WNY’s quiet plan to breed new medical discoveries

Jan. 28--You might not have heard of the University at Buffalo's Clinical and Translational Institute, but if you've been downtown, you've likely seen it.

Behind the scenes, and with millions of dollars in investment, the institute is trying to reshape medical research in Western New York.

It takes up about half of the 10-floor building that opened in 2012 on the Buffalo Niagara Medical Campus that also houses Gates Vascular Institute, a $291 million project by Kaleida Health and UB.

On top of that, the institute in 2015 received a four-year, $15 million National Institutes of Health grant, catapulting it among the approximately 60 medical research institutions in 30 states and the District of Columbia that are part of a major federal effort to speed up the translation of basic laboratory discoveries into new therapies, tests and devices.

Among the goals: train scientists to work across different disciplines, encourage research collaborations, support promising ideas, provide services scientists can share, reduce the bottlenecks that slow progress, look for studies that address the pressing health needs of a community and engage the community in those studies.

[Related: Prospectus 2017/Unveiling the New Buffalo]

"We're trying to raise the level of what we do. There is a lot of expertise in Buffalo, but it is not coordinated," said Dr. Timothy Murphy, director of the institute. "The larger concept is you build local research capacity and that builds national capacity."

In sports, success depends on identifying young talent, building great facilities and effectively managing a complicated business. The same goes for medical research, and the city's major medical and scientific institutions see the center as central to reaching a higher level of achievement.

"In research, you will always have more swings and misses than hits. The center puts you in an elite group and helps you get home runs," said Dr. Gil Wolfe, chairman of the UB Department of Neurology.

Wolfe published a study last year that is emblematic of the quality of research the institute wants to see more of out of Buffalo, Murphy said.

Wolfe and his colleagues answered a long-unsettled issue in myasthenia gravis, an uncommon muscular disorder that makes it difficult to walk, talk, breathe or see. The study found that removing the thymus helps patients who don't have a chest tumor. Knowing this, doctors can recommend the surgery earlier and confidently in individuals who struggle with initial drug treatment.

The research, published in the prestigious New England Journal of Medicine, compared therapies in a randomized study -- the gold standard -- meaning that it can show cause and effect. It was big -- the largest study ever of myasthenia gravis -- involving 67 medical centers in 18 countries. And, the results will make an immediate difference for people with the disease, including about 60,000 in the United States.

Murphy lauded the research for its great size, design and impact.

"You've got a definitive multicenter study that makes an immediate effect on patient care," he said.

More than a year into the grant, the institute's key initiatives are taking shape. Among them:

--Pilot studies: A program to fund promising ideas spent $600,000 on 17 projects from 2010 to 2015 that, in turn, leveraged nearly $8 million in federal grants to expand the research further. The institute spent $600,000 in 2015-16 on eight projects, the largest annual amount it has spent since the program started, and is examining projects for 2017.

--Core services: The institute offers expertise in study design, drug development and biostatistics, as well as laboratory facilities and three imagers, including a PET scanner, a powerful MRI and a micro-MRI. All of this puts researchers in a better position to develop studies more quickly that garner major outside financial support, and that participate in multicenter research initiatives, Murphy said.

--Cutting red tape: The idea is to help researchers navigate the bureaucratic steps from study inception to completion. A recent accomplishment was shortening the time it takes for a study to get examined by UB's Institutional Review Board from 80 to 32 days. Such boards review proposed studies to assess scientific merit, researcher conflicts of interest, and ethics, such as ensuring subjects are protected from harm.

--Engaging the community: One of the key bottlenecks in research is recruitment of patients into clinical trials. Many studies are halted prematurely or take much longer than anticipated because of difficulty recruiting patients. The institute is looking at ways to attract more patients, such as a cellphone app that identifies available studies in the region and a focus on research that addresses pressing health needs in the Buffalo Niagara region, including health disparities.

--Mentoring: The institute is supporting junior faculty members with money and advice to give them time to do research, in addition to their other responsibilities, until they become independently funded investigators. The goal is to nurture young talent in translational studies that attempt to move quickly from basic science to an improvement in medical practice.

"We need to recruit the rising stars and train them here," said Murphy, whose research includes a vaccine for otitis media, a common ear infection in children. "We need a research foundation built on the junior faculty."

So far, the institute has operated largely under the radar, even among the medical community. But Murphy is optimistic knowledge of the institute's work will spread.

"Ultimately, our vision is to increase community engagement in ways that improve outcomes, lower costs and increase patients' satisfaction with their care," Murphy said.

email: [email protected]

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