Championing research into neglected diseases

Worldwide, NTDs affected more than 2 billion people in 2013, according to the Global Burden of Disease study - an initiative run by the University of Washington's Institute for Health Metrics and Evaluation, which tracks the human impact of more than 350 diseases and injuries (J. R. Herricks et al. Several major funding organizations have made NTDs a priority, including the Bill & Melinda Gates Foundation, the Wellcome Trust and the Drugs for Neglected Diseases Initiative (DNDi), a Geneva-based non-profit organization. The full extent of the disruption is not yet known, but inJune, for example, the United Kingdom announced US$5.6 billion in budget cuts that would essentially end the country's Accelerating the Sustainable Control and Elimination of Neglected Tropical Diseases (ASCEND) programme, which provided drugs, clinical training and medical care to tackle five NTDs with high incidence in Africa and south Asia. Matlashewski says that many funders wanted to see a track record of on-the-ground clinical research in NTD-endemic countries before offering support for his fieldwork on the leishmaniasis parasite.

Fix the gene, cure the disease

At least ten trials of gene therapy are now under way, and early data from dozens of patients indicate the potential for long-term disease control and greatly improved quality of life. Sickle-cell disease presents a near-ideal opportunity to tap the power of gene therapy because the disorder typically arises from a mutation in a single nucleotide in one gene. The mutated protein misfolds and assembles into fibrous aggregates, resulting in deformed red blood cells that cause painful and damaging obstructions in blood vessels. Mark Walters, a haematologist and oncologist at the University of California, San Francisco, who was involved in running the trial, reports that treated participants are producing functional haemoglobin and seeing clear clinical benefits, such as not having to visit the hospital for pain relief.