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In this regular feature on Breakthroughs, we highlight some of the most interesting reads in global health research from the past week. Researchers at Rice University have developed a rapid malaria test that uses laser pulse—eliminating the need to draw blood. The test has been found safe in healthy humans and will soon be tested on humans with malaria symptoms. The White House has nominated Dr. Deborah Birx as the next Global AIDS Coordinator and to lead the President’s Emergency Plan for AIDS Relief (PEPFAR). Dr. Birx is currently the director of the Centers for Disease Control and Prevention’s Global AIDS Program and works on PEPFAR implementation at her agency. The US Agency for International Development and GHTC member Population Council have entered into an agreement to develop a non-antiretroviral-based microbicide to block HIV and other sexually transmitted infections. If successful, the partnership could offer a low-risk, easily accessible product for women in low- and middle-income countries. According to the results of an early-stage clinical trial, patients with multi-drug resistant (MDR) or extensively drug-resistant (XDR) tuberculosis (TB) could be treated by using stem cells taken from their own bone marrow. If successful, the treatment could be an alternative for patients with MDR- or XDR-TB who do not respond to conventional drug treatment options.

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 .

In this guest post, Dr. Mary Moran—executive director—and Dr. Penny Farrell—analyst—from Policy Cures shares key findings from the recently released G-FINDER survey. Nine million people still die each year from infectious diseases with lacking or insufficient treatments. The need for research and development (R&D) for new products has never been more urgent. But R&D costs money. The annual G-FINDER survey collects data about funding for neglected disease R&D from over two hundred organizations, including all major public, private, and philanthropic funders. The 2013 G-FINDER report shows that overall funding for neglected disease R&D was $3.2 billion, about half a billion dollars higher than in 2007, with the public sector providing two-thirds of this funding—around $2 billion. But beware—beneath this calm surface figure, there are some dangerous currents, and their direction is changing rapidly. 1. New trends in public funding revealed The headline public finding figure of around $2 billion has been maintained in 2012, but this figure masks marked new funding trends… Public funding for neglected disease R&D with and without the US. Click to enlarge chart. If we focus on the 2011-2012 section of this graph, it’s clear that US funding is rising, but non-US funding has ebbed. In other words, US funding is keeping neglected disease R&D afloat. In 2012, US funding increased by $86 million (up 6.4 percent)—the first increase seen by the US government since 2009. US investment has almost returned to its 2009 peak, when there was a large infusion of funds under the American Recovery and Reinvestment Act, created in response to the global financial crisis (GFC). But between 2009 and 2012, non-US high-income country government funding has decreased by 20 percent. That’s over $90 million lost. Some of this is cyclical. For example the United Kingdom’s Department for International Development disburses funds in generous 3-5 year cycles. But this does not explain all of this worrying trend, and without the United States, public funding for neglected disease R&D would be spiraling downwards. 2. Public funding for R&D needs to change tack 2007-2012 percent change in funding to basic research and product development. Click to enlarge graph. Another dangerous funding current is the post-GFC shift away from product development. Saving lives means not only doing basic laboratory research, but also ensuring that this research is translated into new medicines, vaccines, and tests that will save many of the 9 million lives lost each year to neglected diseases. But, post the GFC, governments have been redirecting their funding away from product development, which is often done abroad, in order to support basic research that can be done, and funded, in academic institutes at home. In the six years prior to 2012, there was a 27 percent increase in public sector funding for traditional basic research, but public funding for urgently needed product development over the same period shrank by nearly 2 percent. For diseases that mainly rely on public funding, such as HIV and AIDS, the result is a skewing of investment away from product development. 3. Funding for product development partnerships (PDPs) took its biggest blow yet in 2012 PDP funding 2007-2012. Click to enlarge graph. PDPs are a not-for-profit funding model that started around the year 2000. PDPs bring public and private organizations together to make products for diseases where there’s a market failure. To date, they have been the most productive product development sector—we have them to thank for nearly three quarters of the 18 new drugs and vaccines created for neglected diseases since 2000. In 2009, PDPs received $530 million—but since then their funding has plummeted by nearly one-third, and 2012 saw the biggest decrease yet. In early 2012, PDPs had at least 91 products for neglected diseases in clinical development. If this funding pattern continues, we will be forced to ask which projects to drop—will it be one working on more reliable treatments for tuberculosis, one developing a cure for African sleeping sickness, or one making a vaccine for malaria? The 6th annual G-FINDER report was launched in London on Dec. 11, 2013, and is available at http://policycures.org/gfinder.html. Conducted by Policy Cures and funded by the Bill & Melinda Gates Foundation, G-FINDER reports on global funding of R&D for new products for neglected diseases.

In this regular feature on Breakthroughs, we highlight some of the most interesting reads in global health research from the past week. The recently released annual G-FINDER survey found that total global funding for neglected disease research in 2012 amounted to $3.2 billion, which included an increase of $92.1 million—or 3.2 percent—over 2011 levels. Unfortunately, non-US government funding fell by 12.4 percent in 2012, with 11 governments either cutting or freezing funding. This drop was offset by an increase in US government funding by 6.4 percent from 2011. After returning from the 8th International Summit of Heads of Medicines Regulatory Agencies in Amsterdam, US Food and Drug Administration (FDA) Commissioner Margaret Hamburg wrote in FDA Voice about the increasing need for international regulatory agencies to work together. The FDA has granted GHTC member Medicines for Malaria Venture and pharmaceutical company GlaxoSmithKline Breakthrough Therapy designation for tafenoquine, an investigational medicine for the treatment and relapse prevention of Plasmodium vivax malaria. The Bill & Melinda Gates Foundation has named Susan Desmond-Hellmann, chancellor of the University of California, San Francisco, as its new CEO. A new article on SciDev.net examines how science is now more influential within the US Agency for International Development than it has been for the last two decades. Civil society groups are reacting to a set of demonstration projects recently selected by a World Health Organization expert panel to find new ways to fund health research for neglected diseases.