Neglected diseases continue to cause significant morbidity and mortality in the developing world. Yet, of the 850 new therapeutic products approved between 2000 and 2011, only 4% (and only 1% of all approved NCEs) were indicated for neglected diseases, even though these diseases account for 11% of the global disease burden. [1]

 

Human African Trypanosomiasis

 

Leishmaniasis

Sleeping sickness or Human African Trypanosomiasis (HAT) is endemic in 37 African countries and around 13 million people are at medium to high risk of being infected. HAT is transmitted by the tsetse fly and is fatal without treatment. Up until 2009, existing treatments for stage 2 of the disease were toxic or difficult to administer. In 2009, DNDi and its partners launched the first new treatment for HAT in 25 years.  

350 million are at risk of leishmaniasis worldwide.The parasite that leads to infection is called Leishmania and transmitted by sandflies. Leishmaniasis is a poverty-associated disease with several different forms; visceral leismaniasis, which is fatal without treatment and cutaneous leishmaniasis are the most common. Existing treatments are difficult to administer, toxic, and costly. Drug resistance is also an increasing problem.

 

Chagas Disease

 

Paediatric HIV

Chagas disease is endemic in 21 countries across Latin America and kills more people in the region than any other parasite-borne disease, including malaria. In total, 70 million people are at risk worldwide and patient numbers are growing in non-endemic countries such as the United States, Australia, and Europe. The disease is transmitted by an insect known as the “kissing bug” and, without treatment, is potentially fatal. Existing treatments have an unsatisfactory cure rate and can have toxic side effects.

 

  There are currently 36.9 million people living with HIV/AIDS worldwide. This figure includes 1.8 million children below 15 years of age, the overwhelming majority of whom live in sub-Saharan Africa. Infants acquire the virus before, during, or after birth, and without access to treatment half of them will die before their second birthday.

Filaria

 

Mycetoma

Filarial diseases such as lymphatic filariasis (elephantiasis), onchocerciasis (river blindness), and loiasis (loa loa) cause chronic illness and life-long disabilities leading to great suffering and social stigmatization. For 20 years, the strategy to control filarial diseases directed by the WHO has been based on mass drug administration (MDA) programmes with drugs such as ivermectin. However these current treatments target the juvenile worm (microfilariae) and need to be repeated: for 10-15 years in the case of onchocerciasis. There is an unmet medical need for a drug that can kill the adult worms (macrofilaricide).  

Mycetoma is characterized by devastating deformities, disability, and high morbidity. It causes stigma and has a serious negative socio-economic impact on those affected. The exact route of infection is unknown. While Mycetoma is endemic in many tropical and subtropical regions, there is only scant data on incidence and prevalence. Treatment requires prolonged courses of ineffective, prohibitively expensive antifungals which have serious side effects, followed by extensive and often destructive surgery (amputation).

 

Hepatitis C

 

Malaria

About 130-150 million people are living with chronic Hepatitis C infection (HCV), which causes inflammatory liver disease. HCV exists in six genotypes and most global research and development efforts are focused on genotypes prevalent in high-income countries, neglecting others which infect the majority (85%) of patients in low- and middle-income countries (LMICs). Competition between companies has hindered the development of optimal pan-genotypic combinations essential for public health use. The newest drugs, Direct Acting Antivirals are very expensive and nearly half of the world’s HCV patients live in LMICs excluded from current licensing agreements.   Malaria kills one child every 1 minute in sub-Saharan Africa and is the leading parasitic cause of morbidity and mortality worldwide. 3.2 billion people are at risk and while effective treatments exist, they have important limitations, including widespread drug resistance. DNDi and its partners have developed two inexpensive, efficacious, field-adapted treatments.

 

[1] Pedrique et al. The drug and vaccine landscape for neglected diseases (2000-2011): a systematic assessment The Lancet 2013; 1(6) e371–e379