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Originally published Sunday, July 22, 2012 at 8:08 PM

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$65M federal grant helps UW turn lab research into medical products

An institute at the University of Washington has won a $65 million grant from the National Institutes of Health to translate researchers' discoveries into useful medical products.

Seattle Times health reporter

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As demands mount to see practical results from publicly funded research, the University of Washington says it's ready to transform researchers' discoveries into useful medical products.

To do that, the UW Institute of Translational Health Sciences (ITHS), which includes other local research institutions, recently received its second five-year, $65 million grant from the National Institutes of Health (NIH).

The money will fund activities that might sound squishy, such as helping scientists engage technical help, run pilot projects and secure the money and expertise to get through clinical trials and start businesses.

In fact, the goal is very concrete and urgent: Move discoveries and inventions out of laboratories and into the hands of people who can benefit from them, in the process returning some of the public's investment to the research institutions and to the region.

For the last many years, that mostly hasn't happened.

"I can't tell you how many ideas that are absolutely fantastic just drop because the inventor had no idea how to take it forward," said Nora Disis, associate dean for translational science in the UW School of Medicine, who heads the ITHS.

With help, ideas and innovations hatched in labs or in the nether regions of technology could become drugs, devices, diagnostic tests, smartphone apps to help patients with particular conditions, or ways to use genetic discoveries to better target drug therapies.

But transforming discoveries into useful products almost always requires a different kind of expertise — one most research scientists don't possess, Disis said.

For example, it might require experience navigating the regulatory maze that awaits a potential drug or device, entrepreneurial skills to set up a business and raise capital, tech chops to tweak smartphone software or medical background in a particular health condition.

It also might require hiring a statistician or a medical ethicist, and gaining the support of a foundation connected to patients and families.

Or all of the above.

Think of the ITHS as creating a giant conveyor belt, Disis says, helping pull great ideas out of the lab, through the regulatory thicket, and into the commercial world of companies and investors to become products helping to improve human health and, hopefully, our regional economy.

This new focus and funding by the NIH has been controversial, criticized by the drug industry and some in Congress as competing with the private sector.

But Disis said ITHS fills a gap that has left great ideas languishing in the lab.

"At the end of the day, people want to see their dollars translated into something that will directly benefit them, their children and their families," she said.

The ITHS conveyor belt recently helped propel a little device that might someday deliver medication to a patient with a brain infection or disease. Like many such bright ideas, it started in a lab — in this case, the lab of UW pharmaceutical researcher Rodney Ho, whose team found a way to bypass the blood-brain barrier that normally prevents medication from reaching the brain.

Seed money from ITHS funded proof-of-concept studies, and helped bring in more grants to keep moving ahead. The ITHS also funded a fellowship for an MBA student to evaluate the market for such a device, which works through the nose, and helped bring in medical experts.

The student, Michael Hite, became the CEO of a company set up to take the discovery the rest of the way. A researcher in Ho's lab, John Hoekman, is now chief scientific officer, and Ho, a co-founder, is a board member.

If the device, now in trials, is successful, the UW likely will benefit from licensing payments and equity in the firm — a typical arrangement, Hite said.

Ideally, he said, he will be able to help other fledgling companies, perhaps even becoming an "angel investor."

"We want to get a virtuous cycle going," Hite said.

Dr. Bonnie Ramsey, a co-investigator with the ITHS, is a pulmonology researcher who knows firsthand how many connections and how much help it takes to get a bright idea to market. In her case, before the ITHS was established, it took a multifaceted collaboration, including a drug company and a private foundation, to do the work behind Kalydeco — the first drug to target the genetic cause of cystic fibrosis.

"It may sound like a simple thing — a scientist finds something in the laboratory, and then they turn that into a pill that saves somebody's lives," Ramsey said. "But it isn't simple."

In the past, Ramsey said, the academic world, often focused on basic research, has lagged behind private industry in pushing products along the lab-to-market pathway.

Never before has there been a coordinated effort to improve efficiency in research, she said.

Lynn Rose, director of scientific administration for the Benaroya Research Institute, notes that the NIH has taken some flak from members of Congress, who are demanding to see results from all those research dollars.

Earlier this year, NIH head Francis Collins said there is a "yawning gap" between discovery and product, and the agency has moved toward bridging that gap. In recent years, Rose said, both drug companies and patent laws have changed, demanding more proof that a discovery works before investing or patenting, but until now, academic institutions haven't had sufficient funding to do that.

Will the push toward commercialization hurt basic research? Collins of NIH has reassured Congress that its basic-research funding will remain steady.

Disis says ITHS isn't duplicating or siphoning off any basic research funds. In fact, everything ITHS hopes to do depends on that "aha!" from the lab or brain of a basic scientist.

The ITHS, which began in 2007 with a similar-size setup grant from NIH, is a collaboration between the UW, Seattle Children's Research Institute, the Fred Hutchinson Cancer Research Center and many other regional organizations.

The institute's mission is ambitious, Disis said, and can only be accomplished in regions such as ours that have a wide range of expertise in fields from biotech to health-outcomes research.

But, she said, it won't be easy because it's never been done before.

"This is a big, hairy, audacious goal. I think people are real interested to see if we can achieve the mission."

Carol M. Ostrom: 206-464-2249 or costrom@seattletimes.com. On Twitter @costrom.

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