail for successful reimbursement of disruptive technologies
10. Foundation for the National Institutes of Health: THE MAL-ED NETWORK – Sharing data and resources to gain a better understanding of child growth and development
11. Give To Cure
12. Hearing Health Foundation and Oregon Health & Science University: The Hearing Restoration Project
13. HemoShear and Children’s National Health System: Rare Disease Drug Accelerator – A new paradigm to accelerate drug discovery for the treatment of rare diseases
14. Human Vaccines Project
15. Indiana Clinical and Translational Science Institute: Strategic Pharma-Academic Research Consortium (SPARC) for Translational Medicine
16. IO Informatics: ASK for PROOF (Applied Semantic Knowledgebases for Prevention of Organ Failure)
17. Johns Hopkins University: The Neurofibromatosis Therapeutic Acceleration Program
18. Locemia Solutions, T1D Exchange, and The Helmsley Charitable Trust: Partnership for development of Dry-Mist Nasal Glucagon
19. Mayo Clinic: Partnership for development of novel treatments for heart valve disease
20. Medical Device Innovation Consortium
21. Medical Research Council Technology: MRCT Dementia Consortium – New models to accelerate novel medicines to patients
22. National Multiple Sclerosis Society: Multiple Sclerosis Outcome Assessments Consortium (MSOAC)
23. The New York Stem Cell Foundation and Beyond Batten Disease Foundation: Multi-stakeholder collaboration to discover cures for juvenile Batten disease
24. The Ontario Brain Institute: An integrated system of partnerships
25. Orion Bionetworks: Hacking brain disease for a cure
26. Patient-Centered Outcomes Research Institute: Engaging patients as partners to create PCORnet
27. Seattle Children’s Research Institute: Alliance for Children’s Therapeutics
28. Solve ME/CFS Initiative: How to build your evidence base
29. Structural Genomics Consortium and CHDI: Open access partnership for Huntington’s Disease research
30. University of California, San Francisco Clinical and Translational Science Institute and Quest: Dementia Program

About FasterCures

FasterCures, a center of the Milken Institute, is an action tank determined to remove barriers to medical progress. We have only one goal: to save lives by speeding up and improving the medical research system. (www.fastercures.org)

http://www.marketwired.com/press-release/top-research-collaborations-explain-how-they-are-speeding-the-path-to-cures-1955901.htm

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By Amanda Koziel in Life in the Heights

Published September 2014

The Beyond Batten Disease Foundation is a foundation near and dear to many individuals and families in the Pemberton Heights neighborhood. Residents Craig and Charlotte Benson established the foundation in August 2008 after their then five- year-old daughter, Christiane, was diagnosed with juvenile Batten disease. Batten disease is a rare, fatal, inherited disorder of the nervous system that usually begins in childhood. The family began tirelessly working with supporters in the neighborhood and beyond to find a cure for this disease and help families everywhere with children with batten disease.

The amount of progress that the foundation has made is impressive, with great strides that have been made since its inception. Beyond Batten Disease Foundation is spearheading a unique strategy that incorporates independent scientific resources with related funding organizations to drive the research into eradicating Batten Disease. Mistakes in the DNA that cause the disease have already been identified, and some researchers have even been able slow the disease in animal models. BBDF has have been able to do this more efficiently than many other groups out there, but there still is much work and research to be done.

The foundation recently developed a Beyond Batten Development Board to help collaborate and create effective strategies for reaching out more broadly to help spread the word and gain more supporters.  Resident Sabrina Brown is the current President of the BBDF and Kelley Daniel is the President -Elect. Kelley Daniel explains “It has been very rewarding to see all of our efforts during this inaugural year come together. From Team Captains, to exchange station coordinators, to kids 2k volunteers, the Development board pulled together and raised $330,000 during the Run to the Sun, which was our main fundraiser this year. I know that with each and every event that the Development Board plans, we will be able to extend our reach and raise both awareness and funds for the research that we need to find a cure.”

When asked about the Run to the Sun, which resident Lance Thompson envisioned, Chair-elect of the event Jimmy Windham explains how fun and meaningful of an experience the event is. The symbolism of running through the night and ending the race in the morning is a symbolic of defeating one of the first noticeable signs of juvenile Batten disease, which is vision loss. The Run to the Sun typically takes place in April and this past spring also included a 2K fun run that is a great way for families to volunteer together.

When asking Kelley what would be something that the neighborhood might not know about the BBDF she had some great feedback. “The Beyond Batten Disease Foundation states that its mission is twofold. First, Prevention and Diagnosis of Bat- tens Disease and, second the Treatment and Cure of Battens Disease. While Battens Disease affects a small number in the population, it is part of a larger group of neurodegenerative diseases some of which are Multiple Sclerosis, Parkinson’s and Alzheimer’s. Our work helps not only research for Battens Disease, but all other neurodegenerative diseases. Our research has revealed that all of us carry 3 and maybe more gene mutations that can cause devastating rare diseases.  So our work at the foundation is far reaching, and directly affects all of us.”

We look forward to seeing the continued progress of this foundation and all it is doing to make waves for the cure of Batten Disease.  For more information on how to support the foundation, please visit beyondbatten.org.

To view the full article in Life in the Heights, click here.

By Bob Gunner in The Paper

Published September 4, 2014

THE WOODLANDS, TX (September 4, 2014) – No treatment. No cure, fatal. These words began the movement of HOPE back in August of 2009 to save then 6 year old resident of The Woodlands, Will Herndon. His diagnosis of the rare, genetic, neuro-degenerative condition called juvenile Batten disease launched a grass roots effort that has since raised $2.5 million locally to fund the most promising research in the history of the disease. This success and progress will be celebrated at the annual Texas chic–themed gala, HOPE Under the Stars on Saturday, September 20, 2014 at The Woodlands Waterway Marriott & Convention Center.

Juvenile Batten disease is a condition that affects an otherwise healthy child with blindness, seizures and memory loss. Overtime, children lose their cognitive functions and mobility, leaving them suffering with dementia and wheelchair-bound, then bedridden. Children that formerly ran, laughed and sang, progressively lose everything. Currently fatal in the late teens or early 20’s, this prognosis is unacceptable for Will Herndon and the hundreds of children affected with this condition.

“We are so blessed to live in a community that has passion to believe that even the impossible, can become possible if you truly believe change can be made,” said Missy Herndon, Will’s Mother and founder of The Will Herndon Research Fund at Beyond Batten Disease Foundation. “I am so grateful for the tremendous support we have received from the families of The Woodlands. Our success is a testimony to the resources, love and faith each of them have shared since day one of Will’s diagnosis. There is no doubt in my mind this community is committed and a life-changing discovery will happen.”

The Will Herndon Research Fund joined forces with Beyond Batten Disease Foundation in 2009 with a shared mission: to find a treatment for juvenile Batten disease. Later that same year, the foundation funded a team of Italian researchers to focus on the disease at the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital. The foundation also funded the largest grant ever designated for juvenile Batten disease research; $2.5 million. Since that time, the foundation has raised over $13.2 million for Batten research through direct donations, co-funding and sponsorships. Currently, these researchers have identified six drug compounds and are finalizing research plans to move those into clinical studies.

HOPE Under the Stars began as a local, grassroots event for The Will Herndon Research Fund for Juvenile Batten Disease at Beyond Batten Disease Foundation in the Rob Fleming Park five years ago with a mission of HOPE to help fund a treatment or cure for this devastating and fatal condition affecting Will Herndon, of The Woodlands. This year, expecting a crowd of more than 800 guests, the 6th Annual HOPE Under the Stars event will be held at the largest indoor venue in our area, to accommodate a growing crowd of continued and new supporters. The Texas chic event will be at The Woodlands Waterway Marriott Hotel and Convention Center in The Woodlands, Texas on Saturday, September 20, 2014. Doors open at 5:30 pm with a cocktail reception and silent auction. Missy and Wayne Herndon, Will’s parents, will kick-off the program at 7 pm with a research update while guests enjoy a southwestern inspired dinner. The celebration will celebrate the amazing support of The Woodlands community raising more than $2.5 million dollars since The Fund’s inception, 5 years ago. A live auction will follow the presentation and much loved New Orleans brass band, The Bucktown All-Stars, will entertain the crowd. Throughout the evening, guests will have the opportunity to ride a mechanical bull, capture the moment with a “Selfie” mirror and pose for a souvenir “butt sketch”. A special “HOPE Lounge” created especially for sports fans, will feature a cigar bar, casino tables and showcase the evening’s SEC Football game. Adjacent to the dance floor will be a “HOPE Hideaway” where guests can take a moment away from the excitement to catch up with friends, new and old. For more information, or for HOPE Under the Stars Tickets and Sponsorships, please visit: www.willherndon.org.

The Will Herndon Research Fund at Beyond Batten Disease Foundation is based in The Woodlands, TX and has multiple fundraising and awareness events locally throughout the year.

To read article on The Paper, click here.

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