Breakthroughs Blog: NTDs

In this regular feature on Breakthroughs, we highlight some of the most interesting reads in global health research from the past week. The GHTC released its 2012 policy report at a Capitol Hill briefing this week. The report provides recommendations for policymakers so they can continue to save lives around the world by supporting research and development for new global health products. This is the third annual report released by the GHTC. The Infectious Disease Research Institute announced that the first clinical trial for a new vaccine for visceral leishmaniasis has begun in Washington State. The US Food and Drug Administration has approved the first-ever 4-in-1 flu vaccine. The Office of Science and Technology Policy has completed its review of final drafts of new federal scientific integrity policies. More than a dozen federal departments and agencies contributed to the process. Secretary of State Hillary Rodham Clinton testified in front of the Senate Appropriations Subcommittee on State and Foreign Operations on Tuesday. She discussed the Global Health Initiative and how it’s “consolidating programs, increasing partners' capacities, and shifting responsibilities to help target our resources where they are most needed and [have the] highest impact.”

GHTC member OneWorld Health (OWH) recently announced that its treatment for kala-azar, or leishmaniasis, was registered for use in Nepal. In this guest post, OWH explains why this announcement is so significant for global health. Today we are thrilled to announce that our antibiotic Paromomycin Intramuscular Injection (PMIM), developed to treat visceral leishmaniasis (VL or kala-azar), has been registered by the government of Nepal. This is the second registration for PMIM in South Asia. It was previously registered with the government of India and added to the National Lists of Essential Medicines of India, Nepal, and Bangladesh. It was also added to the World Health Organization’s Model List of Essential Medicines. Now that the treatment is registered in Nepal, OWH will move forward with our partners on public health studies to help the government determine the best way to distribute and use the treatment in its public health system throughout the VL-endemic regions. What is visceral leishmaniasis? With approximately 500,000 new cases occurring annually, visceral leishmaniasis is the world’s second most deadly parasitic disease after malaria. Fatal if untreated, the official death toll for VL each year is between 50,000 and 60,000. VL is endemic in 79 countries, and primarily afflicts rural, resource-poor populations in India, Nepal, Bangladesh, Ethiopia, Iraq, Sudan, and Brazil, where affordable therapies are needed. Learn more about kala-azar: Kill or Cure: Kala-Azar Now that the treatment is registered in Nepal, OWH will move forward with our partners on public health studies to help the government determine the best way to distribute and use the treatment in its public health system throughout the VL-endemic regions. Photo credit: OneWorld Health What is Paromomycin Intramuscular Injection? An off-patent aminoglycoside antibiotic, paromomycin is an established drug with an extensive history of use and a well-characterized safety profile. OWH developed PMIM as an effective, inexpensive, and safe treatment for VL, and worked with the Indian government and Indian pharmaceutical company Gland Pharma to manufacture and distribute the treatment. PMIM is administered as a once-a-day injection for 21 days. The cost of a course of treatment with PMIM is less than US$20, which is significantly lower than other currently approved VL therapies. Please visit our website for more information – www.oneworldhealth.org.

Danielle Doughman is a program manager for global health research and development (R&D) advocacy at Research!America, the nation's largest not-for-profit public education and advocacy alliance committed to making research to improve health a higher national priority. Research!America is a member of the GHTC. Yesterday in London, some of the biggest players in global health committed to a renewed and intensified focus on neglected tropical diseases (NTDs), a group of diseases affecting the world’s poorest people. One in six people, or 1.4 billion people around the world, has an NTD and one in three people is at risk. Though NTDs don’t always kill, they cause disabilities, making it difficult or impossible for children to attend school and for adults to work and support their families. The international, cross-sector meeting marked collaboration between governments, the World Health Organization (WHO), the World Bank, the Bill & Melinda Gates Foundation, pharmaceutical companies, and global health nongovernmental organizations. It is also a historic pledge that brings new promise and hope to those affected and advances science and health for all. Sir Andrew Witty, CEO of GlaxoSmithKline, noted that the variety of different commitments across sectors demonstrates these collaborations want to “travel far” to end NTDs. “If you want to travel fast, travel alone. If you want to travel far, travel as a group,” he said, quoting an African proverb. Making the best use of existing tools to combat diseases will only get us part of the way. The collaboration's goals are achievable only with new tools—including drugs, vaccines, and diagnostics. Nine of the ten targeted NTDs require new investment in R&D. To take just one example, visceral leishmaniasis (VL) is almost always fatal if untreated, and there are approximately 500,000 cases each year, largely in South Asia and the horn of Africa. The cost of treatment plus loss of income due to one case of VL represents at least 50% of the average spent per family member per year. What’s worse, more than half of people who seek treatment must do so more than once to cure the disease. Available therapies are sometimes toxic or ineffective. New, more effective medicines are needed, at a lower cost and with fewer side effects. A consortium project led by the Drugs for Neglected Diseases initiative, in partnership with California-based OneWorld Health and TDR, WHO’s Special Programme for Research and Training in Tropical Diseases, aims to establish and implement new treatment modalities as successful tools to control and support the elimination of VL in most endemic regions of South Asia. California-based pharmaceutical company Gilead is investing in further research to reduce the cost of treatment. In addition to narrowing funding gaps, new licensing or collaboration agreements have opened up “libraries” of scientific knowledge and research compounds. As a result, one pharmaceutical company recently shared 300 compounds with a lab in Argentina to conduct research on Chagas, an NTD common in rural parts of Central and South America and sometimes found in poor parts of the United States. Something as ambitious as this takes commitment from the public, private, and philanthropic sectors, but the general public must become increasingly engaged as well. The names of diseases like visceral leishmaniasis don’t exactly roll off the tongue of those of us in developed countries, and fewer people could tell you why the condition is so devastating. To increase public awareness and advocacy of NTDs, the Sabin Vaccine Institute-based Global Network’s new END7 campaign aims to end seven of the most common NTDs as a public health threat by 2020. It’s going to take an increased effort on all our parts to make NTDs a thing of the past.