Aim is to develop therapies to the point that they are sufficiently de-risked for others to advance.

Congress is providing $24 million a year for the next five years to start the Therapeutics for Rare and Neglected Diseases program (TRND) at the NIH. This marks a departure from NIH’s focus of backing basic research, as this initiative will support drug discovery, preclinical R&D, as well as seeking partners for further development and eventual commercialization.

The NIH Office of Rare Diseases Research (ORDR) will oversee the program, and TRND’s laboratory operations will be administered by the NHGRI. Additionally the Chemical Genomics Center (NCGC) will be a principal collaborator in TRND, conducting much of the preclinical activities.

A rare disease is one that affects fewer than 200,000 Americans. NIH estimates that more than 6,800 rare diseases afflict more than 25 million Americans. Effective pharmacologic treatments exist for only about 200 of these illnesses. Unlike rare diseases, neglected diseases may be quite common in some parts of the world, especially in developing countries. Private companies seldom pursue new therapies for these types of illnesses because of high costs and failure rates and the low likelihood of recovering investments or making a profit.

“While Congress has previously taken important steps to help these patients, such as providing incentives for drug companies under the Orphan Drug Act, this is the first time NIH is providing support for specific preclinical research and product development,” notes ORDR director, Stephen C. Groft, Pharm.D.

NHGRI acting director, Alan E. Guttmacher, M.D., adds, “Biotechnology and pharmaceutical companies have enormous strength and experience in drug development but to maximize return-on-investment, work primarily on common illnesses. TRND will develop promising treatments for rare diseases to the point that they are sufficiently de-risked for pharmaceutical companies, disease-oriented foundations, or others to undertake the necessary clinical trials.”

TRND is currently setting up an oversight process to decide which projects to pursue. Leadership currently envisions a small number of diseases being studied each year, with strict criteria used to determine which molecules will be studied for which diseases. NIH expects to use existing intellectual property policies to transfer licenses for TRND-discovered drugs to private companies or others for development, clinical testing, and marketing.

TRND will work closely with disease-specific experts on selected projects, leveraging in-house scientific capabilities needed to carry out much of the preclinical development work and contracting out other parts. NCGC will kick-start preclinical activites using its robotic, high-throughput screening system, related assays, and a library of more than 350,000 compounds. TRND will also devote some of its efforts to improving the drug development process itself to make it faster and less expensive.

Beyond the preclinical stage, TRND will work to find a company willing to take the therapy into clinical development. Additionally, it will take advantage of NIH resources to launch human studies, including the NIH Clinical Center, the NIH Rapid Access to Interventional Development, and the Clinical and Translational Science Awards program.

Next articleFIT Biotech and ImmunoVaccine Ally to Enhance HIV Vaccine Candidate