Drs. Scott Larsen and James Shayman Collaborate to Achieve NIH Funding for Moving Small Molecule Into Clinical Development
Scott Larsen, PhD, Joseph Burckhalter Collegiate Research Professor of Medicinal Chemistry, and Director of the Vahlteich Medicinal Chemistry Core (VMCC), and James Shayman, MD, Agnes C and Frank D McKay Professor of Pharmacology and Internal Medicine, have been awarded a UH2/UH3 grant that is associated with the Blueprint Network for Neuroscience Research, a National Institutes of Health funding mechanism, to facilitate translation of basic science discoveries to clinical studies. The purpose of the grant is to fund small molecule drug discovery and development for disorders of the nervous system. Drs. Larsen and Shayman plan to use this grant to fund Investigational New Drug (IND)-enabling studies on their best compounds, which they hope will lead directly to clinical trials.
“Currently, patients with lipid storage diseases of the central nervous system, such as Tay-Sachs, Sandhoff, and Gaucher Type 3, have no treatment options,” said Dr. Larsen. Available treatments have their limitations as well, “Cerdelga, which was just approved by the FDA for treatment of Gaucher Type 1, only works in tissues outside of the central nervous system.”
“Our project really highlights how academic drug discovery collaborations can address an unmet medical need,” continued Dr. Larsen. “In 2007, the newly formed VMCC began a collaboration with Dr. James Shayman, a world-renowned expert on lipid storage diseases and co-inventor of Cerdelga, to modify the chemical structure of Cerdelga in a way that allowed it to penetrate the CNS. This required a level of drug development knowledge and experience on the part of the VMCC that is typically not found in academic labs. It is also important to acknowledge the key contributions made by the U-M Pharmacokinetics Core, directed by Dr. Duxin Sun. The data they provided was critical in designing and selecting our best compounds."
The Blueprint Network is part of NIH’s new commitment to translating basic science discoveries into real medicines. Commenting on this honor, Dr. Larsen said, “We are delighted that they have selected our project for support. Only seven other projects nationally are funded by the BPN. They will supply both drug development expertise and all of the resources necessary to complete an IND application for clinical studies.”
Scott Larsen spent over 22 years in the pharmaceutical industry as a synthetic medicinal chemist before joining U-M in 2007. He is currently Director of the Vahlteich Medicinal Chemistry Core (VMCC), the mission of which is to assist the University of Michigan biomedical research community in the development of biologically active small molecules, usually identified by high throughput screening (HTS). This entails the design and synthesis of new analogs to improve potency and selectivity, and the refinement of physicochemical properties necessary for good cellular and in vivo activity (including solubility, permeability, and metabolic stability).
