A new strategy for developing drugs to fight cancer and other diseases

"Chemically Synthesized Molecules with the Targeting and Effector Functions of Antibodies"
Journal of the American Chemical Society

Promising treatments known as biologics are on the market and under development for many serious illnesses such as cancer, but some of them come with high risks, even lethal ones. Now scientists have produced a novel class of molecules that could be as effective but without the dangerous side effects. They report their work on these compounds, which they tested on prostate cancer cells, in ACS’ Journal of the American Chemical Society.

David A. Spiegel and colleagues explain that biologics are protein-based therapies that have revolutionized cancer treatment over the past decade. These compounds work by latching onto malignant cells and then triggering the immune system to destroy them — an approach known as immunotherapy. More than 400 kinds are currently undergoing testing in clinical trials. Although they’re very effective at clearing out cancer cells, biologics have serious drawbacks — including potentially fatal allergic reactions — that are mainly due to their relatively large size. Spiegel’s team wanted to develop an alternative that would be just as effective but without the risks.

The researchers produced a set of molecules that they call synthetic antibody mimics, or SyAMs. These molecules act like biologics by sparking an immune response but are far smaller. In lab tests, a subgroup called SyAM-Ps worked well against prostate cancer cells. Because of their small size, the researchers suggest that SyAMs could avoid many of the pitfalls that have plagued biologics. The compounds could represent an entirely new direction in immunotherapy for treating cancer and other diseases, the researchers conclude.

The authors acknowledge funding from the National Institutes of Health and Bristol-Myers Squibb.