When I
was a field doctor working in Central America, Southeast Asia, and Africa in
the 1980s, some of the most difficult patients to treat were those infected
with sleeping
sickness, also known as human African trypanosomiasis. In its late stage,
this neglected disease affects the brain, causing severe neurological
disturbances. If left untreated, sleeping sickness is fatal.
At the
time, and even up to just ten years ago, the only treatment option for
late-stage sleeping sickness was melarsoprol, an arsenic-based drug that is
toxic and painful to administer—sometimes referred to as “fire in the
veins”—killing 1 in 20 patients.
You do
not feel very proud to be a doctor when the only option for treating your
patients is a drug that may kill them. I and other clinicians in the field
found this unacceptable. Our frustration with the lack of safe, effective drugs
for neglected diseases like sleeping sickness led to the formation of the Drugs for Neglected Diseases initiative (DNDi).
Two
years ago, the first new treatment for sleeping sickness in 25 years,
nifurtimox-eflornithine combination therapy (NECT),
became available. It was the culmination of years of dedicated field research
by DNDi, Médecins Sans
Frontières/Doctors Without Borders (MSF), Epicentre, the Swiss
Tropical and Public Health Institute (Swiss TPH), and the Ministries of Health
of the Democratic Republic of Congo (DRC) and the Republic of Congo (RoC).
NECT is
a significant therapeutic advance and has helped do away with the use of
melarsoprol. However, it is not ideal: it still requires intravenous infusions,
close monitoring, and hospitalization, all of which are major challenges in the
resource-poor settings and conflict areas in which sleeping sickness is
prevalent.
DNDi is
therefore now working on researching and developing an oral drug option for
sleeping sickness, which could eliminate intravenous infusions,
hospitalization, and painful diagnostic procedures (lumbar punctures) to
determine disease stage. Two compounds are currently in our pipeline: fexinidazole,
in partnership with Sanofi and Swiss TPH; and the oxaborole compound SCYX-7158, in partnership with Anacor
Pharmaceuticals, SCYNEXIS, and Pace University, among others. These
product-development partnerships have helped us create a robust drug-candidate
pipeline for sleeping sickness, potentially bringing new hope to patients and
clinicians.
DNDi
was established in 2003 by MSF, the Indian Council for Medical Research, Brazil`s
Oswaldo Cruz Foundation, the Kenya Medical Research Institute, the Ministry of
Health of Malaysia, and the Pasteur Institute in France, with the UNICEF/UNDP/World
Bank/World Health Organization`s Special Programme for Research and Training in
Tropical Diseases (WHO TDR) as a permanent observer. We are dedicated to
developing new, field-adapted treatments for patients suffering from neglected
tropical diseases, including those with the highest case-fatality rates:
sleeping sickness, Chagas disease, and kala azar (visceral leishmaniasis).
Malaria was also an early focus of DNDi, and new R&D programs for pediatric
HIV and specific helminth infections were added this year.
Our
goal is to deliver 6 to 8 new treatments by 2014—and we`re halfway there, with
4 treatments delivered since 2003: NECT, two fixed-dose antimalarials
(artesunate-amodiaquine [ASAQ] and artesunate-mefloquine [ASMQ]), and a
combination treatment for kala azar in Africa (sodium stibogluconate and
paromomycin [SSG&PM]).
DNDi
exists because of the alarming lack of R&D for the most neglected diseases
in the world, which affect the poorest people but do not represent lucrative
“market potential” for the pharmaceutical industry. Our objective at DNDi is to
address this terrible gap between neglected patient needs and R&D and to
bridge the innovation-to-access divide.
But we
cannot—and do not—do this alone. In fact, we can only accomplish our work
through innovative and collaborative South-South and North-South partnerships.
Our involvement with developing a new treatment doesn`t end with drug approval
or registration; we also take on the responsibility of ensuring access for the
patients who need these new drugs the most. This is done by making sure the
new treatments are affordable and have a robust manufacturing, distribution,
and implementation network behind them, through partnerships with
pharmaceutical and biotech companies, international bodies such as WHO, nongovernmental
organizations like MSF, and governments.
Moreover,
while our primary focus is on delivering new treatments for neglected patients,
a further objective is to bolster local research and health-care capacity in
endemic countries. This is accomplished through our regional disease-specific
R&D platforms, including the Leishmaniasis
East Africa Platform (LEAP), HAT
Platform for sleeping sickness, and Chagas
Clinical Research Platform in Latin America. Such capacity strengthening
includes the building and renovation of hospital wards, clinics, and health
posts; renovation and equipping of clinical laboratories; and training of
health-service personnel in clinical trial methodology, Good Clinical Practice
and Ethics, patient treatment and evaluation, accurate diagnosis and follow-up
by parasitology, and safety.
And
finally, we cannot accomplish our goals without also working to ensure that
sound policies are in place to enable greater patient needs-driven R&D.
This includes appropriate incentives and financing mechanisms, innovative
regulatory pathways that will expedite access, and open innovation approaches
that ensure the widest possible sharing of research knowledge and data.
Establishing
partnerships and enabling policies are key elements of DNDi`s start-to-finish
approach—from drug discovery to treatment implementation—to tackle the urgent
health needs of the most neglected patients.
