Needing teamwork for translation

Science is like football, said National Center for Advancing Translational Sciences (NCATS) Director Chris Austin during the Partnering for Cures panel, “Molecule to Man (and Woman): Speeding Translation to Human Trials.” Translational science needs true teamwork, everyone in the ecosystem coming together – researchers, pharmaceutical companies, venture capital firms, patients groups, and government – in order for new treatments to be brought to trial faster. But, “the incentive has always been for individual achievement,” Austin said. Instead of playing football, the scientific community is playing golf. And many hope the creation of NCATS will help change that.

“NCATS is a different beast,” Austin said. “It was set up as a collaborative instrument. It’s founding principle is that we have to do these very, very important things that no one else can do.” 

Anna Barker of the Arizona State University School of Life Sciences hopes that mission includes creating systems to speed up translation. “We haven’t thought about creating an end-to-end system that starts with the patient and ends with the patient,” she said. And a key to this system is requiring high quality at every level, for which she feels the scientific community needs standards. “We have not thought about how to deliver molecular findings to patients in a high-quality, controlled way,” she said. 

Todd Sherer of the Michael J. Fox Foundation advised caution. “The challenge is finding the balance between overstandardization vs. innovation,” he said.

Some steps that could speed translation involve solving problems that have no return on investment, so it is difficult for researchers or big companies to justify using the resources. “How we use our resources has to be ever more strategic,” said Dale Edgar of Lilly Research Laboratories, who said his company is pursuing success by identifying “pillars of excellence,” such as maintaining quality standards of practice for the data it stores.

Research decisions can’t be made in a vacuum, said Stelios Papadopoulos of Exelixis, especially not in an investment vacuum. Decisions still must be made to get the maximum return on investment. “I have not seen a compelling investment proposition to go in and engage in [translational research],” he said.

NCATS, which is free from needing to find a cure for a particular disease or make a particular return on investment, can help, said Austin. He feels this gives the agency an obligation to work on the general issues that will help advance translation.

The panelists agreed that true collaboration is key. Every stakeholder has to go “all in” for the collaboration to work, cautioned panel moderator Margaret Anderson of FasterCures. Edgar pointed to the Innovative Medicines Initiative in Europe as a good example of all stakeholders working together to find and overcome critical gaps in driving innovation to the patient. He said the exciting thing about NCATS is “thinking about the patient as the end point and not the next grant.” Innovation, he said, “requires a line of sight to the patient from the earliest point.”

“Something NCATS brings to the table is leadership, galvanizing the industry and all of the players,” said Sherer. He advised Austin to plan for some early accomplishments in the next 18-24 months to prove to naysayers that the agency can affect change.

Edgar encouraged NCATS to continue to foster partnerships, because “great things will happen.”

“The way you make these things work increasingly is to be inclusive,” said Barker. “Get the best input you can up front from as many in the community as you can. Get as much buy-in as you can.”

And Austin is doing just that at NCATS. He reported that he is in talks now with stakeholders in “concentric circles” in the process, including institute directors, academic institutions, patient groups, pharma, biotech, and VCs. As the agency develops, his goal is to keep the patient at the center of everything. “Having patient involvement helps you stay on the critical path,” he said.

Anderson concluded with emphasizing the important role that all stakeholders need to play in medical research. “It is unrealistic to think that NCATS can do it alone. We’ve got to do this together.”

NCATS: Improving Outcomes from the Same Inputs

Last week, over 1,000 participants from all sectors of medical research participated in a virtual town hall with the interim leadership of the new National Center for Advancing Translational Science (NCATS) at NIH to discuss the status of the effort. NCATS is the first new Center at NIH in a dozen years.

Convened by FasterCures as part of its Webinar series, it was a follow-up to a previous FasterCures web event held in March 2011 in which NIH director Francis Collins had laid out the vision for this new Center.

Dr. Tom Insel, acting director of NCATS (and director of the National Institute for Mental Health at NIH) laid out the Center’s mission, which in brief is “to pursue opportunities for disruptive innovation” in the process of translating promising discoveries into new diagnostics and therapeutics, and to “study the pathway and reengineer the pipeline” in ways that will benefit all players in the system. NCATS, he said, “is disease agnostic. We’re looking at generic innovations across many different diseases.”

Insel was careful to emphasize that the purpose of NCATS is to “facilitate — not duplicate — other translational research activities supported by NIH; to complement — not compete with — the private sector; and to reinforce — not reduce — NIH’s commitment to basic research.” He said that “most of the pieces of NCATS existed before [within NIH]. We’re trying to improve the outcomes from the same inputs.”
Insel summarized some of the innovative programs that are now housed within or are being contemplated for NCATS, such as :

• The NCATS Pharmaceutical Collection (NPC), a comprehensive resource of 3,800 approved and investigational medicines to facilitate repurposing of medicines;
• Research Electronic Data Capture (REDCap), a Secure Web application that enables investigators to: create standardized surveys, easily transfer data, and export data into a variety of statistical programs;
• The Learning Collaborative, a cross-sector collaboration to repurpose an approved drug for use in leukemia (which will be the topic of a FasterCures Webinar on March 21st);
• An NIH‒FDA‒DARPA collaboration that aims to develop a tissue chip that mimics human physiology to screen for safe, effective drugs; and
• An NIH–industry “compound rescuing initiative,” which would match compounds from pharma’s “virtual medicine cabinet” with NIH-funded scientists’ innovative ideas for new uses.

Margaret Anderson, FasterCures executive director who moderated the web meeting said that NIH’s efforts to establish NCATS is an efficient and effective way to thread the needle of existing work and make headway on de-risking the medical research process.

Dr. Chris Austin, Director of NCATS’s Division of Pre-Clinical Innovation and Dr. Kathy Hudson, NCATS Acting Deputy Director both answered questions from participants about collaboration with other stakeholders. Austin said he sees NCATS “as a collaborative instrument,” and that they are seeking the best ideas and best opportunities from outside researchers and funders. Hudson said “partnerships are an essential ingredient for NCATS,” and that input would be sought via a number of advisory groups, in addition to working with industry and other partners on specific projects.

Dr. Josephine Briggs, Acting Director of NCATS’s Division of Clinical Innovation, answered questions about the future of the Clinical and Translational Science Awards (CTSA) program, a network of 60 academic institutions which is the largest single component of NCATS, representing about 78% of its budget. She said that “the CTSA consortium is absolutely essential in the implementation of best practices” developed through NCATS, and that going forward NIH would be “focused on making sure the CTSAs are truly a networked consortium.”

Insel also noted that “unique and in some cases unprecedented scientific opportunities that exist drove the creation of NCATS.” He emphasized that this new center is a catalyst for medical progress, leveraging larger investments at the 26 other institutes and centers at NIH to make sure they have a greater impact. In the long term, he said, success will be measured on whether NCATS can catalyze clinical research to be “cheaper, faster, and better.”

Patients are waiting.

View archive of the Virtual Town Hall with NCATS leadership.