Dr. Disney’s research confronts diseases that include Alzheimer’s, ALS, muscular dystrophy and, recently, the coronavirus.
JUPITER — A scientist at Scripps Research Institute studying brain diseases and the novel coronavirus has received a hefty grant from the National Institute of Neurological Disorders and Stroke.
The organization, part of the National Institutes of Health, awarded $11 million to Matthew Disney, a Scripps chemistry professor who leads a 25-person lab in Jupiter. Disney’s research centers on RNA, the single-stranded genetic material involved in cell regulation and other actions, and its role in diseases that include muscular dystrophy, ALS and Alzheimer’s.
"This really allows us an opportunity to focus in on the science. ... This stability is unheard of," Disney said. The institute will dole out the $11 million Research Program Award over up to eight years.
>>Cerabino: Wellington equestrian estate prompts pandemic political potshots at Illinois governor
The thrust of Disney’s research consists of identifying shapes and regions of RNA and pairing them with chemicals to be used in drugs, he said.
The hope is that the molecules stick to and manipulate the RNA to alleviate disease concerns. Utilizing a system Disney created for such work, his lab has already identified compounds that are being studied as possible treatments for Fragile X syndrome, muscular dystrophy and inherited ALS, Scripps said in a news release.
It’s sort of like flipping a switch. If regions are disease-driving, Disney said he wants to essentially turn those off. Disney wants to do the opposite if RNA segments are contributing to the disease because they are not being expressed.
Many drugs for these diseases currently target the larger proteins that are built with RNA. Some only address disease symptoms, Disney said, which is different from the strategy he works with.
"What we’re uniformly doing here is we’re targeting the root cause of the disease," Disney said. "And oftentimes that’s RNA."
>>Bartender sues Gov. Ron DeSantis over ’100 percent arbitrary’ closings
"Matt Disney’s work has changed the landscape of what scientists now consider ‘druggable targets,’ and in the process, reinvigorated research on multiple incurable diseases, including muscular dystrophy, ALS and advanced, metastatic cancer," Douglas Bingham, Scripps Research’s executive vice president, said in a statement.
Disney is using the same foundation of research to study the coronavirus, which has an RNA-based genome.
"That technology (we use) is broadly applicable," he said.
Disney and others have mapped the structure of the coronavirus’ RNA genome and are troubleshooting to see what chemicals used in drug treatments can stick to it.
>>Sheltering during a storm may mean taking Ubers to hotels, asking people to stay at home
The ultimate goal is to stop the virus from being able to reproduce and hijack otherwise healthy human cells, he said.
In a preliminary scientific preprint posted on Cold Spring Harbor Laboratory website bioRxiv, Disney and other researchers wrote that there is likely "a huge reservoir of potential drug targets" to treat the coronavirus.
That’s because they think the coronavirus has an "unusually stable" and ordered RNA structure, researchers wrote in an abstract.
>> RELATED: Top virus expert in Jupiter is optimistic about vaccines, therapy
"We’re going after coronavirus pretty aggressively," Disney said.
Finding drugs that can treat the virus goes hand-in-hand with developing a vaccine, Disney said.
He added that researching the coronavirus is a long-term investment — because it establishes an infrastructure that could pay off during the next threat to public health.
"My role here is to try to lay a foundation to have not a vaccine that can be given before you have the disease, but to deliver a medicine these patients can take while they’re suffering because they weren’t vaccinated."