Scientific Publications - Neglected Diseases

2011

Drugs for Neglected Diseases initiative model of drug development for neglected diseases: current status and future challenges by Shing Chang and Jean-Robert Ioset. Future medical chemistry, September 2011, Volume 3, Number 11
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Kinetoplastid Parasites by Von Geldern T, Harhay MO, Scandale I and Don R. Top Med Chem. DOI: 10.1007/7355_2011_17
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Drug discovery and development for neglected diseases: the DNDi model by Chatelain E and Ioset J-R. Drug Design, Development and Therapy, 2011 March, Vol. 5, 175-181.
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Registering New Drugs for Low-Income Countries: The African Challenge by Moran M, Strub-Wourgaft N, Guzman J, Boulet P, Wu L, Pecoul B. PLoS Medicine, 2011 February, Vol. 8, Issue 2, e1000411.
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More about the Regulatory process in the African context here

2010

‘‘Manifesto’’ for Advancing the Control and Elimination of Neglected Tropical Diseases by Hotez P. and Pecoul B. In PLoS NTDs. 2010 May, Vol. 4, Issue 5, e 718
The "manifesto" is meant to mobilise the global community to increase financial support for Neglected Tropical Diseases. All NTDs are "tool ready" but also "tool deficient" as for many diseases tools are suboptimal, incomplete or inadequate. Thus, increased investments in R&D are urgently needed. The eight-point "manifesto" published as an editorial in PLoS NTDs is co-authored by Peter Hotez, President of the Sabin Vaccine Institute, and Bernard Pécoul, Executive Director of DNDi.
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2009

A Business Plan To Help The ‘Global South’ In Its Fight Against Neglected Diseases by Frew S.E, Liu V.Y, and Singer P.A., Health Affairs, 2009, Vol. 28, 6:1760-73
Although neglected tropical diseases (NTDs) threaten the health of those living in the developing world, innovation directed toward addressing NTDs is comparatively meager. Health biotechnology firms in rapidly growing economies in the global South are developing and selling vaccines, diagnostics, and therapeutics for these diseases to local markets. In this paper the authors identify a pipeline of sixty-two NTD products from seventy-eight “Southern” companies. They also propose creation of a Global Health Accelerator—a new nonprofit organization whose mission would be to support and help grow this Southern source of affordable innovation for NTDs.
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Drug discovery for neglected diseases: View of a public-private partnership by Chatelain E, Don R. In Antiparasitic Antibacterial Drug Discovery by Paul M. Selzer (Ed); 2009 Apr: Wiley-Blackwell.
In answer to the lack of modern and effective drugs for diseases such as human African trypanosomiasis (HAT; sleeping sickness) and Chagas disease which present no financial viability for the pharmaceutical industry, new models of drug discovery have been developed.
The Drugs for Neglected Diseases initiative (DNDi) has adopted a model closely related to that of a virtual biotechnology company for identifying and optimizing drug leads.
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2008

Natural Products for Neglected Diseases: A Review by Ioset JR. Current Organic Chemistry. 2008 May; 12 (8): 643-666.
Neglected diseases are responsible for high mortality and morbidity each year in low-income countries. Due to the lack of vaccines and of safe, effective and affordable treatments, there is an urgent need to reinforce the existing therapeutic arsenal against these killers. One of the main opportunities is through the discovery of new molecules from natural origin. The gaps identified in the R&D process aiming to deliver new medicines for neglected diseases however also apply to natural products. A key review of the most promising antiprotozoal molecules recently discovered from natural resources is presented here together with a critical update on their current development status.

2007

Pragmatic and Principled: DNDi's Approach to IP Management by Banerji J and Pecoul B. Intellectual Property Management in Health and Agricultural Innovation: A Handbook of Best Practices (eds. A Krattiger, RT Mahoney, L Nelsen, et al.) 2007.
DNDi's mission is to develop safe, effective, and affordable new drugs for patients suffering from neglected diseases and to ensure equitable access to these drugs. DNDi believes that intellectual property (IP) rights should not pose a barrier to access to these medicines, and that a balanced approach to IP management is therefore critical. DNDi's IP policy articulates the organization’s approach to IP and ensures that products are accessible and affordable to patients who need them most. DNDi recognizes the reality of IP and seeks to implement its humanitarian mission using best, pragmatic practices for IP management. DNDi has already demonstrated that this is feasible, having successfully negotiated with both private and public sector institutions.
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Nature Outlook Neglected Diseases Supplement by various authors. Outlook: Neglected diseases 2007 Sep (449) , No. 7159 pp157-182
Tropical diseases affect more than one billion people, yet there are few effective treatments. And despite much research activity, scientific innovations with therapeutic potential are not making it out of the laboratory. The articles in this Outlook examine what can be done to stimulate the development of effective medicines and deliver them to the people who need them most.
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Neglected diseases: progress in drug development by Croft SL. Curr Opin Investig Drugs. 2007 Feb;8(2):103-4.
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2006

Evaluation of Azasterols as Anti-Parasitics by Gros L, Lorente SO, Jimenez CJ, Yardley V, Rattray L, Wharton H, Little S, Croft SL, Ruiz-Perez LM, Gonzalez-Pacanowska D, Gilbert IH. J Med Chem. 2006 Oct 5;49(20):6094-103.
In this article, the design and synthesis of some novel azasterols is described, followed by their evaluation against Trypanosoma brucei rhodesiense, T. cruzi, Leishmania donoVani, and Plasmodium falciparum, the causative agents of human African trypanosomiasis, Chagas disease, leishmaniasis, and malaria, respectively. Some of the compounds showed anti parasitic activity.
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Endemic tropical diseases: comtemporary health problem due to abandoned diseases in the developing world by Ohta N. Kansenshogaku Zasshi. 2006 Sep;80(5):469-74. Review.
There are two kinds of infectious diseases in the world; diseases being paid attention and neglected diseases. The former diseases include HIV/AIDS, tuberculosis and malaria, the latter group include many parasitic, fungal, bacterial and some of viral infections. "Neglected Infectious Diseases", which have been renamed as Endemic Tropical Diseases by WHO, are endemic in the developing world are not newly appeared diseases, but diseases affecting humans in these decades. However, those diseases were not recognized as serious health problems because of socio-economical and/or scientific reasons. Considering that issues of "Neglected Infectious Diseases" are urgent to be solved and also are challenging for modern medicine and medical sciences, researchers in the developed countries including Japan should make efforts to promote more active researches in this field.
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To Fully Tackle the Gang of Four, Needs-Driven R&D is Essential by Torreele E, Royce C, Don R, Sevcsik AM, Croft S. PLoS Med. 2006 Jun;3(6):e282; author reply e284.
In this correspondence, the authors address the challenges set to the global health audience to address neglected tropical diseases affecting the poor and powerless in resource-poor settings. To tackle the gang of four, adequate and field-adapted health tools must be available, and governments must prioritize needs-driven R&D for those diseases where no such tools exist.
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Global framework on essential health R&D by Chirac P and Torreele E. Lancet. 2006 May 13;367:1560-1.
One of the critical issues to be discussed at the next World Health Assembly ( Geneva , May 22-26) will be a resolution about a global framework on essential health research and development. Over the past years, the crisis in research and development in the worldwide pharmaceutical industry, and in particular the absence of research and development for new medicines targeting diseases that mainly affect people in developing countries (neglected diseases), has become a global concern.
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New initiatives against Africa’s worms by Fenwick A. Trans R Soc Trop Med Hyg. 2006 Mar;100(3):200-7. Epub 2005 Dec 15.
Since 1999, the funding available for the control of diseases of poverty (neglected diseases) has increased mainly due to leverage resulting from donations by the Bill and Melinda Gates Foundation and loans from the World Bank. Many countries have embarked on control programmes on a national scale due to drug donation by pharmaceutical companies through vertical programmes.
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No drugs in an age of plenty: urging governments to redress the balance by Torreele E. Interdisciplinary Science Reviews. 2006 Mar;31(1):3-8.
35,000 people die every day from diseases that disproportionately affect the poor in Latin America, Africa, and Asia , and that have been neglected by the pharmaceutical industry and governments. Although scientific knowledge exists to develop new treatments, political will and the profit-driven pharmaceutical model have not sufficiently supported these efforts. Instead, it has been left to generous philanthropic efforts, but this is unsustainable. Public leadership and support is urgently needed.
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2005

Antitrypanosomal, Antileishmanial, and Antimalarial Activities of Quaternary Arylalkylammonium 2-Amino-4-Chlorophenyl Phenyl Sulfides, a New Class of Trypanothione Reductase Inhibitor, and of N-Acyl Derivatives of 2-Amino-4 Chlorophenyl Phenyl Sulfide by Parveen S, Khan MO, Austin SE, Croft SL, Yardley V, Rock P, Douglas KT. J Med Chem. 2005 Dec 15;48(25):8087-97.
Quaternization of the nitrogen atom of 2-amino-4-chlorophenyl phenyl sulfide analogues of chlorpromazine improved inhibition ~40-fold (3’,4’-dichlorobenzyl-[5-chloro-2 phenylsulfanylphenylamino)-propyl]-dimethylammonium chloride inhibited trypanothione reductase from Trypanosoma cruzi with a linear competitive Ki value of 1.7 ( 0.2 íM). The phenothiazine and diaryl sulfide quaternary compounds were also powerful antimalarials, providing a new structural framework for antimalarial design.
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Government action needed to step up research and development for world's most neglected diseases by Pecoul B. Expert Rev Anti Infect Ther. 2005 Dec;3(6):841-3.
Neglected and most neglected diseases affect millions of people in the world's poorest countries, yet we do not have safe, affordable, and field-adapted vaccines, diagnostics and drugs to tackle them.
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Public-private partnership: from there to here by Croft SL. Trans R Soc Trop Med Hyg. 2005 Oct; 99 Suppl 1:S9-14.
Major changes in research and development (R&D) for drugs to treat tropical and neglected diseases have occurred in the past five years. Public-private partnerships have proved that they can move compounds quickly through the R&D pipeline. The challenge is to ensure that the products are delivered to the people who need them and to ensure that scientists in endemic countries are involved in the whole process.
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DNDi in the British Medical Journal, July 2005: Prioritising Neglected Diseases Related to Poverty by Rhona MacDonald. BMJ. 2005 Jul; 331:12.
Bernard Pécoul, director of the Drugs for Neglected Diseases Initiative, tells Rhona MacDonald how his organisation is hoping to help millions of people living in poverty worldwide.
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Drugs for Neglected Diseases Initiative. Wasunna KM. Afr J Health Sci. 2005 Jan-Jun;12(1-2):i-ii.
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2004

New Drugs for Neglected Diseases: From Pipeline to Patients by Bernard Pécoul. PLoS Med. 2004 Oct; 1(1):e6. Epub 2004 Oct 19.
In wealthy countries,state-funded research has yielded breakthroughs in molecular biology,chemistry,and engineering.These advances have been taken up by the pharmaceutical industry and applied to drug development for a growing range of illnesses and conditions.As a result, patients have access to new drugs that are better tolerated, more specific,and more effective than old ones.
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2003

Recent advances in research and control of malaria, leishmaniasis, trypanosomiasis and schistosomiasis by Croft SL, Vivas L, Brooker S. East Mediterr Health J. 2003 Jul;9(4):518-33. Review.
In the Eastern Mediterranean Region of the World Health Organization (WHO), malaria, schistosomiasis, leishmaniasis and trypanosomiasis are the parasitic diseases of major importance. Our review focuses on recent advances in the control and treatment of these diseases with particular reference to diagnosis, chemotherapy, vaccines, vector and environmental control.
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Initiative launched to develop drugs for neglected diseases by Frankish H. Lancet. 2003 Jul 12; 362(9378):135.
A new initiative that aims to develop drugs to tackle diseases that affect the world's poorest people was launched in Geneva on July 3. The Drugs for Neglected Diseases Initiative (DNDi) aims to identify drugs to combat diseases such as trypanosomiasis, visceral leishmaniasis, and Chagas disease, which affect more than 350 million people every year.
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2002

Reflection & Reaction by Alimuddin Zumla, Lancet Infect Dis 2002 July, 2:393.
Recent global partnership initiatives have focused on stimulating further interest in the development and provision of drugs for the world's top three killer infectious diseases: AIDS/HIV, malaria, and tuberculosis. The “most neglected” diseases (where there are no affordable, effective, easy to use medicines available) continue to cause significant morbidity and mortality in developing countries. This paper is a short introduction to a series of articles outlining research and development priorities for sleeping sickness, visceral leishmaniasis and malaria.
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The world's most neglected diseases. Ignored by the pharmaceutical industry and by public­private partnerships by Yamey G, Torreele E. BMJ. 2002 Jul 27;325(7357):176-7.
The article discusses issues about the “most neglected” diseases as still being ignored not just by the pharmaceutical industry but also by public-private partnerships.
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Drug development for neglected diseases: a deficient market and a public-health policy failure by Trouiller, P. et al., June 2002, Lancet, 359: 2188–94.
There is a lack of effective, safe, and affordable pharmaceuticals to control infectious diseases that cause high mortality and morbidity among poor people in the developing world. This paper analyses outcomes of pharmaceutical research and development over the past 25 years, and reviews current public and private initiatives aimed at correcting the imbalance in research and development that leaves diseases that occur predominantly in the developing world largely unaddressed.
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2001

Neglected diseases of global importance by Ford N, Torreele E. JAMA. 2001 Dec 19;286(23):2943-4.
In this letter to the editor, the authors raise the need of increased responsibility from the private and public sectors in order to ensure that people’s health needs are met, and the importance of non-for-profit drug development initiatives to address the imbalance in the burden of infectious diseases between rich and poor countries.
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Drugs for neglected diseases: a failure of the market and a public health failure? By Trouiller P, Torreele E, Olliaro P, White N, Foster S, Wirth D, Pecoul B. Trop Med Int Health. 2001 Nov;6(11):945-51. Review.
Infectious diseases cause the suffering of hundreds of millions of people, especially in tropical and subtropical areas. Effective, affordable and easy-to-use medicines to fight these diseases are nearly absent. Although science and technology are sufficiently advanced to provide the necessary medicines, very few new drugs are being developed. An urgent reorientation of priorities in drug development and health policy is needed. New and creative strategies involving both the public and the private sector are needed to ensure that affordable medicines for today's neglected diseases are developed.
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Public-private partnerships for health: their main targets, their diversity, and their future directions. Widdus R. Bull World Health Organ. 2001;79(8):713-20. Epub 2001 Oct 24.
The global burden of disease, especially the part attributable to infectious diseases, disproportionately affects populations in developing countries. Inadequate access to pharmaceuticals plays a role in perpetuating this disparity. A large variety of public-private partnerships, combining the skills and resources of a wide range of collaborators, have arisen for product development, disease control through product donation and distribution, or the general strengthening or coordination of health services. Suggestions are made for public, private, and joint activities that could help to improve the access of poor populations to the pharmaceuticals and health services they need.
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Fatal imbalance: the crisis in research and development for drugs for neglected diseases by MSF Campaign for access to medicine, Drugs for Neglected Diseases working group
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1999

Access to Essential Drugs in Poor Countries – A Lost Battle ? ” by Pécoul, B. et al., JAMA, January 27, 1999—Vol 281, No. 4.
This article focuses on the problems of access to quality drugs for the treatment of diseases that predominantly affect the developing world: (1) poor-quality and counterfeit drugs; (2) lack of availability of essential drugs due to fluctuating production or prohibitive cost; (3) need to develop field-based drug research to determine optimum utilization and remotivate research and development for new drugs for the developing world; and (4) potential consequences of recent World Trade Organization agreements on the availability of old and new drugs.
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