Meet the New Medicines, page 2

Pharmaceutical companies changed the world of medicine by using organic chemistry to synthesize small molecules, like aspirin and corticosteroids.

These small-molecule drugs bind to a target protein in the body. When a drug binds to the right target, it changes that protein’s shape and, accordingly, its function.

A major bottleneck in small-molecule research is finding the right target proteins that are involved with disease. Discovering targets is largely achieved by exploratory research, often in universities or public–private partnerships, and turning them into products is the development phase of work that is largely performed by companies.

Manufacturing small-molecule drugs

Small molecules make up around 90% of medicines on the market today. Their composition is so precisely defined that companies can produce them using any number of different manufacturing processes. The challenge is straightforward: make sure that in the end, they’ve produced their molecules to the right specifications.

HSCI’s innovations in small-molecule drugs

HSCI scientists are using stem cells — which have the potential to become many types of cells — to improve the discovery process for small molecules.

Researchers are converting patient stem cells into specific cell types affected by disease — for instance, neurons for neurodegenerative diseases — so that they have a model of the disease in a petri dish. Then, researchers use the cell models to rapidly screen thousands of small molecules, searching for drugs that improve the cells’ condition.

This approach makes drug discovery faster because it allows researchers to test drugs on human cells “in vitro” well before trying them on actual people. It also enables the discovery of drugs that are more specific to patients’ unique genetic mutations.

By understanding how drugs affect certain cell populations, HSCI researchers can also use small molecules to improve the body’s regenerative ability. For example, hearing loss occurs when certain cells in the inner ear get damaged because they cannot repair themselves. Researchers are developing small-molecule drugs that encourage those cells to regenerate.

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