Scientists describe cascading pain of budget cuts

A father came to visit ophthalmology researcher Jean Bennett last week to talk about his son, who is swiftly going blind. Her clinical trial of a one-time injection to cure the inherited disease is scheduled to begin in July.

" 'If this was your son, wouldn't you just take him into your basement and inject him? Why do we have to wait this long?' " Bennett recalled his saying.

She added: "I haven't even told him that it could be delayed."

These are the kinds of stories that scientists tell about how events in Washington - stagnant appropriations followed by sequestration, the government shutdown, and now negotiations on future budget cuts to avoid another crisis early next year - affect them.

Military spending drives some states' economies. Biomedical research is critical to Pennsylvania, which ranked fourth in the nation for federal research funding in fiscal 2012, with $1.4 billion just from the National Institutes of Health. Well over half came to the Philadelphia region and supported more than 20,000 jobs, Sen. Bob Casey said Wednesday while touring laboratories at the University of Pennsylvania.

New Jersey received one-fifth the amount of Pennsylvania's allocation.

With medical costs rising even faster than inflation, flat budgets over 10 years have eroded the NIH's buying power for research by 20 percent, its director has said. Sequestration - the one-size-fits-all, multiyear cuts designed to be so horrible that they would force both parties to agree on an alternative a year ago - slashed an additional 6.4 percent.

That could mean $73 million and more than 1,200 jobs lost in Pennsylvania from NIH cuts alone, according to Rep. Allyson Y. Schwartz (D., Pa.), who introduced a bill Wednesday to restore last year's lost funding and supplement this year's by ending a tax break on corporate jets. Schwartz is seeking the Democratic nomination for governor in the 2014 election.

A conference committee created as part of last week's agreement to end the shutdown is considering whether to keep or reduce the NIH cuts.

Casey emphasized the importance of medical research for jobs and competitiveness, in addition to saving lives. "This is a country that figures out how to cure diseases," he said.

"The competition is rising, particularly in Asia," said Larry Jameson, dean of Penn's Perelman School of Medicine.

Penn is Philadelphia's biggest employer and the third-largest recipient of NIH funding in the nation.

Scientists there Wednesday described long-term effects of even short-term spending reductions. Lost grants mean laid-off researchers and graduate students, who may not return to an ongoing project, they said. The undergraduates who would make up the next generation of scientists may switch career choices.

Cardiovascular researcher Daniel J. Rader, chief of translational medicine and human genetics, described how the sequester had already killed a large proposal that had the potential to speed up commercialization of discoveries, creating new companies and more jobs.

Bennett, an ophthalmology professor at Penn, said in an interview that she has worked for two decades to find a cure for choroideremia, moving from lab experiments to mice, and now to people.

The rare genetic disorder on the male chromosome causes progressive deterioration of the retina, leading to near-blindness by college age. Because it is inherited, young men are devastated by what they know is coming, she said; some have committed suicide.

Bennett said her timeline leading to a clinical trial in July may already have been thrown off by the government shutdown, which delayed consideration of the protocol by an NIH committee. More cuts could threaten the specialized lab that has perfected generating the recombinant virus that will carry a normal copy of the choroideremia gene into the retina to stop and possibly reverse damage caused by the disease.

Re-creating that ability would take two to three years, assuming another lab could get funding, she said. Laid-off researchers would take years of expertise with them.

She fished out an e-mail from a mother whose teenage son had just failed his latest vision test; he couldn't see anything.

"My tears mixed with his as I encouraged him that a treatment is close, gene therapy is coming," she wrote. " 'How soon? Will it be soon enough for me?' " her son asked.

"I didn't know how to respond."

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@DonSapatkin