Translational research: Medicine man

STEPHEN VOSS/REDUX/EYEVINE

In his last role two years ago with the Opera Vivente in Baltimore, Maryland, Christopher Austin played the Calvinist chaplain in Gaetano Donizetti's Lucia di Lammermoor. The story does not lack for drama: the heroine pulls out a knife in her wedding bed and stabs to death the husband who has been forced on her in place of her true love. On the heels of the murder, the chaplain is the guy who is trying to bring order to chaos, says Austin, a bass-baritone who once considered a full-time career in opera.

Austin's most recent stage part has a certain resonance with his new day job. In September, he was appointed as director of the fledgling National Center for Advancing Translational Sciences (NCATS) at the US National Institutes of Health (NIH) in Bethesda, Maryland. In existence since December 2011, the centre has an ambitious some say audacious agenda that channels the central passion of both Austin and his boss, NIH director Francis Collins: to get more successful medicines into more patients, more quickly. That means forcing the agonizingly slow, failure-prone process of 'translational research' the term of art for moving promising discoveries from the lab to the clinic into a higher gear.

Passion runs high among the sceptics, too. Researchers both inside and outside the agency fear that NCATS the first new centre at the NIH in more than a decade, funded at US$575 million last year will encroach on a finite pot of money that they say would be better spent probing the mechanisms of basic biology and disease. Others question the scale of its mission. With the available resources, how are you going to achieve this? asks Thomas Caskey, a molecular geneticist at Baylor College of Medicine in Houston, Texas. To me, you cannot just take this money and be another biotechnology company and you certainly don't have enough money to be a pharmaceutical company.

NCATS will be neither, Austin responds. What will set it apart, he says, is a focus on overcoming obstacles on the road to drug development, from inadequate toxicology methods to inefficient clinical-trial recruitment, rather than actually producing the drugs. In an era in which more than 95% of drug candidates fail, and a novel drug takes 13 years and more than $1 billion to develop, NCATS has to be focused on logarithmic improvements in the process, says Austin. You can't do this in a brute-force way. You have to do it differently. You have to drive the technology development.

Austin's fans say that if anyone has a shot at making this work, it is him. This guy has got clinical training, industry training and scientific training. If you wanted me to pick a quarterback, this is the quarterback I'd pick, says Lee Nadler, director of Harvard Catalyst, the NCATS-funded clinical and translational science centre based at Harvard University in Boston, Massachusetts. But whether quarterback or maestro, Austin has now to give the performance of his career. The biggest risk he faces lies in not delivering something concrete within 1224 months, says Nadler. Everybody is watching him.

Austin learned early, and at first-hand, about the tragic shortcomings of medicine. One night in 1989, when he was a neurology resident on call at Massachusetts General Hospital in Boston, an ambulance brought in a middle-aged man with end-stage amyotrophic lateral sclerosis (ALS), a disease that slowly destroys muscle power but leaves brain function intact. Patients usually die when their breathing muscles give out.

The man had a 'do not resuscitate' order, but, because of a miscommunication, he had been revived by the paramedics. Furious that he had not been allowed to die at home, he demanded that his ventilator be turned off. Austin complied. Watched by his family and Austin, the man died slowly over three hours, in the end turning blue before his heart monitor flatlined. It was like sitting through the crucifixion, Austin recalls. And I just said: 'I can't do this. There has got to be a better way.'

Convinced that he had to do more, Austin began a postdoc in the lab of Connie Cepko, a geneticist at Harvard Medical School in Boston. There, he dived into developmental neurology, using new tracing techniques to reveal the migration of neural progenitor cells in the budding mouse cortex (C. P. Austin and C. L. Cepko Development 110, 713732; 1990).

He was just really driven. He absolutely loves research, says Cepko. She recalls the day that Austin's wife went into labour with the couple's first child at the Brigham and Women's Hospital, around the corner. I went to the lab and there was Chris sitting as his bench, pipetting away. I said, 'Chris, aren't you supposed to be in the delivery room?' He said: 'It'll be a couple hours [yet]'.

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Translational research: Medicine man

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