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Posts Tagged ‘biotechnology

Science Policy Around the Web – April 18, 2017

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By: Nivedita Sengupta, PhD

Source: pixabay

DNA Testing

23andMe Given Green Light to Sell DNA Tests for 10 Diseases

On April 6th, the US Food and Drug Administration (FDA) approved the first at-home genetic test kits, which can be sold over the counter in pharmacies, to determine the risk of developing certain genetic diseases. Since 2006, 23andMe, a company based in California, has been analyzing DNA from saliva samples of its customers to provide genetic insights into their risk of developing 240 different diseases and disorders. However, in 2013, FDA was concerned about customers using test results to make medical decisions on their own, and ordered 23andMe to halt the service. In 2015, FDA eased some of the restrictions and allowed the company to reveal to their customers only the information regarding genetic anomalies that can be transferred to their children, and not any information about the person’s own disease risk.

23andMe now has permission to inform its customers about genetic mutations that are strongly associated with a small group of medical conditions such as Parkinson’s disease, late-onset Alzheimer’s disease, celiac disease and a hereditary blood-clot disorder called thrombophilia. However, it should be noted that the results from these tests are not equivalent to a medical diagnosis, as the development of a disease is also influenced by a person’s family history, lifestyle and environment.

The decision made by the FDA paves the way for a wave of do-it-yourself diagnostic tests, which will be flooding the market in the coming years. “It’s a watershed moment for us and the FDA,” says Kathy Hibbs, chief legal and regulatory officer at 23andMe. However, there are concerns regarding the limits of medical knowledge among common people to understand and interpret the results and the limitations of these tests, which could lead to misinterpretation of the results and complications. (Amy Maxmen, Nature News)

Neonatal Care

Giving Newborns Medicine is a Dangerous Guessing Game. Can We Make it Safer?

Medical emergencies in neonates are on the rise. It might be surprising for many parents to know that 90% of the medications administered in a neonatal intensive care unit are not medically approved by the FDA for use in newborns. Neonates are routinely treated with drugs that are not adequately tested for safety, dosing, or effectiveness. This is a global problem, and many factors contribute to it. Firstly, parents and doctors are afraid of enlisting newborns in clinical trials. Secondly, pharmaceutical companies are afraid to test drugs on neonates as the risk of liability is very high. It is also a small market, so pharmaceutical companies may not make money by getting drugs approved for neonates.

In 2015, an FDA funded nonprofit organization launched two global efforts to encourage clinical trials in newborns. One of which is the International Neonatal Consortium (INC), which published a guide to clinical trials in neonates last year. Dr. Jonathan Davis, Director of INC said, “We’ve got to do something.” Without information on drug data for newborns, “we can’t be certain which drugs, in which doses, to use when.” Under the current system, doctors are making decisions based on either anecdotes or intuition, which essentially means that every sick newborn is an uncontrolled, unapproved study without the guarantee of seeing improvement. No data collection is done, thus not providing any information for treating other infants around the world.

Physicians often take decisions by scaling down from how medications are used in adults. But this can be fatal and lead to disasters as we have seen in the past, with the use of the antibiotic chloramphenicol in the 1950s, and the preservatives benzyl alcohol and propylene glycol in the 1980s. Infants are not tiny adults, and they adsorb, metabolize, and excrete drugs in different ways than adults. The majority of studies done in neonates in recent years have not been able to establish efficacy. More studies need to be done, and this requires proper designing of clinical trials with reduced risk, and eliminating unnecessary interventions. (Megan Scudellari, STATNews)

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April 18, 2017 at 10:45 am

Science Policy Around the Web – April 7, 2017

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By: Kseniya Golovnina, PhD

Cancer Research

RNA-Seq Technology for Oncotargets Discovery

One of the most significant discoveries in cancer research, using the “Big Data” approach with experimental validations, was made recently by Chinese and American scientists together with Splicingcodes.com. They described the first cancer predisposition, familially-inherited, fusion gene, KANSARL, specific to populations with European ancestry, by using advanced RNA-sequencing (RNA-seq) of cancer transcriptomes.

A fusion gene is a hybrid formed from two previously separate genes as a result of chromosomal rearrangements. Often, fusion genes are oncogenes. The first fusion gene abnormality was described in a human malignancy and was called the Philadelphia chromosome. In the early 1980s, scientists showed that a translocation between chromosomes 9 and 22 led to the formation of a fusion gene (BCR/ABL1), which produced a chimeric protein with the capacity to induce chronic myeloid leukemia. KANSARL is the most prevalent cancer gene discovered so far. Scientists systematically analyzed the RNA-seq data of many cancer types from different parts of the world, together with RNA-seq datasets of the 1000 Genome Project. KANSARL fusion transcripts were rarely detected in tumor samples of patients from Asia or Africa, but occurred specifically in 28.9% of the population of European origin.

Scientists from Cancer Genome Anatomy project at the National Cancer Institute (NCI), using sophisticated sequencing techniques, have identified 10,676 gene fusions among cancer-related chromosomal aberrations. Splicingcodes.com has identified over 1.1 million novel fusion transcripts, many of which are likely biomarkers of diseases. Fusion genes play an important role in diagnosis and monitoring of cancer treatment progress by measuring the disappearance of the fusion and, thereby, the disappearance of the tumor tissue. Currently, several clinical trials are aimed at treating fusion-positive patients with a range of targeted therapies, which will hopefully lead to novel therapy development and save patients’ lives. (Splicingcodes)

Biotechnology

Turning Mammalian Cells into Biocomputers to Treat Human Disease

Engineering cells by manipulating DNA and controlling their performance is a growing field of synthetic biology. Scientists have been working with bacterial cells for years to perform different controlled actions, for example, lighting up when oxygen levels drops. Bacterial cells, including Escherichia coli, have a simple genome structure and are relatively easy to manipulate. Using bacterial cells, it was possible also to join several genetic circuits within a single cell to carry out more complex actions.

After successful engineering in bacteria, researchers have aimed to create genetic circuitry to detect and treat human disease in mammalian cells. Most of the attempts have failed due to the complexity of the mammalian genome, until a group of biomedical engineers from Boston and Basel, Switzerland decided to upgrade their DNA “switches”. They used an ability of special enzymes, DNA recombinases, to selectively cut and stitch DNA. The new system in mammalian cells is called ‘Boolean logic and arithmetic through DNA excision’ (BLADE). BLADE founders built a wide variety of circuits (113), each designed to carry out a different logical operation with 96.5% success. This Boolean system has great potential for applications in cell and tissue engineering. One exciting possibility is engineering T-cells with genetic circuits that initiate a suicide response to kill tumors when they detect the presence of two or three “biomarkers” produced by cancer cells. (Robert F. Service, ScienceNews)

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April 7, 2017 at 9:22 am

The Trans-Pacific Partnership and its Impact on Pharmaceutical Affordability

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By: Shakira M. Nelson, PhD, MPH

        For many, the Trans-Pacific Partnership (TPP) was a point of great debate during the 2016 Presidential primaries and election. As a simplified explanation, the TPP is a free-trade agreement involving the United States, Canada, Australia, Japan, New Zealand, Mexico, Chile, Peru, Brunei, Malaysia, Singapore and Vietnam, intended to “level the trading playing field” through the elimination of tariffs and other laws that create trade barriers. In its final form, the TPP would impact up to one-third of world trade and 40% of the global gross domestic product. Many who debated the ramifications of the TPP did so in the context of foreign policy interests. Although aligned with foreign policy, a major part of the TPP deals with intellectual property protection, and pharmaceutical drug development. If implemented, the effects of the TPP could greatly diminish public access to affordable medicines, both domestically and internationally. Moreover, the stronghold the TPP places on intellectual property could limit the development and marketing of less expensive options.

Intellectual property can be divided into two categories: industrial property and copyright. Patents, trademarks, and industrial design fall under industrial property. Patent development is a large part of scientists’ work, seen as almost a necessity to incentivizing innovation. Many argue that, without the ability to patent inventions and significant findings, scientists would not be able to generate profits used to sustain research and development; within the pharmaceutical industry, patents are the proverbial bread-and-butter. When in place, patents create a stronghold around the release of new chemical drugs, which prevents competition by generic brands. The standard length of time of a patent for a chemical drug is 20 years, which starts from the time the drug is invented.

Many new medicines under development today fall under the category of ‘biologics’. As the name suggests, biologics are treatments made from biological sources, and are very different from chemical drugs. Created to treat a multitude of diseases, including Ebola and cancer, biological sources include vaccines, anti-toxins, proteins, and monoclonal antibodies. Given their structural complexity compared to traditional drugs, and use of recombinant DNA technology, biologics are more difficult, and costlier to make. Moreover, manufacturers have a greater burden in ensuring product consistency, quality, and purity over time. This is done through certifying that the manufacturing process remains the same over time. Because of this, it is estimated that the price to manufacture biologics cost on average more than 22 times the price of chemical drugs. Current laws state that generic biologic development, known as biosimilars, cannot be approved until 12 years after the branded product has been approved – this is known as an exclusivity period. This was enacted under the Biologics Price Competition and Innovation Act of 2009, by the Food & Drug Administration (FDA).

The challenge with current policies is establishing a period-of-time that balances the need for companies to generate profits and cash flows, which will incentive them to conduct more research and compensate them for the extensive manufacturing processes, with the need to provide greater access through launching generic drugs and biosimilars. The trouble with the proposed policies of the TPP agreement is that they seem to embolden the pharmaceutical companies by introducing changes that would prevent competition from generics and biosimilars for longer periods of time than the current basic terms. The implications of this are far-reaching, as it may lead to a significant increase in the current costs of pharmaceutical drugs and biologics, hindering the health of the patients who rely upon these treatments.

Critics of the current system of patent length and biologic exclusivity periods fear that rather than incentivizing innovation, companies are being rewarded through their ability to charge higher amounts for drugs without the fear of competition on the market. Health policy experts concur, identifying policies such as the Hatch-Waxman Act of 1984 in allowing for the creation of drug monopolies, and “going too far in compensating the pharmaceutical industry at the public’s expense”. A report released in 2009 by the Federal Trade Commission stated that biosimilar development was more difficult to achieve than traditional generic drugs. For example, development requires comparisons to the original biologic, to prove efficacy and equivalence. Biosimilars must share the same mechanism of action, with no clinically significant differences in terms of safety or potency for the approved condition of use. The steps necessary to achieve this are significant, and therefore imposing a 12-year exclusivity period on biologics may be unnecessary. US Congressmen have pushed to compromise, floating an amendment to the TPP that would lower the exclusivity period to 8 years. However, critics and patients who rely upon drug competition to lower market prices, have protested this amendment stating that costs of new drugs and biologics are too high, and 8 years is too long of a length of time to wait for affordable generics and biosimilars to come on to the market.

The impact of decreasing the length of time it takes for biosimilars to come onto the market can be seen with Neupogen, a leukemia drug that was first approved by the FDA in 1991. Delivered via injection, Neupogen costs patients $3,000 for 10 injections. With injections needed daily, this drug could carry a price tag of well over $100,000 per year. It wasn’t until recently, however, that the first biosimilar was approved on the US market. The biosimilar, Zarxio, was approved as a leukemia drug and is priced at more than $1000 less than Neupogen. This pricing has the potential to decrease the yearly costs of this drug from $100,000 with Neupogen to $55,000-$75,000. Further evidence of these financial savings was provided by the Rand Corporation, which predicted a savings of over $44 billion over 10 years with an increased approval of biosimilars, for patients who rely upon these specific cancer treatments.

Internationally, the policies of the TPP also have far reaching effects on the availability and costs of pharmaceuticals. The 12-year exclusivity period would be imposed upon the other countries involved in the TPP, where currently for some, such as Brunei, there is no current exclusivity protection. By imposing the 12-year period, global competition could become restricted. Additionally, the TPP proposes other key patent protections that play a bigger role on the international market. One protection, known as evergreening, allows drug companies to request patent extensions for new uses of old drugs. The immediate effect of this is an extension of monopolies on drug sales for minor reasons. The second protection allows pharmaceutical companies to request patent extensions if it takes “more than 5 years for an application to be granted or rejected.” Advocacy groups fear that the price of drugs would undermine the efforts of health initiatives, such as the Global Fund to Fight AIDS, Tuberculosis, and Malaria. These initiatives rely upon price competition to manage costs, with the availability of cheap generics helping drive costs down.

Although the current administration has ended the USA’s association with the Trans-Pacific Partnership, it is important to note that other countries may try to implement some of the policies, affecting the availability and affordability of drug treatments. To decrease this burden, the US could work to assist in negotiating exceptions for the poorer and smaller countries, to help them meet any challenges they may come up against. Within the US itself, it is important for policies, laws and any future trade agreements to be modified, with more of a focus on the affordability and regulation of drugs and biologics. Imposing price controls may offer a modest benefit, but may not be a long-term solution. A focus on lowering the patent length for new drugs and biologics can be an immediate step. Although the push back from pharmaceutical lobbyists will be substantial, alleviating the financial burden on families afflicted with cancer and diseases should be the focus.

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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

Genetically Modified Animal Vectors to Combat Disease

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By: Sarah L Hawes, PhD

Mosquito larvae: ©ProjectManhattan via Wikimedia Commons

Diseases transmitted through contact with an animal carrier, or “vector,” cause over one million deaths annually, many of these in children under the age of five. More numerous, non-fatal cases incur a variety of symptoms ranging from fevers to lesions to lasting organ damage. Vector-borne disease is most commonly contracted from the bite of an infected arthropod, such as a tick or mosquito. Mosquito-borne Zika made recent, regular headlines following a 2015-2016 surge in birth defects among infants born to women bitten during pregnancy. Other big names in vector-borne disease include Malaria, Dengue, Chagas disease, Leishmaniasis, Rocky Mountain spotted fever and Lyme.

Vaccines do not exist for many of these diseases, and the Centers for Disease Control (CDC) Division of Vector-Borne Diseases focuses on “prevention and control strategies that can reach the targeted disease or vector at multiple levels while being mindful of cost-effective delivery that is acceptable to the public, and cognizant of the world’s ecology.” Prevention through reducing human contact with vectors is classically achieved through a combination of physical barriers (i.e. bed nets and clothing), controlling vector habitat near humans (i.e. dumping standing water or mowing tall grass), and reducing vector populations with poisons. For instance, the Presidential Malaria Initiative (PMI), initiated under President Bush in 2005, and expanded under President Obama, reduces vector contact through a complement of educating the public, distributing and encouraging the use of bed nets, and spraying insecticide. Now a 600 million dollar a year program, PMI has been instrumental in preventing several million Malaria-related deaths in the last decade.

But what if a potentially safer, cheaper and more effective solution to reduce human-vector contact exists in the release of Genetically Modified (GM) vector species? Imagine a mosquito engineered to include a new or altered gene to confer disease resistance, sterility, or to otherwise impede disease transmission to humans. Release of GM mosquitos could drastically reduce the need for pesticides, which may be harmful to humans, toxic to off-target species, and have led to pesticide-resistance in heavily-sprayed areas. Health and efficacy aside, it is impossible to overturn or poison every leaf cupping rainwater where mosquitos breed. GM mosquitos could reach and “treat” the same pockets of water as their non-GM counterparts. However, an insect designed to pass on disease resistance to future generations would mean persistence of genetic modifications in the wild, which is worrisome given the possibility of unintended direct effects or further mutation. An elegant alternative is the release of GM vector animals producing non-viable offspring – and this is exactly what biotech company Oxitec has done with mosquitos.

Oxitec’s OX513A mosquitos express a gene that interferes with critical cellular functions in the mosquitos, but this gene is suppressed in captivity by administering the antibiotic tetracycline in the mosquitos’ diet. Release of thousands of non-biting OX513A males into the wild results in a local generation of larvae which, in the absence of tetracycline, die before reaching adulthood. Release of OX513A has proven successful at controlling mosquito populations in several countries since 2009, rapidly reducing local numbers by roughly 90%. Oxitec’s OX513A line may indeed be a safe and effective tool. But who is charged with making this call for OX513A and, moreover for future variations in GM vector release?

Policy governing use of genetically modified organisms must keep pace with globally available biotechnology. Regulatory procedures for the use of GM vector release are determined by country, and there is a high degree of international policy alignment. The Cartagena Protocol on Biosafety is a treaty involving 170 nations currently (the US not included) that governs transport of “living modified organisms resulting from modern biotechnology” with potential to impact environmental or human health. The World Health Organization (WHO) and the Foundation for the National Institutes of Health (FNIH) published the 2014 guidelines for evaluating safety and efficacy of GM mosquitos.

Within the US, the 2017 Update to the Coordinated Framework for the Regulation of Biotechnology was published this January in response to a solicitation by the Executive Office of the President for a cohesive report from the Food and Drug Administration (FDA), Environmental Protection Agency (EPA), and US Department of Agriculture (USDA). Separately, biotech industry has been given fresh guidance on whether to seek FDA or EPA approval (in brief):  if your GM product is designed to reduce disease load or spread, including vector population reduction, it requires New Animal Drug approval by FDA; if it is designed to reduce pest population but is un-related to disease, it requires Pesticide Product approval by EPA under the Federal Insecticide, Fungicide, and Rodenticide Act.

Thus, for a biotech company to release GM mosquitos in the US with the intent of curbing the spread of mosquito-borne disease, they must first gain FDA approval. Oxitec gained federal approval to release OX513A in a Florida suburb in August 2015 because of FDA’s “final environmental assessment (EA) and finding of no significant impact (FONSI).” These FDA assessments determined that the Florida ecosystem would not be harmed by eliminating the targeted, invasive Aedes aegypti mosquito. In addition, they affirmed that no method exists for the modified gene carried by OX513A to impact humans or other species. Risks were determined to be negligible, and include the accidental release of a few, disease-free OX513A females. For a human bitten by a rare GM female, there is zero risk of transgene transfer. There is no difference in saliva allergens, and therefore the response to a bite, from GM and non-GM mosquitos. In addition, as many as 3% of OX513A offspring manage to survive to adulthood, presumably by spawning in tetracycline-treated water for livestock. These few surviving offspring will not become a long-term problem because their survival is not a heritable loop-hole; it is instead analogous to a lucky few mosquitos avoiding contact with poison.

Solid scientific understanding of the nature of genetic modifications is key to the creation of good policy surrounding the creation and use of GMOs. In an updated draft of Guidelines For Industry 187 (GFI 187), the FDA advises industry seeking New Animal Drug Approval to include a molecular description of the intentional genetic alteration in animals, method for alteration, description of introduction to the animal, and whether the alteration is stable over time/across generations if heritable, and environmental and food safety assessments. Newer genomic DNA editing techniques such as CRISPR offer improved control over the location, and thus, the effect of genetic revisions. In light of this, the FDA is soliciting feedback from the public on the GFI 187 draft until April 19th, 2017, in part to determine whether certain types of genetic alteration in animals might represent no risk to humans or animals, and thus merit reduced federal regulation.

Following federal clearance, the decision on whether to release GM vectors rests with local government. Currently, lack of agreement among Florida voters has delayed the release of OX315A mosquitos. Similar to when GM mosquito release was first proposed in Florida following a 2009-2010 Dengue outbreak, voter concern today hinges on the perception that GM technology is “unproven and unnatural.” This illustrates both a healthy sense of skepticism in our voters, and the critical need to improve scientific education and outreach in stride with biotechnology and policy. Until we achieve better public understanding of GM organisms, including how they are created, controlled, and vetted, we may miss out on real opportunities to safely and rapidly advance public health.

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February 16, 2017 at 9:46 am

Science Policy Around the Web – December 20, 2016

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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)

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December 20, 2016 at 9:07 am

Science Policy Around the Web – October 25, 2016

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By: Nivedita Sengupta, PhD

Source: pixabay

Clinical Trials

EMA becomes first major drugs agency to publish clinical-study reports online

On 20th October, the London-based European Medicines Agency (EMA) published details of the full clinical-trial data that it received from pharmaceutical companies, some 100 clinical reports, about two EMA-approved medicines, carfilzomib, a cancer drug, and lesinurad, a gout treatment. The disclosures make the EMA the first major drug regulatory agency to completely publish the results of clinical investigations that drug developers submit while applying for the agency’s approval to market medicines in the European Union. “These clinical study reports (CSR) are much more detailed than the papers that drug firms publish in scientific journals. It includes both positive and negative results, and details of drugs’ adverse effects,” says Larry Peiperl, the chief editor of PLoS Medicine.

Under the rules the EMA brought in six years ago, it had released results of such studies only if third parties asked for them using freedom-of-information requests. However, those rules allowed some drug firms to drag the agency to court to try to prevent their data from being released, arguing it as commercially confidential. However, patients and clinicians have waited long, and about 700 medical and patient organizations had lobbied for clinical data release under the All Trials campaign. “The EMA’s CSR policy adopted in 2014 will benefit both academic research and the practice of medicine as a whole,” says EMA executive director Guido Rasi. It will help academicians to independently re-analyze data even after a medicine has been approved, and will help drug developers to learn from the experiences of others.

The EMA intends to release all CSRs in applications that were submitted since 1st January 2015. It will only edit some commercially confidential information like individual patient data before release. After the clearance of backlog, the EMA says that it will offer public access to around 4,500 clinical reports each year.

Some drug firms are still resisting the release of their data by the EMA. In the latest legal battle this July, an interim judicial EU court order blocked the EMA from releasing toxicity studies on a veterinary medicine called Bravecto (fluralaner), and clinical-study reports on Translarna (ataluren), a treatment for Duchenne muscular dystrophy. The two drug firms concerned, Intervet and PTC Therapeutics, argued that the release of data would infringe on their rights to protect commercially confidential information. However, the EMA has appealed against both decisions on 29th September, and says that it sees the cases as a test of its policy. (Alison Abbott, Nature News)

Biotechnology

In a first, mouse eggs grown from skin cells

For the first time, stem cell researcher Katsuhiko Hayashi of Kyushu University in Fukuoka, Japan, and colleagues have reprogrammed fibroblasts from the tip of an adult mouse’s tail to make eggs, which upon fertilization grew into healthy mice. Earlier, adult body cells were reprogrammed to generate stem cells (induced pluripotent stem cells – iPSCs), which were further induced into becoming a wide variety of other cells but never eggs. Egg cells are much trickier as they represent ultimate flexibility which can create all the bits and parts of an organism from raw genetic instructions. “This is very solid work, and an important step in the field,” says developmental biologist Diana Laird of the University of California, San Francisco. This major development could make it possible in near future to study the formation of gametes — eggs and sperm — an unknown process that takes place inside fetuses. Moreover, if the experiments gets extended to human cells, it could make eggs easily available for research and may eventually lead to infertility treatments.

In this experiment, Hayashi and colleagues made artificial ovaries by extracting ovarian support cells from albino mouse embryos, which were then mixed with primordial germ cell‒like cells created from tail-tip skin cells from a normally pigmented mouse. After 11 days of maturation followed by fertilization, the eggs were transplanted into the uteruses of female mice. Six pups with dark eyes were born, indicating that they came from the tail-tip eggs and not eggs accidently extracted from the albino mice along with the ovarian support cells. The baby mice grew up apparently healthy and have produced offspring of their own.

As ovarian cells from mouse embryos were still needed to support the growth of eggs in vitro this could be a potential problem when trying to replicate the experiments in humans. “It’s yet unclear how support cells in ovaries foster egg development. Researchers can’t yet reproduce the supporting cells in the lab and so need to get those cells from embryos,” Hayashi says. (Tina Hesman Saey, ScienceNews)

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Written by sciencepolicyforall

October 25, 2016 at 10:55 am