Drugs for Neglected Diseases Initiative (DNDi): matching needs and opportunities

Neglected diseases such as leishmaniasis, trypanosomiasis, Chagas disease and malaria have a devastating impact on the world's poor. These treatable, infectious tropical diseases have been progressively marginalized by those in charge of making research programme decisions, both in the public and private sectors. Unfortunately, people suffering from these diseases do not constitute a market lucrative enough to attract investment in research and development for new drugs. DNDi is addressing this lack of new, improved drugs for neglected patients with an alternative model of research and development. This model matches patients' needs with gaps identified in the drug development pipeline. Patients' needs: Currently available treatments for each of these diseases leave a great deal to be desired and do not address patients' needs. It is clear that patients in developing countries afflicted by neglected diseases urgently need new treatments that are safe, efficient, short course, easily administered, reasonably priced and accessible. Gaps in the pipeline: At the end of its first year, the Drugs for Neglected Diseases Initiative (DNDi) has a balanced portfolio of nine long, medium, and short-term projects that fill identified gaps in the drug development pipeline for each disease: at the early discovery stage, at the stage before drugs enter clinical development, and at the point where drugs should reach patients but do not. The projects in DNDi's portfolio match needs and opportunities across the drug development pipeline. Four are long-term projects to identify new lead compounds that can kill trypanosomiasis and/or leishmaniasis-causing parasites, and one focuses on combining existing anti-leishmanial drugs. The remaining four are short-term projects working with existing drugs at end of the pipeline: for instance, the registration of paromomycin, an old antibiotic, for visceral leishmaniasis in Africa (in collaboration with Institute of One World Health and WHO/TDR); the evaluation of nifurtimox, a drug used for Chagas disease, in combination with eflornithine to treat second stage sleeping sickness (in collaboration with WHO/TDR and Bayer); and two fixed-dose artesunate combination therapies (FACT) of artesunate/amodiaquine and artesunate/ mefloquine against chloroquine-resistant malaria in Africa and Asia respectively (in collaboration with seven medical research institutes across the world). DNDi continues to build its portfolio by proactively identifying projects and sending out calls for letters of interest to the scientific community via advertisements in scientific journals and on relevant websites. The second call for letters is currently being evaluated and it is hoped that 4-6 new projects will be added to the portfolio by the end of the year. DNDi will lead successful projects through the R&D process to achieve its final objective of giving neglected patients access to new therapies.