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Apr 14

Answer ALS annual meeting emphasizes openness and collaboration as key steps to move the science forward

Nearly one hundred researchers gather in Baltimore to share progress and discuss the future of the project.

Five years after researchers first started meeting to discuss the project that would become known as Answer ALS, they gathered again in Baltimore’s Inner Harbor to share progress over the past year and to discuss the future of the project. Answer ALS is governed by the same ethos as the Packard Center, which emphasizes openness and collaboration as key steps to move the science forward. Researchers are encouraged to share their findings, even if the results aren’t final or they reveal challenges, so that they may receive feedback from their colleagues – such real time peer-review is a critical part of accelerating ALS research.

Since its inception, Answer ALS has enrolled over 1000 participants at 8 centers throughout the U.S. All of the biosamples—blood, cerebrospinal fluid, cells, and more—are all stored and linked. Researchers hope that this detailed clinical and biological data, estimated to average 1 terabyte per patient, will improve our knowledge of the underlying biology of ALS so that researchers can stratify patients into different subgroups based on their genetic and/or biochemical characteristics. This, says Jeffrey Rothstein, Executive Director of Answer ALS, will enable scientists to find the right drug for the right person. Although patient enrollment is complete and much of the clinical phase of the trial has wrapped up, the painstaking work on analyzing the data is still in the early phases.

One of the hallmarks of Answer ALS is the pairing of clinical data, such as slow vital capacity, handheld dynamometry, and speech, with biological data. Nearly every participant has had their genome sequenced, and everyone also donated blood cells for the creation of induced pluripotent stem cells (iPSCs) that will allow scientists to model ALS using a patient’s own cells. To date, scientist Clive Svendsen at Cedars-Sinai Medical Center has initiated hundreds of iPSC lines and completed 619 of them. At the University of California, Irvine, biologist Leslie Thompson is coordinating ’omics analyses of patient cells. ’Omics, which refers to comprehensive, or global, assessment of a set of molecules, such as genes, proteins, RNA, lipids, and metabolism, will enable Thompson and other researchers to perform large network analyses and identify cellular signatures that can help differentiate patients from controls, and groups of patients from each other. Her preliminary analyses can identify modules that correlate with ALSFRS score, the C9orf72 repeat expansion, and bulbar vs limb onset.

Ernest Fraenkel at MIT is coordinating the data analysis for Answer ALS, and thus far his group has stored 88 terabytes on Microsoft Azure, a cloud computing service, which translates to ore than 3 years of continuous high-def streaming video. His goal is to enable the broadest possible community of researchers to identify ALS subtypes and find therapeutic strategies for each. He is working to create an Answer ALS Data Portal to make it easy for scientists to request data or cells. To ensure that this data is widely used, Rothstein is encouraging partnerships with government and non-profit organization, as well as ensuring that everything is open source and freely available.

Other Answer ALS scientists shared their progress on efforts to analyze gene activity and cluster patients into different subgroups, as well as to analyze the genomes of people with sporadic ALS for mutations that might place some small role in disease. Many of these and other studies are using network analysis to identify metabolic pathways that are changed in patients with ALS. For researchers involved in this project, much of the hard work has just begun, but scientists hope that some of these preliminary results will begin to pay dividends soon.

  • The opening session was broadcast via Facebook Live and was viewed by over 2,200 people.

  • Answer ALS program director Emily Baxi moderated the opening session.

  • Ed Rapp, Chairman of the Answer ALS Advisory Board, welcomed the meeting participants.

  • Jeff Rothstein (right), Merit Cudkowicz (center) and Clive Svendsen (left) welcomed questions from those viewing via Facebook and from the meeting participants.

  • James Berry from Massachusetts General and an Answer ALS investigator shared information on the study wrap-up and preliminary results.

  • Jennifer Roggenbuck from Ohio State University explained the RoAR Study progress (Return of ALS Results).