A new molecule
Developing a new drug to treat sleeping sickness is necessary. The development of Pafuramidine maleate (DB289) was interrupted in February 2008. Preclinical studies of a new molecule called fexinidazole are now complete and a clinical trial phase I is ongoing.
Fexinidazole is a nitroimidazole which has shown its efficacy at preclinical stage. It is being investigated for possible future use as an oral drug in early and late stages of both forms of HAT. A new oral drug candidate, the benzoxaborole SCYX-7158 has shown promising results in pre-clinical studies at it is expected to advance soon to clinical development for possible use as in early and late stages.
The Drugs for Neglected Diseases initiative (DNDi) has received a USD 15 million grant from the Bill and Melinda Gates Foundation to undertake clinical development of oral fexinidazole to treat human African trypanosomiasis (HAT).
Fexinidazole – A New Oral Nitroimidazole Drug Candidate Entering Clinical Development for the Treatment of Sleeping Sickness.
SCYX-7158, an Orally-Active Benzoxaborole for the Treatment of Stage 2 Human African Trypanosomiasis
A new molecule to be discovered and developed still needed
The major issue for changing strategies and ensure sustainability of control continues to consist in developing a new drug which will not require any particular skills or care to administer while regimen should not last more than a few days making it manageable by peripheral health staff working in rural areas in an out-patient context.
Ideally such new drug should be effective in both form of the disease and in both stages making lumbar puncture unnecessary; stable at room temperature and having a reasonable volume for easy transport and storage it should thus be possible to integrate patient management in the existing health system.
Cost is also of concern as any new treatment should be affordable by national health systems and patients of endemic countries.
An added problem in the research of new molecules is the challenge of conducting and analizyng data of clinical trials for HAT. WHO organized an informal consultation to develop a consensus framework for clinical trial, which permits direct comparability of data.