Newsroom Archives: Press Releases

Austin, Texas – Beyond Batten Disease Foundation (BBDF) and the New York Academy of Sciences (NYAS) are working together in a new and innovative way to find a cure for a rare disease. While diseases like cancer and Alzheimer’s are widely known, rare diseases like juvenile Batten lack the attention and resulting funds needed to research, develop, and implement treatments. Juvenile Batten even lacks awareness among the medical and research community itself.

To raise awareness among research thought leaders and focus their collective energies on a key topic in juvenile Batten disease research, BBDF Principal Scientist, Danielle Kerkovich, PhD, proposed a unique think-tank of top experts from the Parkinson’s, Alzheimer’s, cancer and Lysosomal Storage Diseases (LSDs) fields to focus on our common drug target—a cell’s lysosome. Lysosomes are the recycling centers of the cell playing vital roles in the removal of cellular waste and keeping critical cell systems in balance. In diseases like juvenile Batten, other LSDs and adult forms of neurodegeneration, lysosome dysfunction compromises multiple cell systems eventually resulting in cell death.

WASHINGTON, DC – March 24, 2015 – Today Genetic Alliance announced the fifteen awardees for Phase I of its initiative to create a ‘white label’ of the Platform for Engaging Everyone Responsibly (PEER). PEER enables individuals to share health information with researchers and each other on their own terms to advance an understanding of health and disease, and to accelerate the development of cohorts for clinical trials. The fifteen selected awardees will work with Genetic Alliance to develop and refine the ‘white label’ PEER while engaging their communities.

Beyond Batten Disease Foundation (BBDF) was one of the fifteen selected awardees. BBDF plans to establish a functional network of existing and emerging Batten registries. Integrating existing registries to standardize and maximize data will provide academic investigators, clinician scientists, Pharma and clinical trialists a critical mass of informative data, accelerate the path to clinical trials, stimulate new research leading to new scientific insights, and provide a platform for patients and families to share their experiences, concerns and wishes.

THE WOODLANDS, TEXAS – Feb. 28. – Where there is a Will, there is a way! Meet the inspirational Will Herndon and his mother Missy at the Woodlands Marathon Saturday, February 28, 2015. Will’s Warriors will run on World Rare Disease Day to support Will, who is fighting a rare disease.

Six years ago, Will and his family received a devastating diagnosis. Will, a loving, bright, energetic 6-year-old, has juvenile Batten disease—a rare, genetic, neurodegenerative disorder. Batten disease attacks an initially healthy child and causes vision loss, loss of cognitive skills, and seizures. Progressively, children suffer loss of memory and speech until they are mentally and physically incapacitated, eventually leaving them wheelchair bound, and then bedridden. With no current treatment or cure, Batten disease is always fatal, often by the late teens or early twenties. Will, now 12, is blind. His family is heart-broken but driven to find a cure.

Austin, Texas– February 4, 2015 – Beyond Batten Disease Foundation (BBDF) https://beyondbatten.org and Brain Canada http://braincanada.ca are pleased to announce a new partnership that will hire researchers in Canada to investigate, explore and find a cure for juvenile Batten disease.

The research project is for $1.5 million over three years. The goal is to further grow the network of international researchers working to find a cure for juvenile Batten Disease. The deadline to submit a letter of intent is February 9, 2015.

“We are pleased to expand our fight against juvenile Batten disease to experts in Canada to transform juvenile Batten disease research and accelerate our timeline to a cure,” said Danielle M. Kerkovich, PhD, BBDF Principal Scientist.

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 6^th 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.

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 5^th 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

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Pianist, vocalist and newest member of Asleep at the Wheel, Emily Gimble has a brand new single, “Mighty Like a Rose,” currently available for downloads on the web at CD Baby.com, iTunes, Amazon and other online distributors. All proceeds benefit Beyond Batten Disease Foundation, a non-profit organization established to eradicate Batten Disease. Often celebrated for her raise-the-roof voice and rambunctious keyboard stylings, Gimble shows her more tender side in this reworking of the 1901 lullaby by Nevin and Stanton. Produced by Floyd Domino, who also takes over on piano, Gimble is accompanied by Ryan Gould on upright bass, J. D. Pendley on guitar and Jonathan Doyle on clarinet.

“This is a most unusual gift for our foundation and has already proved to be a unique fundraiser. For a small amount of money, Emily’s fans can add this gorgeous rendition to their music library and help us spread awareness and research funds for a cure for Batten disease,” said Mary Beth Kiser, president and CEO of the nonprofit foundation.

The musical connections in this story are numerous and unexpected. Emily is the daughter of musician, recording artist and music teacher Dick Gimble and granddaughter of Grammy-winning Texas swing fiddler Johnny Gimble, who played with Bob Wills. Al Strickland also played with Wills and coincidentally taught the finer points of swing piano to Floyd Domino, two-time Grammy winner with Asleep at the Wheel. Additionally, “Mighty Like a Rose” was recorded Bismeaux Studio, which is owned by Ray Benson, Emily’s new boss.

“My mom sang this to me when I was a baby. Her mother sang it to her and I sang it to my two boys,” laughs Mary Beth as she recalls the history of the lullaby in her family. While mastering the song at Terra Nova Digital Audio, owners Jerry and Diane Tubb both remarked that it rang a bell. “That brings back fond memories to me,” said Jerry. The song has been covered over the years but has not sounded so good not since Frank Sinatra’s version from the forties. Floyd and Emily were able to capture the essence of the Harry James classic recording.

Beyond Batten Disease Foundation was established in 2008 by Craig and Charlotte Benson after their five year old daughter, Christiane, was diagnosed with juvenile Batten disease, a rare and fatal inherited disorder that usually begins in childhood. The first symptom is progressive vision loss, followed by personality changes, behavioral problems and mobility issues. Seizures can begin at any time during the course of the disease and tend to worsen with age. There is no treatment or cure.

All proceeds go to the foundation in its quest to find a treatment and a cure for this heartbreaking disease. “Mighty Like a Rose” with vocal by Emily Gimble and an instrumental version can be downloaded at http://www.cdbaby.com/cd/emilygimble12 , Apple’s iTunes Store or on Amazon.

In 2008, local Austin couple Charlotte and Craig Benson received the devastating diagnosis that their then five-year-old daughter, Christiane, had juvenile Batten disease. Batten is a rare, inherited neurological disorder that strikes young children, first causing vision loss and seizures, then cognitive and motor impairment, and ultimately late death by the late teens or 20s. Determined to make a difference, the Benson family established Beyond Batten Disease Foundation (BBDF). The Bensons experienced an outpouring of love and support from their friends and family in the Austin community. Over the past five years, these supporters have continued to give their time, talent and treasure to help the foundation accomplish its mission of eradicating Batten disease. Because of their support, BBDF has been able to make huge strides towards its goal – raising funds and awareness for research and leading development of an easy and inexpensive, groundbreaking blood test to detect the gene mutations that cause Batten disease as well as 700-plus other rare, but serious and often fatal childhood ailments

Beyond Batten Disease Foundation has strong local ties in the Austin community and is proud to announce its participation in Amplify Austin 2014. What began as the inspiration of one child has grown to be the voice for all affected children and their families. BBDF looks forward to accelerating their mission with the help of Amplify Austin and the Austin community. To make a contribution to BBDF through Amplify Austin, schedule a donation today or log on to www.amplifyatx.ilivehereigivehere.org/BeyondBatten on March 20th at 6 p.m. through March 21 at 6 p.m. To learn more about BBDF, visit www.beyondbatten.org

ABOUT AMPLIFY AUSTIN: Last year, the “Amplify Austin” event, powered by Kimbia, raised nearly $3 Million for Central Texas nonprofits in only 24 hours. This hugely successful event benefitted over 300 participating local nonprofits. In today’s changing environment for nonprofits, development executives and community leaders are turning to online-enabled giving days for fresh and highly effective ways to: attract new donors and more funds; drive greater collaboration with nonprofit organizations; and raise community-wide awareness of their work, value and brand. Amplify Austin is an initiative of I Live Here, I Give Here. To learn more, visit www.amplifyatx.ilivehereigivehere.org

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by Jim Cirigliano in Drug Discovery News

published January 10, 2014

NEW YORK—The Alzheimer’s Drug Discovery Foundation has announced a new program that will provide a searchable online marketplace for academic and small biotech researchers seeking contract research organizations (CROs) that provide drug discovery and development services. The program, called ADDF ACCESS, links small biotech and academic research communities to a network of more than 130 CROs that provide industry standard services for central nervous system (CNS) indications.

The program also includes access to drug discovery experts who can provide help in selecting from among the CROs, as well as guidance on the drug discovery process. Registration for the ACCESS program is free.

“The ADDF is regularly approached by scientists in academia and small biotech to provide recommendations for contract research organizations and to provide advice in navigating this sector of the life sciences industry,” says Dr. Rachel Lane, assistant director of scientific affairs at the Alzheimer’s Drug Discovery Foundation. “ADDF ACCESS provides a go-to resource of CROs working in the CNS space and provides guidance on selection and management of CROs.”

CROs have become vital partners for drug discovery and clinical development among programs conducted by academia and small biotech firms that often run on virtual models, Lane notes. CROs are able to offer external validation of data, clinical lab environments that are industry regulated, and drug development expertise.

In addition, CROs have proven valuable to the pharmaceutical industry and private equity groups who use them. Large pharmaceutical companies have developed the means of identifying and managing CRO networks of their own, but academia and small companies typically lack access to these networks or lack the experience to navigate them or use their services effectively.

In addition to providing academic and small biotech researchers access to a selection of CROs, the ADDF program has also negotiated discounted pricing with several of the vendors in the network. Some CROs in the network will provide their services at discounted rates to investigators who are referred through the ACCESS program.

Although the marketplace is not currently a list of “preferred” vendors, the ADDF and a team of consultants are working to perform due diligence on the CROs included in the network. The ADDF has created educational materials designed to guide researchers through best practices for selecting a vendor and managing a CRO contract.

The ADDF is also working to expand the network in the future and has begun building a consortium of nonprofit partners that includes scientists working on many neurodegenerative diseases. BrightFocus Foundation and Beyond Batten Disease Foundation have provided partnership support for the program’s expansion so far, and the ADDF hopes to reach an even broader, multidisciplinary community.

The next step for the program is the completion of its social media platform—a community feature of the site that will allow researchers to relate their experiences with the network of CROs to one another and to engage in an online forum.

“The community feature will provide an online discussion platform and social networking tool to allow users to provide objective feedback on CROs, facilitate discussions on emerging trends in drug discovery for neurodegenerative diseases, allow users to follow companies and create networks of researchers to promote the formation of interdisciplinary teams across CNS therapeutic areas,” says Lane.

The ADDF also looks to continue doing due diligence on the companies in its network to ensure that they are properly categorized according to their core competencies. Looking somewhat farther into the future, the ADDF anticipates developing a platform to highlight promising drug discovery programs within the ADDF portfolio that are available for partnering or licensing opportunities.

The Alzheimer’s Drug Discovery Foundation’s mission is to accelerate the discovery of drugs to prevent, treat and cure Alzheimer’s disease, related dementias and cognitive aging. The ADDF provides funding for drug discovery and clinical development programs in academia and biotechnology companies via a venture philanthropy approach, with funding spanning the translational space from preclinical development through Phase 2 clinical trials.

The ADDF regularly partners with other foundations and industry to leverage resources and funding for its programs. The ADDF has granted nearly $60 million to fund 400 Alzheimer’s drug discovery programs and clinical trials in academic centers and biotechnology companies in 18 countries.

http://www.ddn-news.com/index.php?newsarticle=8044