Noah's Hope Initiatives
With the generous support of people in our community and around the world, Noah’s Hope is making progress in the fight against LINCL-Batten disease. The following are just a few of the initiatives that Noah’s Hope has undertaken. We are eternally grateful for all who have assisted in our efforts to create hope for the future.
- For the past two years, Noah’s Hope has funded projects for Enzyme Replacement Therapy at the Center for Advanced Biotechnology and Medicine at Rutgers University. We hope that this methodology, proven already in other lysosomal storage disorders, can move to clinical trial for LINCL-Batten disease patients by 2014.
- In partnership with Jasper Against Batten, Noah’s Hope funded the creation of a LINCL-Batten disease mouse colony at King’s College London. The work to be done with these mice will help other scientists around the word to better understand how LINCL-Batten disease progresses through the brain and to develop therapeutics that are better targeted.
- Noah’s Hope recruited a new researcher at Rush University in Chicago to look at a new approach to LINCL-Batten disease therapy. This researcher is a Parkinson’s, Alzheimer’s, and Multiple Sclerosis expert who believes that he can apply some of his previous work to LINCL-Batten disease. He will use a drug that has already been approved by the FDA to possibly relieve some of the symptoms of LINCL-Batten disease.
- Noah’s Hope recruited a new researcher at Rice University in Houston to look at how the protein that is responsible for LINCL-Batten disease becomes inoperable. She is testing a new compound in cell cultures to determine if she can make this protein work correctly again.
- Noah’s Hope funded the upgrade of all computer systems at the national Batten Disease Support and Research Association (BDSRA).
- As a result of his efforts on behalf of Noah’s Hope, Tracy VanHoutan, Noah and Laine’s father, was elected by members of the BDSRA to the national BDSRA Board of Directors.
- On behalf of Noah’s Hope, Tracy VanHoutan testified before the U.S. Food and Drug Administration (FDA) in Washington, D.C., in June of 2011. Tracy testified on the challenges that rare diseases face at the regulatory level, and he submitted proposed changes to the FDA.
- On behalf of Noah’s Hope, Tracy VanHoutan participated in numerous conference calls with the National Organization for Rare Disorders (NORD) and the Rare Disease Legislative Advocates (RDLA).
- Noah’s Hope assisted in the recruitment of seven other LINCL-Batten disease foundations to form a small working group dedicated to finding a treatment or cure for LINCL-Batten disease.
- Along with other LINCL-Batten disease family foundations, Noah's Hope coordinated with the BDSRA and the National Institute for Neurological Disorders and Stroke (NINDS) to convene an international LINCL-Batten disease conference at the National Institutes of Health (NIH) in November 2010. The purpose of this conference was to identify gaps in knowledge about LINCL-Batten disease and to develop collaborative efforts among the attending scientists to develop a concrete plan to transition work from the lab into the clinic. We also hope that the NIH will issue a funding request as a result of this conference that could lead to significant funding for LINCL-Batten disease research at the federal level.
- Noah’s Hope is working to develop a project to identify a biomarker or surrogate endpoint for LINCL-Batten disease that will aid in clinical trial design. This project will measure specific proteins in the blood or spinal fluid that will allow us to determine if new therapies are effective.
- On behalf of Noah’s Hope, Tracy VanHoutan made three trips to Washington, D.C., to recruit members of the U.S. House and Senate to join the newly formed “Rare Disease Congressional Caucus.”
- In partnership with Hope-4-Bridget, Noah’s Hope co-funded a project at Sanford Children’s Research Center in South Dakota to develop a test for a high-throughput screening of compounds and pharmaceuticals to determine if any of the therapies might have an impact on LINCL-Batten disease cells.
- Because of his many efforts on behalf of Noah’s Hope, Tracy VanHoutan was invited to the NIH campus in Maryland In September of 2011 to participate as a parent advocate for a workshop on cell-based therapies for pediatric disorders. This workshop addressed strategies to overcome the barriers to advancing the development and delivery of cell-based therapies for pediatric patients, in particular those with rare and life-threatening diseases. The clinical applications of cellular therapies and regenerative medicine, including the ethical considerations and models of clinical trial design, were examined with intent to optimize overall processes for the future.