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Director’s Corner

January 26, 2017: NCATS Academic-Biotech Partnership: A Breath of Fresh Air

Christopher Austin

Readers of this column will know that one of my favorite sayings about translation is that it is a “team sport.” But as any coach knows, teamwork is just that: work. It requires intentionality, goodwill, mutual dependence and knowledge that the team can produce a result that no individual team member could accomplish alone. There is a science to teamwork just as there is to organic chemistry, and since high-functioning teams are so critical to translational success, NCATS strives to understand the general principles of productive scientific teams, and constantly innovates in models of partnership. In fact, developing, demonstrating the usefulness of, and disseminating effective translational team structures is a prominent goal in the new NCATS Strategic Plan. An advance in a potential treatment for a rare and devastating lung disease illustrates why.

Bruce Trapnell, a physician and researcher at Cincinnati Children’s Hospital Medical Center and principal investigator in the NCATS-led Rare Diseases Clinical Research Network, Rare Lung Diseases Consortium, cares for patients with autoimmune pulmonary alveolar proteinosis (aPAP) and seeks to understand and better treat the disease. In aPAP, an immune system malfunction causes proteins and lipids to pile up in the tiny gas-exchange pockets in the lungs, eventually leading to an inability to breathe. The current standard treatment, akin to internal lung-washing, is complicated and dangerous. Patients desperately need a new, more effective therapy.

Dr. Trapnell knew there was evidence that an inhaled version of a protein drug called GM-CSF might effectively treat aPAP, but he had neither the resources nor the expertise to develop the drug so that it might be tested in patients. He partnered with NCATS through its Therapeutics for Rare and Neglected Diseases program. NCATS provided the drug development know-how, resources and regulatory knowledge needed to complement Dr. Trapnell’s disease expertise. However, at that point, the team did not have access to the drug, which belongs to the biopharmaceutical company Sanofi Genzyme, which markets it in injectable form for other diseases. Enter the NCATS Office of Strategic Alliances, which brokered an agreement among Sanofi Genzyme, NCATS and Cincinnati Children’s to enable production and testing of an inhaled version of GM-CSF for testing in aPAP patients. The three partners each had critical expertise and resources which complemented the others, and together they were able to rapidly complete the work required to successfully develop inhaled GM-CSF for a clinical trial. However, another roadblock loomed since the team lacked the study support needed for the clinical trial. So they expanded their team, turning again to NCATS through its Clinical and Translational Science Awards (CTSA) Program. With CTSA Program support, including the recently launched institutional review board (IRB) reliance platform, Trapnell and his colleagues will carry out a safety study of the experimental drug in patients at two centers. The study is set to begin in the coming weeks.

This remarkable story is not yet at its end, but a possible new treatment for patients with aPAP is now within sight. And the success does not stop there: The lessons learned about effective translational teams are now being applied across NCATS and promulgated to the research community. An African proverb says, “If you want to go far, go together”. Together, NCATS’ teams are going farther than ever before, making the translational process more efficient and effective, and thereby getting treatments to patients who so desperately need them.

Christopher P. Austin, M.D.
Director
National Center for Advancing Translational Sciences