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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. A study recently published in Science Immunology found that mice infected with malaria had shorter thigh bones than their control counterparts by ten percent, suggesting if the same effect takes place in human, children could suffer stunted growth due to malaria infection. Researchers found the malaria parasite left behind waste which killed the mice’s bones, resulting in the disruption of cells responsible for breaking down and rebuilding bones. The researchers found a possible solution, however. By giving the mice a derivative of vitamin D, they were able to stimulate more bone regrowth and suppress the breakdown of bone. Fifteen children have died in a rural South Sudanese village after one needle was used over the course of multiple days with a vaccine that was not stored properly. Almost 50 individuals fell ill after being inoculated on the same day, resulting in 15 deaths due to either sepsis or a related toxicity. According to the World Health Organization (WHO), the series of events were a result of a lack of training and qualification from the individual administering the vaccines, who failed to adhere to WHO immunization safety standards. South Sudan has been affected by a years-long civil war which has reduced the ability to carry out health care services. Immunization coverage has fallen from 77 percent before the conflict down to 44 percent.  The National Institute of Allergy and Infectious Diseases and the National Institutes of Health have sponsored a phase 1-2 Chikungunya vaccine trial at three different sites around the United States, including Emory University, Baylor College of Medicine, and the University of Iowa in Iowa City. The study will focus on three groups, one receiving a high-dose vaccine, one receiving a low-dose vaccine, and a control group. The results will help establish the best dosage and timing for the vaccine. If this trial is successful, the vaccine could become an important tool to combat the Chikungunya endemic in Eastern Africa and prevent the spread of cases within the United States.  

In this regular feature on Breakthroughs, we highlight some of the most interesting reads in global health research from the past week. CRISPR-cas9, a revolutionary gene editing tool, has been hailed a breakthrough device for microbiologists and geneticists—but not without certain reservations. In a study recently published in Nature, researchers attempting to target and edit genetic blindness in mice analyzed nucleotides—the single-unit building blocks for DNA—in the edited and control mice in order to evaluate success of the tool. CRISPR successfully targeted the alleles—or mutated nucleotides—causing blindness in the mice, but the editing also caused 1,600 off-target, unpredicted mutations, including 100 “large changes” in the DNA structure. This is important because single-nucleotide mutations are associated with many diseases, such as sickle-cell anemia, Huntington's disease, cystic fibrosis, and more. The researchers stressed that more needs to be done to study off-target mutations before CRISPR can be considered risk-free, but that the outlook for the tool is still positive, as any scientific tool has side effects. The science behind creating an effective vaccine that is capable of completely eradicating a disease is complex and hard-won. The only vaccine that has been used to successfully eradicate a disease in humans is the smallpox vaccine. Edward Jenner used cowpox, a similar but much less dangerous bovine variation of the disease, to create a successful vaccine against smallpox—and now scientists are going back to that methodology in an attempt to eradicate malaria. Scientists plan to genetically modify a rodent-variation of the malaria parasite to then use on healthy human volunteers. The hope is that the variation will result in a protective response in humans. The first phase of trials will begin in 2018 and will involve studying initial protection against the disease, with future phases focusing on continued efficacy of the vaccine. Motivated by a lack of awareness and funding for tuberculosis (TB), Médecins Sans Frontières, or Doctors Without Borders, launched a clinical trial, entitled  TB PRACTECAL, aimed at finding a new, improved treatment for drug-resistant TB. The trial, which officially launched in January 2017, is testing an innovative combination of TB drugs that will only need to be taken for six months. Current treatments for drug-resistant TB are physically and emotionally arduous, painful, and only successful about 50 percent of the time, which is why shorted, improved treatment regimens are needed. The first set of trial results is expected to be released within two years with final results within four. If successful, the organization hopes the new treatment will be rolled out in countries around the world and mark an important step forward in combatting the global epidemic.

As the 70th session of the World Health Assembly (WHA) winds to a close, memberstate delegates and civil society advocates are collectively taking a moment to catch their breath and reflect on an extremely eventful WHA. In a single week, the World Health Organization (WHO) gained a new Director-General—Dr. Tedros Gehbreyesus of Ethiopia—increased mandatory contributions from member states, and passed a landmark cancer resolution, just to name a few key decisions. In addition to these seismic shifts in the landscape of WHO, health research and development (R&D) was also featured prominently across a number of WHA agenda items, some of which saw highly consequential developments. Consultative Expert Working Group on Research and Development: Financing and Coordination (CEWG) The long-running CEWG process finally appears to have reached its end. After more than a decade of exploring approaches to support R&D for neglected diseases and actions to stand up a voluntary R&D-pooled fund at WHO, Assistant Director-General (ADG) Marie-Paule Kieny notified the Assembly that no further work would be done on either the pooled fund or the initial round of demonstration projects associated with it. Her comments came at the close of a lengthy session in which both member states and civil society organizations universally echoed the call for more resources to enable the fund and demonstration projects to operate effectively, as well as bemoaning the lack of contributions to date. In justifying the decision to cease work on these projects, ADG Kieny noted that no additional funding has been raised for the fund beyond the US$11 million pledged over three years ago. With that level of funding, the programs were simply not sustainable, she explained. On a positive note, the WHO Global Observatory on Health R&D—which was created to facilitate R&D coordination and guide prioritization—was spared the ax. While resources are not available to expand its database of R&D projects and data immediately, it is now operational and can be expanded at any time, as additional data and funding become available. Access to medicines As expected, the debate around access to medicines continued to rage at this year’s WHA. Although there was not a dedicated agenda item on follow-up to the United Nations Secretary General’s High-Level Panel on Access to Medicines (HLP) report, a number of countries, including Brazil, India, and Argentina, took every opportunity to raise the report in their remarks on other agenda items. In particular, agenda item 13.3 Addressing the Global Shortage of, and Access to, Medicines and Vaccines—which has historically focused more on supply chain issues—saw particularly heated debate, as well as intricate parliamentary maneuvering. India and Brazil led a concerted push, supported by South Africa, for the WHO Secretariat to initiate a consultation process that could result in WHA adopting “consensus language” on access to medicines. A number of European countries pushed back, noting that the WHA agenda item simply contained a report from the Secretariat for the Assembly to note, thus there was no need for consensus language. Implicit in this objection was a concern on the part of countries opposed to recommendations within the HLP report that any opening created to develop new language on access to medicines would invariably result in references to the HLP report. After extensive debate, including the intervention of WHO’s legal counsel, member states reached a compromise whereby the issue of access to medicines would be included on the agenda for January’s WHO Executive Board (EB) meeting, although that item would not specify discussion of the HLP report. South Africa further proposed a standing EB and WHA agenda item on access, which was vetoed by the United States. Beyond parliamentary maneuvering, the debate around access centered on many of the arguments that have become familiar to those following this issue over the years. On one end, civil society activists and some member states called for a wholesale rethink of the ways in which countries provide incentives for innovation, typically through some form of “delinkage,” wherein R&D costs are no longer related to the costs of the final product. At the opposite end of the spectrum, industry representatives and countries with significant pharmaceutical industries argued that intellectual property is not the primary barrier to access and that attention is better focused on supply chains and countries’ lack of domestic financing for health. Concluding thoughts Overall, this WHA featured one of the most packed agendas I have seen since I started tracking WHO. It is promising that, despite all of the competing agenda items, R&D remained a central focus of vigorous discussion and received significant attention. Even beyond the specific policy developments, it is clear that R&D and innovation remains a priority for the global health community.