Story
Hello, and thank you for being one of Connor's Superheroes!
Because of YOU, we have reached our goal of raising $185,000 for the initial drug repurposing screen!!! However, we're not stopping there. There are several phases of this drug screen and we are working with the A-T Children's Project to continue raising funds for the next phases, as well as other important initiatives. It will be a lifelong journey to find therapies and a cure for Connor and all children with A-T. We have so much work to do and need your help! If you haven’t already donated, please don’t hesitate to do so today. 100% of all money raised will go towards finding therapies and a cure for Connor and all those impacted by A-T.
August 14, 2022: Please see below for an update on the Charles River drug repurposing screen!
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To look at almost 4 ½-year-old Connor is to see a blue-eyed, always happy little boy, a bit wobbly on his feet, who loves the outdoors, superheroes, Legos, monster trucks and adventures.
But Connor is no ordinary toddler.
He was born with ataxia-telangiectasia, or “A-T”, an ultra-rare, progressive, degenerative disease that unleashes a cascade of severe symptoms such as deteriorating muscle control, slurred speech, respiratory infections, feeding issues and a predisposition to numerous types of cancer. The prognosis is grim. Children born with A-T are usually dependent on wheelchairs by the age of 10, having lost the ability to control their muscles, and they rarely survive their 20s. There is no cure and no effective treatments that can manage the neurological aspects of the disease. There are about 400 known cases of A-T in the United States.
While we continue to hold out hope for a brand-new therapy or cure that can reverse Connor's genetic condition- perhaps a gene therapy- we have joined an international effort to locate an existing drug or investigational compound that might slow the degeneration going on in Connor’s brain cells and others like him.
The non-profit A-T Children’s Project is spearheading the search for a cure—ordinarily a needle in a haystack event were it not for the development of technologies that have helped accelerate the process. We are working with the A-T Children’s Project to raise $185,000 needed to fund a repurposing screen with the goal of finding a drug that can be tested in children with A-T to slow the progression of the disease or, ideally, to find a cure. The repurposing screen will begin with the development of customized assays to detect the functional rescue of cells from children with A-T, followed by the screening of large libraries of compounds, both approved and investigational, using those assays. Charles River Laboratories is the scientific institution involved in this critical study.
We (and the A-T Children's Project) are passionate about pursuing this repurposing screen as we know we are in a race against time to find a cure for Connor and all other children affected by this debilitating and deadly disease. We are committed to investigating new ways to extend Connor's life, mitigate any unnecessary hardships he may face, and ideally find a cure, and if not for Connor, then for future generations of children born with A-T or other rare diseases.
Beyond hoping that these efforts will find a treatment from among existing drugs much sooner than if they had to discover them from scratch, the A-T Children’s Project also sees this project as a major milestone for A-T research. They're thrilled that, rather than relying on an academic lab to try to conduct this important effort, a world class organization like Charles River Laboratories is bringing its industrialized, high-throughput approach to this ultra-rare disease. The A-T Children's Project is gaining access to the same technologies and rigor that global pharmaceutical companies receive which is extraordinary, especially in the rare disease world.
We are on a mission to raise $185,000* for this repurposing screen. If you would like to be a part of this groundbreaking effort to find a cure for Connor and all other children with A-T, please consider a tax deductible donation to the A-T Children's Project via the link on this page or the QR code below.
Additionally, if you feel compelled, please share this page with anyone and everyone on all social media platforms and via email, text, etc.! We cannot thank you enough for your support, thoughts, and prayers!
With love,
Dan and Michelle Graban
FYI- please check out this article from Charles River which discusses the repurposing screen we're trying to fund for all children with A-T: https://www.criver.com/eureka/can-existing-drugs-find-new-purpose-ultra-rare-disease. You can also read the A-T Children's Project's update on the screen here: https://www.atcp.org/drug-repurposing-screen-for-a-t-reaches-important-milestone/!
*Please note that when the funds for this project have been raised in full, your donation will be used for future phases of this project or other important A-T Children's Project initiatives.