Science Policy For All

Because science policy affects everyone.

Posts Tagged ‘genetic engineering

Science Policy Around the Web – December 6, 2018

leave a comment »

By: Neetu M. Gulati, Ph.D.

man-2125123_960_720

Source:Pixabay

 

The CRISPR Baby Scandal Gets Worse by the Day

Ethical concerns and controversy came to the forefront last week when news broke that Chinese scientist He Jiankui had supposedly created genetically edited babies using CRISPR technology: a first in the world. CRISPR/Cas9 technology, or CRISPR as it is more commonly known, is a scientific tool that allows researchers to edit (add, subtract, or change) the expression of genes quickly and precisely. This technology could be used to fix mutations that cause human disease. However, there are also risks, using CRISPR for gene editing may have disastrous side effects such as potentially leading to cancer.

He Jiankui claimed that he used the technology to alter a gene called CCR5 to reduce the risk of of HIV infection in embryos before implanting them in a woman, who then gave birth to twin girls. He claimed another CRISPR baby may be on the way from another pregnant woman. It is unclear if He has actually done what he claimed, and he has not yet published his results in a peer-reviewed journal. Nevertheless, the response to He’s claims have been strongly negative. Many people are concerned that He violated ethical norms by editing human embryos, especially because the overall consensus among scientistsin the field of gene-editing was that “there is a need for caution” and to only use the technology after “much more research to meet appropriate risk/benefit standards.”

Since the public has learned about He’s experiments, numerous scientists, including pioneers in the CRISPR field, have spoken out against He’s actions and have called for a temporary moratorium on similar experiments. Southern University of Science and Technology in China, where He has been on unpaid leave since February, has opened an investigation into He after finding out about his research. China’s National Health Commission is also investigating He.

Amid the backlash, He defended himself and his actions at the Human Genome Editing Summit in Hong Kong, claiming to be “proud” of his work. Dr. He has not been seen since the summit, however, and there are now concerns that he may be missing.

(Ed Yong, the Atlantic)

 

Trump emphasizes workforce training in new vision for STEM education

The White House released a new five-year strategic plan for science, technology, engineering, and mathematics (STEM) education this week, with a vision that “all Americans will have lifelong access to high-quality STEM education and the United States will be the global leader in STEM literacy, innovation and employment.” The report emphasizes workforce training in STEM, focusing primarily on opportunities outside of traditional classroom settings, such as apprenticeships. The plan also highlights the need for more diversity in STEM, such as minorities and women.

Overall the strategic plan focuses on four pathways to success: developing and enriching partnerships between educators, employers, and the community; engaging students in trans-disciplinary learning, including advancing innovation and entrepreneurship education; building computational literacy; and operating with transparency and accountability. The plan put forth by the Trump administration diverges from some of the key priorities of the former administration, including efforts focused on traditional academic environments such as training more teachers, and improving STEM instruction in colleges and universities. Instead, this plan appears more focused on how STEM education prepares students for the years after schooling is completed. “STEM education is absolutely critical to supporting the American worker, and this plan brings together a number of programs that are part of our emphasis on the American worker,” said Michael Kratsios, deputy assistant to the president at OSTP.

(Jeffrey Mervis, Science)

 

NASA’s InSight Mars explorer lands safely on the Red Planet

For only the eighth time in human history, a spacecraft has been landed on Mars. The InSight lander touched down on Martian soil on November 26, 2018 after over six months of space travel.

NASA’s InSight mission aims to gather information about Mars, and is part of the NASA Discovery program for focused solar science missions. InSight will study the crust, mantle, and core of Mars, to allow scientists to learn more about the formation of rocky planets in the solar system.

InSight has already begun taking photos of the surface of Mars, which have been posted on social media accounts such as Twitter. The lander has also set up solar panels, which allows InSight to power its cutting edge instruments. In doing so, the lander set an ‘off-world record,’ generating more electrical power than any previous vehicle on the planet’s surface. InSight project manager Tom Hoffman spoke on the importance of this achievement, “The 4,588 watt-hours we produced during sol 1 means we currently have more than enough juice to perform these tasks and move forward with our science mission.” This almost doubles the energy produced in a Martian day produced by NASA’s Curiosity rover, which previously held the record.

InSight will continue taking pictures of the surface of Mars to study its new surroundings and use its robotic arm to set up instruments to place them on the surface of Mars for the next few weeks. It will take two to three months before the lander begins conducting science on the Red Planet.

 

(Sarah Kaplan, Washington Post)

 

 

Have an interesting science policy link? Share it in the comments!

Advertisements

Written by sciencepolicyforall

December 6, 2018 at 5:23 pm

Science Policy Around the Web – November 27, 2018

leave a comment »

By: Allison Dennis, B.S.

forest-1161868_1280

Source: Pixabay

 

California’s Wildfires Could Mean A Generation Of Lung Problems

The acute dangers of uncontrolled wildfires are undeniable, yet chronic dangers remain poorly understood. Changes in air quality due to wildfire smoke may have long-term and widespread health effects that researchers are only beginning to decipher. In 2017, nature provided the near perfect conditions for a much needed experiment. As the Mendocino Complex Fire raged, its smoke drifted over 200 miles to blanket the living space of an outdoor colony of primates bred for research for 10 days. 500 infant rhesus macaques, a commonly used model of human disease, were exposed, allowing respiratory immunologist Lisa Miller to begin an experiment looking for long-term respiratory damage in a pediatric population. Her previous studies of a smaller group of monkeys exposed in 2008 revealed monkeys born in wildfire conditions grew up to have a reduced lung capacity and compromised immune system . Ten years after the fire, monkeys who were infants at the time of the fire have a high incidence of idiopathic pulmonary fibrosis, a fatal human disease associated with environmental pollutants and cigarette smoking. By carefully recreating her impromptu 2008 experiment, Miller is hoping to gain deeper insights into what damage can occur in the developing lung tissue that will lead to possible interventions.

Miller’s research already suggests that a brief exposure to smoke early in life can have a lifetime of consequences. Smoke inhalation is much more widespread than the immediate dangers of fire, and will need to be incorporated into disaster preparedness plans. Financial assistance may be needed to help families temporarily relocate following fires not only due to burned homes, but also to homes blanketed in smoke. Currently, websites like airnow.gov provide up to date measures of air quality and can be used to decide when to limit children’s time outdoors. Parents with other options may need to weigh the potential risks of raising a family in places where wildfires are an annual occurrence.

(Maggie Koerth-Baker, FiveThirtyEight)

 

Genome-edited baby claim provokes international outcry

 

The announcement of the first genome-edited babies is shocking ethicists, scientists, and spectators around the world. He Jiankui of the Southern University of Science and Technology of China in Shenzhen claims to have altered the embryonic DNA of two twin girls born in November 2018 by using CRISPR-cas9 to disable the protein CCR5, known to provide HIV access to human cells. The study has not yet been submitted for publication and will likely undergo extensive peer review for verification. He recruited couples looking to conceive, where the male partner had HIV. The risk of transmission of HIV between father and offspring is very low, and removing semen from sperm before fertilization is commonly used to further mitigate the small risk. However, He thought these would-be parents would especially value the benefits of conferring their child with a lifetime protection against HIV through altered genetics.

Since its first demonstration as a gene-editing technique nearly ten years ago, ethicists have debated the potential application of CRISPR-cas9 to alter human DNA. Because reproductive tissues develop from the edited zygotic cells, it is likely that the twins will pass these changes on to their offspring along with any other possible off-target changes to the genome. While lacking the CCR5 gene has been shown to confer protection against HIV infection, it may subsequently increase the risk of other viral infections. Many still feel that not enough is known about the long-term and generational effects of altering a person’s germ-line using these proteins to justify the risks they could pose over an unborn person’s lifespan. To what extent the institutions that facilitated the experiment were consulted or informed of the true nature of the search remains unclear. The trial was only registered on November 8th, well after the work had begun. However in the race to be first, He appears to understand that any harm to the children arising from the experiment stating these risks are “going to be my own responsibility.”

 

(David Cyranoski, Nature News)

 

Have an interesting science policy link? Share it in the comments!

Written by sciencepolicyforall

November 27, 2018 at 10:24 am

Science Policy Around the Web – November 28, 2017

leave a comment »

By: Patrice J. Persad, PhD

20171128_Linkpost

source: pixabay

Mental Health

Brain Patterns May Predict People at Risk of Suicide

Suicide is the second common cause of death for adults under 27 years old in the United States. In light of this, being able to identify individuals who are more likely to attempt suicide may transform suicide prevention initiatives’ outcomes.

At the end of last month, scientists published that machine learning (ML) algorithms were able to yield accurate prognoses by using functional magnetic resonance imaging (fMRI) profiles to differentiate subjects with suicidal thoughts (17 cases) and those without (17 controls) . After being exposed to a display of thirty words classified as positive (some examples: “kindness,” “carefree,” and “innocent”), negative (some examples: “guilty,” “evil,” and “gloom”), or pertaining to suicide (some examples: “lifeless,” “hopeless,” and “apathy”), each participant’s brain activity, or “neural signature”, for each word was captured by fMRI.

The accuracy of predicting individuals who had experienced suicidal thoughts was calculated as 91%. Upon stratifying the cases, a portion of those who had experienced suicidal thoughts were successfully identified as having attempted ending their lives in the past (nine out of 17). From categorizing individuals by suicide attempt status, the ML algorithm’s predictions were 94% accurate. The study’s authors envision clinical applicability, including improving patient surveillance, which could impact the suicide rate and lead to treatment or therapies concentrating on the cerebral areas identified by the algorithm. However, the researchers counsel that the study must be replicated with more subjects, particularly across different demographic groups and including individuals that have been diagnosed with psychiatric disorders but do not experience suicidal thoughts.

Various ethical concerns are raised by the application of this research, including the complications surrounding valid consent and how the life insurance industry could use the data. Classification of potential thoughts by ML may intrude privacy beyond what volunteers may feel they are consenting to when they agree to be recorded by fMRI or similar technologies. Neural signature classification in patients may have secondary uses, which can expand outside of medicine and healthcare areas, and must be evaluated. Misuse by life insurance companies, inviting discrimination against candidate insurance policy holders because of true and false classification of suicide ideation from fMRI, is a hypothetical scenario.

(Jon Hamilton, National Public Radio)

Species Conservation

‘Gene Drives’ Are Too Risky for Field Trials, Scientists Say

Consider the following illustration. On the island of New Zealand, a clan of non-native stoats, short-tailed weasels, are pulverizing numbers of the endemic takahes, flightless birds. Settlers in the 1800s had set stoats free through the terrain to prey on the rabbits, disturbers and unwanted consumers of agricultural crops. Thus, the stoat in this case may have been thought of as posts in a living rabbit-proof fence. Consequently, impacts on the native ecosystem were and are negative underlining the decline in and harm to takahes and other flightless bird species, such as the kiwi, New Zealand’s national bird. This currently heralds the question “How can population sizes of invasive species, such as these stoats, be reduced?”

A proposed solution to the aforementioned question features the germline genome editing system CRISPR to introduce genetic elements designed to undermine the reproductive success of a particular species. The idea of a gene drive system is to increase the inheritance, or frequency, of a particular variation in a gene. The CRISPR system can be applied to introduce variation in a gene essential for a given species’ reproductive system. When an animal carrying a CRISPR edited copy of a gene mates with an animal carrying a functional copy of the gene, their offspring will inherit one altered copy and one regular copy. Eventually, as this genetic variation becomes frequent enough, two individuals carrying the altered copy are likely to mate, producing infertile offspring. Then the number of invasive species’ members, the stoats for example, would drop as a result.

Are there any caveats to the CRISPR-based gene drive system for species conservation? A research team headed by Kevin M. Esvelt, the first proposer of gene drives for wild populations, used population models and stimulations to revealed a vital finding: if several gene-drive-modified organisms of an invasive species came into contact and bred with wild-type organisms of the same species, the population of the species as a whole in its native habitat would decrease over time. In other words, the encounter between a wild-type organism and gene drive organism is inauspicious if mating transpires in the species’ native habitat. Based on these findings, the researchers stressed prudence in initiating this application in field studies, which echoed discussions lead by the National Academy of Sciences in 2016.

One alternative may be to engineer gene drives to deteriorate after a certain number of generations. However, according to Esvelt in the context of eradicating vector-borne diseases, such as malaria, restricted gene drive systems affecting only several generations (out of many) may be ineffective in vector populations.

Although the opening example’s setting is New Zealand, the future development and employment of CRISPR-based gene drive systems depend on international cooperation plus participation because other nations may experience the impact of an ecosystem beyond their designated borders. For formulating proper safeguards before field testing commencement, voices from the scientific community, political arena, general community, and other domains across potentially affected countries must be amplified and heard for maximizing the protection of all species.

(Carl Zimmer, New York Times)

Have an interesting science policy link?  Share it in the comments!

Written by sciencepolicyforall

November 28, 2017 at 7:03 pm

Science Policy Around the Web – June 10, 2017

leave a comment »

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)

Have an interesting science policy link?  Share it in the comments!

Written by sciencepolicyforall

June 10, 2017 at 11:33 am

Science Policy Around the Web – February 24, 2017

leave a comment »

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)

Have an interesting science policy link?  Share it in the comments!

Written by sciencepolicyforall

February 24, 2017 at 11:23 am

Science Policy Around the Web – December 20, 2016

leave a comment »

By: Liz Spehalski, PhD

Source: Flickr, under Creative Commons

In-Vitro Fertilization

UK Approves Mitochondrial Replacement Therapy Trials For Assisted Reproduction

The UK Human Fertilization and Embryology Authority (HFEA) has given the green light to allow mitochondrial replacement therapy, a type of assisted reproduction that can help families to avoid passing on genetic diseases. The method is controversial because the embryos contain genetic material from three people: two eggs and one sperm. The decision has been widely anticipated and comes after years of debate and a change in the country’s laws in 2015.

Mitochondria are responsible for generating more than 90% of the energy required by the body to sustain life and support organ function. When mitochondria fail, cells generate less and less energy, resulting in cell injury and cell death. The parts of the body that require the most energy; the heart, brain, muscles, and lungs, are the most affected by mitochondrial disease. Mitochondrial disease is difficult to diagnose due to its wide range of symptoms, which can include seizures, strokes, developmental delays, blindness, and heart problems.

Mitochondrial replacement therapy involves exchanging damaged mitochondria for heathy ones by transferring only the nuclear DNA from one egg or fertilized embryo from the mother into a donor egg, whose mitochondrial DNA is intact but nuclear DNA is removed. The technique has already been performed in Mexico and the Ukraine, and John Zhang, a physician at New Hope Fertility Center in New York City, has said that a baby boy conceived by the technique in Mexico seems healthy to this point. Recent work with eggs from affected women, however, found that some of the defective mitochondria from the mother’s egg was transferred to the embryo along with the DNA, raising questions about the effectiveness of the treatment. Currently, congressional action has blocked the FDA from allowing mitochondrial replacement procedures to be attempted in the United States.

On December 15, the HFEA announced that it would allow clinics to apply for licenses to conduct limited trials of the technique, with the goal of preventing mothers from passing down mutations in mitochondria. “Today’s historic decision means that parents at very high risk of having a child with a life-threatening mitochondrial disease may soon have the chance of a healthy, genetically related child. This is life-changing for those families,” said HFEA chair Sally Cheshire. An HFEA spokesperson projected that the UK’s first child with three people’s DNA could be conceived as early as March 2017. (Ewen Callaway, Nature News)

Biotechnology

Commerce Secretary Announces New Biopharmaceutical Manufacturing Institute

Penny Pritzker, US Secretary of Commerce, announced on Friday a new institute to be added to the Manufacturing USA Institute; the National Institute for Innovation in Manufacturing Biopharmaceuticals (NIIMBL). The institute is the eleventh of Manufacturing USA Institute, the first funded by the Department of Commerce (DOC), and the first awarded under the Manufacturing USA “open topic” competition, in which industry was encouraged to propose institutes allocated to any manufacturing area that is not already being tackled.

While pharmaceutical manufacturing relies on chemistry, biopharmaceuticals are a drug product manufactured in or isolated from biological sources. They include vaccines, blood and blood components, stem cell and gene therapies, recombinant proteins, among other biologics. Many of these products are widely used for the treatment of an array of diseases such as cancer, autoimmune disorders and infectious diseases, generating billions of dollars worldwide.

The goals of this institute will be to keep biopharmaceutical manufacturing in the USA and to scale up the production of complex biological drugs. “In communities from coast to coast, the Manufacturing USA network is breaking down silos between the U.S. private sector and academia to take industry-relevant technologies from lab to market,” said Secretary Pritzker. “The institute announced today is a resource that will spread the risks and share the benefits across the biopharmaceutical industry of developing and gaining approval for innovative processes. The innovations created here will make it easier for industry to scale up production and provide the most ground-breaking new therapies to more patients sooner.”

NIIMBL received $70 million from the US Department of Commerce, and will be getting another $129 million from a public-private consortium of 150 companies, academic institutions, and nonprofits, as well as 25 states. The University of Delaware will be coordinating the institute’s partnership with the DOC. The hope is that NIIMBL will help to advance U.S. leadership in the biopharmaceutical industry, foster economic development, improve medical treatments, and ensure a qualified workforce by collaborating with educational institutions to develop new training programs matched to specific biopharma skill needs. (Press Release, Department of Commerce)

Have an interesting science policy link?  Share it in the comments!

Written by sciencepolicyforall

December 20, 2016 at 9:07 am

Science Policy Around the Web – September 30, 2016

leave a comment »

By: Jessica Hostetler, PhD

Source: Flickr, under Creative Commons

Human Genetic Manipulation

World’s first baby born with new “3 parent” technique

On September 27, 2016, the New Scientist reported the birth of a baby born with DNA from three people. The now five-month old healthy baby boy was born in New York to a Jordanian couple who had struggled for years to have a healthy child. The baby’s mother had genes for the lethal Leigh syndrome, a neurological disorder typically resulting in death in 1-3 years after birth, from which her first two children had died. These genes were carried in about 25% of her mitochondria, the energy producers for cells, which contain 37 genes separated from the thousands of other genes held inside the cell’s nucleus. Mitochondrial genes are only passed down from mothers through the mitochondria present in the mother’s egg before being fertilized by a father’s sperm.

The couple worked with US-based fertility expert John Zhang from the New Hope Fertility Center in New York City to undergo an approach for mitochondrial replacement therapy (MRT) called spindle nuclear transfer. Dr. Zhang transferred the nucleus of one of the mother’s eggs into a donor egg, which had the nucleus removed but contained healthy mitochondria. Several of these eggs were then fertilized with the father’s sperm to make 5 embryos with nuclear genes from both the father and the mother and mitochondria from the donor. The only healthy embryo was then implanted into the mother, and resulted in the birth of a healthy baby boy, with 99% healthy mitochondria.

This type of egg manipulation is now legal in the UK, though effectively banned in the US, so the team completed the fertility work in Mexico, which lacks clear regulations for the procedure. While several people such as Sian Harding who reviewed ethics for the UK guidelines, and legal scholar Rosario Isasi (from a Nature article), have acknowledged that Zhang’s group appears to have followed ethical guidelines, questions remain about the ethics, quality and safety of the technique.

The report was covered in a number of additional articles and commentaries, including in the New York Times, Science, and Nature. The commentaries note that researchers are eager for more information on a host of fronts such as the choice of using Mexico as the site of the work (as opposed to a more regulated and rigorous scientific environment) and the threshold of contaminating maternal mitochondria used in transfers (5%). These and other specifics are likely to come up when Dr. Zhang and team report on the case at the American Society for Reproductive Medicine meeting in October, 2016. (Jessica Hamzelou, New Scientist)

Health Policy

Why do obese patients get worse care? Many doctors don’t see past the fat

One in three Americans is obese; despite this fact, doctors and the healthcare system remain ill equipped in “attitudes, equipment and common practices” to treat obese patients. Beyond equipment issues, such as 90% of ERs and 80% of hospitals lacking M.R.I. machines built to accommodate very obese patients, research into bias against obese patients (both conscious and unconscious) shows that healthcare providers spend less time with such patients and refer them for fewer diagnostic tests. The same review reports that doctors feel less respect for obese patients and are more likely to stereotype them as “lazy, undisciplined and weak-willed,” all of which can negatively impact communication in the doctor-patient relationship, which in turn affects quality of care. In an effort to address the problem, the American Board of Obesity Medicine was founded to educate physicians about patient care and provide certification for achieving “competency in obesity care.”

Currently, these attitudes can lead health care providers to misdiagnose symptoms as being obesity-related instead of fully investigating other, potentially life threatening causes. Drug dosing may often be incorrect for obese people, particularly for cancer drug regimens for which obese individuals have worse outcomes across the board. Many orthopedists refuse joint hip and knee replacement surgery for obese patients unless they lose weight, though a review committee from the American Association of Hip and Knee Surgeons recommends a measured approach including options for surgery in some patients after the risks are discussed. The problems obese patients face may be exacerbated by the risk-averse hospital culture where adverse event scores affect Medicare reimbursements; thus pushing hospitals to avoid helping higher-risk patients. Beyond this there is a distinct lack of guidance from drug makers for correct dosing of anethesia drugs, with only a few examples, for instance a report from Dr. Hendrikus Lemmens out of Stanford University. Dr. Lemmens notes that 20-30% of obese-patient stays in intensive care after surgery are due to anesthetic complications and are likely frequently caused by drug dosing errors. Providing quality healthcare will likely only increase as the numbers of obese patients continue to increase in the US. (Gina Kolata, New York Times)

Have an interesting science policy link?  Share it in the comments!

Written by sciencepolicyforall

September 30, 2016 at 9:00 am