The ALS Association’s Early Investment in Antisense Technology Is Having a Real Impact

Our Every Drop Adds Up campaign builds on the idea that people coming together for a common goal can make the impossible happen, just as it did four summers ago when the ALS Ice Bucket Challenge soaked the world. Every person helped, research project funded, story shared, discovery made, piece of legislation passed, and auction bid placed it all adds up!

When Dr. Timothy Miller and his colleagues from Washington University in St. Louis published preclinical data in The Journal of Clinical Investigation last month, showing how second-generation antisense drugs were effective in ALS mouse and rat models, it served as a vivid reminder that every research investment and discovery adds up.

Antisense technology, which The ALS Association has supported from the beginning, represents one of the most promising potential treatments for ALS. The goal is to prevent the production of the toxic proteins by preventing them from being created in the first place. Toxic proteins kill motor neurons, the cells that primarily die in ALS, leading to muscle weakness and, eventually, paralysis.

Doing so could slow or stop disease progression in people living with ALS. Specifically, an antisense drug selectively targets and binds to the mutated messenger RNA linked with ALS and prevents toxic proteins from being made.

Dr. Miller’s research demonstrates how far antisense technology has come since the first-generation drug used in the original phase I trial in 2013.

The ALS Association began supporting this cutting-edge technology in 2004, in the laboratories of Dr. Cleveland and Dr. Richard Smith at the University of California, San Diego. Since then, our translational approach of supporting innovative research has allowed us to invest over $3.5 million and continues to fund top ALS researchers who are focused on making antisense technology work.

The Association’s early investment in this innovative technology has had a significant impact, which only continues to grow. These successes give us great hope for the future of antisense therapies targeting ALS. Here are some examples of antisense technology successes:

• Researchers are currently testing the optimized second-generation antisense drug in the current phase I/II clinical trial for people carrying the SOD1 mutation, which is still enrolling and predicted to end in February 2019. Notably, there is an ongoing open-label extension study that patients can transfer into after trial completion.
• There are plans to begin a clinical trial targeting C9orf72, the most common gene to cause ALS, by the end of 2018 that The ALS Association has been funding for years under the Neuro Collaborative, a partnership between three leading laboratories in California that is dedicated to driving potential ALS therapeutics, like antisense drugs, quickly toward FDA approval.
• Our translational approach facilitates the development of potential antisense treatments not only for ALS, but also for other neurodegenerative diseases. Currently, there are over 20 antisense drugs in preclinical stages or in clinical trials aimed to treat Alzheimer’s disease, Huntington’s disease, Myotonic Dystrophy, and others.
• In December 2016, the FDA approved an antisense drug, targeting spinal muscular atrophy, a common neuromuscular disease, which is the leading genetic cause of death in infants and toddlers. The drug, Spinraza®, is the first approved treatment for this devastating disorder.

Antisense technology is just one example of how the Association’s global research program has maximized donor investments before and after the ALS Ice Bucket Challenge. Your donations allow the Association to invest in the most promising early-stage ideas from all over the world, like antisense technology, that have the potential to cure ALS.