A treatment for Niemann-Pick type C (NPC), an extremely rare and ultimately fatal neurodegenerative disease, performed no differently than placebo in a pivotal trial in 56 children and youths, its corporate sponsor announced on Tuesday. Perplexingly, though, the disease did not progress in either the treatment or placebo groups during the 1-year study, the company said. Normally, the condition, a result of impaired cholesterol metabolism, inexorably worsens, causing loss of balance, difficulty swallowing, seizures, and cognitive disabilities.
The drug, VTS-270, a doughnut-shaped sugar molecule called a cyclodextrin, “did not show a statistically significant separation from placebo,” Steven Romano, Mallinckrodt Pharmaceuticals’s executive vice present and chief scientific officer told investors on a conference call on Tuesday. “But importantly, neither did [patients in the active or placebo arms of the trial] show disease progression as would have been anticipated in the neurodegenerative condition over 52 weeks of observation.” The drug was given by spinal injection into the cerebrospinal fluid, which circulates to the brain.
The news—and the way Mallinckrodt, which has its U.S. headquarters in St. Louis, Missouri, delivered it—came as a shock to families in the NPC community, who learned of it when investors began to tweet about it. (The company did email a letter to NPC disease groups on Tuesday. Mallinckrodt, whose stock is publicly traded, added in a statement emailed to Science that securities laws prevented the company from notifying patients sooner.)
The letter slams the plan’s decision to stop paying for researchers to publish in so-called “hybrid” journals of scientific societies such as the American Chemical Society. Hybrid publications earn revenue from both reader subscription fees and article processing charges (APCs) paid by authors who want to make their papers immediately accessible. “Effectively Plan S would block access to exactly those journals that work with a valuable and rigorous peer-review system of high quality,” the letter says.
Robert-Jan Smits, OA envoy for the European Commission in Brussels and one of the architects of Plan S, says he has “enormous respect” for the work of learned societies, but no tolerance for some journals’ “sometimes outrageous” subscription fees. Hybrid journals were meant as a step to help subscription journals move toward full OA, he says, but they have endured as profitmaking ventures that rely on public funding, without a clear exit in sight.
The researcher at the center of a controversy roiling Cochrane, an international network of doctors and researchers, headquartered in London, that promotes evidence-based medicine, has been suspended as head of the Nordic Cochrane Centre in Copenhagen. Peter Gøtzsche, who was a founding member of Cochrane in 1993, has attracted attention for his outspoken critiques of pharmaceutical companies—and sometimes of Cochrane itself. In September, Cochrane’s governing board voted to remove him for “a consistent pattern of disruptive and inappropriate behaviours.” That decision led four other board members to resign in protest. Two weeks later, Gøtzsche said he would withdraw the Nordic Cochrane Centre from the international organization.
That was unacceptable to the board, however. In an interview with Science last month, Cochrane co-chair Marguerite Koster, a senior manager at Kaiser Permanente, said Cochrane CEO Mark Wilson would try to convince the Rigshospitalet in Copenhagen and the Danish government, which funds the Nordic Cochrane Center, to keep the center within the collaboration. Because he’s been ousted as a member, “Peter Gøtzsche no longer is the director of the Nordic Cochrane Center,” she argued. The board also took control of the website for the center and removed Gøtzsche’s statements about the case from it; he has since posted updates about the fight on his own website.
It’s unclear whether Cochrane’s lobbying has had any effect, but yesterday, the Rigshospitalet, which hosts the Nordic Cochrane Centre, announced it had suspended Gøtzsche. “We're striving to ensure that the Nordic Cochrane Centre continues as part of the international Cochrane Collaboration,” Deputy Chief Executive Per Jørgensen said in a statement. A spokesperson told Science the hospital would not give any further reasons for the suspension. Assistant Director Karsten Juhl Jørgensen has been appointed as acting head of the center, and the hospital has asked the University of Copenhagen to take over supervision of its graduate students.
Every year, after clicking “submit” on the final copies of their Ph.D. dissertations, thousands of scientists answer all sorts of questions—for example, about their age, sex, race, ethnicity, and career plans—as part of the U.S. National Science Foundation’s (NSF’s) Survey of Earned Doctorates (SED). The questionnaire has served as an annual census of U.S. doctoral degree-grantees since 1957 and provides useful demographic information, which can be used to track the success of diversity efforts. In the years ahead, the survey may start covering even more ground: During a meeting last week at NSF’s headquarters in Alexandria, Virginia, the agency said it plans to test the feasibility of adding questions about sexual orientation and gender identity.
NSF’s move was catalyzed by a letter arguing that comprehensive, nationwide data on LGBT (lesbian, gay, bisexual, and transgender) scientists and engineers are needed because the group experiences disadvantages and disparities that are akin to other underrepresented groups, such as racial and ethnic minorities and women. Only a handful of studies have examined LGBT representation in STEM (science, technology, engineering, and math) graduate programs and the scientific workforce, so there’s a clear need for more information, says Jonathan Freeman, an associate professor of psychology at New York University in New York City and the lead author of the letter. The letter writers used LGBT—rather than, say, LGBTQ—because it’s the most generally recognized term, and they didn’t want to confuse audiences who may not be as familiar with others. “In wanting to have a conversation with folks about these issues, oftentimes it’s a way to meet them where they’re at in terms of language,” says letter co-author Laura Durso, the vice president of the LGBT Research and Communications Project at the Center for American Progress in Washington, D.C.
The letter writers asked NSF to include questions about sexual orientation and gender identity on the SED, as well as two biennial surveys administered by the agency: the Survey of Doctoral Recipients and the National Survey of College Graduates (NSCG), both of which are designed to examine the career trajectories of STEM degree holders living in the United States. The letter was cosigned by 251 scientists, engineers, and legal and public policy scholars, as well as 17 scientific organizations (including AAAS, which publishes Science Careers).
If LGBT data were available from these surveys, “you’d have tons of people chewing on these data” to figure out if and where underrepresentation exists and to suggest interventions, Freeman says. Data collected by NSF could help us understand a whole host of questions about STEM’s LGBT community—“whether they’re here, whether they’re being retained, what their work trajectory is, whether they get paid as much”—notes Lauren Esposito, an assistant curator at the California Academy of Sciences in San Francisco and cofounder of the 500 Queer Scientists visibility initiative.
NSF is still in the early planning stages, so details are sparse regarding how it will move forward. But a spokesperson told Science Careers via email that changes to demographic data collection require a “lengthy, deliberate process involving extensive experimentation” in order to ensure that the agency generates “accurate, reliable data sets.” NSF plans to start with the biennial NSCG. The earliest that LGBT questions would be added is 2021 because the window for testing questions to add to the 2019 survey has passed.
Durso, who has worked to add sexual orientation and gender identity questions to federal surveys across the U.S. government, understands why implementing changes to the survey will take time. “There’s actually quite a bit of testing that has to happen,” says Durso, who holds a Ph.D. in clinical psychology and has studied the LGBT community. “These are federal government surveys; you want to do a deliberate and well thought out process.” For instance, it’s important to get the wording right to ensure that people fully understand the question that they’re being asked. Before making any changes to an ongoing survey, statisticians also want to confirm that adding certain questions won’t cause some people to refuse to answer the survey entirely—for instance, because they are offended by the questions.
Esposito is also concerned that collecting this type of information could be risky for the survey respondents themselves. Esposito—who hadn’t read the letter that was sent to NSF until Science Careers emailed her a copy—agrees that there’s a need for the data. “We should be informed and have tools at hand by which we can make policy and bring about change,” she says. But she worries about these kinds of data being in the hands of the federal government. “Sexual orientation and gender are not protected classes federally and in many states in this country,” she notes. When “you can be fired for that information, it seems risky and it seems like a risk that many people would have to think twice before taking,” she says.
Told about Esposito’s concerns, letter co-author Adam Romero—the director of legal scholarship and federal policy at the University of California, Los Angeles School of Law—acknowledged her concerns but expressed confidence that they were not a reason to scrap the survey questions. “In my experience, the federal government does a very good job to keep the personal demographic and other responses of survey takers highly confidential and protected,” he says. In addition, existing federal surveys that ask these kinds of questions usually give an option to decline to answer or to say that you don’t know. “For any particular person who may be uncomfortable, there’s no mandate to disclose their sexual orientation or gender identity.”
Policy decisions in higher education often hinge on information gleaned from federal surveys, notes Bryce Hughes, an assistant professor of education at Montana State University in Bozeman. So if NSF doesn’t collect data on sexual orientation and gender identity, then “we’ll miss opportunities” to make policy decisions that benefit LGBT communities, he says. “I’m just excited to see this moving forward.” Hughes wasn’t involved with the push to nudge NSF to add LGBT questions, but he understands the value of these kinds of data: Earlier this year, he published a study showing that sexual minorities are more likely to leave STEM undergraduate programs than their heterosexual peers.
Freeman—the author who spearheaded the letter—wants to get data into the hands of Hughes and other social scientists because he’s concerned that LGBT issues have been sidelined in STEM diversity discussions. “There is a tendency to see LGBT information … [as an] overly personal demographic detail … that should have no place in science and engineering,” Freeman says. But he says that people shouldn’t view it that way. “This is about a social identity that is like any other, like gender or race or ethnicity.” That’s why it’s important to have LGBT role models and adequate representation across STEM fields, he says.
“These are scientists and engineers, and so numbers speak and data speak, and I think having actual data on this would really change things,” Freeman says. “I think it would trigger a snowballing event of getting more people to study this issue and getting universities and federal funding agencies” to think about LGBT diversity initiatives, he says.
A number of states had climate- and energy-related measures on the ballot yesterday. Here is how two of the most prominent measures fared:
In Washington, carbon tax fails
Voters decisively rejected, by 56% to 43%, a push to become the first U.S. state to tax greenhouse gas emissions for the second time in 2 years. The defeat of Initiative 1631 came after a nearly $32 million opposition campaign funded chiefly by some of the world’s largest oil companies. BP America, the largest donor, pumped in more than $12 million.
For scientists, the new Democratic majority in the U.S. House of Representatives isn’t the only important result from last night’s midterm election. Although some races are still too close to call, and others are awaiting the counting of early votes and absentee ballots, here are some other highlights:
Four House incumbents active on science issues have been defeated, and a fifth is trailing.
Plan S, the open-access (OA) initiative launched by the European Commission and Science Europe in September, has gained two major new members. The Wellcome Trust and the Bill & Melinda Gates Foundation—two of the world’s largest private foundations that support research—announced today they are joining a consortium of 11 European funding agencies in requiring their funded research to be immediately free for all to read on publication.
The two new partners add a lot of funding muscle to the effort to require scientists to publish their papers in journals that make their content free to the public, instead of charging subscriptions. The existing Plan S coalition partners, represented by Science Europe, collectively spend about $8.7 billion on research. Wellcome, based in London, funds about $1.3 billion of biomedical research per year, whereas the Seattle, Washington–based Gates Foundation spends more than $1.2 billion on global health R&D.
The largest part of the policy change is that as of January 2020, Wellcome and Gates will no longer cover the cost of their grantees publishing in so-called hybrid OA journals, which have both subscription and free content. Most scientific journals now follow that hybrid business model, which allows authors to pay a fee if they want to make their articles OA. For the past decade, Wellcome has allowed its grantees to pay these fees, in part because it viewed them as a way to help publishers finance a switch in their business models to full OA. “We no longer believe it’s a transition,” says Robert Kiley, head of open research at Wellcome. “We’re looking to bring about a change where all research is open access.”
UPDATE: The National Science Foundation (NSF) said today that it is “currently reviewing possible future directions” for the Graduate Research Opportunities Worldwide (GROW). Amanda Greenwell, head of NSF’s Office Of Legislative and Public Affairs, said the agency “expects to make an announcement within the coming weeks” but that it “will not be publicly discussing the topic during the decision making process.” Greenwell also said the number of GROW recipients has declined over the past 3 years, from 158 in 2016 to 88 this year.
Here is our previous story from 1 November:
The U.S. National Science Foundation (NSF) has halted a program that each year allows hundreds of the nation’s best graduate students to work with experts in another country. And the agency isn’t saying why—or whether the program will resume.
Begun in 2013, Graduate Research Opportunities Worldwide (GROW) is a perk of NSF’s flagship Graduate Research Fellowship (GRF) program. Students already receiving the $34,000-a-year fellowship can apply for an additional $5000 GROW allowance to cover travel and living expenses incurred while working in one of 17 countries. NSF has budgeted for up to 400 GROW awards a year (2000 GRFs are chosen annually) for foreign collaborations lasting from 3 to 12 months.
The number of monkeys used in U.S. biomedical research reached an all-time high last year, according to data released in late September by the U.S. Department of Agriculture (USDA).
The uptick (see graph below)—to nearly 76,000 nonhuman primates in 2017—appears to reflect growing demand from scientists who believe nonhuman primates are more useful than other animals, such as mice or dogs, for testing drugs and studying diseases that also strike humans.
“I think the numbers are trending up because these animals give us better data. … We need them more than ever,” says Jay Rappaport, director of the Tulane National Primate Research Center in Covington, Louisiana, which houses about 5000 monkeys. The increase also comes amidst a surge in funding from the National Institutes of Health (NIH), which supports much of the nonhuman primate research in the United States.
Scientists who team up with the public to conduct research need to do a better job of including all segments of society. That’s one of the key recommendations in a new report on citizen science by the U.S. National Academies of Sciences, Engineering, and Medicine in Washington, D.C.
“Citizen science project designers must grapple with issues of equity, diversity, power, and inclusion,” says the report, written by a 12-member committee chaired by Rajul Pandya of the American Geophysical Union (AGU), also in Washington, D.C. “They face these issues even if they do not set out to address diversity in their project and even when they are not consciously aware that these factors are at play in their project.”
The phrase “citizen science” covers both projects in which scientists enlist the public—using volunteers for a bird census or to monitor air and water quality, for example—and those in which residents seek help with a problem that requires technical know-how, such as assessing how rising temperatures might affect their community. Millions of people participate in such efforts, which can educate nonscientists about technical topics, show how science can benefit society, and broaden the scope of a particular research project.