Since the first ALS gene was discovered 25 years ago, much has been learned about ALS. The last several years have seen exciting progress in ALS research, with new genetic causes explored and the initial utilization of induced pluripotent stem cells (iPS cells) derived from ALS patients in treatments.
Yet in spite of these discoveries and tools, the lack of new therapies or even good biomarkers yielded from these approaches has proven that single labs working in silos and simple animal models are not going to illuminate the disease’s root cause, identify subtypes of the disease or define the best drug targets. Only a large-scale concerted and coordinated collaborative team effort will be able to make a difference, and make one quickly.
We aren’t asking for answers to single questions, like “What is the next ALS gene to target?” or “What new animal models should we build?” Rather, we are comprehensively evaluating a very large patient population of 1000 participants, generating iPS cells from each and every patient, creating thousands of new and different iPS brain cell lines representing each of those patients, and performing the most comprehensive biological analytics ever—including DNA, RNA, protein and metabolism analysis—to yield a personalized database of thousands of petabytes of new ALS-specific information.
This will be the most comprehensive foundation of ALS data ever amassed, and reflects real patients. In collaboration with experts in machine learning and big data informatics, this wealth of biological data will be mined to uncover ALS causes, subtypes, pathways gone awry and drug targets. It will serve as the foundation for new clinical trials, suggest new ways to subgroup patients to better discover successful drugs and to find drug-responsive biomarkers or diagnostics. Most importantly, this data is and will be publicly available for anyone to access, interpret and mine for valuable insight.
Answer ALS Enrollment
We are encouraged by the continued positive response and strong enrollment rates for Answer ALS. Our participants really keep us going!
Not only are we on a strong track for initial enrollments, our longitudinal efforts are strengthened by the numerous repeat visits.
Cell Line Progress
Genetic Sequencing: What is Genetic Sequencing?
The New York Genome Center is doing the Genetic Sequencing for Answer ALS.
Cell Lines Available to Researchers Around the World
Our Cell lines are already being submitted to the Northeast Amyotrophic Lateral Sclerosis Consortium (NEALS) for distribution to researchers. For more information about NEALS and how to access samples for research: https://www.neals.org/for-als-researchers/neals-sample-repository/
Cedars-Sinai is leading the generation of these unique iPSCs and their derived motor neurons from blood of patients collected across multiple clinical sites in the US along with deep clinical data. These cells will be used in big data projects and large-scale omics investigations. To get information on ordering IPS Cell Lines from Cedars Sinai: