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TSRI Chemist Wins NIH Director’s Pioneer Award of $4.8 Million
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TSRI Chemist Wins NIH Director’s Pioneer Award of $4.8 Million

Matthew D. Disney, a professor on the Florida campus of The Scripps Research Institute (TSRI), has been awarded a prestigious 2015 Pioneer Award from the National Institutes of Health (NIH). The award, one of only 13 given this year, enables scientists to develop groundbreaking approaches with a significant impact on broad areas of biomedical science.

“This program has consistently produced research that revolutionized scientific fields by giving investigators the freedom to take risks and explore potentially groundbreaking concepts,” said NIH Director Francis S. Collins. “We look forward to the remarkable advances in biomedical research the 2015 awardees will make.”

The Pioneer Award, established in 2004, is part of the NIH’s High-Risk, High-Reward Research program supported by the NIH Common Fund designated for scientists of “exceptional creativity.” Disney’s Pioneer Award provides $4.8 million of funding over five years.

“We will use the money wisely to advance precision therapeutics that trick disease-affected cells into making their own drug against diseases for which there are no known cures,” said Disney. “I am honored to receive this award. I have great respect for previous winners and the transformative science that they have accomplished by using support from the Pioneer Award mechanism.”

TSRI Pioneer Award winners have included Professor Thomas Kodadek, Hollis Cline and the late Carlos Barbas III.

Disney’s revolutionary approach uses cells as reaction vessels and a disease-causing defect as a catalyst to synthesize treatments within the disease-affected cell itself. Because the treatment is synthesized only in disease-affected cells, these novel compounds provide highly specific therapeutics that only act when a disease is present, offering potential treatments in a selective, precise and unprecedented manner.

In a 2014 study, Disney and his colleagues used the technology to successfully counteract myotonic dystrophy type 2, a relatively mild and uncommon form of the progressive muscle-weakening disease caused by a type of RNA defect known as a “tetranucleotide repeat,” in which a series of four nucleotides is repeated more times than normal in an individual’s genetic code.

“Given the extremely competitive nature of this award, the scientific community thinks we are on to something potentially transformative to treat and study debilitating neurological diseases,” Disney said. “It has taken us a long time to convince them of this and our persistence has paid off, but there is still much more work to be done!”

Disney’s approach will be applied towards more than 30 incurable diseases, including include amyotrophic lateral sclerosis (ALS, also known as Lou Gehrig disease), fragile X syndrome (the most common cause of autism) and Huntington’s disease.

For more information on Disney’s work, see his laboratory website. For more information on the award, see the NIH Director’s Pioneer Award Program site.

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Professor Matthew Disney will use the award to advance his work on precision therapeutics that trick disease-affected cells into making their own drug. (Photo by James McEntee.)