The Aicardi-Goutières Syndrome Americas Association, LunaPBC, and Genetic Alliance announced a program to assemble a patient-led drug discovery community studying the disease etiology of a rare neurodevelopmental disorder known as Aicardi-Goutières syndrome and support development of therapeutic treatments for this devastating disease.
Program incorporates families to inform research and drug development
SAN DIEGO, Feb. 3, 2022 /PRNewswire/ -- The Aicardi-Goutières Syndrome Americas Association (AGSAA), LunaPBC, and Genetic Alliance today announced a program to assemble a patient-led drug discovery community studying the disease etiology of a rare neurodevelopmental disorder known as Aicardi-Goutières syndrome (AGS) and support development of therapeutic treatments for this devastating disease. The program is being launched in collaboration with Biogen, Inc. and represents Biogen’s second collaboration with Luna and Genetic Alliance.
The program will engage patients and their families, patient advocates, and pharmaceutical partners to create a patient-driven trial design—with patients and their parents as the stewards of their data with the goal of incorporating the optimal clinical and behavioral features and the most desired trial endpoints in the drug discovery process. Luna’s proprietary Community-Driven Innovation technology will align families and researchers on their key priorities and needs.
“The passion of these parents drives research on this condition like never before,” said Sharon Terry, president and CEO of Genetic Alliance. “Genetic Alliance is so excited to support these families. Our work with Luna is the kind of paradigm shift we saw in lots of other industries. People truly at the center, calling the shots.”
AGS primarily affects the developing brain and immune system of infants and toddlers, most often resulting in profound developmental delays, lifelong physical impairments, and persistent neurological changes. Most newborns with AGS do not display any signs or symptoms at birth but go on to develop severe brain dysfunction within the first two years of life, often after months of typical development and good health. In AGS, the body’s immune system turns on itself in a destructive way, targeting myelin, or white matter, in the brain and significantly impacting the nervous system.
Additionally, immune dysfunction associated with AGS can affect many other organs of the body, sometimes in a life-threatening manner. This can include the lungs, the liver, the heart, the skin, blood cells, and the kidneys. Because the signs and symptoms of the disorder are similar to those of a congenital viral infection, AGS is extremely difficult to diagnose.
Although rare, increasing awareness of AGS has revealed higher prevalence than previously ascertained. In order to manage severe progression and improve quality of life for affected individuals and their families, both early identification and timely access to emerging treatments are essential interventions.
The program gives the AGS community the power to shape clinical studies that have the potential to answer their needs more quickly and efficiently. This framework, constructed from deep dives into AGS’s digital world and real-world interviews with parents and patients, helps guide researchers toward the most accurate observations and endpoints that will be built into the study design to increase participant engagement and retention, to ensure interventions meet the goals and needs of patients, to capture real-world and patient-reported insights, and to facilitate comprehensive, longitudinal study.
“This partnership represents a key element of our ‘human-first’ drug discovery approach at Biogen,” said Sally John, Head of Translational Biology at Biogen. “We have learned how important it is to engage patients early in the process. The program will provide a deeper understanding of the natural history of the disease and insight into the families’ lived experiences to help enable discovery of novel medicines and clinical research that best meet the needs of patients.”
The framework also enables children and their parents to participate in studies from the comfort and safety of their homes, thereby reducing the hardship on families, increasing participation, and expediting research.
“This partnership represents an incredible opportunity to not only document and validate the lived experiences of families grappling with Aicardi-Goutières syndrome, but actually use these hard-won insights to shape the future of research and treatment options” said Devon Cordova, vice president of the AGSAA. “With committed community engagement and thoughtful stewardship of our data, we will finally have what we need to tell our story and begin to write a different ending.”
To learn more and determine eligibility to participate, visit https://www.agsamericas.org/ags-patient-registry to join and help accelerate discovery.
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SOURCE LunaDNA
