Breakthroughs Blog: Research Roundup

United States President Barack Obama announced last week his intent to nominate Gayle Smith, senior director for Development and Democracy at the National Security Council, to lead the US Agency for International Development (USAID). Since joining the National Security Council in 2009, Smith has led the Obama administration’s global health work, including the Global Health Initiative and the Global Health Security Agenda. Ambassador Alfonso Lenhardt, deputy director of USAID has served as the acting director since Rajiv Shah stepped down from the post in February. The Universities Allied for Essential Medicines (UAEM)—a global network of students from more than 100 universities in 20 countries—released its second annual University Report Card: Global Equity & Biomedical Research, which evaluates leading US universities on their commitment to neglected disease research. The universities are ranked on innovation, which is measured by investments in neglected disease research; access, or efforts to license new products to ensure affordability and use; and empowerment, which relates to global health education at the university. Johns Hopkins University tops the list, receiving an A- overall, and is followed by Emory University, University of Washington, Harvard University, and University of California San Francisco. The Bill & Melinda Gates Foundation recently brought together nearly 100 representatives from foundations and governments to discuss the impact of Grand Challenges—calls for open innovation in which funding is offered to the best idea(s). The meeting focused on the difficulty of measuring the impact of Grand Challenges, and participants identified key barriers to evaluating and marketing the process. As failure is an inevitable but worthwhile part of innovation, participants have faced difficulties in “selling” Grand Challenges (i.e., to policymakers or donors). Additionally, the complexity and length of the pathway from research and development to utilization of a product makes it hard to pinpoint the exact impact of a Grand Challenge, and while short-term milestones can be assessed, it can take years or even decades before basic research is translated into a lifesaving product.

Scientists at the London School of Hygiene and Tropical Medicine have identified a mutation in Plasmodium falciparum—the parasite responsible for the majority of fatal cases of malaria—that makes the parasite more resistant to quinine and artemisinin, the leading antimalarials worldwide. The mutation was discovered in Kenya in 2013, where children were reported to still have the parasite in their blood after being treated for malaria. The mutation, found in the ap2mu gene, makes the parasite 42 and 32 percent more resistant to quinine and artemisinin, respectively. Congresswoman Martha McSally (R-AZ), Chairman of the House Committee on Homeland Security’s Subcommittee on Emergency Preparedness, Response, and Communications, called on the White House to name a “single coordinator for agencies responsible for responding” to public health crises or bioterrorist attacks in a recent hearing. Former Senator Jim Talent testified before the subcommittee, asserting that the Biomedical Advanced Research and Development Authority has been “chronically underfunded” and that a key lesson learned from the Ebola outbreak is that “it is too late to develop countermeasures after an outbreak or attack has happened.” The co-chairs of the Alliance for Biosecurity echoed this message in an op-ed published in The Hill last week. The authors, who serve as presidents of biotechnology companies Bavarian Nordic and Elusys Therapeutics, call for increased funding for biomedical research and development (R&D), specifically, consistent and multi-year investments in public-private partnerships. Stephen Ostroff, acting commissioner of the US Food and Drug Administration (FDA), does not have any major changes planned for the agency, and will continue former commissioner Margaret Hamburg's efforts to regulate tobacco products and enhance food safety. Dr. Ostroff also stated that the FDA is currently consulting the House Energy & Commerce Committee on the draft of the 21st Century Cures Act. During her final speech as commissioner, Hamburg expressed concern that the legislation could weaken the FDA’s rigorous drug approval process, leading to inefficient or even unsafe products on the market. Newt Gingrich, former United States representative (R-GA), called on Congress to double the National Institutes for Health (NIH) budget in an op-ed in the New York Times last week. Gingrich notes that 92 percent of NIH’s funding goes directly to research and that despite a 50 percent increase in grant applications, funding has either stagnated or declined each year since 2003. Gingrich asserts that “health is both a moral and financial issue” and that support for the NIH should be bipartisan. He admits that he is not usually a keen proponent of increased government spending, but that the government has a “unique” role to play in its ability to mobilize resources and due to the long-term health care savings realized by investing in research for treatments and cures.

India is expected to add all of the drugs currently used in the government’s HIV and AIDS and tuberculosis (TB) control programs to its list of essential medicines within the next six months. These drugs are currently available for free in public clinics; many patients, however, prefer private clinics due to perceived discrepancies in quality of care. The list, which was last updated in 2011, sets price caps to ensure that treatments remain affordable—paramount in a nation where more than 70 percent of the population lives on less than US$2 per day. China’s investments in health-related research and development (R&D) are expected to surpass US funding by 2023. The government of China has invested $1.1 billion in drug development since 2011, due to a policy mandate requiring increased funding for life sciences, including the pharmaceutical industry. Meanwhile, US funding for global health R&D has declined or plateaued each year since 2009. China is currently the world’s biggest manufacturer of active pharmaceutical ingredients and is becoming a leading site for contract research organizations, which support preclinical and clinical research for major pharmaceutical companies. The World Health Organization (WHO) issued a guidance document last week regarding a leading medicine for hepatitis C. The new guidance will inform the design of bioequivalence studies, which determine whether a generic is comparable to a patented product. The drug, sofosbuvir, is patented by Gilead Sciences and can cost up to $1,000 per pill. Proof of bioequivalence will be critical for manufacturers aiming to produce a generic drug and ultimately seek WHO prequalification for their product. In her final speech as commissioner of the US Food and Drug Administration (FDA), Dr. Margaret Hamburg defended the agency’s rigorous drug approval process. “Innovation doesn’t matter if the product doesn’t work,” she noted, highlighting the risks of “unsafe and ineffective medical products.” Hamburg’s remarks come as the House of Representatives Energy and Commerce Committee is in the process of developing legislation—known as 21st Century Cures—that calls on the FDA to expedite the regulatory process using its “breakthrough therapy” designation. This designation, which ensures an accelerated review and increased guidance from the FDA, was granted to 31 drugs in 2014. The 21st Century Cures legislation would allow the FDA to review products based only on preliminary clinical trial data and would allow companies to hold off on long-term safety and efficacy trials until after the product was on the market.

The National Institute of Allergy and Infectious Diseases (NIAID) announced last week that two experimental Ebola vaccines appear to be safe for widespread use, based on early results of clinical trials in Liberia. NIAID will now expand its trials to include as many as 1,500 volunteers, testing the vaccine candidates for efficacy. Volunteers will receive either the vaccine candidate developed by GlaxoSmithKline, another developed by NewLink Genetics Corp, or a placebo. Researchers will examine the immune response to the vaccines to see if participants develop antibodies against Ebola. None of the participants will be intentionally exposed to the Ebola virus. Scientists at NIAID have identified several genetic conditions that affect one’s risk for malaria, and which primarily manifest as red blood cell abnormalities. The researchers followed 1,543 children in Mali for three years, during which time they recorded 4,091 cases of malaria. Genetic disorders that either decreased the production of hemoglobin—the protein that transports oxygen in the blood—or led to the creation of hemoglobin C, an abnormal form of the protein, were associated with increased risk for malaria. Two genetic traits—HbAS and homozygous X-linked G6PD deficiency—appear to protect against malaria. The HbAS gene was not only associated with lower risk for malaria, but with less severe cases of malaria in children who contracted it. Researchers at Cornell University have mapped the genome of Ancylostoma ceylanicum, one of two species of hookworm that infect humans. The study identified 31,000 different genes, including those that are active in hookworms during infection. Scientists believe that as many as 70 of those genes could be promising targets for new drugs or vaccines, and plan to start testing experimental vaccines in hamsters this year.