Advanced medicines from small molecules on the horizon

Large designer collections of new small molecules with the potential to become important in the creation of new medications are under development by three University of Arizona researchers thanks to a new $1.1 million three-year grant from the National Institutes of Health (NIH).

“Small molecules, typically defined as those with molecular weights of 500 or below, are valuable for treating diseases, and most medicines marketed today are from this class of molecules,” said lead UA researcher Christopher Hulme, Ph.D., professor of medicinal chemistry in the College of Pharmacy and member of the BIO5 Institute and the Arizona Cancer Center.

“Since the turn of the century, scientists have implicated thousands of proteins in diseases such as cancer and diabetes, yet we are still in need of small molecule modulators to use what we know about these proteins to create advanced medicines,” Hulme said. “Some of the benefits of drugs developed from small molecules are that they are stable, bio-available, less expensive to manufacture, and can be taken orally.”

This NIH grant is fueling collaborative efforts between Hulme, Victor Hruby, Ph.D., Regents' Professor emeritus of chemistry in the College of Science and a BIO5 member, and Ruben Vardanyan, Ph.D., a research professor in the Department of Chemistry. Drs. Vardanyan and Hruby have more than 30 years of experience creating new drugs for use in patients.

Nathalie Meurice, Ph.D., and Joachim Petit, Ph.D., from the Translational Genomics Research Institute in Phoenix provide chemoinformatics expertise to this newly funded project.

Lead researcher Hulme has extensive experience in each phase of the drug discovery process and had responsibility for more than 50 individual targets over a 13-year period. At Eli Lilly, he led a project concentrated on investigating treatments for Alzheimer’s disease. While working at Amgen, he worked on an Investigational New Drug project for a novel therapy for pain. At RPR (now Sanofi-Aventis), Hulme worked on medicinal chemistry projects in bone metabolism, inflammation and immunology.

The move from industry to the UA three years ago has offered Hulme a collaborative academic environment with an interdisciplinary research approach that spans multiple therapeutic areas, including cancer and diabetes.

“Academic research has the advantage of being very innovative and investigators are continuously more inventive in order to be more competitive for federal grant awards, Industry has the bench-to-bedside experience needed to move a drug candidate through the drug discovery process,” says Hulme. “Partnerships between industry and academia offer a unique opportunity to capitalize on the strengths each area has to offer.”