NIH partners with Eli Lilly, others on rare diseases

In the ongoing hunt to uncover new therapies for rare diseases, the NIH has selected four new preclinical drug development studies to support, the findings of which will be shared with other researchers.

The projects will be funded through the Therapeutics for Rare and Neglected Diseases (TRND) program under NIH's National Center for Advancing Translational Sciences. NIH classifies a rare disease as one that affects fewer than 200,000 Americans. There are more than 6,000 rare diseases, but effective pharmacologic treatments exist for only about 200 of these illnesses, according to NIH.

For the first time, the TRND program will collaborate with a Big Pharma company. NIH selected Eli Lilly ($LLY) as a collaborator in developing a long-acting parathyroid hormone treatment for hypoparathyroidism, a rare condition in which the body does not produce enough of a certain type of hormone.

Another project will focus on developing a potential therapeutic that targets a cardiac disorder associated with LEOPARD syndrome, an extremely rare genetic disease that affects many areas of the body. The two other projects--one involving stem cells--will investigate therapeutic approaches to treat retinitis pigmentosa, a severe form of hereditary blindness.

The TRND program does not award grant money to selected researchers. Rather, participants have access to NIH scientists and the agency's resources to help advance their projects. Because of the collaborative nature of the program, an NIH spokesman told FierceBiotechResearch that the agency is not able to estimate how much the newly selected projects will cost to support.

In the last two years, TRND researchers and collaborators have moved four projects out of the lab and into human clinical trials, evaluating new therapies for sickle cell disease, chronic lymphocytic leukemia, hereditary inclusion body myopathy and Niemann-Pick Type C.

- read the press release
- view all the TRND projects here