Beyond Batten Disease Foundation and the New York Stem Cell Foundation Chosen as a National Innovator by the Milken Institute as They Partner to Find a Cure for Juvenile Batten Disease
The Battle is Personal for a Texas Father Who is Trying to Save His Own Daughter’s Life
New York City, New York – November 17, 2014 – Beyond Batten Disease Foundation (BBDF) and the New York Stem Cell Foundation (NYSCF) have been selected as a national innovator by the Milken Institute and will present their breakthrough findings about juvenile Batten disease at the 6th annual Partnering for Cures, November 16-18 in New York City.
Craig and Charlotte Benson established Beyond Batten Disease Foundation in August 2008 after their then five-year-old daughter, Christiane, was diagnosed with juvenile Batten disease. Together with hundreds of families affected by Batten disease, and many more supporters who share their hope and resolve, they are working tirelessly to create a brighter future for Christiane, and all children with Batten disease.
The Mission to Save Will Herndon
By Tish Williams in Houston Lifestyles & Homes
Published November 1, 2014
The Community Has Contributed $3 Million to Find a Cure for Juvenile Batten Disease
When Missy Herndon received the call with her son’s test results, the genetic counselor cried over the phone. That’s when it hit her that something was really wrong.
Will was a normal kindergartener. When he held papers closer to his face, Missy and her husband Wayne thought he might need glasses. Then he forgot his alphabet and had to relearn it starting from “A,” but kids do silly things. When a second visit to the eye doctor returned a referral for genetic testing, they began to have some doubts. Still, the Herndons looked to a diagnosis for clarity, the first step to fixing the problem and getting Will back on track.
“I remember the day. The kids were at WoW Science Camp when my phone rang. Picking it up, I was quite confident they had found nothing. I thought, ‘I have this active 5-year-old.’ The genetic counselor started crying on the phone,” Missy says. “I just knew it was horrible.”
NYSCF Research Institute announces largest-ever stem cell repository
On Phys.org
Published on October 22, 2014
The New York Stem Cell Foundation (NYSCF) Research Institute, through the launch of its repository in 2015, will provide for the first time the largest-ever number of stem cell lines available to the scientific research community. Initially, over 600 induced pluripotent stem (iPS) cell lines and 1,000 cultured fibroblasts from over 1,000 unique human subjects will be made available, with an increasing number available in the first year. To collect these samples, NYSCF set up a rigorous human subjects system that protects patients and allows for the safe and anonymous collection of samples from people interested in participating in research.
Hair Crafters holds 18th Annual ‘Cut for a Cause’ event
On WDNU.com
Published October 19, 2014
Blue extensions and $20 haircuts drew folks to Hair Crafters Sunday afternoon for the 18th Annual “Cut for a Cause.”
This year the money raised goes towards research for Batten disease, a rare and fatal neurodegenerative disorder that begins in child hood.
NewsCenter 16 has covered the story of Tyler Allman, and “Team Tyler” who have championed awareness for the illness.
Tyler has been fighting batten disease for five years now; he is confined to a wheelchair.
There is no cure for Batten.
However, fundraisers like “Cut for a Cause” help spread awareness to this type of rare disease.
Royer Allman and Heather Allman, “Team Tyler” parents, say, “it was definitely a curveball at first. Tyler was a typical boy that played t-ball was just a normal child, but now he suffers from seizures, he’s blind, he’s lost all of his mobility, so it is a neurodegenerative disease. Today is better than tomorrow, but we just embrace each day and he has such an amazing spirit. And that’s our goal is to keep doing that for him as well as raising money and awareness.”
Hair Crafters raised more than $800 within the first hour of Sunday’s event, and hoped to raise much more throughout the day.
Top Research Collaborations Explain How They Are Speeding the Path to Cures
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)
5th Grade Hope
Back to school always marks the end of summer with anticipation and excitement for a new season for children. Even parents welcome the new routine. New beginnings abound for children of all ages and suddenly we find ourselves in a new place. Kindergarten gives way to first grade, elementary gives way to middle school, and on it goes until our kids finally go off to college or into the real world and we wait breathlessly for the results of all of our hard work.
I once heard that raising children is like having a herd in a green pasture surrounded by a secure fence. They are free to explore and discover within the boundaries of the fence. Every year, our job as parents is to expand their boundaries and responsibility so they can become more and more independent. The idea is to increase their limits and make room for their own decisions so they can discover success and failure, and learn to grow from those experiences. Our job is not to control and dominate them, but rather to allow them the freedom of making their own choices as we continue expanding their pasture until they are capable of functioning independently and responsibly without the fence.
Beyond Batten Disease Foundation partners with NCL Foundation to award $344,742 to research team of Dr. Marco Sardiello, Baylor College of Medicine, Houston
As part of a larger $1.75M grant, BBDF continues to support Dr. Marco Sardiello’s research, investigating the role of CLN3 the juvenile Batten disease gene, CLN3, in Juvenile Neuronal Ceroid Lipofuscinosis (JNCL).
Recognizing the promise of BBDF-funded research, NCL Foundation in Hamburg, Germany, awarded the 5th NCL Research Award to Dr. Marco Sardiello and his team at Baylor College of Medicine in Houston, Texas. The award, which amounts to $133,942, will finance a postdoctoral fellow, Dr. Alberto diRonza, whose work will be dedicated to unraveling the primary role of the normal CLN3 protein and the lysosomal defects that result in Batten disease. NCL’s funding is in conjunction with BBDF’s funding of $210,800 to Dr. Marco Sardiello and his team. With these combined funds, the researchers hope to gain insight into the role of CLN3.
Dr. Marco Sardiello and his team are dedicated to the research and development of innovative therapies to treat lysosomal diseases (LDs), including juvenile Batten disease. LDs are the most common childhood neurodegenerative diseases. The majority of LDs are caused by defects in one of over 60 known soluble lysosomal enzymes or 25 transmembrane proteins in the outer layer of lysosomes. Lysosomes are cellular organelles that play a key role in the degradation and recycling of cellular organelles, proteins, lipids and other substances. Therefore, defects in genes that encode lysosomal proteins result in excessive accumulation and devastating effects.
Complicating treatment, the normal CLN3 gene (when mutated, causes juvenile Batten disease) encodes a transmembrane protein. Therefore, candidate therapies targeting soluble enzyme deficiencies, such as enzyme replacement therapy, bone marrow transplantation, or gene therapy, are generally not considered for treating juvenile Batten.
Dr. Sardiello and his team are investigating transcription factor EB (TFEB). Dr. Sardiello was part of a team which discovered TFEB is a master gene in the network regulating the biogenesis and activity of lysosomes and that inducing TFEB can increase lysosomal clearance in certain disorders. This discovery and its continued study show potential in the treatment of juvenile Batten disease.
About Beyond Batten Disease Foundation
Beyond Batten Disease Foundation (BBDF) is the world’s largest organization dedicated to funding research for a treatment or cure for juvenile Batten disease. Batten disease is a rare, inherited pediatric neurological disorder, which begins with vision loss and seizures, followed by cognitive and motor impairment, and ultimately death by the late teens or 20s. Since its inception in 2008, BBDF has raised over $14.6 million for research through donations, co-funding, leveraging and partnerships. BBDF is spearheading a unique, cohesive strategy, incorporating independent scientific resources and collaboration with related organizations to drive research in Batten Disease. For more information, visit www.beyondbatten.org.
About NCL Foundation
The NCL Foundation was founded 2002 by Dr. Frank Husemann, after his then 6-year-old son Tim was diagnosed with JNCL. Neuronal Ceroid Lipofuscinosis is a rare metabolic disorder which leads to a progressive loss of nerve cells. It is the most common form of childhood dementia. Affected children suffer from neurodegeneration affecting different types of neurons, also in the retina, and this results in early blindness, mental deterioration, loss of motor function and the development of epileptic seizures. Many NCL patients do not survive past their third decade. www.ncl-foundation.com
Press contact: Mary Beth Kiser, President & CEO, Beyond Batten Disease Foundation, 512.275.2600
Making a difference: Beyond Batten Disease Foundation
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.