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The Scientist

Encouraging Development of Therapeutics for Neglected Diseases - Day 1

Conference Proceeding CD Now Available
  • Speaker Presentations
  • Poster Abstracts
  • and More!



7:00 am Registration and Morning Coffee

Challenges and Opportunities

7:55 Chairperson’s Opening Remarks

Susana Mendez, Assistant Professor, Baker Institute for Animal Health, Cornell University

8:00 Keynote Presentation
Onchocerciasis in the Americas: From Arrival to (near) Elimination

Ken Gustavsen, MBA, Director, Corporate Responsibility, Merck

Onchocerciasis (river blindness) is a blinding parasitic disease that threatens approximately 120 million people worldwide. While 99% of the population at-risk for infection from onchocerciasis in Africa, some 500,000 people in the Americas are threatened by infection. A relatively recent arrival to the western hemisphere, onchocerciasis was presumably brought to the New World through the slave trade, migration and exploration. The centuries since its arrival have seen advances in diagnosing, mapping and treating the disease. Once endemic to six countries in the Americas (Brazil, Colombia, Ecuador, Guatemala, Mexico and Venezuela), onchocerciasis is on track for elimination by the year 2012. The success of this public health program is due to a robust partnership involving national governments, local communities, donor organizations, intergovernmental bodies, academic institutions, non-profit organizations and the pharmaceutical industry. Additionally, the lessons learned through the efforts in the Americas are in turn informing the program to control and eliminate onchocerciasis in Africa.

8:30 Delivering New Therapeutics for Neglected Tropical Diseases: Can Product Development Partnerships Do the Job?

Jean-Pierre Paccaud, Director, Business Development, Drugs for Neglected Diseases Initiative

While patients from rich countries have good access to new and improved treatments, lack of available funding for high-quality, groundbreaking research in poor countries results in millions of people suffering the burden of neglected infectious diseases. The Drugs for Neglected Diseases Initiative (DNDi) is a not-for-profit organization working to eliminate this research gap. By working in partnership with the public and private sector, DNDi created the largest ever R&D portfolio for kinetoplastid diseases. Challenges and successes will be discussed.

9:00 Pfizer’s Partnerships to Improve Access to Quality Drugs through Supply Chain Improvement

Martina Flammer, M.D., MBA, Senior Director, Global Access, Pfizer, Inc.

9:30 The Center for World Health & Medicine (CWHM) at Saint Louis University: A Unique Model for the Development of New Therapeutics for Neglected Diseases

Peter Ruminski, Executive Director, Center for World Health & Medicine, Saint Louis University

The CWHM consists of a full team of former pharmaceutical scientists embedded with basic and clinical researchers at an academic institution focused on development of new therapies for neglected diseases. With an emphasis on repurposing existing advanced clinical compounds toward identified mechanisms for these alternate indications, the CWHM collaborates with international experts in the targeted diseases.

Sponsored by
10:00 Using Mechanism of Action Driven Systematic Drug Repositioning to Identify Novel Therapies for any Neglected Disease

Aris Persidis, Ph.D., President, Biovista, Inc.
Mechanism of Action (MoA) can be used to map any drug against any disease and any adverse event. A systematic effort to identify novel repositioned drugs for neglected diseases will be presented. It is based on Biovista’s Clinical Outcome Search Space technology.

10:30 Networking Coffee Break with Poster Viewing


The Case of Leishmaniasis

11:00 The Antituberculosis Drug Pyrazinamide Affects the Course of Leishmaniasis and Increases Immune Activation

Susana Mendez, Assistant Professor, Baker Institute for Animal Health, Cornell University

The leishmaniases are a group of emerging neglected diseases with no vaccine. Drug therapy is inadequate due to expense, intravenous administration, side effects and resistance. We have discovered that pyrazinamide, an oral antibiotic employed in the treatment of tuberculosis, has antileishmanial activity in vivo and in vitro, and in both cutaneous and visceral disease. Moreover, we have found that pyrazinamide analogs have higher antileishmanial activity than the parental compound. The potential drug targets are currently being evaluated.

11:30 A Public Private Partnership for the Development of a New Medicine to Treat Leishmaniasis

Karolina Les, Research Scientist, Chemistry, PolyTherics Ltd.

Visceral Leishmaniasis (VL) is a neglected disease that can be effectively treated with a costly and heat sensitive medicine in a complex lipid-based formulation. Using the same pharmacologically active ingredient, amphotericin B (AmB), we are developing a more cost effective and stable formulation by complexing AmB to a water-soluble polymer, rather than to lipids. This new form of AmB is being developed in a close collaboration between PolyTherics Ltd., the University of London and the not for profit organization, Drugs for Neglected Diseases Initiative (DNDi).

Incentives for Getting Involved

12:00 pm Modernizing the Drug and Diagnostics Evaluation and Regulatory Network

Marc Boutin, Ph.D, Executive Vice President and COO, National Health Council

Due to increasingly scarce resources for health research and the current regulatory system, the status quo is not yielding the treatments needed to address the growing epidemic of chronic diseases. How can we update the regulatory system to remove barriers to invention and provide greater predictability in the search for answers to unmet medical needs? This presentation will discuss ways to encourage scientific inquiry, reward ingenuity, and speed the review and approval of diagnostics and treatments that improve patient outcomes.

Sponsored by
12:30 Luncheon Presentation
Nanoviricides, a Novel Technology for Highly Effective Antivirals Against Neglected Viral Diseases
Anil Diwan, Ph.D., President, NanoViricides, Inc.
A nanoviricide® is a flexible polymer backbone with virus-specific ligands attached. Virus binding to the nanoviricide is believed to occur via specific interaction between the nanoviricide ligands and the viral surface glycoproteins leading to virus neutralization. We have developed a number of virus-specific, yet broadly neutralizing, preclinical nanoviricides for neglected diseases.

1:55 Chairperson’s Remarks

2:00 Incentives for Development of Therapeutics for Neglected Tropical Diseases

Jennifer Hannesschlager, Principal, Scientific Affairs, Tiber Creek Partners, LLC

With the FDA’s Tropical Disease Voucher, the Hilleman Laboratory in India, and financial investments available from the National Institutes of Health and elsewhere, the financial model for advanced development of therapeutics for global health diseases is changing. This talk will discuss these opportunities and incentives, how industry can get involved in these programs, and the challenges that industry participants should be aware of.

Current Strategies for Approaching NTD’s

2:30 Novartis Research Efforts on Neglected Diseases

Brigitta Tadmor, Ph.D., Vice President, Global Head, Diversity & Inclusion and Health Policy, Novartis Institutes for BioMedical Research

Research on diseases of the developing world is part of the Novartis core mission. Our research strategy is driven by high unmet medical need and scientific understanding of disease. Our research portfolio includes diseases that affect currently underserved populations, such as patients with rare disease, patients in the developing world and other groups that traditionally have been neglected by the pharmaceutical industry. Our drug discovery and vaccine efforts focus on tropical infectious diseases, cancer and other major diseases that disproportionately affect people in the developing world.

Supported by
3:00 Eisai’s Approach to NTDs

B.T. Slingsby, M.D., Ph.D., MPH, Director, Global Partner Solutions, Eisai

Eisai is a Japanese pharmaceutical company committed to improving the lives of patients and their families worldwide. Centered on partnerships with public and private entities, Eisai is committed to the research and development of new medicines for diseases of the developing world, and to the creation of new business models that ensure sustainable access to medicines.

3:30 Networking Refreshment Break with Poster Viewing

4:15 Thousands of Chemical Starting Points for Antimalarial Lead Identification

James Brown, Ph.D., Director, Computational Biology, GlaxoSmithKline

No new chemical class of antimalarials has been introduced into clinical practice since 1996 and there is a recent rise of parasite strains with reduced sensitivity to the newest drugs. In 2010, we published in the journal Nature the results of a screen of nearly 2 million compounds from GlaxoSmithKline’s chemical library for inhibitors of Plasmodium falciparum. Over 13,000 compounds were confirmed to inhibit parasite growth and more than 8,000 compounds showed potent activity against a multidrug resistant strain. Most compounds originated from internal company projects and are new to the malaria community. These chemical structures and associated data were made public to encourage further academic-industry partnerships in anti-malarial drug discovery.

4:45 Partnering to Develop New Medicines to Control and Eradicate Malaria

Julie Lotharius, Ph.D., Associate Director, Translational Medicine, Medicines for Malaria Venture

New ways emerged to stimulate research into new, affordable and effective medicines. The most efficient of these is the Product Development Partnership model, exemplified by MMV (Medicines for Malaria Venture), which, over the past decade, has led the renaissance in discovery and development of new medicines to control this terrible disease. The recent call to look beyond malaria control and aim for its eradication has prompted research into critical stages of the life cycle of the malaria parasite that could be targeted by new molecules, and block transmission of the disease from patients to the anopheles vector. Recent advances in genome-based technologies and in vitro screening of whole parasites have broadened the range of therapeutic targets and are accelerating the development of a new generation of treatments for both malaria control and its ultimate eradication.

5:15 Pharmaceutical Development Partnerships (PDP): A Critical Consideration for Discovering and Developing Drugs for Neglected Diseases

Rashmi H. Barbhaiya, Ph.D., CEO & Managing Director, Advinus Therapeutics, Pvt Ltd., India

5:45 Focused Breakout Discussions:

Topic: Drug Repositioning Projects for ND – What does Local Research need from the Developed World

Moderator: Peter Ruminski, Executive Director, Center for World Health & Medicine, Saint Louis University

Topic: Recommendations to Enhance Incentive Funding for NTD R&D

Moderator: Ken Gustavsen, MBA, Director, Corporate Responsibility, Merck

Topic: Evaluation of Current Funding Opportunities and Motivations

Moderator: Jennifer Hannesschlager, Principal, Scientific Affairs, Tiber Creek Partners, LLC

Topic: The Importance of Strategic Partnerships

Moderator: Jean-Pierre Paccaud, Director, Business Development, Drugs for Neglected Diseases Initiative


6:45 End of Day

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