Collaboration is at Our Core…and Spurring Innovation Along the Way

Innovation plays a key role in The ALS Association’s fight to develop treatments and a cure for ALS and to empower people living with the disease to live their lives to the fullest. During June and July, we are celebrating some of the key innovations helping us change the nature of ALS forever.

Since our founding in 1985, we have put collaboration at the forefront of everything we do. The gift of the ALS Ice Bucket Challenge in August 2014 gave us the unique opportunity to up our game in establishing innovative research partnerships around the world.

We continue to foster those partnerships throughout the ALS community today. Our partnerships span to all areas, from academic institutions and researchers, to industry (biotech and pharmaceutical) partners, to government organizations, to other nonprofit organizations.

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The Power of Insights…and Kiddie Pools!

Innovation plays a key role in The ALS Association’s fight to develop treatments and a cure for ALS and to empower people living with the disease to live their lives to the fullest. During June and July, we are celebrating some of the key innovations helping us change the nature of ALS forever.

The ALS Association teamed up with a group of expert data analysts at Mastercard who spent more than 24 consecutive hours to help our national office and chapters gather insights into improving operations and strategies to advance our mission to find a cure for ALS.

Continue reading The Power of Insights…and Kiddie Pools!

How the ALS Ice Bucket Challenge Has Helped Spur Gene Discoveries

Innovation plays a key role in The ALS Association’s fight to develop treatments and a cure for ALS and to empower people living with ALS to live their lives to the fullest. During June and July, we are celebrating some of the key innovations helping us change the nature of the disease forever.

Before the ALS Ice Bucket Challenge, The ALS Association allocated $6 million annually to funding ALS research. After the ALS IBC, the Association has budgeted about $18 million per year to research and has so far committed $84 million to ALS research. From this investment, there has been massive payoff in a significant increase in ALS gene discoveries.

SOD1 was the first ALS gene discovered, in 1993. Since then, over 30 genes have been reproducibly linked to ALS. And in the almost four years since the ALS Ice Bucket Challenge, five new ALS genes have been discovered and validated to contribute to the development of ALS – KIF5A, NEK1, C21orf2, TUBA4A, and TBK1. This rate of gene discovery is the highest in ALS history.

Every new ALS gene discovered represents a new ALS therapeutic target. The more ALS therapeutic targets identified, the more likely that a treatment will be found.

The ALS Association has been committed to gene discovery since its founding in 1985. It has supported research that contributed to all the major gene discoveries in the graph below.

Gene discovery graph.jpg

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Ice Bucket Donations at Work: New TDP-43 Mouse Models Shed Light on ALS Disease Pathways

Researchers funded by The ALS Association, through donations from the ALS Ice Bucket Challenge, discovered new evidence on the role that mutant TDP-43 plays in development and progression of the disease. This important work sheds light on novel aspects of TDP-43 biology and provides valuable tools to gain insight into early stages of ALS disease progression and could lead to the development of new therapies.

Mutations in TDP-43 protein account for approximately five percent of inherited ALS cases. The mis-regulation of the mutated protein is central to disease pathways of ALS, but how these mutations cause ALS is largely unknown: both the loss of TDP-43’s function and the gain of its function has been connected to disease development and progression.

Dr. Elizabeth Fisher from the MRC Centre for Neuromuscular Disease in London and colleagues set out to better understand how TDP-43 causes ALS. They describe new TDP-43 mouse models that demonstrate significant and opposite effects on splicing events, which is validated in cells derived from people with ALS.

“This exciting new mouse model expressing physiologically relevant levels of the mutant protein with an ALS-like disease phenotype is an invaluable tool for the research community to better understand disease mechanism and explore novel therapeutic approaches,” stated Dr. Lucie Bruijn, chief scientist of The ALS Association.

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Ice Bucket Dollars at Work: Researchers Explore Promising New Way to Treat ALS

Researchers at Cedars-Sinai in Los Angeles, funded by The ALS Association with ALS Ice Bucket Challenge donations, are dedicated to finding unique avenues for treating ALS. Using animal models, they transplanted specially engineered neural cells into the motor cortex of the brain, the area responsible for muscle movement.

They found that the transplanted cells protected motor neurons (the cells that die in ALS), delayed disease progression, and extended survival in animal models.

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Ice Bucket Dollars at Work: New Method Developed to Help Improve ALS Clinical Trial Stratification

The ALS Association is committed to helping improve clinical trial design, in order to increase trial efficiency that will more quickly lead to effective therapeutics.

We awarded Dr. David Ennist and colleagues at Origent Data Sciences, Inc. two grants to support research exploring how machine learning algorithms, a type of computational tool, can optimize clinical trial design. Dr. Ennist’s work, recently published in the journal Annals of Clinical Trial and Translational Neurology, looks closely at optimizing patient randomization into clinical trials.

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Amylyx Pharmaceuticals Announces Open Label Extension of CENTAUR Phase II Clinical Trial

The CENTAUR phase II clinical drug trial, sponsored by Amylyx Pharmaceuticals and funded by ALS Ice Bucket Challenge donations, began an open label extension, giving people with ALS who completed their trial period an opportunity to continue taking the drug, AMX0035.

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