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. Two recent announcements occurred regarding US international collaboration on global health research and development (R&D). Earlier this week, the US National Institute of Allergy and Infectious Diseases and the Office of AIDS Research at the National Institutes of Health announced a collaborative project with the Indian Ministry of Science and Technology and Department of Biotechnology to fight tuberculosis in India as part of the Indo-US Vaccine Action Program. On Tuesday, the US Department of Health and Human Services and the European Commission released the first report of the Transatlantic Taskforce on Antimicrobial Resistance (TATFAR), which is a joint commitment by the US and EU to fight antimicrobial resistance. Dr. Lucas Otieno Tina—a physician and researcher at the Kenya Medical Research Institute/US Army Medical Research Unit-Kenya collaboration program—writes in AllAfrica.com about why he has spent his career working to develop a malaria vaccine. He also discusses the promise of the RTS,S malaria vaccine and his involvement in the clinical trials for the vaccine supported by PATH’s Malaria Vaccine Initiative. Last week, the NTD Roundtable—a group of global health partners committed to fighting neglected tropical diseases (NTDs) —hosted briefings in the US House of Representatives and Senate to discuss America’s leadership in the fight against NTDs and the need for greater NTD R&D. The briefings were attended by representatives from key House and Senate committees, the NGO community, and the African diplomatic corps. Among other topics, participants discussed the need for the development of new diagnostic tools and treatments for NTDs. TechCrunch reports about a grant from the Bill & Melinda Gates Foundation to Sure Chill Company in Wales, which has developed a way to store vaccines at a proper temperature without the need for an electrified or gas-powered refrigeration source. The technology reduces problems with vaccine distribution caused by vaccines that lose their efficacy without proper refrigeration.

In this regular feature on Breakthroughs, we highlight some of the most interesting reads in global health research from the past week. This week, pharmaceutical company Sanofi announced that its experimental vaccine against dengue fever has successfully completed its first late-stage clinical trial. The vaccine was proven to reduce the incidence of dengue fever by 56 percent in trial participants in Southeast Asia. Trevor Mundel—president of Global Health at the Bill & Melinda Gates Foundation—wrote this week in Impatient Optimists about the tremendous progress being made in global health research and development (R&D). At this year’s Product Development Forum in Seattle, more than 500 participants gathered together to talk about what is needed to accelerate the development and delivery of high-impact health innovations to those who need them most. Last week, several hundred people came together to celebrate significant achievements in vaccinology over the past two decades and to examine solutions for overcoming remaining hurdles at the Sabin Vaccine Institute’s 20th Anniversary Scientific Symposium. Hosted at the Pan American Health Organization in Washington, DC, it was an energized room filled with visionary leaders from humanitarian and health organizations, nongovernmental organizations, pharmaceutical companies, and academia. Johnson & Johnson has announced its affiliate Janssen Pharmaceuticals has entered into a partnership with the Stop TB Partnership to facilitate access to its tuberculosis treatments. This public-private partnership was established by the newly formed Janssen Global Public Health group.

In this regular feature on Breakthroughs, we highlight some of the most interesting reads in global health research from the past week. During a meeting last week at the United Kingdom’s Royal Society of Medicine, the topic of health research was raised and how it might fit into the post-2015 development agenda. At the meeting, UK Department for International Development Chief Scientific Officer Chris Whitty spoke about the necessity of forward thinking as it relates to global health research. Whitty acknowledged the need to think ahead to what research will be needed from 2020-2040 so that priorities are defined by what the global public health world might look like in the future. Pharmaceutical company GlaxoSmithKline plans to invest up to £130 million in Africa over the next five years to build the continent’s capacity to develop new tools to combat the region’s rising occurrence of noncommunicable diseases (NCDs). New money will go to expanding manufacturing capacity in specific African countries, as well as open the world’s first open-access research and development laboratory for NCDs in Africa. The Bill & Melinda Gates Foundation, the World Bank, and other donors will invest $240 million to fight neglected tropical diseases (NTDs) in low- and middle-income countries. The new funding comes after a pledge by 13 drugmakers two years ago to donate medicines to treat ten NTDs, including river blindness, Guinea worm, and sleeping sickness. Dr. Deborah Birx has been confirmed by the Senate to be the next Global AIDS Coordinator, filling the post to lead the world’s largest health program. Widely praised by global health advocates, Dr. Birx previously led efforts to introduce the President’s Emergency Plan for AIDS Relief-sponsored rollout of life-saving HIV treatments to certain sites in Africa, and launched the first clinical research trials to show proof of concept that an HIV-preventing vaccine was possible.

The US Agency for International Development (USAID) is the nation’s lead foreign assistance agency. Throughout its 50-year history, USAID has worked with other government agencies, the private sector, and nongovernmental organizations to support the development and introduction of affordable health products appropriate for addressing the diseases and health issues in developing countries. The GHTC has launched a new blog series to highlight the impact of USAID’s commitment to global health research and development (R&D). The series will showcase stories from individuals who have benefited from the agency’s support and investment in R&D—as well as people whose lives would vastly improve if USAID were to expand its support for health R&D into new areas. Our third post is by Brian D’Cruz, a volunteer physician who has worked with Médecins Sans Frontières (MSF) in Congo Brazzaville and Central African Republic. Based on his experience with MSF, Brian writes about the urgent need for USAID to support R&D for neglected tropical diseases (NTDs) like sleeping sickness. Q: As an MSF physician working in Congo Brazzaville and Central African Republic, you saw many patients affected by sleeping sickness—an NTD that primarily occurs in the poorest, most rural areas in Africa. Can you tell us about some of your experiences treating this disease during your time with MSF? A: In Central African Republic, I saw firsthand the suffering caused by sleeping sickness. I vividly remember an 11 year old girl named Natacha. She lived in an area where medical care and screening for sleeping sickness had not been available for years due to increasing violence in the area. Our team was the first to treat sleeping sickness there in several years. Several months before we arrived, Natacha started to experience the symptoms of sleeping sickness—including sleep disturbances, memory problems, spasms of her hands, pain all over her body, and seizures. A few weeks before we arrived, she stopped walking. By the time her family was able to get to us, she could no longer speak or feed herself. During this entire time, her family was powerless to help her. By the time they reached our team, we were able to diagnose Natacha and start treatment, but it was too late. She died a few days after reaching us. Brian D’Cruz (second from the left) and other panelists discuss NTDs at a congressional briefing in June 2013. Photo: Research!America Q: What impact does sleeping sickness have on communities and individuals in Congo and Central African Republic? A: Sleeping sickness is fatal if untreated, and what’s more, patients with this disease suffer enormously before their deaths. MSF has been treating patients with sleeping sickness for more than 25 years and we have seen the consequences of this forgotten deadly disease. Natacha’s death is a vivid and terrible example of the suffering that these patients and their families endure. The effort required to find, screen, diagnose, and treat these patients can be enormous, especially as the disease tends to spread in regions with limited access to appropriate care. Sometimes, as in Natacha’s case, the disease spreads because of surrounding violence; other populations are affected because of mass displacement or because they live in very remote locations. Q: As you mentioned, sleeping sickness is fatal if left untreated. As a physician, do you have adequate tools to detect and treat this disease? A: Tools exist to diagnose and treat the disease, but these techniques are cumbersome and require huge resources. To diagnose patients, you need centrifuges, microscopes, and very complex diagnostic tests along with specially trained technicians capable of interpreting the tests. To make a final diagnosis, every patient must have a lumbar puncture, an often very painful procedure which involves collecting spinal fluid from the patient’s back. These tests must often be performed in areas with very little infrastructure (such as lack of electricity) and poor sanitation. Patients in the first stage of the disease may be treated with seven days of daily injections, but treating the advanced stages of the disease is very difficult. Recently, new treatment protocols have been adopted which are safer and easier, but these treatments still have limitations. The current best treatment is a combination therapy, called NECT, developed by the Drugs for Neglected Diseases initiative. This treatment, although a dramatic step forward, still has significant side effects. It also cannot be emphasized enough that active screening and treatment is a constant need; while efforts into research and development for better diagnostic and treatment are essential, we must also ensure that there are people actively finding patients. Q: What kinds of new health tools and products do you think are necessary to help sleeping sickness patients in the Congo and around the world? A: We desperately need a rapid point of care diagnostic test, much like those used for malaria. We need an affordable test which does not require electricity or refrigeration, which could be read by staff with minimal training. This would dramatically increase our ability to screen for this disease. Also, while the disease is curable, current treatment requires intravenous medications administered under close supervision. As the disease tends to spread in areas with very limited medical care, it can be very difficult to give proper care to patients after they’re diagnosed. An oral pill treatment for both stages of the disease would revolutionize our ability to care for patients. New game-changing tools may be available in the near future. Two new oral drugs are now being tested in clinical trials. If they were found to be safe and effective, they could be given to patients who test positive with a rapid test. Just one test and one short-course oral treatment: this simplified protocol would make a huge difference in remote settings. On-going clinical trials with these new tools need to be fully-financed. Q: USAID currently does not invest in research to create new tools for sleeping sickness and other NTDs. How would support from USAID for NTD research help you as a physician? What difference would this support make in the lives of patients? We would save a lot of lives. We would prevent a significant amount of suffering. This disease causes excruciating pain, disability, psychosis, and ultimately death. Increased funding for programs to find patients and treat them, and for better and more patient-adapted diagnostic and treatment tools, would be game-changers. In addition to sleeping sickness, MSF/Doctors Without Borders works on several other of the most neglected of the neglected diseases, including Chagas, Buruli ulcer, and visceral leishmaniasis. These diseases affect very poor, marginalized people, often in some of the remote places. There is no lucrative potential market in developing medicines for people suffering from NTDs. As a result we have limited diagnostics and archaic, sometimes toxic treatments. If we really want to make a difference in the lives of people suffering from NTDs, we will not only need to increase funding for these specific diseases, we’ll have to seriously start looking at ways to create new types of incentives for research and development of new medicines .