Beyond Batten Disease Foundation Announces Formal Sponsorship of Care Beyond Diagnosis

 

Beyond Batten Disease foundation announces formal sponsorship of Care Beyond Diagnosis in effort to establish Clinical Management Guidelines for CLN3 Disease. 

Austin, TX – Raleigh, NC, September 27, 2021 – Beyond Batten Disease Foundation (BBDF) is pleased to announce a sponsorship agreement with Care Beyond Diagnosis (CBD) to establish internationally validated management guidelines for CLN3 Disease, an ultra-rare, fatal neurodegenerative genetic disorder.

Guidelines which have been methodically and transparently developed can both establish and improve upon a standard of care for patient management. Evidence and consensus-based recommendations for care can improve access to effective management and improve the reimbursement process by recognizing medical necessity.

“Having the support of BBDF is a huge achievement for our team. Obviously, the financial commitment is important for us to keep the program going, but looking ahead towards the publication of the guidelines, BBDF will be instrumental in dissemination of the guidelines into family/caregivers’ hands” says Care Beyond Diagnosis Co-CEO, Jeffrey Donohue. “Engagement from the patient community is a necessity in guideline development.”

President and CEO Mary Beth Kiser says, “BBDF has been keenly interested in guidelines for the CLN3 community for many years.  Thanks to the work of Care Beyond the Diagnosis we are able to meet that need and know this will be a valuable resource for our dedicated community of physicians.”

The development program which began in October 2020 is on track to be completed by year end – 2022 ready for submission to a medical journal. CBD predicts a publication will be available for review by Q2 2023.

The effectiveness of clinical guidelines depends on editorial independence, transparency of process and a validated design. CBD organizes its guideline programs in accordance with the Appraisal of Guidelines for Research and Evaluation’s AGREE II Tool, a 23-point assessment used to assess clinical guidelines.

In the rare disease community, research is by nature, is lacking. A guideline development program identifies evidence gaps in existing literature, but through a Modified Delphi Consensus Program, generates new evidence where those gaps exist.

Guidelines for Rare Disease is currently an unmet need within the community. With over 7,000 rare diseases and a vast majority currently without a disease altering therapy, guidelines can offer caregivers and clinicians a tool to provide patients with the highest QoL possible.

CBD published guidelines for CLN2 Disease in April of 2021. The program was assessed by independent reviewers according to the AGREE II Tool and received a 5.7 out of 7, according to Donohue, was the highest assessment for a rare disease guideline publication.

About Beyond Batten Disease Foundation: Beyond Batten Disease Foundation (BBDF) is the world’s largest non-profit organization dedicated to funding research for a treatment and cure for juvenile (CLN3) Batten disease. Since its inception in 2008, over $35 million has been invested in research by leveraging donations, co-funding and strategic partnerships. BBDF is spearheading a unique, coordinated strategy that incorporates independent scientific resources and collaborates with related organizations to drive research in CLN3 disease. More information can be found at www.beyondbatten.org.

About Care Beyond Diagnosis: Care Beyond Diagnosis (CBD) is a non-profit organization dedicated towards supporting patients, families and communities following a rare disease diagnosis. CBD has a vision for improved patient-outcomes through both a  small scale, patient specific approach and through large scale, internationally collaborated Rare Disorder guideline development programs.

www.carebeyonddiagnosis.org

BBDF 101 Wins FDA Investigational New Drug Approval to Pursue Clinical Trials

Lyon, 7 September 2021 – Theranexus, a biopharmaceutical company innovating in the treatment of neurological diseases, and Beyond Batten Disease Foundation (BBDF), announce receipt of Investigational New Drug (IND) approval from the Food and Drug Administration (FDA) to launch a Phase I/II clinical trial of their drug candidate BBDF 101 for juvenile Batten disease, a rare, fatal, genetic disorder.

Batten disease belongs to a group of disorders referred to as neuronal ceroid lipofuscinoses (NCLs).

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BBDF Announces Agreement with Actelion Pharmaceuticals Ltd to Provide Drug Product for BBDF-101

Austin, Texas, March 2, 2021

Beyond Batten Disease Foundation (BBDF) is pleased to announce an agreement with Actelion Pharmaceuticals Ltd, a Janssen Pharmaceutical Company of Johnson & Johnson, to provide Zavesca® (miglustat) for the development of BBDF-101, a proprietary combination of miglustat and trehalose to treat juvenile Batten disease (CLN3). Janssen will provide drug supply for the clinical trials and Extended Access Program, and right of reference to data within the Zavesca New Drug Application (NDA) to support the development of BBDF-101.

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BBDF-101 Announcement

We are excited to announce the following major milestone for BBDF!

The FDA has awarded Orphan Drug and Rare Pediatric Disease designations to Beyond Batten Disease Foundation for BBDF-101. The aim of the program is to facilitate the development of new drugs and biological products for the prevention and treatment of rare pediatric diseases. These designations provide accelerated review for approval, support with the FDA regulatory process and at least seven years of post-approval protection, as well as exemption from filing fees that normally have to be paid to the FDA. These designations do not impact the clinical trial, but are beneficial once the therapy receives New Drug Approval. We are grateful to Theranexus for their continued partnership and commitment to initiating a clinical trial for BBDF-101 as quickly as possible.

THERANEXUS AND BBDF OBTAIN ORPHAN DRUG DESIGNATION (ODD) AND RARE PEDIATRIC DISEASE DESIGNATION (RPDD) FROM THE FOOD AND DRUG ADMINISTRATION (FDA) FOR BBDF-101 FOR BATTEN DISEASE

  • Orphan Drug Designation (ODD) is a status that provides seven years of additional post-approval protection and exemption from filing fees
  • Rare Pediatric Disease Designation (RPDD) qualifies the sponsor at the time of registration for a salable, transferable priority review voucher which can be used to speed up the approval process for another drug candidate

Lyon, August 28, 2020 – Theranexus, a biopharmaceutical company innovating in the treatment of neurological diseases, and Beyond Batten Disease Foundation (BBDF) today announced the decision by the Food and Drug Administration (FDA) to award Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) to the drug candidate BBDF-101 for Batten disease, a rare, fatal, genetic disorder of the nervous system for which there is no treatment.

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BBDF-101 Announcement

We are excited to announce the following major milestone for BBDF! Theranexus, a publicly-traded European pharmaceutical company, has committed the $20 million and expertise necessary to complete the clinical trial and commercialization for our drug discovery, BBDF-101.

Theranexus and Beyond Batten Disease Foundation (BBDF) announce the signing of a worldwide exclusive license for Batten disease drug candidate BBDF-101
  • The agreement covers the development and marketing of the drug following a single clinical trial due to begin in 2020. 
  • If successful, this trial will lead directly to the drug’s approval.

Lyon, 12 December 2019 – Theranexus, a biopharmaceutical company innovating in the treatment of neurological diseases and pioneer in the development of drug candidates modulating the interaction between neurons and glial cells, is pleased to announce the signing of an agreement with Beyond Batten Disease Foundation, granting it a worldwide exclusive license to develop and commercialize the drug candidate BBDF-101 for juvenile Batten disease.

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A Brother, A Sister and the Search for a Deadly Disease’s Cure

 

Below is an article that was written at the beginning of October. It’s a great summary of Garland’s effort to help his sister, Christiane. At the time of the writing, Garland’s fundraising total was about $90,000 and we are excited to report that his total is now over $151,000!

 

Garland Benson, a freckled-face 12-year-old with a winning smile, is on a mission – one that’s very personal, but could have far-reaching implications.

He just needs a little help from 100,000 friends and strangers.

Here’s why: Garland’s 15-year-old sister, Christiane, has juvenile Batten disease, a rare, fatal, inherited neurological disorder. It’s the same disease that was responsible for the July deaths of three siblings over the course of three days in Utah.

Children with the incurable disease suffer progressive vision loss, commonly followed within a couple of years by seizures and, as time passes, the loss of motor functions and speech. That’s followed by premature death, usually in the late teens or early 20s.

In 2008, the parents of Garland and Christiane, Craig and Charlotte Benson, formed the non-profit Beyond Batten Disease Foundation (www.beyondbatten.org) with the goal of helping find a cure. Through research funded by the foundation, progress is now being made on a treatment that, although not a cure, could slow down the disease’s progression, perhaps buying time for young people like Christiane.

The foundation is trying to raise $6 million that’s needed to help hurry that research along by getting it through an expensive validation and development process so that it can receive FDA approval for clinical trials.

Enter Garland, a boy with a big heart for his sister and an understanding that small gifts can add up to extraordinary amounts if the multiplication factor is large enough.

“I decided to raise $1 million of that $6 million on my own,” he says. “If I can get 100,000 people to give $10 each I’ll have $1 million. I think we can easily do that.”

With his goal clear, Garland confidently set out in March to achieve his objective – and do whatever he could for his sister and other young people who have the same disease.

Many donors have left heartfelt messages for him, such as: “Garland, you are the sweetest, best brother a girl could have.” “You inspire me to make a difference.” “Thank you for blessing your sister and so many others.”

Garland quickly found that his self-imposed undertaking wasn’t quite as easy as he hoped, but he has persevered and so far has raised about $90,000. His optimism isn’t faltering yet.

“I think it would be amazing if we find a cure,” Garland says.

Although Batten is genetic, and families can have more than one child with the disease, Garland does not have it. He’s not a carrier either, so he can’t pass it on to his children.

Despite the disease, Christiane is a cheerleader, has joined her family on ski trips and on horseback-riding adventures, and continues to be an inspiration for her younger brother.

“It’s really cool how she can do all that stuff and not be able to see,” Garland says. “But what I’d really like is for her to be able to lead a normal life like me.”

About Garland Benson

Garland Benson, 12, lives in Texas and is trying to raise $1 million to help fund research into juvenile Batten disease, which afflicts his sister, Christiane. The money is being raised through the Beyond Batten Disease Foundation (www.beyondbatten.org), which was launched in 2008 by the parents of Garland and Christiane, Craig and Charlotte Benson. The foundation’s goal is to help eradicate the disease by raising awareness about Batten and helping to fund research that could eventually lead to a cure. Those who want to donate can text the word HERO to 501051 and $10 will be added to their next cell phone bill. Donations can also be made through Garland’s donor page on the Beyond Batten Disease Foundation’s website.

 

 

New York Stem Cell Foundation and Beyond Batten Disease Foundation Announce the Availability of Stem Cell Lines for Research

The New York Stem Cell Foundation (NYSCF) and Beyond Batten Disease Foundation (BBDF) today announce the availability of a unique and comprehensive collection of juvenile Batten (CLN3) disease induced pluripotent stem (iPS) cells. The two organizations partnered to develop this resource for scientists around the world. The collection is a key example of BBDF’s strategy to advance therapy development for Batten disease by creating and disseminating cutting-edge tools and other resources with game-changing potential.

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BBDF Welcomes Lauren Trice as Director of The Will Herndon Research Fund

FOR IMMEDIATE RELEASE

Contact:
Missy Herndon
Founder, The Will Herndon Research Fund
hope@willherndon.org
713-557-0219

LAUREN TRICE NAMED DIRECTOR OF THE WILL HERNDON RESEARCH FUND AT BEYOND BATTEN DISEASE FOUNDATION

 

THE WOODLANDS (January 12, 2017) – Beyond Batten Disease Foundation (BBDF) is pleased to announce its recent hire of Lauren Trice, who joins the team as the Director of The Will Herndon Research Fund. In this role, Trice will oversee the management, development, and operations for the fund.

“I am honored and humbled to serve during a critical time for the research of juvenile Batten disease,” says Trice. “I believe that with the continued support of our community, we will raise awareness and funds to accelerate the research for a treatment or cure.”

Trice, a resident of The Woodlands, comes to BBDF with experience in various marketing and public relations capacities since 2006. Most recently, she served as the Corporate Market Director for the American Heart Association (AHA) in Montgomery County. In this role, she was awarded the Rome Betts Award of Excellence, which is the most prestigious national staff award given by the AHA.

“Lauren brings a wealth of knowledge, skills and a mission-based focus to our organization that we feel confident will help us meet our ultimate goal to fund a life-saving treatment to save Will and the hundreds of other children affected with this disease,” said Missy Herndon, founder of The Will Herndon Research Fund. “Collaborative research efforts have led us to potentially one of the most promising treatments in the history of juvenile Batten disease. We are grateful to have Lauren leading the local efforts to help see this research come to fruition in Will’s lifetime.”

For more information on The Will Herndon Research Fund, visit www.willherndon.org or www.beyondbatten.org.

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ABOUT THE WILL HERNDON RESEARCH FUND

The Fund was established in 2009 by Missy and Wayne Herndon, in honor of their son Will, who was diagnosed with juvenile Batten disease at the age of 6.  This rare, fatal, genetic disease attacks an otherwise healthy child, beginning with vision loss, declining cognitive skills and seizures. Batten disease is fatal, most often by the late teens or early twenties. Our goal is to SAVE WILL, and the hundreds of other children stricken with juvenile Batten disease. We have HOPE, but we need your help in this race against time. To learn more or to get involved, call 832-610-5995, or visit willherndon.org. 

ABOUT BEYOND BATTEN DISEASE FOUNDATION

Beyond Batten Disease Foundation (BBDF) is the world’s largest nonprofit organization dedicated to funding research for a treatment and cure for juvenile (CLN3) Batten Disease. Since its inception in 2008, over $16 million has been invested in research by leveraging donations, co-funding and strategic partnerships. BBDF is spearheading a unique, cohesive strategy, incorporating independent scientific resources and collaboration with related organizations to drive research in juvenile Batten Disease. Today there is a treatment in sight. BBDF funded research has discovered a drug that slows the progression of the disease in Batten models. The foundation has launched a 24 month $6 million campaign to advance the treatment to clinical trials. More information can be found at www.beyondbatten.org.

Beyond Batten Disease Foundation Announces New Funding for Metabolomics to Drive Biomarker Discovery in juvenile Batten disease

Beyond Batten Disease Foundation Announces New Funding for Metabolomics to Drive Biomarker Discovery in juvenile Batten disease

 

AUSTIN, TX –January 9, 2017—Beyond Batten Disease Foundation has launched into high gear in the new year to find ways to measure what is happening to a child’s brain in the throws of juvenile Batten disease. Children across the world are suffering from this devastating illness. The foundation has identified a potential treatment and is preparing to conduct a clinical trial this year.

Before one can conduct a clinical trial to test potential new treatments for a brain disease, one must identify harmless ways of checking up on brain cells and measuring their response to treatment. To ensure progress towards a trial is not slowed due to a lack of ways to measure brain cell responses in juvenile Batten disease, Beyond Batten Disease Foundation (BBDF) has awarded De Montfort and Oxford Universities more than $150,000 to address this problem. Investigators with a rich history of success using advanced analytic tools to measure treatment responses to cancer and other lysosomal storage diseases will apply proton (1H) NMR-linked metabolomic strategies to identify sequential changes in cell activity during disease. Once documented, these changes can be monitored during the course of a clinical trial to confirm that a drug is having its predicted effect or provide early warning signs that something is wrong. Oftentimes, measurements such as these are taken long before a patient would feel different or appear to respond to treatment.

“It is hard to discover what is happening inside individual brain cells. Unlike other parts of the body, we can’t perform biopsies or rely on blood tests to provide that information. It is very elusive.   We have awarded money to a team of researchers with 20+ years of experience and a history of success identifying trial-ready biomarkers so that we can conduct early determination of how effective treatment is while carefully monitoring safety,” said Danielle Kerkovich, PhD, Principal Scientist of the Beyond Batten Disease Foundation.

“We have high hopes because of the stellar records and years of experience of the investigators involved,” said Mary Beth Kiser, President and CEO of Beyond Batten Disease Foundation.

The award, titled “Identification of New Biomarkers for Metabolomics Classification of Juvenile Disease: A Drug-Targeting Strategy”, is part of BBDF’s portfolio to carve a path from here to a cure. The successful completion of this project has the potential to substantially accelerate efforts to treat juvenile Batten disease.

ABOUT BEYOND BATTEN DISEASE FOUNDATION
Beyond Batten Disease Foundation (BBDF) is the world’s largest nonprofit organization dedicated to funding research for a treatment and cure for juvenile (CLN3) Batten disease. Since its inception in 2008, over $16 million has been invested in research by leveraging donations, co-funding and strategic partnerships. BBDF is spearheading a unique, cohesive strategy, incorporating independent scientific resources and collaboration with related organizations to drive research in juvenile Batten Disease. Today there is a treatment in sight. BBDF funded research has discovered a drug that slows the progression of the disease in Batten models. The foundation has launched a 24 month $6 million campaign to advance the treatment to clinical trials. More information can be found at www.beyondbatten.org.

Beyond Batten Disease Foundation, Noah’s Hope, Hope 4 Bridget and Batten Disease Research and Support Association Partner to Support the World’s Largest and most Comprehensive Batten Patient Registry

For Immediate Release

Contact: Angela Hale

Red Media Group

Beyond Batten Disease Foundation, Noah’s Hope, Hope 4 Bridget and Batten Disease Research and Support Association Partner to Support the World’s Largest and most Comprehensive Batten Patient Registry

Austin, TX (September 12, 2016) — Beyond Batten Disease (BBDF), Noah’s Hope, Hope for Bridget and the Batten Disease Support and Research Association (BDSRA) are joining forces to support the expansion of DEM-CHILD, a novel network of prominent NCL clinician scientists and researchers working together to collect the world’s largest and most well-characterized set of patients with juvenile (CLN3), late infantile (CLN2) and 12 other forms of Batten disease. Funding will support the continued collection of patient data in Germany, Norway, Denmark, France, Argentina, Brazil, Italy, Great Britain, Finland, India and the United States with a special focus on standardization of measurement and widespread use of the Unified Batten Disease Rating Scale (UBDRS). “We are co-funding this effort with committed partners because we believe that good and thorough patient registries that identify all of the major and minor characteristics of a disease, along with family contact information, will be critical to the planning, execution and ultimately the success of any clinical trials for Batten disease,” said Mary Beth Kiser, CEO of the Beyond Batten Disease Foundation. The UBDRS was developed at the University of Rochester Batten Center to quantify the physical, behavioral, seizure and functional aspects of juvenile (CLN3) Batten disease. Jonathan W. Mink, MD PhD, FAAN, FANA, FAAP, Frederick A. Horner, MD Endowed Professor in Pediatric Neurology and president-elect of the Child Neurology Society at the University of Rochester Medical Center, will work with Angela Schulz, MD, principal investigator and coordinator of DEM CHILD at the University Medical Center Hamburg-Eppendorf. Together and with their colleagues, Drs Mink and Schulz will add UBDRS data to DEM CHILD, further align their databases, and collaborate using the larger combined data to establish more rigorous natural history baselines for all forms of Batten disease. In the last few years, Dr Schulz has expanded DEM-CHILD from 7 countries to 18. Combined international patient registry development for control data, natural history documentation and biomarker identification is part of BBDF’s larger strategy to prepare for success in clinical trials. ABOUT BEYOND BATTEN DISEASE FOUNDATION Beyond Batten Disease Foundation (BBDF) is the world’s largest nonprofit organization dedicated to funding research for a treatment and cure for juvenile (CLN3) Batten Disease. Since its inception in 2008, over $16 million has been invested in research by leveraging donations, co-funding and strategic partnerships. BBDF is spearheading a unique, cohesive strategy, incorporating independent scientific resources and collaboration with related organizations to drive research in juvenile Batten Disease. Today there is a treatment in sight. BBDF funded research has discovered a drug that slows the progression of the disease in Batten models. The foundation has launched a 24 month $6 million campaign to advance the treatment to clinical trials. More information can be found at www.beyondbatten.org. ABOUT NOAH’S HOPE-HOPE 4 BRIDGET Noah’s Hope-Hope 4 Bridget, a non-for-profit 501(c)3, was established by the parents of Noah VanHoutan, Laine VanHoutan and Bridget Kennicott, who were diagnosed Late Infantile Batten disease (CLN2) in 2009. After founding individual foundations in 2009, the families partnered in 2014 to strengthen their efforts in research and find a treatment and cure for children impacted by the fatal disease. Noah’s Hope-Hope 4 Bridget also works on awareness of Batten disease. Late Infantile Batten disease affects fewer than 450 children in the United States. Children with Late Infantile Batten disease develop normally through their toddler years, giggling, talking, and running circles around their parents. By age three, they begin to have seizures and gradually lose the ability to walk, talk, and feed themselves. Late Infantile Batten disease is ruthless. Noah passed away in March 2016, just a few weeks shy of his 12th birthday. At this time, Late Infantile Batten disease is fatal between the ages of eight and 12, sometimes longer. For more information, about Noah’s Hope visit:www.NoahsHope.com. For more information about Hope 4 Bridget visit: www.Hope4Brdiget.com ABOUT BATTEN DISEASE SUPPORT AND RESEARCH ASSOCIATION The Batten Disease Support and Research Association (BDSRA) serves those with all 14 identified forms of Batten disease through patient education, family and research conferences, consultation and advocacy. For nearly 30 years, BDSRA’s committed families have funded researchers around the world who have developed the research knowledge leading to today’s clinical trials. For more information: www.bdsra.org.

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