The Answer ALS Research Program represents a groundbreaking shift in the study of ALS and neurological diseases, with a particular emphasis on the expansive OMIC data and biological resources it offers to the research community. Traditional approaches to studying the brain and spinal cord have been limited by the inability to biopsy these tissues without causing irreversible damage. However, advances in stem cell technology have transformed this landscape. Through the use of induced pluripotent stem cells (iPSCs) derived from a simple blood sample, scientists can now ""recreate"" a patient's brain and spinal cord cells. These iPSCs can be directed to become spinal motor neurons, the cells most affected by ALS, enabling detailed study of how these neurons differ from those in healthy individuals or other ALS patients. For each participant in the program, a comprehensive suite of data is collected, including longitudinal clinical data, whole-genome sequences, and multi-omics data (transcriptomics, epigenomics, and proteomics) derived from iPSC-generated motor neurons. These datasets are complemented by an extensive collection of biological resources, including patient-derived biofluids, and iPSC lines. All data and resources are standardized and made accessible to the global research community through the Answer ALS Data Portal, known as “Neuromine” (data.answerals.org), a custom-designed platform that integrates knowledge and resources. The Answer ALS repository, rich in multi-omic data, provides an unparalleled resource for researchers worldwide, fostering new insights into ALS and facilitating the discovery of novel therapeutic targets. By openly sharing this wealth of data, biosamples, and research findings, the Answer ALS Research Program has already had a profound impact on ALS research globally. While the primary focus is on ALS, the program is increasingly recognized as a model for collaborative research in other neurological diseases. This innovative program exemplifies the power of partnership between academic institutions, industry, and interdisciplinary science and technology, all united in the mission to answer ALS.
