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NIH's New Drug Pipeline for Neglected Diseases

The National Institutes of Health, a bastion of basic research, is making a foray into developing drugs. NIH leaders today announced a $120 million, 5-year plan to set up a drug development service center at the agency. The center's chemists and toxicologists will modify promising compounds until they're ready to be tested in people. The focus will be on rare and neglected diseases.

"This initiative really is new," said acting NIH Director Raynard Kington in a press conference today. NIH has "never tried to directly develop medications for rare and neglected diseases"—or done much drug development, period. The Therapeutics for Rare and Neglected Diseases (TRND) program builds on NIH's $100-million-a-year Molecular Libraries Program, a small-molecule screening program that some say is replicating work by drug companies. But NIH officials say they will target diseases that industry is ignoring. "It is not TRND's intention to compete with pharmaceutical and biotech companies," said Stephen Groft, director of the NIH Office of Rare Diseases Research.

The program will have space somewhere near NIH’s main campus in Bethesda, Maryland, and will be staffed with scientists recruited from industry. They will take potential drugs discovered at NIH and at universities and tweak their chemistry so the compounds work well in humans without causing toxic side effects—a slow, hard process. Then either NIH or a company will apply to the Food and Drug Administration to test the compounds in clinical trials. NIH could profit from licensing the drugs, but "that's not our goal," said Alan Guttmacher, acting director of the National Human Genome Research Institute. NHGRI will oversee the center, which will get its $24-million-a-year budget from each of NIH's 27 institutes and centers.

One challenge will be choosing the diseases. NIH says there are 6800 rare diseases and others that are considered neglected, mostly infectious diseases that plague the developing world. A panel of scientists and patient advocates will help pick diseases based on scientific opportunity, Groft said.