Biologics: The Next Generation

Even with genomics, these golden apples will remain high-hanging fruit.

MONYA BAKER AND BARRY SHERMAN

The biology behind the first biologic drugs was straightforward. There is no simpler treatment for diabetes than insulin, no simpler cure for dwarfism than growth hormone, no simpler therapy for anemia than erythropoietin. These protein drugs supply the very molecules that patients’ bodies lack, restoring health or lost function. Structurally, the molecules are complex, defying manufacture in a chemical laboratory. That is why, before the first biotechnology companies supplied the drugs, insulin was painstakingly extracted from the pancreases of farm animals, and growth hormone from human cadavers. The ability to generate mass quantities of these molecules by converting cells into chemical factories is perhaps biotechnology’s greatest contribution to medicine. Biotechnology provided an essential tool—recombinant DNA technology—to make these drugs cheaply and reliably. The naturally produced proteins were already effective therapies, or at least well understood, so when their recombinant counterparts were made by genetically engineered cell cultures in the ’80s, their success was no surprise.

Next, biotechnology companies pounced on previously discovered proteins that promised to become medicines. The appropriate genes were quickly identified and cloned, and their uses as therapeutic proteins patented. By the early ’90s, however, the supply of promising proteins waiting to be made into drugs was depleted. But with the publication of the human genome nearly a decade later, scientists, industrialists, and investors delighted once again, proposing that genomics—the study of genes, gene products, and their functions—could bring even more biologic drugs to market. News reports deemed genomics the hottest ticket in biotech and predicted that genomics would help scientists find drugs faster and cheaper, producing hundreds of new biotech drugs by 2010.

Fast-forward to the present day. Now everyone is realizing that genomics offers not a magic wand, but simply the latest tool to probe basic biology. The truth is that the next crop of biologics will have to wait until the science behind explaining each protein’s role in disease is ripe. The harvest will be more difficult and take more time than first thought.