Yale researchers have developed a way to target RNA with small-molecule drugs, creating a new method for tapping into a countless number of biological mechanisms critical to metabolism and gene expression.
“There is tremendous interest in targeting RNA with small molecules,” said Yale biochemist Anna Pyle, whose lab conducted the research. “Despite this potential, there has been limited progress on the development of small-molecule drugs that bind RNA, and a pervasive view that RNA is not a viable therapeutic target.”
Pyle and her colleagues focused on RNA targets within pathogenic yeasts, opportunistic fungal organisms that are especially problematic for patients with compromised immune systems, including patients with implanted devices, neonatal patients and cancer patients.
Pyle’s team identified small molecules that would inhibit a particular group of large, self-splicing ribozymes found in bacteria, plants and fungi — but not found in mammals. These RNA molecules have an elaborate structure that contains tiny, solvent-accessible pockets for a small-molecule drug to bind with the larger molecule.
The researchers also chose this group of RNA molecules because of their crucial role in fungal metabolism.
“Our work establishes that RNA is ‘druggable,’ and that refractory pathogens such as pathogenic fungi and drug-resistant bacteria can be attacked by targeting their unique RNA repertoire,” Pyle said. “It demonstrates that the burgeoning landscape of functional RNA elements contains a wealth of potential targets that can be modulated by small molecules, opening a new frontier in molecular pharmacology.”
The study appeared Oct. 15 in the journal Nature Chemical Biology.
Firm brings disease education to Arabic speakers
Vanessa Research Inc. (VRI) has launched educational website focused on microvillus inclusion disease (MVID) translated into the Arabic language.
The website explains the mechanism of MVID, provides targeted resources for physicians and other caregivers, and publishes new scientific research and other news and information related to this rare genetic disease.
MVID is a genetic disorder causing affected infants to develop persistent and severe diarrhea within hours, days or months after birth. This prevents patients from taking in necessary nutrition and may significantly shorten their lifespan. While MVID is quite rare, many documented cases are in the Middle East, driving VRI to focus on bringing information about the disease to this region.
VRI is a Hamden-based medical research firm.
PatientWisdom makes waves at confab
The New Haven digital health company PatientWisdom Inc. was named “Most Patient-Centered” and an “Audience Favorite” at the second annual Health Tech StandOut! Competition during the 2018 Connected Health Conference Oct. 19 in Boston.
The competition highlighted products and technologies used by patients or consumers of health-care or wellness services outside of the hospital environment, and that addressed the theme of the conference: “Balancing Technology and the Human Element.”
PatientWisdom’s mobile-responsive digital site, which helps patients share “stories” about what matters to them and delivers a one-screen summary via the electronic health record to every member of the care team, was voted the Most Patient-Centered offering demonstrated at the conference.
PatientWisdom’s digital solutions have been adopted by Yale New Haven Health, Froedtert & the Medical College of Wisconsin, OSF HealthCare and other advanced health systems. Learn more at patientwisdom.com.
