This Year’s 10 Biggest Advances in ALS Research

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In 2016, a significant number of ALS research discoveries, advances in clinical trials, collaborations and strategic initiatives all accelerated the pace of discovery in finding treatments and a cure for ALS.

We’ve pulled together what we think are 10 of 2016’s biggest advances in ALS research that gave us, and people living with ALS, hope this year!


The discovery of the NEK-1 gene, now known to be among the most common genes that contribute to the development of ALS, made headlines around the globe. More than 80 researchers in 11 countries conducted this largest-ever study of inherited ALS – read more about how Ice Bucket Challenge donations helped The ALS Association invest in this important work conducted by Project MinE.


The Neurological Clinical Research Institute (NCRI) imaging team at MGH, led by Dr. Nazem Atassi, used PET imaging to successfully scan the first person living with ALS to measure inflammation in the brain, a promising first step in this imaging biomarker study. Since then, many more people have participated in the study.


The U.S. Food and Drug Administration (FDA) accepted, and is currently reviewing, a New Drug Application (NDA) for Edaravone for the treatment of ALS. We’re eagerly anticipating news from the FDA in 2017!


Dr. Aaron Gitler and his researcher colleagues, supported by The ALS Association, identified a new therapeutic target for C9orf72-associated ALS. We interviewed Dr. Gitler on our blog about the therapeutic potential to treat ALS.


Investigators at Cedars-Sinai gained approval from the FDA to test the safety of a combination stem cell-gene therapy in a clinical trial. The ALS Association previously certified Cedars-Sinai as a Treatment Center of Excellence, meeting the Association’s rigorous standards with their comprehensive, collaborative approach to patient care and services.


Brainstorm announces positive results for NurOwn stem cell phase II trial reported in the U.S. and then announces larger phase III trial to begin in 2017.


IBM’s Watson supercomputer discovers 5 new ALS genes. IBM’s collaboration with the Barrow Neurological Institute in Phoenix shows the power of Big Data and the potential for advanced computing to speed up progress toward treatment and a cure.


One of the nation’s largest precision medicine programs began enrolling in October, with 9 centers at universities and hospitals across the United States. The ALS Association committed $3.5 million in Ice Bucket Challenge-raised money to this exciting collaborative effort.


At the International ALS/MND Symposium in Dublin, a leading University of Miami ALS researcher and the pharmaceutical company Orphazyme announced the successful completion of phase II trial of arimoclomol in SOD1 ALS.


Announcements of global collaborations, ALS ONE and NeuroLINCS, supported by millions in funding from The ALS Association. These two initiatives will help generate the data researchers need to continue their important work.

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Submitted by: Nicolas E. on Sat, 02/19/2022

Thank you

Submitted by: Nicolas E. on Sat, 02/19/2022

Thank you

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