IRVINE — Dr. David Scharp figures he just got the chance of a lifetime. For two decades, the noted St. Louis surgeon has been working on new treatments for diabetics. He joined Neocrin Inc. recently and found himself in the thick of a race to develop a new method for attacking diabetes.
The small research firm is on the cutting edge of technology as it experiments with an implant that can act like a pancreas and eliminate the need for insulin injections.
"This is the assault on the summit!" says 49-year-old Scharp, as the company's new chief scientific officer escorts a visitor through Neocrin's spotless laboratory. "This is the chance to realize a 20-year dream."
Scharp's appointment at the Irvine company comes just after Neocrin secured $12.5 million last month in fresh funds from venture capitalists to continue its studies on large mammals, such as dogs and baboons.
Indeed, investors share Scharp's enthusiasm, pouring a total of $28.5 million into Neocrin since it was formed three years ago.
"The potential could be staggering," said Dr. Philip Noguchi, a federal Food and Drug Administration official, "but that's only if it works, of course."
With more than 1 million diabetics in the United States and Europe spending billions of dollars on insulin, needles and related items, the market for something that can replace the drug is huge. If all goes well, Neocrin believes its device eventually could generate more than $1 billion a year in revenue.
To be sure, other companies are working on new treatments, and the race to develop an artificial pancreas is difficult, with major clinical hurdles ahead.
Neocrin is one of the major front-runners with plans to begin clinical trials in human beings next year. Before it does, the privately held company plans to go public to raise the cash it needs for what will likely be four more years of testing.
Neocrin wants to perfect an artificial pancreas, containing insulin-producing islet cells, that would better control a diabetic's insulin requirements than injections. Islets are a cluster of cells in the normal pancreas that produce the insulin the body needs.
Company researchers believe that among its other benefits, the implantable device could help prevent blindness, amputations and other severe complications that often debilitate diabetics later in life.
Industry experts say Neocrin's experienced executives and scientists, loyal investors and laboratory successes are keeping it on track.
Scharp joined the company as an executive vice president after spending a year advising it while on leave from Washington University in St. Louis. Scharp and his collaborator, Dr. Paul E. Lacy, a Washington University researcher who is on Neocrin's scientific advisory board, have completed more than a dozen human-islet transplants.
Those transplants of human islets into diabetics have reduced the patients' need for insulin shots. Though that's a medical victory, Scharp concedes, it has little hope of widespread use because of the scarcity of human pancreases.
At Neocrin, Scharp will oversee the scientific development of its artificial pancreas, shepherding it through large animal trials and, assuming the tests succeed, human trials as well.
Neocrin's prototype isn't impressive to the naked eye.
"Like a stay for a shirt collar?" Scharp suggested as he picked up a thin, two-inch bit of white polymer that could be part of the final product. That tiny item could contain the cells that the diabetics need to produce insulin in the body.
Like scientists elsewhere, Neocrin's researchers are experimenting with the idea of implanting pig islet cells in human diabetics. Though pig insulin long has been used as an effective treatment for human diabetes, researchers must find a way to protect pig islets cells from being rejected by the human immune system.
Last year, Neocrin took a step toward safeguarding pig islets.
Researchers tested a new membrane containing pig islets in rats, mice and dogs. Test results suggest the membrane has pores small enough to block the host's large anti-body proteins from attacking the islets. With a prior version of membrane that had pores too large to exclude antibodies, Neocrin figured it would have to limit its initial testing to a small number of diabetics who take drugs to suppress the immune system.
With the new membrane, Neocrin will try to persuade federal regulators to permit tests in a wider population of patients, mostly those who contract the disease in childhood and must use insulin injections but not immune-blocking drugs.
Such advances are attracting investors. The test results are a "milestone," said Dr. Alix Marduel, a general partner with Sofinnova Inc., a San Francisco venture capital firm that invested in Neocrin's recent financing.