Dr Graeme Bilbe, Research & Development Director, DNDi
DNDi and other not-for-profit drug research and development (R&D) organizations have explored new R&D pathways to develop and deliver safe, adapted, and affordable treatments for neglected diseases. Most of these new pathways have shown signs of success, while several have faced challenges that span across the drug discovery and development processes.
Leishmaniasis is transmitted by the bite of a sand fly. It is a complex disease with over a million new cases occurring every year and 350 million people living at risk worldwide in 98 countries. The disease presents several different forms, the most common of which are visceral leishmaniasis (VL), which is fatal without treatment, and cutaneous leishmaniasis (CL).
Chagas disease is transmitted by the ‘kissing bug’ and also by blood transfusion, organ transplantation and congenital transmission. The disease is endemic in 21 countries in Latin America, where 100 million people are at risk.
Filarial diseases include onchocerciasis (also known as River Blindness), lymphatic filariasis (also known as elephantiasis), and loiasis. They are caused by parasitic worms which are transmitted by insect vectors to humans, mainly in Africa and Southeast Asia. About 168 million people are infected by the three diseases.
Human African trypanosomiasis (HAT), also known as ‘sleeping sickness’, is transmitted by the tsetse fly. While currently its prevalence is declining, HAT is still a threat to millions of people across Sub-Saharan Africa with 83% (2013) of all cases in the Democratic Republic of Congo (DRC).
Malaria is transmitted by mosquitoes. It kills one child every 30 seconds in sub-Saharan Africa and is the leading parasitic cause of morbidity and mortality worldwide. 3.2 billion people are at risk. DNDi with its partners have delivered two fixed-dose combination (FDC) of the five artemisinin-based drugs (ACTs), FDC’s that were recommended by WHO for the treatment of uncomplicated falciparum malaria.
HIV is transmitted to children during pregnancy, delivery and through breastfeeding. An estimated 3.3 million children below the age of 15 were living with HIV in 2012, more than 90% of whom were in Sub-Saharan Africa.
The GHIT Fund announced in June 2014 a funding award opportunity for the development of new medicines, vaccines, or diagnostics for neglected tropical diseases (NTDs) that are prevalent in the developing world.
The Drugs for Neglected Diseases initiative (DNDi) sends its sincerest condolences to the families, friends, and loved ones of those on board Malaysia Airlines Flight MH17.
Dr. Wilfried Mutombo, Coordinating investigator, Fexinidazole study for HAT
At the time DNDi and its partners are extending the clinical trial on fexinidazole, the first new oral treatment tested for sleeping sickness, to the early stages of the disease in adults and to children between 6 and 14 years of age, it is important to remember how clinical trials in remote areas of a country such as the Democratic Republic of the Congo (DRC) can be a daunting challenge, but can also bring lasting benefits to local communities and researchers, and to the health system overall.
Filaria is a group of neglected tropical diseases infecting over 150 million people in sub-Saharan Africa, Asia, and Latin America. The two main filarial diseases, onchocerciasis (river blindness) and lymphatic filariasis (elephantiasis), devastate the lives of patients, causing debilitating symptoms and social discrimination. In March 2014, DNDi traveled to rural Ghana to meet and interview patients with filaria and the physician-researchers treating them.
New York – June 14, 2014 – The Drugs for Neglected Diseases initiative (DNDi) wishes to extend its deepest condolences to the Rockefeller Family for the tragic loss of Dr. Richard Rockefeller, who was killed in a plane crash the morning of June 13.
Robert Don, Discovery & Preclinical Director, and Charles Mowbray, Head of Drug Discovery, DNDi
Important advances have been made in recent years to optimize the use of existing medicines to treat neglected tropical diseases (NTDs) but, while providing urgently needed improvements on previous treatments, they are often still not ideal. There remains an urgent need to design and develop modern drugs to treat these diseases.
A new paper recently commissioned by DNDi to examine the possibilities of creating a pooled international R&D fund for the Demonstration Projects that were selected as part of the process following on the report of the WHO Consultative Expert Working Group on Research and Development (CEWG): “Demonstration Financing: Considerations for a Pilot Pooled International Fund for R&D.”
On 7 April 2014, the Director-General: Health, Ms P Matsoso and the Executive Director: DNDi, Dr Bernard Pécoul, signed a Partnership Agreement on Improving Access to Paediatric HIV Treatment in South Africa. DNDi’s paediatric HIV project, in partnership with Cipla Ltd., and with the support of the UNITAID, in addition to the French Development Agency (AFD), Médecins Sans Frontières/Doctors Without Borders (MSF), and the UBS Optimus Foundation, is to develop two 4-in-1 lopinavir-based fixed dose combinations as well as a solid formulation of ritonavir booster that are suitable for young children infected with HIV, as well as with tuberculosis and HIV. These formulations, ultimately, will be in solid ‘granular’ form that is palatable and requires no refrigeration, alleviating much of the current treatment burden on the children, their mothers and caregivers, and healthcare workers.
Silvia Gold, President, Mundo Sano Foundation
American Trypanosomiasis, more commonly known as Chagas disease, no longer reflects the geographical scope of the disease. Today, Chagas disease affects people around the world. It is hard to imagine that a disease, discovered over a century ago, which today can be easily diagnosed and for which effective treatment exists, continues to be a problem of severe health impact.
Bernard Pécoul, Executive Director, DNDi
The field of neglected disease R&D today is experiencing what could be characterized as a phase of shifting sands: after long inaction for decades, we are now experiencing both remarkable advances and rude set-backs. The innovative ideas, incentives, and R&D partnerships, such as DNDi, that emerged over the last decade are right at the nexus of these movements – of governments, industry, philanthropy, and civil society, among others – and they thus impact directly on our work. They provide a constant reality check that reminds us of just how fragile the field of not-for-profit drug development for neglected diseases is, be it in times of advances or in times of set-backs. We have to take this seriously into account and feed our reflections, debates, and efforts to secure the sustainability of the environment in which we work to solve, in the long term, the problems of millions of patients.
Jean-François Alesandrini, Fundraising & Advocacy Director
WHO Member States and experts, after weeks of consultations, are in the process of selecting ‘demonstration projects’ to address the unmet medical needs of developing countries. The overall objective of this process is to evaluate and assess new innovative mechanisms to coordinate and fund public health research and development (R&D).
One of the most important achievements of DNDi Latin America is the beginning of the Lead Optimization Latin America project (LOLA), with a focus on the first steps of Research & Development (R&D) for new medicines: the discovery of new compounds. This is a crucial step in the expansion of R&D projects in the region.
Eric Stobbaerts, Head of DNDi Latin America
As DNDi marks its 10 years of existence, the time is ripe to assess the achievements and difficulties experienced in order to move into a new decade with new approaches. For Latin America, in particular, this period is unique, even transformative, as it is marked by unprecedented economic and social strengthening of the region. This growth brings with it the crucial role of Latin America’s public and private sectors in contributing meaningfully to the neglected disease research and development landscape. The activities of DNDi Latin America are focused on contributing to this process and have expanded with increasing opportunities to partner with ministries of health and national control programmes, but also other non-governmental and private-sector actors, to address the needs of patients suffering from neglected tropical diseases endemic in the region.