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Oct 17

Answer ALS Scientists Announce Major Milestone

Packard Center News
The public release of a huge range of biological specimens is now available and openly accessible to researchers

At this year’s annual meeting of the Northeast ALS Consortium (NEALS), scientists from the Answer ALS initiative announced the public release of a huge range of biological specimens openly accessible to researchers. Packard Center and Answer ALS Director Jeffrey Rothstein, also a neurologist at Johns Hopkins University, says that this is an “unprecedented” availability for such samples.

 “Never before have so many biological samples, ranging from blood specimens to stem cells created from our patients, been provided to global researchers. This has been one of the major goals of Answer ALS, and we anticipate that this is only the first of many other specimens of use to scientists,” Rothstein says.

Answer ALS, the largest ALS research initiative to date, involves the combined resources of seven universities across the United States, all engaged in the recruitment of 1000 ALS patients and 200 controls. Once enrolled, these individuals will provide a variety of biological specimens, including blood, cerebrospinal fluid, and fibroblast cells. Researchers will use the latter to create lines of induced pluripotent stem cells derived from each ALS patient to study ALS in all its different forms. They will also use a wide range of cutting-edge techniques, including genomics, epigenomics, metabolomics, proteomics, and transcriptomics, to better understand why ALS starts and how it progresses. The study began recruitment in December 2015 and is currently ahead of schedule.

At the October NEALS meeting, Rothstein and his fellow Answer ALS co-directors Clive Svendsen, Director, Board of Governors Regenerative Medicine Institute, Cedars-Sinai Hospital, and Merit Cudkowicz, Chief of Neurology and Director of ALS Program, Massachusetts General Hospital, announced several major milestones. To date, they have released:

  • 34 well defined ALS iPS cell lines released through the Answer ALS iPSC repository website
  • Over 27,000 serum, blood and CSF samples released to the global ALS research community
  • 564 patients currently enrolled in the Answer ALS study
    • 82 participants completed all 5 visits
    • 146 have completed 4 visits
    • 361 are beyond the 1st visit
  • The Answer ALS App to monitor patient participant motor, speech, breathing and cognition progression is now available.
  • The Les Turner ALS Research and Patient Center at Northwestern Medicine announced it has joined Answer ALS as the 7th clinical site in the nation.

By obtaining samples and creating stem cell derived motor neurons from such a large range of patients, followed by extensive biological analytics of this surrogate “brain biopsy,” Answer ALS hopes to use these tools and data to one day tailor treatments for specific groups of patients, which may help improve success and slow disease progression, as well as advancing the understanding of the basic biology of ALS.

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