The purpose of the new, Congressionally mandated, NIH Therapeutics for Rare and Neglected Diseases (TRND) Program is to build on the chemical genomics efforts of the NIH Roadmap to develop new drugs for patients with rare and neglected diseases. TRND will focus not only on developing drugs for such diseases, but also on scientific paradigm and technology development to improve success rates in the preclinical stage of drug development generally. Specifically, TRND?s disease-specific goal will be to generate clinical candidate compounds and data packages on them that are sufficient for Investigational New Drug (IND) Applications to the FDA. TRND will thus focus on the most scientifically challenging stages of drug development, the stages that lead to meeting the requirements set out by the FDA for human testing of new drugs. TRND will leverage both public and private sector investments to bring new drugs to patients with rare and neglected diseases. The activities that TRND will accomplish include: 1) reiterative chemical modification of promising starting compounds. 2) testing of these compounds for properties required for safety and efficacy in cells, animals, and ultimately humans. 3) testing optimized compounds in pharmacological and toxicology models required by FDA. 4) performing IND-enabling testing in animal models of safety and efficacy. 5) cellular and animal testing of current FDA-approved drugs to identify new diseases that may be treated with them, leading to potentially rapid therapeutic advances. TRND will work in support of research for the National Cancer Institute. TRND will establish a new and innovative model that combines the complementary strengths of the NIH extramural, NIH intramural, including the National Cancer Institute, and biopharmaceutical sectors to bring new treatments to rare and neglected diseases.
