NCATS Announces New Research Projects to Improve Treatments for Rare and Neglected Diseases

NCATS recently began work on three new preclinical drug development projects aimed at finding treatments for rare blood disorders and infectious diseases. The research, supported through the Center's Therapeutics for Rare and Neglected Diseases (TRND) program, also is designed to provide insights that will broadly improve and accelerate the translational science process.

TRND researchers will work on projects to develop treatments for malaria and Lassa fever, two infectious diseases that affect hundreds of thousands of people each year around the world, predominantly in developing countries. Malaria is a parasitic disease that spreads through the bite of an infected mosquito, and Lassa fever is a viral hemorrhagic virus, one of a group of viruses that also includes Ebola.

The third project is designed to develop a drug candidate with the potential to treat two blood disorders: beta-thalassemia and sickle cell disease. Both are caused by defects in hemoglobin, the protein in red blood cells that carries oxygen throughout the body.

Often, there is a perceived high risk of developing treatments for rare or neglected diseases due to anticipated lower returns on investment. TRND researchers seek to advance preclinical drug development projects to first-in-human trials, an approach known as "de-risking." This strategy aims to make candidate drugs more commercially viable and thus attractive to outside partners who can invest in their further development. For example, a sickle cell drug called Aes-103 that advanced to clinical testing with TRND program resources was acquired in July 2014 by Baxter International's BioScience business for further clinical development.  

"The success of Aes-103 is a validation of the NCATS model, which fosters a novel collaborative approach designed to de-risk therapeutic development programs and attract private-sector investment," said NCATS Director Christopher P. Austin, M.D. "We look forward to applying this model to the new TRND projects in hopes of advancing additional treatment candidates for patients."

TRND researchers also have advanced three other projects to human clinical trials. These included studies to evaluate treatments for chronic lymphocytic leukemia, hereditary inclusion body myopathy and Niemann-Pick disease type C. These projects have reached the stage at which partners, such as pharmaceutical, biotechnology or disease advocacy groups, are being sought to move the treatments into the next phases of clinical development.

"With so much recent success, TRND researchers are highly motivated to tackle these new projects," said John C. McKew, Ph.D., director of the TRND program. "With millions of patients in need of better treatments, our team is excited to continue this important work."

A rare disease is one that affects fewer than 200,000 Americans. It is estimated that there are more than 6,000 rare diseases. However, effective pharmacologic treatments exist for only about 200 of these illnesses. Neglected diseases are conditions that inflict severe health burdens on low-income populations. Many of these conditions are infectious diseases that are most prevalent in tropical climates, particularly in areas with unsafe drinking water, poor sanitation, substandard housing, and little or no access to health care.

TRND collaborations offer an opportunity to partner with TRND researchers and gain access to rare and neglected disease drug development capabilities, expertise and clinical/regulatory resources in a collaborative environment with the goal of moving promising therapeutics into human clinical trials.

TRND projects are adopted through a formal solicitation process. To learn more about TRND, its projects and clinical research studies, and how to apply, visit the TRND page.


Posted October 2014