Four teams of researchers have received $150,000 to pursue translational research projects through CTSI’s Translational Accelerator Awards. The funding agreements last twelve months, and they’re designed to help quickly move innovations through intermediary stages, such as proof-of-concept, and to develop data to support larger studies in the future.
Epigenetic Therapy of Prader-Willi Syndrome
This project comprises a newly-forged partnership with ConduITS, the Institutes for Translational Sciences at Icahn School of Medicine at Mount Sinai. The project is led at Duke by Yong-Hui Jiang, MD, PhD, a physician scientist with the Duke Department of Pediatrics who has spent the last ten years working with children who have Prader-Willi Syndrome (PWS). His collaborator at Mount Sinai, Jian Jin, PhD, is a chemical biologist in the Biophysics and Systems Pharmacology program.
The project is a collaborative effort to develop a novel epigenetic therapy for PWS, a genetic disorder that affects approximately 1 in 12,000 newborns. According to Jiang, this disease manifests in early feeding difficulties, childhood obesity, intellectual development issues, and behavior problems. While physicians can diagnose this condition through early genetic testing, no effective molecular therapy is available to those affected.
The researchers plan to optimize one of two compounds they have identified that shows promise in mouse models. The lead compound, UNC0642, was identified through a small molecule drug screen program in collaboration with Bryan Roth, MD, PhD. The compound was first synthesized by Dr. Jian Jin at UNC before he began working with Mount Sinai. While UNC0642 is promising, it has not been assessed fully for safety and off-target (unintended) effects, and does not show optimal central nervous system penetration or oral bioavailability, meaning it may not be effective as an orally-administered drug.
The funding through Duke CTSI and ConduITS will allow Jin’s team at Mount Sinai to create a derivative compound that may have better central nervous system penetration, lower toxicity, and few off-target effects, while Jiang’s team at Duke will examine the derivative compound for efficacy and toxicity in mouse models of PWS and determine the optimal dose regimen of the novel therapy.
To learn more about the other three projects selected for funding, including:
Integrated Musculoskeletal Physical Therapy in the Patient Centered Medical Home (IMPaC)
Beyond Drills, Cautery, and Suction: Automating the Next Generation of Surgery
Bivalent Influenza Viruses as Next-Generation Vaccines
Please visit the Duke Clinical & Translational Science Institute website.