March 2011

Drug discovery: major challenges

In addition to industry-funded trials, more than 25,000 patients participate in the National Cancer Institute’s clinical trials annually. Following a careful review by the Institute of Medicine, the NCI currently is working hard to improve the speed and efficiency of the design, launch and conduct of clinical trials, hoping to incorporate innovative science and trial design into cancer trials, improve prioritization, selection, support and completion of clinical trials, and incentivize the participation of patients and physicians. Given an annual clinical trial investment of more than $800 million, these goals will be important for NCI to achieve.

Beyond cancer, the goals of the National Institutes of Health Clinical and Translational Science Awards program are to speed the translation of laboratory discoveries into treatments for patients, to engage communities in clinical research efforts, and to train a new generation of clinical and translational researchers. This consortium includes 55 medical research institutions located throughout the U.S. The CTSA consortium presently is funded by the NIH National Center for Research Resources.

Recently, NIH Director Francis Collins proposed the creation of a new National Center for Advancing Translational Sciences that would oversee the CTSA program. According to available information, “NCATS is not intended to be a drug company. It is a facilitator of translational research across the NIH and complementary to translational research already being conducted and supported on a large scale in the individual NIH Institutes and Centers. NCATS will seek ways to leverage science to bring new ideas and materials to the attention of industry by demonstrating their value.” In principle, it makes sense to house the CTSA program in an organizational unit that will do everything possible to facilitate drug discovery.

The creation of NCATS also has raised concerns, however, in part because it will disband the National Center for Research Resources that oversees critical, long-term technology development. NCRR supports the development of new technologies, including instrumentation, software and methods for biomedical research through a constellation of programs including Biomedical Technology Research Centers. NCRR also supports Shared Instrumentation and High-End Instrumentation Grant Programs. Currently under discussion is where to house these programs; the American Society for Biochemistry and Molecular Biology wants to be sure that they continue to be well nurtured, as they support critical, cutting-edge mass spectrometry, synchrotron X-ray technologies, molecular dynamics computation, optical and laser technology, and fluorescence spectroscopy.

All of us want cures for a long list of debilitating illnesses, and we hope that NCATS will focus on what NIH does best. Basic research is essential for disease target identification, such as BRAF in melanoma. When patients relapse, basic science also will be required to explain the molecular basis for therapeutic resistance and drug-target bypass. At the same time, the pharmaceutical industry has invested billions of dollars in drug screening and medicinal chemistry, and they are experts in drug design. It makes obvious sense to try to leverage all the expertise that industry can provide. In cancer, NCATS can encourage innovation in clinical trials and promote both industrial and industry-academic collaborations. Combination therapies are going to be essential given the recalcitrance of tumors to targeted intervention, and collaboration will be important in this regard. The U.S. Food and Drug Administration also will be an important partner as combination regimens are evaluated differently, and revisiting those guidelines may benefit all of us.

ASBMB President Suzanne Pfeffer ( is a biochemistry professor at the Stanford University School of Medicine.


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