UT Southwestern doctors: Moving discoveries from test tube to treatment

Dr. Burgess hosts roundtable discussion at UT Southwestern on biomedical innovation and the 21st Century Cures Act.

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Washington, September 2, 2015 | comments

It’s too expensive and takes too long to move medical discoveries to the patient's bedside, and federal support and funding for biomedical research in the United States has dropped more than 22 percent in inflation-adjusted dollars since 2003.

Medical doctors, Ph.Ds and politicians gathered Tuesday at UT Southwestern Medical Center in Dallas to discuss the 21st Century Cures Act. The legislation to support biomedical research recently passed the U.S. House and is being considered by the Senate. Its broad aim is to accelerate the discovery, development and delivery of promising new treatments for patients

“The goal of the 21st Century Cures Act is to give you the capability to cut through the red tape to decide what’s the most most innovative, game-changing research, and get it done,” U.S. Rep. Joe Barton told UT Southwestern doctors, scientists and department heads at the event.

Barton and fellow Texas Republican U.S. Rep. Michael C. Burgess, an ob-gyn and a former resident at UT Southwestern, joined UT Southwestern President Daniel K. Podolsky and others in the roundtable discussion.

Barton said the prognosis for the 21st Century Cures Act is good.

“I will guarantee the people in this room that the 21st Century Cure Act is going to become law,” Barton said.

Dr. Marc Diamond, a neurologist recently recruited from Washington University to UT Southwestern, said consistency needs to be built into the research funding pipeline.

Burgess, an architect and co-sponsor of the legislation, said consistency is one aim of the act.

Barton told Diamond “you can’t go year-to-year, I get that,” but added that, because of the budget deficit, “you can’t expect increases every year.”

“The medical research budget is competing against the defense budget and the highway budget and all of those,” Barton said.

Diamond, professor of neurology and neurotherapeutics and director of the Center for Alzheimer’s and Neurodegenerative Diseases, said in addition to prolonging human life and easing suffering, there are big economic advantages to funding medical research.
“I wanted to emphasize the implications on the general economy,” Diamond said. “If we can keep people healthy, keep people’s brain functioning at a higher level for longer, it can have many, many benefits.”

Eric Olson, professor of molecular biology and chairman of that department, said UT Southwestern is making great strides in the fight against muscular dystrophy through “genome editing,” in which DNA is inserted, replaced, or removed from a genome. Muscular dystrophy is a hereditary condition typically striking men in their 20s and marked by progressive weakening of the muscles.

“Changing a single letter in the genetic code can change a progressive disease,” said Olson, a National Academy of Sciences member. “A single letter in the book of life can essentially cause muscular dystrophy. We believe this (genome editing) approach can provide benefit and maybe cure 80 percent of children who have this disease.”

Burgess said the legislation would establish an Innovation Fund at the National Institutes of Health to renew federal support and funding for biomedical research. The legislation also would set new guidelines for faster FDA approvals of drugs and medical devices.

“It is crucial that America remains at the front lines of biomedical research and care,” Burgess said. “These investments lead to exponential advances in medicine.”

Recently, regenerative medicine researchers at UT Southwestern identified a cell that replenishes adult heart muscle. Researchers have been trying for decades to find the specialized cells that make new muscle cells in the heart, because most cells of that type don’t replenish themselves after a heart attack or other significant heart muscle damage.

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