DEMONSTRATE PERSISTENCE

Pete Lollar, an Emory hematologist, recalls seeing a cartoon that illustrated the long odds; thousands of pills were pouring into the open end of a huge funnel, while one measly survivor made it out of the bottom. The image resonated with him because he lived through the process with a drug he invented to treat acquired hemophilia A, a rare condition that usually strikes older people.

Lollar started investigating Factor VIII, a protein that helps blood clot, four decades ago. In 1992, he and his lab partners at Emory discovered a way to synthesize the substance to treat hemophilia patients. After failing to find a pharmaceutical company to license the technology, Lollar co-founded a company with the university to license the technology and develop a medication.

Twenty years later, after several pharmaceutical firms had licensed or sublicensed the technology, the drug had still not won federal approval. It was in the wilderness between research and commercialization, what people in drug development call “the valley of death.” Lollar blames economics. The market for the medication was relatively small—in pharmaceutical parlance, it was an “orphan drug”—so none of the successive companies felt an urgent need to spend the money to finish testing. Lollar kept at it. Finally, in 2014—twenty-two years after Lollar disclosed his invention to Emory—Baxter International won FDA approval for his hemophilia drug, obizur.

“What I learned,” Lollar says, “is that getting a drug to market is an idiosyncratic process. There are no easy formulas, and there are many routes to failure. Once you disclose an invention, the university has to like it well enough to spend money to prosecute the patents. And then it has to try to find a company to license it. The company has to be willing to spend the money to perform preclinical work and do clinical trials. The longer it goes, the more expensive it gets.”

Even when a drug is approved, the struggle isn’t over.

Christian P. Larsen, professor of surgery in Emory’s Department of Transplantation, co-developed a drug called belatacept that reduces the chance of kidney transplant rejection. After nineteen years of development, Bristol Meyers Squibb secured FDA approval for the medication in 2011, and Larsen and his co-discoverer, Thomas C. Pearson, executive director of the Emory Transplant Center, marked the triumph by performing the first kidney transplant using the drug.

But that was just the beginning.

“We tell the story of belatacept like it was linear,” Larsen says, “but it wasn’t. There were so many fits and starts, peaks and valleys, other candidates that we decided not to pursue. And then when you get the drug approved, there really isn’t a moment when you get to declare victory.”

He continues. “That was a big revelation for me. It’s never over. You have to do follow-up studies, keep investigating the best way to use it, find the most effective companion drugs. If you take the long view of solving problems for patients, drug discovery is an ongoing process.”

A NEW GENERATION OF ‘ACADEMIC ENTREPRENEURS’

One of the biggest changes in drug development has been the role of private industry. Research universities used to think their work had ended after scientific discovery; it was the job of pharmaceutical companies, not academia, to develop innovations into medications.

Now institutions such as Emory embrace partnerships with biotech and pharmaceutical companies.

“Ultimately, a lot of the ideas we’ve had only move forward if someone in the for-profit sector wants to move them forward,” says Vikas P. Sukhatme, dean of the Emory School of Medicine. “Most drugs are developed by indus try. And most devices are developed by industry. So, we do