Massachusetts General Hospital
2018 Harrington Scholar-Innovator
Current osteoporosis treatments focus on slowing down the rate at which bone tissue breaks down, a process called bone resorption. Only one type of drug, parathyroid hormone analogs, is approved as a therapeutic to build new bone. However, it requires daily injections.
After years of research, Dr. Wein identified a small molecule inhibitor targeting salt inducible kinases which was shown to stimulate bone formation and decrease bone resorption. He and his team began tests on genetically modified mice in order to build confidence in the target, and later designed new compounds to identify small molecules that were pharmaceutically acceptable.
With the funding Dr. Wein received from the Harrington Scholar-Innovator award, he and his team initiated a medicinal chemistry campaign and synthesized over a hundred compounds to improve the small molecule, eventually demonstrating subnanomolar potency and selectivity against the target.
By the end of the program, Dr. Wein and his team had a lead compound with dramatic in vitro properties.
After the 2018-2019 Harrington Scholar-Innovator program, Dr. Wein and his institution formed a sponsored research agreement to continue looking for clear evidence of increased bone formation and bone mass. Currently, Dr. Wein is in licensing discussions with the company to continue the work into large organisms.
”We ended the Harrington Discovery Institute project with a really powerful set of tools and were able to continue the work with this industry collaboration.”