DNDi Key Achievements in Neglected Disease Treatments
The Drugs for Neglected Diseases initiative (DNDi) has established a robust drug pipeline dedicated to combating neglected diseases. By focusing on both the improvement of existing medications and the creation of entirely new chemical entities, DNDi addresses critical gaps in global healthcare. Their work ensures that life-saving treatments are not only scientifically effective but also affordable and accessible to the populations that need them most.
Key Facts
- Diverse Pipeline: Developed treatments for malaria, sleeping sickness, visceral leishmaniasis, Chagas disease, HIV, and Hepatitis C.
- Accessibility: Focuses on non-patented, affordable options, such as ASAQ malaria treatment costing as little as $0.05 for children.
- Innovation: Introduced the first entirely oral treatment for sleeping sickness (Fexinidazole) and a pending single-dose oral treatment (Acoziborole).
- Paediatric Focus: Created specialized dosages for infants and children for Chagas disease and HIV.
- Global Collaboration: Partners with the WHO, pharmaceutical companies like Sanofi and Cipla, and various national health ministries.
Combatting Malaria with Fixed-Dose Combinations
DNDi has significantly improved malaria treatment through fixed-dose combinations—medications that combine two or more active ingredients into a single tablet to simplify dosing and improve efficacy.
ASAQ (Artesunate/Amodiaquine)
Launched in 2007 in partnership with Sanofi, ASAQ is a highly affordable treatment produced in Morocco. It follows a simple regimen of one or two tablets daily for three days. Registered in 32 African countries, India, Ecuador, and Colombia, it is included in the WHO Model List of Essential Medicines. To date, over 437 million treatments have been distributed.
ASMQ (Artesunate/Mefloquine)
Introduced in 2008 via the FACT Project Consortium, ASMQ offers a high compliance rate and a three-year shelf-life. Produced in Brazil by Farmanguinhos/Fiocruz and later by Cipla through South-South technology transfer, it was registered in multiple countries across Asia and Africa by 2015, with over one million treatments distributed.
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Advancements in Sleeping Sickness (HAT)
Human African Trypanosomiasis (HAT), commonly known as sleeping sickness, has seen a paradigm shift in treatment over the last two decades.
NECT (Nifurtimox-Eflornithine Combination Therapy)
Launched in 2009, NECT was the first improved treatment for stage 2 (advanced) HAT in 25 years. It is significantly safer than the previous arsenic-based melarsoprol, which had a 5% mortality rate. NECT is now used for 100% of stage 2 HAT patients across all 13 endemic countries.
Fexinidazole and Acoziborole
In 2018, DNDi and Sanofi introduced Fexinidazole, the first entirely oral treatment for Trypanosoma brucei gambiense, requiring a 10-day course of tablets. Looking forward, Acoziborole is expected by 2026 as the first single-dose oral treatment for both stages of the disease. This innovation allows for treatment at the village level, supporting the goal of disease elimination by 2030.
Treating Visceral Leishmaniasis (VL)
DNDi has worked to reduce the toxicity and duration of treatments for visceral leishmaniasis, a severe form of leishmaniasis.
Regional Strategies in Africa and Asia
In East Africa, the SSG&PM (sodium stibogluconate plus paromomycin) combination was introduced in 2010 as a cost-efficient, shorter-course first-line treatment. In Asia, DNDi promoted combinations of amphotericin B, miltefosine, and paromomycin. In India, the 2014 National Roadmap for Kala-Azar Elimination shifted the first-option treatment to single-dose amphotericin B, moving away from miltefosine monotherapy.
HIV Co-infection and Latin American Guidelines
For patients co-infected with HIV and VL, the WHO recommended a combination of liposomal amphotericin B and miltefosine in 2022. Similarly, in Latin America, the Pan American Health Organization (PAHO) updated its 2022 guidelines to recommend liposomal amphotericin B over pentavalent antimonials in Brazil due to lower toxicity.
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Specialized Paediatric and Co-infection Care
Treating children requires specific formulations to ensure safety, accuracy, and adherence.
Chagas Disease and Paediatric HIV
Launched in 2011, the paediatric dosage of benznidazole is the only treatment available for infants and children under two years old (up to 20 kg) infected congenitally. For paediatric HIV, DNDi and Cipla developed a 4-in-1 fixed-dose combination (lopinavir, ritonavir, lamivudine, and abacavir). This heat-stable, taste-masked granule capsule was approved by SAHPRA in June 2022.
TB/HIV Super-boosting
To solve a drug interaction where a tuberculosis (TB) medication reduces the effectiveness of the HIV drug ritonavir, DNDi sponsored a study in South Africa. The results led the WHO to strengthen recommendations for "super-boosting"—adding extra ritonavir to the regimen for co-infected children.
Other Global Health Initiatives
- GARDP: Launched in 2016 as a collaboration between the WHO and DNDi, the Global Antibiotic Research and Development Partnership is a not-for-profit entity developing new antibiotic treatments.
- Ravidasvir: In 2021, Malaysia granted conditional registration for ravidasvir, part of a combination treatment (ravidasvir + sofosbuvir) for Hepatitis C that showed a 97% cure rate in trials.
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Treatment Summary Table
| Treatment | Target Disease | Key Innovation | Year |
|---|---|---|---|
| ASAQ | Malaria | Affordable fixed-dose combination | 2007 |
| ASMQ | Malaria | High compliance, 3-year shelf-life | 2008 |
| NECT | Sleeping Sickness | Safer alternative to arsenic-based drugs | 2009 |
| SSG&PM | Visceral Leishmaniasis | Shorter-course, cost-efficient (East Africa) | 2010 |
| Benznidazole (Paediatric) | Chagas Disease | First age-adapted dosage for infants | 2011 |
| Fexinidazole | Sleeping Sickness | First entirely oral treatment | 2018 |
| Ravidasvir | Hepatitis C | High-efficacy DAA combination | 2021 |
| 4-in-1 Combination | Paediatric HIV | Heat-stable, taste-masked granules | 2022 |
| Acoziborole | Sleeping Sickness | Single-dose oral treatment | 2026 (Est.) |
Frequently Asked Questions
What is a fixed-dose combination?
A fixed-dose combination is a single medication that contains multiple active pharmaceutical ingredients. This approach simplifies the treatment regimen for the patient and often improves adherence and efficacy.
How has the treatment for sleeping sickness evolved?
Treatment has progressed from dangerous arsenic-based injections (melarsoprol) to the safer NECT combination, then to the first all-oral treatment (Fexinidazole), and finally toward a single-dose oral cure (Acoziborole).
Why is the 4-in-1 HIV treatment significant for children?
It replaces complex regimens with a single, heat-stable, and taste-masked granule capsule that can be sprinkled on food, making it easier to administer to infants and young children.
What is the role of GARDP?
The Global Antibiotic Research and Development Partnership (GARDP) is a not-for-profit organization launched by the WHO and DNDi to develop and deliver new or improved antibiotic treatments to address global public health needs.
How does "super-boosting" help children with TB and HIV?
Super-boosting involves adding extra ritonavir to a child's treatment to counteract the effect of certain TB drugs that would otherwise make the HIV medication less effective.