In a world where science is constantly providing new drug targets and where miraculous new therapies are proclaimed almost daily in the media, very few of these advances have been for “orphan” or neglected diseases. One approach to solving this problem has been through the establishment of public/private partnerships. In this case the pharmaceutical companies are not expected to shoulder the full load of drug discovery and development.

Five years ago a consortium led by the University of North Carolina at Chapel Hill (UNC-CH) was brought together to discover and develop new drugs for the treatment of human African trypanosomiasis (HAT) and visceral leishmaniasis. The new Consortium for Parasitic Drug Development (CPDD), received initial funding from the Bill & Melinda Gates Foundation (BMGF), followed by two new recently awarded 5-year grants.

During the first five years the consortium discovered and developed DB289 (pafuramidine), the first orally active drug and the first new drug in the last 50 years for the treatment of early stage HAT. Pafuramidine is an orally active prodrug of the pentamidine analog, furamidine.  Under the guidance of the CPDD scientists the drug was moved from early drug discovery through preclinical and Phase I and II clinical trials to Phase III clinical trials (now under way). The efficacy trials have been performed under very difficult conditions in the Democratic Republic of the Congo, Angola and Sudan.

CPDD accomplishments include the discovery of potential new drugs that can penetrate the blood-brain barrier to treat late stage trypanosomiasis (CNS HAT) and new analogs effective against visceral leishmaniasis. The newly funded BMGF grants are designed to continue development of these new drug candidates.

CPDD’s partners include Georgia State University, Swiss Tropical Institute, London School of Hygiene and Tropical Medicine, Ohio State University, University of South Florida, University of Glasgow, Gorgas Memorial Institute (Panama), Kenya Agricultural Research Institute, and Immtech Pharmaceuticals Inc.

The consortium represents a new direction in academic-led research. The idea of translational research in academia is not new, but the implementation of all the aspects needed to successfully carry out the discovery and development of new therapeutic agents is still in its early and formative stages.