For those familiar with the field of neglected diseases, it goes by the name of the 10/90 gap: only 10 per cent of global health research was devoted to conditions that accounted for 90 per cent of the global disease burden. It was to counteract this imbalance that the Drugs for Neglected Diseases initiative (DNDi) came into being in 2003. Coinciding with its tenth anniversary year, the organization has been granted the BBVA Foundation Frontiers of Knowledge Award in the Development Cooperation category. Its Executive Director, Bernard Pécoul, announced this morning at a press conference that new drugs DNDi is working on “could dramatically change the management of some of these neglected diseases.”
The organization and its partners are studying new, easier-to-administer oral drugs against diseases such as sleeping sickness, Chagas disease and leishmaniasis, which both cause and result from poverty. DNDi has already delivered improved treatments against these diseases by combining or reformulating existing medicines, and now has new chemical compounds at different stages of the testing process.
Also under development is a new formulation of antiretroviral treatment for pediatric HIV. Pécoul remarked at the conference that 350,000 children each year need antiretroviral treatment and that of this number 90% are born in Africa. Although HIV is a widely studied disease, “not enough is being done specifically for infants and toddlers in developing countries.” Today’s treatments are neither child friendly – they taste bad and contain alcohol – nor suitable for patients in certain areas – they need refrigeration. Two years ago, DNDi started a project to combine four drugs in one; a pleasant-tasting formulation that doesn’t require the cold chain and which, according to Pécoul, “will hopefully reach the market in a couple of years.”
Pécoul will receive the award this Thursday, on June 20th, at a formal ceremony in the BBVA Foundation’s Madrid headquarters.
DNDi is a not-for-profit research and development organization working to deliver new treatments for what are commonly known as neglected diseases, a set of 17 neglected tropical diseases and other conditions that affect hundreds of millions of people who are collectively too poor to offer a lucrative market to the pharmaceutical industry. It focuses particularly on sleeping sickness or human African trypanosomiasis, Chagas disease, leishmaniasis, filarial infections, malaria, and pediatric HIV.
DNDi was set up on the combined initiative of seven public and private institutions: Médecins Sans Frontières/Doctors Without Borders, the Indian Council for Medical Research, the Kenya Medical Research Institute, the Malaysian Ministry of Health, France’s Institut Pasteur, Brazil’s Oswaldo Cruz Foundation (Fiocruz), and the Special Program for Research and Training in Tropical Diseases of the World Health Organization.
Its strategy is to coordinate and synergize the efforts of public and private partners – including pharmaceutical companies – in the development, production and distribution of new treatments. DNDi has offices in Switzerland, Brazil, the Democratic Republic of the Congo, Kenya, India, Malaysia, the United States and Japan, and coordinates a partnership network that liaises with the public and private institutions involved in each project.
“DNDi’s innovative collaborative model,” Pécoul remarks, “has secured the increasing engagement of public and private actors, as a result of growing awareness of these urgent and neglected public health issues.”
Among the organization’s partners are a number of Spanish institutions including Médecins Sans Frontières Spain, the Barcelona Centre for International Health Research (CRESIB), Carlos III University of Madrid, and GSK Tres Cantos. DNDi has also received financial support from the Spanish development cooperation agency AECID.
Six approved treatments
Pécoul offers figures to illustrate just what is meant by neglected diseases: between 1975 and 1999 only 16 of the total 1,393 new drugs developed by the pharmaceutical industry were specifically developed for neglected tropical diseases plus malaria or tuberculosis. In the last decade “progress has been made,” but, he is quick to point out, “the fatal imbalance persists.” Only 3.8% of the products, excluding vaccines, approved between 2000 and 2011 were targeted for neglected diseases, as was the case for only 1.4% of all clinical trials conducted in the same period.
After ten years of research and development, DNDi has delivered six treatments: two antimalarials, a combination therapy for late-stage sleeping sickness, a combination therapy for visceral leishmaniasis in Africa, a set of combination therapies for visceral leishmaniasis in Asia, and a pediatric dosage form of benznidazole for Chagas disease.
Also, the organization has helped establish three clinical research platforms in Africa and the Americas: for leishmaniasis in Kenya, Ethiopia, Sudan, and Uganda; for sleeping sickness in the Democratic Republic of the Congo and other endemic countries in the region; and for Chagas disease in Latin America. “Strong regional networks such as these,” Pécoul affirms, “help strengthen research and treatment implementation capacity in neglected disease-endemic countries.”
NECT, the new sleeping sickness treatment developed by DNDi and its partners, is the first new product in 25 years for the late stage of the disease. It is far safer than what had previously been standard treatment, melarsoprol, an arsenic derivative so toxic that it kills 5% of those it is supposed to cure. The new treatment is already available in the twelve African countries which account for 99% of reported sleeping sickness cases.
DNDi is also working with partners on two new chemical entities for this disease: oxaborole SCYX-7158 and fexinidazole. The first is an oral compound which is currently concluding Phase I clinical studies, while fexinidazole, also an oral treatment, began Phase II/III studies in the Democratic Republic of the Congo and Central African Republic in October 2012. “A safe, effective, oral-only treatment would simplify the way the disease is managed,” Pécoul explains, “as well as supporting the WHO’s target to eliminate sleeping sickness by 2020.”
Leishmaniasis and Chagas
DNDi is also about to start Phase II studies – testing efficacy and safety – on the use of fexinidazole in visceral leishmaniasis patients in East Africa.Ten years ago, the standard treatment for visceral leishmaniasis was a month-long course of painful daily injections, a treatment schedule that is difficult to manage in poorly equipped health centers in the remote areas where the disease is found. The combined therapy delivered by DNDi has already shortened the treatment duration to 17 days, but the goal remains to come up with an oral treatment.
In the case of Chagas disease, a decade ago there were no pediatric formulations. DNDi’s dosage form for children up to the age of two is oral, inexpensive, and easy-to-use. It is also designed for home administration: an important pro-compliance factor in what is a lengthy two-month treatment.
Another oral drug, E1224, is currently being tested on adult patients in Bolivia. “If successful,” Pécoul says, “this drug could be the oral, easy-to-use, affordable and safe treatment option that Chagas patients need and deserve.”
These ongoing projects are joined by an early-stage pipeline of twelve entirely new chemical entities.
“Receiving this prestigious award by the BBVA Foundation is a great honor for us and our partners and recognition of our collective achievements of the past 10 years,” says Pécoul. “It is also a strong encouragement to pursue our mission and to continue to develop and deliver new and affordable treatments to neglected patients in dire need.”
Violaine Dallenbach: firstname.lastname@example.org +41 22 906 92 47 / +41 79 424 14 74 (mobile)
About Drugs for Neglected Diseases initiative (DNDi)
DNDi is a not-for-profit research and development (R&D) organization working to deliver new treatments for the most neglected diseases, in particular sleeping sickness (human African trypanosomiasis), Chagas disease, leishmaniasis, filarial (parasitic worm infections), and paediatric HIV.
Since its inception in 2003, DNDi has delivered six treatments: two fixed-dose antimalarials (ASAQ and ASMQ), nifurtimox-eflornithine combination therapy (NECT) for late-stage sleeping sickness, sodium stibogluconate and paromomycin (SSG&PM) combination therapy for visceral leishmaniasis in Africa, a set of combination therapies for visceral leishmaniasis in Asia, and a paediatric dosage form of benznidazole for Chagas disease in Latin America.
DNDi was established in 2003 by MSF, the Indian Council of Medical Research, Brazil’s Oswaldo Cruz Foundation, the Kenya Medical Research Institute, the Ministry of Health of Malaysia, and the Institut Pasteur in France, with the Special Programme for Research and Training in Tropical Diseases (WHO-TDR) as a permanent observer.
About ‘Connect to Fight Neglect’
As part of its 10th anniversary, DNDi has launched a special advocacy website to give voice and attention to neglected patients and those working to develop and deliver life-saving treatments for them. The ‘Connect to Fight Neglect’ website is a multimedia web portal where videos, photos, audio, testimonials, stories, and opinions can be shared about some of the world’s most neglected diseases, the people whose lives are diminished and threatened by these conditions, and the research and care efforts under way around the world. Among the people featured are patients, doctors, researchers, public health officials, policymakers, funders, and activists.