Medicinal Chemistry

Research Focus

We use advanced technologies to discover new therapeutic chemicals that can be developed into disease-fighting drugs.

Millions of people live with life-altering diseases that have few, if any, medicinal treatments, and no cures. By focusing on the invention of new drug compounds for common and rare diseases, Indiana Biosciences Research Institute (IBRI) medicinal chemists seek to make a difference in people’s lives. 

Creating new molecules to treat diseases

The innovative Dr. August M. Watanabe Drug Discovery Laboratory at the IBRI serves as ground zero for drug discoveries. Here, IBRI medicinal chemists and biologists work together to create and test pharmacologically active compounds that have the potential to be developed into disease-fighting drugs.

It starts with our medicinal chemists designing molecules and preparing them by chemical synthesis. Our medicinal chemists tap the expertise of the IBRI’s computational chemists to gain a better understanding of how proposed molecules might interact with their target proteins. Once a new molecule is prepared, medicinal chemists work collaboratively with pharmacologists to test the drug’s activity in biochemical- and cell-based assays.

When a compound shows desirable activity in pharmacological assays, additional tests may take place to advance it to key studies in disease-related animal models. The goal is to create and optimize molecules that demonstrate efficacy and safety in these preclinical studies.

Medicinal chemistry projects at the IBRI

The development of new drug therapies is a long and complex process that requires collaboration among a team of scientists with specialized expertise. The IBRI partners with outside institutions and scientists to advance the development of new drugs.

Finding treatments for rare childhood diseases

In collaboration with Riley Hospital for Children researchers at Indiana University (IU) Health, our medicinal chemists are working to develop treatments for neurofibromatosis type 2 (NF2).

This rare pediatric disease causes benign (noncancerous) tumors to grow along the auditory nerve that carries information between the brain and the inner ear. Symptoms, such as hearing loss, tinnitus and dizziness, appear during childhood or early adulthood. Tumors outside the auditory system cause cataracts, limb weakness and fluid buildup in the brain.

There is no highly effective treatment or cure for NF2. It’s considered an orphan disease, which means it’s too rare (affecting approximately 1 in 60,000 people worldwide) for large pharmaceutical companies to invest efforts in drug development. The IBRI is helping to bridge this gap by exploring small molecule therapies that can target the gene mutation that causes NF2 and stop tumors before symptoms occur.

Developing new therapies for common diseases

Medicinal chemistry has the potential to develop drugs for many diseases, from the rare to the common. IBRI medicinal chemists and colleagues at the IU School of Medicine are collaborating with physician-scientists at the IU School of Medicine to develop new therapies for Alzheimer’s disease and cancers like osteosarcoma. In addition, IBRI investigators are pursuing cutting-edge research in diabetes.

Community partners in drug advancements

We designed the Watanabe Drug Discovery Lab at the IBRI with other chemists in mind. Experienced innovators and start-up companies can use our state-of-the-art lab to safely conduct medicinal chemistry research and design new drugs. We welcome community partners who would benefit from access to the latest equipment and technology or who want to tap the expertise of experienced IBRI chemists.