In 1990, Stephen A. Roth, a tenured biology professor at the University of Pennsylvania, didn't dream that he might one day become CEO of a cutting-edge biopharmaceutical company. But his research was starting to attract interest. Roth had figured out how to make certain complex sugar molecules cheaply and in large enough quantities to be used commercially--as medicines or food additives. Abbott Laboratories was interested, and in 1992, it invested in his startup, Neose Technologies Inc. in Horsham, Pa.
Neose's process could lead to new treatments for cancer, bacterial and viral infections, and other diseases. Two other giants--Johnson & Johnson and Bristol-Myers Squibb Co.--have established partnerships. On its own, Neose is already in human trials with new drugs for ulcers and ear infections--and early results are promising.
Industry experts are impressed. "Their technology has enabled a whole range of products that really weren't possible before," says Stephen J. Catani, a vice-president at McNeil Specialty Products Co., a unit of Johnson & Johnson.
John S. Sonnier, an analyst at Vector Securities International Inc., says Neose's greatest strength is its interest "in so many different areas, from vaccines to food supplements to anti-infectives." And more "anti-infectives"--so called because they remove germs rather than kill them--are on the way. "There are more than a dozen pathogenic bacteria with known cell-surface targets," says Roth. "Each one is a potential target for Neose."
Scientists have known for decades that complex sugar molecules play a critical role in many biological processes--from infections to the fertilization of egg cells by sperm. Bacteria and viruses have evolved ways to infect cells by binding to the sugars that occur naturally on their surfaces. Neose's synthetic sugars can thwart that process. When the sugars are delivered to the site of infection, they act as decoys. Germs are fooled into latching on to them instead of the cells they normally infect. The germs and decoys are then cleared from the system--and the infection is blocked.
It sounds simple, but making drugs out of sugars was far from straightforward. Because simple sugars can be linked together in several ways, it is almost impossible to build a compound from its individual components. If even one element in the chain is misconfigured, a different sugar--with a different biological function--is created. Sometimes, the sugars can be extracted from natural sources, such as breast milk or plants. But the process is inefficient and can result in products of varying quality.
FAST AND CHEAP. It's also too expensive. For years, Abbott Labs considered supplementing infant formula with a sugar that boosts the immune system. But just a teaspoonful would have cost several thousand dollars. Then Roth devised an ingenious way to make the sugars using naturally occurring enzymes. Now, in just weeks, Neose can make kilos of Abbott's sugar at a cost that won't drive up the price of formula.
Roth's patented process uses enzymes called glycosyltransferases to string sugars together in correct orientations. Individual subunits are added in a series of steps, each of which relies on a different transferase. "It is an elegant and simple technology," says Barry D. Shur, a carbohydrate biologist at Emory University School of Medicine.
For patients, this elegant technology could lead to new treatments. For Roth, it offers the sweet smell of success.