ALS Association-Funded Researcher Don Cleveland Wins 2018 Breakthrough Prize in Life Sciences

Dr. Don Cleveland of University of California, San Diego received the 2018 Breakthrough Prize in Life Sciences, an eminent honor to our long-time-funded ALS researcher. He received a $3 million prize, the largest individual monetary prize in science, during “The Oscars of Science” gala in Silicon Valley hosted by Morgan Freeman and aired on the National Geographic channel. He will use the Prize to continue his vital ALS research projects. The Prize was founded in 2013 and honors “transformative advances towards understanding living systems and extending human life,” according to officials. Notable individuals sponsor the Prize, including Priscilla and Mark Zuckerberg, Sergey Bin, Pony Ma, Yuri and Julia Milner, and Anne Wojcicki.

On  receipt on the honor, Dr. Cleveland said: “It’s an extraordinary honor that recognized the efforts of my colleagues and trainees in determining what goes wrong in inherited ALS and the development of a designer DNA drug approach for suppressing the disease-causing gene. Almost no one thought that this gene silencing approach could be made to work in the nervous system. I’m incredibly grateful to The ALS Association for their support – right from the beginning and continuing to today – that enabled the success that is now being celebrated.”

Dr. Cleveland leads an illustrious research career and is well-respected in the ALS field. He was awarded the Prize for his numerous accomplishments in neurodegenerative disease research. Dr. Cleveland played a large role in discovering the molecular disease pathways of inherited ALS, in particular SOD1, which is the second most common inherited cause of ALS. He also is the first to elucidate the role of glia (support cells of the central nervous system) in neurodegeneration. He was also recognized for establishing antisense oligonucleotide (ASO) therapy in animal models of ALS and another neurodegenerative disorder, Huntington’s disease.

The ALS Association is proud to fund and recognize his research achievements for many years. In 1999, The ALS Association, along with the American Academy of Neurology, awarded Dr. Cleveland with the prestigious Sheila Essey Award for his significant contributions to research. This includes his discovery of disease protein tau and helped identify key steps that trigger neurodegenerative disease. Notably, his team pioneered ASO therapy, a project The ALS Association has funded since its infancy in 2004. From our initial investment, this gene silencing therapy for ALS has propelled all the way from an academic laboratory to partnerships with industry to testing the approach in the clinic. Currently, a clinical trial for people carrying the SOD1 mutation is ongoing, with plans to begin a clinical trial targeting C9orf72, the most common genetic cause of ALS, in the near future. His successes in antisense technology extends beyond ALS to other neurodegenerative diseases. In December 2016, the FDA approved antisense technology targeting spinal muscular atrophy, which is the leading cause of death in infants and toddlers. Today, we continue to fund Dr. Cleveland’s cutting-edge research through the Neuro Collaborative, a unique academic-industry program in California that is focused on curing ALS.

Check out a video about Dr. Cleveland’s role in antisense technology.

Thank you to Dr. Cleveland for being a long-time advisor and supporter of The ALS Association, especially to the Milton Safenowitz Postdoctoral Fellowship Program. We congratulate Dr. Cleveland for this great honor and look forward to seeing more exciting news out of his laboratory!

Learn more about the Neuro Collaborative and how The ALS Association supports antisense technology.

These are just some examples of the many ALS breakthroughs we champion that were made possible by you. Donate today to help us keep up the momentum towards finding a cure!




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