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Archive for July 2017

Science Policy Around the Web – July 25, 2017

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By: Allison Dennis B.S.

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Gene Drives

With Great Power There Must Also Come–Great Responsibility!

 

On the horizon of life-changing biotechnology up for ethical debate, nestled between CRISPR and whole genome sequencing, are gene drives, which have the potential to alter genes for better or for worse across generations. During sexual reproduction each of the two versions of a gene carried by a parent has a 50% chance of being inherited by each offspring. The frequency of each version of a gene across a population is influenced by rates of mutation, migration, genetic drift, and natural selection. Gene drives present the technology to circumvent these natural forces. By introducing molecular machines capable of damaging a particular version of a gene along with the version they prefer to the cells that give rise to eggs or sperm in an organism, scientists can shift the likelihood that their version will be inherited by that organism’s offspring from 50% to 100%. Upon fertilization the undesired gene will be damaged by the molecular machine and the desired gene will used as a template to repair the damaged copy, allowing two copies of the desired gene to be permanently introduced in the offspring and inherited by the next generation. Clever applications have been proposed to design mosquitoes resistant to malaria, mice unable to transmit lyme’s disease, or salmon able to grow to full size in half the time. More bold applications would use the technology to render female mosquitoes sterile, the ultimate insecticide. However, for each one of these beneficial applications exists the devastating opposite, which could be employed to accelerate the spread of disease. Altering population genetics of one species could accidentally devastate ecosystems.

U.S. defense organizations have taken notice of this powerful technology. DARPA, the Defense Advanced Research Project Agency, has launched the Safe Genes program in an effort to anticipate and address potential risks of introducing uncontrolled or undesired gene drives. The program awarded a collective $65 million to seven labs hoping to develop counter technologies including self-fizzling drives, chemical control methods, and gene drive vaccines. This summer, to delve deep into the intellectual discussion JASON, tackled the issue. This independent group of scientists, holding stellar academic records and top-secret clearances, meets once a year to address questions posed by the U.S. Department of Energy, Department of Defense, CIA, and FBI. However, their report is likely to be classified. (Ewen Callaway, Nature News)

Violence Against Women

Beginning to Understand the Nature of Intimate Partner Violence Through Data Curation

Careful evaluation of the nature of homicides of women has revealed that 55% result from intimate partner violence (IPV). The study conducted by the US Center for Disease Control (CDC) looked into the circumstances surrounding the deaths of 10,018 women over the age of 18 between 2003 and 2014 across 18 states. In addition to cases where the victims were intimate partners of the suspect, IPV-related homicides included cases where the female victims were friends, family, or those who intervened during an incident of IPV.

Nationwide political attention was drawn to the issue of IPV starting in the 1990s. The Violence Against Women Act was passed by Congress in 1994 and sought to legally define domestic violence as a crime external to the purview of private family matters. Research has revealed several risk factors associated with intimate partner violence, including threats with weapons, stalking, obsessive jealousy, sexual assault, and controlling behavior. However, the effectiveness of political and public health interventions remain unclear due to the overall decline in violence over the last decade and believed underreporting of individual incidence.

In an effort to more broadly understand the “who, when, where and how” surrounding violent deaths that occur in the United States, including those connected with IPV, the CDC created the National Violent Death Reporting System in 2002. By pooling information gathered by local law enforcement officers, coroners, medical examiners, and state agencies the CDC is hoping learn more about “why” so many violent deaths occur, towards the goal of developing and evaluating public health interventions. At its inception, funding only supported the participation of six states. However, involvement has been increasing from 17 states in 2006 and 42 in 2016 with the goal of eventually including all 50 states, U.S. territories, and the District of Columbia.

This study confirmed that homicide as a result of IPV occurs across all age groups and racial ethnic groups. However, young black and Hispanic women are disproportionately affected compared with white and Asian women of the same age group. Overall, black and indigenous women experienced significantly higher higher homicide rates, including non IPV related cases, than women of other races. Women died as a result of the use of firearms in 53.9% of all cases. While the “why” still remains unclear, this 15 year glance back sheds some light on the groups most affected by violence inflicted by their own partners, providing opportunity for targeted prevention. (Camila Domonoske, NPR)

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July 25, 2017 at 6:42 pm

Science For All – Effective Science Communication and Public Engagement

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By: Agila Somasundaram, PhD

Image: By Scout [CC0], via Wikimedia Commons

         In 1859, Charles Darwin published the Origin of Species, laying the foundation for the theory of evolution through natural selection. Yet more than 150 years after that discovery and despite a large volume of scientific evidence supporting it, only 33% of the American population believes that humans evolved solely through natural processes. 25% of US adults believe that a supreme being guided evolution, and 34% reject evolution completely, saying that humans and all other forms of life have co-existed forever. Similarly, only 50% of American adults believe that global climate change is mostly due to human activity, with 20% saying that there is no evidence for global warming at all. A significant fraction of the public believes that there is large disagreement among scientists on evolution and climate change (the reality being there is overwhelming scientific evidence and consensus), and questions scientists’ motivations. Public skepticism about scientific evidence and scientists extends to other areas such as vaccination and genetically-modified foods.

Public mistrust in the scientific enterprise has tremendous consequences, not only for federal science funding and the advancement of science, but also for the implementation of effective policies to improve public and global health and combat issues such as global warming. In her keynote address at the 2015 annual meeting of the American Society for Cell Biology, Dr. Jane Lubchenko described the Science-Society ParadoxScientists need society, and society needs science. How then can we build public support for science, and improve public trust in scientists and scientific evidence?

Scientists need to be more actively involved in science outreach and public engagement efforts. Communicating science in its entirety, not just as sensational news, requires public understanding of science, and familiarity with the scientific process – its incremental nature, breakthrough discoveries (that don’t necessarily mean a cure), failures, and limitations alike. Who better to explain that to the public than scientists – skilled professionals who are at the center of the action? In a recent poll, more than 80% of Americans agree that scientists need to interact more with the public and policymakers. But two major hurdles need to be overcome.

Firstly, communicating science to the public is not easy. Current scientific training develops researchers to communicate science in written and oral formats largely to peers. As scientists become more specialized in their fields, technical terms and concepts (jargon) that they use frequently may be incomprehensible to non-experts (even to scientists outside their field). The scientific community would benefit tremendously from formal training in public engagement. Such training should be incorporated into early stages of professional development, including undergraduate and graduate schools. Both students and experienced scientists should be encouraged to make use of workshops and science communication opportunities offered by organizations such as AAAS, the Alan Alda Center for Communicating Science, and iBiology, to name a few. Secondly, federal funding agencies and philanthropic organizations should provide resources, and academic institutions should create avenues and incentives, for scientists to engage with the public. Both students and scientists should be allowed time away from their regular responsibilities to participate in public outreach efforts. Instead of penalizing scientists for popularizing science, scientists’ outreach efforts should be taken into consideration during promotion, grants and tenure decisions, and exceptional communicators rewarded. Trained scientist-communicators will be able to work better with their institutions’ public relations staff and science journalists to disseminate their research findings more accurately to a wider audience, and educate the public about the behind-the-scenes world of science that is rarely ever seen outside. Engaging with the public could also benefit researchers directly by increasing their scientific impact, and influence research directions to better impact society.

While increasing science outreach programs and STEM education may seem like obvious solutions, the science of science communication tells us that it is not so simple. The goals of science communication are diverse – they range from generating or sharing scientific excitement, increasing knowledge in a particular topic, understanding public’s concerns, to actually influencing people’s attitudes towards broader science policy issues. Diverse communication goals target a diverse audience, and require an assortment of communicators and communication strategies. Research has shown that simply increasing the public’s scientific knowledge does not help accomplish these various communication goals. This is because people don’t solely rely on scientific information to make decisions; they are influenced by their personal needs, experiences, values, and cultural identity, including their political, ideological or religious affiliations. People also tend to adopt shortcuts when trying to comprehend complex scientific information, and believe more in what aligns with their pre-existing notions or with the beliefs of their social groups, and what they hear repeatedly from influential figures, even if incorrect. Effective science communication requires identifying, understanding and overcoming these and other challenges.

The National Academies of Sciences, Engineering, and Medicine convened two meetings of scientists and science communicators, one in 2012 to gauge the state of the art of research on science communication, and another in 2013 to identify gaps in our understanding of science communication. The resulting research agenda outlines important questions requiring further research. For example, what are the best strategies to engage with the public, and how to adapt those methods for multiple groups, without directly challenging their beliefs or values? What are effective ways to communicate science to policymakers? How do we help citizens navigate through misinformation in rapidly changing internet and social media? How to assess the effectiveness of different science communication strategies? And lastly, how do we build the science communication research enterprise? Researchers studying communication in different disciplines, including the social sciences, need to come together and partner with science communicators to translate that research into practice. The third colloquium in this series will be held later this year.

Quoting Dr. Dan Kahan of Yale University, “A central aim of the science of science communication is to protect the value of what is arguably our society’s greatest asset…Modern science.” As evidence-based science communication approaches are being developed further, it is critical that scientists make scientific dialogue a priority, and make use of existing resources to effectively engage with the public – meet people where they are – and bring people a step closer to science – why each person should care – so that ‘post-truth’ doesn’t go from being merely the word of the year to a scary new way of life.

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Written by sciencepolicyforall

July 22, 2017 at 11:27 pm

Science Policy Around the Web – July 21, 2017

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By: Rachel F Smallwood, PhD

Source: pixabay

Cancer

Engineered Cell Therapy for Cancer Gets Thumbs Up from FDA Advisers

A panel of advisers has recommended that the FDA approve chimeric antigen receptor T-cell (CAR-T) therapy for treatment of acute B-cell lymphoblastomic leukemia. The committee unanimously agreed that the risk to benefit ratio was favorable enough to proceed with approval of the drug (tisagenlecleucel), manufactured by Novartis. CAR-T therapy utilizes a patient’s own immune cells to find and attack cancer cells. In a recent trial in humans, 82.5% of patients went into remission following treatment with the drug; there have also been promising results from its use in glioblastoma treatment. The treatment would specifically be for pediatric and young adult patients who did not respond well to initial treatments or who relapsed from being in remission.

Despite have strong positive effects, there are potential risks posed by CAR-T therapy. In the study mentioned above, almost half of the patients experienced an inflammatory reaction called cytokine release syndrome. Although all of those cases were treatable, the condition can be life-threatening. Novartis also reported neurological problems. Other CAR-T trials have had several deaths due to brain swelling, but those were in adult populations and were some differences in the therapies.

The FDA often does take the recommendations of its advisers, but there is much to consider in this decision. It would essentially be approving a living drug that is individualized to each patient; the patients’ own blood cells are sent to a manufacturing center, where they are genetically engineered to target leukemia cells. The cell population is then allowed to proliferate, and the entire process takes around twenty-two days. This process presents a quality assurance and control problem to the FDA. However, the target population typically has a poor prognosis and very few options, so the panel considers the potential for increased survival and quality of life to be worth the risks. (Heidi Ledford, Nature News)

Stem-Cell Therapy

Unapproved Stem-Cell Treatments Touted on Federal Database Clinicaltrials.Gov

ClinicalTrials.gov is an online database, curated by the National Library of Medicine and the National Institutes of Health, that logs clinical studies occurring around the country and allows them to be searched by patients, family members, healthcare providers, and researchers. The information on the site is provided by the researchers or sponsors of the individual studies themselves. It allows patients and healthy people to become aware of opportunities to participate in medical research. These studies involve a wide range of treatments, including drugs, devices, behavioral therapies, and procedures.

A recent study found that the database is being abused by clinics advertising for stem cell trials. These trials target individuals looking for treatment for a variety of conditions, and all of them charge for participation. There are very few FDA-approved stem cell therapies, and most clinics that utilize stem cell therapies assert that they do not need FDA approval since they are practicing medicine and do not substantially alter the stem cells (although that is disputed).  Since the researchers themselves indicate in the database whether they need FDA approval, there is little oversight to ensure these studies are correctly representing the risks and benefits of their treatment.

Although a disclaimer was added this spring that informs visitors that the presence of a trial in the database does not indicate government endorsement of it, many people do not realize that they could potentially be participating in a for-profit procedure that does not have the proper oversight to ensure patient safety. In one such case, three women were blinded who paid to receive stem cell therapy for macular degeneration. Most legitimate research studies will not require payment for participation, although travel and lodging costs associated with participation may be incurred.

While many patients may receive treatment at one of these clinics without an adverse event or even with a positive result, critics of these types of clinics are calling for regulation of entries into the ClinicalTrials.gov system. They assert that a federal resource for medical research should not be used to advertise for for-profit clinics that are utilizing therapies that have not been studied or reviewed for safety and efficacy. (Laurie McGinley, Washington Post)

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July 21, 2017 at 10:08 am

The Economic Impact of Biosimilars on Healthcare

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By: Devika Kapuria, MD

          Biologic drugs, also defined as large molecules, are an ever-increasing source of healthcare costs in the US. In contrast to small, chemically manufactured molecules, classic active substances that make up 90 percent of the drugs on the market today, biologics are therapeutic proteins that undergo production through biotechnological processes, some of which may require over 1000 steps. The average daily cost of a biologic in the US is $45 when compared with a chemical drug that costs only $2. Though expensive, their advent has significantly changed disease management and improved outcomes for patients with chronic diseases such as inflammatory bowel disease, rheumatoid arthritis and various forms of cancer. Between 2015-2016, biologics accounted for 20% of the global health market, and they are predicted to increase to almost 30% by 2020. Worldwide revenue from biologic drugs quadrupled from US $47 billion in 2002 to over US $200 billion in 2013.

The United States’ Food and Drug Administration (FDA) has defined a biosimilar as a biologic product that is highly similar to the reference product, notwithstanding minor differences in clinically-inactive components, and for which there are no clinically meaningful differences between the biologic product and the innovator product in terms of safety, purity and efficacy. For example, CT-P13 (Inflectra) is a biosimilar to infliximab (chimeric monoclonal antibody against TNF-α) that has recently obtained approval from the FDA for use of treatment of inflammatory bowel disease. CT-P13 has similar but slightly different pharmacokinetics and efficacy compared to infliximab. With many biologics going off patent, the biosimilar industry has expanded greatly. In the last two years alone, the FDA approved 4 biosimilar medications: Zarxio (filgrastim-sndz), Inflectra (infliximab-dyyb), Erelzi (etanercept-szzs) and Amjevita (adalimumab-atto).

Unlike generic versions of chemical drugs (small molecules that are significantly cheaper than their branded counterparts), the price difference between a biosimilar and the original biologic is not huge. This is due to several reasons. First, the development time and cost for biosimilars is much more than for generic medications. It takes 8-10 years and several hundred million dollars for the development of a biosimilar compared to around 5 years and $1-$5 million for the generic version of a small molecule drug. With only single entrants per category in the US, biosimilars are priced 15-20% lower than their brand name rivals, which, though cheaper, still amount to hundreds of thousands of dollars. By the end of 2016, the estimated global sales from biosimilars amounted to US $2.6 billion, and nearly $4 billion by 2019. Estimates for the cost savings of biosimilars for the US market are variable; the Congressional Budget Office estimated that the BPCI (Biologics Price Competition and Innovation) Act of 2009 would reduce expenditures on biologics by $25 billion by 2018. Another analysis from the Rand Corporation estimated that biosimilars would result in a $44.2 billion reduction in biologic spending between 2014 and 2024.

In the United States, a regulatory approval pathway for biosimilars was not established till the Patient Protection and Affordable Care Act of 2010. However, biosimilars have been used in Europe for over a decade, and this has led to the development of strategies for quicker adaptation, including changes in manufacturing, scaling up production and adapting to local healthcare policies. These changes have led to a competitive performance of biosimilars in the European market, with first generation biosimilars taking up between 50-80% of the market across 5 European countries, with an expected cost savings of $15 to$44 billion by 2020. One example that demonstrates a significant discount involves the marketing of Remsima, a biosimilar of Remicade (infliximab). In Norway, an aggressive approach towards marketing of Remsima was adopted with a 69% discount in comparison to the reference product. After two years, Remsima has garnered 92.9% of the market share in the country.

The shift to biosimilars may be challenging for both physicians and patients. While safety concerns related to biosimilars have been alleviated with post marketing studies from Europe, there still remains a significant lack of awareness about biosimilars amongst healthcare providers, especially about prescribing and administering them. Patient acceptance remains an important aspect as well, with several patients loyal to the reference brand who may not have the same level of confidence in the biosimilar. Also, like with generics, patients may believe that biosimilars are, in some way, inferior to the reference product. Increased reporting of post marketing studies and pharmacovigilance can play a role in alleviating some of these concerns.

In 2015, the FDA approved the first biosimilar in the US, after which, it has published a series of guidelines for biosimilar approval, under the BPCA act, including demonstrating biosimilarity and interchangeability with the reference product. This includes a total of 3 final guideline documents and 5 draft guidance documents. Starting in September 2017, the World Health Organization will accept applications for prequalification into their Essential Medication list for biosimilar versions of rituximab and trastuzumab, for the treatment of cancer. This program ensures that medications purchased by international agencies like the UNICEF meet standards for quality, safety and efficacy. Hopefully, this will increase competition in the biosimilar market to reduce price and increase access to medications in low-income countries.

Both human and economic factors need to be considered in this rapidly growing field. Increasing awareness among prescribers and patients about the safety and efficacy of biosimilars as well as improving regulatory aspects are essential for the widespread adaptation of biosimilars.

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July 19, 2017 at 10:42 am

Science Policy Around the Web – July 7, 2017

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By: Leopold Kong, PhD

Food Policy

Food and Microbiota in the FDA Regulatory Framework

More and more probiotic food products, or microbiota-directed foods, claiming to “improve” the body’s microbiota have been hitting the shelves, with sales valuing over US$700 million in the US alone and US$36.6 billion globally this past year. However, there is little framework regulating their ingredients or guaranteeing the scientific accuracy of their health claims that has resulted in costly legal action. For example, in September 2009, Dannon settled a US$35 million consumer class action suit challenging the claimed health benefits in their ads. A similar class action suit against Procter & Gamble’s Align probiotic has been certified and set for Oct. 16, 2017. A paper recently published in the journal Science calls for greater clarity in policy regulating probiotic products. Importantly, the authors urge that probiotics should be clearly classified as a dietary supplement, a medical food, or a drug. If classified as a dietary supplement, probiotics can make claims on nutrient content and effect on health, but not on treatment, prevention or diagnosis of disease. If classified as a medical food, probiotics must contain ingredients that aid in the management of a disease or condition, with “distinctive nutritional requirements”, that is scientifically recognized. Finally, if classified as a drug, probiotics will require clinical trials to prove its medical claims. An alternative, and perhaps cheaper, way forward is to regulate probiotics as a kind of over-the counter medical food, requiring testing only for their active ingredients that can be used in a variety of products. (Green et al., Science)

Antibiotic Resistance

Untreatable Gonorrhoea on the Rise Worldwide

Over 78 million people are infected with gonorrhea each year, a sexually transmitted disease that has traditionally been treated effectively with anti-microbials. However, recently published data from 77 countries show that antibiotic-resistant gonorrhea is getting more pervasive and harder to cure. “The bacteria that cause gonorrhea are particularly smart. Every time we use a new class of antibiotics to treat the infection, the bacteria evolve to resist them,” said Dr. Teodora Wi, Medical Officer, Human Reproduction, at the WHO. The data found widespread resistance to ciprofaxacin, azithromycin, and even to the last-resort treatments, oral cefixime and injectable ceftriaxone. New drugs are under development, including a phase III trial of a new antibiotic, zoliflodacin, launched by the non-governmental organization Drugs for Neglected Diseases Initiative and Entasis Therapeutics, a biotech company in Waltham, Massachusetts. Better prevention through education on safer sexual behavior and more affordable diagnostics will also be needed moving forward. (Amy Maxmen, Nature News)

Maternal Health

U.S. has the Worst Rate of Maternal Deaths in the Developed World

A recent six-month long investigation by NPR and ProPublica has found that more women in the US are dying of pregnancy related complications than any other developed country. Surprisingly, this rate is increasing only in the US, which stood at ~ 26.4 deaths per 100,000 births in 2015, translating to nearly 65,000 deaths annually.  This is three times worse than for women in Canada, and six times worse than for women in Scandinavian countries. Reasons include older new mothers with more complex medical histories, unplanned pregnancies, which are the case half the time in the US, greater prevalence of C-sections, and the fragmented health system. This is in contrast with progress in preventing infant mortality, which has reached historic levels in the US. Better medical training for maternal emergency and more federal funding for research in this area may improve the situation for American mothers. (Nina Martin and Renee Montagne, NPR)

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Science Policy Around the Web – July 7, 2017

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By: Liu-Ya Tang, PhD

Source: pixabay

Autism

Is There Such a Thing as an Autism Gene?

Autism has become a global burden of disease. In 2015, it was estimated to affect 24.8 million people globally. Significant research efforts are underway to investigate the causes of autism. Autism is highly heritable – there is an 80 percent chance that a child would be autistic if an identical twin has autism. The corresponding rate is about 40 percent for fraternal twins.

However, is there such a thing as a single autism gene? Researchers haven’t found one specific gene that is consistently mutated in every person with autism. Conversely, 65 genes are strongly linked to autism and more than 200 others have weaker ties, many of which are related to important neuronal functions. Mutations in a variety of these genes can collectively lead to autism. The mutations could be from single DNA base pair, or copy number variations, which are deletions or duplications of long stretches of DNA that may involve many genes. Most mutations are inherited, but some mutations could also happen in an egg or sperm, or even after conception.

Besides genetic factors, maternal lifestyle and environmental factors can also contribute to autism. Exposure to air pollution during pregnancy or a maternal immune response in the womb may increase the risk of autism. While there is speculation on the link between vaccines and autism, it is not backed by scientific evidence.

Since both genetic and non-genetic factors play a role in the development of autism, establishing the underlying mechanism is complicated. There is no single specific test that can be used for screening autism. However, some tests are available to detect large chromosomal abnormalities or fragile X syndrome, which is associated with autism. (Nicholette Zeliadt, Washington Post)

STEM Education

New Florida Law Lets any Resident Challenge What’s Taught in Science Classes

A new law was signed by Florida Gov. Rick Scott (R) last week, and has taken effect starting July 1. The law requires school boards to hire an “unbiased hearing officer” to handle complaints about teaching materials that are used in local schools. Any county resident can file a complaint, and the material in question will be removed from the curriculum if the hearing officer thinks that the material is “pornographic,” or “is not suited to student needs and their ability to comprehend the material presented, or is inappropriate for the grade level and age group.”

There are different voices in the new legislation, which affects 2.7 million public school students in Florida. Proponents argue that it gives residents more right in participating in their children’s education. A sponsor, state Rep. Byron Donalds (R-Naples), said that his intent wasn’t to target any particular subject. However, Glenn Branch, deputy director of the National Council for Science Education, is worried that science instruction will be challenged since evolution and climate change have been disputed subjects. A group called Florida Citizens for Science asked people to pay close attention to classroom materials and “be willing to stand up for sound science education.”

Like the new law in Florida, the legislature in Idaho rejected several sections of the state’s new public school science standards related to climate change and requested a resubmission for approval this fall. Since the Trump administration began, there has been “a new wave of bills” targeting science in the classroom. To protect teacher’s “academic freedom,” Alabama and Indiana adopted non-binding resolutions that encourage teachers to discuss the controversy around subjects such as climate change. A supporter of the resolution, state Sen. Jeff Raatz (R-Centerville), told Frontline, “Whether it be evolution or the argument about global warming, we don’t want teachers to be afraid to converse about such things”. (Sarah Kaplan, Washington Post)

 

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Written by sciencepolicyforall

July 7, 2017 at 1:32 pm