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Posts Tagged ‘Stem Cells

Science Policy Around the Web – October 10, 2017

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By: Kseniya Golovnina, PhD.

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source: pixabay

Gene Therapy

In a First, Gene Therapy Halts a Fatal Brain Disease

 The first historic gene therapy approval by the U.S. Food and Drug Administration in August 2017 opened a new era for the treatment of serious and life-threatening diseases. One month later Bluebird Bio announced the successful start of Lenti-D therapy in the clinical trial and gave a flutter of hope to cure cerebral adrenoleukodystrophy (CALD) also known as Lorenzo’s Oil.

Adrenoleukodystrophy (ALD), a rare disorder that affects one in 21,000 male births worldwide, is caused by mutations in the ABCD1 gene that lead to the subsequent accumulation of very long chain fatty acids in tissues, including the myelin of the central nervous system. The most severe form of ALD, known as cerebral ALD (CALD), involves the progressive destruction of the protective sheath of the nerve cells in the brain that are responsible for thinking and muscle control. This leads to deafness, blindness, seizures, loss of muscle control and dementia, resulting in permanent disability or death. Symptoms of CALD progress rapidly if untreated and the only current treatment is stem cell transplantation.

Gene therapy is a technique for correcting defective genes responsible for disease development.  It utilizes viruses to deliver unmutated copies of the genes, such as the ABCD1 gene, to the cells of the patient’s body. First, blood from the patient is collected by apheresis, depleting immune response T cells and enriching progenitors of all blood cells (hematopoietic stem cells, HTS). The HTS cells are then infected with a virus carrying a functional copy of the gene, before returning the cells to the body.

Bluebird Bio is now pursuing Lenti-D therapy which uses lentiviruses to deliver a functional copy of the ABCD1 gene to patients with ALD. Results published in the New England Journal of Medicine, reported that 15 out of 17 patients (88%) were free from major functional disabilities two years after the hematopoietic stem-cell gene therapy. These results demonstrate the therapy’s efficacy over the 76% benchmark established by radiotherapy-free survival at 24 months. Bluebird’s Chief Medical Officer David Davidson expressed excitement about the patients’ progress. He announced that the first four patients treated in the expansion cohort are also doing well, as measured by their amount of the functional ABCD1 gene.

(Gina Kolata, The New York Times)

Drug pricing

FDA acts to encourage generic competition for complex drugs

What kind of feelings do you have when pharmaceutical companies announce their prices for upcoming exciting gene therapies and other innovative, life-changing bio pharmaceuticals? Positive news about development and success of first-of-their-kind drugs can be undermined by anxieties that patients will not be able to afford them. High drug prices can prevent accessibility of new therapies vital for many patients, and market analysts predict rapid inflation in healthcare spending over the next few years due to the aging US population.

The FDA is conscious of the stress expensive drugs put on both patients and the entire healthcare system. Under the leadership of current Commissioner, Dr. Scott Gottlieb, one of key goals of the the FDA is to bring more competition from generics to help drive prices down. On October 2, 2017 the agency prepared draft guidance specifically aimed at copycats of complex therapies and therefore trying to clear the way for generic drug makers to the market.

Gottlieb wrote in his blog post that the agency is looking for more efficient regulatory pathways with robust reviews and communication with pharmaceutical companies for abbreviated new drugs applications (ANDAs). He highlighted that “early and better meetings between FDA and sponsors can improve development timelines. The agency acknowledged that the complexity of the approval process may be discouraging to generic drug makers. The new guidance aims to clarify these complexities by outlining how genetic makers can prove “sameness” by showing that there is no difference in response to generic drug compared with the original. This is the second update for ANDAs process and Dr. Gottlieb assured that FDA will continue to progress in this reformation.

(Linda A. Johnson, The Associated Press)

 

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October 10, 2017 at 10:11 pm

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Science Policy Around the Web – July 21, 2017

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By: Rachel F Smallwood, PhD

Source: pixabay

Cancer

Engineered Cell Therapy for Cancer Gets Thumbs Up from FDA Advisers

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

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

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

Stem-Cell Therapy

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

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

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

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

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

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July 21, 2017 at 10:08 am

Science Policy Around the Web – June 13, 2017

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By: Nivedita Sengupta, PhD

By Mikael Häggström, used with permission. [Public domain], via Wikimedia Commons

Stem Cell Therapy

Texas on Track to Become First State to Explicitly Back Stem Cell Therapies

On 30th May, Texas passed a bill  authorizing unapproved stem cell therapies, making Texas the first state to openly recognize experimental treatments. The bill will make the use of unapproved stem cell therapies legal for patients and is currently awaiting the approval of Governor Greg Abbott, who already supports the measure. Experimental stem cell therapies for terminal and chronic conditions have struggled for years to gain support without much success. Until now, no state has provided legal validation for these kind of therapies and the current stem cell procedures are mostly done under strict regulations.

Amendments were added to the bill, which require that the treatments be delivered by doctors with the approval of an institutional review board, which deals with human research. It will also add another amendment that will allow patients to have authority to sue in case the treatments go wrong. Many scientists and advocates opposed the measure stating that unapproved stem cell therapies can be harmful rather than beneficial. They state that though the amendments add protection to the patients, there are a few aspects of the bill that make them uncomfortable. Two other bills focused on patient access to experimental therapies, also known as “right-to-try” policies, failed to pass in the Texas Senate. (Andrew Joseph, STATNews)

Research Funding

NIH Scraps Plans for Cap on Research Grants

US National Institutes of Health (NIH) decided to drop the controversial proposal of capping the number of grants that an investigator can have at a time. The initial capping attempt was suggested to gather funds for younger researchers by NIH in May. The proposal was based on studies that suggested that a lab’s productivity decreases once it holds too many grants. Younger scientists often face more difficulties in obtaining NIH RO1 grants compared to their older more experienced colleagues. As a result, many researchers applauded the NIH’s effort to provide more funding for younger scientists. Yet the capping proposal received major adverse response from the scientific community stating that the NIH’s interpretation of the productivity study data does not apply to all labs, especially to the collaborative lab groups with four or five R01s that are more productive than labs with only one. Researchers also complained that the proposed point-based scoring system will also make collaborations difficult thus hampering productivity in the long run.

NIH director Dr. Francis Collins stated that the original idea was still a work in progress and NIH is going to put a hold on it. Instead of the cap, on 8th June, NIH announced the creation of the special fund, the Next Generation Researchers Initiative (NGRI), starting with US$210 for funding young researchers. The initiative will focus on investigators with less than 10 years of experience as NIH- funded principal investigators, and on high score grant proposals that were rejected because of lack of money. The initiative will grow up to $1.1 billion over the next five years. According to NIH principal deputy director Larry Tabak, NIH will immediately start creating an inventory of investigators who meet these criteria and expects that this approach will allow more than 2,000 additional R01 grants to be funded to younger scientists compared to the cap-based plan, which would have supported only 1600 awards. Nonetheless, the current proposal is still going to generate controversy as it will affect the older researchers because of NIH’s diversion of funding. (Sara Reardon, Nature News)

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June 13, 2017 at 7:08 pm

Science Policy Around the Web – May 12, 2017

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By: Liu-Ya Tang, PhD

Source: pixabay

Biosafety

Basic Scholarship in Biosafety Is Critically Needed

While a significant amount of money funds primary research in life sciences, the portion allotted in biosafety assessment is almost neglected, which can be detrimental to biomedical research. In a recent paper in mSphere, an open-access journal published by the American Society for Microbiology (ASM), the authors reported the status of practicing biosafety in U.S. labs and pointed out the urgent need for funding in this field.

They identified human errors as the dominant component of laboratory biosafety risk, but there was limited data to support a quantitative analysis of human failure rates. Publicly available risk assessments were only focused on mechanical failure rates. They also found that historical biosafety incident data is not adequate, and incidents reporting systems are not sufficiently standardized. So the same mistakes could likely happen in multiple labs. In contrast, other industries, such as the power and transportation industries, have been investing heavily in maintaining safety records and have benefited from doing so. The authors cite an example from the airline industry to address the importance of incident reporting system. After a flight crash outside Washington’s Dulles airport in 1974, the Federal Aviation Administration (FAA) created a no-fault system of reporting aviation incidents and mistakes. FAA has maintained this system ever since, which has helped reduce accident rates by two-thirds compared to that in the early 1970s.

Even though funding for biosafety assessment is much less than that in other industries, the consequences of a potential infectious disease outbreak can be much bigger than any other accidents. Therefore, such funding is urgently needed for three aspects: “(i) development of a national incident reporting system, (ii) primary research programs focused on human reliability assessments, equipment failures, and decontamination efficiencies, and (iii) sharing of best practices.” Investing in biosafety and biorisk management will help enhance laboratory safety practices and improve work performance of our research enterprise in the long run. (Ryan Ritterson and Rocco Casagrande, mSphere)

Human Stem-Cell Research

Attitudes Towards Stem-Cell Research in Europe, Canada and the United States

Human embryonic stem-cell research has caused many political and public debates over moral concerns while providing benefits to human health. In science policy making, public opinion has great impact. To investigate factors that affect international public opinion towards stem-cell research, Allum N. and colleagues analyzed representative sample surveys in Europe and North America, fielded in 2005, when it was a highly contested issue.

The authors found that public attitudes towards stem-cell research has been affected by government decisions, especially in the U.S. During the Bush administration, federal funding only allowed the use of a small number of existing cell lines in stem-cell research. These limitations were removed by an Executive Order from President Barack Obama that expanded NIH support for human stem-cell research. In response to government guidance, public support for stem-cell research in the U.S. rose from 40 percent in 2002 to around 65 percent in 2010. About 65 percent of Europeans and Canadians supported human stem-cell research on the condition that it is tightly regulated. The other influential factor is religion. The authors showed that in all the regions examined, approval for stem-cell research decreased with increasing religious commitment. This pattern was more pronounced in the U.S. and Canada than in Europe. But interestingly, half of even the most religious public supported stem-cell research, which indicated that perhaps the benefits of stem-cell research are being more appreciated. Overall, the majority of people in the surveyed areas hold positive attitudes towards human stem-cell research. (Nick Allum et al, PLOS ONE)

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May 12, 2017 at 11:07 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 – November 22, 2016

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By: Rachel Smallwood, PhD

Photo source: pixabay

Federal Research Funding

US R&D Spending at All-Time High, Federal Share Reaches Record Low

Recently released data from the National Science Foundation (NSF) showed trending increases in scientific research funding in the US across the past several years. Estimates of the total funding for 2015 put the value at an all-time high for research and development (R&D) funding for any country in a single year. In 2009, President Obama stated a goal to devote 3% of the USA’s gross domestic product (GDP) to research, and we have been making slow progress to that point; in 2015, 2.78% of the GDP went to research. Businesses accounted for the largest portion of overall scientific funding, contributing 69% of the funds. The second largest contributor was the federal government; however, it had the lowest percentage share of the total since the NSF started tracking funding in 1953, and the actual dollar amount contributed has been declining since 2011. Therefore, although the overall percentage of GDP going to research is increasing, that increase is driven by businesses, whereas the GDP percentage contributed by the federal government has dropped to almost 0.6%.

When taking a closer look at types of research, the federal government is the largest funding source for basic science research, covering 45% of the total. However, businesses make up the majority of the funding for applied research (52% in 2014) and experimental development (82% in 2014). This disproportionality in funding types combined with the decreases in federal research spending are concerning for the basic science field. There is more competition for less money, and this concern is compounded by uncertainty and questions about President-Elect Trump’s position on and plans for scientific funding. Aside from a couple of issues, primarily concerning climate change and the environment, he has said very little about science and research. Many scientists, institutions, and concerned citizens will be watching closely to see how science policy develops under Trump’s administration and its effects on federal spending and beyond. (Mike Henry, American Institute of Physics)

Biomedical Research

‘Minibrains’ Could Help Drug Discovery for Zika and for Alzheimer’s

A group of researchers at Johns Hopkins University (JHU) is working on a promising tool for evaluating disease and drug effects in humans without actually using humans for the tests. ‘Minibrains’ are clusters of human cells that originated as skin cells, reprogrammed to an earlier stage of development, and then forced to differentiate into human neural cells. They mimic the human brain as far as cell types and connections, but will never be anywhere near as large as a human brain and can never learn or become conscious.

A presentation earlier this year at the American Association for the Advancement of Science conference showcased the potential utility for minibrains. A large majority of drugs that are tested in animals fail when introduced in humans. Minibrains provide a way to test these drugs in human tissue at a much earlier stage – saving time, money, and animal testing – without risking harm to humans. Minibrains to test for biocompatibility can be made from skin cells of healthy humans, but skin cells from people with diseases or genetic traits can also be used to study disease effects.

A presentation at the Society for Neuroscience conference this month demonstrated one such disease – Zika. The minibrains’ growth is similar to fetal brain growth during early pregnancy. Using the minibrains, Dr. Hongjun Song’s team at JHU was able to see how the Zika virus affected the cells; the affected minibrains were much smaller than normal, a result that appears analogous to the microcephaly observed in infants whose mothers were infected with Zika during the first trimester.

Other presentations at the meeting showcased work from several research groups that are already using minibrains to study diseases and disorders including brain cancer, Down syndrome, and Rett syndrome, and plans are underway to utilize it in autism, schizophrenia, and Alzheimer’s disease. Though there might be a bit of an acceptance curve with the general public, minibrains potentially offer an avenue of testing that is a better representation of actual human cell behavior and response, is safer and more affordable, and reduces the need for animal testing. (Jon Hamilton, NPR)

Health Policy

A Twist on ‘Involuntary Commitment’: Some Heroin Users Request It

The opioid addiction epidemic has become a significant healthcare crisis in the United States. Just last week the US Surgeon General announced plans to target addiction and substance abuse. He also stated the desire for a change in perception of addiction – it is a medical condition rather than a moral or character flaw. Earlier this year, the Centers for Disease Control published guidelines that address opioid prescribing practices for chronic pain, strongly urging physicians to exhaust non-pharmacologic options before utilizing opioids. In response to the rising concern over prescription opioid abuse, steps have been taken to reduce prescriptions and access. This has resulted in many turning to heroin – which is usually a cheaper alternative anyway – to get their opioid fix.

One of the first steps in treatment and recovery for addiction and dependence is detoxing. However, opioids are highly addictive and many people struggle with the temptation to relapse. Additionally, many of the programs designed to help with the initial detox have long wait lists, are expensive, and may not be covered by insurance, further deterring those with addiction and dependence from getting the help they need. These factors have caused many to start turning to their states, asking to be voluntarily committed to a program on the basis that they are a danger to themselves or others because of their substance abuse. This is currently an option in 38 states. These programs can be held in either privately-run institutions or in state prisons. However, this practice is controversial because if the person’s insurance does not cover their stay, it falls to tax payers to foot the bill. While this is unpopular with some, advocates say the civil commitment laws are important options while there may be no other immediate ways for an individual to get help. (Karen Brown, NPR)

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November 22, 2016 at 9:00 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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October 25, 2016 at 10:55 am