Neglected Tropical Diseases
Neglected tropical diseases (NTDs) are a set of 20 diseases that disproportionately affect more than one billion people living primarily in the developing world.
In 2019 we provided $500,000 in financial support to The Carter Center for the Onchocerciasis Elimination Program for the America, to support the elimination of onchocerciasis in the Americas
As a leading cause of disability, NTDs carry with them significant social and economic burdens. However, as the World Health Organization (WHO) reports, many of these diseases can be effectively controlled and, in many cases, eliminated. Our company has a long-standing commitment to research and access to treatment for NTDs. We initiated research on MECTIZAN® (ivermectin) for use in humans for the NTD onchocerciasis (river blindness) in 1978, leading to the creation of the groundbreaking MECTIZAN Donation Program.
Through a range of in-house programs and external partnerships, we continue to conduct research and enable access to address the burden of NTDs today.
Programs and initiatives
Since 2008, G-FINDER has reported on global investments in neglected disease research and development (R&D) from a range of public and private institutions, and is considered a unique source of current information and insights into ongoing trends for stakeholders engaged in NTDs.
To contribute to global awareness and advocacy on R&D for NTDs, we participate in the annual G-FINDER survey. We are pleased to note that industry investment in neglected disease R&D has increased in each of the last five years and reached new record highs in each of the last three years.
Reported industry investment has increased by nearly 50 percent since 2008, and this growth in industry investment contributed to a dramatic increase in funding for clinical development and post-registration studies. The most recent analysis of global funding for basic research and product development for neglected diseases from public and private sector sources topped the U.S. $4 billion mark and is the highest level recorded by the G-FINDER survey.
The London Declaration
We are an original signatory to the London Declaration, a collaborative effort launched in 2012 to accelerate progress toward eliminating or controlling 10 NTDs by the end of the decade.
Our company joined 12 other global pharmaceutical companies and many other stakeholders, including endemic country governments, WHO, the Bill & Melinda Gates Foundation, the U.S. Agency for International Development (USAID), the UK Department for International Development (DFID), nongovernmental organizations (NGOs) and other organizations in this effort.
Together with the other pharmaceutical companies, we committed to continuing or increasing donations of medicines to treat or prevent these diseases. Donors committed financial resources, and NGOs agreed to support implementation needs. The partners came together under the banner of “Uniting to Combat NTDs” to track progress and identify gaps that need to be addressed in order to reach the goals of the London Declaration. Through the MECTIZAN Donation Program, we are helping to achieve the goals of eliminating the transmission of onchocerciasis (river blindness) and eliminating lymphatic filariasis (LF) as a public health problem.
To help provide the necessary ongoing technical support for country-led NTD programs, including support for onchocerciasis and LF elimination programs in Africa, in 2018, our company provided $500,000 in financial support to WHO’s Expanded Special Program to Eliminate NTDs (ESPEN). To support the elimination of onchocerciasis in the Americas, in 2019, we provided $500,000 in financial support to The Carter Center for the Onchocerciasis Elimination Program for the Americas.
Although existing tools are having a major impact, several NTDs require new or improved drugs and diagnostics to achieve the goals of the London Declaration. We are engaged in various efforts to advance progress toward developing new drugs and diagnostics. For example, together with several other companies, we are providing access to compound libraries with external researchers through the World Intellectual Property Organization’s Re:Search (WIPO Re:Search) consortium and Drugs for Neglected Diseases Initiative (DNDi).
Research and development
Our company is one of the founding members of WIPO Re:Search, a consortium of public and private organizations that facilitates research on NTDs, malaria and tuberculosis. Our inaugural collaboration through this consortium was with investigators at the University of California, San Diego (UCSD), and included providing scientists with a series of compounds for screening that have the potential to lead to better and safer treatments for patients suffering from schistosomiasis. This disease is caused by a blood-borne parasite and affects millions of people living in the developing world.
Also through WIPO Re:Search, and as described in the section on Malaria of this report, we entered into a collaboration with researchers at the Walter and Eliza Hall Institute of Medical Research (WEHI) in Australia in late 2014 that is focused on the research and development of antimalarial drugs that could potentially impact the replication and transmission of malaria.
In 2015, our joint team was successful in applying for and receiving a Wellcome Trust Pathfinder Award. Initial screening yielded quality hits with excellent antimalarial potency and in vivo proof-of-concept activity. With the encouragement and support of the BioVentures for Global Health Partnership Hub component of WIPO Re:Search, the team has applied and successfully competed for further rounds of funding from the Wellcome Trust (WT). Such support has and continues to accelerate the team’s collaborative research program, with the objective to have a novel and potentially transformational antimalarial candidate ready for testing in the clinic by early 2022.
In furtherance of our commitment to the WIPO Re:Search mission and community, we continue to provide relevant expertise to other WIPO Re:Search members as requested and appropriate, and to contribute to the development and implementation of the consortium’s vision and strategy. We also continue to consider other collaboration opportunities where our company’s contributions can provide unique and significant impact.
Emerging pathogens with potential to generate severe epidemics
We have a long-running collaboration with the United States Army Medical Research Institute of Infectious Diseases (USAMRIID) to perform targeted screening of the company’s compounds. The primary focus of this effort has included viral pathogens such as Ebola, Zika and related viruses that have the potential to generate severe epidemics and pose a threat to civilian and military populations.
There is not a more timely example of the importance of this type of partnership than is illustrated by the current COVID-19 pandemic and the ability to quickly pivot to focus the assets and expertise of both parties on viruses like SARS-CoV-2 as well on future emerging viral pathogens with the potential to cause pandemics.
One example has been the testing of hit molecules from a USAMRIID screen against MERS as potential starting points for a program against COVID-19. Follow-up to these screening efforts is ongoing.
Read more about our response to COVID-19.
Nontuberculous Mycobacterial (NTM) Disease
Through our company’s membership and collaborative activities in the TB Drug Accelerator (TBDA) consortium, we are connected with investigators who are pursuing research programs that are complementary to those directed toward TB, although they are outside of the scope of the TBDA’s remit. This provides an opportunity to leverage learnings from our TBDA drug discovery efforts to make impactful contributions to these programs. One such example is our NTM disease focused collaboration with researchers at Hackensack Meridian Health’s Center for Discovery and Innovation, as part of their NIH Center of Excellence in Translational Research.
NTM disease is a collection of tuberculosis (TB)-like symptomatic infections caused by relatives of Mycobacterium tuberculosis (Mtb), the causative agent of TB. Pulmonary infection is the most common‒and the most difficult to treat‒presentation of NTM disease. As such, NTM infection is an important emerging threat to cystic fibrosis (CF) patients and poses significant threat to other immunocompromised patient populations.
Current treatment options for NTM disease are limited and usually include use of antibiotics that have not been developed against NTM pathogens or tested through clinical trials in NTM patients. Multidrug therapy is of long duration‒comparable to that for multi-drug resistant TB‒and the cure rates are comparable, or worse. For some NTM pathogens, such as M. abscessus (Mab), there is no reliable cure. Even though, in the U.S., the incidence rate of NTM disease has surpassed that of TB, there is little focus on drug discovery and development for NTMs, mostly owing to the recalcitrant nature of the infection.
Our collaborative research program aims to elucidate new preclinical development candidates optimized against NTM pathogens (e.g., M. abscessus/M. avium), leveraging drug discovery efforts from the TBDA program to rapidly progress advanced compound series.
Learn more about our efforts to fight Tuberculosis.
- Bill & Melinda Gates Foundation
- Drugs for Neglected Diseases Initiative (DNDi)
- The Carter Center for the Onchocerciasis Elimination Program for the Americas
- Hackensack Meridian Health’s Center for Discovery and Innovation
- U.S. Agency for International Development (USAID)
- U.S. Army Medical Research Institute of Infectious Diseases (USAMRIID)
- UK Department for International Development (DFID)
- Wellcome Trust (WT).
- World Health Organization (WHO)
- World Intellectual Property Organization’s Re:Search (WIPO Re:Search) consortium