Breakthroughs Blog: NTDs

As the XIX International AIDS Conference continues in Washington this week, research for new tools to prevent and treat HIV and AIDS is standing out as a major theme. Below, we provide a summary of various sessions that have touched on research and product development topics. The US Congress and the Global AIDS Epidemic: Amid a group of protestors on Wednesday, former Sen. Bill Frist (R-TN) moderated a session with Sen. Chris Coons (D-DE), Sen. Mike Enzi (R-WY), Rep. Barbara Lee (D-CA), and Senator Marco Rubio (R-FL). When discussing her newly released bill—the Ending the HIV and AIDS Epidemic Act of 2012—Lee said that even in these “difficult” financial times, “we must move forward toward an AIDS-free generation.” Several other panelists stressed that a large part of achieving an AIDS-free generation will involve research for new products to prevent, test, and treat the disease. Speaking about recent scientific advances to create an HIV and AIDS cure, Rubio said that increased investment in innovation is critical to advancing these efforts. Coons echoed these comments, saying, “We need to double-down, we need to invest in basic science, invest in translational science, a vaccine, and new drugs. We need to innovate.” Rep. Barbara Lee (D-CA) at the International AIDS Conference. Photo credit: PATH. Effective Solutions to Combat HIV: Increasing Evidence of the Impact of Neglected Tropical Diseases on HIV Transmission and Disease Progression: In a session that examined the link between HIV and AIDS and neglected tropical diseases (NTDs), Giuseppe Pantaleo, chief of the Division of Immunology and Allergy at the University of Lausanne, said that 25 percent of people living with HIV worldwide are also co-infected with an NTD. He added that because of the high co-infection rate, there is a great need to focus on research, such as the clinical links between HIV and NTDs. Maria Aparecida Shikanai Yasuda from the University of Sao Paulo reiterated this point, saying that research is needed to develop new NTD drugs that are less toxic and more efficient. Astrid-Christina Koch, with the Delegation of the European Union to the United States, said that the second phase of the European and Developing Countries Clinical Trials Partnership will fund more stages of research. Currently the partnership covers research Phases II and III but will expand to cover Phases I through IV. This expansion could go a long way in addressing product development for NTDs and other global health diseases. Microbicides, pre-exposure prophylaxis (PrEP), and vaccines continue to take center stage at AIDS 2012. At a session to discuss The Role of Vaccines in Ending the Pandemic, panelists stressed that vaccines have proven to be one of the best, cost-effective global health solutions, and that research needs to continue to develop a vaccine for HIV and AIDS. Margaret McCluskey from the US Agency for International Development quoted Sen. John Kerry (D-MA), who has called an HIV and AIDS vaccine the “holy grail.” During a session on Wednesday on Microbicides and PrEP: Back to Basics, Ian McGowan from the University of Pittsburgh emphasized that a range of microbicide and PrEP formulations—from intra-muscular injections to vaginal and rectal products, rings, and oral pills—are needed to provide men and women worldwide with a range of HIV prevention choices that best fit their specific needs. Kim Lufkin is the GHTC’s communications officer.

In this regular feature on Breakthroughs, we highlight some of the most interesting reads in global health research from the past week. GHTC member the American Foundation for AIDS Research has issued a brief analyzing the human impact that widespread budget cuts, or sequestration, would have on US-funded global health programs. The brief concludes that a “proportionate cut in US Global Health Initiative funding through sequestration would have minimal impact on deficit reduction … The human impact of such cuts in US investments, however, would be devastating.” The director of the National Institutes of Health (NIH), Francis Collins, also discussed the effect of sequestration on biomedical research. He testified before the Health Subcommittee of the Energy and Commerce Committee, saying that sequestration “could do serious damage to the progress that we now see in medical research [at the NIH].” The European Medicines Agency has issued a recommendation that the Hexaxim childhood vaccine is safe for use outside of the European Union. Developed by Sanofi, the vaccine offers protection against six World Health Organization priority diseases. A recent article in USA Today looks at the potential of a widely used HIV/AIDS drug, Truvada, as a possible tool to prevent HIV transmission. In an effort to make foreign assistance more transparent, the US Agency for International Development has made its foreign assistance obligation and expenditure data available online on the Foreign Assistance Dashboard. An experimental four-in-one HIV and AIDS drug called Quad is found to be safe and effective after going through a three-phase clinical process. The Lancet published the research following a recommendation in May by a US Food and Drug Administration (FDA) advisory panel to approve Quad for previously untreated adults infected with HIV-1. A final decision is expected from the FDA by August.

In this regular feature on Breakthroughs, we highlight some of the most interesting reads in global health research from the past week. The White House Office of Science and Technology Policy and the Results for Development Institute have released new reports that examine incentives for global health research and development. Pharmaceutical company AstraZeneca and the Drugs for Neglected Diseases initiative have announced an agreement to collaborate on drug-compound screening for the neglected tropical diseases leishmaniasis, Chagas disease, and sleeping sickness. Todd Park, US chief technology officer, discusses innovation efforts undertaken by the US Government, including the Entrepreneurs in Residence Program at the US Food and Drug Administration. The Institute of Medicine has released a report on how to make clinical trials more efficient and effective.

Rachel M. Cohen, MPP, is the regional executive director of the Drugs for Neglected Diseases initiative (DNDi) North America. One billion people. That’s roughly how many people live in North America and Europe combined. And that’s also roughly how many people have at least one neglected tropical disease (NTD). NTDs, as defined by the World Health Organization, are a set of 17 infectious diseases that severely debilitate or kill people, most of whom are poor, marginalized, or in other ways vulnerable. NTDs such as sleeping sickness and kala azar are 100 percent fatal if left untreated. Parasitic-worm diseases like river blindness and elephantiasis can blind and deform people, young and old. Chagas disease almost exclusively infects those in Latin America, but kills more people in this region than malaria. The United States Government has played a major role in ensuring that patients with certain NTDs receive urgently needed treatments through the US Agency for International Development (USAID) NTD Program, while simultaneously being the largest funder of basic research for NTDs through the National Institutes of Health. This commitment has spanned several presidential administrations, receiving widespread support from both Democrats and Republicans alike in Congress. However, today US Government funding for NTDs is under threat. The recently announced US fiscal year (FY) 2013 budget request from the Obama Administration has slashed the USAID NTD Program budget, which was already miniscule at $89 million, by nearly 25 percent to $67 million. That is one of the single largest percentage cuts in global health. This isn’t trimming the fat—it’s cutting into muscle. While the USAID NTD Program has made an enormous impact for millions of people, the current initiative only focuses on five of the 17 NTDs. It does not fund diagnosis or treatment of the NTDs with the highest death rates —sleeping sickness, Chagas disease, and visceral leishmaniasis—and it does not fund any research into new health tools, unlike every other disease-specific program at USAID. USAID must urgently invest in research and late-stage product development for NTDs in order to bring new, life-saving health technologies to patients. Photo: PATH/Evelyn Hockstein. If the requested budget cuts go through, existing programs will have to be cut, and there will be no room for urgently needed program expansions. This will have direct consequences in terms of lives lost. The NTD Program budget line needs to be increased to President Obama’s FY 2012 request of $100 million. USAID must also urgently invest in a broader scope of diseases, including the aforementioned deadly NTDs, and in late-stage product development for NTDs in order to bring new, life-saving health technologies to patients. But why are new drugs needed? Can we not make do with the drugs already available to treat NTDs? In short, no. Here is just one reason. Today, filarial parasitic-worm diseases (onchocerciasis, also known as river blindness, and lymphatic filariasis, also known as elephantiasis) are treated with the drug ivermectin. But in people co-infected with another filarial disease, African eye worm (Loa loa, or loaisis), this treatment, alone or in combination with another called albendazole, can lead to brain damage—or even death. More than 14 million people are at high risk. For these patients, a new treatment is needed. My organization, the not-for-profit research and development organization Drugs for Neglected Diseases initiative (DNDi), is developing a drug called flubendazole that, if successful, would be safe and effective in these patients. Without such a drug, control and elimination of river blindness and elephantiasis will not be possible. USAID has publicly committed to contributing to the global goals of elimination and control of these and other NTDs, both through the US Global Health Initiative targets and, most recently, by signing on to the “London Declaration on NTDs” on January 30 at the high-level event “Uniting to Combat NTDs.” But rather than sustaining or expanding its commitment to NTDs, USAID appears to be retreating. For nearly all other communicable diseases, USAID funds late-stage product development (phase IIb or later), to bring needed innovations through the pipeline and, ultimately, to patients. This is the case for malaria, tuberculosis, and HIV and AIDS, for example. But not for NTDs. USAID must without delay begin investing into late-stage product development for NTDs. This approach would align NTDs with other disease-specific USAID programs that currently allocate a percentage of their funding for late-stage product development, and would be entirely consistent with USAID’s vision of promoting science and technology to advance international development. In order to bridge the gap between innovation and equitable access to the benefits of scientific research, late-stage product development is crucial. USAID must act now to prioritize the needs of the world’s most neglected patients who urgently need new drugs, diagnostics, and vaccines. Without new tools, these patients will fall through the cracks and die in silence.