News from the A-T Children's Project
We have just awarded funding to Margot Mayer-Proschel, PhD at the University of Rochester to characterize a new mouse model that her lab has developed for A-T.
With a research grant from the A-TCP, Ronald Hart, PhD at Rutgers University in New Jersey will create induced pluripotent stem cells (iPSCs) using blood samples taken from kids with A-T.
Brad Margus has been tapped to co-chair the oversight board for the Network for Excellence in Neuroscience Clinical Trials, or NeuroNEXT, a new, innovative program at the National Institutes of Health.
On February 28, friends and families of A-T kids across the U.S. and Canada raised funds for A-T research by celebrating Rare Disease Day.
The A-T CureTeam weekend from January 11-13 proved to be a huge success – for both crossing finish lines and raising funds.
The A-T Children's held a workshop that focused on finding treatments for A-T's debilitating neurological symptoms. All researchers and families were invited to attend.
With funding from the A-T Children’s Project and the Australian-based BrAshA-T organization, a team of researchers in Australia is using neuroimaging technology to learn more about abnormal circuitry and inflammation in the brains of A-T patients, possibly providing clues for therapeutic targets and uncovering imaging biomarkers for use in clinical trials.
The A-T Children’s Project welcomes Dr. Phillip LoGrasso to its Scientific Advisory Board. Dr. LoGrasso is a Professor in the Molecular Therapeutics Department and Senior Director of Drug Discovery in the Translational Research Institute within The Scripps Research Institute where he has been since 2005.
With help from scientists who specialize in brain imaging, the A-T Children’s Project is seeking ways to treat the abnormal brain circuitry in ataxia-telangiectasia (A-T). For the first time, a comprehensive PET and fMRI study of the brains of adults with A-T has begun.
Three papers have been published recently in the scientific literature describing: 1) gastrostomy tube (G—tube) safety and satisfaction in patients with A-T; 2) the care of critically ill A-T patients and 3) the care of patients with A-T undergoing operations and the possible risks associated with the use of anesthesia for surgical procedures.
This type of “disease-in-a-dish” model can be used to screen hundreds of thousands of compounds for their ability to diminish or reverse certain characteristics of the disease in culture. Promising compounds or drugs can then be developed for future clinical trials.
A post doc at Louisiana State University will be studying neurological dysfunction in A-T for the Fellowship Award that he received from the A-T Children's Project.
The A-T Children's is funding two new research grants that may provide insights regarding brain function in A-T. The Florida-based Wobbly Feet Foundation has agreed to co-fund part of each grant in an on-going partnership to accelerate A-T research.
The A-T Children's Project, together with the UK-based A-T Society, will fund a clinical study led by Emma Ross, PhD from the University of Brighton in England. This study will examine the effects of inspiratory muscle training (IMT) on lung function and quality of life in individuals with A-T.
Lung problems cause sickness and death in many patients with ataxia-telangiectasia (A-T). However, a lack of information from following A-T patients’ lung disease over time has limited doctors’ knowledge of these critical problems. With the goal of providing expert advice on the diagnosis, evaluation, and management of lung disease in A-T, Dr. Sharon McGrath-Morrow and her colleagues at the A-T Clinical Center recently published a state-of-the-art paper in the journal Pediatric Pulmonology.
Since the late 1990s, Paul K.Y. Wong, PhD, from the University of Texas MD Anderson Cancer Center has been exploring what goes wrong in blood and brain cells when the A-T protein is missing. Although A-T is not the primary research focus for Dr. Wong, a leading investigator in the area of mouse retroviruses as a model for HIV associated dementia, his laboratory has produced more than 10 A-T related papers in the past decade.
Over the last few years, the Children’s Neurobiological Solutions (CNS) Foundation and the A-T Children’s Project (A-TCP) began to realize that a critical gap existed in the path to bringing potential therapies to the clinic for children with neurological disorders. An organized network of multidisciplinary, well-funded clinicians experienced in performing clinical trials for rare, pediatric neurological disorders, like ataxia-telangiectasia
(A-T), did not exist.