CHALLENGE ME to revolutionize the treatment of ALS
By Don Cleveland, Ph.D.
Ludwig Institute for Cancer Research, University of California, San Diego
Principal Investigator, The Neuro Collaborative
Committee Member, California ALS Research Network
There was a lot of skepticism in the research community when we proposed development of “designer” DNA drugs as a way to treat ALS. This therapy is an approach called “antisense” oligonucleotide technology (ASO), which works to silence genes that are known to cause a particular disease. The ALS Association was the first funding organization to invest in the research and development of ASO therapies in the treatment of neurodegenerative disease.
From the very beginning, The ALS Association has been the boldest supporter of antisense drug technology, which has been transformative for the development of these promising new therapies. In addition, the cutting-edge research being done through the Neuro Collaborative, a unique academic-industry program in California launched by the Golden West Chapter, received the first $5 million dollars from the 2014 ALS Ice Bucket Challenge, thanks to the generous outpouring of support of our ALS community
With these donations, generated by people like you, the Association has been able to double down on its investment in this research. This includes ASO drugs targeting the two most common genetic causes of ALS, mutation in the SOD1 or C9orf72 genes. These potential therapies are now in clinical trials, and we’re encouraged by the data gathered thus far. The funds from the 2014 ALS Ice Bucket Challenge also enabled identification of new genes that are affected in essentially all people with ALS and that are ideal targets for these drugs.
We are pleased that the Association is continuing with a vision that can see beyond short-term strategies and is continuing to enable the kinds of approaches that could be truly field redirecting. Their dedicated support of antisense technology is a great example of their forward thinking.
There has never been a more encouraging environment to advance the development of therapies, like ASO drugs, for people living with ALS.
The Challenge Me campaign picks up where the ALS Ice Bucket Challenge left off. On behalf of our Chapter’s Board of Directors and Staff, we invite you to join us and the ALS community in challenging others to participate, advocate, and donate to fuel the search for effective treatments and cures for ALS.