A Jupiter scientist’s ambitious proposal to take on more than 30 incurable illnesses including Lou Gehrig’s Disease has won a prestigious $4.8 million, five-year award announced Tuesday by the National Institutes of Health.
Matthew D. Disney, a professor on the Florida campus of The Scripps Research Institute, has been awarded a 2015 Pioneer Award, one of 13 given this year.
The award is part of the federal agency’s High Risk, High Reward research program, officials said. Disney pitched a plan to defuse devastating illnesses by getting diseased cells to open their doors to chemical con artists that seek out and work only on them.
“Really what we want to do is show this works not just for one disease but many diseases,” Disney told The Palm Beach Post. “This is an immense challenge.”
The idea, as he put it in a statement that formally announced the award, is to “trick disease-affected cells into making their own drug against diseases for which there are no known cures.”
Still, a lot of effort lies ahead to see if promising early work can turn the tide against unforgiving diseases.
In 2014, Disney and colleagues showed the technology can successfully work against myotonic dystrophy type 2, described as a relatively mild and uncommon form of the progressive muscle weakening disease. It is said to be caused by a defect in genetic material in which some coding is repeated more times than normal.
Now he wants to target more than 30 incurable diseases that include amyotrophic lateral sclerosis , or ALS, also known as Lou Gehrig disease; fragile X syndrome, the most common cause of autism; and Huntington’s disease.
Money raised by the ice bucket challenge online fad — Disney and fellow researchers doused themselves at one point — led to a $500,000 grant from the ALS Association announced in July. It will be shared between Scripps Florida in Jupiter, where Disney works, and the Mayo Clinic in Jacksonville.
“It’s exciting, but also sad,” Disney said in August. For those who have the disease, “time is not on their side. That’s what keeps me up at night.”
The NIH award ups the ante and widens the horizon. The program established in 2004 “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 in a statement. “We look forward to the remarkable advances in biomedical research the 2015 awardees will make.”
The award represents “a great honor not only for Matt and his lab, but for The Scripps Research Institute as well,” said institute president-elect Steve Kay. “Matt’s work represents the kind of research the institute is known for — bold, imaginative and aimed at helping those people with the greatest medical needs. Our congratulations to Matt on this well-deserved achievement.”
Dale Boger, chair of Scripps’ department of chemistry, said “we are thrilled” by the news.
Disney said the honor of the highly-competitive award suggests “the scientific community thinks we are on to something” to treat and study debilitating neurological diseases.
“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 said.\