Finding Better Treatments for Neglected Diseases
08 January 2020

The World Health Organization goal of eliminating cases of sleeping sickness by 2030 seems to be within reach, but we’ve been here before.

Proven new treatments for the deadly disease are needed, and the Drugs for Neglected Diseases initiative, with support from the Stavros Niarchos Foundation (SNF) and others, is leading an international network striving to find and implement them.

------------------------------------------------------------------------------------------------------------------------------------------

It begins with fever, body aches, severe headache, and fatigue. As it progresses, serious neuropsychological effects begin to take hold, leading to personality changes that include confusion, aggressive behavior, disruption of sleep patterns, and psychosis. Left untreated, it leads to death.

The Drugs for Neglected Diseases initiative (DNDi), a nonprofit drug research and development organization, estimates that approximately 8.5 million people across 36 countries in Africa face medium to high risk of infection with sleeping sickness. In 2012, the World Health Organization (WHO) set a goal of sustainably eliminating cases of sleeping sickness, also known as human African trypanosomiasis, by 2030, and this goal seems to be within reach.

But we’ve been here before. By the mid-1960s, prevalence of the disease had fallen from many tens of thousands of cases each year to fewer than five thousand through systematic treatment efforts. As prevalence ebbed, resources for active management withdrew, and in the 1990s, a combination of regional trends set the stage for a powerful resurgence of the disease.

 

Graph

To achieve the WHO goal of eliminating sleeping sickness altogether, new treatments were required. This is where DNDi stepped in.

“Less than ten years ago we were still treating this disease with an arsenic derivative that killed 5% of all patients,” said Dr. Viktor Kande.

The treatment Dr. Kande mentions could also cause burns on skin so severe they could lead to amputation, and had such a fearsome reputation that patients would avoid treatment after diagnosis. The other available treatment was prohibitively complex, necessitating 56 separate intravenous transfusions over the course of a two-week hospitalization.

The lack of safe, effective treatments for sleeping sickness prompted Médecins Sans Frontières (MSF) to convene the Drugs for Neglected Diseases working group in 1999, and MSF earmarked the money that came with its Nobel Prize win that year for combatting neglected diseases. Out of that working group developed the Drugs for Neglected Diseases initiative, formally established in 2003 by seven organizations from around the world, including the Kenya Medical Research Institute, the Malaysian Ministry of Health, and the Indian Council for Medical Research.

In 2009, a new treatment developed by a collaborative including DNDi offered medical professionals a significantly better option. But this new treatment, though an important improvement in efficacy and treatment experience, remained far from ideal, requiring patients to undergo lumbar puncture and hospitalization and entailing difficulties in transportation. Were the disease to be—and remain—fully vanquished, further advances were required.

Leading a research and development effort in collaboration with a range of partners around the world, DNDi introduced fexinidazole, a ten-day exclusively oral treatment not necessitating extended hospitalization. Dr. Kande, who was principal investigator on a clinical trial for fexinidazole, says that in the new drug “now we have a treatment that is safe, effective, and simple.” Fexinidazole has been added to the WHO Model List of Essential Medicines and recommended by the European Medicines Agency.

Sleeping sickness is classed by the WHO among neglected tropical diseases, which collectively affect over a billion people a year and sap billions of dollars from developing economies. One element of the “neglect” these diseases face is of traditional market incentives to find, prove, and distribute effective treatments. This is where DNDi comes in, driven instead by patient needs—including, when those patients are young children, strawberry flavored H.I.V. medication.

DNDi continues to push forward on research into a one-dose oral drug, acoziborole, that could work in tandem with fexinidazole to extinguish sleeping sickness. A 2018 grant from the Stavros Niarchos Foundation (SNF) supports DNDi in continuing to pursue its sleeping sickness program.

Results from the final phases of a study on acoziborole in the Democratic Republic of Congo and Guinea are expected by the end of this year. 

Learn more:
• Read DNDi’s excellent visual story on the sleeping sickness project, “A doctor’s dream
• Learn more about the context and history of the project in DNDi’s sleeping sickness disease brief
• Find in-depth information on the disease from the WHO
• Learn about other neglected tropical diseases from the WHO
• Read the article from which data in the chart above is sourced