A report from GHTC and Policy Cures Research examines how US federal funding for global health R&D is delivering impact both abroad and at home.

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R&D across health areas | Neglected Tropical Diseases

R&D for Neglected Tropical Diseases

How new tools can transform the fight

Neglected tropical diseases (NTDs) are a group of diseases that exert a crushing burden in poor and underserved communities around the world. Named for the limited attention they have historically received, NTDs are inextricably linked to poverty and inequality. These diseases exact a heavy toll, causing chronic illness, severe disfigurement, blindness, or even death. They can also exacerbate malnutrition and delay cognitive development, as well as reduce economic productivity, trapping families and communities in poverty. Increased attention in recent years has brought new resources to the fight against NTDs and fueled research breakthroughs. Yet, very significant gaps remain in the arsenal of tools needed to control and eliminate these diseases, underscoring the need for the research and development (R&D) of new tools.

  • 1 billion+people
    worldwide are affected by NTDs
  • 179 countries
    reported an NTD in 2021
  • 21 conditions
    are classified as NTDs

Research successes

Technologies have transformed the fight against NTDs:

  • The world’s first vaccine for chikungunya, a disease causing debilitating joint pain, which was developed with DoD support, was approved in 2023.
  • A second dengue vaccine that is safe for use in individuals with and without prior infection was approved in 2022.
  • The first all-oral cure for sleeping sickness, a fatal neurological disease, was approved in 2018. Fexinidazole cures all stages of the disease in ten days, replacing prior treatments that involved two weeks of intravenous injections at a hospital.
  • The first new drug for river blindness in 20 years, moxidectrin, was approved in 2018 to treat this disfiguring and blinding disease.
  • Child-friendly drugs for schistosomiasis and Chagas disease were approved in 2024 and 2011, respectively, enabling children to receive appropriate care for these painful parasitic diseases.
  • New shorter, more effective combination therapies for visceral leishmaniasis—a disease causing spleen and liver damage—, introduced in the 2000s, are improving care.
  • New diagnostics, including rapid tests for river blindness and elephantiasis, developed with NIH research, and a test that detects all four types of dengue, developed by CDC, have been introduced.

Continued progress is possible, not inevitable Continued progress is possible, not inevitable

Continued progress is possible, not inevitable

Key missing tools Key missing tools

To eliminate NTDs, we need new and improved tools, including:

  • New and improved diagnostics to rapidly detect infection at the point-of-care in low-resource settings and, in some cases, to distinguish between different strains or stages of a disease.
  • Vaccines to prevent and treat infection. Beyond dengue and chikungunya, no vaccines exist for the remaining 20 NTDs prioritized by the World Health Organization.
  • Antivenoms to treat snakebites.
  • New vector and biological control tools, like space spray insecticides and Wolbachia bacteria that disrupt reproduction, to reduce mosquito and other vector populations.
  • New and improved treatments and cures, including therapies designed for children and shorter, simplified regimens with fewer side effects. While there are low-cost, effective interventions to combat some NTDs, in many cases, these available treatments are still lengthy and burdensome with significant side effects.

Breakthroughs on the brink Breakthroughs on the brink

  • An all-oral, single-dose treatment for sleeping sickness, acoziborole, which is in late-stage development, could simplify treatment administration, laying the groundwork for the eventual elimination of this deadly disease. A pediatric version of this medicine is also in development.
  • An oral treatment for dengue, developed with NIH support, showed promising results in Phase 2 clinical trials, offering hope that in the coming years, we will see the first-ever treatment for this disease.
  • Researchers have created a synthetically produced antibody that can neutralize a neurotoxin found in the venom of four different deadly snake species, laying the foundation for the eventual development of a universal antivenom for snakebites.
  • Several vaccines for schistosomiasis—a disease that causes liver damage or kidney failure if left untreated—are in development, including a candidate now in Phase 2 trials developed with NIH and DoD support.
  • Fosravuconazole, a potential new weekly treatment for eumycetoma, a fungal disease that can cause severe deformity of the limbs, demonstrated promising results in Phase 2 trials. Existing treatments for this disease are toxic, costly, only cure a fraction of patients, and require daily dosing.
  • Two NIH-funded vaccine candidates against hookworm infection are in clinical development, including one in Phase 2 development. This parasitic infection causes gastrointestinal issues and protein deficiency and is linked to impaired learning in children.
  • Several diagnostic tests for Buruli ulcer—a disease that causes painful ulcers and swelling—are in development, including an instrument-free, point-of-care test and other tools for remote health care settings. Today, diagnosis is typically done by appearance, which leads to high rates of misdiagnosis and delayed treatment.
  • Mosquitoes infected with Wolbachia, a bacteria that impact their reproduction and reduce population size, have been released to fight dengue and other mosquito-borne diseases in locations ranging from Indonesia to the Florida Keys, with early results showing promise. NIH and USAID have funded research into this approach.

US government investment in NTD R&D (in 2022) US$ millions

Breakthroughs on the brink
US Government R&D efforts US Government R&D efforts

The US government is advancing R&D to control and eliminate NTDs through a whole-of-government approach:

  • National Institutes of Health (NIH) conducts R&D for new treatments, vaccines, diagnostics, vector control products, and other tools to combat NTDs.
  • Centers for Disease Control and Prevention (CDC) advances research to develop new and improved diagnostics and interventions to strengthen NTD control and elimination efforts.
  • Department of Defense (DoD) undertakes R&D to create vaccines, drugs, diagnostics, and vector control products for NTDs that threaten US service members stationed abroad.
  • US Agency for International Development (USAID) has in the past supported the development of new drugs and diagnostics for a select group of NTDs as part of its flagship NTD Program but largely focuses on deploying existing interventions.
  • Food and Drug Administration administers the Tropical Disease Priority Review Voucher Program to incentivize investment in products for NTDs.
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Introduction

1 billion people affected; 21 conditions*: World Health Organization. Neglected tropical diseases page. Accessed July 17, 2024. https://www.who.int/health-topics/neglected-tropical-diseases

*WHO designated NTDs include: Buruli ulcer; Chagas disease; dengue and chikungunya; dracunculiasis; echinococcosis; foodborne trematodiases; human African trypanosomiasis; leishmaniasis; leprosy; lymphatic filariasis; mycetoma, chromoblastomycosis and other deep mycoses; noma; onchocerciasis; rabies; scabies and other ectoparasitoses; schistosomiasis; soil-transmitted helminthiases; snakebite envenoming; taeniasis/cysticercosis; trachoma; and yaws.

179 countries reported: More countries eliminate neglected tropical diseases but investments key to sustain progress. Press release. World Health Organization; January 30, 2023. https://www.who.int/news/item/30-01-2023-more-countries-eliminate-neglected-tropical-diseases-but-investments-key-to-sustain-progress

Research successes

World’s first vaccine for chikungunya: FDA Approves First Vaccine to Prevent Disease Caused by Chikungunya Virus. Press release. US Food and Drug Administration; November 9, 2023. https://www.fda.gov/news-events/press-announcements/fda-approves-first-vaccine-prevent-disease-caused-chikungunya-virus

Second dengue vaccine: Takeda’s QDENGA®▼ (Dengue Tetravalent Vaccine [Live, Attenuated]) Approved in Indonesia for Use Regardless of Prior Dengue Exposure. Press release. Takeda; August 22, 2022. https://www.takeda.com/newsroom/newsreleases/2022/takedas-qdenga-dengue-tetravalent-vaccine-live-attenuated-approved-in-indonesia-for-use-regardless-of-prior-dengue-exposure/

First all-oral cure for sleeping sickness: Drugs for Neglected Diseases initiative. Sleeping Sickness: Fexinidazole for T.b. gambiense. Accessed July 17, 2024. https://dndi.org/research-development/portfolio/fexinidazole/

New drug for river blindness: First new treatment for river blindness approved by U.S. FDA in 20 years. Press release. World Health Organization Special Programme for Research and Training in Tropical Diseases; June 14, 2018. https://tdr.who.int/newsroom/news/item/14-06-2018-first-new-treatment-for-river-blindness-approved-by-u-s-fda-in-20-years

Child-friendly drugs for schistosomiasis: The European Medicines Agency adopts positive opinion of our treatment option for schistosomiasis in preschool-aged children. Press release. Pediatric Praziquantel Consortium; December 15, 2023. https://www.pediatricpraziquantelconsortium.org/newsroom/european-medicines-agency-adopts-positive-opinion-our-treatment-option-schistosomiasis

Child-friendly drugs for Chagas disease: Drugs for Neglected Diseases initiative. Chagas disease: Paediatric Benznidazole. Accessed July 17, 2024. https://dndi.org/research-development/portfolio/paediatric-benznidazole/

Combination therapies for visceral leishmaniasis: Drugs for Neglected Diseases initiative. Visceral leishmaniasis. Accessed July 17, 2024. https://dndi.org/research-development/portfolio/ssg-pm/

Rapid tests for river blindness and elephantiasis: Innovative partnership brings to market new tools for neglected tropical diseases. Press release. PATH; April 11, 2016. https://www.path.org/our-impact/media-center/innovative-partnership-brings-to-market-new-tools-for-neglected-tropical-diseases/

Test that detects all four types of dengue: CDC Begins Distribution of New Dengue Fever Tests. *Global Biodefense. June 18, 2012. Accessed July 17, 2024. https://globalbiodefense.com/2012/06/28/cdc-begins-distribution-of-new-dengue-fever-tests/

Continued progress is possible, not inevitable

Bill & Melinda Gates Foundation. *Goalkeepers 2024 Report. The Race to Nourish a Warming World. Bill & Melinda Gates Foundation; 2024. https://www.gatesfoundation.org/goalkeepers/report/2024-report/

Key missing tools

Bill & Melinda Gates Foundation. *Goalkeepers 2024 Report. The Race to Nourish a Warming World. Bill & Melinda Gates Foundation; 2024. https://www.gatesfoundation.org/goalkeepers/report/2024-report/

Breakthroughs on the brink

Single-dose treatment for sleeping sickness: Drugs for Neglected Diseases initiative. Sleeping sickness: Acoziborole. Accessed July 17, 2024. https://dndi.org/research-development/portfolio/acoziborole/

Oral treatment for dengue: Janssen Announces Promising Antiviral Activity Against Dengue in a Phase 2a Human Challenge Model. Press release. Johnson & Johnson; October 20, 2023. https://www.jnj.com/media-center/press-releases/janssen-announces-promising-antiviral-activity-against-dengue-in-a-phase-2a-human-challenge-model

Antivenom: Wilcox, C. Powerful new antivenom raises hopes for a universal solution to lethal snakebites. *Science. February 21, 2024. Accessed July 17, 2024. https://www.science.org/content/article/powerful-new-antivenom-raises-hopes-universal-solution-lethal-snakebites

Several vaccines for schistosomiasis: Infectious Disease R&D Tracker. Policy Cures Research; 2023. Accessed July 17, 2024. https://www.policycuresresearch.org/pipeline-database/

Potential new weekly treatment for eumycetoma: Drugs for Neglected Diseases initiative. Mycetoma: Fosravuconazole. Accessed July 17, 2024. https://dndi.org/research-development/portfolio/fosravuconazole/

Vaccine candidates against hookworm: Infectious Disease R&D Tracker. Policy Cures Research; 2023. Accessed July 17, 2024. https://www.policycuresresearch.org/pipeline-database/

Vaccine candidates against hookworm, candidate in phase II development/NIH funding: Efficacy of Na-GST-1/Alhydrogel Hookworm Vaccine Assessed by Controlled Challenge Infection. ClinicalTrials.gov identifier. NCT03172975. Accessed July 17, 2024. https://clinicaltrials.gov/study/NCT03172975

Diagnostic tests for Buruli ulcer: Infectious Disease R&D Tracker. Policy Cures Research; 2023. Accessed July 17, 2024. https://www.policycuresresearch.org/pipeline-database/

Diagnostic tests for Buruli ulcer, instrument-free, point-of-care test: FIND. Rapid tests for Buruli ulcer control. Accessed July 17, 2024. https://www.finddx.org/what-we-do/projects/rapid-tests-for-buruli-ulcer-control/

Mosquitoes infected with *Wolbachia: World Mosquito Program. Accessed July 17, 2024. https://www.worldmosquitoprogram.org/

Mosquitoes infected with *Wolbachia, NIH support: National Institute of Health National Institute of Allergy and Infectious Diseases. Neglected Tropical Diseases Scientific Findings. Accessed July 17, 2024. https://www.niaid.nih.gov/research/neglected-tropical-diseases-scientific-findings

Mosquitoes infected with *Wolbachia, USAID support: USAID. *Combating Zika and Future Threats: A Grant Challenge for Development: Using Wolbachia to prevent disease transmission. USAID; 2021. https://www.usaid.gov/sites/default/files/2022-05/2a_-_Lessons_Learned_on_Supporting_Crisis-Related_Innovations_-_MAY_2021_-_WMP_Success_Story_FINAL_1.pdf

US government investment in NTD R&D

G-FINDER data portal. Policy Cures Research; 2023. Accessed May 14, 2024. https://gfinderdata.policycuresresearch.org/

US government R&D efforts

Kaiser Family Foundation. The U.S. Government and Global Neglected Tropical Disease Efforts. Accessed July 17, 2014. https://www.kff.org/global-health-policy/fact-sheet/the-u-s-government-and-global-neglected-tropical-diseases/

US Food and Drug Administration. Tropical Disease Priority Review Voucher Program. Accessed July 17, 2014. https://www.fda.gov/about-fda/center-drug-evaluation-and-research-cder/tropical-disease-priority-review-voucher-program

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