Profile: NCATS—new kids on the block

It's not easy being the new kid on the block. Just ask Christopher Austin who has recently been appointed director of the US National Institutes of Health's (NIH) newest centre—the National Center for Advancing Translational Sciences (NCATS). Devoted to translational medicine, his mission is to cut the time, cost, and failure rate of bringing laboratory research through to successful clinical medicine. Even with a career spanning private industry with Merck to directing NIH's Therapeutics for Rare and Neglected Diseases programme, Austin is under no illusions. “This is the most difficult scientific problem that currently exists”, he says. Other researchers may disagree, but Austin cites the 13—15 years development timescale, more than US$1 billion cost, and 99% failure rate of new drugs as the scale of the problem. “That's not something that serves anyone well.”

But how can NCATS circumvent the bureaucracy, conflicts of the interest, legalities—to say nothing of the scientific challenges—and bring health care to those that need it in their lifetimes? “The ecosystem by which one can do this has changed dramatically in the last 10 years”, says Austin. Academia and industry were not interested in translational problems, he adds, but there is now a proper recognition that, as a multifactorial problem that cuts across disciplines, the relevant groups are more than keen to work together.

Austin refers to NCATS' $20 million Discovering New Therapeutic Uses for Existing Molecules programme as an example of this new willingness to share. It brings eight major drug companies together along with 58 of their drugs that have passed human safety trials, but did not work for their original indication. The hope is that new treatments can be found, including for neglected diseases. Austin is optimistic, saying he never thought he would see drug companies divulging this amount of data on their own molecules in his lifetime. However, sceptics might argue that it's public money being spent on failed drugs to fail again.

Good or bad, the new kid often gets a hard time. And NCATS' inception has not been free of controversy. How will NCATS deliver with taxpayers' money where the leaner, meaner, and more manoeuvrable private sector has not? Publicly funded institutions do not have shareholders to answer to; they do not have to pursue short-term gains that will bring revenues. This, Austin says, will allow NCATS to focus on the bigger problems that are holding everyone back, such as developing 3D human tissue chips to better predict toxicity in new compounds, or undertaking improved clinical trials.

“I'm very positive it will facilitate unlocking some of the bottlenecks in translational research that stop us having a continuum for a development pipeline”, says Patrick Johnston, Director of the Institute of Health Sciences, Queen's University Belfast, UK. “At present the right mechanisms for partnership and funding aren't there.”

Johnston, who is also Director of the UK Medical Research Council's Translational Research Group, thinks a focus on genomics and translational medicine can help treat diseases by redefining them, naming success with the four scales of breast cancer, luminal to basal, which already dictate treatment.

But NCATS is not the only recent development in translation medicine. In September, 2012, TransCelerate, a non-profit organisation made up of ten major biopharmaceutical companies that aims to solve common drug development problems, was launched. Austin does not see this private equivalent as a competitor, and says it will be natural for them to collaborate when they need access to patients in NIH centres for their studies.

Support is also forthcoming from perhaps unexpected places. NCATS' creation led to the disbanding of NIH's National Center for Research Resources (NCRR); its former director Barbara Alving says NCATS will be influential. “It's like when someone throws a pebble and there are a lot of ripples”, she says. “NCATS is not big enough to do this alone but can help establish a culture of collaboration.”

Austin himself notes a challenge in how his new centre meets its mission with a limited budget—80% of which carries on the work of the Clinical and Translational Science Awards previously administered by Alving's NCRR. But Alving says it's time to move forward. “The fact that it's non-categorical and not devoted to a single disease means that it can be more of an evolutionary centre.”

Just 2 weeks into the job at time of writing, Austin is yet to detail specific changes. But he's clear in his mission that NCATS has to do the work of all the ongoing programmes it has inherited in translational research “faster, cheaper, better” than before. Unfortunately, that was the mantra of NASA Administrator Daniel Golding, who pioneered a lower cost approach to spaceflight that proved controversial in latter years as successive missions to Mars failed.

But medicine is a different animal to rocket science, and the new kid around town may yet unblock the holes in big pharma's research and development pipeline. “The time is right for this type of organisation”, says Austin. “If we can't do this better in 5 to 10 years, we have a problem.”

Time will tell.