February 27, 2020: Resource for Systemizing Serendipity Turns 10
NCATS celebrated a very special anniversary recently. The NCATS Pharmaceutical Collection (NPC), a compilation of every drug approved for human use by major regulatory agencies worldwide, turned 10 years old. The NPC was developed to enable drug repurposing — using a drug approved for one indication to treat another — both to identify potential new treatments more rapidly and, on a broad scale, to better understand the workings of the human body by identifying all drugs that can affect a given physiological process. Repurposing has been a focus of NCATS since its founding, given its potential to identify treatments rapidly and thus make the therapeutic development process more efficient. The New Therapeutic Uses program provides another paradigm for repurposing, and Translator is being built to help researchers identify which drugs and drug candidates to test.
The NPC has become a remarkable engine of scientific and medical discovery, but the impetus for its creation did not come from a scientist. It came from a mother of a child with a rare disease. She approached me for help in finding a treatment for her son, whose life expectancy was less than five years. I gave her a tour of the robotic wizardry in the labs, explaining how it allowed us a jump-start on the approximately 15-year process of making a new drug. Instead of being carried away by the extraordinary science, she was perplexed. Her son did not have 15 years to wait for a drug, she said. The only alternative to creating a new drug was to find a currently approved one that worked for her son’s disease — i.e., repurposing. At that time, repurposing was a serendipitous affair, based on chance clinical observations by clinicians. We asked ourselves how we could systematize serendipity, testing every drug currently approved in a cellular assay of her son’s disease. That’s when we realized that no such comprehensive collection of drugs had ever been created. So, we decided to create one, and it became the NPC.
After five years of work, the information in the NPC was made public. In the ensuing 10 years, the NPC was utilized in more than 200 projects in such diverse areas as rare diseases, infectious diseases and cancer. Examining the patterns of response across these hundreds of projects has provided insights into fundamental biology and connections among diseases previously thought to be unrelated. Several projects produced drugs with new potential uses that have entered clinical trials. The NPC experience is summarized in a recent publication.
The NPC data are now a part of a suite of resources at the NCATS Inxight: Drugs — a web portal that aggregates reliable, curated drug development data from multiple existing sources in one place. All the data generated through this effort are deposited in PubChem.