DIGITAL HEALTHCARE

ZYNTEGLO is the first gene therapy approved for transfusion-dependent β-thalassemia (TDT) European marketing authorization for ZYNTEGLO follows the fastest assessment of an advanced therapy medicinal product (ATMP) as part of the European Medicines Agency’s Priority Medicines (PRIME) program ZYNTEGLO is bluebird bio’s first gene therapy to gain regulatory approval bluebird bio, Inc. announced today that the European Commission (EC)

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Source: Nature Study of almost half a million people links mutation that protects against HIV infection to an earlier death. The scientist who edited the genomes of twin girls in an attempt to make them resistant to HIV might have inadvertently shortened their life expectancy. People with two disabled copies of the CCR5 gene — the version

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Source The Guardian International Life Sciences Institute used by corporate backers to counter public health policies, says study An institute whose experts have occupied key positions on EU and UN regulatory panels is, in reality, an industry lobby group that masquerades as a scientific health charity, according to a peer-reviewed study. The Washington-based International Life Sciences

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Source ARM Organovo Collaborates With MCRI and Leiden University Medical Center to Develop Stem Cell-Based Bioprinted Tissue Treatments for Kidney DiseaseMay 30, 2019 – (Organovo) – This multi-organizational effort integrates Organovo’s leading bioprinting platform with MCRI’s advanced stem cell differentiation technology and LUMC’s cell lines and clinical expertise.  The collaboration has been made possible through generous

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Cancer drugs are being approved by the FDA at an alarmingly fast rate after preliminary studies show encouraging results. However, after more careful follow-up trials were done, most of these drugs did not show any clear benefits. Cancer drugs are usually granted accelerated approval in the hopes of providing patients faster access to medicine that

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Future topics To give potential applicants as much time as possible to form consortia and prepare their proposals, IMI regularly publishes information on possible future topics well in advance of the official Call launch. To ensure you get the latest information on forthcoming Calls, sign up to our newsletter, follow us on Twitter, or join our LinkedIn group.

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In light of additional data from ongoing trials of onasemnogene abeparvovec (Zolgensma®, Novartis/AveXis), as well as the treatment’s final FDA label and just-announced launch price, the Institute for Clinical and Economic Review (ICER) today published an addendum to its Final Evidence Report on treatments for spinal muscular atrophy (SMA). Since the publication of ICER’s Final Evidence Report on

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Wake Forest Institute for Regenerative Medicine (WFIRM) researchers have shown the feasibility of bioengineering vascularized functional renal tissues for kidney regeneration, developing a partial augmentation strategy that may be a more feasible and practical approach than creating whole organs. In the proof-of-concept study published online this month in Acta Biomaterialia journal, the scientists created a novel

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Johns Hopkins researchers have developed a way to map retinal cell development and potentially advance regenerative medicine Johns Hopkins Medicine researchers report they have created a method of mapping how the central nervous system develops by tracking the genes expressed in cells. The technique, demonstrated in mouse retinas for this study, follows the activity of

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Source The Economist Western countries, as well as poor ones, are demanding transparency in the cost of drugs THESE DAYS it is hard to find a government that is not struggling with the high price of medicines. In England, the government is fighting Vertex, a drug company, over the cost of a drug for cystic

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Source: TMF The next telecommunication revolution is just around the corner: the promises of high bandwidth, low latency and low-power-low-cost of 5G will open the gate to a flood of new inventions and the implementation of ideas, which have already been for long in the public consciousness – such as stable augmented reality or truly

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For the first time, immunologists from The University of Texas at Austin have captured on video what happens when T-cells – the contract killers of the immune system, responsible for wiping out bacteria and viruses – undergo a type of assassin-training program before they get unleashed in the body. A new imaging technique that allowed

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Individuals who use healthcare apps such as fitness trackers, weight loss, wellness, exercise, etc., BEWARE!A couple of recent developments have highlighted the fact that most apps are not subject to HIPAA, which means that with broadly-worded privacy policy these healthcare apps can and do readily share healthcare and other data collected by the apps with third

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Machine learning algorithm predicts sudden death in heart failure patients for the first time Artificial intelligence (AI) has shown promise to select heart failure patients for expensive treatments to prevent lethal arrhythmias, reports a study presented today at ICNC 2019. (1) The study is the first to use a machine learning algorithm to predict sudden

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Last year, the birth of the first genetically modified babies shook the world. The German Ethics Council now presents a comprehensive ethical investigation into possible interventions in the genome of human embryos or germ cells. The Council does not deem the human germline to be inviolable. It does, however, consider germline interventions to be ethically

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A recent editorial in the Journal of the American Medical Association (JAMA) addresses the growing problem of medical misinformation, such as anti-vaccine views, fake treatments, unproven alternative products and services, and others. The authors, Armstrong and Naylor, make some good recommendations, but unfortunately are about 20 years behind the times when it comes to confronting scientific misinformation.

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An advance in the groundbreaking cancer treatment known as CAR T-cell therapy appears to eliminate its severe side effects, making the treatment safer and potentially available in outpatient settings, a new USC study shows. “This is a major improvement,” said Si-Yi Chen of the USC Norris Comprehensive Cancer Center, professor in the Department of Molecular Microbiology

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A state-of-the-art brain-machine interface created by UC San Francisco neuroscientists can generate natural-sounding synthetic speech by using brain activity to control a virtual vocal tract – an anatomically detailed computer simulation including the lips, jaw, tongue and larynx. The study was conducted in research participants with intact speech, but the technology could one day restore the

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Germany and France are two flourishing AI hubs, but the UK has been named the European AI “powerhouse” in a new report from London-based investment firm MMC Ventures. Published earlier this month, the analysis sheds light on the hype around the technology, but identifies healthcare as an area of increasing focus for AI entrepreneurs as systems

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Augmented Reality can change brain surgery thanks to powerful diagnostic platforms, revolutionize radiology, and open new doors to reconstructive surgery. Augmented Reality (AR), also known as spatial computing –a merging of digital and physical spaces,– is one of the current technology trends that, together with Virtual Reality (VR) and Mixed Reality (MR), is changing all industries, includinghealthcare and medical education. 

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Researchers have developed a high-tech support system that can keep a large mammalian brain from rapidly decomposing in the hours after death, enabling study of certain molecular and cellular functions. With funding through the National Institutes of Health BRAIN Initiative, researchers developed a way to deliver an artificial blood supply to the isolated postmortem brain of

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NIH scientists and funding contributed to development of experimental treatment A small clinical trial has shown that gene therapy can safely correct the immune systems of infants newly diagnosed with a rare, life-threatening inherited disorder in which infection-fighting immune cells do not develop or function normally. Eight infants with the disorder, called X-linked severe combined

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Researchers have infused cells edited using CRISPR-Cas9 into two patients in a trial conducted at the University of Pennsylvania, NPR reports today (April 16). A university spokesperson confirmed in an emailed statement to The Scientist that the trial is underway and that two patients, one with multiple myeloma and one with sarcoma, have been treated so far. The study is

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Cardiovascular disease (CVD) remains the in the United States. And one of the most effective preventive measures to lower the risk of a CVD event are statins — a class of cholesterol-lowering medications. It’s well established that statins save lives, but a recent study finds that even after two years, half of all people prescribed statins don’t achieve healthy cholesterol levels.

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Hundreds of smartphone apps promise to make managing diabetes easier, but very few offer real-time guidance on what to do for dangerously high or low blood sugar, a US study suggests. Researchers looked at 5,185 apps for phones running Google’s Android software or Apple’s iOS system.  Out of this total, they found 371 apps that claimed

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Researchers discovered thousands of genes essential for cancer’s survival and ranked which ones show the most promise as drug targets for developing new treatments In one of the largest studies of its kind, researchers used CRISPR technology to disrupt every gene in over 300 cancer models from 30 cancer types and discover thousands of key

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Diets that replaced red meat with healthy plant proteins led to decreases in risk factors for cardiovascular disease (CVD), according to a new study from Harvard T.H. Chan School of Public Health and Purdue University. The study is the first meta-analysis of randomized controlled trials examining the health effects of red meat by substituting it for

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It was once a central tenet of biology that RNA molecules did their work inside the cell. But it’s now clear that RNA molecules are also active outside the cell, with potentially major implications for our health. To learn more about these unrecognized roles, the NIH Common Fund has launched the Extracellular RNA (exRNA) Communication Program. This month,

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A new analysis published on 3 April in the Lancet links 20 per cent of global deaths to poor diet, equating to around 11 million deaths per year (1). The biggest risk factors were a high intake of sodium (salt) or low intake of whole grains, fruit, nuts and seeds, vegetables, and other healthy foods. The Global Burden of Disease

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The biomolecule ribonucleic acid (RNA) is pivotal to cell function. RNA plays various roles in determining how the information in our genes drives cell behavior. One of its roles is to carry information encoded by our genes from the cell nucleus to the rest of the cell where it can be acted on by other

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OVERVIEW Shifting from treating symptoms to curing chronic diseases by making the transformative promise of Advanced Therapies a reality for the benefit of patients and society and by making Europe a spearhead of Advanced Therapies in Science,Clinics and Biomedical Industry, that is the vision of the international project RESTORE Health by Advanced Therapies, coordinated by

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New research reveals a potential revolutionary way to treat eye injuries and prevent blindness – by softening the tissue hosting the stem cells which then helps repair wounds, inside the body. The team discovered that the simple application of a tissue-softening enzyme, collagenase, prevents the loss of corneal stem cells following an injury and could

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Your diet plays an important role in your health. An unhealthy diet is one of the leading risk factors for cardiometabolic diseases, such as heart disease, stroke, and diabetes. Eating a healthy diet can lower your risk for some of these conditions. A healthy eating plan includes vegetables, fruits, whole grains, fat-free or low-fat dairy

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In the past three decades, in vitro fertilization (IVF) has gone from an experimental procedure to being more common. Pregnancies enabled by IVF frequently have more difficulties, with children born earlier and smaller even among singleton births. University of Minnesota researchers conducted the largest study of childhood cancer after conception by IVF to date. This

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The first gene therapy to treat a rare blood disorder is one step closer to approval Friday following a recommendation by European officials. Lentiglobin, the gene therapy for beta-thalassemia developed by Cambridge, Mass.-based Bluebird Bio, was recommended for approval by the Committee for Medicinal Products for Human Use (CHMP), the drug-reviewing arm of the European Medicines Agency.

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The European Commission is announcing the winners of a pan-European competition open to researchers addressing grand scientific and technological challenges that could change our future. The six preparatory actions now launched will have one year to prepare science and technology agendas in areas of strategic importance for Europe. These may contribute to defining and launching

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A new study led by scientists in the Center for Cancer Research (CCR) at the National Cancer Institute (NCI) sheds light on one way tumors may continue to grow despite the presence of cancer-killing immune cells. The findings, published March 29, 2019, in Science, suggest a way to enhance the effectiveness of immunotherapies for cancer

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With modern, guideline-directed therapy,1 which includes angiotensin-converting enzyme (ACE) inhibitors, angiotensin II receptor blockers (ARBs), β-receptor blockers, mineralocorticoid receptor blockers, and angiotensin receptor neprilysin inhibitors, up to 40% of patients with dilated cardiomyopathy (heart failure with reduced ejection fraction) may experience a return of left ventricular ejection fraction (LVEF) and ventricular geometry to normal.2 This encouraging result

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An important achievement in stem cell research was recognized in 2012, when the Nobel Prize in Physiology or Medicine was awarded to two scientists who transformed the field: Shinya Yamanaka and John Gurdon. Together, they received the award for “the discovery that mature cells can be reprogrammed to become pluripotent.” The impact of this breakthrough

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As artificial intelligence continues to evolve, diagnosing disease faster and potentially with greater accuracy than physicians, some have suggested that technology may soon replace tasks that physicians currently perform. But a new study from the Google AI research group shows that physicians and algorithms working together are more effective than either alone. It’s one of

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https://www.nature.com/articles/s41591-019-0375-9 Reports of old brains’ decrepitude have been greatly exaggerated, scientists reported on Monday, unveiling results that contradict a much-discussed 2018 study and instead support the idea that human gray matter is capable of generating new neurons up to the ninth decade of life. The research, published in Nature Medicine, also found that old brains from

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Statistical significance has become an infallible doctrine of scientific research. However, many scientists and statisticians argue that long-held beliefs about statistical significance have, in fact, harmed the scientific community. In hypothesis testing, the p-value gives the probability or likelihood that the null hypothesis is true and is frequently used as a measure of “statistical significance.”

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A mouse study finds that when blood platelets are activated during exercise, they release factors that increase the number of newborn neurons in the hippocampus. It’s well established that exercise is good for the mammalian brain. As early as 1999, researchers discovered considerably more newborn neurons in the hippocampi of mice that had access to

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Overall death rates from breast cancer fall in the EU, but rise in Poland Death rates from breast cancer are predicted to fall in all European Union (EU) countries in 2019 with the exception of Poland, according to new research published in the leading cancer journal Annals of Oncology In their annual predictions for cancer deaths

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Study uncovers genes that control process of whole-body regeneration A new study looks at how three-banded panther worms are able to regenerate their whole bodies and how this regeneration could be applied to humans. Video by Yi-Jyun Luo When it comes to regeneration, some animals are capable of amazing feats. If you cut off a

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It was surprisingly simple. University of California, Berkeley, scientists inserted a gene for a green-light receptor into the eyes of blind mice and, a month later, they were navigating around obstacles as easily as mice with no vision problems. They were able to see motion, brightness changes over a thousandfold range and fine detail on

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New results of a gigantic study testing how well the Apple Watch detects important heart-rhythm changes offer a tantalizing glimpse of how consumer devices might change medicine and lead to new ways to conduct science — but little more. Unfortunately, the data don’t tell us much about whether anyone should buy an Apple Watch, or

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SUPPORT RESTORE AND DIFFUSE INFORMATION USING DOWNLOAD MATERIAL AT https://www.restore-h2020.eu/documents/download/restore_pr_slides_short_update_14_03_2019.pdf Large Scale Research Initiative RESTORE is a “place” where medicine, basic research, technology development and engineering meet, communicate and work together. Advanced Therapies (Advanced Therapies Medicinal Products and Biologised Medical Devices – ATMP/bio-MD) are a potential game changer in health care: They aim to transform

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Eric Lander, Françoise Baylis, Feng Zhang, Emmanuelle Charpentier, Paul Berg and specialists from seven countries call for an international governance framework. We call for a global moratorium on all clinical uses of human germline editing — that is, changing heritable DNA (in sperm, eggs or embryos) to make genetically modified children. By ‘global moratorium’, we

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Here’s why that’s undermining personalized medicine In a paper published last week, scientists led by Dr. Pui-Yan Kwok of the University of California, San Francisco, analyzed 154 genomes from 26 ethnic populations, from Han Chinese and Tuscans to Yoruba, Esan, Puerto Ricans, and Peruvians. They found 60 million bases in one or more of these populations

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A feature-length documentary chronicling the story of CRISPR premiered at the SXSW festival yesterday. From executive producer Dan Rather and director Adam Bolt, the co-writer and editor of the Oscar-winning film Inside Job, comes the story of the biggest tech revolution of the 21st Century. And it isn’t digital, it’s biological. A breakthrough called CRISPR has

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Discovery made while editing genetic defect behind Duchenne muscular dystrophy Scientists have developed a method to boost the efficiency of CRISPR gene editing in Duchenne muscular dystrophy (DMD), according to a study that could have implications for optimizing gene therapies for other diseases. The finding stemmed from research at UT Southwestern in which a single-cut gene-editing

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Cell sheets have previously shown greater efficacy for tissue repair than injections of single stem cells. New research conducted by scientists at the Los Angeles Biomedical Research Institute and Emmaus Life Sciences, Inc. (both CA, USA) has demonstrated the possibility of producing scaffold-free engineered cell sheets. These cell ‘patches’ could enable better organ repair follow

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In a paper published in the journal Nature, an international research team led by researchers at Karolinska Institutet in Sweden report that bone growth in mice takes place in accordance with the same principles as when new cells are constantly produced in blood, skin and other tissue. This contradicts the previous understanding that bone growth depends

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Advanced Therapies are new treatment modalities (e.g. cell and gene therapy, tissue engineering) that aim to consign the never-ending treatment of chronic ailments to the past and instead offer sustained improvement and even cures. To enable Europe to become a competitive leader in this field, RESTORE is defining an Advanced Therapies roadmap 2021-2030 for Europe.

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February 27 2019 P.M.Fornasari At the MWC Barcelona, one of the largest mobile events in the world, Royal Philips (NYSE: PHG, AEX: PHIA), a global leader in health technology today unveiled a unique mixed reality concept developed together with Microsoft Corp. (NASDAQ: MSFT) for the operating room of the future. Based on the state-of-the-art technologies

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February 27 2019 P.M. Fornasari Yale researchers have developed a new way to efficiently engineer immune cells, an advance which enhances the ability to fend off cancer and other diseases, they report in the Feb. 25 issue of the journal Nature Methods. A number of new life-saving therapies rely on reconfiguring human immune cells to act

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Highlights •Microbiome-derived NO promotes widespread S-nitrosylation of the host proteome •Interspecies S-nitrosylation regulates miRNAs, gene expression and host development •Microbiota control host function by shaping the post-translational landscape Summary Bioactive molecules can pass between microbiota and host to influence host cellular functions. However, general principles of interspecies communication have not been discovered. We show here

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Advanced Therapies (Advanced Therapies Medicinal Products and Biologised Medical Devices – ATMP/bio-MD) are a potential game changer in health care: They aim to transform the current focus on “treatment of disease” into one that concentrates on “regeneration of health”. The unifying goal of RESTORE is the implementation of newly developed Advanced Therapies in clinical routine

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Allogeneic CAR-Ts could serve an important niche – particularly for patients who can’t receive autologous CAR-Ts – but the earliness of the data makes conclusions tough to draw. Since their transition from small, academic clinical trials to commercial production, CAR-T cell therapies have won recognition as a novel and highly effective therapeutic modality, particularly in

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Research led by Johns Hopkins investigators has uncovered the roles of two types of cells found in the vessel walls of fat tissue and described how these cells may help speed bone repair. The study found that one of these perivascular stem cell types, pericytes, induces growth of new blood vessels, while another type, adventicytes,

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The hype around AI applications in healthcare spans decades, but the latest wave of AI tech suggests real-world traction. Artificial intelligence is already promising big things at Massachusetts General Hospital and Brigham and Women’s Hospital in Boston, where researchers have been building machine learning models over the past couple of years. Those models now help

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Data is pouring in from more than 100 million wearable devices worldwide that can track your steps, sleep patterns, blood pressure, heart rate, energy levels and a whole range of other measures. There’s just one problem, says Dr. Karl Poterack, director of applied clinical informatics at Mayo Clinic: “We don’t know if this data can

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REGENHEALTHSOLUTIONS is a strong supporter of RESTORE, one of the six large scale research initiatives selected by European Commission in areas from health and energy to artificial intelligence and cultural heritage—to compete to become one of its next billion-euro ‘flagship’ science initiatives. On 1 March, each team will receive €1 million (US$1.1 million) to develop a

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Nutrigenomics — the nexus of nutrition, genetics and genomics — is still in its infancy, but USC researcher Cary Kreutzer sees vast potential in its ability to help people take control of their health. ne day, you may walk into your doctor’s office and come out with a diet that’s been personalized for your unique

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National Institutes of Health scientists have used human skin cells to create what they believe is the first cerebral organoid system, or “mini-brain,” for studying sporadic Creutzfeldt-Jakob disease (CJD). CJD is a fatal neurodegenerative brain disease of humans believed to be caused by infectious prion protein. It affects about 1 in 1 million people. The

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We have arrived at a special moment in health care. Innovative, life-changing gene therapies are here that will cure or ease debilitating diseases. Yet these expensive treatments are entering a market structure that was not built to price them. Congress will likely need to play a part in developing a new paradigm for financing such

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The FDA is now aware of bacterial infections caused by multi-drug resistant organisms (MDROs) that have occurred due to transmission of a MDRO from use of investigational fecal microbiota for transplantation (FMT): Two immunocompromised adults who received investigational FMT developed invasive infections caused by extended-spectrum beta-lactamase (ESBL)-producing Escherichia coli (E.coli). One of the individuals died. FMT

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Bluebird bio Inc on Friday set a price for its gene therapy, Zynteglo, at 1.58 million euros ($1.78 million) over five years, after winning conditional approval in Europe this month to treat a rare genetic blood disorder. The company proposed an installment plan, with 315,000 euros paid up front and four additional annual payments due

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Large Scale Research Initiative RESTORE The visionary unifying goal of Large Scale Research Initiative RESTORE is to make the transforming promise associated with Advanced Therapies a reality for the benefit of patients and society. RESTORE sees itself as a “place” where medicine, basic research, technology development and engineering meet, communicate and work together. Through this

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Animal study finds large bone defects could be regenerated through stem cell implantation and allowing movement To treat large gaps in long bones, like the femur, which result from bone tumor removal or a shattering trauma, researchers at Penn Medicine and the University of Illinois at Chicago developed a process that partially recreates the bone

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Data released May 9 by the Alliance for Regenerative Medicine (ARM) illustrates the growth of clinical activity where gene therapy is concerned. ARM’s Quarterly Regenerative Medicine Global Data Report for the first quarter of 2019 showed 372 gene therapy clinical trials were in progress as of the end of Q1. Interestingly, a majority (217 or 58%) were

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This report includes an overview of regenerative medicine approaches to treating a variety of cancer types, along with a list of ARM member companies active in developing these therapies, global financings, an overview of the oncology-focused clinical pipeline, as well as commentary from select therapeutic developers active in this area. Key findings include: There are 416 companies worldwide active in

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A new phase of the IGNITE Network will conduct clinical trials of genomic medicine interventions. The National Institutes of Health will fund clinical trials to assess the benefits, applicability and efficacy of applying genomic medicine interventions to improve management of diseases such as high blood pressure, depression and chronic pain. The trials are part of

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