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 (genomics), RNA (transcriptomics) and protein (proteomics) 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 openly available through the Answer ALS Data Portal for anyone to access, interpret and mine for valuable insight.
Overview of Progress
Enrollment is now closed.
Cell Line Progress
Induced pluripotent stem cells (iPSCs) and motor neurons are produced at Cedars Sinai under the leadership of Dr. Clive Svendsen and Dr. Dhruv Sareen. Cell lines can be ordered by visiting Cedars-Sinai Biomanufacturing Center.
Charts track progress sequencing patient-derived samples across the whole genome, epigenome, transcriptome and proteome. Data are released via the Answer ALS Data Portal as soon as they pass routine quality control.
The New York Genome Center is doing the Genetic Sequencing for Answer ALS.
Epigenomics: What is epigenomics?
Epignomics is performed at Diagenode and Massachusetts Institute of Technology under the leadership of Dr. Ernest Fraenkel.
Transcriptomics: What is transcriptomics?
Transcriptomics is performed at University California, Irvine under the leadership of Dr. Leslie Thompson.
Proteomics: What is proteomics?
Proteomics is performed at Cedars Sinai under the leadership of Dr. Jenny Van Eyk.