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Posts Tagged ‘CRISPR

Science Policy Around the Web – September 26, 2017

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By: Rachel F Smallwood, PhD

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source: pixabay

Public health

Air Pollution Tied to Kidney Disease

A new study has reported a link between kidney disease and air pollution. Using data collected from over 2.4 million veterans, researchers were able to examine this relationship by consulting NASA and EPA pollution data. They found that glomerular filtration rate, a measure of kidney function that quantifies how much blood is passing through the kidneys to be filtered, decreased as levels of fine particulate matter less than 2.5 microns in diameter (PM 2.5) increased. These particles are small enough to enter the bloodstream where they then enter the kidneys. The authors estimate that 44,793 cases of chronic kidney disease and 2,438 cases of end-stage renal disease can be attributed to PM 2.5 levels that exceed EPA standards.

Kidney disease is just the latest disease that can be partially attributed to air pollution. Pulmonary conditions, cardiovascular disease, and stroke have been established as being contributed to and exacerbated by air pollution. Earlier this year, it was reported that air pollution also increased the risk of Alzheimer’s and dementia. With so many researchers reporting on such a wide variety of adverse health effects due to air pollution, it is becoming more imperative to address these issues and not lose ground in the struggle for cleaner air. Citizens and policymakers need to be educated about the importance and vigilant about the risks of air pollution. They need to work together to find ways to reduce pollution levels, not just for the planet for future generations, but for the health of today’s.

(Nicholas Backalar, The New York Times)

Biomedical Research

Scientists grow bullish on pig-to-human transplants

Speaking of kidney disease, the list of people in the United States waiting to receive a kidney transplant has almost 100,000 entries. One solution that scientists and physicians have long considered is xenotransplantation – harvesting donor kidneys and other organs from animals like pigs, which naturally have human-like anatomies. Until now, there have never been any demonstrations that have come close to being acceptable for trials in humans. However, that may be changing. A few research groups are reporting that they are close to moving into human trials and have begun early talks with the FDA.

They have been testing their methods by implanting pig kidneys and hearts into monkeys. The monkeys typically have an extreme immune response, which is what the groups have been attempting to ameliorate. Researchers have not been able to completely eliminate the immune response, but recently they reported that a transplanted kidney lasted over 400 days in a macaque before rejection. A transplanted heart lasted 90 days in a baboon before experimental protocol required that they stop the experiment. A previous experiment demonstrated the ability to keep a pig heart viable when implanted into an immune-suppressed baboon’s stomach for over two years, though it was just to test the biocompatibility and the baboon still had its autogenous heart.

This success is partially attributable to CRISPR technology, which has allowed scientists to remove portions of the pig DNA that intensify the immune response. The International Society for Heart and Lung Transplantation laid out bare minimum of requirements for moving xenotransplantation into human trials. They require at least 60% survival of life-supporting organs for at least 3 months with at least 10 animals surviving. Scientists also need to provide evidence that survival could be extended out to 6 months. These experimental results and minimum requirements are informative for expectations of xenotransplantation: it is not a permanent solution (at least not any time soon). However, they may provide temporary solutions that give patients more time while they are waiting on transplants from human donors. This is good news; over 7000 people died on an organ transplant waiting list in 2016. For those just trying to get through with dialysis or who just need a few more months before receiving their heart, these xenotransplants could mean the difference between life and death.

(Kelly Servick, Science)

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September 26, 2017 at 4:58 pm

Science Policy Around the Web – September 5, 2017

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By: Sarah L. Hawes, PhD

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Image: By Simon Caulton [CC BY-SA 3.0], via Wikimedia Commons

Gene therapy

FDA approves breakthrough gene therapy for childhood leukemia

Last week, the FDA approved use of gene therapy for the first time, to be used against resistant or relapsed acute lymphoblastic leukemia (ALL) originating in B-cells. The treatment, called Kymriah, was made by Novartis Pharmaceuticals in collaboration with University of Pennsylvania. It is a form of CAR T-cell therapy, in which a patient’s own immune cells are extracted and genetically modified to better identify and attack cancer-laden B-cells before being infused back into the patient.

Because the cellular feature which modified T-cells use to seek and destroy cancerous cells is also present on healthy B-cells, treatment carries risks including hypoxia, hypotension, and suppressed immune function. A life-threatening immunological reaction called cytokine release syndrome appears more commonly in adults, and may explain patient age-restriction (25 and under) on FDA’s approval.

For patients with otherwise intractable cancer, Kymriah may be a literal life-saver. In a recent clinical trial on 63 patients with drug-resistant or recurring ALL, Kymriah lead to remission in 83% of cases three months post-treatment.

While announcing approval of Kymriah, FDA Commissioner Scott Gottlieb asserted that the FDA is “committed to helping expedite the development and review of groundbreaking treatments that have the potential to be life-saving.” This has been substantiated for Kymriah in particular using both Priority Review and Breakthrough Therapy mechanisms. These speed FDA approval, thereby shortening pharmaceutical companies’ delay to profit, and have helped to drive activity in the promising CAR T-cell research arena in recent years.

Despite the success of these mechanisms in bringing a breakthrough cancer therapy to market faster, Novartis insists that a $475,000 price tag for one-time treatment is conservative considering the high cost of drug development and low number of candidate patients. This sobering figure is made worse by the fact that some cases indeed recur several months following Kymriah. Novartis is currently working with Medicare on a plan for outcome-based pricing, so that the pharmaceutical company is only paid if patients respond to the therapy.

(FDA News Release; Jessica Glenza, The Guardian)

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Image: By NOAA, via Wikimedia Commons

Emergency preparedness

Hurricane Harvey illustrates the importance of disaster preparedness for research institutions (again) 

The US National Academies of Sciences, Engineering, and Medicine released a report just last month highlighting weaknesses in disaster preparedness in biomedical research facilities, and issuing recommendations to enhance the resilience and continuity of research in the face of adversities including natural disasters, fires, and cyber threats. Costs of unpreparedness are high. In In 2012 Hurricane Sandy is estimated to have caused NYU more than $20 million in research equipment, and killed thousands of mice housed in New York laboratories, including many transgenic strains which took decades to develop and existed nowhere else on earth.

Hurricane Harvey’s toll on the scientific community is similarly, incalculably high. University of Houston’s infant rhesus monkeys ran out of formula and had to be weaned early. Loss of refrigeration capability jeopardized precious tissue and reagents, not to mention rendering some agents hazardously unstable. The University of Texas at Austin Marine Science Institute lost the roof off a microbial-ecology lab, forcing trainees to abandon their work and move to alternative institutions.

Some fared better due to advanced planning. For instance, Baylor College of Medicine was protected from Harvey by a wall installed around their campus after 2001 Tropical Storm Allison cost them 60,000 breast-cancer samples and thousands of laboratory animals. To support less fortunate Texas researchers, the broader scientific community has used hashtag #SciHelpTX on Twitter to advertise sharable resources such as open lab space, computers, and animal colony husbandry.

Hopefully Harvey has driven home the message that preparedness is a necessary investment going forward. Enacting preparations remains up to individual institutions’ policies; a list of recommendations by the National Academies can be found here.

(Emma Marris, Nature News)

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Image: Wikimedia Commons

Gene therapy

Correction of a pathogenic mutation in human embryos? Maybe! The exploration continues

An August 2017 publication in Nature reports success using CRISPR-Cas9 to delete targeted sections of gene responsible for producing familial hypertrophic cardiomyopathy from human zygotes. The study, led by Dr. Shoukhrat Mitalipov, involved collaboration between the Salk Institute, Oregon Health and Science University (OHSU) and Korea’s Institute for Basic Science. By introducing a short-lived version of CRISPR, an enzyme, and a repair template into a healthy egg prior to fertilization but simultaneously with sperm bearing the targeted genetic defect, authors believe they ensured the gene excisions would take place early, and be carried throughout all following cell divisions. They believe this technique avoids unintended edits and mosaicism, in which both diseased and repaired cells exist side by side in the organism. The team found both the deleted genes and the template for replacement absent, and believe the genome repair used the healthy genes from the egg. They suggest this is due to certain evolutionary resiliencies associated with early stage eggs.

Other researchers responded by emphasizing the remaining uncertainties and importance of maintaining a focus on research as opposed to pushing too quickly toward application of germline editing techniques with the potential for producing heritable genetic changes. Complex ethical questions remain around germline editing even should techniques be perfected for any specific section of the genome. This research could not receive government funding due to the creation and destruction of human embryos.

Within three weeks, a preprint article questioned the likelihood of the egg serving as a template for repair of the genome’s deleted genes. The authors state that following fertilization the egg and sperm DNA are not in close enough contact for such borrowing, and propose two alternative scenarios: Either the egg failed to incorporate the sperm DNA which is sometimes seen with in vitro fertilization, or failed to replace the missing segment at all. Either would have resulted in an absence of the targeted paternal or template genes. Mitalipov has promised to respond point by point.

(Kelly Servick, Science Magazine)

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September 5, 2017 at 5:29 pm

Science Policy Around the Web – June 10, 2017

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By: Allison Dennis, BS

Source: pixabay

Animal Testing

Lack of Clarity Puts Chemical Safety and Animal Welfare at Odds

In the lineup of American stereotypes, the health-nut who cares about the chemicals in his shampoo is often the same person who cares if that shampoo was tested on animals or not. However, a bill signed June 22, 2016, known as the Frank R. Lautenberg Chemical Safety for the 21st Century Act, may be placing those two views at odds. The bill requires the U.S. Environmental Protection Agency (EPA) to implement a risk-based process to evaluate the safety of chemical substances currently being used in the marketplace and approve the use of new chemicals before their introduction. The bill was passed with bipartisan support and offered EPA the new-found power to fully regulate the use of well-known carcinogens like asbestos.

Yet the pathway forward for the EPA is daunting. More than 62,000 substances find their way into and onto our bodies through the products we use and our environment. While many of these substances have become associated with disease over time, how can the EPA certify the risks associated with different exposures to varying amounts of each substance on such an extensive list? The Act itself suggested that once the EPA has evaluated the existing information on the 62,000 substances currently in use, it spend the next twelve months triaging chemicals according to their potential risk. Next, the highest priority chemicals will be evaluated on a three-year deadline to develop knowledge of their toxicity and guidelines for their regulation. Ultimately, by clearly cataloging the risk of common chemicals the Frank R. Lautenberg Chemical Safety for the 21st Century Act promises to greatly reduce the amount of animal testing needed in the long-term.

In the meantime, however, the companies that use to-be-regulated substances in their products may be inclined to undertake independent toxicity testing, collecting enough data to guarantee that their favorite substances meet the low-risk criteria and avoid a drawn-out evaluation. Defining toxicity requires careful experimentation, which can sometimes be carried out in human cells outside of the body, but often require evaluation in animals. Animal rights groups like the Human Society find concern with the lack of transparency in the pre-prioritization process. They fear the eagerness of companies to provide data without any clear guidelines about how that data will be evaluated or what substances will require extensive evaluation could result in extensive and unnecessary animal testing. Further they suggested that the EPA require any new pre-approval data obtained by companies to be collected using non-animal methods. (Maggie Koerth-Baker, FiveThirtyEight)

CRISPR

Small Study may Reveal Big Concerns over CRISPR-Based Therapy

A one-page letter published in Nature Methods last week reports unexpectedly high levels of unintended changes to the genomes of mice that underwent a CRISPR-based therapy. Since it’s renaissance as a therapeutic tool in 2012, CRISPR has occupied the imaginations of scientists, doctors, patients, investors, and ethicists. CRISPR technology provides a relatively straight-forward and reproducible means to gene editing on the cellular level, but its applications to create heritable mutations in the human germ line is on hold until more is understood about the long-term effects such treatments would have.

The original study sought to explore potential long-term effects of germline manipulation by CRISPR in a mouse model. Guide RNA along with the Cas9 enzyme were injected into mouse zygotes, which introduced a correction in a mutation in the rd1 gene of otherwise blind mice. Initiating this change before the first cell division enabled this corrected mutation to be inherited by all cells arising in the developing mouse, consequently restoring the ability for the eyes to develop normally. In a follow-up experiment described in their one page letter, the researchers looked for mutations in the genomic DNA of two CRISPR-treated adult mice compared with a control mouse, revealing over 2,000 unintended mutations following CRISPR treatments. None of these mutations appeared to affect the mice, suggesting that deep genomic sequencing may be required to reveal unanticipated changes in an outwardly healthy mouse. Further, the nature of these unintended mutations offered few clues explaining how they might have occurred.

This result stands in contrast with other reports quantifying the extent of these unintended changes, which found CRISPR to be highly specific. While the CRISPR-Cas9 system has been observed to sometimes alter off-target regions of the genome, this activity can usually be curbed through the careful design and evaluation of the guide RNA. The limitations of this small study have been discussed extensively since its publication. However, the findings have sparked the need for further investigation into the long-term-whole-animal effects of germline-editing by CRISPR. As human germline-editing creeps closer to reality, the FDA will be tasked with developing an entirely new means of evaluating the safety of such technologies (Megan Molteni, Wired)

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June 10, 2017 at 11:33 am

Science Policy Around the Web – February 24, 2017

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By: Alida Palmisano, PhD

Source: usda [Public domain], via Wikimedia Commons

Public Access to Data

Lawsuit Aims to Force USDA to Repost Scrubbed Animal Welfare Records

“Put the records back on the internet.”

An article published in Science discusses a lawsuit filed on February 13 against the U.S. Department of Agriculture (USDA) by an animal law expert at Harvard University. According to the plaintiffs, USDA violated the federal Freedom of Information Act (FOIA) when it removed thousands of animal welfare inspection reports and other records from a publicly accessible website. USDA generated records that document animal facility inspections, enforcement actions, animal censuses, and other information collected by the agency in the course of enforcing the federal Animal Welfare Act.  The law covers animals in more than 7800 facilities, including zoos, roadside circuses, and research laboratories at government agencies and academic medical centers.

The decision to remove the public access to these records may have been a response to a lawsuit involving another law, the Horse Protection Act. The plaintiffs in a 2016 Texas lawsuit accused USDA of violating their rights under the Privacy Act by posting inspection documents required by the Horse Protection Act. A resulting USDA review of all its public postings led the agency to scrub from its website documents generated under both the Horse Protection Act and the Animal Welfare Act.  In the future, the agency announced, people who want access to those records will need to file a FOIA request. The agency’s most recent FOIA report states that it takes an average of 94 days for the agency to respond to a simple FOIA request and 234 days on average for more complicated requests.

In February 13’s lawsuit, the plaintiffs invoke a section of FOIA that requires agencies to make publicly available electronically all records that it has released under FOIA which “because of the nature of the subject matter, the agency determines have become or are likely to become the subject of subsequent requests for substantially the same records.” (Meredith Wadman, ScienceInsider)

Science and Immigration

Grad Students, Postdocs with U.S. Visas Face Uncertainty

While U.S. courts are busy handling President Donald Trump’s travel ban on immigration from seven majority-Muslim countries, the temporary shut down of the executive order, the appeal to reinstate the travel ban, the rejection of the immediate restoration of the ban, and more appeals and rulings, graduates and postdoctoral students already in the United States are weighing their options and trying to plan rationally in an unpredictable and fluid situation.

Many scientists in the U.S. are on student or other working visas. All these visas may not be renewable, depending on future executive orders and regulations. The dilemma “simply ruins their future. It’s a catastrophe,” says a Yemeni biologist who is on a university faculty on an H-1B, a 3-year visa for professionals. For years, lawmakers in Washington have tried to reform abuses of visa regulations by companies using visas to bring workers to the U.S. to learn the ropes, and then send the trained workers to other countries where the job can be done cheaply. The H-1B system is contentious: on one side labor advocates want the exploitation of the H-1B system to stop supporting an outsourcing business model. On the other hand, tech companies like Google and Facebook say they can’t get enough visas for top foreign talent, as the cap on the number of H-1Bs issued every year means that sometimes foreign graduates from top U.S. universities, places like the Massachusetts Institute of Technology and the University of California, Berkeley, can’t get one. The travel ban already has harmed the top universities in the U.S., stranding students, faculty and scholars abroad, and making foreign schools more attractive to some of the world’s brightest students.

In papers filed in Brooklyn federal court, the schools (that include Columbia, Duke, Harvard, Johns Hopkins, Princeton, Stanford, Yale, Massachusetts Institute of Technology and several more) said that the order blocking travel from seven predominantly Muslim countries threatens their abilities to educate future leaders from every continent. They said the executive order has “serious and chilling implications” and that the ban “casts doubt on the prospect and value of studying and working here for everyone,” the papers said. (Meredith Wadman, Richard Stone, Science)

Genetic Engineering

US Science Advisers Outline Path to Genetically Modified Babies

“Scientists should be permitted to modify human embryos destined for implantation in the womb to eliminate devastating genetic diseases such as sickle-cell anaemia or cystic fibrosis — once gene-editing techniques advance sufficiently for use in people and proper restrictions are in place. That’s the conclusion of a 14 February report from the US National Academies of Science, Engineering, and Medicine.”

The report follows a 2015 National Academies summit between scientists, ethicists, legal experts and patient groups from around the world. At the time of the meeting, given the outstanding scientific, ethical and legal questions surrounding the issue, the organizers concluded that scientists shouldn’t yet perform germline editing on embryos intended for establishing a pregnancy. However, the organizers also stated that altering human embryos for basic research was acceptable.

The latest iteration of this ongoing CRISPR debate moves the bar a little further. The report recommends restricting the technique to severe medical conditions for which no other treatment exists. Eric Lander, president of the Broad Institute of MIT and Harvard, said, “It’s a very careful, conservative position that’s just a little bit beyond an absolute bar.” In the report, the committee also called for international cooperation, strict regulatory and oversight framework, public input into decisions and long-term follow-ups of children who have edited genomes. The report adds that for now, genome editing should not be used for human enhancement, such as improving a person’s intelligence or giving them super-strength.

The report drew immediate criticism from a California-based non-profit organization called the Center for Genetics and Society. “This report is a dramatic departure from the widespread global agreement that human germline modification should remain off limits,” said Marcy Darnovsky, executive director of the center. “It acknowledges many of the widely recognized risks, including stigmatizing people with disabilities, exacerbating existing inequalities, and introducing new eugenic abuses. Strangely, there’s no apparent connection between those dire risks and the recommendation to move ahead.” (Sara Reardon, Nature)

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February 24, 2017 at 11:23 am

Science Policy Around the Web – February 17, 2017

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By: Thaddeus Davenport, PhD

Source: pixabay

CRISPR

Decision in the CRISPR-Cas9 Patent Dispute

This week, Heidi Wedford from Nature News reported that the United States Patent and Trademark Office (USPTO) made a decision on the disputed patents for the gene editing technology known as CRISPR-Cas9 in favor of the Broad Institute of MIT and Harvard. The CRISPR-Cas9 system has been widely publicized, and this publicity is arguably not out of proportion with the potential of this technology to simplify and accelerate the manipulation of DNA of both microbial (prokaryotic) and higher order (eukaryotic) cells for research and therapy. A simplified, programmable version of CRISPR-Cas9 for use in gene editing was initially described by Charpentier and Doudna, and it was rapidly translated for use in eukaryotic cells by Zhang and colleagues at the Broad Institute in parallel with Doudna, Charpentier, and others.

The USPTO decision follows a dramatic and ongoing dispute over whether the patent application submitted by the University of California on behalf of Doudna and Charpentier – which was submitted before that of the Broad Institute, and described the technology in broad terms as a method of cutting desired DNA sequences – was sufficient to protect the CRISPR-Cas9 intellectual property when the Broad Institute later filed a fast-tracked patent application describing the use of CRISPR-Cas9 for use in eukaryotic cells. Because the Broad Institute’s application was expedited, it was approved before the University of California’s application. In January of 2016, the University of California filed for an ‘interference’ proceeding, with the goal of demonstrating to the USPTO that Doudna and colleagues were the first to invent CRISPR-Cas9, and that the patent application from the Broad Institute was an ‘ordinary’ extension of the technology described in the University of California application.

On February 15th of this year, the USPTO ruled that the technology described in the Broad Institute’s application was distinct from that of the University of California’s. The importance of this decision is that the patents granted to the Broad Institute for the use of CRISPR-Cas9 in mammalian cells will be upheld for now. It also creates some complexity for companies seeking to license CRISPR-Cas9 technology. Because of the overlapping content of the CRISPR-Cas9 patents held by the University of California and the Broad Institute, it is possible that companies may need to license the technology from both institutions. The University of California may still appeal the USPTO’s decision, but this is a significant victory for the Broad Institute for the time being. For many scientists, this dispute is a dramatic introduction to the inner workings of the patent application process. We would do well to familiarize ourselves with this system and ensure that it works effectively to accurately reward the discoveries of our fellow scientists and to facilitate the transfer of technology to those who need it most, without imposing undue economic burden on companies and consumers. (Heidi Wedford, Nature News)

Scientific Publishing

Open Access to Gates Foundation Funded Research

Also this week, Dalmeet Singh Chawla reported for ScienceInsider that the Bill and Melinda Gates Foundation had reached an agreement with the American Association for the Advancement of Science (AAAS) that will allow researchers funded by the Gates Foundation to publish their research in the AAAS journals Science, Science Translational Medicine, Science Signaling, Science Immunology, and Science Robotics. This agreement follows an announcement in January in which the Gates Foundation decided that research funded by the foundation would no longer be allowed to be published in subscription journals including Nature, Science, and New England Journal of Medicine, among others, because these journals do not meet the open access requirements stipulated by the new Gates open-access policies. The new Gates Foundation policy requires its grant recipients to publish in free, open-access journals and to make data freely available immediately after publication for both commercial and non-commercial uses. A similar policy is being considered by the nascent Chan Zuckerberg Initiative.

In the agreement with AAAS, the Gates Foundation will pay the association $100,000 in order to make Gates-funded published content immediately freely available online. Convincing a journal as prominent as Science to make some of its content open-access is a step in the right direction, but it is perhaps more important as a symbol of a changing attitude toward publishing companies. Michael Eisen, co-founder of the Public Library of Science (PLoS) open-access journals, was interviewed for the ScienceInsider article and noted, “[t]he future is with immediate publication and post-publication peer review, and the sooner we get there the better.” This sentiment seems to be increasingly shared by researchers frustrated with the hegemony of the top-tier journals, their power over researchers’ careers, and the constraints that subscription-based journals impose on the spread of new information. Funding agencies including the Gates Foundation, Howard Hughes Medical Institute, and the National Institutes of Health are in a unique position to be able to dictate where the research they fund may be published. A collective decision by these agencies to push the publishing market towards an improved distribution of knowledge – through open-access publishing and post-publication peer review – and away from the historical and totally imagined importance of validation through high-tier journal publication would enrich the scientific ecosystem and accelerate innovation. In this regard, the efforts by the Gates Foundation are laudable and should be extended further. (Dalmeet Singh Chawla, ScienceInsider)

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February 17, 2017 at 12:44 pm

Science Policy Around the Web – August 26, 2016

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By: Leopold Kong, PhD

Adipose Tissue  Source: Wikipedia Commons, by Blausen.com staff, “Blausen Gallery 2014“.

Health Policy

Is there such a thing as ‘fat but fit’?

Nearly 70% of American adults are overweight or obese, raising their risk for health problems such as heart disease, diabetes, and high blood pressure. However, about a third of obese individuals appear to have healthy levels of blood sugar and blood pressure. Whether these ‘fat but fit’ individuals are actually “fit” has been controversial. A recent study published in Cell Reports has sought to dissect differences in the fat cells of the ‘unfit’ obese versus the ‘fit’ obese using tools that probe the patterns of genes being turned on or off. Fat from non-overweight people were also examined in the study. Interestingly, fat of non-overweight individuals and obese individuals differed in over 200 genes, regardless of ‘fitness’. However, the fat of ‘fit’ versus ‘unfit’ obese individuals only differed in two genes. Dr. Mikael Rydén, the lead author of the study commented: “We think that adds fuel to the debate. It would imply that you are not protected from bad outcomes if you are a so-called fit and fat person.” The study also highlights the complexity of fat’s influence on health, and raises the possibility of ‘fat’ biopsies. For example, fat from normal weight individuals following an unhealthy lifestyle may have marked differences that are diagnostic of future obesity. With the rising cost of treating chronic diseases associated with being overweight, further studies are warranted. (Lindzi Wessel, Stat News)

Biomedical Research

Half of biomedical research studies don’t stand up to scrutiny

Reproducible results are at the heart of what makes science ‘science’. However, a large proportion of published biomedical research appears to be irreproducible. A shocking study by scientists at the biotechnology firm Amgen aiming to reproduce 53 “landmark” studies showed that only 6 them could be confirmed. The stakes are even higher when it comes to pre-clinical cancer research. In fact, they are $30 billion higher, according to a recent study, suggesting that only 50% of findings can be reproduced. Primary sources of irreproducibility can be traced to (1) poor study design, (2) instability and scarcity of biological reagents and reference materials, (3) unclear laboratory protocols, and (4) poor data analysis and reporting. A major stumbling block may be the present culture of science, which does not reward publishing replication studies, or negative results. Higher impact journals generally prioritize work that demonstrates something new and potentially groundbreaking or controversial. When winning grant money and academic posts hinges on impact factor, reproducibility suffers. However, with such high potential for wasting substantial funds on medically significant areas, radical changes in science policy towards publishing, peer review and science education is urgently needed. The recent reproducibility initiative aiming “to identify and reward high quality reproducible research via independent validation” may be a step in the right direction. However, a paradigm shift in scientists’ attitudes towards what constitutes important research might be necessary. (Ivan Orannsky, The Conversation)

Biotechnology

In CRISPR fight, co-inventor says Broad Institute misled patent office

The intellectual property dispute over the multibillion-dollar CRISPR gene editing technology has grown increasingly heated in the last months. With the FDA giving the go-ahead for the first U.S. clinical trial using CRISPR and with China beginning a clinical trial this month using this technology, the tension is high. On one side of the dispute is University of California’s Jennifer Doudna whose initial work established the gene-editing technology in a test tube. On the other side is Broad Institute’s Feng Zhang, who within one year made the technology work in cells and organisms, and therefore broadly applicable for biotechnology. Was Zhang’s contribution a substantial enough advance to warrant its own patents? Was Doudna’s work too theoretical and basic? This week, a potentially damning email that emerged from the legal filings of the dispute was made public. The email is from a former graduate student of Zhang’s, Shuailiang Lin, to Doudna. In addition to asking for a job, Lin wrote that Zhang was unable to make the technology work until the 2012 Doudna publication revealed the key conceptual advances. Lin adds: “I think a revolutionary technology like this […] should not be mis-patented. We did not work it out before seeing your paper, it’s really a pity. But I think we should be responsible for the truth. That’s science.” A spokesperson for the Broad Institute, Lee McGuire, suggested that Lin’s claims are false, and pointed out that Lin was in a rush to renew his visa, and had sent his explosive email to Doudna after being rejected for a new post at the Broad Institute. With CRISPR technology promising to change the face of biotechnology, the drama over its intellectual property continues to escalate. (Antonio Regalado, MIT Technology Review)

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August 26, 2016 at 9:00 am

Science Policy Around the Web – August 9, 2016

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By: Thaddeus Davenport, Ph.D.

Infectious Diseases

Local Transmission of Zika Virus in Miami

Most of the world became aware of Zika virus earlier this year when doctors in Brazil noticed a correlation between Zika virus infection in pregnant women and microcephaly in their newborn children. Recent articles in the New York Times on July 29th and August 1st report a notable shift in the course of the Zika virus epidemic – local transmission of the virus within the United States. In the last year, there have been over 1600 cases of Zika virus detected in the United States, but nearly all of these were imported infections, in which individuals became infected while traveling in Zika-endemic regions or through sexual transmission of the virus from people traveling outside of the United States. Because the Zika-carrying mosquito species, Aedes Aegypti is resident in parts of the continental United States, many people expected that it was only a matter of time before Zika virus would be transmitted locally within the United States. On July 29th, the Centers for Disease Control and Prevention and the Florida State Department of Health reported that Zika virus was transmitted to at least four individuals in the Wynwood neighborhood of Miami. By August 1st, the number of individuals infected in the neighborhood had risen to fourteen, and the CDC issued a travel advisory discouraging pregnant women from traveling to the affected area. Though Congress went to recess last month without passing a bill to provide funding for the fight against Zika virus, perhaps, and hopefully, the news of local Zika transmission will motivate bipartisan action in Congress to fund mosquito control efforts and basic research on this still poorly understood virus. (Pam Belluck, New York Times)

Clinical Research

First CRISPR-based clinical trial set to begin this month

The gene-editing technique commonly referred to as CRISPR-Cas9 has received significant attention over the last year because of its impressive potential to target, cut, and modify nearly any sequence of interest within a genome. David Cyranoski reported for Nature News that scientists in China are poised to be the first to use CRISPR-Cas9 technology in a human clinical trial. The trial will enroll individuals with metastatic, non-small cell lung cancer, for whom other treatment options have failed. T cells, immune cells capable of killing infected, cancerous, or otherwise afflicted cells, will be removed from these individuals and modified using CRISPR-Cas9 to delete the gene for a protein called PD-1, which plays a role in downregulating the immune response. It is hoped that removing PD-1 will make the participants’ T cells better able to mount an immune response against cancer cells. As an additional measure of safety, the genome of modified cells will be sequenced in order to confirm that there are no off-target modifications outside of the PD-1 gene that might impact the safety or validity of the study. Initially the trial will test the safety of introducing CRISPR-Cas9-modified cells into ten individuals at three different dosages, while also monitoring the effect of the treatment on cancer progression. A similar trial is set to begin in the United States later this year. These will be important proof-of-concept studies to show that CRISPR-Cas9 can be applied safely and effectively in the treatment of disease. (David Cyranoski, Nature News)

Drug Development – Antibiotic Resistance

New Funding for Antibiotics Development

Bacterial resistance to antibiotics is a significant and emerging threat to public health. Methicillin resistant Staphylococcus aureus (MRSA) – a commonly reported and widely feared strain of S. aureus – is one example of a bacterium that was once readily treatable with penicillin and related antibiotics, but which became difficult to treat after acquiring resistance genes. As current antibiotics become less effective against pathogenic bacteria, doctors are running out of tools to treat infections. An important hurdle to addressing the problem of antibiotic resistance is obtaining the funding necessary to support basic research. Unlike drugs to treat chronic health problems, effective antibiotics designed to cure infections of limited duration, are not necessarily a good investment for pharmaceutical companies. Asher Mullard reported for Nature News that CARB-X – Combating Antibiotic Resistant Bacteria Biopharmaceutical Accelerator- a public-private partnership supported primarily by the US government, Wellcome Trust, and the UK-based Centre for Antimicrobial Resistance hopes to provide US $350 million to motivate and accelerate the development of new antibiotics over the next five years. While many biotechnology companies support the initiative, some researchers, including Kim Lewis a researcher at Northeastern University, worry that CARB-X is too heavily focused on drug development instead of the discovery of novel antibacterial compounds. Despite their limitations CARB-X and other similar programs will likely provide valuable incentives for biotechnology and academic researchers to work towards better antibiotics for the common good. (Asher Mullard, Nature News)

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Written by sciencepolicyforall

August 9, 2016 at 9:00 am