NEW YORK, Aug. 21, 2024 (GLOBE NEWSWIRE) -- Project Mosaic, a pioneering consortium of ALS nonprofits, is proud to announce the launch of a groundbreaking initiative aimed at revolutionizing the development of treatments for Amyotrophic Lateral Sclerosis (ALS). This collaborative effort, in partnership with leading industry experts, seeks to advance next-generation, cell-based models of ALS that better reflect the disease's complex and heterogeneous biology.
If successful, the initiative will be a critical first step to creating the ALS-equivalent of cancer biopsies at industrial scale.
For decades, the fight against ALS has been marked by high failure rates in drug development, largely due to the inadequacy of existing preclinical models. These models, often based on genetically triggered forms of ALS like C9ORF72 and SOD1, fail to represent the majority of ALS cases, particularly the 80% of cases with no clear genetic cause. This has resulted in a slow pace of innovation and a lack of effective therapies, leaving thousands of patients with limited treatment options.
Project Mosaic aims to change this paradigm by moving a new class of cell-based ALS disease models from academic labs to industry. These models will be created from induced pluripotent stem cells (iPSCs) derived from ALS patients, allowing researchers to study the disease in a more representative and meaningful way. The project will focus on standardizing and validating already-demonstrated methods that recapitulate TDP43 pathology, a hallmark of ALS found in 97% of cases, using motor neurons derived from patient tissues.
The goal is a standardized, open-source methodology available to researchers and commercially-available assays that drug makers can use to determine if a drug candidate is likely to work for subgroups of patients before human trials.
"We are excited to embark on this ambitious journey to make ALS a livable disease," said Bernie Zipprich, Program Director of Project Mosaic, who is also a person living with ALS. "By leveraging cutting-edge techniques and collaborating with top scientists and industry leaders, we aim to create more accurate models that can lead to the discovery of targeted therapies for different ALS subgroups. This initiative has the potential to transform the way we approach ALS drug development and significantly improve patient outcomes."
The initiative will unfold in two stages. The first stage involves replicating and validating two promising methods for modeling sporadic ALS, followed by a thorough evaluation of their accuracy against postmortem brain tissue samples. The second stage will explore the commercial potential of these models by using them to screen drug candidates and identify "responders" in clinical trials.
The initiative has the backing and support of the ALS Association, Project ALS, Answer ALS, Target ALS, Everything ALS and the ALS Hope Foundation, as well as a number of leading ALS and stem cell researchers.
For more information or to participate in this transformative initiative, please contact Bernie Zipprich at bernie.zipprich@projectmosaic.org.
About Project Mosaic
Project Mosaic represents a consortium of ALS nonprofits and industry stakeholders dedicated to accelerating the development of effective treatments for ALS by moving a promising new class of ALS disease models from academic labs to industry. By making it possible for drugmakers to develop and test targeted treatments for subgroups of patients, Project Mosaic aims to overcome a key barrier to ALS drug development and transform the fight against this brutal disease.
Media Contact:
Veronika Stattin
Chief of Staff, Project Mosaic
info@projectmosaic.org
