Where do your DONATIONS go?
Philanthropy matters. Anyone can give anytime in any amount. The Foundation operates without permanent staff members and Directors serve without compensation for administrative and grant evaluation processes. We pride ourselves at maximizing gifts to benefit research, individual children, education and advocacy.
Thanks to our sponsors, we raised over $86,000 in 2015. In eight years we have raised over $760,000. We are so proud of this accomplishment.
March 2015 – $100,000 was awarded to support a Natural History and Biorepository Database for Infantile and Late Infantile Batten Disease. Dr. Jonathan Mink from the University of Rochester and Dr. Katherine Sims of Harvard University and Massachusetts General Hospital are the recipients of this gift. The registry and biorepository will provide valuable information on the natural history of CLN1 and CLN2 and the possibility to correlate changes in cells, their gene and proteins with clinical outcomes. This type of information will make it possible to compare the outcomes of future treatment strategies with what we would expect from the natural history of the disease. OPTN has promised an additional $100,000 each year for 2016 and 2017 to fulfill the entirety of the project of $300,000 with the hope that new revenue streams will support this project past 2017.
March 2015 – $25,000 was awarded to Dr. Michelle Hastings from the Rosalind Franklin University School of Medicine and Science, Chicago and Dr. Jill Weimer from Sanford Research Center, South Dakota. Their study is called Stop Codon Read-Through and Nonsense Suppression for the Treatment of Infantile and Late Infantile Batten Disease. The gene defects in infantile and late infantile Batten Disease result in unstable proteins. A new strategy will be tried to produce more stable protein products from the mutated genes by manipulating the genes with small drugs that cause the ribosomes (where genes are expressed into proteins) to make a more stable protein. These drugs work by bypassing the mutation, and allowing the ribosomes to read through (or around) the defect. These dollars are to support an exciting ‘proof of concept,’ focusing on stabilizing the mutated proteins that cause Batten Disease. If the proof of concept is achieved, OPTN would support future dollars to allow these scientists to further their research.
December 2015 – $2,000 was awarded to The Nicholas Giving Tree Project. Sponsorship was given to 22 families who have children with various types of Batten Disease all over the United States. With the support of 40 donors OPTN sponsored personal care items, over-the-counter medications, clothes and toys.
June 2015- $6,300 was awarded to support expert nursing care at the Batten Disease Support Research Association’s (BDSRA) Annual Conference held in Chicago. Over 40 children with Batten Disease in need of expert medical attention were given care and support during this 3 day conference. This allowed their families to attend the support meetings and conferences as well as get some respite knowing their children were in good hands. OPTN was proud to offer our skills and expertise to these loving children. Four volunteers of OPTN, who financed their own travel, attended the conference to support the nurses and provide individual one-on-one companion care.
March 2016- $12,000 was awarded to sponsor the 3rd International Scientific Workshop through the sister organization of OPTN, the Batten Research Alliance (BRA) in Bethesda, Maryland. This two day conference fosters a discussion by leaders in the field of Batten Disease. More than two dozen scientists will collaborate and share their findings in the latest hope for evolving fields providing direction in research, create new ideas and partnerships in a forum that otherwise would be impossible to reproduce. The outcome of the conference includes request for proposals (RFPs). Proposals are then independently reviewed and scored so that an educated determination can be made to financially support the best and most promising research proposal.
OPTN is looking forward to supporting exciting research into 2016.
OPTN has committed to continue to support the Natural History and Biorepository data base over the next two years offering $100,000 for each year in 2016 and 2017. Dr. Jonathan Mink from the University of Rochester (Biorepository) and Dr. Katherine Sims of Harvard University and Massachusetts General Hospital (Natural History Data Base) are the recipients of this gift. Giving scientists access to demographics, dates regarding treatment and tissue samples is an important step in the search for a cure. The ongoing registry and biorepository will provide valuable information on the natural history and the possibility to correlate changes in cells, their genes and proteins with clinical outcomes. This type of information will make it possible to compare the outcomes of future treatment strategies with what we would expect from the natural history of the disease. It is the first of its kind for Batten Disease in the United States.
OPTN is hosting the 3rd bi-annual Batten Disease Workshop in Bethesda, Maryland on March 30th, 2016. This three-day workshop is the combined effort of 10 family foundations coming together to review findings from past research projects and latest findings. Scientists will be encouraged to work in teams to come up with innovative research proposals. Proposals will then be accepted through the BRA to determine possible financial awards, should their application meet the high expectations for promising outcomes. OPTN will receive the findings from the independent reviewers and will look to offer financial support.
OPTN will continue to attend the BDSRA’s Annual Conference in 2016 held this year in St. Louis, Missouri. We will continue to offer our support in the nursing care and respite of the individuals who attend the conference.